No antibiotics worked, so this woman turned to a natural enemy of bacteria to save her husband’s life | CNN



CNN
 — 

In February 2016, infectious disease epidemiologist Steffanie Strathdee was holding her dying husband’s hand, watching him lose an exhausting fight against a deadly superbug infection.

After months of ups and downs, doctors had just told her that her husband, Tom Patterson, was too racked with bacteria to live.

“I told him, ‘Honey, we’re running out of time. I need to know if you want to live. I don’t even know if you can hear me, but if you can hear me and you want to live, please squeeze my hand.’

“All of a sudden, he squeezed really hard. And I thought, ‘Oh, great!’ And then I’m thinking, ‘Oh, crap! What am I going to do?’”

What she accomplished next could easily be called miraculous. First, Strathdee found an obscure treatment that offered a glimmer of hope — fighting superbugs with phages, viruses created by nature to eat bacteria.

Then she convinced phage scientists around the country to hunt and peck through molecular haystacks of sewage, bogs, ponds, the bilge of boats and other prime breeding grounds for bacteria and their viral opponents. The impossible goal: quickly find the few, exquisitely unique phages capable of fighting a specific strain of antibiotic-resistant bacteria literally eating her husband alive.

Next, the US Food and Drug Administration had to greenlight this unproven cocktail of hope, and scientists had to purify the mixture so that it wouldn’t be deadly.

Yet just three weeks later, Strathdee watched doctors intravenously inject the mixture into her husband’s body — and save his life.

Their story is one of unrelenting perseverance and unbelievable good fortune. It’s a glowing tribute to the immense kindness of strangers. And it’s a story that just might save countless lives from the growing threat of antibiotic-resistant superbugs — maybe even your own.

“It’s estimated that by 2050, 10 million people per year — that’s one person every three seconds — is going to be dying from a superbug infection,” Strathdee told an audience at Life Itself, a 2022 health and wellness event presented in partnership with CNN.

“I’m here to tell you that the enemy of my enemy can be my friend. Viruses can be medicine.”

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How this ‘perfect predator’ saved his life after nine months in the hospital

During a Thanksgiving cruise on the Nile in 2015, Patterson was suddenly felled by severe stomach cramps. When a clinic in Egypt failed to help his worsening symptoms, Patterson was flown to Germany, where doctors discovered a grapefruit-size abdominal abscess filled with Acinetobacter baumannii, a virulent bacterium resistant to nearly all antibiotics.

Found in the sands of the Middle East, the bacteria were blown into the wounds of American troops hit by roadside bombs during the Iraq War, earning the pathogen the nickname “Iraqibacter.”

“Veterans would get shrapnel in their legs and bodies from IED explosions and were medevaced home to convalesce,” Strathdee told CNN, referring to improvised explosive devices. “Unfortunately, they brought their superbug with them. Sadly, many of them survived the bomb blasts but died from this deadly bacterium.”

Today, Acinetobacter baumannii tops the World Health Organization’s list of dangerous pathogens for which new antibiotics are critically needed.

“It’s something of a bacterial kleptomaniac. It’s really good at stealing antimicrobial resistance genes from other bacteria,” Strathdee said. “I started to realize that my husband was a lot sicker than I thought and that modern medicine had run out of antibiotics to treat him.”

With the bacteria growing unchecked inside him, Patterson was soon medevaced to the couple’s hometown of San Diego, where he was a professor of psychiatry and Strathdee was the associate dean of global health sciences at the University of California, San Diego.

“Tom was on a roller coaster — he’d get better for a few days, and then there would be a deterioration, and he would be very ill,” said Dr. Robert “Chip” Schooley, a leading infectious disease specialist at UC San Diego who was a longtime friend and colleague. As weeks turned into months, “Tom began developing multi-organ failure. He was sick enough that we could lose him any day.”

Patterson's body was systemically infected with a virulent drug-resistant bacteria that also infected troops in the Iraq War, earning the pathogen the nickname

After that reassuring hand squeeze from her husband, Strathdee sprang into action. Scouring the internet, she had already stumbled across a study by a Tbilisi, Georgia, researcher on the use of phages for treatment of drug-resistant bacteria.

A phone call later, Strathdee discovered phage treatment was well established in former Soviet bloc countries but had been discounted long ago as “fringe science” in the West.

“Phages are everywhere. There’s 10 million trillion trillion — that’s 10 to the power of 31 — phages that are thought to be on the planet,” Strathdee said. “They’re in soil, they’re in water, in our oceans and in our bodies, where they are the gatekeepers that keep our bacterial numbers in check. But you have to find the right phage to kill the bacterium that is causing the trouble.”

Buoyed by her newfound knowledge, Strathdee began reaching out to scientists who worked with phages: “I wrote cold emails to total strangers, begging them for help,” she said at Life Itself.

One stranger who quickly answered was Texas A&M University biochemist Ryland Young. He’d been working with phages for over 45 years.

“You know the word persuasive? There’s nobody as persuasive as Steffanie,” said Young, a professor of biochemistry and biophysics who runs the lab at the university’s Center for Phage Technology. “We just dropped everything. No exaggeration, people were literally working 24/7, screening 100 different environmental samples to find just a couple of new phages.”

While the Texas lab burned the midnight oil, Schooley tried to obtain FDA approval for the injection of the phage cocktail into Patterson. Because phage therapy has not undergone clinical trials in the United States, each case of “compassionate use” required a good deal of documentation. It’s a process that can consume precious time.

But the woman who answered the phone at the FDA said, “‘No problem. This is what you need, and we can arrange that,’” Schooley recalled. “And then she tells me she has friends in the Navy that might be able to find some phages for us as well.”

In fact, the US Naval Medical Research Center had banks of phages gathered from seaports around the world. Scientists there began to hunt for a match, “and it wasn’t long before they found a few phages that appeared to be active against the bacterium,” Strathdee said.

Dr. Robert

Back in Texas, Young and his team had also gotten lucky. They found four promising phages that ravaged Patterson’s antibiotic-resistant bacteria in a test tube. Now the hard part began — figuring out how to separate the victorious phages from the soup of bacterial toxins left behind.

“You put one virus particle into a culture, you go home for lunch, and if you’re lucky, you come back to a big shaking, liquid mess of dead bacteria parts among billions and billions of the virus,” Young said. “You want to inject those virus particles into the human bloodstream, but you’re starting with bacterial goo that’s just horrible. You would not want that injected into your body.”

Purifying phage to be given intravenously was a process that no one had yet perfected in the US, Schooley said, “but both the Navy and Texas A&M got busy, and using different approaches figured out how to clean the phages to the point they could be given safely.”

More hurdles: Legal staff at Texas A&M expressed concern about future lawsuits. “I remember the lawyer saying to me, ‘Let me see if I get this straight. You want to send unapproved viruses from this lab to be injected into a person who will probably die.’ And I said, “Yeah, that’s about it,’” Young said.

“But Stephanie literally had speed dial numbers for the chancellor and all the people involved in human experimentation at UC San Diego. After she calls them, they basically called their counterparts at A&M, and suddenly they all began to work together,” Young added.

“It was like the parting of the Red Sea — all the paperwork and hesitation disappeared.”

The purified cocktail from Young’s lab was the first to arrive in San Diego. Strathdee watched as doctors injected the Texas phages into the pus-filled abscesses in Patterson’s abdomen before settling down for the agonizing wait.

“We started with the abscesses because we didn’t know what would happen, and we didn’t want to kill him,” Schooley said. “We didn’t see any negative side effects; in fact, Tom seemed to be stabilizing a bit, so we continued the therapy every two hours.”

Two days later, the Navy cocktail arrived. Those phages were injected into Patterson’s bloodstream to tackle the bacteria that had spread to the rest of his body.

“We believe Tom was the first person to receive intravenous phage therapy to treat a systemic superbug infection in the US,” Strathdee told CNN.

“And three days later, Tom lifted his head off the pillow out of a deep coma and kissed his daughter’s hand. It was just miraculous.”

Patterson awoke from a coma after receiving an intravenous dose of phages tailored to his bacteria.

Today, nearly eight years later, Patterson is happily retired, walking 3 miles a day and gardening. But the long illness took its toll: He was diagnosed with diabetes and is now insulin dependent, with mild heart damage and gastrointestinal issues that affect his diet.

“He isn’t back surfing again, because he can’t feel the bottoms of his feet, and he did get Covid-19 in April that landed him in the hospital because the bottoms of his lungs are essentially dead,” Strathdee said.

“As soon as the infection hit his lungs he couldn’t breathe and I had to rush him to the hospital, so that was scary,” she said. “He remains high risk for Covid but we’re not letting that hold us hostage at home. He says, ‘I want to go back to having as normal life as fast as possible.’”

To prove it, the couple are again traveling the world — they recently returned from a 12-day trip to Argentina.

“We traveled with a friend who is an infectious disease doctor, which gave me peace of mind to know that if anything went sideways, we’d have an expert at hand,” Strathdee said.

“I guess I’m a bit of a helicopter wife in that sense. Still, we’ve traveled to Costa Rica a couple of times, we’ve been to Africa, and we’re planning to go to Chile in January.”

Patterson’s case was published in the journal Antimicrobial Agents and Chemotherapy in 2017, jump-starting new scientific interest in phage therapy.

“There’s been an explosion of clinical trials that are going on now in phage (science) around the world and there’s phage programs in Canada, the UK, Australia, Belgium, Sweden, Switzerland, India and China has a new one, so it’s really catching on,” Strathdee told CNN.

Some of the work is focused on the interplay between phages and antibiotics — as bacteria battle phages they often shed their outer shell to keep the enemy from docking and gaining access for the kill. When that happens, the bacteria may be suddenly vulnerable to antibiotics again.

“We don’t think phages are ever going to entirely replace antibiotics, but they will be a good adjunct to antibiotics. And in fact, they can even make antibiotics work better,” Strathdee said.

In San Diego, Strathdee and Schooley opened the Center for Innovative Phage Applications and Therapeutics, or IPATH, in 2018, where they treat or counsel patients suffering from multidrug-resistant infections. The center’s success rate is high, with 82% of patients undergoing phage therapy experiencing a clinically successful outcome, according to its website.

Schooley is running a clinical trial using phages to treat patients with cystic fibrosis who constantly battle Pseudomonas aeruginosa, a drug-resistant bacteria that was also responsible for the recent illness and deaths connected to contaminated eye drops manufactured in India.

And a memoir the couple published in 2019 — “The Perfect Predator: A Scientist’s Race to Save Her Husband From a Deadly Superbug” — is also spreading the word about these “perfect predators” to what may soon be the next generation of phage hunters.

VS Phages Sanjay Steffanie

How naturally occurring viruses could help treat superbug infections

“I am getting increasingly contacted by students, some as young as 12,” Strathdee said. “There’s a girl in San Francisco who begged her mother to read this book and now she’s doing a science project on phage-antibiotic synergy, and she’s in eighth grade. That thrills me.”

Strathdee is quick to acknowledge the many people who helped save her husband’s life. But those who were along for the ride told CNN that she and Patterson made the difference.

“I think it was a historical accident that could have only happened to Steffanie and Tom,” Young said. “They were at UC San Diego, which is one of the premier universities in the country. They worked with a brilliant infectious disease doctor who said, ‘Yes,’ to phage therapy when most physicians would’ve said, ‘Hell, no, I won’t do that.’

“And then there is Steffanie’s passion and energy — it’s hard to explain until she’s focused it on you. It was like a spiderweb; she was in the middle and pulled on strings,” Young added. “It was just meant to be because of her, I think.”

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Urinary tract infections in men: Here are 10 things to know | CNN

Editor’s Note: Dr. Jamin Brahmbhatt is a urologist and robotic surgeon with Orlando Health and president of the Florida Urological Society.



CNN
 — 

While urinary tract infections are more common in women, men can still get what’s commonly known as a UTI. Here are 10 things I’d like you to know about urinary tract infections, including who’s more at risk and how to get treatment.

UTI is short for urinary tract infection. It’s an infection of the organs in your body – I call them pipes – that are meant to funnel your urine out of your system and into the urinal. Most UTIs are caused by bacteria that work their way into the urethra, prostate, bladder or kidneys.

Way more women than men are diagnosed with UTIs. Anatomically, we feel this happens because women have a shorter urethra – the tube that connects the bladder to the outside world. The shorter length makes it easier for bacteria to travel to the urinary system. Men have longer urethras and therefore can be protected against urinary infections.

But the length of the urethra alone cannot protect men against UTIs – over their lifetimes, 12% of men will get urinary symptoms linked to a UTI. This by no means implies a urethra or penis are short or small. In men, there is usually a more clear pathologic cause to the infection beyond just the length of the urethra.

There are many reasons why a guy may get a UTI – all of them we take seriously and should not be ignored.

Men older than 50 tend to get more infections than younger men. As a urologist, I see men get recurrent infections when they do not properly empty their bladder because of an enlarged prostate. Beyond the prostate, men may not empty their bladder if they have nerve damage from stroke, uncontrolled diabetes or injury to the spine.

Men can also get infections that start from the prostate or testicles that seed up into the bladder, or the opposite can happen where the infection goes from the bladder to the other organs. Kidney stones can also be a cause of infection. (I know this from personal experience – I’ve had a kidney stone myself!)

Younger men may also present with urinary infections because of sexually transmitted diseases. Men can also get an infection if they have a recent procedure done in the urinary system.

4. What are the signs and symptoms of a UTI?

Burning with urination (dysuria), increased urinary frequency, urgency, incontinence, foul smell, blood in the urine, fevers, chills, pain in the abdomen near the bladder. Believe it or not, some men may have zero symptoms and still get diagnosed with a UTI based on urine cultures done for other purposes.

UTI is diagnosed by sending your urine off for a culture. This is when a sample of your urine is processed and evaluated for various strains of bacteria. The most common bacteria identified in urinary tract infections is E.coli. Once the culture is done, the results can guide treatment, which is usually oral antibiotics. There is a test called a urine analysis which can be done quickly in our office which can suggest an infection. However, the best test is an actual culture.

Doctors do not wait for the culture results – which can take one to three days – to start treatment. If an infection is suspected, an antibiotic will be started immediately and then adjusted based on the culture results.

UTIs generally are treated with oral or IV antibiotics. Most infections can be treated with oral antibiotics. However there are superbugs that may be resistant to what we can give you by mouth that may require the use of stronger antibiotics through an IV. Most treatments last seven to 10 days, but can be longer.

In severe cases of infection that has spread to the bloodstream, strong IV antibiotics are started immediately to control the infection. Patients are placed in the hospital to start these strong treatments. You do not have to stay in the hospital for weeks if you have infection in your bloodstream. As long as you are doing well – no fever, normal labs, heart and pulse OK – then you may continue these IV treatments from home. Each treatment is tailored to your condition.

As a doctor, my answer is: No. Men should not try to treat infections on their own. If you have symptoms, get yourself to a doctor or emergency room.

The best prevention is making sure first there is nothing anatomical that needs to be corrected, such as an enlarged prostate, kidney stone or blockage.

Proper hygiene can help prevent infections. Men with uncircumcised penises should make sure they can retract the foreskin and clean under the foreskin and the glans properly. Cranberry supplements have been shown to help prevent infections. Staying hydrated by drinking enough fluids/water during the day can also help. Making sure you don’t hold your urine can help, too. Staying in good health to avoid chronic medical conditions such as diabetes and heart disease will also protect against infections.

9. My infection is gone. Are there any long-term effects on my body?

Recurrent, untreated infections could cause strictures, or tight scars, in your urethra that would slow your stream and make it difficult to empty your bladder. Infections could also cause the bladder to lose its ability to fill and empty properly. In the long run, if you are getting constantly treated with antibiotics, we may run out of antibiotics to give you due to resistance.

The first priority is to clear the infection with antibiotics.

From there, we do a full workup with a detailed history, evaluation of chronic medical problems and exam of the genitals to look for anatomic issues such as a foreskin that won’t retract back. Imaging may include a CT scan of the abdomen and pelvis to look for kidney stones, blocked tubes and other abnormalities.

If you see a urologist, you will likely get a cystoscopy, where we place a camera inside of a small tube into the urethra to look at the inside of your urine channel. The cystoscopy helps look for strictures, large obstructing prostates and changes to the bladder walls. Once a cause is found, it’s aggressively treated with either medication or surgery.

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Using melatonin for sleep is on the rise, study says, despite potential health harms | CNN

Editor’s Note: Sign up for the Sleep, But Better newsletter series. Our seven-part guide has helpful hints to achieve better sleep.



CNN
 — 

More and more adults are taking over-the-counter melatonin to get to sleep, and some may be using it at dangerously high levels, a study has found.

The overall use among the US adult population is still “relatively low,” but the study does “document a significant many-fold increase in melatonin use in the past few years,” said sleep specialist Rebecca Robbins, an instructor in the division of sleep medicine for Harvard Medical School. She was not involved in the study.

The study, published in the medical journal JAMA, found that by 2018 Americans were taking more than twice the amount of melatonin than a decade earlier.

Melatonin has been linked to headaches, dizziness, nausea, stomach cramps, drowsiness, confusion or disorientation, irritability and mild anxiety, depression and tremors as well as abnormally low blood pressure. It can also interact with common medications and trigger allergies.

While short-term use for people with jet lag, shift workers and those who have trouble falling asleep appears to be safe, long-term safety is unknown, according to the National Center for Complementary and Integrative Health at the National Institutes of Health.

“In an associational study we found that older adults who reported frequent use — every night or most nights — of a sleep aid (over the counter or prescription) had a higher risk of incident dementia and early mortality,” Robbins said.

However, researchers could not determine which type of sleep aid — over-the-counter medications, such as melatonin, or prescription medications — was responsible for the findings.

Since 2006, a small but growing subset of adults are taking amounts of melatonin that far exceed the dosage of 5 milligrams a day typically used as a short-term treatment, the study found.

However, pills for sale may contain levels of melatonin much higher than what is advertised on the label. Unlike drugs and food, melatonin is not fully regulated by the US Food and Drug Administration, so there are no federal requirements that companies test pills to ensure they contain the amount of advertised melatonin.

“Previous research has found that that melatonin content in these unregulated, commercially available melatonin supplements ranged from — 83% to +478% of the labeled content,” said Robbins, who coauthored the book “Sleep for Success! Everything You Must Know About Sleep But are Too Tired to Ask.”

Nor are there any requirements that companies test their products for harmful hidden additives in melatonin supplements sold in stores and online. Previous studies also found 26% of the melatonin supplements contained serotonin, “a hormone that can have harmful effects even at relatively low levels,” according to the National Center for Complementary and Integrative Health.

“We cannot be certain of the purity of melatonin that is available over the counter,” Robbins said.

Taking too much serotonin by combining medications such as antidepressants, migraine medications and melatonin can lead to a serious drug reaction. Mild symptoms include shivering and diarrhea, while a more severe reaction can lead to muscle rigidity, fever, seizures and even death if not treated.

Because it is purchased over the counter, experts say many people view melatonin as an herbal supplement or vitamin. In reality, melatonin is a hormone made by the pineal gland, located deep within the brain, and released into the bloodstream to regulate the body’s sleep cycles.

“There is a view that if it’s natural, then it can’t hurt,” Robbins told CNN in an earlier interview on the impact of melatonin on children. “The truth is, we just really don’t know the implications of melatonin in the longer term, for adults or kids.”

Another reality: Studies have found that while using melatonin can be helpful in inducing sleep if used correctly — taking it at least two hours before bed — but the actual benefit is small.

“When adults took melatonin, it decreased the amount of time it took them to fall asleep by four to eight minutes,” Dr. Cora Collette Breuner, a professor in the department of pediatrics at Seattle Children’s Hospital at the University of Washington, told CNN in 2021.

“So for someone who takes hours to fall asleep, probably the better thing for them to do is turn off their screens, or get 20 to 40 minutes of exercise each day, or don’t drink any caffeinated products at all,” Breuner said.

“These are all sleep hygiene tools that work, but people are very reticent to do them. They rather just take a pill, right?”

There are other proven sleep tips that work just as well, if not better than sleeping aids, experts say. The body begins secreting melatonin at dark. What do we do in our modern culture? Use artificial light to keep us awake, often long past the body’s normal bedtime.

Research has found that the body will slow or stop melatonin production if exposed to light, including the blue light from our smartphones, laptops and the like.

“Any LED spectrum light source may further suppress melatonin levels,” said Dr. Vsevolod Polotsky, who directs sleep basic research in the division of pulmonary and critical care medicine at Johns Hopkins University School of Medicine, in an earlier CNN interview.

So ban those devices at least an hour before you want to fall asleep. Like to read yourself to sleep? That’s fine, experts say, just read in a dim light from a book or use an e-reader in night mode.

“Digital light will suppress the circadian drive,” Polotsky said, while a “dim reading light will not.”

Other tips include keeping your bedroom temperature at cooler temperatures — about 60 to 67 degrees Fahrenheit (15 to 20 degrees Celsius). We sleep better if we’re a bit chilly, experts say.

Set up a bedtime ritual by taking a warm bath or shower, reading a book or listening to soothing music. Or you can try deep breathing, yoga, meditation or light stretches. Go to bed and get up at the same time each day, even on weekends or your days off, experts say. The body likes routine.

If your doctor does prescribe melatonin to help with jet lag or other minor sleep issues, keep the use “short term,” Robbins said.

If you are planning to use melatonin for a short-term sleep aid, try to purchase pharmaceutical grade melatonin, she advised. To find it, look for a stamp showing that the independent, nonprofit US Pharmacopoeial Convention Dietary Supplement Verification Program has tested the product.

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HIV/AIDS Fast Facts | CNN



CNN
 — 

Here’s a look at the origins, treatments and global response to HIV and AIDS.

HIV stands for human immunodeficiency virus.

AIDS stands for acquired immunodeficiency syndrome.

HIV/AIDS is spread through sexual contact with an infected person, sharing needles with an infected person, through transfusions of infected blood or through an infected mother.

People infected with HIV go through three stages of infection:

  1. Acute infection, or acute retroviral syndrome, which can produce flu-like symptoms in the first month after infection.
  2. Clinical latency, or asymptomatic HIV infection, in which HIV reproduces at lower levels.
  3. AIDS, in which the amount of CD4 cells fall below 200 cells per cubic millimeter of blood (as opposed to the normal level of 500-1,500).

HIV-1 and HIV-2 can both cause AIDS. HIV-1 is the most common human immunodeficiency virus; HIV-2 is found mostly in western Africa.

Antiretroviral therapy (ART) involves taking a cocktail of HIV medications used to treat the virus. In 1987, Azidothymidine (AZT) became the first FDA-approved drug used to attempt to treat HIV/AIDS.

from UNAIDS:

38.4 million – Number of people living with HIV/AIDS worldwide in 2021.

5.9 million – Approximate number of people living with HIV globally that are unaware of their HIV-positive status in 2021.

160,000 – Newly infected children worldwide in 2021.

1.5 million – New infections worldwide in 2021.

650,000 – Approximate number of AIDS-related deaths worldwide in 2021.

Of the 4,500 new infections each day in 2019, 59% are in sub-Saharan Africa.

40.1 million – Approximate number of AIDS-related deaths worldwide since the start of the epidemic.

Sub-Saharan Africa is comprised of the following countries: Angola, Benin, Botswana, Burkina Faso, Burundi, Cameroon, Cape Verde, Central African Republic, Chad, Comoros, Democratic Republic of the Congo, Ivory Coast, Djibouti, Equatorial Guinea, Eritrea, Ethiopia, Gabon, The Gambia, Ghana, Guinea, Guinea-Bissau, Kenya, Lesotho, Liberia, Madagascar, Malawi, Mali, Mauritania, Mauritius, Mozambique, Namibia, Niger, Nigeria, Republic of the Congo, Rwanda, Sao Tome and Principe, Senegal, Seychelles, Sierra Leone, Somalia, South Africa, Sudan, South Sudan, Swaziland, Tanzania, Togo, Uganda, Zambia and Zimbabwe.

1981 The Centers for Disease Control and Prevention (CDC) publish the first reports of men in Los Angeles, New York and San Francisco who were previously healthy and are suffering from rare forms of cancer and pneumonia, accompanied by “opportunistic infections.”

1982 The CDC refer to the disease as AIDS for the first time.

1983 French and American researchers determine that AIDS is caused by HIV.

1985 Blood tests to detect HIV are developed.

December 1, 1988 – First World AIDS Day.

1999 Researchers in the United States find evidence that HIV-1 most likely originated in a population of chimpanzees in West Africa. The virus appears to have been transmitted to people who hunted, butchered and consumed the chimpanzees for food.

January 29, 2003 In his State of the Union speech, US President George W. Bush promises to dramatically increase funding to fight HIV/AIDS in Africa.

May 27, 2003 – Bush signs H.R. 1298, the US Leadership Against HIV/AIDS, Tuberculosis and Malaria Act of 2003, also known as PEPFAR (US President’s Emergency Plan for AIDS Relief), that provides $15 billion over the next five years to fight HIV/AIDS, tuberculosis and malaria abroad, particularly in Africa.

July 30, 2008 H.R. 5501, The Tom Lantos and Henry J. Hyde United States Global Leadership Against HIV/AIDS, Tuberculosis, and Malaria Reauthorization Act of 2008, becomes law and authorizes up to $48 billion to combat global HIV/AIDS, tuberculosis and malaria. Through 2013, PEPFAR plans to work in partnership with host nations to support treatment for at least four million people, prevention of 12 million new infections and care for 12 million people.

October 2011 – In his book, “The Origins of AIDS,” Dr. Jacques Pepin traces the emergence and subsequent development of HIV/AIDS to suggest that initial AIDS outbreaks began earlier than previously believed.

July 24, 2012 – Doctors announce during the 19th International AIDS Conference that Timothy Ray Brown, known as the “Berlin patient,” has been clinically “cured” of HIV. Brown, diagnosed with leukemia, underwent a bone marrow transplant in 2007 using marrow from a donor with an HIV-resistant mutation. He no longer has detectable HIV.

March 3, 2013 Researchers announce that a baby born infected with HIV has been “functionally cured.” The child, born in Mississippi, was given high doses of antiretroviral drugs within 30 hours of being born. A year later, the child now has detectable levels of the virus in her blood, 27 months after being taken off antiretroviral drugs, according to scientists involved with her case.

June 18, 2013 Marking the 10th anniversary of PEPFAR, Secretary of State John Kerry announces that the millionth child has been born HIV-free due to prevention of mother-to-child transmission programs (PMTCT).

March 14, 2014 – The CDC reports on a case of likely female-to-female HIV transmission. Unlike previous announcements of other cases involving female-to-female transmission, this case excludes additional risk factors for HIV transmission.

July 24, 2017 – A 9-year-old child from South Africa is reported to have been in remission for over eight years without treatment, according to Dr. Avy Violari, who spoke at the 9th International AIDS Society Conference on HIV Science in Paris.

November 2018 – According to PEPFAR’s website, they have “supported life-saving antiretroviral treatment (ART) for more than 14.6 million men, women and children” since 2003.

March 5, 2019 – According to a case study published in the journal Nature, a second person has sustained remission from HIV-1. The “London patient” was treated with stem cell transplants from donors with an HIV-resistant mutation. The London patient has been in remission for 18 months since he stopped taking antiretroviral drugs. The study also includes a possible third remission after stem cell transplantation, this person is referred to as the “Düsseldorf patient.”

May 2, 2019 – A study of nearly 1,000 gay male couples, where one partner with HIV took antiretroviral therapy (ART), found no new cases of transmission to the HIV-negative partner during sex without a condom. The landmark, eight-year study, published in the Lancet medical journal shows that the risk of passing on the HIV virus is eliminated with effective drugs treatment.

October 7, 2019 – Governor Gavin Newsom signs a bill making HIV prevention drugs available without a prescription in California starting on January 1, 2020. The medications covered by the new legislation are pre-exposure prophylaxis (PrEP) and post-exposure prophylaxis (PEP), which both help prevent HIV infections. California becomes the first state in the country to allow pharmacists to provide the drugs without a physician’s prescription.

November 6, 2019 – According to a study published in the Journal of Acquired Immune Deficiency Syndromes, a team of scientists has detected a new strain of HIV. The strain is a part of the Group M version of HIV-1, the same family of virus subtypes to blame for the global HIV pandemic, according to Abbott Laboratories, which conducted the research along with the University of Missouri, Kansas City.

June 15, 2020 – A study is published in the journal JAMA Network Open showing that the life expectancy of people with HIV approaches that of people without the virus, when antiviral therapy is started early in infection. However, disparities still remain in the number of chronic health problems that people with HIV endure.

July 7, 2020 – Scientists presenting at the 23rd International AIDS Conference announce a new study that found an injection of the investigational drug cabotegravir every eight weeks was more effective at preventing HIV than daily oral pills. It is also announced that a Brazilian man might be the first person to experience long-term HIV remission after being treated with only an antiviral drug regimen – not stem cell transplantation.

November 16, 2021 – A new study finds a second patient whose body has seemingly rid itself of HIV. The international team of scientists reports in the Annals of Internal Medicine that the patient, originally from the city of Esperanza, Argentina, showed no evidence of intact HIV in large numbers of her cells, suggesting that she may have naturally achieved what they describe as a “sterilizing cure” of HIV infection. The 30-year-old woman in the new study is only the second patient who has been described as achieving this sterilizing cure without help from stem cell transplantation or other treatment.

December 20, 2021 – The US Food and Drug Administration announces that it has approved the first injectable medication for pre-exposure prophylaxis (PrEP) to lower the risk of getting HIV through sex.

February 15, 2022 – A US woman becomes the third known person to go into HIV remission, and the first mixed-race woman, thanks to a transplant of stem cells from umbilical cord blood, according to research presented at a conference on Retroviruses and Opportunistic Infections.

December 1, 2022 – An experimental HIV vaccine, called eOD-GT8 60mer, has been found to induce broadly neutralizing antibody precursors among a small group of volunteers in a Phase 1 study. The clinical trial results, published in the journal Science, suggest that a two-dose regimen of the vaccine, given eight weeks apart, can elicit immune responses against the human immunodeficiency virus.

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Opioid Crisis Fast Facts | CNN



CNN
 — 

Here’s a look at the opioid crisis.

Experts say the United States is in the throes of an opioid epidemic. An estimated 9.2 million Americans aged 12 and older misused opioids in 2021, including 8.7 million prescription pain reliever abusers and 1.1 million heroin users.

Opioids are drugs formulated to replicate the pain-reducing properties of opium. Prescription painkillers like morphine, oxycodone and hydrocodone are opioids. Illegal drugs like heroin and illicitly made fentanyl are also opioids. The word “opioid” is derived from the word “opium.”

Nearly 110,000 people died of drug overdoses in 2022, and more than two-thirds of those deaths involved a synthetic opioid.

Overdose deaths have been on the rise for years in the United States, but surged amid the Covid-19 pandemic: Annual deaths were nearly 50% higher in 2021 than in 2019, CDC data shows.

Prescription opioid volumes peaked in 2011, with the equivalent of 240 billion milligrams of morphine prescribed, according to the market research firm, IQVIA Institute for Human Data Science.

Alabama, Arkansas, Louisiana and Tennessee had the highest opioid dispensing rates in 2020.

Opioids such as morphine and codeine are naturally derived from opium poppy plants more commonly grown in Asia, Central America and South America. Heroin is an illegal drug synthesized from morphine.

Hydrocodone and oxycodone are semi-synthetic opioids, manufactured in labs with natural and synthetic ingredients.

Fentanyl is a fully synthetic opioid, originally developed as a powerful anesthetic for surgery. It is also administered to alleviate severe pain associated with terminal illnesses like cancer. The drug is up to 100 times more powerful than morphine. Just a small dose can be deadly. Illicitly produced fentanyl has been a driving factor in the number of overdose deaths in recent years.

Methadone is another fully synthetic opioid. It is commonly dispensed to recovering heroin addicts to relieve the symptoms of withdrawal.

Opioids bind to receptors in the brain and spinal cord, disrupting pain signals. They also activate the reward areas of the brain by releasing the hormone dopamine, creating a feeling of euphoria or a “high.”

Opioid use disorder is the clinical term for opioid addiction or abuse.

People who become dependent on opioids may experience withdrawal symptoms when they stop using the medication. Dependence is often coupled with tolerance, meaning that users need to take increasingly larger doses for the same effect.

A drug called naloxone, available as an injection or a nasal spray, is used as a treatment for overdoses. It blocks or reverses the effects of opioids and is often carried by first responders.

More data on overdose deaths

The 21st Century Cures Act, passed in 2016, allocated $1 billion over two years in opioid crisis grants to states, providing funding for expanded treatment and prevention programs. In April 2017, Health and Human Services Secretary Tom Price announced the distribution of the first round of $485 million in grants to all 50 states and US territories.

In August 2017, Attorney General Jeff Sessions announced the launch of an Opioid Fraud and Abuse Detection Unit within the Department of Justice. The unit’s mission is to prosecute individuals who commit opioid-related health care fraud. The DOJ is also appointing US attorneys who will specialize in opioid health care fraud cases as part of a three-year pilot program in 12 jurisdictions nationwide.

On October 24, 2018, President Donald Trump signed opioid legislation into law. The SUPPORT for Patients and Communities Act includes provisions aimed at promoting research to find new drugs for pain management that will not be addictive. It also expands access to treatment for substance use disorders for Medicaid patients.

State legislatures have also introduced measures to regulate pain clinics and limit the quantity of opioids that doctors can dispense.

1861-1865 – During the Civil War, medics use morphine as a battlefield anesthetic. Many soldiers become dependent on the drug.

1898 – Heroin is first produced commercially by the Bayer Company. At the time, heroin is believed to be less habit-forming than morphine, so it is dispensed to individuals who are addicted to morphine.

1914 – Congress passes the Harrison Narcotics Act, which requires that doctors write prescriptions for narcotic drugs like opioids and cocaine. Importers, manufacturers and distributors of narcotics must register with the Treasury Department and pay taxes on products

1924 – The Anti-Heroin Act bans the production and sale of heroin in the United States.

1970 – The Controlled Substances Act becomes law. It creates groupings (or schedules) of drugs based on the potential for abuse. Heroin is a Schedule I drug while morphine, fentanyl, oxycodone (Percocet) and methadone are Schedule II. Hydrocodone (Vicodin) is originally a Schedule III medication. It is later recategorized as a Schedule II drug.

January 10, 1980 – A letter titled “Addiction Rare in Patients Treated with Narcotics” is published in the New England Journal of Medicine. It looks at incidences of painkiller addiction in a very specific population of hospitalized patients who were closely monitored. It becomes widely cited as proof that narcotics are a safe treatment for chronic pain.

1995 – OxyContin, a long-acting version of oxycodone that slowly releases the drug over 12 hours, is introduced and aggressively marketed as a safer pain pill by manufacturer, Purdue Pharma.

May 10, 2007 – Purdue Pharma pleads guilty for misleadingly advertising OxyContin as safer and less addictive than other opioids. The company and three executives are charged with “misleading and defrauding physicians and consumers.” Purdue and the executives agree to pay $634.5 million in criminal and civil fines.

2010 – FDA approves an “abuse-deterrent” formulation of OxyContin, to help curb abuse. However, people still find ways to abuse it.

May 20, 2015 – The DEA announces that it has arrested 280 people, including 22 doctors and pharmacists, after a 15-month sting operation centered on health care providers who dispense large amounts of opioids. The sting, dubbed Operation Pilluted, is the largest prescription drug bust in the history of the DEA.

March 18, 2016 – The CDC publishes guidelines for prescribing opioids for patients with chronic pain. Recommendations include prescribing over-the-counter pain relievers like acetaminophen and ibuprofen in lieu of opioids. Doctors are encouraged to promote exercise and behavioral treatments to help patients cope with pain.

March 29, 2017 – Trump signs an executive order calling for the establishment of the President’s Commission on Combating Drug Addiction and the Opioid Crisis. New Jersey Governor Chris Christie is selected as the chairman of the group, with Trump’s son-in-law, Jared Kushner, as an adviser.

July 31, 2017 – After a delay, the White House panel examining the nation’s opioid epidemic releases its interim report, asking Trump to declare a national public health emergency to combat the ongoing crisis

September 22, 2017 – The pharmacy chain CVS announces that it will implement new restrictions on filling prescriptions for opioids, dispensing a limited seven-day supply to patients who are new to pain therapy.

November 1, 2017 – The opioid commission releases its final report. Its 56 recommendations include a proposal to establish nationwide drug courts that would place opioid addicts in treatment facilities rather than prison.

February 9, 2018 – A budget agreement signed by Trump authorizes $6 billion for opioid programs, with $3 billion allocated for 2018 and $3 billion allocated for 2019.

February 27, 2018 – Sessions announces a new opioid initiative: The Prescription Interdiction & Litigation (PIL) Task Force. The mission of the task force is to support local jurisdictions that have filed lawsuits against prescription drugmakers and distributors.

March 19, 2018 – The Trump administration outlines an initiative to stop opioid abuse. The three areas of concentration are law enforcement and interdiction; prevention and education via an ad campaign; and job-seeking assistance for individuals fighting addiction.

April 9, 2018 – The US surgeon general issues an advisory recommending that Americans carry the opioid overdose-reversing drug, naloxone. A surgeon general advisory is a rarely used tool to convey an urgent message. The last advisory issued by the surgeon general, more than a decade ago, focused on drinking during pregnancy.

May 1, 2018 – The Journal of the American Medical Association publishes a study that finds synthetic opioids like fentanyl caused about 46% of opioid deaths in 2016. That’s a three-fold increase compared with 2010, when synthetic opioids were involved in about 14% of opioid overdose deaths. It’s the first time that synthetic opioids surpassed prescription opioids and heroin as the primary cause of overdose fatalities.

May 30, 2018 – The journal Medical Care publishes a study that estimates the cost of medical care and substance abuse treatment for opioid addiction was $78.5 billion in 2013.

June 7, 2018 – The White House announces a new multimillion dollar public awareness advertising campaign to combat opioid addiction. The first four ads of the campaign are all based on true stories illustrating the extreme lengths young adults have gone to obtain the powerful drugs.

December 12, 2018 – According to the National Center for Health Statistics, fentanyl is now the most commonly used drug involved in drug overdoses. The rate of drug overdoses involving the synthetic opioid skyrocketed by about 113% each year from 2013 through 2016.

January 14, 2019 – The National Safety Council finds that, for the first time on record, the odds of dying from an opioid overdose in the United States are now greater than those of dying in a vehicle crash.

March 26, 2019 – Purdue Pharma agrees to pay a $270 million settlement to settle a historic lawsuit brought by the Oklahoma attorney general. The settlement will be used to fund addiction research and help cities and counties with the opioid crisis.

July 17, 2019 – The CDC releases preliminary data showing a 5.1% decline in drug overdoses during 2018. If the preliminary number is accurate, it would mark the first annual drop in overdose deaths in more than two decades.

August 26, 2019 – Oklahoma wins its case against Johnson & Johnson in the first major opioid lawsuit trial to be held in the United States. Cleveland County District Judge Thad Balkman orders Johnson & Johnson to pay $572 million for its role in the state’s opioid crisis. The penalty is later reduced to $465 million, due to a mathematical error made when calculating the judgment. In November 2021, the Oklahoma Supreme Court reverses the decision.

September 15, 2019 – Purdue files for bankruptcy as part of a $10 billion agreement to settle opioid lawsuits. According to a statement from the chair of Purdue’s board of directors, the money will be allocated to communities nationwide struggling to address the crisis.

September 30, 2019 – The FDA and DEA announce that they sent warnings to four online networks, operating a total of 10 websites, which the agencies said are illegally marketing unapproved and misbranded versions of opioid medicines, including tramadol.

February 25, 2020 – Mallinckrodt, a large opioid manufacturer, reaches a settlement agreement in principle worth $1.6 billion. Mallinckrodt says the proposed deal will resolve all opioid-related claims against the company and its subsidiaries if it moves forward. Plaintiffs would receive payments over an eight-year period to cover the costs of opioid-addition treatments and other needs.

October 21, 2020 – The Justice Department announces that Purdue Pharma, the maker of OxyContin, has agreed to plead guilty to three federal criminal charges for its role in creating the nation’s opioid crisis. They agree to pay more than $8 billion and close down the company. The money will go to opioid treatment and abatement programs. The Justice Department also reached a separate $225 million civil settlement with the former owners of Purdue Pharma, the Sackler family. In November 2020, Purdue Pharma board chairman Steve Miller formally pleads guilty on behalf of the company.

March 15, 2021 – According to court documents, Purdue files a restructuring plan to dissolve itself and establish a new company dedicated to programs designed to combat the opioid crisis. As part of the proposed plan, the Sackler family agrees to pay an additional $4.2 billion over the next nine years to resolve various civil claims.

September 1, 2021 – In federal bankruptcy court, Judge Robert Drain rules that Purdue Pharma will be dissolved. The settlement agreement resolves all civil litigation against the Sackler family members, Purdue Pharma and other related parties and entities, and awards them broad legal protection against future civil litigation. The Sacklers will relinquish control of family foundations with over $175 million in assets to the trustees of a National Opioid Abatement Trust. On December 16, 2021, a federal judge overturns the settlement.

February 25, 2022 – Johnson & Johnson and the three largest US drug distributors – McKesson Corp, Cardinal Health Inc and AmerisourceBergen Corp – finalize a $26 billion nationwide opioid settlement.

March 3, 2022 – The Sackler families reaches a settlement with a group of states the first week of March, according to court filings. The settlement, ordered through court-ordered mediation that began in January, requires the Sacklers to pay out as much as $6 billion to states, individual claimants and opioid crisis abatement, if approved by a federal bankruptcy court judge.

November 2, 2022 – CVS and Walgreens agree to pay a combined $10 billion, over 10 and 15 years, to settle lawsuits brought by states and local governments alleging the retailers mishandled prescriptions of opioid painkillers.

November 15, 2022 – Walmart agrees to the framework of a $3.1 billion settlement, which resolves allegations from multiple states’ attorneys general that the company failed to regulate opioid prescriptions contributing to the nationwide opioid crisis.

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Covid-19 is no longer a public health emergency, but others remain | CNN



CNN
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The Covid-19 pandemic hit a major milestone this month as public health emergency declarations were ended by both the United States government and the World Health Organization. Emergency declarations for mpox also recently ended.

This doesn’t mean Covid-19 and mpox are no longer of concern, but it does mark the end of the availability of certain logistical capabilities to manage them.

Still, other critical health challenges were identified as health emergencies years before the start of the Covid-19 pandemic and continue to be so: the opioid crisis in the US and the global spread of poliovirus.

Some public health concerns can be serious health threats without a formal emergency declaration, said Dr. Tom Frieden, former director of the US Centers for Disease Control and Prevention.

It largely boils down to the logistics of government operations.

“When there’s an unusual situation that requires multiple parts of an agency or multiple parts of the government or multiple parts of society to come together and coordinate, collaborate and work efficiently, then an emergency declaration can be a useful tool,” said Frieden, president and CEO of Resolve to Save Lives, an organization focused on global epidemic prevention and cardiovascular health.

“Sometimes, an emergency is declared to make the point that it’s a big problem, to get people’s attention. Sometimes, an emergency is declared to get things done because that’s the only way you can bring certain governmental capacities to bear.”

According to WHO, a public health emergency of international concern is “an extraordinary event” that poses public health risk through the international spread of disease.

It creates an agreement between countries to abide by WHO’s recommendations for managing the emergency, often requiring a “coordinated international response.” Each country, in turn, declares its own public health emergency – declarations that carry legal weight. Countries use them to marshal resources and waive rules in order to ease a crisis.

In the US, the secretary of the Department of Health and Human Services can declare a public health emergency for diseases or disorders that pose a threat, including “significant outbreaks” of an infectious disease, bioterrorist attacks or otherwise.

This triggers the availability of a set of resources and actions for the federal government, such as additional funds and data and reporting requirements.

Emergency declarations typically last up to 90 days, with formal renewal required as necessary every three months after that.

The opioid crisis was determined to be a public health emergency in October 2017, during the Trump administration, driven by the rising rates of opioid-related deaths and opioid use disorder.

The declaration has been renewed for more than five years, most recently at the end of March.

According to the CDC, the opioid epidemic started in 1999 with a rise in prescription opioid overdose deaths. Deaths started to increase precipitously as synthetic opioids – particularly fentanyl – started to take over in 2013.

In 2021, overdose deaths reached record levels in the US, and about three-quarters – more than 80,000 deaths – involved opioids.

Within the first year of the opioid emergency declaration, HHS used expanded authorities to field a survey about treatment for opioid use disorder among providers and to expedite research on the topic.

Public health emergencies are also declared to help with recovery after natural disasters, most recently for severe storms that hit Mississippi in March.

However, the Government Accountability Office considers both federal management of the public health emergency system and efforts to combat drug misuse to be “high-risk” areas that are vulnerable or in need of broad reform.

In a recent report, the federal watchdog group said that it has found “persistent deficiencies in HHS’s leadership role preparing for and responding to public health emergencies” and no demonstrated progress in federal agencies’ actions to address drug misuse.

WHO has considered poliovirus a public health emergency of international concern since 2014.

A committee formed to address the emergency reviewed the most recent data on cases and spread this month and voted unanimously that ongoing risks merited an extension of the emergency declaration, which the director-general formalized Friday.

The committee was “encouraged by reported progress” but says that risks remain high for factors including weak vaccination rates that could have been affected by the Covid-19 pandemic.

WHO identified seven counties with potential risk for international spread. And the US is among a group of 37 countries with recently detected cases.

In July, a polio case identified in New York became the first in the US in nearly a decade. The identified case, along with several positive wastewater tests in nearby communities, met WHO criteria to consider the US a country with circulating poliovirus.

Experts warned that it could just be the “tip of the iceberg,” with hundreds of cases spreading silently.

Childhood vaccination rates in the US dropped during the Covid-19 pandemic. A CDC report found that about 93% of kindergarteners enrolled in the 2021-22 school year got the required vaccines, including measles, mumps and rubella (MMR); diphtheria, tetanus and acellular pertussis (DTaP); and polio. Coverage fell for the second year in a row amid the pandemic, from about 94% the previous year and below the federal target of 95%.

While no longer under a formal emergency declaration, Covid-19 continues to be part of the “landscape of health threats,” Frieden said.

But the efficiency and coordination that the formal declaration helps facilitate should always be the goal.

“I think there are really important lessons from Covid, including the need to have a much more resilient public health system so that we can find problems quickly and implement effective solutions quickly,” Frieden said.

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FDA advisers narrowly vote in favor of experimental gene therapy for rare muscle disease | CNN



CNN
 — 

Most parents wouldn’t be thrilled with the idea of their kids getting hooked up to an IV bag filled with trillions of viruses.

But for Melanie Hennick, whose son, Connor, has Duchenne muscular dystrophy, it was an opportunity she hoped would change his life.

“We knew this wasn’t a cure,” Hennick said. “But it was a chance.”

Connor is one of just dozens of kids to have received SRP-9001, an experimental gene therapy that aims to slow or stop the progression of Duchenne muscular dystrophy, or DMD. Current treatments for the disease – which primarily affects boys because of the way it’s inherited – include steroids and, later, heart drugs. But none stop it.

SRP-9001 uses viruses to ferry a copy of a gene to muscles to help make up for one that’s causing the disease. Hennick and many other parents like her advocated for the treatment’s accelerated approval Friday in a meeting of outside advisers to the US Food and Drug Administration.

The advisers voted 8-6 in favor of approving the treatment, and the FDA will now decide whether to follow their advice.

“The decision the FDA has to make doesn’t just affect the patients in study 301 [an ongoing confirmatory trial that Sarepta is running]; it affects the entire field of drug development for Duchenne,” said Dr. Caleb Alexander, a professor of epidemiology and medicine in the Bloomberg School of Public Health at Johns Hopkins University, who voted against recommending approval. “The totality of evidence … simply doesn’t rise to the threshold that’s required for accelerated approval.”

Dr. Raymond Roos, a neurology professor at the University of Chicago Medical Center, voted in favor. “The downside of the gene therapy here is relatively small compared to whether it really helps the patient, and for this reason, I voted yes,” he said.

Dr. Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, said his agency will take the recommendation and “do something that we have to do every day at FDA. … We have to manage through the uncertainty here.”

The FDA’s decision, expected by the end of the month, will have implications not just for families like Connor’s but for how the agency regulateths treatments like this one more broadly: It would be the first of its kind of medicine – one-time treatments delivering a gene to try to fix a disease – to get accelerated approval, a faster track through the regulatory process. Its approval would set a precedent for other drugs like this based on so-called surrogate endpoints, a measure of what the drug does in the body, before further clinical evidence is available.

“Approval of a gene therapy for Duchenne muscular dystrophy will be huge,” said Jeffrey Chamberlain, a professor at the University of Washington School of Medicine who helped pioneer gene therapy approaches for the disease. “This, I think, will spur further research and further development of gene therapies for other diseases.”

DMD patients don’t have a lot of time to wait. Kids with Duchenne typically lose the ability to walk before they’re teenagers and often don’t live well into their 30s, Chamberlain said. He’s not directly involved with SRP-9001, which is being developed by Sarepta Therapeutics, and is on the scientific advisory board for another company working on DMD gene therapies, Solid Biosciences.

“Gene therapy appears to be a really good approach to try to treat this disease, because it’s a genetic disease,” Chamberlain said. “The cause of the disease is a mutation in a single gene.”

That gene is responsible for the production of dystrophin, a protein key to the structure of muscle cells.

“It’s kind of like the two-by-fours that make up your house,” Chamberlain said. “It’s really important for just holding everything together.”

SRP-9001, invented at Nationwide Children’s Hospital in Columbus, Ohio, before being licensed for development by Sarepta, delivers a miniaturized version of the dystrophin gene to cells, aiming to help them make a version of the muscle-preserving protein.

In a key clinical trial, the therapy appeared to do that. But it didn’t meet another main goal: showing a benefit on a measure of muscle function, complicating SRP-9001’s path through the FDA.

Sarepta blamed the outcome on an imbalance in how the trial separated patients into the placebo and treatment groups. But key FDA reviewers appear unconvinced.

“The clinical studies conducted to date do not provide unambiguous evidence that SRP-9001 is likely beneficial for ambulatory patients with DMD,” agency reviewers wrote in briefing documents released ahead of Friday’s meeting, referring to patients who can still walk – the group who will initially be eligible for the treatment if it gets approved.

Family after family who participated in Sarepta’s trials, like the Hennicks, disagree with the reviewers. They say they believe that the treatment has helped keep their kids walking and running in ways they never would have without it.

“It’s really miraculous,” said Nate Plasman, whose son Andrew got SRP-9001 as part of the trial in January 2019, at age 4.

Sara, left, Andrew and Nate Plasman on the day he was dosed in the trial in January 2019.

Andrew was away from school for more than two months when he got the experimental therapy, Plasman said, and when he returned, “his teachers at the preschool were blown away,” he recalled. “They’re like, ‘Who is this kid?’ He’s running. He’s jumping. He’s pedaling the tricycle. He’s getting up and down off the ground” – all things he couldn’t do as well before the therapy.

Marit Sivertson, mom to 9-year-old Brecken, agrees.

“We’ve seen the incredible changes with our son,” she said. “He’s not just walking around. He’s running; he’s swimming; he’s diving. He’s truly living the life that every 9-year-old boy ought to be living.”

Dr. Jerry Mendell of Nationwide Children's Hospital in Ohio, who developed the gene therapy, left, with Brecken Kinney.

Sivertson and Plasman also spoke at Friday’s meeting. Their goal isn’t to secure the therapy for their own kids; because it’s designed as a one-time treatment, they wouldn’t take it again. They say they’re speaking up on behalf of children who are still waiting.

That wait is especially painful for Daniel and Lindsey Flessner, who have two sons with DMD. Their 5-year-old son, Mason, is in the SRP-9001 clinical trial. Their 2-year-old, Dawson, is still too young.

“With every trip, every fall, every time he stands up by walking up his legs using his hands to help stabilize him, it just keeps chipping away at us,” Flessner said. “It’s very painful as the parents watching your children struggling knowing all you can do is wait, when waiting is what you don’t have time for.”

Lindsey and Daniel Flessner's sons, Mason and Dawson, both have DMD.

In addition to questions about how well the treatment works, the FDA reviewers raised concerns about safety, particularly “related to the possibility of administering an ineffective gene therapy.”

The reviewers focused on opportunity cost: Because of the viruses used to deliver the gene, patients can develop an immune response that could render future doses ineffective.

Chamberlain said work is underway to find ways to be able to administer more doses, if needed, but it’s currently a one-and-done treatment.

For now, he thinks this approach is the best hope for DMD patients.

“It’s not perfect,” he acknowledged. “It’s not a complete cure, but from what I can gauge from the clinical results that have been released by Sarepta and some of the other companies, I think the micro-dystrophin gene therapy is working better than any other drug that’s been tried for Duchenne muscular dystrophy.”

It’s unclear how long the effects will last; Sarepta is continuing trials, and a confirmatory study would be required as part of accelerated approval. Sarepta has proposed a trial that it’s already running to satisfy that requirement, with results expected later this year.

For families facing DMD, there’s an opportunity cost to waiting, too. In its briefing documents for the FDA meeting, Sarepta estimated that accelerated approval would speed up broad access to SRP-9001 by at least a year, a time in which about 400 boys could lose the ability to walk and another 400, whose disease is more advanced, would die.

Melanie Hennick said Connor was admitted to the trial just weeks before he’d have aged out, at 8 years old. She said she believes the therapy is the reason Connor’s doing so well.

“We had the opportunity to see Connor grow as an 8-, 9-, 10-, 11- and 12-year-old with more capacity than we ever dreamed,” she said. “He climbs stairs unaided; he runs around; he plays football; he plays hockey; he plays on a baseball team. … Those are things that we never thought we would be able to see him do, especially at 12.”

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How a medication abortion, also known as an ‘abortion pill,’ works | CNN



CNN
 — 

While legal battles over access to mifepristone, one of two drugs used for medication abortions, play out in court, the drug continues to be available in states which consider abortion legal.

“While many women obtain medication abortion from a clinic or their OB-GYN, others obtain the pills on their own to self-induce or self-manage their abortion,” said Dr. Daniel Grossman, a professor of obstetrics, gynecology and reproductive sciences at the University of California, San Francisco.

“A growing body of research indicates that self-managed abortion is safe and effective,” he said.

Mifepristone blocks the hormone progesterone, which is needed for a pregnancy to continue. The drug is approved to end a pregnancy through 10 weeks’ gestation, which is “70 days or less since the first day of the last menstrual period,” according to the FDA.

In a medication abortion, a second drug, misoprostol, is taken within the next 24 to 48 hours. Misoprostol causes the uterus to contract, creating cramping and bleeding. Approved for use in other conditions, such as preventing stomach ulcers, the drug has been available at pharmacies for decades.

Together, the two drugs are commonly known as the “abortion pill,” which is now used in more than half of the abortions in the United States, according to the Guttmacher Institute, a research group that supports abortion rights.

“Some people do this because they cannot access a clinic — particularly in states with legal restrictions on abortion — or because they have a preference for self-care,” said Grossman, who is also the director of Advancing New Standards in Reproductive Health, a research group that evaluates the pros and cons of reproductive health policies and publishes studies on how abortion affects a woman’s health.

What happens during a medication abortion? To find out, CNN spoke with Grossman. The conversation has been edited for clarity.

CNN: What is the difference between a first-trimester medication abortion and a vacuum aspiration in terms of what a woman experiences?

Dr. Daniel Grossman: A vacuum aspiration is most commonly performed under a combination of local anesthetic and oral pain medications or local anesthetic together with intravenous sedation, or what is called conscious sedation.

An injection of local anesthetic is given to the area around the cervix, and the cervix is gently dilated or opened up. Once the cervix is opened, a small straw-like tube is inserted into the uterus, and a gentle vacuum is used to remove the pregnancy tissue. Contrary to what some say, if the procedure is done before nine weeks or so, there’s nothing in the tissue that would be recognizable as a part of an embryo.

The aspiration procedure takes just a couple of minutes; then the person is observed for one to two hours until any sedation has worn off. We also monitor each patient for very rare complications, such as heavy bleeding.

Grossman: A medication abortion is a more prolonged process. After taking the pills, bleeding and cramping can occur over a period of days. Bleeding is typically heaviest when the actual pregnancy is expelled, but that bleeding usually eases within a few hours. On average people continue to have some mild bleeding for about two weeks or so, which is a bit longer than after a vacuum aspiration.

Nausea, vomiting, fever, chills, diarrhea and headache can occur after using the abortion pill, and everyone who has a successful medication abortion usually reports some pain.

In fact, the pain of medication abortion can be quite intense. In the studies that have looked at it, the average maximum level of pain that people report is about a seven to eight out of 10, with 10 being the highest. However, people also say that the pain can be brief, peaking just as the pregnancy is being expelled.

The level of cramping and pain can depend on the length of the pregnancy as well as whether or not someone has given birth before. For example, a medical abortion at six weeks or less gestation typically has less pain and cramping than one performed at nine weeks. People who have given birth generally have less pain.

CNN: What can be done to help with the pain of a medication abortion?

Grossman: There are definitely things that can be used to help with the pain. Research has shown that ibuprofen is better than acetaminophen for treating the pain of medication abortion. We typically advise people to take 600 milligrams every six hours or so as needed.

Some people take tramadol, a narcotic analgesic, or Vicodin, which is a combination of acetaminophen and hydrocodone. Recent research I was involved in found medications like tramadol can be helpful if taken prophylactically before the pain starts.

Another successful regimen that we studied combined ibuprofen with a nausea medicine called metoclopramide that also helped with pain. Other than ibuprofen, these medications require a prescription.

Another study found that a TENS device, which stands for transcutaneous electrical nerve stimulator, helps with the pain of medication abortion. It works through pads put on the abdomen that stimulate the nerves through mild electrical shocks, thus interfering with the pain signals. That’s something people could get without a prescription.

Pain can be an overlooked issue with medication abortion because, quite honestly, as clinicians, we’re not there with patients when they are in their homes going through this. But as we’ve been doing more research on people’s experiences with medication abortion, it’s become quite clear that pain control is really important. I think we need to do a better job of treating the pain and making these options available to patients.

CNN: Are there health conditions that make the use of a medication abortion unwise?

Grossman: Undergoing a medication abortion can be dangerous if the pregnancy is ectopic, meaning the embryo is developing outside of the uterus. It’s rare, happening in about two out of every 100 pregnancies — and it appears to be even rarer among people seeking medication abortion.

People who have undergone previous pelvic, fallopian tube or abdominal surgery are at higher risk of an ectopic pregnancy, as are those with a history of pelvic inflammatory disease. Certain sexually transmitted infections can raise risk, as does smoking, a history of infertility and use of infertility treatments such as in vitro fertilization (IVF).

If a person is on anticoagulant or blood thinning drugs or has a bleeding disorder, a medication abortion is not advised. The long-term use of steroids is another contraindication for using the abortion pill.

Anyone using an intrauterine device, or IUD, must have it removed before taking mifepristone because it may be partially expelled during the process, which can be painful.

People with chronic adrenal failure or who have inherited a rare disorder called porphyria are not good candidates.

CNN: Are there any signs of trouble a woman should watch for after undergoing a medication abortion?

Grossman: It can be common to have a low-grade fever in the first few hours after taking misoprostol, the second drug in a medication abortion. If someone has a low-grade fever — 100.4 degrees to 101 degrees Fahrenheit — that lasts more than four hours, or has a high fever of over 101 degrees Fahrenheit after taking the medications, they do need to be evaluated by a health care provider.

Heavy bleeding, which would be soaking two or more thick full-size pads an hour for two consecutive hours, or a foul-smelling vaginal discharge should be evaluated as well.

One of the warning signs of an ectopic pregnancy is severe pelvic pain, particularly on one side of the abdomen. The pain can also radiate to the back. Another sign is getting dizzy or fainting, which could indicate internal bleeding. These are all very rare complications, but it’s wise to be on the lookout.

We usually recommend that someone having a medication abortion have someone with them during the first 24 hours after taking misoprostol or until the pregnancy has passed. Many people specifically choose to have a medication abortion because they can be surrounded by a partner, family or friends.

Most people know that the abortion is complete because they stop feeling pregnant, and symptoms such as nausea and breast tenderness disappear, usually within a week of passing the pregnancy. A home urine pregnancy test may remain positive even four to five weeks after a successful medication abortion, just because it takes that long for the pregnancy hormone to disappear from the bloodstream.

If someone still feels pregnant, isn’t sure if the pregnancy fully passed or has a positive pregnancy test five weeks after taking mifepristone, they need to be evaluated by a clinician.

People should know that they can ovulate as soon as two weeks after a medication abortion. Most birth control options can be started immediately after a medication abortion.

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FDA advisers vote unanimously in support of over-the-counter birth-control pill | CNN



CNN
 — 

Advisers for the US Food and Drug Administration voted unanimously on Wednesday in support of making the birth-control pill Opill available over-the-counter, saying the benefits outweigh the risks.

Two FDA advisory panels agreed that people would use Opill safely and effectively and said groups such as adolescents and those with limited literacy would be able to take the pill at the same time every day without help from a health care worker.

The advisers were asked to vote on whether people were likely to use the tablet properly so that the benefits would exceed the risks. Seventeen voted yes. Zero voted no or abstained.

Opill manufacturer Perrigo hailed the vote as a “groundbreaking” move for women’s health.

“Perrigo is proud to lead the way in making contraception more accessible to women in the U.S.,” Murray Kessler, Perrigo’s president and CEO, said in a statement. “We are motivated by the millions of people who need easy access to safe and effective contraception.”

The FDA doesn’t have to follow its advisers’ advice, but it often does. It is expected to decide whether to approve the over-the-counter pill this summer.

If it’s approved, this will be the first birth-control pill available over the counter in the United States. Opill is a “mini-pill” that uses only the hormone progestin.

At Wednesday’s meeting, Dr. Margery Gass of the University of Cincinnati College of Medicine thanked the FDA for its consideration of switching Opill to an over-the-counter product.

“I think this represents a landmark in our history of women’s health. Unwanted pregnancies can really derail a woman’s life, and especially an adolescent’s life,” she said.

The FDA has faced pressure to allow Opill to go over-the-counter from lawmakers as well as health care providers.

Unwanted pregnancies are a public health issue in the US, where almost half of all pregnancies are unintended, and rates are especially high among lower-income women, Black women and those who haven’t completed high school.

In March 2022, 59 members of Congress wrote a letter to FDA Commissioner Dr. Robert Califf about OTC contraception.

“This is a critical issue for reproductive health, rights, and justice. Despite decades of proven safety and effectiveness, people still face immense barriers to getting birth control due to systemic inequities in our healthcare system,” the lawmakers wrote.

A recent study showed that it’s become harder for women to access reproductive health care services more broadly – such as routine screenings and birth control – in recent years.

About 45% of women experienced at least one barrier to reproductive health care services in 2021, up 10% from 2017. Nearly 19% reported at least three barriers in 2021, up from 16% in 2017.

Increasing reproductive access for women and adolescents was a resounding theme among the FDA advisers.

“We can take this opportunity to increase access, reduce disparities and, most importantly, increase the reproductive autonomy of the women of our nation,” said Dr. Jolie Haun of the James A. Haley Veterans’ Hospital and the University of Utah.

Dr. Karen Murray, deputy director of the FDA’s Office of Nonprescription Drugs, said the agency understands the importance of “increased access to effective contraception” but hinted that the FDA would need more data from the manufacturer.

Some of the advisers and FDA scientists expressed concern that some of Perrigo’s data was unreliable due to overreporting of “improbable dosing.”

Murray said the lack of sufficient information from the study poses challenges for approval.

“It would have been a much easier time for the agency if the applicant had submitted a development program and an actual use study that was very easy to interpret and did not have so many challenges. But that was not what happened for us. And so the FDA has been put in a very difficult position of trying to determine whether it is likely that women will use this product safely and effectively in the nonprescription setting,” she said. “But I wanted to again emphasize that FDA does realize how very important women’s health is and how important it is to try to increase access to effective contraception for US women.”

Ultimately, the advisers said, they don’t want further studies of Opill to delay the availability of the product in an over-the-counter setting.

“I just wanted to say that the improbable dosing issue is important, and I don’t think it’s been adequately addressed and certainly leads to some uncertainty in the findings. But despite this, I would not recommend another actual use study this time, and I think we can make a decision on the totality of the evidence,” said Kate Curtis of the US Centers for Disease Control and Prevention.

Curtis said she voted yes because “Opill has the potential to have a huge positive public health impact.”

Earlier in the discussion, Dr. Leslie Walker-Harding of the University of Washington and Seattle Children’s Hospital said the pill is just as safe as many other medications available on store shelves.

“The safety profile is so good that we would need to take every other medicine off the market like Benadryl, ibuprofen, Tylenol, which causes deaths and people can get any amount of that without any oversight. And this is extremely safe, much safer than all three of those medications, and incorrect use still doesn’t appear to have problematic issues,” she said.

Dr. Katalin Roth of the George Washington University School of Medicine and Health Sciences also emphasized the safety of the pill over the 50 years it has been approved as a prescription drug in the US.

“The risks to women of an unintended pregnancy are much greater than any of the things we were discussing as risks of putting this pill out out over-the-counter,” she said. “The history of women’s contraception is a struggle for women’s control over their reproduction, and we need to trust women.”

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Millions of people are prescribed antidepressants for chronic pain. Do they work? | CNN

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CNN
 — 

Around one-third of people globally live with chronic pain — pain experienced for more than three months — and millions of people are prescribed antidepressants to relieve the condition.

However, a new review of prior research published Tuesday has found that most antidepressants used to relieve chronic pain are being prescribed without sufficient reliable evidence of their effectiveness. What’s more, potential harms haven’t been well studied.

A two-year study by the nonprofit group Cochrane found that only one antidepressant, duloxetine, was effective for short-term pain relief based on the available evidence. Cochrane is an international collaboration of researchers that produces the Cochrane Library, which includes a database of systematic summaries addressing key questions in health care.

Sold under the brand names Irenka and Cymbalta, duloxetine is a serotonin and norepinephrine reuptake inhibitor, or SNRI, and also boosts levels of the feel-good neurochemical dopamine.

“This is a global public health concern,” said lead author Tamar Pincus, a professor and chronic pain researcher at the University of Southampton in the United Kingdom.

“Chronic pain is a problem for millions who are prescribed antidepressants without sufficient scientific proof they help, nor an understanding of the long-term impact on health.”

The review included 176 studies with a total of 28,664 participants and looked at 25 different antidepressants. The studies mainly investigated three types of chronic pain: fibromyalgia, nerve pain and musculoskeletal pain.

The average length of the study was 10 weeks, and the studies were randomized controlled trials — regarded as the gold standard in medical research. Seventy-two of the studies were funded by pharmaceutical companies.

The most commonly prescribed antidepressant for chronic pain globally was amitriptyline, the study said. Sold in the United States under the brand names Elavil and Vanatrip, the antidepressant was approved in 1961 by the US Food and Drug Administration to treat depression in adults. The medication has significant side effects, so it is not commonly used for depression, but is prescribed to treat migraines and chronic pain such as diabetic neuropathy.

However, the authors found most of the studies on amitriptyline’s effectiveness were small and the evidence was not reliable.

Milnacipran, which is approved by the FDA for fibromyalgia, was also effective at reducing pain, the review found, but the scientists were not as confident about this drug compared with duloxetine due to limited studies with few people.

Anyone taking antidepressants for chronic pain relief should speak to their doctor before stopping their medication due to concerns over the new report, the authors stressed.

Antidepressants are thought to help with pain because the bodily systems that regulate mood and pain overlap, explained Ryan Patel, a research fellow studying chronic pain at the Wolfson Centre for Age-Related Diseases at King’s College London.

He said the key question for researchers to answer was not whether antidepressant drugs were effective for treating pain but “for whom are antidepressants effective?”

“Even when the cause of chronic pain is the same, the biological changes that occur in the nervous system are varied and so it is no surprise that pain presents differently from person to person, and not everyone will respond to the same drugs,” said Patel, who wasn’t involved in the review.

“What this comprehensive analysis demonstrates is that when clinical trials are designed poorly under the assumption that everyone’s experience of pain is uniform, most antidepressants appear to have limited use for treating chronic pain,” Patel added in a statement.

Even for the antidepressant duloxetine, there was no research looking at long-term use of the drug, the review found.

“Though we did find that duloxetine provided short-term pain relief for patients we studied, we remain concerned about its possible long-term harm due to the gaps in current evidence,” Pincus said.

The report said future research should address any unwanted effects of using antidepressants for chronic pain, noting that the existing data on this was “poor.”

“It (duloxetine) does look really good at the moment for short term pain relief, but I want to emphasize that patients aren’t prescribed duloxetine or any antidepressant for three weeks, four weeks, six weeks, they’re prescribed it for six months. So it’s really shocking that we don’t have any evidence for long term use of even duloxetine,” Pincus said.

Dr. Cathy Stannard, the clinical lead for the UK National Institute for Health and Care Excellence (NICE) guideline for chronic pain, and a pain specialist for NHS Gloucestershire’s Integrated Care Board in the UK, said that it was important to emphasize the social and psychological influences on how people experience pain and the importance of a patient’s relationship with their doctor.

“There is good evidence that for people with pain, compassionate and consistent relationships with clinicians remain the foundations of successful care,” Stannard, who wasn’t involved in the research, said in a statement.

“Research shows that what people want most is a strong, empathic relationship with their care provider. They want time to discuss what matters to them and they want easy access to support and to be partners in their care.”

Non-pharmaceutical interventions, such as support with mobility, debt management, trauma and social isolation, were also likely to help people living with pain, she added.

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