Doctors diagnosed Katrina Barry with pulmonary arterial hypertension after she suffered a heart attack at age 25.

(Credit: Courtesy of Katrina Barry)

(CNN) --

When doctors told Katrina Barry she had a rare and serious condition called pulmonary arterial hypertension, or PAH, they warned her not to look up information on the Internet.

Come on, he thought;

Did they want a young woman heading to graduate school, who had survived a transatlantic flight while she had a heart attack and now open heart surgery, not to pursue the condition that kept trying to kill her?

Waiting for her on the internet was disturbing information.

PAH affects approximately 500 to 1,000 Americans each year, often women between the ages of 30 and 60, according to the American Lung Association.

Barry, who was 25 years old at the time, discovered in his reading that he had two to five years to live, depending on the severity of his condition at the time.

Then his medical team offered him a potential lifeline: a one-of-a-kind experimental drug called sotatercept that traps a growth factor that is overproduced in people with PAH, potentially changing the underlying biology of the disease.

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She enrolled in a study to test the medication, which is given every three weeks by injection under the skin.

He began treatment at the dawn of the Covid-19 pandemic, in March 2020. Barry said he diligently wore an N95 mask and two layers of clothing and washed after each trip to the hospital for his shots to avoid contracting Covid along the way. .

"I had to go to the hospital because I was dying if I didn't get this medicine," he said.

"This was the only chance I had to survive."

This Tuesday, the U.S. Food and Drug Administration (FDA) approved the medication Barry has taken for four years, a drug she credits with keeping her alive and allowing her to resume many of the activities she enjoyed before her diagnosis.

Merck, the company that makes the drug, said it will be sold under the brand name Winrevair.

Barry, now 29, is a patient consultant for Merck, but said she has not received compensation for telling her story.

Winrevair was approved by the FDA on Tuesday to help treat pulmonary arterial hypertension.

(Credit: Courtesy of Merck)

Winrevair is a biologic medication designed to trap and retain proteins called activins that are overproduced in people who have PAH.

These proteins cause the walls of a person's blood vessels to continue growing and thickening over time.

As blood vessels narrow, the heart is forced to work harder to pump blood to the lungs.

This stress eventually damages the heart, causing it to fail.

Without treatment, people with PAH usually live about two to three years after their diagnosis.

Treatment with a combination of medications that dilate or relax blood vessels may improve this outlook, but they are not a cure.

"This drug has a very different mechanism of action, and it can actually rebalance some of the growth that occurs in the very small pulmonary arterioles where the disease begins," said Dr. Vallerie McLaughlin, a cardiologist at the University of Michigan who helped study sotatercept. .

"That may attack the disease from a different angle and allow for what we call reverse remodeling," he said, meaning patients could improve over time while taking the drug.

Winrevair is the first new type of drug to emerge for PAH patients in a long time, said Dr. Panagis Galiatsatos, a specialist in pulmonary and critical care medicine at Johns Hopkins Bayview Medical Center and a spokesman for the American Lung Association.

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In animal studies conducted before human trials, the drug seemed to be able to do more than just treat symptoms: It seemed like it could stop blood vessels from thickening and perhaps prolong patients' lives, but those benefits have not been realized. demonstrated in humans.

Instead, Galiatsatos said, the FDA approved the drug based on other signs of effectiveness, including whether patients could walk farther in six minutes than they could before starting the drug, a clinical measure called the six-minute walk test. minutes.

In a study that included 323 people who took sotatercept or a placebo for six months, those who received the drug were able to walk 34 meters, about half a city block, farther in six minutes than they could before starting treatment, while people in the placebo group were able to walk only about 1 meter further than they could when they started the study.

Both groups were also taking standard medications for the condition, which help relax blood vessels to improve blood flow.

At the end of the study, nine participants, or about 5%, in the sotatercept group had died or their disease had worsened, while 42 patients, or about 26%, in the placebo group had died or worsened.

The FDA was willing to move quickly to approve the drug because the disease, Galiatsatos said, is a "time bomb" for those who cannot wait for longer studies.

The study that led to the drug's approval was published in the New England Journal of Medicine last year.

"Many of us are excited about a drug like this, to be able to offer our patients something that we believe is better than their current standard therapy," said Galiatsatos, who noted that neither he nor the American Lung Association have any financial interest in the medicine.

But Galiatsatos said that as promising as the drug seems, there are still many unknowns, including whether the drug will benefit all PAH patients equally.

Although a significant number of people diagnosed with the disease are African-American, he noted, only seven people in the clinical trial, representing 2% of participants, were black.

"I tend to hold my breath" with any new medication, Galiatsatos said, adding that he's "waiting for the real-world data to come in to see how well this worked in more diverse patients."

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Like "a miracle"

In July 2019, Barry had rented an apartment for the summer in Greece, where she was eager to volunteer at a children's hospital.

But while she was abroad, the shortness of breath she had been asking doctors about for more than two years suddenly worsened.

He woke up one morning gasping for air and realized the situation was serious.

He knew that he had to return home to Boston for help.

She called her mother, and they booked a flight for her to return to the United States.

He landed at the airport and went directly to the hospital.

There, doctors performed a CT scan and found what looked like dozens of blood clots in the tiny vessels that run through the lungs.

Her heart had been severely damaged by a heart attack.

"The cardiologist said my level of heart damage wasn't found until the autopsy," Barry said.

"And all the doctors said he shouldn't have survived that flight home."

A delicate operation to remove the clots followed, but surgeons who opened his chest discovered that his blood vessels had actually collapsed in many areas, mimicking blood clots.

The blood pressure inside his heart was through the roof, and that's when they finally knew what they were dealing with: PAH, she said.

Because the disease is rare and often affects young women, delays in diagnosis are unfortunately common, Barry said.

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While doctors were warming Barry's body to get her out of surgery, her heart wouldn't start again.

She spent a week in an induced coma on a heart-pulmonary bypass machine.

Her medical team told her parents that she would probably need an immediate heart-lung transplant.

After a few days on the bypass machine, however, her body began to recover, and doctors were able to bring her out of the coma.

When he woke up, he learned of his diagnosis of pulmonary hypertension.

At 25 years old, facing a much shorter life than expected, Barry saw everything differently.

"Why am I saving if I'm not going to have a retirement?" he asked himself.

"Let me enjoy the life I have now."

But there wasn't even much life left to enjoy, he said.

Katrina Barry walking in Greece in 2022. (Credit: Courtesy of Katrina Barry)

She was strapped to oxygen tanks and always wondered how many she would need for however long she wanted to be away from home.

There was also a large new medication pump that lived in a fanny pack that she could never take off, not even to shower.

The pump delivered a continuous flow of medication through a catheter inserted directly into a large vein in his body to help keep his blood vessels open.

If something happened to the bomb, he was told he had three hours to get to the nearest hospital for help.

Being alive was nice and all, Barry thought, but what about wearing a dress?

What about going swimming?

"The list of all the things I couldn't do went on," he said.

Barry was diagnosed shortly after her surgery in October 2019, and took the opportunity to try sotatercept in the spring of 2020. The improvements weren't instantaneous, but after a few months, she went from not being able to even fold her clothes to being able to make a little more.

She was able to take short walks and paddle a little.

After a year, Barry said, he was able to stop using continuous oxygen and use it only at night.

He eventually was able to stop using it at night as well.

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After about 18 months, doctors were able to remove his central line and bulky med pump, allowing him to switch from IV versions of his vasodilator medications to pill forms.

“For me, that was so life-changing,” Barry said.

He improved so much that he was able to return to Greece in 2022 and hike a volcano.

Last month, she went skiing.

Barry still has PAH.

His lungs burn and he becomes breathless if he exerts himself too much.

"But it's gotten better over time," Barry said.

She is enrolled in a master's program in public health, and was able to wear a dress to her brother's wedding.

She has not needed the heart and lung transplant that doctors thought she might need.

"It's a miracle drug, honestly," he said of sotatercept.

"It sounds strange to say it, but it gave so many positive things and not so many negative things."

He knows that at least one person in the tight-knit HAP community experienced bleeding while taking the medication and had to stop taking it.

Bleeding and dizziness among the risks

In clinical trials, people taking sotatercept had more nosebleeds due to ruptured blood vessels in their noses, and developed more spider veins under their skin.

Experts say the drug can stimulate the growth of new blood vessels.

Other side effects included dizziness and an increase in hemoglobin, the molecule that carries oxygen in the blood.

Some people with PAH already have elevated hemoglobin levels as their bodies try to compensate for the condition, so adding the medication on top of that could be too much for them, experts said.

Sotatercept may also decrease platelets, the cells that help form clots, increasing the risk of bleeding.

"There have been some reports of gastrointestinal bleeding and even bleeding within the brain," said Dr. Kristin Highland, a lung specialist at the Cleveland Clinic who treats people with PAH.

"So bleeding is probably a real side effect."

Doctors who have tested sotatercept believe it could be a breakthrough.

But they also acknowledge that there are still many unknowns, including whether people taking sotatercept will need to continue taking the combination of drugs that help relax their blood vessels and lower their blood pressure, Highland said.

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"That's the million-dollar question," he said.

"There's certainly a lot of hope that maybe this would allow for lower doses or possibly elimination of one of those therapies. But I don't think we have the answer to that."

Merck says it plans to charge a wholesale cost of $14,000 per vial and dosing is based on the patient's weight.

The medication will come in single or double vial kits that can be used at home.

Based on data from clinical trials, Merck says it expects most patients to need a single vial every three weeks, at an annual cost of about $243,000.

The wholesale cost is not typically what patients pay.

The final cost for individuals is determined after insurance providers negotiate the price.

Still, that's significantly more than some analysts expected to see.

The nonprofit Institute for Clinical and Economic Review said the drug's benefits would be worth the cost about $35,000 a year.

Merck said in a news release that it expected the drug to be in specialty pharmacies by the end of April.

At least initially, patients have to receive their sotatercept injections in a clinic, where they can be monitored.

Over time, Barry said, he was able to start giving himself the injections at home, which has been more convenient.

Dr. Aaron Waxman, Barry's doctor and director of the pulmonary vascular disease program at Brigham and Women's Hospital in Boston, said he has seen other patients who have been as successful as her in the six years they have been studying the drug. .

Those people have also been able to get off their med pumps and switch to pills for their other therapies.

One of those pills, Opsynvi, a new combination of two commonly taken medications made by Johnson & Johnson, received FDA approval Monday to treat PAH.

"We are seeing a marked improvement that would suggest a reversal" of the disease with sotatercept, Waxman said.

"I've never seen this kind of improvement in the disease before," he said.

"I think this will change the game."

Barry says the medication has already been a beacon for people who may not have much to fight for.

"It has brought so much hope to people who have lost all hope due to this fatal disease," he said.

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