FDA Approves A Drug For A Rare Disease That Causes Children To Age Quickly : Shots

Sam Berns and Audrey Gordon, executive director of The Progeria Research Foundation and Berns’s aunt, attend The New York Premiere Of HBO’s “Life According To Sam” on October 8, 2013 in New York City.

Thos Robinson/Getty Images for HBO


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Thos Robinson/Getty Images for HBO

Sam Berns and Audrey Gordon, executive director of The Progeria Research Foundation and Berns’s aunt, attend The New York Premiere Of HBO’s “Life According To Sam” on October 8, 2013 in New York City.

Thos Robinson/Getty Images for HBO

The Food and Drug Administration has approved a drug that extends the lives of children with an extremely rare genetic disorder that causes them to grow old before they grow up.

The disorder, progeria, ages cells rapidly and prematurely. As a result, affected children remain small and begin to look frail and old by the time they reach school age. Most die of heart disease in their early teens.

But the drug, Zokinvy, slows down the decline.

“Zokinvy is a treatment. It’s not a cure,” says Dr. Leslie Gordon, an assistant professor of pediatrics at Brown University and medical director of The Progeria Research Foundation. “But what we can say is that, so far, we know that it increases the average lifespan by about two-and-a-half years.”

Gordon and her family played a crucial role in making Zokinvy possible.

Their goal was to find a treatment for Gordon’s son, Sam Berns, who was diagnosed with progeria in 1998 and died in 2014.

“We started this for Sam, and Sam is always here, always,” Gordon says.

Sam Berns gave a TEDx talk about living with progeria.

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As a teenager, Sam became the public face of progeria. He spoke to NPR and other media about his condition, was the subject of an HBO documentary, and gave a TEDx talk about how to live a happy life that has been viewed more than 40 million times.

Sam was one of the first children with progeria to receive Zokinvy and Gordon says the drug seemed to extend her son’s life.

“I think Sam felt that way,” she says. “And looking at the data, and as parents, we felt that way too.”

Since Sam’s death, his family has continued to operate The Progeria Research Foundation and to search for a cure.

“We make a really great team,” says Audrey Gordon, who is Sam’s aunt and the foundation’s president. “Leslie [is] in charge of the science aspect, I’m in charge of the fundraising and her husband, Scott, is the chairman of the board.”

The approach reflects a philosophy embraced by Sam, who was a big sports fan, Audrey Gordon says.

“Whenever someone asked, who’s your favorite player on your beloved Patriots or your Boston Bruins, he always refused because he felt like it wasn’t any one person who made the team,” she says “It was the team as a whole.”

Team Sam published the first evidence that Zokinvy worked in 2012. But getting FDA approval required a whole new

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Coronavirus spreads quickly among household members

If one person in a household has Covid-19, there’s a good chance it’ll spread to others — and quickly.

A report from the Centers for Disease Control and Prevention, published Friday, took a close look at how the virus spread throughout people’s homes.

Full coverage of the coronavirus outbreak

The study included 101 households in Nashville, Tennessee, and Marshfield, Wisconsin. All households had one index patient — a confirmed case of Covid-19. At the time the index patient first reported symptoms, no one else in their household reported any.

The 101 index patients (each in their own household) lived with a total of 191 household contacts, 102 of whom — or 53 percent — went on to test positive for Covid-19, according to the report.

What’s more, the spread occurred “rapidly,” the authors wrote: “Approximately 75 percent of infections [were] identified within 5 days of the index patient’s illness onset.”

The report noted that the age of the index patient didn’t matter — adults, children and teens all spread the virus to others in their households.

Family clusters were reported to be a source of spread early on in the pandemic. The first instance of person-to-person spread in the country was between a husband and his wife in Chicago. One of the first coronavirus cases in New York, a man in his 50s, spread the virus to his wife and two of his children.

As early as February, the World Health Organization noted that most cases in China occurred in family clusters.

Isolate immediately

The new report highlights the importance of isolating family members as soon as possible if they suspect they have Covid-19.

“Isolation should begin before seeking testing and before test results become available because delaying isolation until confirmation of infection could miss an opportunity to reduce transmission to others,” the authors wrote. At the same time, the authors added, everyone in the home should start wearing a mask.

If feasible, the individual should isolate in a separate bedroom and use a separate bathroom.

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The report noted that 69 percent of the index patients, however, reported spending at least four hours in the same room with other household members the day before getting sick, and 40 percent said they spent the same amount of time with others the day after getting sick.

Similarly, 40 percent of the index patients shared a bedroom before getting sick, and 30 percent shared a bedroom after getting sick.

The report also underscored the importance of quarantining. Less than one half of the household contacts who tested positive for the coronavirus had symptoms when they were tested, and many reported no symptoms over the next seven days. These individuals should quarantine to avoid spreading the infection to others.

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General plans campaign to deliver coronavirus vaccine quickly

The Pentagon general spearheading President Trump’s drive to ensure a coronavirus vaccine gets to the American people in record time said Tuesday that Operation Warp Speed can only work if there is full buy-in from the public when the vaccine becomes available.

Army Gen. Gustave Perna, a military logistics specialist who got a shoutout from Mr. Trump himself at last week’s presidential debate, described his mission at a session organized by the conservative Heritage Foundation was “our world’s best scientists and doctors working beside the world’s best military with the support of American industry and academia.”

“It’s the collective effort that will ensure we’re successful. I’m proud to be a part of it,” Gen. Perna said.

Operation Warp Speed is pushing ahead in the face of an at times angry political debate over the government’s handling of the virus crisis and whether the urgency of the mission will affect the safety of the process.

Thousands of volunteers will be needed after pharmaceutical giants AstraZeneca and Johnson & Johnson announced this week they were restarting U.S. clinical trials on their coronavirus vaccine candidates following a temporary stand-down over possible safety issues. Officials at Operation Warp Speed — a public-private partnership to develop therapeutics and ultimately a vaccine for COVID-19 — said locating a large number of volunteers for the trials is absolutely crucial.

In the past, about 5,000 to 8,000 people agreed to take part in clinical trials, Dr. Matthew Hepburn, head of program’s vaccine development, told the Heritage gathering.

“Our standard has been 30,000 volunteers for our clinical trials. We want to gather as much safety information as we possibly can,” Dr. Hepburn said Tuesday. “We want to know if [the vaccine candidates] work. The more people you enroll, the sooner you’re going to be able to evaluate the effectiveness.”

Dr. Hepburn said biotech firm Moderna has already completed its Phase 3 clinical trials with more than 30,000 volunteers enrolled in the testing. The New York-based pharmaceutical company Pfizer has signed up more than 40,000 people to take part in currently ongoing testing.

“That shows me that over 60,000 Americans have decided to say, ‘I will volunteer. I don’t know if I’m going to receive the vaccine or if I receive a placebo. But I’m going to do my part,’” Dr. Hepburn said. “We’re very proud of the spirit of volunteerism that we see in America.”

Medical researchers will be keeping in contact with those who received the initial COVID-19 vaccine candidates.

“The people that are vaccinated in the clinical trial now, our intention is to follow them for up to two years so we can get a better idea on that critically important question of duration of protection,” Dr. Hepburn said.

Past campaigns against viruses such as smallpox, polio and the measles made it possible for researchers to move more quickly to the coronavirus finish line, officials said.

“These efforts have gone on for decades. We stand on the shoulder of giants,” Dr. Hepburn said. “There was massive progress

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