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.

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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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Peter Thiel backs ATAI’s psychedelics mission in $125 million round

Peter Thiel

Adam Jeffery | CNBC

LONDON — Billionaire tech investor Peter Thiel has backed a Berlin start-up aiming to make psychedelic drugs to treat mental health disorders in a $125 million funding round.

ATAI Life Sciences, which describes itself as a drug development platform, was set up to acquire, incubate and develop psychedelics and other drugs that can be used to treat depression, anxiety, addiction and other mental health conditions. The company — founded in 2018 by entrepreneurs Christian Angermayer, Florian Brand, Lars Wilde and Srinivas Rao — announced the funding on Monday.

“ATAI’s great virtue is to take mental illness as seriously as we should have been taking all illness all along,” Thiel, who co-founded Palantir and PayPal, said in a statement shared with CNBC. “The company’s most valuable asset is its sense of urgency.”

Thiel made a 10 million euro ($12 million) investment in ATAI through his venture firm, Thiel Capital, while the rest of the series C funding round came from Apeiron Investment Group (Angermayer’s family office), Catalio Capital Management, Future Ventures, Galaxy Investment Partners, Falcon Edge Capital, and Pura Vida.

Total investment in the company now stands at over $210 million.

ATAI, which has roughly 35 staff in offices across Berlin, New York and San Diego, is currently partnered with around 10 drug development companies. In exchange for a majority stake in the drugs they’re developing, ATAI helps the scientists to raise money, work with the regulators, and conduct clinical trials.

None of ATAI’s drugs have been formally approved by regulators to date.

There is growing interest in certain psychedelics after recent clinical studies suggested that some could help patients with a number of mental illnesses, either in combination with traditional solutions or in cases where nothing else has worked.

“The current treatments (for mental health issues) which are out there are definitely not sufficient,” company co-founder Angermayer told CNBC via Zoom ahead of the announcement. “I don’t want to say they don’t work as all because some people are helped by them but they’re not sufficient.”

Almost a billion people suffer from mental health problems worldwide, according to the World Health Organization. Of the 322 million people globally that have depression, roughly a third are treatment-resistant, according to fellow company co-founder Brand.

ATAI said it will use the new funding to pay for the clinical development of drugs that ATAI has already backed. This includes ar-ketamine, which is being developed at Perception Neuroscience for treatment-resistant depression, and ibogaine, which is being developed by DemeRx to treat opioid addiction. The funding will also be used to identify new drugs and support their development.

Earlier this year, the U.S. Food and Drug Administration approved Johnson & Johnson’s ketamine-like nasal spray for depression and there are a number of other start-ups trying to get psychedelic drugs to market including MindMed and Beckley Psytech.

Psychedelic trips

Angermayer said that people “meet themselves” when they have a psychedelic trip. “Since we’re born, society imposes on us how we should

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Another Study Finds COVID Usually Mild in Kids | Health News

By Robert Preidt, HealthDay Reporter

(HealthDay)

MONDAY, Nov. 23, 2020 (HealthDay News) — COVID-19 is mild is most children, a new study says, but certain children have a higher risk of severe illness.

Of more than 135,000 children tested for the new coronavirus (SARS-CoV-2) at seven children’s hospitals in the United States up to September, 4% were found to be infected.

Those most likely to test positive included children from ethnic minorities, teens, those with history of public insurance, and those with certain underlying medical conditions.

Similar risk factors were noted in the 6.7% of infected children who developed severe COVID-19 and were hospitalized with respiratory, cardiovascular or COVID-19-specific symptoms. Of those, 27.6% required intensive care and 9.2% required mechanical ventilation.

Eight of the children who tested positive died, a fatality rate of 0.15%. The risk of death was strongly associated with having numerous complex preexisting medical conditions.

Children with a progressive long-term medical condition were nearly six times more likely to develop severe illness, and the risk was 1.5 to three times higher among Black children, those younger than 1 and older than 12, and those with a history of public insurance.

The researchers also found that Black, Hispanic and Asian children were less likely to get tested, but were two to four times more likely to test positive than white children.

“While the overall risk is low in this group of children, we see significant disparities in those who are testing positive and developing severe disease, which follows what we see in adults,” said study author Hanieh Razzaghi, assistant director of the PEDSnet Data Coordinating Center at Children’s Hospital of Philadelphia.

PEDSnet is national pediatric health network that includes more than 7 million patients.

“Future studies need to evaluate to what extent the higher rate of positive test results reflects different testing strategies across patient groups, as well as different social determinants of risk, like exposure to air pollution and likelihood of family continuing to work at in-person essential jobs,” Razzaghi said in a hospital news release.

“Similarly, it is important to understand differences in the biology of infection that cause different rates of symptoms between patients, so we can best protect children at higher risk,” she added.

The study was published Nov. 23 in the journal JAMA Pediatrics.

SOURCE: Children’s Hospital of Philadelphia, news release, Nov. 23, 2020

Copyright © 2020 HealthDay. All rights reserved.

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Good Sleep Habits Tied to Lower Risk of Heart Failure

A combination of healthy sleep habits may help reduce the risk for heart failure, new research suggests.

Scientists studied 408,802 generally healthy people aged 27 to 73 between 2006 and 2010, collecting information on their sleep habits. Each person got a zero-to-five “healthy sleep score,” based on five healthy sleep practices: being a “morning person”; sleeping seven to eight hours a night; rarely or never snoring; rarely having insomnia; and rarely being excessively sleepy during the day.

Over an average follow-up of 10 years, there were 5,221 cases of heart failure. Compared with people who scored zero or one, those who scored two had a 15 percent reduced risk for heart failure; those who scored three had a 28 percent reduced risk; and those who scored four a 38 percent risk reduction. Those who scored a perfect five had a 42 percent lower risk of heart failure compared with those who scored zero or one.

The study, in the journal Circulation, controlled for smoking, alcohol intake, physical activity, diabetes, hypertension and other variables. It is an observational study, however, so it does not prove causality.

“We should consider all of these sleep behaviors together rather than treating them as separate phenomena,” said the senior author Dr. Lu Qi, a professor of epidemiology at Tulane University. “People regulate their sleep as a whole, not as separate events.”

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Oxford Vaccine Demonstrates 70% Protection Against SARS-CoV-2

The Sars-CoV-2 vaccine developed by the University of Oxford demonstrated average efficacy of 70.4%, according to results of clinical trials.

The interim analysis of phase 2/3 trials in the UK and Brazil found efficacy of 62% following two full doses given at least one month apart.

However, vaccine efficacy was 90% when ChAdOx1 nCoV-19, also known as AZD1222, was given to a subset of participants as a half dose, followed by a full dose at least one month later.

No hospitalisations or severe cases of the disease were reported in participants who received the vaccine, Oxford’s partner AstraZeneca said in a press release this morning.

The data also suggested a half dose and full dose regime could help to prevent transmission of the virus, according to a press release from the University of Oxford.

Sarah Gilbert, professor of vaccinology at Oxford, said: “The announcement today takes us another step closer to the time when we can use vaccines to bring an end to the devastation caused by SARS-CoV-2.”

The announcement follows promising interim data from Pfizer-BioNTech and Moderna, which recently demonstrated protection of around 95% against developing COVID-19 with their messenger RNA (mRNA) vaccines

However, the Oxford vaccine is cheaper to produce, and unlike the other vaccines that require a demanding cold chain storage, can be kept at standard refrigerator temperatures of between 2C and 8C.

‘Exciting’ News

Prof Andrew Pollard, director of the Oxford Vaccine Group, said: “These findings show that we have an effective vaccine that will save many lives.

Excitingly, we’ve found that one of our dosing regimens may be around 90% effective and if this dosing regime is used, more people could be vaccinated with planned vaccine supply.”

Asked why giving a half dose followed by a full dose appeared to be more effective, Prof Pollard told a briefing hosted by the Science Media Centre: “We think that by giving a smaller first dose that we’re priming the immune system differently, we’re setting it up better to respond.”

He added: “What we don’t know at this moment is whether that difference is in the quality or the quantity of the immune response, and that’s something we are doing to be digging into.”

Pascal Soriot, AstraZeneca’s chief executive Officer, said using a halved first dose and a standard second dose would mean “we can vaccinate more people, faster”.

AstraZeneca Prepares to Seek Regulatory Approval

The company said it would immediately prepare regulatory submission of the data to authorities around the world.

Trial results will be submitted to a scientific journal for peer review.

The pooled analysis included data from the COV002 Phase 2/3 trial in the UK and COV003 phase 3 trial in Brazil, and involved more than 23,000 participants.

Dr Zania Stamataki, viral immunologist at the University of Birmingham, commented: ” People will be tempted to compare efficacy between the different vaccines but we must remember that these are early data designed to achieve approval. Efficacy is not the same as effectiveness and the early numbers

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Bulgaria Plans Lockdown to Contain Coronavirus Infection Surge | World News

SOFIA (Reuters) – Bulgaria plans to close schools, restaurants and shops and ban all sports events, private celebrations and excursions as it struggles to contain a coronavirus case surge.

The Balkan country’s health minister Kostadin Angelov said on Monday that the measures, to be debated by the centre-right government on Wednesday, were aimed at preventing a struggling health system from being overwhelmed.

New coronavirus cases have doubled in the past week to 23,569, Bulgarian health ministry data showed, bringing the total number to 121,820 in the country of 7 million.

Some 6,350 people are in hospitals, 1,000 more than a week ago, and more than 400 are in intensive care.

Hospitals are strained, with many short-staffed due to rising infections among medics, while ambulances have been searching for coronavirus beds in major cities.

Bulgaria’s COVID-19 fatalities per 100,000 people are the third highest in the European Union in the past 14 days, data showed. In total, 2,880 people have died from COVID-19.

“No matter how prepared, no (health) system can withstand such a pressure,” Angelov told reporters.

“We cannot afford to lose the lives of young people, of old people, of doctors and of teachers,” he added.

Under the plan, which if approved will be enforced from Nov. 27, schools and universities will switch to online studies, while kindergartens and nurseries will be closed.

Sports and cultural events will be banned, including seminars, conferences and private celebrations. All restaurants, bars and cafes will be shut, as well as all shops except for pharmacies, food stores and banks.

Tourist trips both at home and abroad will also be banned.

Earlier, Prime Minister Boyko Borissov said the measures should be balanced to keep the small and open economy going and Angelov said he hoped the measures could allow some easing for the Christmas holidays.

(Reporting by Tsvetelia Tsolova; Editing by Alexander Smith)

Copyright 2020 Thomson Reuters.

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Here’s how the U.S. government plans to distribute the first Covid-19 vaccines.

In the wake of a steady stream of positive results indicating the effectiveness of several coronavirus vaccines, the official in charge of the federal coronavirus vaccine program explained on Sunday news shows how the vaccines might be distributed to Americans as early as next month.

Dr. Moncef Slaoui, head of the administration’s Operation Warp Speed, said that within 24 hours after the Food and Drug Administration approves a vaccine, doses will be shipped to states to be distributed. “Within 48 hours from approval,” the first people would likely receive injections, Dr. Slaoui said on ABC’s “This Week With George Stephanopoulos.”

Two companies, Pfizer and Moderna, announced this month that their vaccines were about 95 percent effective, and Pfizer formally submitted an application to the F.D.A. for emergency approval. Regulators at the agency will spend about three weeks reviewing the application. On Dec. 10, an outside advisory board on vaccines will meet to discuss the application, and the agency is expected to make a decision shortly after that meeting. Moderna is expected to submit its own application soon.

Even if the first vaccine is authorized in mid-December, officials and company representatives have estimated that there will only be enough doses available to treat about 22.5 million Americans by January. Each vaccine requires two doses, separated by several weeks.

Dr. Slaoui said vaccines would be shipped to states, proportioned according to their population, and that states would decide how and where to distribute the doses. He said that likely within a day after a vaccine receives F.D.A. authorization, a committee at the Centers for Disease Control and Prevention would issue recommendations for which groups should be first to receive a vaccine.

High-priority groups are likely to include frontline medical workers and residents of nursing homes. Dr. Scott Gottlieb, a former F.D.A. commissioner, said on the CBS show “Face the Nation” that those groups would likely be followed by other older adults and then expanded to younger adults in the spring. Both he and Dr. Slaoui estimated that tens of millions of adults could be vaccinated by sometime in May.

Immunizations for children would follow. Dr. Slaoui said on the CNN show “State of the Union” that the youngest participants in the clinical trials so far have been 12 to 14 years old and that approval for younger children and toddlers would likely not occur until late in 2021, after clinical trials for those age groups are conducted.

On “Face the Nation,” Larry Merlo, the chief executive of CVS Health, said that pharmacists and other medical staff from CVS plan to immunize residents of more than 25,000 long-term care facilities, beginning about 48 hours after a vaccine is approved. He said that for several years, CVS has been going to nursing homes to administer the seasonal flu vaccine, so “we have the systems, we have the processes, and we have built the logistics directly for the Covid vaccine.”

Mr. Merlo, whose company runs 10,000 pharmacies across the country, also said as the supply

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A photographer grapples with the legacy of Alzheimer’s disease

Jalal Shamsazaran’s father was first diagnosed with Alzheimer’s disease more than five years ago. “I found out that it’s inherited in our family,” the Iranian photographer said. “My grandfather had Alzheimer’s, my father had Alzheimer’s, and my aunt also has Alzheimer’s.” So when Shamsazaran decided to photograph his father’s struggles with the degenerative disease, he did it with the expectation that he might be documenting the changes in behavior and personality that he may, one day, experience himself. “Maybe it can be said that I am somehow photographing myself,” he said.

Shamsazaran’s images, which chronicled the last years of his father’s life before his death in early 2020, have been selected as this year’s recipient of the Bob and Diane Fund, a grant designed to promote work “that increases the understanding of the disease and inspires research to end it,” said Gina Martin, the fund’s creator.

“Jalal captures his father’s decline into dementia until his death with such tenderness and respect,” she added. “His beautiful black-and-white images with his distinct style and voice shows the power of visual storytelling.”

For Shamsazaran, the grant is another opportunity to raise awareness about the disease, which affects more than 50 million people in the world, and 750,000 in Iran alone. “Any father with Alzheimer’s can be my father, and I am the child of all of them,” Shamsazaran said.


Jan. 17, 2020: “The slim hands of my father rest on blanket as he sleeps. These hands are part of our country’s history to me and they make me to remember the hardships he went through for his family.” (Jalal Shamsazaran/NVPImages)
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‘Half-measure’ Virus Vaccine Intrigues Experts

Evidence suggesting an initial half dose of the vaccine being developed by drugs firm AstraZeneca and the University of Oxford is more effective than a full dose is counterintuitive, and even took the researchers by surprise.

Why would less be better than more when it comes to triggering an immune response?

Andrew Pollard, the director of the Oxford Vaccine Group, described the findings from the Phase 3 clinical trial as “intriguing”.

They showed that the vaccine had an efficacy of 62 percent among the people given two full doses a month apart.

But this rose to 90 percent for another group who received a half-dose first and then a full dose after a month.

“I think all of us expected that the two high doses would be the best response,” said Pollard, who noted researchers had only seen the details of the results over the weekend and would now start digging into the data.

“We think that by giving a smaller first dose, that we’re priming the immune system differently. We’re setting it up better to respond,” he told a press briefing.

Sarah Gilbert, professor at Oxford’s Nuffield Department of Medicine, said the better result with a smaller initial dose could be because this better “mimics what happens in a real infection”.

Essentially a vaccine uses a safe method to trick the immune system into believing it is dealing with a dangerous infection, triggering an immune response and an immune memory that can activate if the body comes across the real pathogen.

“It could be that by giving a small amount of the vaccine to start with and following up with a big amount, that’s a better way of kicking the immune system into action and giving us the strongest immune response,” Gilbert told reporters.

Clinical trials suggested that an initial half-dose was better than a full one Clinical trials suggested that an initial half-dose was better than a full one Photo: AFP / JUAN MABROMATA

The Astra/Oxford vaccine employs what is known as a “viral vector”, using engineered viruses to deliver genetic cargo into cells, giving them instructions on how to fight SARS-CoV-2.

The strategy uses the transporting virus as a “Trojan Horse”, said Colin Butter, Associate Professor at the University of Lincoln.

It is “complex and usually achieved experimentally: a luxury not available in the present situation”.

The technology itself may be the reason why an initial half-dose could work better, according to several scientists commenting on the results, with the immune system acting against the virus being used as a delivery vehicle.

“It may seem confusing that a higher initial dose gives a less favourable response, but this may just be due to a residual response in some patients to the disabled ‘vehicle’,” a snippet of chimpanzee virus used to deliver the vaccine “payload”, said Stephen Griffin, Associate Professor in the School of Medicine, University of Leeds.

But he said this could be “easily fixed” by using the adjusted dose.

Pollard said researchers would be looking to find out if the issue was the quantity or quality of the immune

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‘Hidden’ Prostate Cancer on Biopsy Usually Means Good Outcome: Study | Health News

By Robert Preidt, HealthDay Reporter

(HealthDay)

MONDAY, Nov. 23, 2020 (HealthDay News) — Negative biopsies among early-stage prostate cancer patients who’ve chosen active surveillance are associated with a low risk of disease progression, but they aren’t a sign that their cancer has completely vanished, a new study indicates.

Active surveillance refers to close monitoring for signs of cancer progression — what’s often called “watchful waiting.” Patients sometimes get regular prostate-specific antigen (PSA) testing, prostate exams, imaging and repeat biopsies.

The objective of active surveillance is to avoid or delay treatment and its side effects without putting patients at risk of cancer progression and death.

Sometimes, active surveillance patients have negative biopsies that show no evidence of prostate cancer. While some of these patients may believe that their cancer has “vanished,” they most likely have low-volume or limited, hidden areas of prostate cancer that weren’t detected in the biopsy sample, according to the authors of the study published recently in The Journal of Urology.

“While a negative biopsy is good news, the long-term implications associated with such ‘hidden’ cancers remain unclear,” said study author Dr. Carissa Chu, from the University of California, San Francisco.

For the study, Chu and colleagues analyzed data from 514 men undergoing active surveillance for early-stage prostate cancer between 2000 and 2019. All of them had at least three surveillance biopsies after their initial prostate cancer diagnosis. Median follow-up time was nearly 10 years.

Of those patients, 37% had at least one negative biopsy, including 15% with consecutive negative biopsies, according to the report.

Men with negative biopsies had more favorable disease characteristics, including low PSA density and fewer samples with cancer at the initial prostate biopsy. Negative biopsies were also associated with good long-term outcomes, the researchers said.

After 10 years, rates of survival with no need for prostate cancer treatment (such as surgery or radiation) were 84% for men with consecutive negative biopsies, 74% for those with one negative biopsy and 66% for those with no negative biopsies.

After adjusting for other factors, the researchers concluded that men with one or more negative biopsies were much less likely to have cancer detected on a later biopsy.

“For men undergoing active surveillance, negative biopsies indicate low-volume disease and lower rates of disease progression,” Chu said in a journal news release. “These ‘hidden’ cancers have excellent long-term outcomes and remain ideal for continued active surveillance.”

SOURCE: The Journal of Urology, news release, Nov. 17, 2020

Copyright © 2020 HealthDay. All rights reserved.

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