Tag: treatment

Categories
Health

Dr. Ounceshas ties to hydroxychloroquine corporations as he backs Covid therapy

Dr. Oz has ties to hydroxychloroquine companies as he backs Covid treatment

Republican Senate candidate from Pennsylvania, Dr. Mehmet Oz, has financial ties to at least two pharmaceutical companies that supply hydroxychloroquine, an antimalarial drug he circulated as a possible treatment for Covid-19.

Oz, a physician and veteran television host who is up against Democrat John Fetterman in the race for the Pennsylvania Senate seat, owns with his wife at least $615,000 in Thermo Fisher Scientific stock, according to its financial disclosure. Thermo Fisher Scientific’s website lists hydroxychloroquine sulfate as one of the available products. It’s unclear when Oz and his wife bought the stock or if they owned it, as Oz promoted hydroxychloroquine as a Covid treatment early in the pandemic.

Oz and his wife also own between $15,001 and $50,000 in McKesson Corporation stock, according to the disclosure. According to the FDA, the company labels and sells hydroxychloroquine sulfate. It’s also unclear when they bought McKesson stock.

Hydroxychloroquine sulfate is the anti-malarial drug commonly known as hydroxychloroquine, according to the Food and Drug Administration. Doctors across the country, helped in part by support from former President Donald Trump and conservative media figures, have been offering the drug to patients as a Covid treatment, despite its questionable effectiveness against the virus.

Oz’s financial ties to a manufacturer and distributor of the drug, and his promotion of it as a potential Covid treatment, raise questions about what he would benefit from its wider use during the pandemic. If he wins the Senate election, he could also face conflicts of interest as Congress grapples with a still-evolving coronavirus pandemic.

In a statement responding to CNBC questions about Oz’s ties with companies that manufacture or distribute hydroxychloroquine, including when he and his wife bought shares in Thermo Fisher Scientific, Oz campaign spokeswoman Brittany Yanick, does not affect the financial interests of the candidate.

“At the beginning of the pandemic, Dr. Mehmet Oz with healthcare professionals worldwide who are considering hydroxychloroquine and azithromycin as viable treatment options for critically ill COVID patients. He offered to fund the clinical trial at Columbia University,” she said.

The FDA has approved hydroxychloroquine to fight malaria but warned that it “has not been shown to be safe or effective for treating or preventing COVID-19.”

Oz took bold steps early in the pandemic to promote its use as a treatment. He urged Trump administration officials in 2020 to support a study he wanted to fund at Columbia University Medical Center on the effect of hydroxychloroquine on Covid-19 patients, according to emails obtained by the select subcommittee of the House of Representatives on the coronavirus crisis have been received and published.

Oz also has ties to a third company, which it says has divested hydroxychloroquine from its US portfolio.

Sanofi, which is headquartered in France and previously manufactured hydroxychloroquine, supported Oz’s nonprofit HealthCorps for years, according to the group’s annual disclosure reports. Between 2009 and 2018, Sanofi was listed as either a sponsor or donor in kind to the Oz-funded group, which owns aims to help teenagers with their health and well-being. In 2013, Sanofi is listed as one of the group’s “School Sponsors”. HealthCorps’ website states that a school sponsor must donate $100,000 to qualify.

Sanofi announced in April 2020 that it would donate 100 million doses of hydroxychloroquine to 50 countries around the world as studies evaluated the drug’s effectiveness in treating Covid-19.

A spokesman for Sanofi told CNBC that the company was not involved in Oz’s comments about Covid-19 or hydroxychloroquine. He explained that Sanofi divested hydroxychloroquine from its US portfolio in 2013 and was investigating the drug’s use as a potential way to fight the virus early in the Covid pandemic. After it was deemed ineffective against Covid-19, the company’s work on it was suspended.

The spokesperson also explained that the company’s last financial contribution to HealthCorps was in 2011. The company representative later corrected himself in a follow-up email to CNBC after the publication of this story, saying that 2013 was actually the last year that Sanofi made a financial donation to HealthCorps.

Oz’s ties to companies that would benefit from wider use of hydroxychloroquine could pose problems for the Republican if he wins the Senate seat. Kedric Payne, an ethics attorney at the Campaign Legal Center, told CNBC in an email that Oz could choose to walk away from the companies if he defeated Fetterman in November.

“He could have a rude awakening if elected because ethics rules could bar him from the job. Senators cannot use their positions to promote goods or services that benefit them financially,” Payne said. “Oz could voluntarily divest the shares if elected or stop promoting anything tied to his shares.”

A spokesman for Thermo Fisher Scientific declined to comment. A McKesson representative did not respond to a request for comment prior to publication.

Since launching his campaign late last year, Oz has downplayed warnings from the FDA and other experts against the use of hydroxychloroquine as a Covid treatment. He suggested political animus against Trump endorsing the drug as a treatment and Oz in the Senate election, motivating criticism of the drug as a way to combat Covid.

“Well let me say this real quick, I really don’t know if it works or not, we haven’t been able to prove to this day if it works [hydroxychloroquine] works or not, which is a shame because we should have known by now whether a cheap 70-year-old drug used by a billion people works or not,” Oz said at a campaign event earlier this year. “But we don’t know. t which is a problem in itself. However, I mentioned it and then President Trump mentioned it in a press conference and suddenly the whole world hated hydroxychloroquine without testing it, without knowing it.”

Before launching his campaign, Oz championed hydroxychloroquine more explicitly. During an interview with Fox News in March 2020 at the height of the pandemic, Oz said that “hydroxychloroquine has a role” in fighting the virus. An on-screen graphic while Oz was being interviewed called the anti-malarial drug “promising” as a treatment option for Covid-19.

Oz also sought White House help to get the hydroxychloroquine trial going, which he wanted to fund at Columbia, where he was once vice chairman of the department of surgery. He has since said the study never got off the ground.

The Pennsylvania nominee’s communications with White House officials were released last month by the House’s select subcommittee on the coronavirus crisis. In an email dated March 2020 Deborah Birx, former Trump White House coronavirus response coordinator, told Oz he would recruit patients and pay for the hydroxychloroquine trial himself.

Also in March 2020, Oz Trump’s son-in-law and adviser Jared Kushner emailed that “we must make the completion of this study a national priority and insist on immediate enrollment,” according to correspondence obtained by the House Committee and has published. Kushner replied to Oz the same day, “What do you recommend to speed it up?”

The New York Post reports that Oz spent $8,800 on hydroxychloroquine tablets for the study at the time and offered to spend $250,000.

Oz, during his campaign for the Pennsylvania Senate seat, accused then-New York Governor Andrew Cuomo of stopping the study after effectively banning the anti-malarial drug as a Covid treatment.

Oz’s financial ties could pose a bigger problem for him if he wins the Pennsylvania race, one of a few contests to decide which party will control the Senate next year. A Real Clear Politics poll average shows Fetterman leading Oz by almost 7 percentage points.

Share ownership in Congress will come under increased scrutiny. Some lawmakers in Congress have proposed a ban on individual stock deals that would require lawmakers to invest assets in a blind trust or to divest them outright.

Business Insider has identified at least 71 lawmakers who have violated the Stop Trading on Congressional Knowledge Act, or STOCK Act. The law aims to prevent members of Congress from trading stocks using inside information gained from their work as legislators.

By and large, however, members of Congress had little impact on lucrative stock deals.

Categories
Health

Florida, Texas open Covid antibody remedy facilities as delta surge overwhelms hospitals

Florida, Texas open Covid antibody treatment centers as delta surge overwhelms hospitals

Florida Governor Ron DeSantis holds a press conference to announce the opening of a monoclonal antibody treatment center to help recover COVID-19 patients at Camping World Stadium in Orlando.

Paul Hennessy | LightRakete | Getty Images

Florida and Texas are opening free monoclonal antibody centers to treat a surge in Covid-19 patients in both states in the hopes that early intervention will help keep people out of hospitals and save more lives – even if they do The governors of both states are fighting local officials with mask and vaccination regulations.

Texas is building nine antibody infusion centers, Governor Greg Abbott announced on Friday, while Florida opened its fifth site on Wednesday. With the delta variant spike, coronavirus patients were occupied by more than 46% of Texas intensive care beds and more than half of Florida intensive care units as of Thursday, compared with 27% nationwide, according to the Department of Health and Social Affairs.

“What takes you to the hospital is the inflammation. People get inflammation in their lungs,” said Dr. Arturo Casadevall, Chair of Molecular Microbiology and Immunology at the Bloomberg School of Public Health at Johns Hopkins University, told CNBC in an interview. “So what these antibodies do is, if you give them to a patient early, they neutralize the virus.”

Abbott has firsthand experience of the treatment. His office announced Tuesday that he was receiving monoclonal antibody treatment from Regeneron after testing positive for Covid despite being fully vaccinated.

Although monoclonal antibodies like Regeneron and GlaxoSmithKline treatments are one of the few proven ways to fight the virus and reduce hospital stays, they were rarely used during the pandemic because they are awkward to administer. Monoclonal antibody treatments must be injected directly into the vein via an IV infusion, which requires time and dedicated medical staff, often using the same equipment reserved for chemotherapy patients.

The Food and Drug Administration issued emergency clearances to Regeneron’s treatment in November, saying it reduced hospital admissions for Covid “in patients at high risk for disease progression within 28 days of treatment.” GlaxoSmithKline just received emergency approval for its treatment with Vir Biotechnology in May and said it has reduced hospital stays and deaths in high-risk patients by about 85%.

The FDA approved both companies’ treatments for use in patients 12 years of age and older.

“Many patients who are examined by their doctors and referred for a monoclonal antibody infusion are less likely to be hospitalized,” said Teresa Farfan, spokeswoman for the Texas Division of Emergency Management, in an email to CNBC . “This will help ensure that resources are available in the hospitals to treat those with the most severe cases of the virus.”

Treatment centers couldn’t get there early enough as the Delta variant is driving cases to record highs in Florida. The state, which publishes its cases once a week on Fridays, last reported a record seven-day average of nearly 21,700 new infections, 12.6% more than a week ago, according to a CNBC analysis of data compiled by Hopkins.

Texas has been moving closer and closer to its record highs of more than 23,000 average cases per day in January in recent weeks, reporting a seven-day average of just over 15,400 new infections on Thursday, up from a seven-day average of around 3,000 a last month.

“Let me be very clear on this – both monoclonal and vaccines save lives,” said Christina Pushaw, spokeswoman for Florida Governor Ron DeSantis, in an email to CNBC. “They certainly aren’t mutually exclusive.”

More than 34% of the 50,706 registered inpatients in Florida have the coronavirus, as does over a quarter of the 51,337 registered inpatients in Texas, as measured Thursday. Abbott called 2,500 medical workers from across the country last week to help fight the virus and urged hospitals to build capacity by postponing election procedures.

A box and vial of the Regeneron monoclonal antibody can be seen at a new COVID-19 treatment site opened by Florida Governor Ron DeSantis at Camping World Stadium in Orlando following a press conference.

Paul Hennessy | LightRakete | Getty Images

While both Abbott and DeSantis have urged residents to get vaccinated, they still strictly oppose mask or vaccination regulations, saying it violates personal freedoms. Republican governors have banned local governments and school districts from requiring face-covering. Abbott has threatened $ 1,000 fines for those who fail to comply, and DeSantis said it will withhold pay from educators who prescribe masks.

With many children returning to classrooms this fall, local officials are pushing back. Several school districts in both states have defied their governors’ orders and restored their mask mandates, with appeals courts in Dallas and San Antonio issuing injunctions last week to circumvent the ban.

The Texas Supreme Court on Sunday blocked the injunctions, sided with Abbott and prevented school districts from issuing their own guidelines. Local officials say they plan to continue fighting Abbott in court, and President Joe Biden on Wednesday directed the education secretary to intervene “to protect our children.”

“This includes using all of its regulators and, if necessary, taking legal action against governors who try to block and intimidate local school officials and educators,” said Biden.

Dr. Bruce Farber, chief of infectious diseases at Northwell Health in New York, said states that don’t allow schools to prescribe masks are at great risk this fall.

“These states are gambling as I see it,” he said in an interview. “By not allowing masking and preventing masking and leaving it to the parents, (they) are really playing with fire.”

Categories
Politics

Rand Paul’s spouse purchased shares in Covid therapy maker Gilead as virus unfold

Rand Paul's wife bought shares in Covid treatment maker Gilead as virus spread

U.S. Sen. Rand Paul (R-KY) listens to Dr. Anthony Fauci, director of the National Institute of Allergy and Infectious Diseases, during a Senate Health, Education, Labor and Pensions Committee hearing to discuss the on-going federal response to COVID-19, at the U.S. Capitol in Washington, D.C., U.S., May 11, 2021.

Greg Nash | Reuters

WASHINGTON — Republican Sen. Rand Paul and his wife had not bought or sold stock in an individual company in at least 10 years when Kelley Paul purchased shares of the drug company Gilead Sciences in early 2020.

The purchase came early in the novel coronavirus’ initial wave through the United States — and one day after the first U.S. clinical trial began for Gilead’s remdesivir as a treatment for Covid-19, according to records reviewed by CNBC.

That purchase and its timing made headlines Wednesday when the Kentucky senator disclosed it for the first time in a mandatory Senate filing — more than 16 months after the legal deadline for reporting it had passed.

Rand Paul has been one of the leading opponents of Covid mask mandates and other preventative measures, calling for people to “resist” them. YouTube suspended his official account Tuesday over his claims that masks don’t prevent infections. Paul called the suspension a “badge of honor.”

The purchase of up to $15,000 worth of Gilead shares was made three weeks before the World Health Organization declared Covid a pandemic. On Feb. 26, 2020, the day Kelley Paul bought the shares, there were only 14 confirmed cases of Covid in the United States.

The 2012 STOCK Act requires members of Congress to disclose the purchase and sale of individual stocks, bonds and commodity futures within 45 days of the transaction.

Other assets — such as mutual funds, EIFs and T-bills — are exempt from the 45-day requirement and need to be disclosed only once a year. The different reporting schedules prioritize the disclosure of trades that could be used to profit from nonpublic information.

Since 2012, Paul has disclosed 187 transactions involving mutual funds, EIFs, trusts and government bonds in his annual reports. But he has disclosed only one transaction in an individual stock: Gilead.

Paul’s office said he filled out a disclosure form about the Gilead purchase on time in 2020, but through an oversight it was not transmitted to the Senate records office.

It is not out of the ordinary for a U.S. senator such as Paul or his spouse to buy stock in a publicly traded company like Gilead. But for Rand and Kelley Paul, Gilead is the first and only individual stock that the lawmaker has reported he or his wife buying or selling during his 10 years in the Senate.

Paul is a member of the Senate health committee, which received a private briefing in January 2020 on the threat of the coronavirus from Trump administration officials. A Paul spokesperson said the senator did not attend any Covid committee briefings.

A prominent Washington ethics lawyer, who declined to be named because his clients are both Republican and Democratic elected officials, told CNBC, “If the [Securities and Exchange Commission] were conducting an insider trading investigation of this transaction they would see the sudden purchase of individual stocks when the subject of the investigation had not purchased individual stocks before and had recently had access to market-moving information as a huge red flag.”

Last year, federal prosecutors investigated stock sales made in advance of a coronavirus-fueled market plunge by and connected to Sen. Richard Burr, R-N.C., Sen. Jim Inhofe, R-Okla., then-Sen. Kelly Loeffler, R-Ga., and Sen. Dianne Feinstein, D-Calif.

Those probes ended without charges being filed — but the investigations and details about the controversial trades were widely publicized at the time. Loeffler was defeated in a runoff election in January.

By not disclosing the purchase, Paul avoided becoming the subject of an investigation like the ones that targeted his fellow senators last year.

Paul’s disclosure Wednesday was first reported by The Washington Post. But the fact that the Gilead shares were the couple’s one and only stock buy in the last decade has not been reported until now.

A spokeswoman for Paul said the senator and his wife “lost money” on the Gilead stock.

While it’s true that the price of Gilead is lower now than when Kelley Paul bought the shares, she has not sold the Gilead stock yet, meaning she has not realized any losses or gains from it.

CNBC asked Paul’s spokeswoman, Kelsey Cooper, if the senator or his wife had bought or sold any stocks in the year since the Gilead purchase. She did not answer.

CNBC Politics

Read more of CNBC’s politics coverage:

The price of Gilead stock has fluctuated since Kelley Paul bought shares at $74.70, climbing as high as $83.99 and falling as low as $56.56.

Gilead shares were trading at $70.65 late Thursday.

Exactly how many shares Kelley Paul owns is unclear. Senators are required to report the value of transactions by them or their spouses only within a range of dollar values. In this case, Kelley Paul bought between $1,001 and $15,000 worth of shares, Sen. Paul’s disclosure said.

Last month, Sen. Tommy Tuberville, R-Ala., disclosed stock and stock option trades valued at a total of between $894,000 and $3.5 million from January through May.

Like Paul, Tuberville made his disclosure after the expiration of the deadline set by the STOCK Act.

Tuberville’s trades included a Jan. 25 sale of stock put options for Alibaba Group Holding Limited, the giant Chinese e-commerce company. Tuberville is a leading critic of China.

A Tuberville spokeswoman told CNBC last month that the senator had not even known about the individual stock and stock option trades and therefore also had not known they needed to be disclosed by the STOCK Act’s deadline.

She said Tuberville has financial advisors who handle his stock trading. She would not identify those managers when asked who they were.

Correction: This article has been updated to reflect the correct spelling of remdesivir.

Categories
Health

The Rationing of a Final-Resort Covid Remedy

The Rationing of a Last-Resort Covid Treatment

The results vary widely between hospitals. Overall, however, survival rates have decreased over time, including in large US and European hospitals. From January to May 2020, according to the international register, less than 40 percent of Covid patients died in the first 90 days after the start of ECMO. But more than half died in the months that followed. “The patients seem to be doing significantly worse,” said Dr. Barbaro.

He and his colleagues analyze whether this is related to factors such as new virus variants, less experienced care centers or changes in the treatment of patients before ECMO.

ECMO is offered in a few community hospitals that care for most Americans. The exception is Saint John’s, the Santa Monica facility where the doctor and police sergeant were treated.

An ECMO program started about a year before the advent of Covid-19. The 266-bed hospital provided therapy to 52 Covid patients during the pandemic, much like the entire Northwell healthcare system in New York, which has more than 6,000 hospital and long-term care beds.

The Saint John’s Charity Foundation, supported by the area’s affluent donor base, helped fund the ECMO program and its expansion. The hospital accepted some uninsured Covid patients for ECMO, while those patients elsewhere were often turned down despite a federal program that reimburses hospitals for their treatment.

“There are just so many inequalities,” said Dr. Hammond, director of the Saint John intensive care unit. And for every Covid patient who has survived with ECMO, there are “probably three, four, five people who will die on the waiting list”.

She and other doctors said the pandemic highlighted the need to make ECMO more widely available and less resource intensive. Until then, “we really need a sharing system,” she said. There are allocation systems for transplant organs and trauma care.

Categories
Health

Biogen Alzheimer’s drug and the brand new battle over dementia remedy

Biogen Alzheimer’s drug and the new battle over dementia treatment

Aduhelm from Biogen

Source: Biogen

The FDA approval of Biogen’s Alzheimer’s drug Aduhelm marked a milestone in Dr. Paul Aisen. The director of the Alzheimer’s Therapeutic Research Institute at USC has focused on the treatment of neurodegenerative disease for the past three decades and in recent years has helped guide this particular drug through the various phases of clinical trials.

But as he sat in his sun-drenched San Diego office in early June, he felt slightly puzzled by the way the Food and Drug Administration approved their use in early June on an “accelerated” basis, normally reserved for cancer drugs. This meant that the clinical benefit was considered likely, but approval for long-term use would be the subject of a larger study in a fourth phase of studies.

Aisen, who works as a paid advisor to Biogen, emphasizes the “unusual nature” of the regulator’s green light, as an advisory board of experts voted and publicly opposed the approval, and insists that there were still “many questions the “I have – they still have no answers.”

Three members of the FDA’s panel that oversees the research have resigned since it was approved this week, including Dr. Aaron Kesselheim, a medicine professor at Harvard Medical School, who said in a letter the agency’s decision on Biogen was “probably the worst drug approval decision in history.” recent US history. “

Last November, that panel said in an 8-1 vote that Biogen’s late-stage phase did not provide “strong evidence” showing that aducanumab is effective in treating Alzheimer’s; two other panellists said the data was “uncertain”.

While Aisen sees Aduhelm as an “effective treatment” for a disease that affects millions of Americans, he also has concerns about the impact of the FDA’s decision on the range of other potential treatment options that are in the late stages of development.

An immediate challenge facing other teams working on a broader Alzheimer’s drug pipeline, he said in a recent video call, would be to keep participants in ongoing studies, let alone attract new ones.

“In most cases,” he said, many people with Alzheimer’s disease would drop out of other drug trials to begin treatment with the newly approved Aduhelm. Leaving them would make the study data for these alternative drugs less useful, even if the drugs in question might one day prove to be safer, more effective, or more suitable for different stages of the disease. But perhaps pervertedly, he still regards Aduhelm’s approval as “a boost to these efforts – a powerful boost”.

Over 6 million Americans suffer from Alzheimer’s

In the past few years, some large pharmaceutical companies have abandoned brain disease research efforts, including Pfizer and Boehringer Ingelheim in 2018 – in fact, Biogen Aduhelm had given up at one point during clinical trials in 2019 before reversing its decision – after decades of failure in search of a breakthrough.

The controversy surrounding the Biogen drug, including its potential cost, is hitting a massive, unmet need for dementia treatment and a disease that costs the US up to $ 259 billion annually. The Alzheimer’s Association estimates that more than 6 million Americans have Alzheimer’s or some other form of dementia, and by 2050 that number could reach over 12 million people, which costs a trillion dollars a year.

Because of this, some dementia drug experts are focusing on the new attention and funding, rather than the potential downsides of Biogen approval, said Dr. Jeffrey Cummings, a neurologist at the University of Nevada, Las Vegas, who does an annual review of. publishes Alzheimer’s drug development pipeline. His research consistently showed that drug failure rates prior to Biogen’s approval were 99.6 percent, a stark contrast to 1 in 5 successful cancer drugs (20%).

Cummings says that any short-term adverse side effect for other drug trials “will be overcome, if at all, by increased interest from companies, venture capital, and biotechnology once they see that there is a way to get approval for a particular drug”. Illness.”

In recent history, the National Institutes of Health spent two to three times more research on heart disease and cancer than they did on dementia, and the lack of qualified participants in clinical trials also slowed progress.

Next in the dementia drug pipeline

For the handful of other Alzheimer’s drugs in development hoping to overcome the same regulatory hurdles and prove their effectiveness – including Eli Lillys Donanemab, Roches Gantenerumab, and Eiseis Lecanemab – there could be a silver lining, the first mover advantage to cede to Aduhelm.

After decades of expensive but largely unsuccessful research attempts, pharmaceutical giant Eli Lilly’s CEO David Ricks said that after a series of positive phase two results for its Donanemab offering, his company is “getting closer and closer to the goal”.

Speaking at CNBC’s Healthy Returns Summit in May, a month before the FDA approved Aduhelm’s rival Biogen, he said his team felt “good about the probability of success” and said he wanted an “accelerated” route too explore what he called “adaptative avenues for the FDA to consider earlier study of data” that “should be used in a serious and widespread disease like Alzheimer’s”.

However, he conceded that recruiting for the next phase of the studies would require a significantly larger cohort of participants, and since it would take 18 months, he did not expect a new approved product until late 2023 at the earliest.

Several experts told CNBC that the Biogen drug’s unique threshold for regulatory approval, with the treatment potential appearing to trump uncertain real-world benefits, efforts of competitors like Lilly, who are focused on drug development on relatively based on similar techniques.

Aduhelm’s own clinical study data had shown that the drug successfully attacked and cleared clusters of a certain type of protein that many researchers believe may be responsible for Alzheimer’s disease. But it didn’t offer enough evidence to prove that the drug provided cognitive benefits to patients.

Debate on targeting amyloid beta formations

Known by scientists as aducanumab, it works by offering a set of identical antibodies that are cloned from white blood cells. These antibodies are chosen for their targeting abilities, as they can identify specific proteins called beta-amyloids that have built up certain formations in the body.

There is ample evidence that these beta-amyloid formations, also known as “pathological aggregates” or “plaques,” are a major cause of Alzheimer’s disease, although the exact causal mechanisms are not yet fully understood, according to Christian Pike von USC’s Leonard Davis School of Gerontology. Nonetheless, he says the antibodies can help prevent these plaques from forming before other particles are caused to break them apart, a process that is clearly identifiable in before and after neural imaging.

As an analogy, it may be helpful to think of the amyloid beta proteins as young people walking through a city during the day, where the city is the human body and the day is a human lifespan. In certain cities, when afternoon turns into evening, individual young people gather, and some of these gatherings can become toxic and begin to cause problems. The antibodies supplied by Aduhelm act like police officers arriving at the scene, identifying disruptive gatherings, surrounding them, separating them, and then instructing bystanders to disperse the young people.

“When you say, ‘Well, hey, the FDA is buying in that general concept,'” Pike said on a phone call, “if we can remove beta-amyloid from the brains of people with the disease, even if we can there is limited evidence of cognitive benefits, “he continued,” there could be a variety of different therapies that would qualify under these criteria.

The long string of past failures within the Alzheimer’s pipeline that targeted beta-amyloid will continue to weigh on optimism until conclusive evidence is produced – something this week’s controversy over the first new approved Alzheimer’s drug in decades shows has not yet been done.

“What we’re going to find out by using this drug one way or another is whether or not the amyloid clearing hypothesis is correct,” says USC health economist Darius Lakdawalla, who argues that Biogens will continue to test it Drug will prove useful to this confirmatory experiment.

“If it’s right, then I think it opens the door to a lot of innovation, a lot of drug candidates that will try to remove amyloid in the pursuit of that hypothesis in the future.”

Categories
Health

Psychedelic drug increase in psychological well being remedy nears actuality

Psychedelic drug boom in mental health treatment nears reality

Magic mushrooms are seen in a grow room in the Netherlands in this 2007 file photo.

Peter Dejong | AP

Entrepreneur Dick Simon has never shied away from speaking up about business topics other CEOs might find too stigmatized to touch. He has spent years dedicated to improving U.S. business relations with Iran, and more recently, the Boston-based CEO has embraced another passion: improving the market for and medical community’s understanding of how psychedelic drugs can be used to treat mental illness. It’s a health, and emerging health business, that Simon came to appreciate through the firsthand frustration of watching people in his life suffering — not just from mental illness, but from the failure of existing and costly medical treatments.

Drugs long stigmatized, such as psilocybin and MDMA, are rising in profile as mental illness treatment options. Just last week, results from a phase 3 trial of MDMA combined with talk therapy for post-traumatic stress disorder showed results that were impressive.

“This is a pivotal event,” said Elemer Piros, a biotech analyst at Roth Capital Partners who covers the emerging alternative mental health treatment space. “It may not seem humongous, but it is one of the best and most rigorously executed trials in the space. And the results corroborate what we have seen time and time again from smaller studies over the past two decades,” he said, referencing remission rates double that of a placebo. “The magical experiences kept showing up, but no one had the courage to take it through to regulators.”

Join CNBC’s Healthy Returns Summit on May 11

The annual Healthy Returns Summit is on Tuesday, May 11. Meg Tirrell speaks with CDC’s Dr. Rochelle Walensky, Pfizer’s head of vaccine R&D, and Eli Lilly CEO David Ricks. Bertha Coombs talks mental health care with Centene CEO Michael Neidorff and dives into the future of telehealth with Teladoc CEO Jason Gorevic. Jim Cramer closes the day with Abbott CEO Robert Ford. Register now.

The results of the MDMA study, whose senior author is Rick Doblin, Ph.D., founder and executive director of the Multidisciplinary Association for Psychedelic Studies (MAPS), are expected to be published in Nature Medicine on Monday and FDA approval could come by 2023, according to a New York Times report.

A recent Imperial College London study of psilocybin use in depression reported in The New England Journal of Medicine also produced positive results. Before the end of the year, clinical results also are expected from a study involving Compass Pathways — which IPO’d late last year — using its approach of guided psilocybin experiences as a treatment for drug-resistant depression.

“People still believe that ‘your brain on drugs’ commercial is the truth rather than all scientific evidence on major therapeutic benefits,” said Simon, who heads the Psychedelic Medicines for Mental Health Group at entrepreneurial network YPO and also serves on an advisory council at Mass General Hospital on the topic. (Dr. Sharmin Ghaznavi, Mass General Hospital Assoc. Director, Center for the Neuroscience of Psychedelics, will speak at the CNBC Healthy Returns Summit on Tuesday, May 11.)

A focus on depression treatment outcomes

There are example of stigmatized drugs in FDA-approved medical usage, ketamine, for example, as an anesthesia since the 1970s, and ultimately, used on an “off-label” basis to treat depression based on the existing FDA authorization. In 2019, a Johnson & Johnson ketamine-derived treatment for drug-resistant depression was the first new approach for the mental health condition specifically approved by the FDA in decades.

The current treatment approach of helping people to live with depression and PTSD, and on medication, creates a patient population and cost factor that is a burden on the health-care system. That may ultimately help the new drug companies gain acceptance if the clinical trials results continue to be positive.

A close friend of Simon’s almost lost a child suffering from mental illness. The individual was looking at a prognosis of never going back to school, never being able to work, at best not being a danger to themselves with medication. “That was not a prognosis you want for a 20-year-old,” he said. “They had tried everything, and eventually out of complete desperation, they started learning about the potential for psychedelic-assisted therapies, and it worked,” he told CNBC in an interview conducted late last year.

Now, he says, that person is off medication, in a relationship and leading a normal professional life.

Mental illness is among the most costly medical expenses in the U.S., and it has a high cost to employers in lost productivity. In 2019, 51.5 million adults were living with a mental illness in the U.S., and the number of people suffering and drug costs, already in the tens of billions of dollars annually, are projected to grow in the years ahead, with Covid-19 compounding mental health issues globally.

Roughly 7% of Americans suffer depressive episodes annually, and roughly 1% are resistant to treatment, the latter associated with a significantly higher economic burden including hospitalization. Americans who suffer depressive episodes have additional bouts within 2-5 years at a rate exceeding 40%, according to a recent Cowen & Company research report on Compass Pathways, and that risk increases with each new depressive episode.

“Covid has done a lot of terrible things, but it has elevated mental health visibility, and as a result of that there is lots of interest,” Simon said.

Public vs. professional acceptance of illegal drugs

Denver became the first city in the U.S. to decriminalize psychedelic mushrooms in 2019, and in a 2020 ballot measure, voters in Oregon made it the first state to decriminalize mushrooms and legalize them for treatment purposes. But investors behind the new drug treatment approaches are not focused on public acceptance, the trend of microdosing (for which they say data remains slim) or consumer recreational market potential, though many do find ideas about these drugs to be outdated.

“Consciousness is not the key here,” Simon said. “For purely medical use, there is a tremendous amount of data and traction for expanding use, which is where I’m focused.”

One of the biggest investors in the emerging field is Atai Life Sciences, a holding company for multiple biotech start-ups pursuing alternative treatments for depression, anxiety and addiction based on stigmatized drugs, and backed by venture capitalist Peter Thiel. It recently filed for an IPO.

Atai’s chairman Christian Angermayer — who says he has never touched a beer even though he comes from Bavaria where it is “our daily nutrition,” or smoked a joint or cigarette — is a personal believer in the power of psychedelics to have a positive influence on life. He described his first experience with psychedelics as “the single most meaningful thing” in his life.

“Nothing else even comes close,” Angermayer told CNBC in an interview conducted late last year.

But his personal experience is distinct from his role as an investor and executive focused on the mental health market needs. Angermayer was an early investor in Compass Pathways, where one of the founders, Lars Christian Wilde, suffered from drug-resistant depression and found help in psychedelics.

“We want to bring it back to the legal realm, but in the shamanistic setting of today, and that is with a therapist. We want to make it legal, but solely for doctors or psychotherapists in a clinical setting,” said Angermayer, who will speak at CNBC Healthy Returns on Tuesday. “These are not drugs you can take alone and not everyone can afford to go to the Amazon and see a shaman. We need to bring it into the medical system.”

Investment risks

A common thread among those closely watching, and investing in this space, is the personal experience with family and friends suffering from mental illness and struggling to find a successful medical treatment. “These people have been suffering for decades,” said Piros, who has a family member now struggling with depression and who has not yet found an effective medical therapy.

The new companies come with a high level of investment risk, common in the biotech space, with early trials showing promise but the business generating no revenue today. Advocates and investors in these alternative drug treatments say the economic argument is compelling when compared to current options.

Piros, who has spent more than two decades analyzing biotech companies, says investors need to be mindful that when you get involved with a development stage company it is not about the money being made already, but factors including how long the companies will have IP protection, when they can be expected to enter the market, and potential cash flows over a period between a decade to 15 years.

I’m not a medical professional or a researcher, but as a CEO and entrepreneur, I’m someone who is used to making things happen.

Dick Simon, heads the Psychedelic Medicines for Mental Health Group at entrepreneurial network YPO

Unlike biotechs working with brand new compounds which have a failure rate as high as 90%, the trials using psychedelic drugs that have been studied for decades are less likely to result in outright failures. Still, Piros said that the proper way to think about this new theme is as part of an existing investment risk tolerance for the biotech sector, and these new drugs should be no more than 10% of that existing allocation.

“It’s not chronic medicine, which as a business model is reasonably predictable and a great business model. It remains to be seen how this business model works, but … if we only need treatment for depression twice a year to be in remission that is a thousand times better than anything we can offer today, and PTSD has no approved drug,” Piros said. “It’s not like a crapshoot anymore.”

If a company like Compass makes it to market, its treatment approach could reach millions of Americans — estimates range from roughly 2 million to 4 million — not being served well by the current class of depression drugs. Pricing of the treatment could be $10,000, according to Cowen estimates, or as high as a $20,000, according to Piros, which he said is closer to the cost of current treatments. Depending on the number of patients who are resistant to current drugs that the therapy reaches, a 5%-7% market share could be worth billions. According to a Cowen estimate, $1 billion in annual sales; according to Berenberg Capital Markets, more than $2.5 billion; and according to Piros, possibly as much as $5 billion for a new, successful entrant at peak.

“We don’t expect 5% penetration two years after launch, more like five to seven years after launch, and going beyond 5% is crazy. But that is still a very large chunk of value,” Piros said. “We don’t need to go to the consumer angle.”

Many factors would influence the overall size of the market, from patients who are designated as good candidates for the new treatments, to the number of treatments needed, the infrastructure required for the guided sessions, which need to be in controlled environments like existing treatment centers that currently administer ketamine, and physician acceptance. Compass is creating 100 centers to train medical professionals and offer guided therapy, and plans to grow to 3,800 centers in a peak year.

Medical resistance

Getting the medical establishment to embrace these treatments may be among the most difficult parts of the journey. Piros said he has discussed alternative treatments with psychiatrists on behalf of his family, but they told him they would not be interested until there are decades of placebo-controlled trial data behind the drugs. “These were young doctors, fully up to date on the latest trials and literature. It’s going to be a long road before full acceptance.”

Cowen expects the existing anti-depression drugs in the SSRI (selective serotonin reuptake inhibitor) class, which account for upwards of 75% of prescriptions, to remain the first line of choice in therapy, but its analysts also wrote in a recent report that surveying and interviews it has conducted with doctors indicate roughly 30% of patients are resistant to these drugs and as many as 1 in 4 patients might be considered for new treatment alternatives.

The executives in the field know the history, and even with seven decades of research into use of psychedelics culminating in the latest, more rigorous work, they expect continued resistance. But they are determined, and now with a growing body of clinical trial data to back up psychedelics.

“There are those who have been out there in the wilderness metaphorically, major institutions carrying on research over the last decade. How do I help them get past the stigma society still has around this work?” Simon asked. “How do you get veterans groups who don’t like the fact that 22 veterans a day commit suicide, and each year more die in suicide than in all wars since 9/11, how do you engage them, across the political spectrum? I’m not a medical professional or researcher, but as a CEO and entrepreneur, I’m someone who is used to making things happen.”

After his initial psychedelic trip, Angermayer said the first thought he had was that he needed to call his parents and tell them how much he loved them. The second thought: “This must be legal as a treatment. … We’re several years away. It’s not tomorrow, but it’s not in ten years,” he said. 

If you are having thoughts of suicide, call the National Suicide Prevention Lifeline at 1-800-273-8255 (TALK) or go to SpeakingOfSuicide.com/resources for a list of additional resources.

For more exclusive insights from our reporters and speakers, sign up for our Healthy Returns newsletter to get the latest delivered straight to your inbox weekly. For a front row seat at CNBC Events, you can hear directly from the visionary executives, innovators, leaders and influencers taking the stage in “The Keynote Podcast.” Listen now, however you get your podcasts.

Categories
Health

MDMA Reaches Subsequent Step Towards Approval for Therapy

MDMA Reaches Next Step Toward Approval for Treatment

However, in the early 1980s, MDMA fled the clinic to the dance floor, where it became known as ecstasy. In 1985 the Drug Enforcement Administration criminalized MDMA as a List I substance, defined as “currently unaccepted medical use and high potential for abuse”.

Some mental health workers continued to administer MDMA-based therapies underground, but most stopped. The number of scientists completing studies with MDMA also decreased. Some people, including Dr. Doblin, who formed his association in 1986 to focus on developing MDMA and other psychedelics into FDA-approved drugs, continued to be heavily involved in MDMA research. It took nearly two decades to overcome alarmist claims about Ecstasy’s dangers, including the fact that it had eaten holes in users’ brains, to finally get approval to start college. Animal and human studies confirm that MDMA does not cause neurotoxic effects at the doses used in clinical studies.

Ecstasy or molly, on the other hand, can be adulterated with other potentially dangerous substances, and users can take doses far higher than is safe. According to a database maintained by the Drug Abuse and Mental Health Administration up to this year, MDMA accounted for 1.8 percent of all visits to the US emergency room in 2011. In Europe, MDMA was responsible for 8 percent of drug-related emergency visits to 16 major hospitals in 10 countries from 2013 to 2014.

Scientists still do not fully understand the source of MDMA’s therapeutic effects. The substance binds to proteins that regulate serotonin, a neurotransmitter that can, among other things, improve mood. Antidepressants like Prozac bind to the same proteins and block their reabsorption of serotonin. However, MDMA continues this process and causes the proteins to pump serotonin into synapses and strengthen their chemical signal.

MDMA also increases levels of oxytocin, dopamine, and other chemical messengers and creates feelings of empathy, trust, and compassion.

The primary therapeutic effect, however, may be due to the apparent ability to reopen what neuroscientists refer to as “critical phase”, the window in childhood when the brain has the superior ability to create and recreate new memories to save. A mouse study published in Nature in 2019 found that MDMA may restore the adult brain to this earlier state of malleability.

An estimated 7 percent of the US population will suffer from PTSD at some point in their life, and up to 13 percent of combat veterans will have the disease. In 2018, the U.S. Department of Veterans Affairs spent $ 17 billion on disability payments for over one million veterans with PTSD.

Categories
Health

Regeneron to request FDA clearance for antibody drug as preventative remedy

Regeneron to request FDA clearance for antibody drug as preventative treatment

View of Regeneron Pharmaceuticals corporate, research and development headquarters on Old Saw Mill River Road in Tarrytown, New York.

Lev Radin | LightRocket | Getty Images

Regeneron Pharmaceuticals announced Monday that it would ask the Food and Drug Administration to approve the use of its Covid-19 antibody therapy as a preventative treatment.

The therapy, given to former President Donald Trump shortly after he was diagnosed with Covid-19 last year, has already been approved by the FDA to treat adults with mild to moderate Covid-19 and pediatric patients aged 12 and over approved age who tested positive for the virus and is at high risk of serious illness.

Regeneron said it plans to expand the use of its treatment in the United States after a Phase 3 clinical study jointly conducted by the National Institutes of Health found the drug reduced the risk of symptomatic infections in individuals by 81%.

The company also said that people who were symptomatic and treated with the drug resolved their symptoms an average of two weeks faster than those who received a placebo.

“As more than 60,000 Americans continue to be diagnosed with COVID-19 every day, the REGEN-COV antibody cocktail can help provide immediate protection to unvaccinated people exposed to the virus,” said Dr. George Yancopoulos, President and Chief Scientific Officer of Regeneron, said in a press release.

The study included 1,505 people who were not infected with the virus but lived in the same household as someone who recently tested positive. Participants received either a dose of Regeneron therapy or a placebo.

The company said 41% of the people in the study were Hispanic and 9% were Black. Additionally, 33% of the participants were obese and 38% were 50 years and older, according to the company.

Regeneron therapy belongs to a class of treatments known as monoclonal antibodies, which act as immune cells to fight infections. Monoclonal antibody treatments attracted widespread attention after it was revealed that Trump had received Regeneron’s drug in October.

In recent months, public health officials have raised concerns that emerging, highly contagious variants of coronavirus could threaten monoclonal antibodies on the market. Dr. However, Myron Cohen, who leads monoclonal antibody efforts for the NIH-sponsored COVID Prevention Network, said the drug has shown that it will retain its effectiveness against new strains.

As the world’s attention has shifted to giving Covid-19 vaccines, health experts say treatments are also crucial to ending the pandemic, which, according to compiled data, has topped 31.1 million in just over a year Infected Americans and killed at least 561,800 people from Johns Hopkins University.

Categories
Health

Liesbeth Stoeffler, 61, Runner Stored Going by Uncommon Lung Therapy, Dies

Liesbeth Stoeffler, 61, Runner Kept Going by Rare Lung Treatment, Dies

Liesbeth Stoeffler’s doctors had to make a courageous decision in 2009. Ms. Stoeffler was on a ventilator and deeply sedated after cystic fibrosis destroyed the lungs that had once given her the ability to run and hike.

She needed a double lung transplant, but doctors feared that prolonged ventilatory time could make her too weak or malnourished to be eligible for a transplant.

Doctors at Columbia University’s Irving Medical Center took her off the ventilator in about a day and hooked her to an extracorporeal membrane oxygenation machine (ECMO) that pumped blood out of her body, removed carbon dioxide, and flowed oxygen-rich blood back into her. In fact, it looked like an artificial lung.

It was a rarely known and risky use of the machine, which not only enabled Ms. Stoeffler to wake up from the calm. She was also able to eat, talk on her smartphone, exercise in bed, and walk on the spot while connected – an unusually long 18 days for the transplant to take place.

“The ECMO was the bridge between my respiratory failure and the transplant,” Ms. Stoeffler told USA Today in 2009.

ECMO – a treatment for viruses that damage the lungs – has proven extremely helpful in the past in cases of H1N1 flu (or swine flu) and is used, according to Columbia and other ECMO centers around the world. A study published in the medical journal The Lancet last September showed that 62.6 percent of 1,035 seriously ill Covid-19 patients survived after ECMO treatment.

Ms. Stoeffler’s transplanted lungs worked well for almost a decade, allowing her to hike in the mountains near her parents’ home in Austria and complete two New York marathons, half marathons, an Ironman bike course, and a sprint triathlon.

But her body eventually refused the transplanted lungs, and she underwent another transplant in 2019. It didn’t work that well or lasted so long. Ms. Stoeffler died of cystic fibrosis at Irving Medical Center on March 4, said her brother Ewald Stoffler. She was 61 years old.

Liesbeth Stoeffler was born on June 18, 1959 in Hermagor, Austria, a town at the foot of the Carnic Alps. Her father Johann was a truck driver; Her mother, Margarethe (Strempfl) Stoeffler, was a housewife.

After graduating from business school, she left Austria in 1977 for an au pair job in Manhattan, where she had hoped to move since she was a teenager, her brother said in an email.

Updated

March 26, 2021 at 12:43 am ET

“During the first three years that Liesbeth spent in New York, she refused to speak a single word of German,” wrote Stoeffler, “so that she could learn English as quickly and as well as possible.”

She took courses in computers and graphic design and was hired by Deutsche Bank, Blackstone Group, and eventually investment management firm Sanford C. Bernstein (now AllianceBernstein). She worked there for nearly 20 years, rising to vice president and presentation specialist, creating graphics for marketing and sales documents.

During her time at Bernstein, she developed breathing problems and found out in 1995 that she had cystic fibrosis. But she kept this largely to herself.

“She always coughed and got her staff to ask her to check it out,” said Christina Restivo, a close friend she met in Bernstein and who headed a support team of friends who looked after her. “She kept it private until she got to the point where the only way to live was a double transplant.”

In June 2009, after a routine blood test in the hospital, Ms. Stoeffler felt too exhausted to return home. One of her doctors, David Lederer, a pulmonologist, admitted it.

“She was in intensive care and on a ventilator within 48 hours,” he said in a video of her case created by Irving Medical Center. He added, “She didn’t really improve the vent support we provided for her so we knew we had to do something for her.”

Using the ECMO helped her remain eligible for the transplant. “About five days later she told me it was the best thing she’d felt in years,” said Dr. Matthew Bacchetta, who also treated Ms. Stoeffler, an online publication in Columbia.

In less than two years, Ms. Stoeffler started running seriously. Starting with the Fred Lebow Classic, a five-mile race in Central Park in January 2011 (named after the founder of the New York City Marathon), she finished 47 different races hosted by the New York Road Runners Club. Their last was an 8-kilometer event in August 2017.

Ms. Restivo said her friend’s running likely extended the life of her transplanted lungs.

“Because your immune system is so suppressed by a transplant, she was told not to work out in a gym where she could pick up bacteria,” she said. “She used nature to exercise her lungs.”

In addition to her brother Ewald, three sisters, Gabriele and Birgit Stoeffler and Waltraud Wildpanner, Mrs. Stoeffler survive. and another brother, Hannes.

Ms. Restivo, who is Ms. Stoeffler’s executor, said Ms. Stoeffler would sometimes write to the doctors with instructions. Another text arrived on her last day.

“I got a call to go to the hospital at 3:30 am,” she said. “Liesbeth was still vigilant with her oxygen mask, texting me as usual, telling me what to do and keeping me informed of her status. Fully aware at all times. “

Categories
Health

Sickle Cell Therapy Not Linked to Most cancers, Researchers Say

Sickle Cell Treatment Not Linked to Cancer, Researchers Say

Just weeks after promising sickle cell disease gene therapy appeared to hit a roadblock, the outlook for treatment is now looking brighter. Preliminary data suggesting it could cause cancer has not held up.

In gene therapy, scientists insert a normal gene into the patient’s DNA to correct sickle cell disease caused by a devastating mutation. The cutting-edge treatment could prove to be a cure, and a company testing the treatment, Bluebird Bio, was on track to apply for approval from the Food and Drug Administration next year.

However, on February 16, Bluebird Bio announced that a sickle cell patient treated in a clinical trial five years ago had developed acute myeloid leukemia. Another patient developed acute myelodysplastic syndrome, a form of cancer that is often a precursor to leukemia.

The company stopped its studies of sickle cell patients and those with another blood disorder called beta thalassemia while its researchers tried to understand whether gene therapy was flawed.

On Wednesday, Bluebird Bio reported that it had found no evidence that gene therapy caused the sickle cell patient’s leukemia.

The gene inserted into the patient’s DNA did not interfere with the function of other genes, the company said. And the gene wasn’t inserted into the genome near anyone else known to be involved in leukemia.

Bluebird Bio is still investigating whether its treatment is related to acute myelodysplastic syndrome, but officials have asked the Food and Drug Administration to allow their clinical trials to continue.

A separate sickle cell study at Boston Children’s Hospital was also discontinued when Bluebird Bio announced the two cancers at the request of the National Institutes of Health, which is paying for the study.

Dr. David Williams, a hematologist at Boston Children’s and lead researcher on the study, said the researchers are asking permission from the NIH to resume their work.

Like Bluebird Bio investigators, Dr. Williams and his colleagues used a disabled lentivirus to deliver a gene to sickle cell patients. Lentiviruses are considered safe – hundreds of patients in other gene therapy studies have been treated with them and no blood cancers have been reported. The possibility that lentiviruses may not be safe was a matter of great concern.

The leukemia patient in the Bluebird Bio study had genetic abnormalities related to leukemia, which could explain why they developed.

Philip Gregory, the company’s chief scientist, said it was not yet clear whether the patient diagnosed with myelodysplastic syndrome actually had it. So far, Bluebird Bio has not been able to find any cancer cells in its bone marrow.

“He may have been diagnosed prematurely,” said Dr. Gregory. If cancer cells are found in the patient’s marrow, the company will perform the same detailed molecular analysis it did for the leukemia patient, added Dr. Gregory added.

Dr. John Tisdale, director of cellular and molecular therapeutics at the National Heart, Lung and Blood Institute, was cautiously optimistic.

“These data actually dismiss the vector as causal,” he wrote in an email. He added that the researchers need a better understanding of the study participants’ illnesses before they can exhale one last sigh of relief.