The U.S. Food and Drug Administration hasn’t approved a new medication for Alzheimer’s since 2003, but a new study published by the New England Journal of Medicine on Saturday is showing hopeful initial results.
Donanemab, an antibody treatment produced by drug-maker Eli Lilly and Company, targets a modified form of amyloid-β (Aβ) peptide — a plaque that accumulates in the brains of people with Alzheimer’s.Early-stage clinical trial results showed that by 52 weeks in, the amyloid plaque levels of those receiving the experimental drug achieved a negative status. In other words, their amyloid plaque levels were the same as the average person’s.
To set the study up, half of the 275 participating patients received the drug for 76 weeks as part of the trial, while the other half received a placebo. Those involved were people with early symptomatic Alzheimer’s disease.
“We are confident in the results of the TRAILBLAZER-ALZ study,” Daniel Skovronsky, Lilly’s chief scientific officer and president of Lilly Research Laboratories, said in a statement. “This is the first late-stage study in Alzheimer’s disease to meet its primary endpoint at the primary analysis. Donanemab has the potential to become a very important treatment for Alzheimer’s disease.”
By monitoring memory and the ability to perform daily tasks, researchers also discovered that the drug seems to slow cognitive decline by about 32%.
Overall, about 40% of participants treated with donanemab achieved amyloid negativity as early as six months after starting treatment, and 68% reached this goal by 18 months in.
“We were pleased to see not only slowing of cognitive and functional decline, but also very substantial clearance of amyloid plaques and slowing of spread of tau pathology,” Skovronsky said. “The constellation of clinical and biomarker results indicates the potential for long-term disease modification. We are grateful to the patients, caregivers, and investigators who participated in this landmark study.”
Though results are promising, researchers said longer and more expansive trials are still necessary going forward to ensure donanemab’s safety.
According to the Centers for Disease Control and Prevention, Alzheimer’s is one of the top 10 leading causes of death in the U.S., and the fifth leading cause of death among adults aged 65 or older.
The FDA is also currently undergoing a review of another, separate experimental drug from Biogen, a Cambridge-based company, and Japanese partner Eisai Co.
Only a handful of drugs are approved to manage Alzheimer’s symptoms, and there are none to treat the cause.
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The race to find vaccines for COVID-19 has dominated the headlines, but there’s been less news about how to keep people with COVID out of the hospital. Tonight, we’re going to tell you a story about one possible treatment. It’s called fluvoxamine. The generic drug was developed 40 years ago as an antidepressant and has been primarily used to treat obsessive-compulsive disorder. Now, a small but ingenious clinical trial and a series of coincidences have led scientists to look closely at fluvoxamine as a possible tool to keep newly diagnosed COVID-19 patients from becoming severely ill. So how did a pill that costs 60 cents become a dark horse to treat COVID? We went to a place that knows all about long shots to find out.
Golden Gate Fields is in Berkeley, California. The stands have been empty since COVID hit last year, but the races go on. There are 1,200 thoroughbreds here – trained and cared for by more than 500 people. Last November, COVID went on a rampage in the barn area where many workers live.
Horses race at California’s Golden Gate Fields
Dr. David Seftel: We had four cases that were initially reported and because we have a community living back there, we decided to test everybody. And that’s when we saw the first round of testing reveal 200 positive individuals.
Sharyn Alfonsi: Wow. What was your reaction when you heard 200 positive cases right here?
Dr. David Seftel: Shock and dismay.
Dr. David Seftel has been the physician for employees and their families at Golden Gate Fields for 20 years.
He is originally from South Africa and is Harvard-educated.
Sharyn Alfonsi: Who was sick? Was it the jockeys, was it the guys who work in the stable, their families?
Dr. David Seftel: It was really across the entire spectrum. And what’s interesting about our community is that it really is a mirror image of the community that is most affected by COVID, predominantly Latino community, incredibly hardworking. They don’t have the luxury of working from home or working on Zoom. They have to be out there every single day.
Dr. David Seftel
But there are few early treatment options for COVID. The handful of drugs that have been approved are for high risk patients and must be delivered intravenously, often in a hospital.
Dr. David Seftel: When I looked at this community, I said I know the numbers, I know the stats. There are gonna be deaths and there’s gonna be disability unless I take action.
Sharyn Alfonsi: Is that what you were thinking as the numbers kind of rolled in?
Dr. David Seftel: This was a disaster in the making.
Dr. Seftel felt his only choice to keep his patients from getting sicker was to act on a tip he got just hours before. The doctor offered them the antidepressant fluvoxamine. To understand why, you have to go back to the starting gate of our story.
Eight months earlier in March, Dr. Angela Reiersen, a child psychiatrist at Washington University in St. Louis, was home sick with COVID symptoms. And thinking about old medical studies she’d read.
Sharyn Alfonsi: Most people when they’re home sick with COVID, they say, “Look, I just wanna sit on the couch, and ride this out.”
Dr. Angela Reiersen: Well, I didn’t wanna just sit there and be sick. I was really kind of driven to try to find answers.
Dr. Reiersen remembered a study published a year earlier by these researchers at the University of Virginia on mice. They found fluvoxamine stopped sepsis. Sepsis is a runaway immune response in which inflammation gets out of control, damages organs and can be deadly. It’s believed a similar phenomenon occurs in COVID patients.
Dr. Angela Reiersen
Dr. Angela Reiersen: And I thought, well, I wonder if we could use fluvoxamine to treat COVID and prevent that clinical deterioration?
Sharyn Alfonsi: You thought, this is something that might be able to stop inflammation from going into overdrive?
Dr. Angela Reiersen: Right. Either stop the inflammation from going into overdrive or shut it down once it had started to prevent our own bodies from destroying ourselves basically. So, then I emailed Eric Lenze, and just kind of explained the whole rationale behind it in an email.
Dr. Eric Lenze is also a psychiatrist at Washington University. He specializes in finding new uses for drugs already approved by the Food and Drug Administration.
Sharyn Alfonsi: Did you have some skepticism at first?
Dr. Eric Lenze: Amazingly, I did not.Angela presented a very compelling and innovative case for this drug. And it turns out that there’s a lot of properties of– psychiatric drugs like… safety and ease of use… and the fact that they can get into the body quickly that makes ’em actually ideal for repurposing.
Dr. Eric Lenze
The doctors got $20,000 from Washington University last April to launch a small randomized clinical trial on fluvoxamine. But getting patients to try an antidepressant for COVID was hard.
Sharyn Alfonsi: How’d you sell it?
Dr. Eric Lenze: Yeah, and that was a real steep learning curve for us as well that we’re doing with this antidepressant drug that we usually use for obsessive-compulsive disorder. Imagine you’re a patient at home, sick with COVID and you get a phone call like that.
Patients who agreed didn’t have to leave their homes. Researchers would drop off a paper bag containing fluvoxamine pills to half of the COVID patients. The other half would get a placebo, with instructions to take the pills for 15 days.
Dr. Eric Lenze: Our team was acting like– couriers, or, if you will, delivery men, dropping it off at their house. And then we would work with them through the phone and the internet. By May we were kind of running on fumes, as far as– funding went. Fortunately it was at that point that I– read in the New York Times, of all places, about the COVID Early Treatment Fund.
Steve Kirsch is the founder of the group Lenze read about. Kirsch is a Silicon Valley entrepreneur who made a fortune developing the optical computer mouse. He put up a million dollars of his own money and then assembled a panel of scientists to decide which covid research he should fund.
Sharyn Alfonsi: Tell me about the first conversation you had with Dr. Lenze.
Steve Kirsch: You know, we were like, oh, we– we got a grant application. This is thrilling to us. And it’s for $67,000… and so it’s a very modest amount, so we ran it through the scientific advisory board and they said, you know, this is novel.
Steve Kirsch
Steve Kirsch cut the check, which allowed Dr. Lenze to finish recruiting the 152 patients he needed for his trial. It was completed in August.
Dr. Eric Lenze: So the results were really pretty incredible. Out of the 80 people who received fluvoxamine, none, zero of them deteriorated versus– 8% of the people– who got placebo.
You heard that right. The patients on fluvoxamine did not deteriorate to the point of severe lung damage.
Steve Kirsch: And he goes over the results and I’m like, holy moly.
Sharyn Alfonsi: You probably wanted to scream it from the rooftops at that point.
Steve Kirsch: Oh, absolutely–
Sharyn Alfonsi: And how did Dr. Lenze and his colleagues react?
Steve Kirsch: He said, “Well, look, we have to get this published or nobody’s gonna believe it. We want to submit it to JAMA – the Journal of the American Medical Association because that is the top journal for this once you put it in JAMA, and they publish it, then everybody will believe it.”
OCD pill being tested for COVID-19 patients
02:38
It was published in November. But while the editors offered high praise for the study’s methodology – they said the results “should not be used as the basis for current treatment decisions.” That’s because the editors wanted confirmation in a larger trial. Not the speed Steve Kirsch is used to in Silicon Valley.
Sharyn Alfonsi: I imagine you think the next morning, you’re gonna be front-page news on the New York Times–
Steve Kirsch: And everybody starts taking it and all the doctors start– you know, people start demanding it.
Sharyn Alfonsi: But that did not happen.
Steve Kirsch: No–
Sharyn Alfonsi: You ended up on a webinar.
Steve Kirsch: Yes. I ended up on a webinar.
This is the webinar that night about COVID.
It was for Harvard Business School alumni and the host was David Seftel. The race track doctor. It was just hours after Dr. Seftel hosted the group that he learned about the massive outbreak at Golden Gate Fields.
Sharyn Alfonsi: Had he heard about fluvoxamine at that point?
Steve Kirsch: No.
Sharyn Alfonsi: So he hears it from you on the webinar?
Steve Kirsch: He hears about it from me on his webinar.
Sharyn Alfonsi: Were you skeptical at all about what he was saying?
Dr. David Seftel: Absolutely. And I’m a born skeptic. Right after the webinar, I took a deep dive into the science. And then I looked at Eric Lenze’s paper. A paper that was selected out of 10,000 other papers by the JAMA for publication because its methodology was strong. This is something that I felt comfortable with taking to patients.
COVID drug studies look at repurposed drugs
02:39
So Dr. Seftel decided to offer a 15-day prescription for fluvoxamine to the track workers with COVID.
Sharyn Alfonsi: Did you feel like you were placing a bet on these patients at all?
Dr. David Seftel: No, because I weighed the risk and reward. And in this particular circumstances, strong biochemistry, great initial clinical results, minimal downside. I felt I had to act.
Dr. Seftel’s decision to use a prescription drug off-label is an accepted medical practice – with patient consent. The most common side effect of fluvoxamine is slight nausea.
Sharyn Alfonsi: How many of them ended up taking the fluvoxamine and what was the outcome?
Dr. David Seftel: Sixty-Five patients elected to take fluvoxamine. Forty-eight declined. 12.5% of all those who refused fluvoxamine ended up hospitalized and one died. In the group that did take fluvoxamine, none of them were hospitalized.
Once again – none of the covid patients taking fluvoxamine deteriorated.
Sharyn Alfonsi: Could it have been a fluke?
Dr. David Seftel: I don’t believe so. You cannot influence a virus that is as wily and as wicked as COVID with a fluke.
But to be trusted by the wider medical community, fluvoxamine needed a larger trial. So Steve Kirsch’s fund put a half million dollars behind a new trial led by Dr. Eric Lenze. Paper bag deliveries have been replaced by FedEx boxes that the team plans to ship to more than a thousand COVID patients around the country and Canada.
Dr. Eric Lenze: I have to be a scientist about this. We’ve tested it in one study. But– in my view, it needs to be confirmed in a larger study.
Sharyn Alfonsi: Is it reasonable to think that this drug could be an answer?
Dr. Francis Collins: Fluvoxamine could certainly be something you wanna put in the tool chest. ‘Cause it looks as if it has the promise to reduce the likelihood of severe illness.
Dr. Francis Collins is the director of the National Institutes of Health. As part of the pandemic response, Collins oversees the federal effort to identify drugs to repurpose for COVID treatment. It’s a priority because of concerns that new COVID variants could make vaccines less effective.
Dr. Francis Collins
Dr. Francis Collins: Dr. Lenze is a great example of a physician scientist who probably never planned to work on an infectious disease. And yet approaches it with appropriate skepticism about anything that isn’t absolutely certain because you don’t want to make that recommendation unless you know for sure.
Sharyn Alfonsi: And how closely will you be watching– what he reports?
Dr. Francis Collins: The whole scientific community is watching his study and trying to see whether there’s a way we can help in our own trials. We’re strongly considering adding an arm to one of those trials to test fluvoxamine. To further add to the data that could be generated.
There’s been great caution about recommending repurposed drugs for COVID after the malaria drug hydroxychloroquine was promoted as a potential “game-changer” by former President Trump – before it was tested in a large clinical trial on COVID patients.
Sharyn Alfonsi: What is the bar for a drug like fluvoxamine to be widely used?
Dr. Francis Collins: Well, it’s the FDA who will have the job of figuring out whether to give an approval– for this use of that drug. And it will be about benefit and risk. And the benefit is maybe even a reduction of 20% of the chances that you’re gonna end up in the hospital, that’s probably a good thing. That should be added to the mix.
The first results from the national trial could come next month, a little more than a year after Dr. Angela Reiersen sent Dr. Eric Lenze that email about fluvoxamine.
Sharyn Alfonsi: Your colleague had to read the study. Silicon Valley guy had to step in. Then there’s some people at a race track that are gonna try it out. It seems unbelievable.
Dr. Eric Lenze: If you had told me what the odds were at the start of this, I might have reconsidered doing this.
Sharyn Alfonsi: It was a long shot.
Dr. Eric Lenze: For sure.
Produced by Guy Campanile. Associate producer, Lucy Hatcher. Broadcast associate, Elizabeth Germino. Edited by Peter M. Berman.
said he asked her if she had a boyfriend, called her sweetheart, touched her on her lower back at a reception and once kissed her hand when she rose from her desk.
Ana Liss, now 35 years old, served as a policy and operations aide to Mr. Cuomo between 2013 and 2015. She said the actions by Mr. Cuomo were unsolicited and occurred in the first year while she sat at her desk, which was near his office in the Executive Chamber of the New York State Capitol in Albany.
In an interview with The Wall Street Journal, Ms. Liss said she initially perceived Mr. Cuomo’s conduct as harmless flirtations. Over time, she said, she has come to see it as patronizing, and she added it diminished her from an educated professional to “just a skirt.”
“It’s not appropriate, really, in any setting,” she said.
In response to questions about Ms. Liss, Rich Azzopardi, a senior adviser to Mr. Cuomo said Saturday: “Reporters and photographers have covered the governor for 14 years watching him kiss men and women and posing for pictures. At the public open-house mansion reception, there are hundreds of people, and he poses for hundreds of pictures. That’s what people in politics do.”
At his last public appearance on Wednesday, Mr. Cuomo said this behavior was customary for him.
“I understand that sensitivities have changed and behavior has changed, and I get it. And I’m going to learn from it,” he said.
Ana Liss displays a pin she earned while working in the governor’s office.
Photo:
libby march for The Wall Street Journal
Ms. Liss is the third former female aide to accuse Mr. Cuomo of inappropriate behavior in the workplace. The two other former aides have said he sexually harassed them. Mr. Cuomo has apologized for making people uncomfortable. He has said he never touched anyone inappropriately.
“It was unintentional, and I truly and deeply apologize for it,” he said Wednesday. “I feel awful about it and, frankly, I am embarrassed.”
Ms. Liss and other current and former administration officials said the governor regularly asked them about their dating lives, touched them and commented about their physical appearance. Longtime staffers told some women they should wear high heels when the governor was in Albany, according to Ms. Liss and other former staffers. Mr. Azzopardi said no one was compelled to wear high heels.
The Journal spoke with more than 30 officials who either work or have worked for Mr. Cuomo during his 10 years as governor. All of those officials, who include current and former agency heads, described a high-pressure environment where seven-day workweeks were common.
Several people described the working environment as toxic. Many former staffers recalled the governor’s actions more endearingly. Once on Valentine’s Day, Mr. Cuomo had roses delivered to the female employees, they said. Two women who received the flowers said they appreciated the gesture.
When asked about the criticism of working conditions, Mr. Azzopardi said: “The people of this state elected the governor to represent them four times during the last 14 years, and they know he works day and night for them. There is no secret these are tough jobs, and the work is demanding, but we have a top-tier team with many employees who have been here for years, and many others who have left and returned.”
One former aide, 25-year-old Charlotte Bennett, recently said Mr. Cuomo asked about her sex life and whether she had relationships with older men.
Another former adviser, Lindsey Boylan, said in a Feb. 24 Medium post that Mr. Cuomo tried to kiss her on the lips in his office and, during a 2017 flight on his plane, suggested they play strip poker. A spokeswoman for Mr. Cuomo has said Ms. Boylan’s accusations are false.
The governor is facing mounting pressure over the accusations, as well as how the state handled Covid-19 in nursing homes. State Attorney General Letitia James is overseeing an investigation into the accusations by Mses. Bennett and Boylan. Federal prosecutors are interested in how the governor’s top advisers pushed to alter a Health Department report to include a lower tally of deaths in those facilities, people familiar with the matter said.
Republicans and an increasing number of Democrats have called for Mr. Cuomo’s resignation or impeachment, but senior Democratic state lawmakers are resisting until Ms. James’s review is complete.
Ms. Liss said she decided to come forward after Mses. Bennett and Boylan accused Mr. Cuomo of sexual harassment. Ms. Liss said the governor’s response to their accusations has been inadequate.
Ms. Liss won a competitive fellowship in 2013 and joined Mr. Cuomo’s team to work on economic-development programs. She already had a master’s degree from the University of Pennsylvania and had been working at a business-development firm in Rochester. She said she was proud of her role in the Executive Chamber but was dismayed that the governor never asked her about her work, focusing instead on personal questions or her appearance.
Ana Liss keeps in her office a framed picture of her and Gov. Andrew Cuomo, taken at a 2014 reception, showing his arm around her waist.
Photo:
Libby March for The Wall Street Journal
Ms. Liss recalled working at a May 6, 2014, reception at the Executive Mansion in Albany, which is Mr. Cuomo’s official residence. Mr. Cuomo was in a living room on the north side of the mansion’s first floor and noticed Ms. Liss, she recalled.
“He came right over to me and he was like, ‘Hey, Sweetheart!’” she said.
She said the governor hugged her, kissed her on both cheeks and then wrapped his arm around her lower back and grabbed her waist. They turned to a photographer, who took a picture that shows Mr. Cuomo’s hand around her waist.
In the Medium post, Ms. Boylan described a similar encounter with the governor at a Jan. 6, 2016, event at Madison Square Garden. She said Mr. Cuomo stopped to talk with her after a speech, and she was soon informed by her boss that the governor had a crush on her.
“It was an uncomfortable but all-too-familiar feeling: the struggle to be taken seriously by a powerful man who tied my worth to my body and my appearance,” Ms. Boylan wrote.
Ms. Liss said she never made a formal complaint about the behavior of the governor or anyone else. She said she eventually asked for a transfer to another office.
Ms. Liss said her experience working for the governor prompted her to begin mental-health counseling in 2014. She said she drank heavily that year, and she left the Executive Chamber in 2015 to take a position at Cornell University as a corporate-relations manager. Ms. Liss now works as the director of the Department of Planning and Development for Monroe County in upstate New York.
The Journal interviewed two other Empire State Fellows who said they observed Ms. Liss drinking heavily and skipping social engagements when she worked for the governor.
Peter Walke, a fellow who now serves as Vermont’s environmental conservation commissioner, said in a recent interview that he noticed Ms. Liss became more withdrawn over time.
After the allegations by Mses. Boylan and Bennett, Mr. Walke contacted Ms. Liss. She relayed her own experiences to him, Mr. Walke said.
Ms. Liss said she was proud of the work she did during her time in Albany, and still keeps in her office that picture of her and Mr. Cuomo at the reception. She supports the policies he has enacted.
“I just wish—I wish that he took me seriously,” she said.
—Leslie Brody and Jim Oberman contributed to this article.
Write to Jimmy Vielkind at Jimmy.Vielkind@wsj.com, Deanna Paul at deanna.paul@wsj.com and Khadeeja Safdar at khadeeja.safdar@wsj.com
MALVERN, Pa., Feb. 23, 2021 (GLOBE NEWSWIRE) — Ocugen, Inc., (NASDAQ: OCGN), a biopharmaceutical company focused on discovering, developing, and commercializing gene therapies to cure blindness diseases and developing a vaccine to save lives from COVID-19, today announced that on the recommendation of the European Medicines Agency (EMA), the European Commission has granted orphan medicinal product designation for OCU400 (AAV5-hNR2E3), for the treatment of both retinitis pigmentosa (RP) and Leber Congenital amaurosis (LCA).
The prevalence of RP in Europe is estimated at approximately 165,000 patients and the prevalence of LCA in Europe is estimated at approximately 40,000 patients. Globally, the number of people suffering from RP and LCA is estimated to be around 2.0 million and 0.2 million, respectively.
“We believe the granting of this designation by the European Commission validates the potential of our modifier gene therapy platform to treat many inherited retinal diseases (IRDs). IRDs associated with RP and LCA diseases are caused by mutations in over 175 genes, and it is impractical to develop therapies that are specific to each gene. OCU400 has the remarkable potential to address a significant number of patients globally who are in desperate need of rescue from these blindness diseases and we are working diligently to move this program to clinic,” said Dr. Shankar Musunuri, Chairman of the Board, Chief Executive Officer, and Co-founder of Ocugen.
“RP and LCA are chronically debilitating groups of IRDs characterized by severe impairment in visual functions starting as young as infancy, often progressing into night blindness and tunnel vision and eventually causing total blindness as early as the patient’s mid-40s. Since the existing approved therapy only addresses a small percentage of this population, there is an unmet need for new treatment options addressing a wider population of patients with IRDs,” said Dr. Mohamed Genead, Chair of Retina Scientific Advisory Board and Acting Chief Medical Officer of Ocugen.
Nuclear Hormone Receptors such as NR2E3 are important modulators of retinal development and function acting as “master genes” in the retina. NR2E3 is delivered to target cells in the retina using an adeno-associated viral (AAV) vector. As a potent modifier gene, expression of NR2E3 within the retina may help reset retinal homeostasis, potentially stabilizing cells and rescuing photoreceptor degeneration. Preclinical results published in Nature Gene Therapy demonstrate the potency of modifier gene therapy to elicit broad-spectrum therapeutic benefits in early and advanced stages of RP including vision rescue in early and advanced stages of the disease.
Orphan medicinal product designation in Europe offers certain benefits to drug developers while they develop drugs intended for safe and effective treatment, diagnosis, or prevention of rare diseases or conditions that impact fewer than 5 in 10,000 patients in the European Union. Benefits include protocol assistance, reduced regulatory fees, research grants, and 10 years of market exclusivity following regulatory approval.
About Retinitis Pigmentosa Retinitis pigmentosa is a clinically and genetically heterogeneous group of IRDs characterized by diffuse progressive dysfunction of predominantly rod photoreceptors, with subsequent degeneration of cone photoreceptors, and retinal pigment epithelium (RPE). Visual impairment usually manifests as night blindness and progressive visual field loss. Its prevalence is 1 in 3,000 to 1 in 5,000. RP may be seen in isolation (typical RP) or in association with systemic disease. Over 150 gene mutations have been associated with RP and this number represents only 60% of the RP population. The remaining 40% of RP patients cannot be genetically diagnosed, making it difficult to develop individual treatments.
About Leber Congenital Amaurosis Leber congenital amaurosis is a family of congenital retinal dystrophies that results in severe vision loss at an early age. Patients present usually with nystagmus, sluggish or near-absent pupillary responses, severely decreased visual acuity, photophobia and high hyperopia. It is the most severe retinal dystrophy causing blindness by the age of 1 year. This dystrophy is a genetically heterogeneous recessive disease affecting 1 in 30,000 to 1 in 81,000 subjects. Mutations in one of more than two dozen genes can cause LCA.
About OCU400 OCU400 is a novel gene therapy product candidate with the potential to be broadly effective in restoring retinal integrity and function across a range of genetically diverse IRDs. OCU400 is the first program that Ocugen is advancing based on its breakthrough modifier gene therapy platform developed by Dr. Neena Haider, Associate Professor of Ophthalmology at Harvard Medical School and Associate Scientist at the Schepens Eye Research Institute (SERI) of Massachusetts Eye and Ear. Ocugen obtained an exclusive worldwide license from SERI to develop and commercialize ophthalmology products based on specified nuclear hormone receptor genes, including NR2E3. Consisting of a functional copy of the nuclear hormone receptor gene NR2E3, OCU400 is delivered to target cells in the retina using an AAV vector. As a potent modifier gene, expression of NR2E3 within the retina may help reset retinal homeostasis, stabilizing cells and potentially rescuing photoreceptors from degeneration.
About Ocugen, Inc. Ocugen, Inc. is a biopharmaceutical company focused on discovering, developing, and commercializing gene therapies to cure blindness diseases and developing a vaccine to save lives from COVID-19. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with one drug – “one to many” and our novel biologic product candidate aims to offer better therapy to patients with underserved diseases such as wet age-related macular degeneration, diabetic macular edema, and diabetic retinopathy. We are co-developing Bharat Biotech’s COVAXIN™ vaccine candidate for COVID-19 in the U.S. market. For more information, please visit www.ocugen.com.
Cautionary Note on Forward-Looking Statements This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from our current expectations. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events or otherwise, after the date of this press release.
Ocugen Contact: Ocugen, Inc. Sanjay Subramanian Chief Financial Officer and Head of Corporate Development ir@ocugen.com
Media Contact: For Ocugen: LaVoieHealthScience Emmie Twombly etwombly@lavoiehealthscience.com +1 857-389-6042
New York Magazine writer Jonathan Chait says Hollywood’s treatment of conservative actress Gina Carano is reminiscent of the Hollywood blacklist in the 1950s.
Chait’s Friday essay for the Intelligencer argued there was no “principled distinction” between Carano’s recent termination from future episodes of The Mandalorian and her representation and the Hollywood blacklist against communists.
Carano was dropped by talent agency UTA this week after sharing a social media post comparing the current political climate to the Nazi era. Lucasfilms, which produces The Mandalorian, said it had no plans for her.
In the simplest terms, obesity is the product of a body’s energy output being less than its energy input. But in reality, there’s nothing simple about this complex and mysterious disease.
Obesity, which has skyrocketed in recent decades – now defining the body mass of over 40 percent of adult Americans – isn’t just difficult for people to endure and scientists to understand. It’s also incredibly hard to treat.
Beyond commitment to sustained lifestyle changes – healthy eating and exercise, effectively – there are really only two potential options that may help: bariatric surgery and weight-loss medications.
The former is invasive and carries various risks and complications. As for the drugs, they don’t always work, and can have their own adverse effects too.
However, an experimental treatment recently trialled by scientists and detailed in a study published this week could open new doors for treating obesity patients with a weight-loss drug.
In the study, which involved almost 2,000 obese adults across 16 different countries, participants took a weekly dose of a drug called semaglutide, an existing medication already used in the treatment of type 2 diabetes.
A control group took only a placebo, in place of the medication. Both groups received a lifestyle intervention course designed to promote weight loss.
At the end of the trial, the participants who took the placebo lost a small but clinically insignificant amount of weight. But for those who took semaglutide, the effects were pronounced.
After 68 weeks of treatment with the drug – which suppresses appetite due to a variety of effects on the brain – participants taking semaglutide lost on average 14.9 percent of their body weight. And over 30 percent of the group lost more than 20 percent of their body weight.
Broadly speaking, this makes the drug up to twice as effective as existing medications for weight loss, the researchers say, approaching the kind of efficacy of surgical interventions.
“No other drug has come close to producing this level of weight loss – this really is a game-changer,” says obesity researcher Rachel Batterham from University College London.
“For the first time, people can achieve through drugs what was only possible through weight-loss surgery.”
In addition to losing weight, participants registered improvements in other areas, showing reductions in various cardiometabolic risk factors, and reporting quality of life improvements.
While the results are compelling, semaglutide dosage for anti-obesity effects does come with some drawbacks.
Mild-to-moderate effects were reported by many participants (in both the semaglutide and placebo groups), including nausea and diarrhoea. While the effects were temporary, they were enough for nearly 60 of participants to discontinue their treatment, compared with just five in the placebo group.
At present, the drug requires a weekly injection to work – whereas an oral form of the medicine would likely be preferred by patients.
More significantly, we don’t yet have data on what happened to the participants after the drug regimen ceased at the end of the trial.
For at least one individual, however, who spoke to The New York Times, her weight began to creep up after the trial was over.
“While drugs like this may prove useful in the short term for obtaining rapid weight loss in severe obesity, they are not a magic bullet for preventing or treating less severe degrees of obesity,” says nutritionist Tom Sanders, an emeritus professor at King’s College London, who wasn’t involved with the study.
“Public health measures that encourage behavioural changes such as regular physical activity and moderating dietary energy intake are still needed.”
Nobody would deny the wisdom of that, but if further analysis of semaglutide turns out to be positive, we could also be looking at an important new pharmaceutical option to help combat obesity.
And that option might arrive sooner than we think.
The study, funded by pharmaceutical company Novo Nordisk – which sells semaglutide as an anti-diabetic medication – is now being tendered as evidence to international health regulatory authorities, in support of an application to market the drug as an obesity treatment.
The US FDA, along with its counterparts in the UK and Europe, is currently assessing the data.
The findings are reported in The New England Journal of Medicine.
ST. BERNARD PARISH – A Louisiana resident went viral on social media after she posted a video explaining how she hasn’t been able to do anything with her hair due to the fact that it’s stuck to her head.
She goes by the name, Tessica, on her Instagram. Three days ago Tessica hit over a million views on Instagram after she claimed that her hair has been stuck in the style of a braided ponytail for about a month after she sprayed Gorilla Glue on her actual hair.
Tessica says she has tried to wash her hair 15 times and even used coconut oil as a last resort, but came out with no luck.
On Saturday she posted a picture of her at the St. Bernard Parish Hospital to receive treatment to undo her mistake.
Tessica’s video has gone viral on Instagram, Twitter, and TikTok with views topping of a million and thousands of comments.
A representative for the actor said on Thursday that Diamond, known for playing Samuel “Screech” Powers on the hit ’90s teen series, has completed his first round of chemotherapy treatment, with another to follow.
“Dustin is looking forward to spending more time with his girlfriend, playing his bass guitar/video games, as well as making videos for his fans on social media,” a statement from Roger Paul, Diamond’s representative, said.
Diamond, who revealed his diagnosis last week, will begin physical therapy soon, Paul added.
Diamond played Screech for more than a decade, appearing in four different incarnations of the “Saved by the Bell” franchise.
Peacock is home to a reboot of the series, which Diamond has not appeared in.
