Scale AI founder Alexandr Wang says he's waiting for Elon Musk's Neuralink before he has kids
And that means he's not having kids anytime soon.
On the Shawn Ryan Show on Thursday, Wang said he wants to wait to have kids until Neuralink or other brain-computer interfaces are available.
Neuralink, one of Elon Musk 's most futuristic endeavors, is developing coin-sized microchips that can be embedded into human brains. These chips will not only be able to record brain activity, but also stimulate it.
Still in clinical trials, Neuralink has so far been embedded in three patients. One of those patients, Brad Smith, who has ALS, said he was able to edit a video using his Neuralink brain chip.
While Neuralink has received a ton of buzz, it's not the only one developing these interfaces. Synchron, backed by Bill Gates and Jeff Bezos, is already working with Apple to help those with disabilities, like ALS patients, use their iPhones. Motif Neurotech is developing a neurostimulator system that works like a pacemaker for the brain and is now used for treating severe depression.
Wang also believes these devices will have profound implications for child development. "In your first like seven years of life, your brain is more neuroplastic than at any other point in your life," he said. "When we get Neuralink and we get these other technologies, kids who are born with them are gonna learn how to use them in like crazy, crazy ways."
Neuroplasticity refers to the brain's ability to adapt and change — whether that means rewiring its structure, shifting how it functions, or forming new connections — in response to things happening inside us or around us.
It's often enhanced in children because the "organization of networks of neuronal synapses as well as white matter pathways remain 'under construction' well into adolescence and even later," according to a 2009 article published in the journal Brain Dev. This is why children are also able to learn new skills quickly and recover from injuries faster.
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Forbes
34 minutes ago
- Forbes
This Secretive Company Built An Empire By Hawking Bad Financial And Health Advice On Facebook
T he roughly fifty-seven minute video, titled 'URGENT WARNING for Americans Born Before 1979', begins with a white-haired man in a checkered shirt sitting in a blurred hallway. He speaks slowly: 'With Donald Trump taking the White House and RFK Jr. now positioned to dismantle the uniparty's corrupt FDA …. multibillion dollar drug conglomerates, some of Big Pharma's oldest and most prominent, will be in a desperate race to flood our nation with poison.' The man urges 'seniors like you and me' to join the online 'Health Sciences Institute,' where they can learn about alleged 'remarkable medical breakthroughs' that the government and pharmaceutical companies 'have been intentionally hiding.' (The Institute's website contains a disclaimer stating that its content should not be interpreted as personal medical advice.) The man's video is one of many of longform advertisements produced by The Agora and its sprawling conglomerate of subsidiaries, which have spent decades cashing in on Americans' growing distrust in government, financial institutions and healthcare providers. The Agora makes its money by selling subscriptions to 'natural health' newsletters that are a vector for its wellness supplements business, as well as investment advice newsletters that can cost thousands of dollars a year. Across the internet, the company spends millions to place its outlandish claims in front of American social media users. A current spate of ads insist that President Trump will soon 'unleash' a $150 trillion 'secret trust' that has been hidden from the American people, and you can get rich from trading on it, if you just subscribe. The Agora has spent at least $500,000 promoting the claim on Meta's platforms alone, according to the Meta ad library. The Agora's business is huge. It says it is a '1.5bn+ company,' and its subsidiaries pour millions each month into advertising on Facebook and Instagram. Agora-related channels have more than 100 million views on YouTube, and the company's top-performing newsletters boast millions of subscribers. The consumer protection group Truth in Advertising estimated that it pulled in approximately $500 million in revenue in 2021 alone. But a Forbes investigation into hundreds of Agora-linked ads, domains, and opaque social media accounts — and interviews with six people who have worked for Agora companies— reveal a company that has found success by misleading vulnerable people. On websites maintained by TrustPilot and the Better Business Bureau, a sprawling network of companies under the Agora umbrella have amassed hundreds of one-star reviews. In them, customers describe spending thousands of dollars on lifetime subscriptions to newsletters that abruptly ended without refunds, and/or losing money by taking the newsletters' investing advice. In one review, a former customer describes 'wasting' more than $15,000 on Agora's products — including a 'special VIP membership' with exclusive newsletter updates, alerts, and videos, that cost more than $9,500. The customer declined to provide more detail when reached for comment. 'This is the perfect storm for a company like Agora to manipulate consumers in the U.S.' Bonnie Patten, Executive Director of Truth In Advertising One former employee of an Agora company said that after a week of onboarding at The Agora's Baltimore offices, she began to worry the company's business was predicated on taking advantage of older people. She said she looked up her own elderly family members in the company's customer database to ensure that none of them were buying its products. For more than three decades, regulators have tried to stop The Agora from engaging in what they have called 'false promises' in its marketing. In 2003, the SEC sued several Agora subsidiaries for advertising insider trading tips, which caused a judge to order $1.5 million in restitution and civil penalties. (The judge found that Agora Inc., the parent company, had not engaged in wrongdoing.) In 2016, a different Agora subsidiary was sued by the attorneys general of Pennsylvania and Oregon for misleading consumers, falsely claiming that they could receive benefits from big tobacco settlements made many years before. The company settled with both states. In 2019, the FTC sued several Agora entities over products that promised a sham cure for Type 2 diabetes, and information about a nonexistent government benefits program. In late 2021, the company settled with the FTC, and was forced to refund nearly 35,000 Americans who had paid for certain products. The refunds totaled more than $2 million. The company did not admit wrongdoing, and told the Baltimore Sun that it was relieved to get back to publishing new content for its customers. Despite regulatory action, The Agora seems to have done little to change its ways — and it's poised to continue its practices at a moment when Trump has gutted consumer protection enforcement, social media companies have taken their proverbial feet off the gas when it comes to taking down misleading messages, and mistrust in institutions is at an all-time high. As Bonnie Patten, Executive Director of the nonprofit consumer protection group Truth in Advertising (TINA), told Forbes , 'This is the perfect storm for a company like Agora to manipulate consumers in the U.S.' The Agora did not respond to repeated requests for interviews or to a request for comment. Bill Bonner and Addison Wiggin, The Agora's founders, have been selling doomsday investment advice since 1979 — the advent of the internet itself. In the early 2000s, the pair co-authored two New York Times bestsellers, Financial Reckoning Day and Empire of Debt . Bonner later became a Business Insider contributor, and Wiggin a Forbes contributor (he has not published since 2015). Today, from a block of swanky rowhomes in downtown Baltimore, the company they built offers more than a hundred investment and 'natural' health-focused newsletters, which are distributed through a network of more than 20 publisher subsidiaries. Bonner and The Agora's current CEO, Erika Nolan, did not respond to interview requests or requests for comment on this story. Wiggin initially responded and scheduled an interview with Forbes, but didn't show up. He then said in an email that he had forwarded our correspondence to the Agora Companies legal team, and did not respond to additional requests for an interview. Many people's first encounters with The Agora start with a Facebook or Instagram ad teasing some shocking new risk or discovery. Its strategy is known as 'direct response copywriting': writing advertisements that convince someone to buy something on the spot. When they click on the ad, they're taken to a page hosting a longform video, known internally at Agora as a VSL, or 'video sales letter,' which can run for an hour or longer. Each features a finance or health 'guru' — a personality that The Agora has cultivated as a face of its operations. The Agora has tapped numerous public figures to become 'gurus' across its lifetime, making use of their reputations to convert potential customers. Agora gurus have included Robert Kiyosaki, the now-bankrupt motivational speaker and author of Rich Dad, Poor Dad ; James Altucher, a cryptocurrency podcaster who founded the former finance social network, StockPickr and authored the financial self-help book Choose Yourself ; and Whitney Tilson, a Democratic candidate for New York City mayor who previously helped found Teach For America. (Kiyosaki, Altucher, and Tilson did not respond to interview requests.) In the Agora playbook, 'gurus' first sell viewers on a modestly priced newsletter subscription. In one current promotion, a balding man in a sport coat sits in front of an American flag and a shelf of gold-leaf lettered books explaining that President Trump can and will run for a third term. (Legally, he cannot.). The man says he's coming forward at great personal risk, to reveal trading secrets that will make you rich, if you sign up for his newsletter for $49.00. Once a guru makes that initial sale, known as the 'front end,' the purchaser starts receiving pitches for Agora's 'back end', which involves opportunities to, say, join a $10,000 exclusive investment club, or attend a $2,000 stock-picking webinar. The 'back end' is where Agora makes its real money. In addition to a panoply of special-offer newsletters and exclusive clubs, Agora subsidiaries also sell dietary supplements and other wellness products, through more than a dozen brands. Offerings include colorfully named cocktails like Memotex (for memory), Virasurge (sexual function) and Ultra Vital Gold (anti-aging). One Agora company also sells a water purifier called Turapür that promises to 'transform average tap water into the antioxidant fountain of energy.' In an Ask Me Anything video on YouTube in 2017, one Agora employee explained how the system works. 'One of the promotions that you may have seen is that a German scientist discovered a cure for cancer in 1944, and the powers that be have hidden it from us until now,' Agora marketing director Matt Lazas explained. 'We use a model of big ideas and longform sales newsletters to acquire the customers, and then on the back end, we have a whole stable of supplements that we monetize those customers on.' Lazas did not respond to a request for comment. The Agora has become a titan of social media, running millions of dollars' worth of ads on Facebook and Instagram from opaque accounts with names like 'America's Secret Fund.' "Over the last few weeks, roughly 30 million ounces of gold have been quietly moved from London vaults to the U.S.,' began one Agora ad running from a Meta page called Freedom Asset Advocates earlier this month. 'My sources tell me this massive gold movement is tied to an upcoming shift in the global monetary system. The kind that happens once every 50-100 years ... I've identified 4 gold mining stocks perfectly positioned to potentially return 100x when this plays out." Meta spokesperson Erin Logan told Forbes that the company is now reviewing a list of Agora-related pages for violations of its policies, and the Ad Library showed that at least 11 pages running ads for Agora subsidiaries have now been 'deleted or unpublished.' On YouTube, a recent Agora ad promised 'a new wave of AI wealth' that one guru said 'will be 14x bigger than chatbots, generative AI, and quantum AI combined.' Placed from a channel with the handle @novileanlifedaily5807 and the title Opportunistic Trader, the ad had been viewed more than 840,000 times. A Google text ad promises a 'proven method' to make '300%+ Stock Gains at 9:30 AM.' Google removed both ads after receiving a Forbes inquiry about them. Both Meta (which owns Facebook and Instagram) and Google (which owns YouTube) have slackened their enforcement of policies banning harmful misinformation in recent months. Meta has removed many of The Agora's ads, and some of the pages and accounts that run them. But it has continued to run hundreds more. Earlier this year, the Wall Street Journal found that Meta rarely severs ties completely with big-money advertisers, and the platform acknowledged that it was facing 'an epidemic of scams' of increasing complexity. The New York Times recently reported that YouTube had relaxed its enforcement of policies that have for years banned harmful misinformation from its platform. In many ways, The Agora's social media strategy mirrors its corporate structure. Today, The Agora says it includes more than 20 companies, though at various points in its 45-year history, it has included even more than that. On LinkedIn, one former staffer described a phenomenon that he deemed 'The Octopus Model.' Operating as a cluster of subsidiaries, rather than a centralized company, drives internal competition, he explained — and it also means that when the company gets in trouble, the trouble often doesn't go all the way to the top. 'Neither Bill Bonner nor Mark Ford [another Agora leader] has ever been arrested or accused of fraud or wrongdoing,' he wrote to conclude his 'Octopus Model' description. Bonnie Patten at Truth In Advertising also commented on Agora's tentacled strategy — but she chose a slightly more colorful creature to compare it to: 'Agora is a multi-headed hydra. They have dozens and dozens of publications that they are using to deceive and manipulate consumers.' In a text message, Wiggin told Forbes that he thought the Octopus Model 'sounds less promising than the 'free market' model Agora historically used.' Asked what The Agora's 'free market' model was, Wiggin sent Forbes Google search results for two economists at George Mason University before acknowledging that neither has ever been affiliated with The Agora. Forbes asked Wiggin about The Agora's specific history with free market principles, to which he replied, 'I probably would [share] but I don't know you.' Wiggin noted that he hadn't been party to the SEC or FTC lawsuits, and said a message from Forbes was the first he'd heard of Truth in Advertising. Agora founder Bill Bonner now conducts much of his business — and the occasional Agora retreat — from a chateau in France that he and his wife bought and remodeled in 2009. Since then, Bonner has hosted copywriting events and Agora staff at the chateau. He opened a library in the house for members of The Agora's 'Oxford Club,' and gives those same members the option to rent out the entire property (prices are only visible to Oxford Club members). In addition to Bonner's chateau, The Agora also maintains a burgeoning empire around the world. In the U.K., it has operated under the subsidiary Southbank Research, offering access to many of the same 'gurus' it advertises in the US. In India, Agora affiliate EquityMaster offers dozens more newsletters with names like Hidden Treasure, Double Income, and Lazy Millionaire, with prices that reach up to more than a hundred thousand rupees. Additional Agora branches offer similar products in Germany, Japan, Ireland, Australia, and France. The company has also purchased and restored more than a dozen historic buildings in Baltimore, though it expressed plans to sell some of those properties early last year. 'If a Democrat's in office, sell on fear; if a Republican's in office, sell on greed.' internal Agora slogan In the U.S. The Agora's business has often dabbled in politics. It has rented the email lists of prominent conservative political influencers like Mike Huckabee and Newt Gingrich, and has also employed at least one conservative political figure directly as a guru: Trumpworld figure Sebastian Gorka was among the newsletter authors for Stansberry Research, a former Agora subsidiary, in 2020 and 2021. Last year, Paradigm Press, another Agora subsidiary, ran ads on Facebook and Instagram depicting Vice President Kamala Harris with devil horns, forcibly injecting children with vaccines. Just days before the election, with voting underway, Agora ads told Americans that actually, the election might be, or might have been, cancelled or postponed — and that, of course, their newsletters would tell you how to capitalize on it. Two former Agora employees recalled a slogan that they said was popular in the company's Baltimore offices: 'if a Democrat's in office, sell on fear; if a Republican's in office, sell on greed.' The Agora's publications have expressed varying opinions on the policies of Donald Trump — from comically positive to apocalyptically negative — but they all seem to agree that Trump is good for content. Not all The Agora's products have a political angle. In addition to its core copywriting business, The Agora also runs an 'administrative services' company, sells 'stock screening' software, , and manages a luxury resort in Nicaragua that offers timeshares, holotropic breathwork workshops, and a 'Path to Vitality Longevity Experience.' In the course of reporting this story, Forbes set up a new email address to sign up for two free Agora newsletters. Several emails sent to the address promised that attendees of a promoted event would learn a 'daily teaspoon secret that crushes' Type 2 diabetes, a 'lost arthritis cure shown to reverse pain in 82% of patients,' and a 'resurrection' cancer treatment ('15 times more powerful than chemotherapy, radiation and surgery'). The event was free, but it upsold attendees on a $2,460 'Masterplan For Fighting Disease.' Another email promotion promised to reveal a 'Natural Cancer Flush' which '[S]imply flushes it out. The cancer starts dying off.' Viewers of this promotion are upsold on an 'Ultimate Cancer-Defeating Protocol' produced by Dr. Richard Gerhauser, and available for just $279. The 'Cancer-Defeating Protocol' is not Gerhauser's first rodeo with The Agora. In fact, it appears to echo another Agora product that ended in disrepute. In 2019, Dr. Gerhauser was sued by the FTC alongside several Agora entities over a different 'protocol': his 'Doctor's Guide to Reversing Diabetes in 28 Days.' At the time, the consumer protection agency's spokesperson said that Gerhauser and Agora 'stole money from older adults with lies,' and the agency ultimately required Agora to issue refunds for 'Doctor's Guide' customers. Forbes contacted the FTC about Gerhauser's 'cancer-defeating protocol,' but it declined to comment on the company's current conduct. Gerhauser did not respond to a request for comment. More from Forbes Forbes How This Chicago Private Equity Firm Scored The Biggest Exit Of 2025 By Hank Tucker Forbes Red States–And AI–Are Big Losers From Trump's Clean Energy Massacre By Christopher Helman Forbes An Arms Dealer Joins Silicon Valley's Military Boom By David Jeans Forbes This Startup Built A Hospital In India To Test Its AI Software By Amy Feldman Forbes Why The U.S. Should Copy Canada To Fix Its Broken Air Traffic Control System By Jeremy Bogaisky
USA Today
an hour ago
- USA Today
ALS Community Files Citizens' Petition asking FDA to Approve NurOwn Stem Cell Therapy
New and Unprecedented Survival, Respiratory, and Biomarker Data Prove that NurOwn Helps People with ALS Live Longer and Live Stronger On July 4, 1939, Lou Gehrig delivered his iconic 'luckiest man' speech, announcing his retirement from the New York Yankees. On that day, ALS ended his Hall of Fame career. Less than two years later, ALS ended his life at just 37 years old. In the last 86 years, the lethal outcome has not changed. ALS is a cruel, paralyzing and 100% fatal disease. But today, the ALS community has hope. A coalition of ALS patients and family members has filed a Citizens' Petition with the FDA, requesting the approval of NurOwn, a neurotrophically-enhanced stem cell therapy. Backed by a decade of real-world data from the NurOwn trials and Expanded Access Program (EAP), the 309-page Citizens' Petition details the unprecedented survival, respiratory, and biomarker data for the FDA's consideration. The new evidence is supported by testimony from top ALS neurologists who were the trial's principal investigators, and the 'totality of the evidence' from the Phase 3 trial. And, it aligns with real-world evidence where trial participants (now-unblinded) and their treating neurologists have proclaimed that NurOwn improves how people with ALS 'feel, function and survive.' NurOwn: A Revolutionary Approach to ALS Treatment Developed by BrainStorm Cell Therapeutics , NurOwn combines the restorative potential of autologous mesenchymal stem cells with the regenerative power of neurotrophic factors, which are like 'Miracle-Gro' for dying motor neurons. NurOwn uses a patient's own stem cells that work like a FedEx truck, delivering nano-packages of neurotrophic factors and immunomodulatory cytokines directly to damaged motor neurons. The results are profound. Within days, trial participants reported halting of symptoms like fasciculations, cramping and clonus; and some improvements in function. With additional doses, the EAP data confirm NurOwn's ability to slow lethal ALS progression, improve function, restore breathing, and extend survival – offering a lifeline to those battling this 100% fatal and paralyzing disease. Unprecedented Survival and Respiratory Data Survival data have long been the gold standard for FDA approvals; and as Commissioner Makary has emphasized: 'gold standard science and common sense' will guide this FDA's decisions. To that end, Petitioners have submitted survival data derived from their own real-world evidence over the past decade. These survival data are unprecedented in ALS clinical trial history. Five-Year Survival: 100% in NurOwn EAP vs. 20% in ALS natural history. All EAP participants (n=10) achieved five-year survival without tracheostomies. Extended Tracheostomy-Free Survival (TFS): 7-year median tracheostomy-free survival (range from 5 to 8.5 years), far surpassing the 2.5- to 3-year median in ALS natural history data. Progression-Free Survival (PFS): When patients received NurOwn, they experienced PFS ranging from a few months up to 17 months. ( See Petition's Emergent Fact section C at pg 19-33). At the FDA Advisory Committee meeting for NurOwn in 2023, Dr. Anthony Windebank of Mayo presented the clinical trial data and shared his expert opinion about the progression-free survival that he and other experienced trial investigators had witnessed – unprecedented in their prolific 40+ year neurology practices: 'I think this data is compelling & it should be approved…. While not everyone responds to the treatment, there are clearly a significant number who do. I have clearly seen some people stabilize in a way that I have never seen in any other trial . In fact, in the small number of people who participated in EAP and received 6-9 treatments, there were people who stabilized while on NurOwn in the trial. In the interval before they were in the EAP – which was over a year or more in some cases – these participants deteriorated, then again stabilized in the additional [EAP] treatment period. There were some who IMPROVED their score. Other investigators who have been working 'hands on' with the participants in the trial have seen similar responses….' Dr. Windebank's testimony underscores the unprecedented impact of NurOwn on people with ALS. And the NurOwn survival data is buttressed by other compelling efficacy data also detailed in the Citizen's Petition: Long-term Preservation of Respiratory Function : A 5- to 8-year delay in the need for non-invasive ventilation (NIV) over a 15-month natural history; and significant stabilization or improvement in Forced Vital Capacity (FVC), both key predictors of ALS survival. ( See Emergent Fact section D at pg 34-44). Long-term Slowing of ALS Progression: Up to an 85% slowing in ALS progression rate, from a trial qualification of a minimum loss of 1 point per month to 0.15 points per month after receiving NurOwn. ( See Emergent Fact section F at pg 44-46). Biomarker Evidence: 23 CSF biomarkers demonstrate statistically significant changes and NurOwn's target engagement across pathways of neuroprotection, neuroinflammation, and neurodegeneration. ( See Fact section M at pgs 156-166). Real-World Evidence and Patient Experiences Our Citizens' Petition also leverages real-world evidence (RWE) and real-world data (RWD) from the EAP and Right to Try – consistent with the Congressional intent of the 21st Century Cures Act. Multiple trial participants testified, submitted Public Comments and shared their RWE, which aligns with the type of efficacy evidence specified in the ALS Guidance Document and 21st Century Cures. At the time of the advisory committee meeting in 2023, many trial participants reported tangible improvements in how they felt and functioned, and hence, an improved quality of life. (See sections H & I, pgs. 91-127). Their testimony was supported by video evidence documenting those improvements and by the opinions from multiple treating neurologists outside the clinical trial. For example, neuromuscular specialist Dr. Danielle Geraldi-Samara submitted a Public Comment to the FDA about what she observed in many of her patients participating in the NurOwn Phase 3 trial and EAP: 'The real world evidence could not be more striking. I have known patients nearly immobile who gained some functionality in their gait, patients with severe dysarthria become intelligible, patients who could not manage the fine motor skill needed to button or zipper, finally able to dress independently. I have patients with solid plateaus [in ALSFRS-R scores] over the course of a year.' Her clinical observations of progression-free survival after the NurOwn trial mirror those of Dr. Windebank and the other investigators during the trial and EAP. Now that the Phase 3 trial has been unblinded, multiple trial participants have confirmation that NurOwn halted their lethal progression and helped some people regain function. Our lived patient experiences now have both validation and vindication. When people are becoming paralyzed, it's common sense that we know when a therapy helps us function. Our lived patient experiences aren't anecdotal hyperbole; they are evidence. And as Commissioner Makary recently said at the Gene and Cell Therapy Forum, there is value in learning from 'n of 1' cases. Combined, the EAP 'n of 10' and the right to try 'n of 1' illustrate compelling and consistent, dose-dependent evidence of efficacy. Reinforcing the efficacy data, Navy pilot Matt Bellina shared the RWE and RWD contained in his VA medical records in his blog and on social media . Matt too experienced unprecedented clinically meaningful improvements after receiving 7 doses of NurOwn via Right to Try. Although he was a slow progressor, diagnosed in 2011, Matt's ALS had progressed significantly. He was choking on food, using NIV to breathe at night; had little use of his hands; and could not stand without assistance. His data are informative, supporting evidence of efficacy because he is the only person in the US who received 6 consecutive doses; because he was the only 'slow progressor' to receive NurOwn; and because his baseline score was 21/48 on the ALS Functional rating scale. Matt's large magnitude, dose-dependent improvement in function was immediate and obvious. ( See section J at pgs 128-133). Matt has video documenting him standing out of a wheelchair unassisted – the first time in two years. He stopped choking on food . He i mproved his functional score by 6 points . His FVC stabilized and he stopped using NIV to breathe for more than 4 years. NurOwn interrupted Matt's lethal trajectory to death. Commissioner Makary has repeatedly offered that the FDA, under President Trump, 'believes in both the spirit and the letter of right to try. ' Thus, Petitioners hope that this FDA will consider and believe the RWE from the very veteran for whom President Trump's Right to Try law was named . Totality of Evidence Methodology for Rare Diseases To determine if a therapy can meet the approval threshold of 'substantial evidence,' the FDA asks if a therapy improves how people 'feel, function or survive.' Regulators look principally at the trial's primary endpoint at one fixed point in time at the end of the trial. But in heterogeneous rare diseases with small populations and short placebo-controlled trials, efficacy signals can be missed. Hence, it's much more likely to result in a Type II statistical error: delaying or denying approval of a drug that does work. In a terminal disease like ALS, Type II errors cause ongoing paralysis and death. Thus, the Citizens' Petition reasserts the propriety of the FDA's use of the 'totality of evidence' statistical methodology to assess NurOwn's efficacy. This approach – widely accepted in oncology for evaluating therapies in heterogeneous, rare populations – strengthens the case for NurOwn's approval by highlighting the consistent benefits in the subgroup of ALS patients earlier in ALS progression (akin to a drug working on stage I and II cancer patients). When including the trial population with the most advanced ALS (akin to stage III/IV cancer), the trial did not meet its endpoints. But when looking at the patients earlier in ALS progression, NurOwn met statistical significance. Using the 'totality of the evidence' methodology, renowned biostatistician and Wilkes Award winner, Dr. Lee-Jen Wei of Harvard / Dana Farber analyzed the multiple trial endpoints, across multiple functional scale domains, at multiple time points throughout the 28-week trial. He testified at the Advisory Committee meeting that these p-values were: 0.045, 0.021, 0.007 and 0.005; thus providing more supporting evidence of NurOwn's efficacy. Meeting FDA Approval Thresholds The Citizens' Petition asserts that NurOwn achieves the statutory thresholds for multiple FDA approval pathways: 1. Traditional Approval NurOwn's survival data, including the five-year survival, TFS, PFS and OS, meet the 'substantial evidence' threshold of one well-controlled trial plus supporting evidence. This conclusion aligns with the FDA's recognition that survival data are the gold standard in FDA approvals. Thus the diversity and magnitude of NurOwn's survival outcomes fulfills both the 'quality' and 'quantity' requirements of 'substantial evidence.' ( See Emergent Fact section C, pgs. 19-33). 2. Accelerated Approval NurOwn meets the 'reasonable likelihood' threshold for accelerated approval. The survival data from the 'n of 10' EAP are 'reasonably likely to predict' a favorable impact on irreversible mortality of the 32,000 people with ALS. This survival data far surpasses survival data supporting the accelerated approval of many cancer therapies. ( See comparison at Memorandum section I, pgs. 191-209 and Exs. A & B). NurOwn's respiratory data, including delays in time-to-tracheostomy, time-to-NIV, and improved FVC, are also reasonably likely to predict a favorable impact on mortality. ( See Emergent Fact section D, pgs. 34-43 and Memorandum II.C pgs. 219-222). NurOwn's CSF biomarker data are also reasonably likely to predict a 'clinically meaningful' effect. NurOwn caused statistically significant changes in first-in-class CSF biomarkers – regardless of disease severity and only in the NurOwn treatment arm. Of the 45 pre-specified biomarkers tested, 23 had statistically significant changes and 15/23 had p-values ≤0.001. These CSF biomarkers provide objective biological evidence of target engagement across pathways of neuroinflammation, neurodegeneration, and neuroprotection. ( See Petition Facts section M, pgs. 156-166 and Memorandum section II.C pgs. 219-222). Additionally, Brainstorm Cell has shared neurofilament light biomarker data in a poster presentation at the 2024 NEALS conference. As the FDA has acknowledged, as ALS progression advances, harmful NfL increases, reflecting more diseased and dying motor neurons. At the end of the Phase 3 trial, there was a 9.4% delta between the NurOwn and placebo arm (p=.037). But in those 10 from EAP who were earlier in progression at the start of the Phase 3 trial, the delta between the NurOwn and placebo arms was more apparent. At the end of Phase 3, the 4/10 on placebo had a 37% increase in harmful NfL whereas the 6/10 on NurOwn had a 4% decrease in NfL. With the additional dosing in EAP, the 4/10 in the placebo-crossover group finally experienced a 5% decline in harmful NfL, whereas people on NurOwn maintained a 36% decrease from baseline. Not surprisingly, those who received the most doses of NurOwn and received it earliest in ALS progression had the largest magnitude functional changes and as well as the largest decrease in NfL levels – with two people who received 9 total doses having a decrease of ≥60% in harmful NfL levels. ( See table in section II.C.2.a on page 222). As such, the changes in CSF biomarkers are reasonably likely to predict a clinically meaningful benefit, and thus, the third way that NurOwn can meet the threshold for accelerated approval. 3. Conditional Approval NurOwn aligns with Commissioner Makary's proposed 'plausible mechanism of action' threshold for conditional approval. Both stem cell technology and neurotrophic factors are plausible mechanisms of action in ALS; and NurOwn's CSF biomarker data confirms biological plausibility. ( See Petition Fact section N, pgs. 173-176 and Memorandum section II.G at pgs. 234-241). A Call for De Novo Review and Expedited Action The Citizens' Petition requests a de novo review by the FDA. The Center for Biologics Evaluation and Research (CBER) has not ever considered the EAP survival, respiratory, or biomarker data, nor has it considered the Right to Try data from Navy pilot Matt Bellina, nor the unblinded and now corroborated RWE/RWD from people who have benefitted from NurOwn since 2011. The Petitioners also request that CBER use the Commissioner's new Priority Voucher to expedite review. The Citizens' Petition also proposes that FDA consider the far-reaching benefits of a Phase 4 post-marketing study, including a biorepository and natural history/exposome database, which aligns with the FDA Priorities outlined by Doctors Makary and Prasad. A Historic Moment for the ALS Community At the recent 2025 Gene and Cell Therapy Forum , Secretary Kennedy shared that the FDA will do everything it can to 'accelerate approvals for rare diseases.' And in their Joint OpEd for JAMA Viewpoints , Commissioner Makary and Director Prasad said the FDA is committed to 'rapidly usher to market new products with transformational potential.' In furtherance of that commitment, Director Prasad told the rare disease community that the FDA will: 'approve anything that is an incremental advancement' accelerate therapies by 'taking action at the first sign of promise for rare diseases' and at the 'earliest sign of statistical evidence' monitor people post-market to 'ensure people live longer, stronger.' The Citizens' Petition argues that NurOwn has more than transformational potential . Rather, the survival and respiratory data, along with 8 years of RWE, demonstrate its already transformational impact on people living with ALS. Thus, the ALS community calls on the FDA to approve NurOwn, honoring its commitment to marry 'gold standard science and common sense.' ALS is stealing decades from our lifespans. Just as the FDA acts with urgency for people with terminal cancer, the Citizens' Petition asks the FDA to act with the same urgency as ALS is killing our motor neurons. Please don't let another generation of people with ALS die waiting when we know a stem cell therapy can help us live. About ALS ALS is a 100% fatal, heterogeneous, rare neurodegenerative disease. As motor neurons die, the brain can no longer communicate with the voluntary muscles, which slowly become paralyzed. For reasons researchers don't fully understand, ALS impacts only the motor neurons, not the sensory neurons. Thus, people with ALS still feel cramping, sensations, fasciculations and pain, but they can't move to respond to them. Ultimately, people lose the ability to walk, talk, move, eat, drink, swallow, and eventually, breathe. About the Petitioners The Petitioners are a coalition of people who received NurOwn and others with ALS who could not. We are committed to advancing research, treatment access, and policy changes for ALS. Petitioners: Nicholas Warack, Esq. Matt Klingenberg – Phase 3 & EAP Eric Stevens – Phase 3 & EAP Joshua Smith – Phase 3 & EAP Estate of Roberto Muggli – Phase 3 & EAP Lesley Krummel – Phase 3 Estate of Kade Simons – Phase 3 Estate of Justin Rogers – Phase 3 Terri Pickering Saenz – Phase 2 Tara Collazo Mayuri Saxena Estate of Jamie Rose Berry Estate of Patricia Manhardt Shahriar Minokadeh, MD Contact: SOURCE: NurOwn Citizen's Petition View the original press release on ACCESS Newswire
Associated Press
an hour ago
- Associated Press
ALS Community Files Citizens' Petition asking FDA to Approve NurOwn Stem Cell Therapy
New and Unprecedented Survival, Respiratory, and Biomarker Data Prove that NurOwn Helps People with ALS Live Longer and Live Stronger BREMERTON, WA / ACCESS Newswire / July 7, 2025 / On July 4, 1939, Lou Gehrig delivered his iconic 'luckiest man' speech, announcing his retirement from the New York Yankees. On that day, ALS ended his Hall of Fame career. Less than two years later, ALS ended his life at just 37 years old. In the last 86 years, the lethal outcome has not changed. ALS is a cruel, paralyzing and 100% fatal disease. But today, the ALS community has hope. A coalition of ALS patients and family members has filed a Citizens' Petition with the FDA, requesting the approval of NurOwn, a neurotrophically-enhanced stem cell therapy. Backed by a decade of real-world data from the NurOwn trials and Expanded Access Program (EAP), the 309-page Citizens' Petition details the unprecedented survival, respiratory, and biomarker data for the FDA's consideration. The new evidence is supported by testimony from top ALS neurologists who were the trial's principal investigators, and the 'totality of the evidence' from the Phase 3 trial. And, it aligns with real-world evidence where trial participants (now-unblinded) and their treating neurologists have proclaimed that NurOwn improves how people with ALS 'feel, function and survive.' NurOwn: A Revolutionary Approach to ALS Treatment Developed by BrainStorm Cell Therapeutics, NurOwn combines the restorative potential of autologous mesenchymal stem cells with the regenerative power of neurotrophic factors, which are like 'Miracle-Gro' for dying motor neurons. NurOwn uses a patient's own stem cells that work like a FedEx truck, delivering nano-packages of neurotrophic factors and immunomodulatory cytokines directly to damaged motor neurons. The results are profound. Within days, trial participants reported halting of symptoms like fasciculations, cramping and clonus; and some improvements in function. With additional doses, the EAP data confirm NurOwn's ability to slow lethal ALS progression, improve function, restore breathing, and extend survival - offering a lifeline to those battling this 100% fatal and paralyzing disease. Unprecedented Survival and Respiratory Data Survival data have long been the gold standard for FDA approvals; and as Commissioner Makary has emphasized: 'gold standard science and common sense' will guide this FDA's decisions. To that end, Petitioners have submitted survival data derived from their own real-world evidence over the past decade. These survival data are unprecedented in ALS clinical trial history. (See Petition's Emergent Fact section C at pg 19-33). At the FDA Advisory Committee meeting for NurOwn in 2023, Dr. Anthony Windebank of Mayo presented the clinical trial data and shared his expert opinion about the progression-free survival that he and other experienced trial investigators had witnessed - unprecedented in their prolific 40+ year neurology practices: 'I think this data is compelling & it should be approved…. While not everyone responds to the treatment, there are clearly a significant number who do. I have clearly seen some people stabilize in a way that I have never seen in any other trial. In fact, in the small number of people who participated in EAP and received 6-9 treatments, there were people who stabilized while on NurOwn in the trial. In the interval before they were in the EAP - which was over a year or more in some cases - these participants deteriorated, then again stabilized in the additional [EAP] treatment period. There were some who IMPROVED their score. Other investigators who have been working 'hands on' with the participants in the trial have seen similar responses....' Dr. Windebank's testimony underscores the unprecedented impact of NurOwn on people with ALS. And the NurOwn survival data is buttressed by other compelling efficacy data also detailed in the Citizen's Petition: Real-World Evidence and Patient Experiences Our Citizens' Petition also leverages real-world evidence (RWE) and real-world data (RWD) from the EAP and Right to Try - consistent with the Congressional intent of the 21st Century Cures Act. Multiple trial participants testified, submitted Public Comments and shared their RWE, which aligns with the type of efficacy evidence specified in the ALS Guidance Document and 21st Century Cures. At the time of the advisory committee meeting in 2023, many trial participants reported tangible improvements in how they felt and functioned, and hence, an improved quality of life. (See sections H & I, pgs. 91-127). Their testimony was supported by video evidence documenting those improvements and by the opinions from multiple treating neurologists outside the clinical trial. For example, neuromuscular specialist Dr. Danielle Geraldi-Samara submitted a Public Comment to the FDA about what she observed in many of her patients participating in the NurOwn Phase 3 trial and EAP: 'The real world evidence could not be more striking. I have known patients nearly immobile who gained some functionality in their gait, patients with severe dysarthria become intelligible, patients who could not manage the fine motor skill needed to button or zipper, finally able to dress independently. I have patients with solid plateaus [in ALSFRS-R scores] over the course of a year.' Her clinical observations of progression-free survival after the NurOwn trial mirror those of Dr. Windebank and the other investigators during the trial and EAP. Now that the Phase 3 trial has been unblinded, multiple trial participants have confirmation that NurOwn halted their lethal progression and helped some people regain function. Our lived patient experiences now have both validation and vindication. When people are becoming paralyzed, it's common sense that we know when a therapy helps us function. Our lived patient experiences aren't anecdotal hyperbole; they are evidence. And as Commissioner Makary recently said at the Gene and Cell Therapy Forum, there is value in learning from 'n of 1" cases. Combined, the EAP 'n of 10" and the right to try 'n of 1" illustrate compelling and consistent, dose-dependent evidence of efficacy. Reinforcing the efficacy data, Navy pilot Matt Bellina shared the RWE and RWD contained in his VA medical records in his blog and on social media. Matt too experienced unprecedented clinically meaningful improvements after receiving 7 doses of NurOwn via Right to Try. Although he was a slow progressor, diagnosed in 2011, Matt's ALS had progressed significantly. He was choking on food, using NIV to breathe at night; had little use of his hands; and could not stand without assistance. His data are informative, supporting evidence of efficacy because he is the only person in the US who received 6 consecutive doses; because he was the only 'slow progressor' to receive NurOwn; and because his baseline score was 21/48 on the ALS Functional rating scale. Matt's large magnitude, dose-dependent improvement in function was immediate and obvious. (See section J at pgs 128-133). Matt has video documenting him standing out of a wheelchair unassisted - the first time in two years. He stopped choking on food. He i mproved his functional score by 6 points. His FVC stabilized and he stopped using NIV to breathe for more than 4 years. NurOwn interrupted Matt's lethal trajectory to death. Commissioner Makary has repeatedly offered that the FDA, under President Trump, 'believes in both the spirit and the letter of right to try. " Thus, Petitioners hope that this FDA will consider and believe the RWE from the very veteran for whom President Trump's Right to Try law was named. Totality of Evidence Methodology for Rare Diseases To determine if a therapy can meet the approval threshold of 'substantial evidence,' the FDA asks if a therapy improves how people 'feel, function or survive.' Regulators look principally at the trial's primary endpoint at one fixed point in time at the end of the trial. But in heterogeneous rare diseases with small populations and short placebo-controlled trials, efficacy signals can be missed. Hence, it's much more likely to result in a Type II statistical error: delaying or denying approval of a drug that does work. In a terminal disease like ALS, Type II errors cause ongoing paralysis and death. Thus, the Citizens' Petition reasserts the propriety of the FDA's use of the 'totality of evidence' statistical methodology to assess NurOwn's efficacy. This approach - widely accepted in oncology for evaluating therapies in heterogeneous, rare populations - strengthens the case for NurOwn's approval by highlighting the consistent benefits in the subgroup of ALS patients earlier in ALS progression (akin to a drug working on stage I and II cancer patients). When including the trial population with the most advanced ALS (akin to stage III/IV cancer), the trial did not meet its endpoints. But when looking at the patients earlier in ALS progression, NurOwn met statistical significance. Using the 'totality of the evidence' methodology, renowned biostatistician and Wilkes Award winner, Dr. Lee-Jen Wei of Harvard / Dana Farber analyzed the multiple trial endpoints, across multiple functional scale domains, at multiple time points throughout the 28-week trial. He testified at the Advisory Committee meeting that these p-values were: 0.045, 0.021, 0.007 and 0.005; thus providing more supporting evidence of NurOwn's efficacy. Meeting FDA Approval Thresholds The Citizens' Petition asserts that NurOwn achieves the statutory thresholds for multiple FDA approval pathways: 1. Traditional Approval NurOwn's survival data, including the five-year survival, TFS, PFS and OS, meet the 'substantial evidence' threshold of one well-controlled trial plus supporting evidence. This conclusion aligns with the FDA's recognition that survival data are the gold standard in FDA approvals. Thus the diversity and magnitude of NurOwn's survival outcomes fulfills both the 'quality' and 'quantity' requirements of 'substantial evidence.' (See Emergent Fact section C, pgs. 19-33). 2. Accelerated Approval NurOwn meets the 'reasonable likelihood' threshold for accelerated approval. The survival data from the 'n of 10" EAP are 'reasonably likely to predict' a favorable impact on irreversible mortality of the 32,000 people with ALS. This survival data far surpasses survival data supporting the accelerated approval of many cancer therapies. (See comparison at Memorandum section I, pgs. 191-209 and Exs. A & B). NurOwn's respiratory data, including delays in time-to-tracheostomy, time-to-NIV, and improved FVC, are also reasonably likely to predict a favorable impact on mortality. (See Emergent Fact section D, pgs. 34-43 and Memorandum II.C pgs. 219-222). NurOwn's CSF biomarker data are also reasonably likely to predict a 'clinically meaningful' effect. NurOwn caused statistically significant changes in first-in-class CSF biomarkers - regardless of disease severity and only in the NurOwn treatment arm. Of the 45 pre-specified biomarkers tested, 23 had statistically significant changes and 15/23 had p-values ≤0.001. These CSF biomarkers provide objective biological evidence of target engagement across pathways of neuroinflammation, neurodegeneration, and neuroprotection. (See Petition Facts section M, pgs. 156-166 and Memorandum section II.C pgs. 219-222). Additionally, Brainstorm Cell has shared neurofilament light biomarker data in a poster presentation at the 2024 NEALS conference. As the FDA has acknowledged, as ALS progression advances, harmful NfL increases, reflecting more diseased and dying motor neurons. At the end of the Phase 3 trial, there was a 9.4% delta between the NurOwn and placebo arm (p=.037). But in those 10 from EAP who were earlier in progression at the start of the Phase 3 trial, the delta between the NurOwn and placebo arms was more apparent. At the end of Phase 3, the 4/10 on placebo had a 37% increase in harmful NfL whereas the 6/10 on NurOwn had a 4% decrease in NfL. With the additional dosing in EAP, the 4/10 in the placebo-crossover group finally experienced a 5% decline in harmful NfL, whereas people on NurOwn maintained a 36% decrease from baseline. Not surprisingly, those who received the most doses of NurOwn and received it earliest in ALS progression had the largest magnitude functional changes and as well as the largest decrease in NfL levels - with two people who received 9 total doses having a decrease of ≥60% in harmful NfL levels. (See table in section II.C.2.a on page 222). As such, the changes in CSF biomarkers are reasonably likely to predict a clinically meaningful benefit, and thus, the third way that NurOwn can meet the threshold for accelerated approval. 3. Conditional Approval NurOwn aligns with Commissioner Makary's proposed 'plausible mechanism of action' threshold for conditional approval. Both stem cell technology and neurotrophic factors are plausible mechanisms of action in ALS; and NurOwn's CSF biomarker data confirms biological plausibility. (See Petition Fact section N, pgs. 173-176 and Memorandum section II.G at pgs. 234-241). A Call for De Novo Review and Expedited Action The Citizens' Petition requests a de novo review by the FDA. The Center for Biologics Evaluation and Research (CBER) has not ever considered the EAP survival, respiratory, or biomarker data, nor has it considered the Right to Try data from Navy pilot Matt Bellina, nor the unblinded and now corroborated RWE/RWD from people who have benefitted from NurOwn since 2011. The Petitioners also request that CBER use the Commissioner's new Priority Voucher to expedite review. The Citizens' Petition also proposes that FDA consider the far-reaching benefits of a Phase 4 post-marketing study, including a biorepository and natural history/exposome database, which aligns with the FDA Priorities outlined by Doctors Makary and Prasad. A Historic Moment for the ALS Community At the recent 2025 Gene and Cell Therapy Forum, Secretary Kennedy shared that the FDA will do everything it can to 'accelerate approvals for rare diseases.' And in their Joint OpEd for JAMA Viewpoints, Commissioner Makary and Director Prasad said the FDA is committed to 'rapidly usher to market new products with transformational potential.' In furtherance of that commitment, Director Prasad told the rare disease community that the FDA will: The Citizens' Petition argues that NurOwn has more than transformational potential. Rather, the survival and respiratory data, along with 8 years of RWE, demonstrate its already transformational impact on people living with ALS. Thus, the ALS community calls on the FDA to approve NurOwn, honoring its commitment to marry 'gold standard science and common sense.' ALS is stealing decades from our lifespans. Just as the FDA acts with urgency for people with terminal cancer, the Citizens' Petition asks the FDA to act with the same urgency as ALS is killing our motor neurons. Please don't let another generation of people with ALS die waiting when we know a stem cell therapy can help us live. About ALS ALS is a 100% fatal, heterogeneous, rare neurodegenerative disease. As motor neurons die, the brain can no longer communicate with the voluntary muscles, which slowly become paralyzed. For reasons researchers don't fully understand, ALS impacts only the motor neurons, not the sensory neurons. Thus, people with ALS still feel cramping, sensations, fasciculations and pain, but they can't move to respond to them. Ultimately, people lose the ability to walk, talk, move, eat, drink, swallow, and eventually, breathe. About the Petitioners The Petitioners are a coalition of people who received NurOwn and others with ALS who could not. We are committed to advancing research, treatment access, and policy changes for ALS. Petitioners: Contact: SOURCE: NurOwn Citizen's Petition press release
