Author: nurown citizens petition

The Real-World Evidence in the Citizens’ Petition Supports the Accelerated Approval of the Stem Cell Therapy that Was Helping People with ALS Live Longer and Breathe Better

NEW YORK CITY, NY / ACCESS Newswire / July 14, 2025 / Every neurologist who specializes in neuromuscular medicine remembers their first patient… the first time you have to deliver an ALS diagnosis. How do you tell someone that they will lose the ability to walk, talk, move, eat, drink, swallow, and eventually, breathe? There is no easy way to tell someone that ALS is always fatal and there are no disease-modifying treatments.

In every physician’s heart there is that first patient that you always remember. And then another patient comes along and imprints on your soul. For me, after 15 years of practice, that patient was Kade Simons. I share his story jointly and with the permission of his mother.

Kade Simons died of ALS on August 4, 2024 – just a week before his 27th birthday. His passing was the most devastating in all my years as a neurologist. Some patients just settle in your heart, make a home there and never leave. Kade did that.

Kade, his mom Kandy and I met at our first telehealth appointment in December of 2022. Right away, Kade made it clear that he didn’t care much for doctors. But somehow we clicked. He saw I was wearing black nail polish and this resonated with him, so he took a liking to me. At the next appointment, my nails were no longer black. With his mischievous grin and his twinkling blue eyes, he gave me a little side-eye. It made me think about those small things in life that we, as humans, find connection in. In hindsight, I realize just why he liked my nail polish and it crushes my soul. Why? Because he was a mere 6 years older than my own daughter. SIX YEARS. He looked at me the way my own children look at me when maybe I am a little on trend and they find me relatable. And now I realize why Kade took a special place in my spirit; because he was a child and this tore at my maternal core.

No mother should ever have to bury a child. I wish that everyone on the NurOwn Advisory Committee two years ago would have been forced to look into the eyes of a vibrant 20-something year-old and reconcile that they ignored his compelling testimony. That is the testimony Kade and Kandy shared with me.

Kade’s Experience with NurOwn
At that first appointment, I sat in amazement as Kandy and Kade shared the story about Kade’s participation in the NurOwn stem cell trial at Mayo. Kade received 3 doses of NurOwn from July to November of 2019. Although the trial had not yet been unblinded, Kade and his mom were adamant that he was in the treatment arm.

Kade felt NurOwn work almost immediately. He told me that his fasciculations stopped. They showed me videos. After his first dose, he felt his foot “popping up” when he walked. Translation: his foot drop was improving as his brain’s messages were getting delivered again to his foot muscles. Kandy then narrated a video where Kade exclaimed that he was having to get used to the renewed strength in his legs that he hadn’t experienced in a while. With every dose of NurOwn, Kade felt stronger, walked easier, had more range of motion, less spasticity, clonus and cramping. His physical therapist agreed.

The more we talked, the more I became intrigued. Kade had ALS onset in three regions: bulbar and both upper and lower limbs. Consistent with his multi-loci onset and ALS natural history, Kade was initially a fast progressor. But his medical records showed that his lethal progression abruptly slowed after he started receiving NurOwn.

And so important and “clinically meaningful” to any 24-year-old, Kade was still able to use his hands to text and to play video games with his brother and friends – maintaining that social connection with his peers. In my professional experience, all of this was extremely unusual for someone four years post-diagnosis.

Kade boasted that he was still able to eat pizza and Jersey Mike’s subs – something that both the neurologist and the Italian girl in me appreciated. Eating without limitations four years after diagnosis is highly unusual in ALS. But it’s almost unheard of in someone who began their ALS journey with bulbar onset.

But most of all, I was astounded that Kade was still breathing on his own without the assistance of non-invasive ventilation. He was sleeping laying flat (something that doesn’t happen when the nerves innervating your diaphragm muscles start to die). Kade’s pulmonologist agreed about the rarity of Kade’s normal respiratory function.

Kade had been a college baseball player. As an athlete, Kade made quite clear to me that he knew his body. He knew when it worked and he knew when it didn’t. He was insulted that people would doubt when he said NurOwn improved how he functioned. Kandy too said, “I watched Kade improve on NurOwn.” Just as Kade knew his body, a mom knows her child.

Kade asked me if I believed them. I did.

In the months after that first appointment with Kade, I had the chance to meet with several other patients in the NurOwn trial. Many of these patients reported similar improvements and their medical records mirrored those experiences. The more patients I met, the more records I reviewed, the more I believed in NurOwn’s efficacy. The people who thought they were in the placebo arm (or were non-responders) had medical records that reflected their decline; but those who were as adamant as Kade that they were in the NurOwn arm had medical records that reflected a stark contrast.

My Expert Opinion as a Neuromuscular Specialist with Documented RWE
I promised Kade – in life – that I would try my best to advocate for him and all others brutalized by this disease.

When it came time for the NurOwn Advisory Committee meeting in 2023, I was compelled to share, not just my clinical observations outside the trial (evidence the FDA calls “real-world evidence”), but also my expert opinion as a neuromuscular specialist who has treated well over a thousand people with ALS. Because of my tele-neurology practice, I suspected I had seen more NurOwn recipients than any other neurologists besides the trials’ investigators. And uniquely, I saw them years after the 28-week trial ended. Thus, my observations could provide clinical evidence about the durability of my patients’ responses on NurOwn.

I submitted a Public Comment to the FDA sharing my professional opinion that NurOwn worked on some people.

“I have been working in the ALS clinical space and in ALS multidisciplinary clinics for over 15 years….
I have seen the full breadth of clinical constellations playing out over time. What I have not seen, though,
is anyone with significant functional improvement from a declining baseline;
I have not seen patients rise from plateaus. It does not happen in the natural course of ALS.
It did happen with the introduction of NurOwn.
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 implored the FDA to consider the risk of a Type II error – failing to approve a drug that does work.

“I implore you to open your minds and to take my clinical observations and this real world evidence
just as you would any solid data set from a trial. Consider this evidence as if it were your patient
you observed in your own clinic…. Not all evidence manifests in the confines of a trial.
Listen to the narratives of these patients and their physicians. NurOwn works.
The science of the trial, the art of our clinical observations and attention to our patients’ accounts
demonstrates the truth of this statement.… Please allow this treatment to come to market
with a phase 4 post-marketing trial. Let other neurologists witness what I have seen in my own patients.
Let thousands more patients have a chance to benefit from NurOwn.”

I knew death would be imminent for so many of my patients if they couldn’t get more NurOwn. They had already defied the odds. I knew the ALS clock wouldn’t wait five more years for another Phase 3 trial. Kade had already lived nearly four years past his last dose of NurOwn in 2019.

The Citizens’ Petition
Kade was strong, determined, and courageous, and so too is his mother Kandy-who, despite her grief over the loss of her son, is a Petitioner in the recent Citizens’ Petition. This 309-page legal document seeks accelerated approval of NurOwn based on powerful survival and respiratory data, along with real-world evidence from the now-unblinded trial participants.

The Citizens’ Petition relies upon the promise of the 21st Century Cures Act, wherein Congress encouraged the FDA to use real-world evidence and patient experiences – data from actual patients like Kade and the EAP participants. In 100% terminal and heterogeneous rare diseases, every story matters and every type of evidence matters.

When the trial was unblinded after the Advisory Committee meeting, I was not surprised to learn that – almost without exception – every one of my patients accurately predicted if they received NurOwn or placebo. As the Citizens’ Petition states:

“When people are dying, they know when a drug helps them live.
When people are becoming paralyzed, they know when a drug helps them move again.
And when people can’t breathe without a non-invasive ventilator,
they know when a drug helps them breathe again.”

The new data in the Citizens’ Petition illustrates that the FDA’s focus on the primary endpoint alone overlooked the totality of the evidence, and I believe resulted in a Type II statistical error. The totality of the evidence included: the data of responders earlier in progression, objective biomarker data, compelling patient experiences, neurologists’ observations, and expert opinions, like those detailed in the 309-page Citizens’ Petition. For example, the FDA failed to heed the opinions of esteemed ALS clinical trialists with four decades of experience like Dr. Robert Brown of UMass Chan School of Medicine and Dr. Anthony Windebank of Mayo Clinic. They opined that a “significant number” of people had a “clinically meaningful” response to NurOwn, that it caused progression-free survival in some, and even helped some regain function – just as Kade told me.

Although Kade was not chosen to receive additional doses in the Expanded Access Program (EAP), his data too is informative and it was included in the Citizens’ Petition. I can corroborate Kade’s submitted data:

Kade had symptom onset in three regions concurrently. Kade was a fast progressor, losing 1 point per month
before receiving NurOwn. Kade’s first bulbar symptoms occurred in May of 2018…. By the time Kade received
NurOwn in July of 2019, his ALSFRS-R score had plummeted to 34…. NurOwn caused the most durable impact
on Kade’s respiratory and bulbar function…. Kade didn’t get a feeding tube until February of 2024, and even then
he still continued to eat things like pizza and Jersey Mike’s Subs up until a few months before his death in
August of 2024. Most impressively, Kade did not use NIV up until the time of his death.

In addition to the long-term data from people in the Phase 3 trial and EAP, the Citizens’ Petition also submitted the following unprecedented survival and respiratory data:

• 100% five-year survival in EAP vs. 20% natural history
  (p-value = ~0.000335 (one-sided) or ~0.000671 (two-sided)

• EAP Median ≈ 7 year Tracheostomy-free survival (60-103 month range) vs. 30 month median in ALS natural history

• Progression-free survival (range up to 17 months)

• Long-term slowing of ALS progression of up to 85%

• Halting of respiratory decline

• Range of 5-8+ years in time-to-NIV vs. 15-27 months natural history

Notably, comparing the above survival data to exhibit A & B in the Petition, the NurOwn survival data meets or beats the survival data supporting the accelerated approval of dozens of cancer therapies.

The Citizens’ Petition also cites multiple studies that conclude that almost no one with multi-loci ALS onset survives five years. But Kade did.

Kade beat the bulbar onset natural history data as well. On pages 29-32, the Petition reports that the median lifespan for people with bulbar onset is approximately 2 years from symptom onset and 1.5 years from diagnosis. This is consistent with my professional experience. The Petition reports that NurOwn recipients who had bulbar onset lived approximately 3.5 years longer than the median in ALS natural history.

Applying these above peer-reviewed criteria to Kade, I can confirm, as Kade’s treating neurologist, that:

• Kade Lived “trach-free” 78 months (6.5 years) from symptom onset vs. the 2-year average for bulbar onset

• Kade’s progression slowed from more than 1 point per month on the ALSFRS-R down to 0.16 points per month

• Kade chose not to use a bi-pap to breathe and thus at the time of his death (68 months from diagnosis), Kade’s time-to-NIV dwarfed the ALS natural history of 15 months for fast progressors and the 13.5 months for people with bulbar onset.

As Kade’s treating neurologist, these data align with the Public Comment that I submitted in 2023. They also align with Kandy’s Public Comment and testimony that she shared at the NurOwn Advisory Committee meeting. These data in the Citizens’ Petition further strengthen my expert opinion and resolve that NurOwn helped Kade breathe better and live longer.

It is a tragedy unlike anything I have experienced in my career that Kade was denied continued access to the stem cell therapy that was helping him live.

Gold Standard Science and Common Sense Support Approval
I am heartened to read that Commissioner Makary is committed to marry gold standard science and common sense.

As such, I implore the FDA to consider the new and unprecedented survival and respiratory data that is reasonably likely to predict a clinically meaningful impact. Listen to the opinions of the neurologists who treated the patients. But most of all, listen to the patients. They are the only ones who know what it feels like to die a little more each day. And the new data proves that they were right. They knew when NurOwn helped them live.

Thus, substantial evidence supports the approval of NurOwn. Please approve NurOwn with a Phase 4 post-marketing study. Please show my patients with ALS the same compassion and humanity that the Oncology Center of Excellence shows cancer patients.

My Promise to Kade
Today, with this Press Release, I renew my promise to Kade. As I did during his life, I will continue to do after his death. For whatever number of years God blesses me to continue helping people with ALS, I will move forward with the same resolve that Kade and his family have shown.

Please hold space in your heart today for the 26-year-old man with the resolve and courage that I could never fathom possessing.
God rest him.

About Danielle Geraldi-Samara, MD
For over 15 years, Dr. Geraldi-Samara has diagnosed and treated people with ALS. She attended medical school at the State University of New York at Syracuse and completed her residency and neuromuscular fellowship at the Mount Sinai School of Medicine. With the goal of bringing ALS care closer for local patients, Dr. Geraldi-Samara helped establish an ALS/Neuromuscular clinic from the ground up at NYU-Brooklyn. She also served as a supervising Neuromuscular Specialist at Northwell Health’s multidisciplinary ALS clinic in New York.

Dr. Geraldi-Samara counts patient relationships as the driving force in her career. Because of the inequity in ALS regulatory policy and law – and Kade’s inability to get more of the drug that was helping him live – Dr. Geraldi has resumed her academic career, where she is currently working on a master’s degree in bioethics. While continuing her neuromuscular practice, she also wants to work to transform regulatory policy for patients with terminal rare diseases like ALS.

About Kandy Simons
Kandy Simons is the mother of Kade Simons. She has become a patient advocate to right the wrongs that were inflicted upon her son and her family. She lives in Oklahoma City, Oklahoma with her husband Mitchel Simons as well as her only remaining child, Kruz Simons.

About NurOwn
Developed by Brainstorm Cell Therapeutics, NurOwn is a mesenchymal stem cell therapy marries the restorative powers of stem cells with the regenerative powers of neurotrophic factors. NurOwn is made from your body’s own stem cells harvested from a bone marrow aspiration. They are then treated in a lab and supercharged to release neurotrophic factors. Once injected directly into the CSF, your stem cells work like a Fed Ex truck, delivering nano-packages of neurotrophic factors and immunomodulatory cytokines directly to the sites of the motor neurons damaged by ALS. The neurotrophic factors act like “Miracle-Gro” for your neurons.

Contact :

1. Danielle Geraldi-Samara, MD
   (mail to: DGSneuro@gmail.com)

2. Nicholas Warack, Esq.
   (mail to: Veterans4NurOwn@gmail.com)

3. Mitze Klingenberg, BSN RN
   (mail to: NurOwnWorks@gmail.com)

Copy of Citizens’ Petition and Exhibits Filed with the FDA:
Join Dr. Geraldi, Kandy Simons and many others in urging the FDA to approve NurOwn for people with ALS. The 309-page Citizens’ Petition includes the data supporting approval as well as easy-to-read charts and graphs that show how NurOwn extended lifespan and improved function for people with ALS. You can see the data and graphics, as well as Petitioners’ stories at: https://www.nurownworks.com/

SOURCE: NurOwn Citizen’s Petition

View the original press release on ACCESS Newswire

Matt Bellina Regained Function After Receiving NurOwn via the Right to Try Law He Fought to Pass and Now He is Fighting for All Americans with ALS to Get the Same Chance He Did

BREMERTON, WA / ACCESS Newswire / July 11, 2025 / The Backstory

Twenty-two years ago, Matt Bellina and Jamie Warack, along with a group of ten college midshipmen, checked onto a destroyer in Yokosuka, Japan. They set off on a navy summer cruise to visit a war memorial in Busan, South Korea. As with many experiences in the navy, they left each other after a month with shared experiences and memories, not knowing if their paths would ever cross again. Both Matt and Jamie went on to become naval aviators: Matt on the EA-6b Prowler, and Jamie on the P3 Orion. But neither Matt nor Jamie could have imagined they would meet again through the shared heartbreak of ALS.

Matt was diagnosed with ALS in 2011. Jamie’s husband, Nick, also a P3 Orion pilot, was diagnosed with ALS in the fall of 2020.

ALS is a cruel disease. As motor neurons die, the brain can no longer communicate with the voluntary muscles, which slowly become paralyzed. Ultimately, people lose the ability to walk, talk, move, eat, drink, swallow, and eventually, breathe. These two naval airmen who had once controlled our country’s most elite aircraft would soon lose the ability to control their own bodies.

As only the few unlucky people who have navigated this diagnosis can know, Nick and Jamie waffled through feelings of despair and hopelessness, but hung onto every word the doctors said, hoping for a change in tone or the possibility that they got the diagnosis wrong, hoping for any new drug that could slow the lethal march to death.

Hope was soon to come in an unexpected way, not from a doctor’s office. Shortly after his diagnosis, Nick learned of a Navy pilot from Whidbey Island who had become a powerful advocate for ALS patients, especially for the Right to Try law and a stem cell treatment called NurOwn. And then came the day that hope was restored. Nick showed Jamie a video of that same man with ALS standing up from his wheelchair for the first time in two years.

Even then, Jamie didn’t realize the man in the wheelchair was the same Matt Bellina she’d known from that summer decades before. It wasn’t until Nick publicly shared his diagnosis on social media that the connection clicked. Matt, already years into his own battle with ALS, commented on Nick’s post, offering prayers and heartfelt support. Tucked away in boxes were photos of the much younger Matt and Jamie. The young Matt in those photos was the same naval aviator, Matt Bellina, who stood up out of his wheelchair.

That moment, that unexpected connection, and Matt’s journey with NurOwn, gave Nick and Jamie a spark of hope when they needed it most. In the years since, their path with ALS has been filled with uncertainty and heartache, but hope has remained at the center of their story – thanks in part to Matt Bellina’s courage and the possibilities offered by the groundbreaking stem cell therapy called NurOwn.

Nick’s Fight

As Nick said to his family and friends when sharing the news of his ALS diagnosis:

“Today is but one day in my fight. Tomorrow will be the next.”

Nick and Jamie believe tomorrow holds the promise of more time, more memories, and more strength – for all the people and families fighting this disease. They are holding on to that promise – believing in the power of tomorrow. But as pilots they also knew that the hope for tomorrow is based on the actions you take today. So a few weeks after the heartbreaking FDA Advisory Committee, Jamie reached out to another ALS advocate and pronounced:

“We are not the type of people to sit back and do nothing, especially when so much is on the line….
We want to do something drastic to get some positive attention for ALS and
the treatments…. This sounds crazy, but we are prepared to
do something crazy to hopefully facilitate change….
We are in this fight with you now.”

That “something crazy” was about to come to fruition. A few months earlier, when Nick had published a story in the American Bar Association Journal, talking about his law firm’s pro bono program, he never anticipated how much their guidance would play a role in his fight to get access to the stem cell therapy that could help him live.

Nick and the pro bono team at Davis, Wright & Tremaine spent months meeting, strategizing, researching, reading Supreme Court briefs and listening to oral arguments. They repeatedly challenged the status quo that the FDA could deny approval of a drug that had compelling evidence of helping some people live longer and live better. They were unwilling to accept the answer that there was no hope for Nick.

When Matt Bellina stood up out of his wheelchair, he showed the world that the impossible was now possible in ALS. Was it also possible that the real-world data gathered by citizens with ALS could ever support an FDA approval, and if so, how could they get that evidence before the FDA?

The legal answer was the Citizen’s Petition filed with the FDA one week ago.

Just as they served our country for years – fighting to protect all of us – these naval aviators are still fighting today … fighting to change regulatory law … fighting to protect the 32,000 people with ALS… and still fighting for approval of the stem cell therapy that can help them live. The Citizens’ Petition launches their fight.

Matt’s Right to Try Story

In 2007, Brig. General Tom Mikolajcik testified before Congress and warned that the risk of ALS was skyrocketing. According to a recent Military Medicine study, in post-9/11 veterans, the highest risk is among pilots like Matt, Nick and the several C-141 pilots in Gen. Mik’s own squadron who were stationed at the Charleston AFB.

General Mik demanded that we “give our soldiers, sailors, airmen, and marines an opportunity to fight this disease with a medical arsenal.” Then he challenged Congress and the FDA to step up its commitment to veterans and to act with urgency:

“We owe our veterans treatment now.
If these soldiers were dying in the field rather than quietly at home… we would leave no stone unturned.
We would use the best existing resources to make sure they had
whatever they needed to survive … to ensure that no man or woman is left behind.”

Just four years later, when Matt was diagnosed with ALS, there were no disease-modifying drugs in the medical arsenal. As he described in the 2016 HBO Vice documentary, “Die Trying: the battle for ALS treatments,” promising drugs were stuck in clinical trials, but Matt was unable to qualify for any of those trials from the moment he was diagnosed. So Matt did what veterans do; he fought to change the law.

On May 30, 2018, President Trump signed the Matt Bellina Right to Try bill into law. President Trump tweeted:

“With Right to Try, patients with life-threatening illnesses
will finally have access to experimental treatments
that could improve their conditions.”

The Citizens’ Petition reports that’s precisely what happened for Matt Bellina.

Matt’s NurOwn Story

The Citizens’ Petition documents Matt Bellina’s real-world evidence (RWE) and real-world data (RWD) on NurOwn. He received 7 doses of NurOwn via Right to Try from December 2018 through 2020. Matt is the only person in the US who received 6 consecutive doses at the recommended two-month dosing interval. The seventh was received nine months later. But sadly, after his seventh dose, Matt couldn’t get more. It’s now been nearly 5 years since his last dose of the stem cell therapy that was helping him live. Today Matt is dying waiting.

Matt has publicly released clinical data from his VA medical records in both his blog and on social media. Before NurOwn, Matt was losing about 0.45 points per month on the 48-point functional scale. Although he had been a slow progressor, his ALS was already advanced as demonstrated by his functional score of 21/48. Matt had already lost over half of his measurable function.

But once he received NurOwn, that lethal trajectory changed. Matt’s VA medical records document that instead of losing nearly 3 points in 6 months, he regained 6 points of function. This is a 9-point delta. This doesn’t happen in ALS. But it did on NurOwn.

And those changes started happening almost immediately. Just two weeks after his first NurOwn dose, Matt shared that his legs were stronger; his family propped him up against the kitchen counter and he was able to stand. In February 2019, Matt posted on Facebook and shared his improvements in all four domains on the functional rating scale used for ALS trial endpoints: gross and fine motor function, bulbar function and most importantly breathing function:

“I have been given a gift…. Only one month after my first round of treatment,
I have improvement in the clinical strength of my right deltoid and my left bicep.
My forced vital lung capacity is 23% higher and I am seeing subjective improvement in my speech & swallowing.
I no longer need a bi pap at night. Due to increased core strength & coordination,
I am now able to pull myself up to standing.”

By May 2019, Matt publicly shared videos demonstrating his profound increase in function and added this commentary:

“What is remarkable is that I was not able to get out of my chair on my own before NurOwn.
After my second treatment I was able to pull up to standing using both my legs and my arms.
Since the third treatment I am able to stand from my chair without the aid of my arms.
I have not been able to do this for over 2 years and it feels great.
We all need to push the FDA to approve this treatment.
It is simply unacceptable that I am the only one receiving this treatment outside of the trial.
All people with ALS deserve this chance.”

Additionally, before NurOwn, Matt’s ability to swallow food and liquids was also becoming compromised. In her Public Comment, Matt’s mother described a choking incident documented in his VA hospital records in December 2018. After receiving NurOwn, Matt’s choking stopped. He was once again able to enjoy his favorite foods and was still able to enjoy a cold beer on a hot Philly summer day. Matt didn’t get a feeding tube until last month – that’s 6.5 years after his first dose of NurOwn.

As profound as all these changes were, Matt’s improvements in breathing function were the most clinically meaningful as they were life-sustaining. After 6 doses of NurOwn, Matt’s lung capacity was 37% higher than it was before his first injection and Matt stopped using a non-invasive ventilator to breathe at night. This doesn’t happen in the normal lethal and unrelenting progression of ALS. It did happen on NurOwn.

Matt’s Real-World Evidence Is Supporting Evidence of Efficacy for FDA Approval

The Citizens’ Petition asserts that Matt’s Right to Try data is “supporting evidence” of efficacy demonstrating that NurOwn works on some people with 100% fatal ALS.

At multiple patient-focused drug development meetings and in hundreds of emails with former FDA leadership, Matt and the entire ALS community repeatedly shared his data and videos – documenting his unprecedented improvements in function. And yet, Matt’s data was not discussed in the FDA’s briefing documents or presentation at the NurOwn Advisory Committee (AdComm) meeting in September 2023. The AdComm voted against approval and instead recommended another Phase 3 trial limited to the trial population that had shown hypothesis-generating promise in the post hoc analysis.

Ultimately, the AdComm vote was based only on data from the 28-week trial and did not include the RWE/RWD over the 8 years for those in EAP nor the real-world data from Matt Bellina’s Right to Try dosing.

The prior FDA Administration ignored Matt Bellina’s evidence. Consider this irony. The Navy entrusted Matt with a $50 million aircraft and the lives of fellow crew members, but the former FDA didn’t trust him to know if a stem cell therapy was helping his own paralyzed body regain function.

Matt’s real-world data is critical, not only because of his profound improvement, but it is also informative because it provides unique evidence not captured in the NurOwn Phase 3 trial data. As detailed in the Citizens’ Petition:

• Matt is the only person in the US to receive 6 consecutive doses and his functional improvements evidence a dose-dependent impact in both durability and magnitude of response.

• Matt’s baseline score of 21/48 illustrates that NurOwn can work on some people later in ALS disease progression.

• Matt was a slow progressor and this population was excluded from the NurOwn trial (and most ALS trials) because it’s hard to assess changes in slow progressors in short 6-month trials; and thus it tells us that NurOwn can work on some people in this subset of slower ALS progressors.

• Matt received his first dose of NurOwn more than 7 years after onset, demonstrating that it can work on some people like his friend and fellow naval aviator, Nick Warack, who has had ALS for 5 years.

Matt’s RWD from Right to Try also aligns with the clinically meaningful impact and “progression-free survival” that trial investigators observed in the NurOwn Phase 3 trial and EAP. At the FDA Advisory Committee meeting, Mayo’s Dr. Anthony Windebank opined that NurOwn works:

“I would now like to provide my clinical perspective on NurOwn ….
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….
There were some who IMPROVED their score!”

In 40+ years working as a neurologist and clinical trialist, Dr. Windebank said NurOwn caused improvements like he had never seen before. Similarly, when people with ALS saw videos of a wheelchair-bound man rising out of his wheelchair, and taking steps with a walker again, they too knew it was like nothing they had ever experienced or witnessed before. And just as Jamie and Nick said, Matt’s video evidence provided a ray of hope in a disease that has had none since ALS was discovered in 1869.

Commissioner Makary recently told the roomful of esteemed scientists at the Gene and Cell Therapy Forum that researchers and regulators can learn things from “n of 1” data. And as one former CBER official said, when making decisions about a therapy’s efficacy, regulators like to see a treatment response so obvious that you don’t need to be a statistician to interpret it.

Matt Bellina’s Right to Try data exemplifies the obvious. NurOwn works.

Thus, the Citizens’ Petition is asking this new FDA to consider Matt’s real-world data derived from his Right to Try dosing. Compare it to the NurOwn Phase 2 or 3 randomized controlled data that demonstrated efficacy in some. Compare it to the ALS clinical trial database; compare it to the natural history databases. How many times in ALS natural history or ALS clinical trials have people regained 6 points in function and risen up out of a wheelchair? How many times have people with ALS stopped needing a bi-pap to breathe?

The Citizens’ Petition urges the FDA to compare Matt’s “n of 1” RWD to whatever data you want. But at least consider it. In a 100% fatal and paralyzing disease, any evidence of efficacy – all evidence of efficacy – should be considered. Gold standard science and common sense demand nothing less.

In 2022, Matt so believed in NurOwn’s efficacy that he co-authored a press release asking the FDA for an Advisory Committee meeting so veterans’ voices could be heard.

“Veterans with ALS have a unique stake in the fight for a NurOwn AdComm.
We sacrificed our lives for every citizen’s right of due process.
It is the antithesis of all we fought for if we, now, were denied that same right.”

Today the Citizens’ Petition makes this same request. Please give Matt his due process; give Matt his first opportunity to be heard; give the 5,000+ veterans with ALS a chance to fight the disease they got from serving our country; give the new CBER team the first chance to consider Matt’s unprecedented improvements before deciding the fate of Matt, Nick and the other 32,000 Americans with ALS.

And then, just as General Mik implored Congress, the Citizens’ Petition implores the FDA to please add NurOwn to the medical arsenal for all Americans battling ALS.

About these Naval Aviators
Matt Bellina served as a Naval Officer and aviator for nearly 10 years. As an Electronic Attack Pilot in Whidbey Island, Washington, Matt flew the EA-6B Prowler, and deployed to Europe, the Middle East, Africa and Asia. Following his flying career he worked in Operations intelligence, before medically retiring due to ALS in 2014.

Nick and Jamie (Nelson) Warack both served as Naval Officers and aviators, serving as flight instructors and mission commanders on the P-3 Orion. Jamie ultimately rose to the position of Weapons and Tactics Instructor (the P3’s equivalent of “Top Gun”). Nick and Jamie flew operational missions in Iraq, the Horn of Africa, South East Asia, and the Mediterranean, combining for over 100 combat missions and earning eight strike flight air medals between them.

Matt, Nick and Jamie are approaching ALS with the same dedication and tenacity that carried them through their time in the Navy. They now lead a coalition of people who have been advocating for approval of NurOwn. They are committed to expediting access to all promising treatments for ALS, and to implementing policy changes to benefit everyone in the ALS community.

Contact:

1. Nicholas Warack, Esq.
   (mail to: Veterans4NurOwn@gmail.com)

2. Mitze Klingenberg, BSN RN
   (mail to: NurOwnWorks@gmail.com)

Copy of Citizens’ Petition and Exhibits Filed with the FDA:
(https://www.nurownworks.com/)

About the FDA Approval Pathways

Nick, Jamie and Matt assert that the survival, respiratory and functional data from the EAP, Right to Try and randomized controlled trials are part of the “totality of the evidence” that support NurOwn’s approval.

1. Traditional Approval
   Survival data are the gold standard in FDA approvals. NurOwn’s survival data meet both the quality and quantity requirements of “substantial evidence.” (See Petition’s Emergent Fact section C and Memorandum section II).

2. Accelerated Approval
   NurOwn meets the standards for accelerated approval. The survival and respiratory data are “reasonably likely to predict” a favorable impact on the mortality of the 32,000 people with ALS. This survival data far surpasses survival data supporting the accelerated approval of many cancer therapies. (See Petition’s Emergent Fact section C & D; Memorandum section I and II.C; and Exs. A & B).

3. Conditional Approval
   NurOwn’s “plausible mechanism of action” also meets the threshold for Commissioner Makary’s proposed conditional approval pathway. (See Petition Fact section N, pgs. 173-176 and Memorandum section II.G at pgs. 234-241).

SOURCE: NurOwn Citizen’s Petition

View the original press release on ACCESS Newswire

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.

• 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 hisblog and onsocial 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 improved 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:

1. Nicholas Warack, Esq.
   (mail to: Veterans4NurOwn@gmail.com)

2. Mitze Klingenberg
   (mail to: NurOwnWorks@gmail.com)

SOURCE: NurOwn Citizen’s Petition

View the original press release on ACCESS Newswire