Matt Bellina

“We want to focus the agency on cures and meaningful treatments. We believe in the letter and the spirit of Right to Try.” -Commissioner Marty Makary (FOXNews - May 8, 2025)

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We do too. In 2018, “Right to Try” became law. Named after Navy pilot Matt Bellina, it was only fitting that Matt was one of the first people to benefit from it. This was possible only because the tiny start- up pharma company, Brainstorm Cell and the VA, covered the cost of manufacturing and administering NurOwn.

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From December 2018 through 2020, Matt Bellina received 7 doses of NurOwn via Right to Try; of which 6 were consecutive doses at two-month dosing intervals. The seventh was received 9 months after the initial 6 doses. Matt is the only person in the US who received six consecutive doses. 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 is helping him live.

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Matt Bellina’s results via Right to Try are not mentioned in the FDA Briefing Document. They are, however, publicly available and were provided by the Bellinas to the FDA during multiple PFDD meetings. Thus, his RWD/RWE and PROs constitute new supporting evidence of efficacy and it should be considered by the FDA.

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Matt has released clinical data from his VA medical records in his blog and on social media. In February 2019, Matt posted on Facebook:

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“I have been given a gift. Many of you read last June that Brainstorm would be treating me with the experimental treatment of Nurown under the new federal Right to Try law.... 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 & 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. Because this is an investigational therapy we don't know what tomorrow will bring but for now, we are feeling incredibly blessed.”

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On NurOwn, Matt’s ALSFRS-R score increased by 6 points on the 48-point scale and he stopped using a non-invasive ventilator to breathe at night. In May 2019, Matt again posted videos on Facebook demonstrating a profound increase in function and added this commentary:

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“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 pALS deserve this chance.”

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In 2020, other rare disease advocates were still sharing Matt’s hopeful story:

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“On the morning of December 27th, 2018, American Airlines flight 1776 flew from Boston to Philadelphia with a small, temperature-controlled box containing my personal mesenchymal stem cells in the form of the treatment known as NurOwn.... Within two weeks, I felt the overwhelming urge to stand up out of my wheelchair, so my family propped me up against the kitchen counter and I stood! Since then, I have had six injections and I have regained the ability to stand on my own without assistance. My lung capacity is 37% higher than it was before my first injection, so I no longer need the assistance of a breathing machine. I have gained enough mobility in my arms to scratch my face and even take my glasses off. All of these are improvements from where I was before the treatment.”

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Anyone with ALS experience knows that Matt’s response is not typical in ALS. Matt’s real- world data is critical, not only because of his profound improvement, but also because of many unique factors that are illustrative as supporting evidence:

• Only person in the US to receive 6 consecutive doses

• Matt’s baseline score of 21 was 6 points below the data that met statistical

  significance in the Phase 3 trial

• Matt was a slow progressor and this population was excluded from the NurOwn trial

• Petitioners acknowledge that Matt is an “n of 1” – but not the only “n of 1.” Matt has a squadron of people who, individually, are also an “n of 1.” Combined there is power in numbers. 

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Matt so believed in NurOwn’s efficacy that he also co-authored a press release asking the FDA for an AdComm so veterans’ voices could be heard:

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“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.”

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Although the FDA had actual knowledge of Matt’s unprecedented improvements, his mother Deb Bellina was not chosen to speak at the FDA AdComm. Matt got this terminal disease from serving our country and the FDA’s lottery process didn’t give him an opportunity to be heard. A patient advocate wrote to the FDA in advance of the AdComm to alert them of this inequity (See Exhibits). Nonetheless, the FDA refused to allow the Bellinas to testify during the Open Public Hearing. No veterans with ALS were chosen to speak despite comprising approximately 1 in 6 people afflicted with ALS.

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So instead, Deb Bellina went to the media. Just before the FDA’s AdComm on NurOwn, Deb authored an OpEd with Fox News:

“Mothers of children with ALS are helplessly watching our children wither away under the burden of this disease. For those who have seen the benefits of this treatment, every day that passes without a decision is like a slow death unto itself.”

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The night before the AdComm, Deb Bellina and Paula Smith were on Cincinnati affiliate WLWT sharing their story about fighting for their sons’ lives. Deb spoke about the blessing and tragedy of being in this fight with Matt for over a decade:

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“I’ve watched a lot of people die over and over and over again, and every time that happens, I think how different it could have been and how different it can be."

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Even worse than denying the Bellinas a chance to tell Matt’s story at the AdComm, the FDA also failed to discuss or believe his real-world evidence. Matt Bellina’s results are not even mentioned in the FDA Briefing Document.

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Consider this irony. The Navy entrusted Matt with a $50 million aircraft and the lives of fellow crew members, but the FDA didn’t trust him to know if a drug was helping him live.

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As a Navy pilot, Matt had to inspect his plane before every flight to ensure it would function properly. His pre-flight checklist was outlined in Naval Air Training and Operating Procedures Standardization Flight Manual, Navy model EA-6B Aircraft (“NATOPS”). For example, the Navy entrusted Matt to be responsible for:

• Inspecting critical weapon systems, such as the AGM-88 HARM (High-speed Anti-Radiation Missile), a tactical, air-to-surface anti-radiation missile, was a key responsibility for Matt. (NATOPS III-7-6). Matt was required to be knowledgeable not only about his aircraft, but also about the weapon systems that complemented it. This depth of knowledge highlights Matt’s ability to understand complex systems and their interactions.

• Closely inspecting the aircraft’s struts and landing gear, noting that if the anti-skid drive cap lacks all four rivets, the aircraft will experience anti-skid system failure during landing rollout. (NATOPS III-7-9). The consequence of missing even a single rivet could result in system failure. The Navy entrusted Matt to maintain an exceptional level of attention to detail, even for seemingly minor components, in a manner demanded by few professions.

• Conducting a 29-step ejection seat preflight inspection. (NATOPS III-7-11). Failure to perform this inspection correctly would result in the certain death of both the pilot and crew in the event of a catastrophic aircraft failure. The Navy aircrew relied on his judgment when inspecting a system designed to save their lives when there is no time to think.

• Memorizing 28 “boldface” emergency procedures for each phase of flight. (NATOPS V-12-1). As Pilot-in-Command, he was required to diagnose and execute over two dozen emergency procedures from memory without reference aids. The Navy, Matt, and his crew depended on his training, critical thinking, quick reaction, and level-headedness during emergencies, as their lives hinged on his abilities.

 

This begs the question: if Matt can be trusted to execute missions in a $50 million aircraft and to diagnose complex, interconnected systems; inspect both mundane and critical components; and execute multi-step emergency procedures from memory to save lives, why didn’t the prior FDA trust him to know if a therapy caused him to regain function in his own paralyzed body?