Latest news with #ALS
Yahoo
3 hours ago
- Health
- Yahoo
Palm Beach physician pioneering new advances in ALS and inflammatory disease treatment
A Palm Beach physician-scientist is leading efforts to create breakthrough treatments for Parkinson's, ALS, Alzheimer's and other diseases through a biotech company she established nearly a decade ago. Dr. Simrit Parmar, a Northwestern University-trained oncologist and research scientist, launched Cellenkos Inc., in 2016 to accelerate the clinical development of "T regulatory cell" therapies for the treatment of autoimmune diseases and inflammatory disorders. T regulatory cells, or Tregs (pronounced tee-regs), are natural immune system regulator cells that suppress the activity of other immune cells to prevent excessive inflammation and autoimmune reactions. Before launching Cellenkos, Parmar conducted extensive research on these cells and developed therapies based on them. While working as an oncologist/hematologist in the Department of Stem Cell Transplantation and Cellular Therapy at the University of Texas's MD Anderson Cancer in Houston in 2009, she developed treatments using T regulatory cells derived from umbilical cord blood to help prevent graft-versus-host disease (GVHD), a complication that can occur after an allogeneic stem cell or bone marrow transplant. Parmar told the Daily News it was 'very discouraging' to watch patients die from GVHD, which motivated her to focus her research on controlling the inflammation caused by donor cells, starting with the discovery of T regulatory cells. As she continued to conduct her research, she discovered that these cells had uses beyond preventing GVHD. "We found that the application of these cells is beyond just that one niche," she told the Daily News. "It can be applied to any autoimmune disease, to any inflammatory disorder." In 2016, the cell technology Parmar developed at MD Anderson was licensed to Cellenkos, which now supports her ongoing research through a sponsored research agreement. Since then, Parmar has focused on her work advancing targeted Treg cell therapies designed to ease inflammation and regulate immune function. Cellenkos is currently in clinical trials for various inflammatory and autoimmune diseases, including ALS, bone marrow failure, severe COVID-19 and myelofibrosis. Results have been positive so far, Parmar said, noting that ALS patients have seen a delay in disease progression and functional decline and an improvement in quality of life. "We have made some great strides in neurodegenerative disease," she said. "We just published a paper in the New England Journal of Medicine Evidence on ALS, where we showed not only disease halting, but slight improvement in resolving inflammation. That research has applications beyond just ALS. We are hoping as we are garnering more funding, we can go after Parkinson's, Alzheimer's and multiple sclerosis." Inflammation is the root cause of many diseases including cardiac, respiratory, neurological and hematological disorders, Parmar said, and it also drives many autoimmune diseases. Parmar's cell therapies restore immune balance by targeting harmful T cells — a type of white blood cell called lymphocytes — that can mistakenly attack healthy cells in the body, and retraining the body's own defense mechanisms. "We have a drug that heals inside out," she said. "We believe that we neutralize the bad player, which may not have initiated the inflammation, but definitely plays a key role in the propagation of widespread inflammation." Parmar, a mother of three who lives in Palm Beach's North End and travels regularly to Houston, where Cellenkos is based, said she remains committed to developing cell therapies targeting a broad range of autoimmune and inflammatory diseases. Cellenkos is pursuing funding for larger ALS research studies in the U.S., and it is also preparing for the next phase of clinical trials required for federal Food and Drug Administration approval. Parmar said she sees Palm Beach County's emergence as a neuroscience hub as a key benefit of her move, and she hopes it will lead to new research, development, and partnership opportunities. "We're incredibly excited about the momentum building in the Palm Beach and Jupiter area around neuroscience innovation," she said. "With new opportunities for R&D collaboration in the region, it presents an ideal environment for us to expand. We see great potential not only to develop a manufacturing facility for neurological compounds … but also to establish a dedicated treatment center that brings these breakthroughs directly to patients.' Jodie Wagner is a journalist at the Palm Beach Daily News, part of the USA TODAY Florida Network. You can reach her at jwagner@ This article originally appeared on Palm Beach Daily News: Palm Beach physician-scientist pioneering groundbreaking ALS therapy
Miami Herald
4 hours ago
- Health
- Miami Herald
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. Five-Year Survival: 100% in NurOwn EAP vs. 20% in ALS natural history. All EAP participants (n=10) achieved five-year survival without 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 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 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 ApprovalNurOwn'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 ApprovalNurOwn 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 ApprovalNurOwn 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 ALSALS 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, Klingenberg - Phase 3 & EAPEric Stevens - Phase 3 & EAPJoshua Smith - Phase 3 & EAPEstate of Roberto Muggli - Phase 3 & EAPLesley Krummel - Phase 3Estate of Kade Simons - Phase 3Estate of Justin Rogers - Phase 3Terri Pickering Saenz - Phase 2Tara CollazoMayuri SaxenaEstate of Jamie Rose BerryEstate of Patricia ManhardtShahriar Minokadeh, MD Contact: Nicholas Warack, Esq.(mail to: Veterans4NurOwn@ Klingenberg(mail to: NurOwnWorks@ SOURCE: NurOwn Citizen's Petition
USA Today
7 hours ago
- Health
- 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
7 hours ago
- Health
- 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
Associated Press
7 hours ago
- Health
- Associated Press
PathMaker Neurosystems Announces Publication of First Study Demonstrating Increased Survival in an ALS Mouse Model After Treatment With Non-Invasive Neuromodulation
Boston, MA and Providence, RI, July 07, 2025 (GLOBE NEWSWIRE) -- PathMaker Neurosystems Inc. ('PathMaker'), a clinical-stage neurotechnology company developing a breakthrough non-invasive approach for treating Amyotrophic Lateral Sclerosis (ALS, Lou Gehrig's disease), today announced the publication of groundbreaking research in Frontiers in Neurology demonstrating that its proprietary multi-site direct current stimulation (Multi-Site DCS) technology significantly extended survival time and reduced motor neuron degeneration in the widely used SOD1-G93A mouse model of ALS. This important study marks the first demonstration of any non-invasive neuromodulation technique delivering statistically significant survival benefits in an ALS mouse model. The results are compelling, with treated mice exhibiting a 74% increase in survival time from disease onset compared to untreated counterparts. The published paper, ' Multi-path direct current spinal stimulation extended survival in the SOD1-G93A model of amyotrophic lateral sclerosis,' has as senior author, Dr. Zaghloul Ahmed, Professor and Chairman, Department of Physical Therapy and Professor, Center for Developmental Neuroscience, CUNY and Scientific Founder of PathMaker Neurosystems. Beyond extending survival and mitigating motor neuron degeneration, this NIH-funded research highlights that Multi-Site DCS treatment also reduced spinal excitability, decelerated motor dysfunction progression, activated protein degradation pathways and lowered SOD1 protein levels in motor neurons. 'These results provide compelling evidence that our non-invasive treatment approach can meaningfully impact disease progression,' said Nader Yaghoubi, M.D., Ph.D., Co-Founder and Chief Executive Officer of PathMaker Neurosystems. 'The mechanistic insights from this paper underscore the potential of non-invasive Multi-Site DCS as a transformative disease-modifying intervention for ALS. Our ongoing efforts are directed towards translating this innovative treatment into clinical development, with the CALM trial, Controlling Amyotrophic Lateral Sclerosis Motor Neuron Excitability, our second ALS clinical trial, now in progress ( NCT06649955 ). This trial, funded by the U.S. Department of Defense ALSRP program, represents a significant advancement in our journey to bring this novel modality to clinical use. About PathMaker Neurosystems Inc. PathMaker Neurosystems is a clinical-stage neuromodulation company developing a breakthrough non-invasive approach to the treatment of amyotrophic lateral sclerosis (ALS). More than 34,000 people in the U.S. and 350,000 people worldwide are estimated to suffer from ALS. PathMaker is collaborating with world-class institutions to develop and bring to market first-in-class products for treating ALS and other neurological disorders with very high unmet medical need. The MyoRegulator® platform is based on PathMaker's proprietary technology for multi-site direct current stimulation (Multi-Site DCS). MyoRegulator® has been previously designated by the US Food and Drug Administration (FDA) as a 'FDA Breakthrough Device.' MyoRegulator® and MyoRegulator® ALS are investigational medical devices and are limited by Federal law to investigational use only. For more information, please visit the company website at Source: PathMaker Neurosystems Inc. Media contact: PathMaker PR (617) 535-7696 [email protected]
