Latest news with #LouGehrig
Yahoo
3 hours ago
- Business
- Yahoo
Klotho Neurosciences, Inc. Granted FDA Orphan Drug Designation for KLTO-202 for Treatment of Amyotrophic Lateral Sclerosis ("ALS" or "Lou Gehrig's Disease")
NEW YORK, July 10, 2025 /PRNewswire/ -- Klotho Neurosciences, Inc. (Nasdaq: KLTO), a gene and cell therapy company focused on the treatment of neurodegenerative and other aging-related diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to the company's novel secreted-Klotho (s-KL) promoter, gene and delivery system (KLTO-202, or for the treatment of ALS. The FDA grants Orphan Drug Designation to drugs and biologics that are intended for safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S. The Orphan Drug Designation provides certain incentives, such as tax credits, toward the cost of human clinical trials and a waiver for the payment of the GDUFA User Fee for market applications. Additionally, Orphan Drug Designation of the product provides the developers seven years of US market exclusivity and independent from the Company's intellectual property protection. "Receiving the Orphan Drug Designation for for the early treatment of ALS underscores the importance of bringing new treatment options to patients suffering from this rare, universally fatal disease" said Dr. Joseph Sinkule, Klotho's Chief Executive Officer. "My cousin Karen died from this horrific disease. We aim to deliver the first gene replacement therapy addressing the neurologic insult resulting in motor neuron damage and the potential neurologic protection induced by providing therapeutic blood, brain, and muscle concentrations of the s-KL protein. After the FDA's review of the data leading to the Orphan Drug Designation, we believe this ODD designation provides strong validation of our science and our approach to treat this disease" concludes Dr. Sinkule. ALS is sometimes referred to as Lou Gehrig's disease. Lou Gehrig, who played for the New York Yankees for 17 years in the 1920s and 1930s, stunned players and fans by retiring from baseball at the age of 36 after being diagnosed with ALS. Prior to this diagnosis, Gehrig played in a record-breaking 2,130 consecutive games, was referred to as the "Iron Horse," and was considered one of the greatest baseball players of all time. Less than two years later, at the age of 37, Gehrig died of complications from ALS. ALS is also referred to as Motor Neuron Disease in the UK and elsewhere. ALS is considered a rare disease and affects fewer than 200,000 people in the US, with around 5,000 new cases diagnosed each year. Klotho Neurosciences will have completed "proof of concept" studies in two animal models of human ALS and the Company is currently initiating manufacturing of the ALS-targeted product candidate, followed by meetings with the U.S. FDA and EMA in Europe to concur with the development path going forward. KLTO-202, the company's lead product candidate targeting motor neuron diseases and muscular dystrophies, is composed of a muscle-specific promoter called "desmin," driving the expression of the s-KL gene transcript and s-KL protein, with targeted delivery of the gene therapy to the neuromuscular junction - the interface between the spinal cord and the muscles. At this time, KLTO-202 is not approved for human use by any regulatory authority. About Klotho Neurosciences, Neurosciences, Inc. (NASDAQ: KLTO), is a biogenetics company focused on the development of innovative, disease-modifying cell and gene therapies using a protein derived from a patented form of the "anti-aging" human Klotho gene (s-KL), and its novel delivery systems to transform and improve the treatment of neurodegenerative and age-related disorders such as ALS, Alzheimer's, and Parkinson's disease. The Company's current portfolio consists of its proprietary cell and gene therapy programs using DNA and RNA as therapeutics and genomics-based diagnostic assays. The Company is managed by a team of individuals and advisors who are highly experienced in biopharmaceutical product development and commercialization. For more information, please visit the company's website at Investor Contact and Corporate Communications: - Jeffrey LeBlanc, CFOir@ Cautionary Note Regarding Forward-Looking StatementsThis press release contains forward-looking statements. These statements are made under the "safe harbor" provisions of the U.S. Private Securities Litigation Reform Act of 1995. These forward-looking statements generally are identified by the words "believe," "project," "expect," "anticipate," "estimate," "intend," "strategy," "future," "opportunity," "plan," "may," "should," "will," "would," "will be," "will continue," "will likely result," and similar expressions. Without limiting the generality of the foregoing, the forward-looking statements in this press release include descriptions of the Company's future commercial operations. Forward-looking statements are predictions, projections and other statements about future events that are based on current expectations and assumptions and, as a result, are subject to risks and uncertainties. Many factors could cause actual future events to differ materially from the forward-looking statements in this press release, such as the Company's inability to implement its business plans, identify and realize additional opportunities, or meet or exceed its financial projections and changes in the regulatory or competitive environment in which the Company operates. You should carefully consider the foregoing factors and the other risks and uncertainties described in the documents filed or to be filed by the Company with the U.S. Securities and Exchange Commission (the "SEC") from time to time, which could cause actual events and results to differ materially from those contained in the forward-looking statements. Copies of these documents are available on the SEC's website, All information provided herein is as of the date of this press release, and the Company undertakes no obligation to update any forward-looking statement, except as required under applicable law. View original content to download multimedia: SOURCE Klotho Neurosciences, Inc.
Yahoo
3 days ago
- Sport
- Yahoo
Cody Bellinger hits 3 home runs, gets robbed of 4th during rout of Cubs in first game against former team
All that stood between Cody Bellinger and Lou Gehrig in Yankee Stadium Friday night was a leaping Kyle Tucker. The Chicago Cubs right fielder robbed Bellinger of what ultimately would have been the first four-homer game by a New York Yankees player since Gehrig's in 1932. A three-homer performance was quite the consolation prize for Bellinger, especially during an 11-0 win in his first game against his former team. Advertisement Bellinger, who played for the Cubs the previous two seasons before he was traded to the Yankees in a salary dump this past December, got a curtain call in the eighth inning after his third blast narrowly cleared the wall in right-center field. "I know his time there was really good," Yankees manager Aaron Boone said of Bellinger's stint in Chicago, via The Athletic. 'I don't want to make it like anything like that, but it's always good to go against your old teammates that you spent a lot of time with, [who] you know and respect. "To perform right away against them, I'm sure probably is a little cherry on top of it all." All three of Bellinger's home runs scored two runs. He tied his single-game career high with 6 RBI. Advertisement The 6-foot-3 lefty hails from Scottsdale, Arizona, but he might as well have rent in right field because that's where he was living Friday. The two-time All-Star deposited two of his dingers there, and it was also the scene of the crime for Tucker's seventh-inning theft. Bellinger's final swing launched his third big fly to right-center, where he was assisted by a young fan who gave Cubs All-Star center fielder Pete Crow-Armstrong a taste of Tucker's medicine, robbing PCA of a spectacular catch. Bellinger extended his now-16-game hitting streak. He also became the third Yankees player this season to deliver a three-homer game, joining Aaron Judge and Jasson Domínguez in making franchise single-season history. Three three-homer games is a first for the storied club. Judge accomplished the feat on March 29 against the Milwaukee Brewers. Domínguez did it on May 9 versus the Athletics. Advertisement Speaking of Judge, he put on a web gem clinic in right field Friday. Judge robbed Crow Armstrong — an NL MVP candidate — of a home run in the fourth inning. Additionally, he made a pair of diving grabs, one in the fourth and another in the eighth. Meanwhile, fresh off the news he'll replace teammate Max Fried in this year's All-Star Game, Yankees pitcher Carlos Rodón spun eight scoreless frames. It was a star-studded Yankees performance. Bellinger was the headliner with a trio of home runs against his former team.
Miami Herald
07-07-2025
- 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
Yahoo
07-07-2025
- Sport
- Yahoo
This Date in Baseball - Travis Hafner 1st player in MLB history to hit 5 grand slams pre All-Star
July 7 1923 — Lefty O'Doul, pitching for the Boston Red Sox, allowed 13 runs in the sixth inning to the Cleveland Indians, who won 27-3. In 1928, he was to return to the majors as a great hitting outfielder. Advertisement 1936 — The NL won its first All-Star game 4-3 at Braves Field in Boston. 1937 — Lou Gehrig drove in four runs with a home run and a double to pace the AL to an 8-3 victory over the NL in the All-Star game at Washington's Griffith Stadium. In attendance was President Franklin D. Roosevelt. 1959 — At Forbes Field in Pittsburgh, the first of two All-Star games played that season went to the NL, 5-4. The NL scored the tying and winning runs in the bottom of the eighth when Hank Aaron singled in a run and scored on a triple by Willie Mays. 1964 — The NL beat the AL 7-4 in the All-Star game on Johnny Callison's two-out, three-run homer off Dick Radatz in the bottom of the ninth inning at New York's Shea Stadium. The win pulled the NL even with its rivals (17-17-1) for the first time since the series began. Advertisement 1998 — Coors Field lived up to its billing as a hitter's haven as the American League beat the Nationals 13-8 at Coors Field in the highest-scoring All-Star game in major league history. The 21 runs broke the record set in the AL's 11-9 win in 1954. 2006 — Cleveland Indians designated hitter Travis Hafner became the first player in major league history to hit five grand slams before the All-Star break when he connected in the second inning of a 9-0 win over Baltimore. 2009 — Alan Embree earned the win in Colorado's 5-4 victory over Washington without throwing a pitch. He entered with two outs in the eighth and picked off Austin Kearns, who had singled off Joel Peralta. It was the first time a major leaguer had gotten a win without throwing a pitch since B.J Ryan for Baltimore at Detroit on May 1, 2003. 2011 — Dustin Pedroia hit a three-run shot and Boston added three consecutive home runs in the seventh in a 10-4 win over Baltimore. Six different Boston players homered, including the three straight by David Ortiz, Josh Reddick and Jarrod Saltalamacchia. Advertisement 2011 — A Texas Rangers fan died after falling about 20 feet onto concrete reaching out for a baseball tossed his way by All-Star outfielder Josh Hamilton during a game. Shannon Stone, 39, was at the game with his young son, who watched as his dad tumbled over the outfield railing after catching the ball. The accident happened in the second inning after Oakland's Conor Jackson hit a foul ball. Hamilton retrieved the ball and tossed it into the stands as players routinely do. 2016 — Colorado's Trevor Story tied an NL rookie record for most home runs before the All-Star break, homering twice and boosting his total to 21 as the Rockies beat Philadelphia 11-2. 2018 — Mark Reynolds homered twice and drove in a career-high 10 runs and the Washington Nationals beat the Miami Marlins 18-4. Reynolds (5 for 5) tied his career high for hits and equaled the Nationals' RBI record. 2021 — In what has clearly been the 'Year of the No-Hitter″, five Rays pitchers combine to pitch one against the Indians in the second game of a doubleheader that goes seven innings. _____
USA Today
07-07-2025
- 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
