.@FDACBER @SecKennedy, It's 83 DAYS since our #NUROWN Citizens' Petition Survival data: #GoldStandard for FDA approvals. As @DrMakaryFDA said, 'gold standard science & common sense' this FDA #NUROWN survival data UNPRECEDENTED in ALS history & UNAVAILABLE at ADCOMM 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. Every day of delay dooms more patients. The suffering is immeasurable. @VPrasadMDMPH: The science is in. The time for waiting is over. End 156 years of hopelessness. Give us a chance to live. #ApproveNurOwn #NurownWorks #ALS #BeyondSignsOfPromise #AcceleratedApproval Read the evidence: nurownworks.com/read-the-pet… @realDonaldTrump @RepGuthrie @RepLarryBucshon @michaelcburgess @RepGusBilirakis @RepBillJohnson @RepRichHudson @RepBuddyCarter @DrNealDunnFL2 @cathymcmorris @LeaderJohnThune @RepDebDingell @RepMMM @RobertKennedyJr @SenatorWicker @RepRobinKelly @RepBarragan @DrOz @DrOzCMS @GovBraun @BernieSanders @SenBobCasey @SenatorBaldwin @timkaine @SenatorHassan @SenTinaSmith @SenMarkey @SenBillCassidy @SenMullin @lisamurkowski @RogerMarshallMD @RepJasonCrow @TeamCalvert @RepBrianFitz @ChrisCoons @RepAndyHarrisMD @DickDurbin @RepDianaDeGette @RepMikeQuigley @RepSchakowsky @rosadelauro @RandPaul @RepDonBacon @SenJohnCurtis @BillCassidy

No progress yet, unfortunately Previous FDA leadership always made ALS patients wait until the very last minute before answering. So many people would die waiting, just like they are now. I'm hoping that the new FDA leadership, @DrMakaryFDA @FDACBER @SecKennedy have meant what they have said publicly. The ALS community has been burned so many times by the FDA that I am getting more nervous as time goes by without an answer. New & UNPRECEDENTED survival data detailed in Nurown Citizens' Petition submitted 75 DAYS AGO, biomarker evidence, and the preponderance of RWE undeniably deserve an invite to @BrainstormCell to resubmit its BLA so we can have due process using the totality of the evidence. The question is whether we can take the public statements of @VPrasadMDMPH, @MartyMakary, and @RobertKennedyJr at face value. Will this administration be different? I pray that it will. It's the obvious decision if they are considering "Gold standard science and common sense" @realDonaldTrump @RepGuthrie @RepLarryBucshon @michaelcburgess @RepGusBilirakis @RepBillJohnson @RepRichHudson @RepBuddyCarter @DrNealDunnFL2 @cathymcmorris @LeaderJohnThune @RepDebDingell @RepMMM @SenatorWicker @RepRobinKelly @RepBarragan @RepAngieCraig @RepKimSchrier @RepLoriTrahan @BernieSanders @SenBobCasey @SenatorBaldwin @SenatorHassan @SenTinaSmith @SenMarkey @SenBillCassidy @SenMullin @lisamurkowski @RogerMarshallMD @RepJasonCrow @TeamCalvert @RepBrianFitz @ChrisCoons @RepAndyHarrisMD @DickDurbin @RepDianaDeGette @RepMikeQuigley @RepSchakowsky @rosadelauro @RandPaul @RepDonBacon @SenJohnCurtis

.@FDACBER @SecKennedy, It's 61 days since our #NUROWN Citizens' Petition Survival data: #GoldStandard for FDA approvals. As @DrMakaryFDA said, 'gold standard science & common sense' guide this FDA #NUROWN survival data UNPRECEDENTED in ALS history & UNAVAILABLE at ADCOMM 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. Every day of delay dooms more patients. The suffering is immeasurable. @VPrasadMDMPH: The science is in. The time for waiting is over. End 156 years of hopelessness. Give us a chance to live. #ApproveNurOwn #NurownWorks #ALS #BeyondSignsOfPromise #AcceleratedApproval Read the evidence: nurownworks.com/read-the-pet… @realDonaldTrump @RepGuthrie @RepLarryBucshon @michaelcburgess @RepGusBilirakis @RepBillJohnson @RepRichHudson @RepBuddyCarter @DrNealDunnFL2 @cathymcmorris @LeaderJohnThune @RepDebDingell @RepMMM @RobertKennedyJr @SenatorWicker @RepRobinKelly @RepBarragan @DrOz @DrOzCMS @GovBraun @BernieSanders @SenBobCasey @SenatorBaldwin @timkaine @SenatorHassan @SenTinaSmith @SenMarkey @SenBillCassidy @SenMullin @lisamurkowski @RogerMarshallMD @RepJasonCrow @TeamCalvert @RepBrianFitz @ChrisCoons @RepAndyHarrisMD @DickDurbin @RepDianaDeGette @RepMikeQuigley @RepSchakowsky @rosadelauro @RandPaul @RepDonBacon @SenJohnCurtis @BillCassidy

.@DrMakaryFDA @FDACBER @SecKennedy, It’s 60 days since we filed our #NUROWN Citizens' Petition For 156 years, ALS has had ZERO survivors Survival data have long been the gold standard for FDA approvals; and as Commissioner @MartyMakary has emphasized: "gold standard science and common sense" will guide this FDA's decisions These survival data are unprecedented in ALS clinical trial history AND WAS NOT AVAILABLE AT THE NUROWN ADCOMM HEARING. 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. Every day of delay dooms more patients. The suffering is immeasurable. @VPrasadMDMPH: The science is in. The time for waiting is over. End 156 years of hopelessness. Give us a chance to live. #ApproveNurOwn #NurownWorks #ALS #BeyondSignsOfPromise #AcceleratedApproval Read the evidence: nurownworks.com/read-the-pet… @realDonaldTrump @RepGuthrie @RepLarryBucshon @michaelcburgess @RepGusBilirakis @RepBillJohnson @RepRichHudson @RepBuddyCarter @DrNealDunnFL2 @cathymcmorris @LeaderJohnThune @RepDebDingell @RepMMM @RobertKennedyJr @SenatorWicker @RepRobinKelly @RepBarragan @DrOz @DrOzCMS @GovBraun @BernieSanders @SenBobCasey @SenatorBaldwin @timkaine @SenatorHassan @SenTinaSmith @SenMarkey @SenBillCassidy @SenMullin @lisamurkowski @RogerMarshallMD @RepJasonCrow @TeamCalvert @RepBrianFitz @ChrisCoons @RepAndyHarrisMD @DickDurbin @RepDianaDeGette @RepMikeQuigley @RepSchakowsky @rosadelauro @RandPaul @RepDonBacon @SenJohnCurtis @BillCassidy

.@DrMakaryFDA @FDACBER @SecKennedy, Please read my OpEd: newsweek.com/doctors-plea-na… The new FDA leadership now arrives with bold promises of change, posing a question that haunts everyone touched by an untreatable neurological disease: Will this time be different? Health and Human Services (HHS) Secretary @RobertKennedyJr vows to "sweep away barriers" and to "figure out new ways of accelerating approvals for drugs and treatments that treat rare diseases." FDA Commissioner @MartyMakary has several times asked why it takes 10 years for a drug to get to market and proposes a "conditional approval" pathway based on a "plausible mechanism." Center for Biologics Evaluation and Research (CBER) Director @VPrasadMDMPH promised to "take action at the first sign of promise for rare diseases." For the ALS community, these words are now a crisis of credibility. In a disease that has a median survival of 2-3 years, we are asking for regulatory flexibility for a treatment that began its Phase 1 trial in 2011 and was granted FDA Fast Track designation in 2014. As a petitioner on the July 3 citizens' petition for a stem cell therapy known as NurOwn, my community has presented the FDA with a clear test. Ultimately, we are seeking accelerated approval based on new, unprecedented survival data from an expanded access program—data that exceeds the extension of survival of many approved cancer therapies. But the initial request is simpler: invite the sponsor (@BrainstormCell ) to resubmit its application for a full review. Ours is a request for due process to give a voice to the voiceless—the lowest possible bar for the Trump administration to demonstrate its promised flexibility It has been 57 days of continued #ALS deaths & horrific suffering, since we submitted the July 3rd Nurown citizens' petition. Please invite them to resubmit the BLA and take the first step to ending 156 years of hopelessness. #ManySignsOfPromise #ManyPlausibleMechanisms #CriticalUnmetNeed #100PercentFatal #AcceleratedApproval @realDonaldTrump @RepGuthrie @RepLarryBucshon @michaelcburgess @RepGusBilirakis @RepBillJohnson @RepRichHudson @RepBuddyCarter @DrNealDunnFL2 @cathymcmorris @LeaderJohnThune @RepDebDingell @RepMMM @SenatorWicker @RepRobinKelly @RepBarragan @RepAngieCraig @RepKimSchrier @RepLoriTrahan @BernieSanders @PattyMurray @SenBobCasey @SenatorBaldwin @SenatorHassan @SenTinaSmith @SenMarkey @SenBillCassidy @SenMullin @lisamurkowski @RogerMarshallMD @RepJasonCrow @TeamCalvert @RepBrianFitz @ChrisCoons @RepAndyHarrisMD @DickDurbin @RepDianaDeGette @RepMikeQuigley @RepSchakowsky @rosadelauro @RandPaul @GovBraun @SenJohnCurtis

.@DrMakaryFDA @FDACBER @SecKennedy, It’s been 57 days since we filed our Citizens' Petition For 156 years, ALS has had ZERO survivors. We now have a therapy, NurOwn, with unprecedented, gold-standard survival data. @GovBraun Every day of delay dooms more patients. The suffering is immeasurable. @VPrasadMDMPH: The science is in. The time for waiting is over. End 156 years of hopelessness. Give us a chance to live. #ApproveNurOwn #NurownWorks #ALS #BeyondSignsOfPromise #AcceleratedApproval Read the evidence: nurownworks.com/read-the-pet… @realDonaldTrump @RepGuthrie @RepLarryBucshon @michaelcburgess @RepGusBilirakis @RepBillJohnson @RepRichHudson @RepBuddyCarter @DrNealDunnFL2 @cathymcmorris @LeaderJohnThune @RepDebDingell @RepMMM @RobertKennedyJr @MartyMakary @SenatorWicker @RepRobinKelly @RepBarragan @DrOz @DrOzCMS @BernieSanders @SenBobCasey @SenatorBaldwin @timkaine @SenatorHassan @SenTinaSmith @SenMarkey @SenBillCassidy @SenMullin @lisamurkowski @RogerMarshallMD @RepJasonCrow @TeamCalvert @RepBrianFitz @ChrisCoons @RepAndyHarrisMD @DickDurbin @RepDianaDeGette @RepMikeQuigley @RepSchakowsky @rosadelauro @RandPaul @RepDonBacon @SenJohnCurtis @BillCassidy.

.@DrMakaryFDA @FDACBER @SecKennedy, It’s 58 days since we filed our #NUROWN Citizens' Petition For 156 years, ALS has had ZERO survivors Survival data have long been the gold standard for FDA approvals; and as Commissioner @MartyMakary has emphasized: "gold standard science and common sense" will guide this FDA's decisions These survival data are unprecedented in ALS clinical trial history AND WAS NOT AVAILABLE AT THE NUROWN ADCOMM HEARING. 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. Every day of delay dooms more patients. The suffering is immeasurable. @VPrasadMDMPH: The science is in. The time for waiting is over. End 156 years of hopelessness. Give us a chance to live. #ApproveNurOwn #NurownWorks #ALS #BeyondSignsOfPromise #AcceleratedApproval Read the evidence: nurownworks.com/read-the-pet… @realDonaldTrump @RepGuthrie @RepLarryBucshon @michaelcburgess @RepGusBilirakis @RepBillJohnson @RepRichHudson @RepBuddyCarter @DrNealDunnFL2 @cathymcmorris @LeaderJohnThune @RepDebDingell @RepMMM @RobertKennedyJr @SenatorWicker @RepRobinKelly @RepBarragan @DrOz @DrOzCMS @GovBraun @BernieSanders @SenBobCasey @SenatorBaldwin @timkaine @SenatorHassan @SenTinaSmith @SenMarkey @SenBillCassidy @SenMullin @lisamurkowski @RogerMarshallMD @RepJasonCrow @TeamCalvert @RepBrianFitz @ChrisCoons @RepAndyHarrisMD @DickDurbin @RepDianaDeGette @RepMikeQuigley @RepSchakowsky @rosadelauro @RandPaul @RepDonBacon @SenJohnCurtis @BillCassidy

.@DrMakaryFDA @FDACBER @SecKennedy, It’s been 56 days since we filed our Citizens' Petition For 156 years, ALS has had ZERO survivors. We now have a therapy, NurOwn, with unprecedented, gold-standard survival data Every day of delay dooms more patients. The suffering is immeasurable. @VPrasadMDMPH: The science is in. The time for waiting is over. End 156 years of hopelessness. Give us a chance to live. #ApproveNurOwn #NurownWorks #ALS #BeyondSignsOfPromise #AcceleratedApproval Read the evidence: nurownworks.com/read-the-pet… @realDonaldTrump @RepGuthrie @RepLarryBucshon @michaelcburgess @RepGusBilirakis @RepBillJohnson @RepRichHudson @RepBuddyCarter @DrNealDunnFL2 @cathymcmorris @LeaderJohnThune @RepDebDingell @RepMMM @RobertKennedyJr @MartyMakary @SenatorWicker @RepRobinKelly @RepBarragan @DrOzCMS @DrOz @RepKimSchrier @RepLoriTrahan @BernieSanders @SenBobCasey @SenatorBaldwin @timkaine @SenatorHassan @SenMarkey @SenBillCassidy @SenMullin @lisamurkowski @RogerMarshallMD @RepJasonCrow @TeamCalvert @RepBrianFitz @ChrisCoons @RepAndyHarrisMD @DickDurbin @RepDianaDeGette @RepMikeQuigley @RepSchakowsky @rosadelauro @RandPaul @RepDonBacon @SenJohnCurtis @BillCassidy