Brainstorm announced its 15 Phase 3B trial sites in US.
ALS Trial called "ENDURANCE"
https://t.co/0BNnhf5Tim
I know multiple people who HALTED their progression on #NurOwn in Phase 2, Phase 3A and EAP. It may not work on everyone, but it has given many people more time to spend with their families.
If I were newly diagnosed with #ALS, I would move heaven & earth to enroll in this trial.
TRIAL DETAILS:
• 24 wk Trial
• 50-50 placebo
• 24 wk Open Label Extension where everyone gets the therapy
LOCATIONS:
• AZ- Barrow Neuro (Phoenix)
• CA - UC San Diego
• CA - USC (Los Angeles)
• CA - California Pacific Medical Center
• CA - UC San Francisco
• CO - U Colorado Anschutz
• FL - Nova Southeastern U (Ft. Lauderdale)
• FL - Mayo (Jacksonville)
• FL - U of South Florida (Tampa)
• IL - Northwestern Medicine (Chicago)
• MA - Healey Ctr at MassGen (Boston)
• MA - UMass Medical School (Worcester)
• MN - Mayo Clinic (Rochester)
• OR - Providence ALS Center (Portland)
• PA - Temple (Philadelphia)
Trial is NOT yet recruiting.
Anticipated start date June 30, 2025
Anticipated completion date = 2029
Thank you @BrainstormCell for never giving up on the ALS Community.
NurOwn is an autologous mesenchymal stem cell therapy (made from your own stem cells) derived from a bone marrow aspiration. They are sent to a lab and treated with neurotrophic factors then reinjected directly into the CSF where it can work directly on the damaged motor neurons.
#EndALS $BCLI #MND
#alsawarenessmonth #ALSAwareness
#LouGehrigsDisease #Neurotwitter #Neurology #Neurodegenerative #stemcells #RegenerativeMedicine #stemcelltherapy
"We are not data & numbers & spreadsheets. We are human beings with no hope to survive. #ALS is 100% fatal. If you have the power to change that, there should be no hesitation."
~ @JamieRoseBerryy
Delays have consequences.
💔Jamie died on November 9, 2022 -- without ever getting to #NurOwn, the mesenchymal stem cell therapy that has given so many others years more time with their families.
#LivingLongerLivingBetter
#EndALS #ALSAwarenessMonth #ALSawareness
#LouGehrisDisease
@MartyMakary@DrMakaryFDA@VPrasadMDMPH@SecKennedy@POTUS
"Lives lost every month that we are delayed."
Agreed. NIH funding must be restored immediately.
But we also must stop blindly trusting the FDA and instead start asking how many lives are lost because of the delay caused by the FDA's Type II errors?
Tragically, over 6,000 people with 100% terminal #ALS have died waiting each year since 2015 because of the hubris & paternalism of @FDACBER.
People at the FDA -- with ZERO expertise in ALS:
• Ignored the opinions & clinical observations about efficacy from PI neurologists Tony Windebank of Mayo & Bob Brown of UMass who collectively have 500+ peer reviewed studies and h-index scores of 92 & 133.
• Ignored the opinion of the top ALS biomarker expert in the US, Bob Bowser of Barrow Neuro with 120+ peer-reviewed studies ... a researcher so esteemed that he just received a one year $16.7M grant from NIH to create a national ALS research consortium.
• Ignored the opinion of renowned biostatistician Lee Jen-Wei of Harvard who innovated a "totality of the evidence methodology appropriate for rare disease drug trials.... combined with his 300+ peer-reviewed studies, this methodology earned him the Wilks Memorial award in 2009.
• Failed to allow top biostatistician Don Berry to testify about adaptive Bayesian methodology in rare diseases. Dr. Berry has authored 400+ peer-reviewed studies, and has an h-index of 133. He consulted with FDA’s CDRH to set scientific & quality standards for using a Bayesian approach -- but CBER's Celia Witten & Peter Marks didn't deem his opinion worth hearing.
• Failed to allow Jesse Cedarbaum, a Yale neurologist, to testify about the flaws in the COA used for ALS trial endpoints -- even though Dr Cedarbaum designed the COA. Dr Cedarbaum has 30+ yrs of drug development & trial experience, serves on the @NINDS ALS Common Data Elements Project & the @NIH Neuroscience Biomarkers Steering Committee.... but CBER's Celia Witten & Peter Marks didn't deem his opinion worth hearing.
Meanwhile some people with ALS regained function with just 1 dose of a mesenchymal #stemcell therapy in the P2 in 2015... with 3 doses in the P3 in 2019-2020... with 7 doses via RTT in 2019-2020 ... and with 6 more doses in EAP in 2021-2022. And once again, CBER's Celia Witten & Peter Marks didn't deem their experiences worth believing.
That's 54,000 lives lost prematurely -- so far -- because of the FDA's delay.
@MartyMakary@SecKennedy@HeidiOverton@vincehaley@theomerkel@ScottCentorino@SenBillCassidy@ChrisCoons@SenTomCotton@SenWhitehouse@SenatorDurbin@lisamurkowski@SenatorWicker@SenAmyKlobuchar@SenTinaSmith@SenWarren@SenMarkey@SenMarkKelly@SenRubenGallego@SenatorBennet@SenBlumenthal@SenatorWarnock@ChrisVanHollen@SenatorShaheen@SenLBR@SenJackyRosen@SenJeffMerkley@SenSchumer@SenatorCantwell@SenAngusKing@JohnBoozman@SenJohnKennedy@SenatorCollins@MarshaBlackburn@SenCapito@johnthune@RepGuthrie@RepDianaDeGette@RepAndyHarrisMD@DrNealDunnFL2@RepJohnJoyce@RepGregMurphy@RepMikeQuigley
He passed so peacefully with Kandy and Kruz and my hands all on him in a beautiful place over looking the Vail mountains. He passed on his terms. He will be forever loved and missed. I can’t imagine going a day without him. I love you son.
It is with the deepest sadness that I say my oldest son Kade has passed away this morning in Vail, Colorado. He was the most beautiful and toughest person that I have ever known. It gives me peace to know that he is in a much better place and not suffering anymore.
@RainConsulting They also didn’t see my son’s video of him walking after his first injection stating how GOOD he felt. “Almost too good” because how much stronger his weakened leg was & having to adjust to new strength. Oh that’s right… no one from the AdCom making a decision watched the video
@RainConsulting Well said. Kade knew from his 1st injection it worked. We witnessed it w/our eyes. We received confirmation he received NurOwn & not placebo. He deserved a chance at more. I have much disdain for not just the FDA but those in this community who spoke against his chance at more.
@LorriCav And it’s not going to be effective for EVERYONE… no drug or treatment ever is. However, you can’t keep denying access for the people it DOES work for. They deserve that chance to see improvements and stability in this horrific f*cking disease.
Average person with #ALS loses 1 pt/mo in function on the ALSFRS-R scale. Before the 1st #NurOwn injection, this trial participant was a “fast progressor,” losing 1.43 pts/mo.
After receiving NurOwn, this person almost immediately HALTED THEIR PROGRESSION -- consistent with the testimony & “Real World Evidence” of other EAP & trial participants and trial PI Tony Windebank of @MayoClinic.
This person was also a “Responder” as measured by the Primary Endpoint, which assessed whether someone had a large magnitude 1.25-point change in ALSFRS-R slope PER MONTH.
This person’s change in slope was 1.52 pts PER MONTH – a massive slowing in disease progression.
The statutory test for efficacy is whether a therapy has a “clinically meaningful” impact on how someone ”feels and functions.” In ALS, where 50% die in 18 months, nearly 10 points of preserved function over 6 months is not just clinically meaningful, it is life-changing.
#NurOwnWorks.
Our regulatory process doesn't.
People with ALS are #DyingWaiting.
@POTUS@VP@SecBecerra@DrCaliff_FDA
@SenatorBraun @ChrisCoons @RepAnnaEshoo @cathymcmorris@SenSanders@SenBillCassidy@SenJohnThune@SenatorLankford@SenMullin@RepAndyHarrisMD@rosadelauro@RepSchakowsky@RepMikeQuigley@DorisMatsui@SenatorWicker@SenAmyKlobuchar@SenSchumer@SenGillibrand@RepJasonCrow@RepBarragan @RepLBR @RepJohnCurtis @RepGusBilirakis@RepPaulTonko
#EndALS #LouGehrig
More proof that #NurOwn works in #ALS!
Biomarker paper published in @MuscleAndNerve
🔹CSF samples taken 7x during 28 wk trial to analyze impact of 3 doses on 45 pre-specified #biomarkers
🔹3 biomarkers were predictive of clinical outcomes:
1️⃣ Galcetin-1 ... (p=0.01)
2️⃣ LAP/TGFb1 ... (p=0.05)
3️⃣ Neurofilaments light (NfL) ... (p=0.05)
🔹With just 3 doses, NurOwn caused "significant changes" in 64% of the 45 biomarkers analyzed:
1️⃣ 89% neuroprotection
2️⃣ 63% neuroinflammation
3️⃣ 50% neurodegeneration
🔹With just 3 doses, "Debamestrocel significantly reduced NfL from baseline compared with placebo (11% vs. 1.6%, p = .037)"
🔹Senior authors on the study were trial PIs & renown #ALS specialists: Dr. Merit Cudkowicz of the Healey Center at @MGHMedicine & Dr. Bob Brown of @UMassChan
According to PI & renowned specialist in #regenerative medicine, Dr. Anthony Windebank of @MayoClinic:
"The publication of these findings is important, because it demonstrates a potential biologic mechanism by which modified mesenchymal #stemcells (debamestrocel) may benefit patients with ALS.... "There was a STRONG SIGNAL in the phase III trial suggesting a benefit of these cells in a sub-group of patients with less advanced disease...."
Bravo @BrainstormCell
Thank you for being the 1st company that had the foresight to analyze CSF biomarkers in #ALS across a trial's duration. Thank you @Cylebo for your commitment to the ALS community & your impact on changing the future of ALS research & clinical trials.
⬇️READ PAPER:
https://t.co/DuWpuBQlI1
#neurotwitter
#Neurology #LouGehrig #LouGehrigsDisease #EndALS #motorneurondisease #neurodegenerative