Top Tweets for #diedWaiting
Agree with @houmanhemmati & @PeterDiamandis
Most unethical thing in medicine are the patients who #DiedWaiting for a treatment that worked on them but wasn't approved yet. This happened in #ALS.
CBER refused to approve a neurotrophically-enhanced #stemcell therapy delivered via intrathecal injection directly to dying motor neurons. Renowned @MayoClinicNeuro & @UMassChanNeuro PIs told FDA some patients stabilized & regained function - something they had never seen in 40+yrs of practice. Patients had videos proving it restored function. FDA refused to consider RWE.
In short 28-wk P3 trial with just 3 doses, data showed it worked on subgroup with early stage ALS (akin to stage I/II cancer). CBER's OTAT/OTP said "Do another trial to prove post hoc subgroup' ... tacitly acknowledging they were ok with people dying in the meantime.
Now patients -- led by an attorney & #veteran with ALS @NicholasWarack -- have taken it upon themselves to file a Citizens' Petition with 7+ yrs of unprecedented and now mature EAP survival data -- confirming what patients & PIs have been saying since P2 in 2015: #NurOwnWorks.
🔹7+ yrs median trach-free survival vs 2.5 yrs in ALS natural history
🔹5-yr EAP survival: 100% vs <20% natural history
🔹Progression-free survival up to 18 months after just 3 doses
🔹Survival data exceeds approved ALS therapies and many cancer therapies
🔹Unprecedented survival data still maturing as 5/10 from EAP still alive & not trached
🔹ORR of 35% on primary endpoint & 73% on secondary endpoint in early progression subgroups
🔹CSF Biomarker data: 23 met stat significance & 15/23 p<0.001
🔹Extended dosing in EAP showed up to 60% decrease in neurodegeneration biomarker Neurofilament Light
Under Prasad, CBER didn't bother responding to 300 pages of evidence in the Citizens Petition -- a response was due Dec 30th.
Since 2020, over 30k Americans with ALS have died waiting. Since the Citizens' Petition was filed, nearly 6k have died waiting.
Houman is right: We need a "COMMON SENSE & patient-first approach to how drugs are regulated." Regulatory rigidity is killing people with #rarediseases
@LeaderJohnThune @SenatorWicker @RonJohnsonWI @SenRonJohnson @SenRickScott @SenGillibrand @RandPaul @DrRandPaul @ChrisCoons @SenTomCotton @SenBillCassidy @ChuckGrassley
@RepGusBilirakis @DorisMatsui @KenCalvert @RepAndyHarrisMD @RepJohnJoyce @RepMGriffith @RepMMM @RepGuthrie @RepDonBacon @RepSchakowsky @TomColeOK04
I totally agree with my friend @PeterDiamandis - it's DEEPLY unethical to deny or delay patients access to medicines that can save or dramatically improve their lives for bureaucratic or technical reasons.
Last week, I openly challenged former head of ophthalmology at FDA on this, frustrated that FDA has made it impossible to develop preventative treatments for intermediate macular degeneration (earlier form of AMD just BEFORE you are permanently blinded) by demanding treatments that dramatically improve vision before you've lost any vision.
Watch the video below. I argue that FDA's behavior has been akin to denying Stage 1 cancer patients a drug that works, forcing them to develop Stage 3 or 4 cancer so the clinical trial can demonstrate a larger potential benefit. This is backwards thinking.
The former FDA official defends his position by saying you can't create a preventative drug that doesn't work in 100% of people. WHAT? We give cholesterol and blood pressure medicines to tens of millions of people, many of whom will still get a heart attack or stroke (albeit often delayed), and many of whom will die of something else. Why take that CHOICE away from physicians and patients?
As long as I am alive, I am committed to bringing back COMMON SENSE & a patient-first approach to how drugs are regulated. The federal government is not the patient, nor it is the physician.
The FDA's role is clearly to be one important gatekeeper - to review clinical and other data to be sure there's a meaningful chance of efficacy outweighing safety risks and making those facts available to the public.
Once a drug is approved, there are THREE other layers of protection:
-Patients decide whether or not to take a drug
-Physicians decide whether or not to prescribe a drug
-Insurance companies decide whether or not to pay for a drug
I urge a move to this common sense approach that empowers the American PUBLIC to make informed decisions about their health as opposed to having very smart/committed & very well-meaning government officials limit our choices.
Our lives, our health, our vision, our ability to enjoy a high quality life depend on it.
Thank you @ArshadKhanani for moderating such an incredible panel & conference last weekend!
.@FDACBER @VPrasadMDMPH, 132 DAYS you've had our Nurown Citizens petition=SILENCE
Laura Jean is one of tens of 1000s who have #DiedWaiting WITHOUT HOPE leaving behind her husband & 3 young children
@SecKennedy @DrMakaryFDA, Every day of delay=more HORRIFIC deaths.
No words can convey how HORRIFIC the suffering is while we wait on the FDA
You're MISTAKEN if you believe 100% FATAL #ALS can't happen to YOU.
GRANT ACCELERATED APPROVAL for NurOwn NOW.
Our Citizens' Petition, submitted on July 3rd and now ignored for 127 days, demands it. This autologous stem cell therapy—our own cells engineered to flood our brains with life-saving neurotrophins—is ALS's FIRST BREAKTHROUGH in 156 years of hell. The Phase 3b trial was cleared in May 2025, and the data from the 2022 ADCOMM screams for approval.
Irrefutable Evidence
Biomarkers PROVE It Works: Reduced Neuroinflammation: Significant decrease in MCP-1 cytokines (p<0.05). Reduced Neurodegeneration: Significant drop in Neurofilament Light Chain (NfL) (p<0.05). Increased Neuroprotection: Massive surge in neuroprotective growth factors like GDNF/VEGF (p≤0.001). A total of 23 signals confirm target engagement.
Real-World Survival SHATTERS Norms: The Expanded Access Program (EAP, n=10) shows 100% 5-YEAR survival without tracheostomies, compared to only 20% in the natural history of the disease (p=0.006). The median tracheostomy-free survival is extended to 7 YEARS (a 2-3x extension). Progression was halted for up to 17 months, and 85% of participants saw a SLOWED decline (ALSFRS-R: 0.15 pts/mo vs. natural freefall). Navy veteran Matt Bellina GAINED 6 points on the ALSFRS-R scale after 7 doses.
ALS Trials are Rigged Against Hope: Current trials suffer from small sample sizes (n=200), extreme patient heterogeneity, and the flawed ALSFRS-R questionnaire primary endpoint. NurOwn has shown success despite these barriers.
The TOFERSEN Precedent: Tofersen was approved based on an NfL drop alone for just 2% of the ALS population. NurOwn has demonstrated broader, better survival benefits supported by extensive real-world evidence & ALSO HAD A STATISTICALLY SIGNIFICANT REDUCTION in Nfl.
A conditional approval with a Phase 4 follow-up is the clear path forward.
A Direct Appeal to FDA Leadership
@MartyMakary: You have vowed to combine "gold standard science + common sense" to give rare disease warriors faster access. We are asking you to deliver on that promise for cell therapies.
@RobertKennedyJr: Your crusade for stem cell therapies and your goal to end the FDA's "war" on them directly targets the crisis facing veterans and the underserved who are massacred by ALS.
Thousands are dying during these delays. Fulfill your promise.
Bureaucracy KILLS. Evidence SAVES.
Read the petition: https://t.co/8bjpTsn80q
Convene a meeting TODAY. Give us a fighting chance to LIVE.
#BeyondSignsOfPromise #ApproveNurOwn #EndALS156YearsOfHopelessness
@realDonaldTrump @POTUS @RepGuthrie @RepLarryBucshon @michaelcburgess @RepGusBilirakis @RepBillJohnson @RepRichHudson @RepBuddyCarter @DrNealDunnFL2 @cathymcmorris @LeaderJohnThune @RepDebDingell @RepMMM @SenatorWicker @RepRobinKelly @RepBarragan @DrOz @DrOzCMS @GovBraun @BernieSanders @SenBobCasey @SenatorBaldwin @SenatorHassan @SenTinaSmith @SenMarkey @SenBillCassidy @SenMullin @lisamurkowski @RogerMarshallMD @RepJasonCrow @TeamCalvert @RepBrianFitz @ChrisCoons @RepAndyHarrisMD @DickDurbin @RepDianaDeGette @RepMikeQuigley @RepSchakowsky @rosadelauro @RandPaul @RepDonBacon @SenJohnCurtis @BillCassidy
.@FDACBER @VPrasadMDMPH, you brought hope with your words, but did you mean what you said?
104 DAYS you've had our NUROWN Citizens petition=SILENCE
ALS=100% FATAL since 1869. Patricia #DiedWaiting
@SecKennedy @DrMakaryFDA, Every day of delay=more HORRIFIC deaths WITHOUT HOPE.
No words can convey how HORRIFIC the suffering is while we wait on the FDA
GRANT ACCELERATED APPROVAL for NurOwn NOW.
Our Citizens' Petition, submitted on July 3rd and now ignored for 99 days, demands it. This autologous stem cell therapy—our own cells engineered to flood our brains with life-saving neurotrophins—is ALS's FIRST BREAKTHROUGH in 156 years of hell. The Phase 3b trial was cleared in May 2025, and the data from the 2022 ADCOMM screams for approval.
Irrefutable Evidence
Biomarkers PROVE It Works: Reduced Neuroinflammation: Significant decrease in MCP-1 cytokines (p<0.05). Reduced Neurodegeneration: Significant drop in Neurofilament Light Chain (NfL) (p<0.05). Increased Neuroprotection: Massive surge in neuroprotective growth factors like GDNF/VEGF (p≤0.001). A total of 23 signals confirm target engagement.
Real-World Survival SHATTERS Norms: The Expanded Access Program (EAP, n=10) shows 100% 5-YEAR survival without tracheostomies, compared to only 20% in the natural history of the disease (p=0.006). The median tracheostomy-free survival is extended to 7 YEARS (a 2-3x extension). Progression was halted for up to 17 months, and 85% of participants saw a SLOWED decline (ALSFRS-R: 0.15 pts/mo vs. natural freefall). Navy veteran Matt Bellina GAINED 6 points on the ALSFRS-R scale after 7 doses.
The Case for Approval
ALS Trials are Rigged Against Hope: Current trials suffer from small sample sizes (n=200), extreme patient heterogeneity, and the flawed ALSFRS-R questionnaire as the primary endpoint. NurOwn has shown success despite these barriers.
The TOFERSEN Precedent: Tofersen was approved based on an NfL drop alone for just 2% of the ALS population. NurOwn demonstrated broader, better survival benefits supported by extensive real-world evidence & ALSO HAD A STATISTICALLY SIGNIFICANT REDUCTION IN Nfl.
Accelerated Approval with a Phase 4 follow-up is the clear path forward.
A Direct Appeal to FDA Leadership
@MartyMakary: You have vowed to combine "gold standard science + common sense" to give rare disease warriors faster access. We are asking you to deliver on that promise for cell therapies.
@RobertKennedyJr: Your crusade for stem cell therapies and your goal to end the FDA's "war" on them directly targets the crisis facing veterans and the underserved who are massacred by ALS. Thousands are dying during these delays. Fulfill your promise.
Bureaucracy KILLS. Evidence SAVES.
Read the petition: https://t.co/8bjpTsn80q
Convene a meeting TODAY. Give us a fighting chance to LIVE.
#BeyondSignsOfPromise #ApproveNurOwn #EndALS156Year
@realDonaldTrump @POTUS @RepGuthrie @RepLarryBucshon @michaelcburgess @RepGusBilirakis @RepBillJohnson @RepRichHudson @RepBuddyCarter @DrNealDunnFL2 @cathymcmorris @LeaderJohnThune @RepDebDingell @RepMMM @SenatorWicker @RepRobinKelly @RepBarragan @DrOz @DrOzCMS @GovBraun @BernieSanders @SenBobCasey @SenatorBaldwin @SenatorHassan @SenTinaSmith @SenMarkey @SenBillCassidy @SenMullin @lisamurkowski @RogerMarshallMD @RepJasonCrow @TeamCalvert @RepBrianFitz @ChrisCoons @RepAndyHarrisMD @DickDurbin @RepDianaDeGette @RepMikeQuigley @RepSchakowsky @rosadelauro @RandPaul @RepDonBacon @SenJohnCurtis @BillCassidy
@MinoShah @DrMakaryFDA @SecKennedy @FDACBER You need to take action @DrMakaryFDA @SecKennedy It’s a crime against humanity 10’s of thousands #ALS patients have #DiedWaiting since another petition got 160K+ signatures begging for #NurOwn approval after unprecedented results over 9 years ago. What are you waiting for?
.@DrMakaryFDA @FDACBER @SecKennedy: @pjgreen testified for #NUROWN, was denied by #FDACBER & #DiedWaiting
Delaying the July 3rd citizens petition & @BrainstormCell #BLA refile continues #FDA's history of apathy for the immense suffering & death of ALS patients
If Phil was YOUR family member, facing CERTAIN DEATH but blocked by the #FDA from a treatment that WORKED, what would YOU do?
@realDonaldTrump @RepGuthrie @RepLarryBucshon @michaelcburgess @RepGusBilirakis @RepBillJohnson @RepRichHudson @RepBuddyCarter @DrNealDunnFL2 @cathymcmorris @LeaderJohnThune @RepDebDingell @RepMMM @SenatorWicker @RepRobinKelly @RepBarragan @RepAngieCraig @POTUS @RobertKennedyJr @MartyMakary @SenBobCasey @SenatorBaldwin @SenatorHassan @SenTinaSmith @SenMarkey @SenBillCassidy @SenMullin @lisamurkowski @RogerMarshallMD @RepJasonCrow @TeamCalvert @RepBrianFitz @ChrisCoons @RepAndyHarrisMD @DickDurbin @RepMikeQuigley @RepSchakowsky @rosadelauro @RandPaul @RepDonBacon @SenJohnCurtis
@Cylebo @BrainstormCell @MaryKayTurner1 At what point do pALS deserve to know where NurOwn stands? Upon death? 10's of thousands have #diedwaiting! Upon loss of all function? 10's of thousands have! Upon diagnosis? 20 pALS are diagnosed EVERY DAY in US alone! So, when?
King’nazir was determined to walk before he couldn’t anymore. CHILDREN DIE FROM ALS TOO!! #childhoodALS #diedwaiting #sptlc2 #raredisease
Why early access to whole genome sequencing is needed. #endpediatricALS #diedwaiting #longlivekingnazir
Best treatment discovery in 30yrs & 15mths on still awaiting results publication. Average life expectancy with MND is 2yrs the context screams WRONG on all levels! No wonder those who can afford are buying privately #healthinequality #evensaintsarentthispatient #diedwaiting
We are asking @MNDpatients & supporters to email their MP to ensure the patient voice is heard at @APPGonMND next Weds. Our statement & template message here https://t.co/9z8pEbyMJq Pls read to find out more. @DHSCmedia @NIHRresearch @VictoriaAtkins @BBCBreakfast @joncoates73
Why do we race to #endals ?! For starters, because 2 of the 4 people in this picture I just ran across from Sept of 2021 are gone… And for everyone else who has lost someone to ALS, and for those still fighting. RIP gentlemen.
…
#als #mnd #Racing #diedwaiting
Beautiful tribute by @SRuhle of @11thHour for #healthcare advocate @AdyBarkan
Thx @MSNBC for reminding us that we can all #BeAHero.
Sadly Ady #DiedWaiting for promising drugs stuck in clinical trials. He still had so much more to accomplish. Gone Too Soon.
Prayers for Rachael & their kids.
Is @adybarkan on your mind today? Take 3 minutes to watch the @11thhour @msnbc segment honoring him.
Thank you @sruhle and @mauratweets, Ady would have loved this. ❤️
https://t.co/duRXNstDgT
My husband #diedwaiting
Shame on @DrCaliff_FDA & Director of the @FDACBER, Peter Marks.
There is the blood of #ALS patients on both of your hands.
#NurownWorks #NurownIsSafe #CriticalUnmetNeed #BloodOnYourHands
You killed an effective treatment for hopeless #ALS patients.
Congress, it may be you or your loved one tomorrow who is diagnosed with this DEATH SENTENCE because of this continuation of the CRUEL #FDACBER regulatory RIGIDITY for a disease WITH NO SURVIVORS since 1st identified in 1869.
@PatriciaManhar1 @ShereeWLWT @RepGuthrie @RepLarryBucshon @RepAnnaEshoo @michaelcburgess @RepGusBilirakis @RepBillJohnson @RepRichHudson @RepBuddyCarter @DrNealDunnFL2 @cathymcmorris @RepDebDingell @RepMMM @RepAnnieKuster @RepRobinKelly @RepBarragan @RepAngieCraig @RepKimSchrier @RepLoriTrahan @SenatorWicker @PattyMurray @SenBobCasey @SenatorBaldwin @timkaine @SenatorHassan @SenTinaSmith @SenMarkey @SenBillCassidy @SenMullin @lisamurkowski @SenatorBraun @RogerMarshallMD @RepJasonCrow @TeamCalvert @RepBrianFitz @ChrisCoons @RepAndyHarrisMD @DickDurbin @SenJohnThune @RepDianaDeGette @RepMikeQuigley @RepSchakowsky @rosadelauro @RepJohnCurtis @DorisMatsui @chairmang @RepDonBacon
@DrMayl @onein300 @BrainstormCell @US_FDA Approve with much larger, longer PM study to confirm. Then there will be much clearer picture. Need to get the ball rolling ASAP. Dying patients don’t have time to waste. Some have been fighting 8+ years to access, many have already #diedwaiting , 100+ US MDs wrote yes.
"This is what ALS remission looks like...
Approve NurOwn Now"
~ @MuggliRoberto
https://t.co/sRn2hhWJWz
• Feb 2019, he received 1st dose of #NurOwn at Mayo
• Oct 2019, he recorded this video in MN
• May 2023, he #DiedWaiting
How is this okay in our country?
#NurOwnWorks #NurOwnPreservesFunction🛟
@POTUS @neeratanden @VP
@nadia_sethi Former advocate.. that's me right now, too. I think we need a minute to recupe, ya know? It was damn hard to watch my Trav die. I'm taking my minute.
#hisfightismyfight #diedwaiting #IamALS #TravisLuck #Sayhisname #FALS
CONGRESS & @DrCaliff_FDA, #Nurown (Debamestrocel) AdComm hearing IN 3 DAYS!
Below are just a few examples of the #DiedWaiting & #DyingWaiting.
#ALS can happen to ANYONE at ANY AGE. I hope you will see that #ALS is EVERYONE'S problem, not just those dying now.
Grant NUROWN Accelerated Approval with a Phase 4 postmarketing study.
#NurownWorks #NurownIsSafe #CriticalUnmetNeed
WATCH AdComm HERE: https://t.co/pg9cJCiI3P
ALS IS NOT RARE & is rapidly becoming more common.
The incidence is expected to increase by 70% by 2040.
>90% of diagnosed have NO family history.
You or your loved one could be next.
Ensure an END to the CRUEL @FDACBER regulatory RIGIDITY for 100% PROGRESSIVE & FATAL #ALS.
@RepGuthrie @RepLarryBucshon @RepAnnaEshoo @SenBillCassidy @SenMullin @lisamurkowski @SenatorBraun
@RepJasonCrow @TeamCalvert @RepBrianFitz @RepTerriSewell @ChrisCoons @RepAndyHarrisMD @PattyMurray @SenatorDurbin @SenatorHassan @RepDonBacon @SenSanders @RepMMM @RogerMarshallMD @RepDianaDeGette @RepMikeQuigley @janschakowsky @rosadelauro @RepJohnCurtis @DorisMatsui @RepBarragan @RepAngieCraig @RepKimSchrier @RepLoriTrahan @cathymcmorris @SenAmyKlobuchar @SenatorWicker @SenKevinCramer @JohnBoozman @SteveDaines @RepStephenLynch @RepMariaSalazar @RepJenniffer @RepPaulTonko @RepJohnJoyce @Congressman_JVD @RepDavids @RepChrisPappas @RepGusBilirakis @neeratanden46 @michaelcburgess @SenMarkey
My brother was taken from us almost 8 months ago. He should be here to deliver this message in person #fuckALS #diedwaiting @US_FDA #futuretweet #scheduledtweet
Today is my #daughters 15th #birthday! Very proud of the young lady she is! Never forget how much I Love You! #Forever #futuretweet
The NurOwn AdComm is coming up in just two weeks (September 27th)! Remind the FDA that the ALS community is watching. Sign on to our statement by adding your name, and please SHARE this post to help us reach our goal of 10,000 sign-ons. https://t.co/2wp9RyKfjS
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