Yani Stavrev

[Yani Stavrev @yanistavrev]

Today, America celebrates 250 years of freedom and independence.

But what exactly are you celebrating @POTUS, while thousands of Americans with ALS cannot even stand for the National Anthem? While healthy people dance, laugh, eat and celebrate, ALS patients are dreaming of being able to take even one single step. While some families celebrate, others are watching someone they love lose every function of the human body.

That is the brutal irony of Independence Day: the people who have lost every ounce of independence are forced to watch everyone else celebrate it.

Is this what you meant @SecKennedy, when you promised to Make America Healthy Again?

Today also marks 87 years since Lou Gehrig’s speech. Eighty-seven years later, people with ALS are still dying the same horrific death.

Decades of awareness. Decades of fundraising. Billions raised in the name of ALS. But let me make something very clear: awareness is not a treatment. A fundraiser is not a treatment. Another speech is not a treatment.

ALS patients today do not need promises for the future. They need treatment now — before they lose their voice, their ability to swallow, their breathing, and their last chance.

@FDA_KyleD, every day of delay can mean a feeding tube, a ventilator, a wheelchair, a funeral.

NurOwn should be granted accelerated approval while a confirmatory trial continues. And while you stay silent, people are living inside a nightmare — and dying.

So while America celebrates 250 years of independence, remember the people with ALS who cannot stand, cannot speak, cannot eat, cannot breathe, and cannot even say “Happy Independence Day.”

America, what the hell are you celebrating?

@realDonaldTrump @RobertKennedyJr @JDVance @DrJBhattacharya @DrOz @houmanhemmati @HHSGov @US_FDA @FDACBER

#NurOwnWorks — Approve NurOwn with a Phase 4 confirmatory study for #ALS, a fatal disease with no time to waste.
https://t.co/W8gmyjBASN

#EndALS #DoTheRightThing #PleaseAct #ApproveNurOwn #MAHA #FDADelaysKill

[Suzee Q @SusieM414141]

What it takes to get Steve Gleason on a plane.

This ex-NFL safety has raised more than $65 million for ALS research. He is a hero for what he has done. And I love that he can still smirk. Do you know anyone with ALS?

[Yani Stavrev @yanistavrev]

37 years old.

Brooke Eby didn’t need more “ALS awareness.” She needed a cure.

She didn’t need another conference, another grant, another research program, another promise that scientists are “getting closer,” or another organization telling us about “progress.”

She needed to live.

ALS progressively took away what a human body should be able to do, and at 37 years old, it took her life.

And somehow we have normalized this.

We call it a “battle.” We celebrate “awareness.” We announce “promising research.” We ask for another five years of funding.

Enough.

After decades of research and billions spent, people with ALS are still dying from the same disease.

Stop asking us to celebrate progress while people like Brooke are dying.

Humanity failed to give her what she actually needed: a cure.

Rest in peace, Brooke.

You should still be here.

[Yani Stavrev @yanistavrev]

So this is the victory? Another five years of funding for “research”?

ACT for ALS was passed in 2021. Now another five years, through 2031, have been secured for more research, more grants, more programs, and more promises about accelerating toward a cure.

I hope I’m wrong, but I already know what I expect to see in 2031:

No cure. Another memorial list. And another campaign asking Congress to reauthorize ACT for ALS for another five years.

Then 2036. Then 2041.

At what point do we stop calling the continuation of research infrastructure a victory and start demanding an actual outcome?

I watch ALS organizations constantly asking for ACT for ALS, more programs, more funding, more advocacy, more research.

Yet I almost never see the same uncompromising demand:

CURE ALS.

People with ALS are not research programs. They don’t have five-year authorization cycles.

They are dying NOW.

[Yani Stavrev @yanistavrev]

“Free from the illness” because she died is exactly the point.
Death is not treatment. It is not recovery. And it is not the kind of “freedom” anyone with ALS should have to wait for.
ALS took her health, her independence, and finally her life.
Maybe saying “she’s free” brings some comfort. I understand that.
But the harder truth is this:
She should still be here.
She deserved treatment. She deserved recovery. She deserved more time with the people she loved.
The goal should not be to make death easier to accept.
The goal should be to stop ALS from killing people.

[Rain Consulting Group @RainConsulting]

Thousands continue to die of this disease. It’s progressive and 100% fatal. The FDA absolutely needs to approve the NurOwn Citizen Petition based on the totality of evidence. It’s long overdue for a response from FDA. It’s currently the only treatment known to pause progression and improve function in ALS.

[Yani Stavrev @yanistavrev]

@alsadvocacy @limpbroozkit Relyvrio turned out to be about as useful for ALS as your account and your posts about ALS: a lot of noise, a lot of confidence, and zero actual value. The difference is that Relyvrio at least had the decency to get withdrawn after the evidence failed.

[Yani Stavrev @yanistavrev]

NAME THE GROUPS.

If advocacy organizations actually urged against NurOwn approval at the last minute, patients and families deserve to know exactly who they were. Not someday. Now.

Because the deeper I dig into the ALS establishment, the harder it becomes to ignore what is happening right in front of us.

Everyone should read this new Neurology paper:

“Repairing the Amyotrophic Lateral Sclerosis Trial Credibility Gap.”

The authors lecture the ALS community about “one-drug advocacy,” “demands for immediate access,” and the supposed “significant harm” caused when desperate patients begin viewing regulators as barriers rather than “accountability partners.”

And what do they cite for that claim?

REFERENCE 26.

THE NurOwn CITIZEN PETITION.

Let that sink in.

A petition from people fighting for access to a treatment for a relentlessly progressive, fatal disease is literally used as an example in a section called “Real-World Harms.”

What exactly is the “harm” here?

Patients demanding that FDA examine the TOTALITY OF EVIDENCE?

Patients refusing to quietly deteriorate and die while another multi-year trial runs?

Patients questioning a regulatory system after watching generation after generation of ALS patients lose their ability to walk, speak, swallow and breathe while being told to wait for “more rigorous evidence”?

THAT is the problem?

ALS itself apparently isn't enough “real-world harm.”

Dying while waiting apparently isn't enough “real-world harm.”

Years of regulatory delay apparently aren't enough “real-world harm.”

But patients becoming angry and demanding access? Now THAT earns a warning in Neurology.

Give me a break.

And there is another irony here.

The paper warns about a “clinical trial industrial complex” and “perverse incentives.” Fine. Then let's examine incentives everywhere — including inside the establishment making this argument.

The paper's own disclosures list consulting relationships, industry affiliations, intellectual-property relationships and leadership positions involving pharmaceutical companies and ALS organizations among several of its authors.

That does NOT automatically mean anyone did anything wrong.

But if you are going to lecture dying patients about “perverse incentives,” then you had better be prepared for patients to examine YOUR incentives too.

Scientific rigor matters.

Primary endpoints matter.

Randomization matters.

Nobody is asking scientists to manufacture evidence that does not exist.

But scientific rigor is NOT the same thing as pretending that the risk of doing nothing is zero.

In ALS, delay is not neutral.

WAITING IS AN INTERVENTION.

And its adverse events are paralysis, loss of speech, loss of swallowing, respiratory failure and death.

NurOwn did not meet its Phase 3 primary endpoint in the overall population. That is a fact.

But that is NOT the entire evidence base.

There are randomized data, biomarker data, subgroup signals, responder analyses, expanded-access experience and subsequent evidence that deserve scientific examination — not rhetorical dismissal of the patients asking FDA to look at the totality of evidence.

If the authors believe the NurOwn Citizen Petition is scientifically wrong, then challenge its evidence point by point.

Show us where the petition is wrong.

Show us which evidence is invalid.

Show us which analysis cannot be reproduced.

Show us why conditional access with mandatory continued data collection would create greater risk than forcing patients to wait years for another trial.

But stop treating desperate patients exercising their legal right to petition their government as though THEY are the credibility problem in ALS.

The credibility problem is much bigger than that.

After decades of research, enormous funding, countless conferences, grants, papers, biomarkers, models and trials, ALS patients still face a disease with no general cure and devastating mortality.

The system is entitled to demand evidence from drug developers.

Patients are equally entitled to demand RESULTS from the system.

So @RainConsulting — if your source is correct that @realDonaldTrump wanted NurOwn approved, and advocacy organizations urged against it at the last minute…

NAME THEM.

Patients deserve to know who spoke in their name, what they said, what evidence they relied on, and why.

Sunlight works both ways.

@kelsieswrites — you owe ALS patients an explanation.

@nadia_sethi — you owe ALS patients an explanation.

@VPResearch_ALS — you owe ALS patients an explanation.

@profcjmcdermott — you owe ALS patients an explanation.

@Rothstein_Lab — you owe ALS patients an explanation.

Michael Benatar, Kuldip Dave and Thos Cochrane — you owe ALS patients an explanation too.

You chose the NurOwn Citizen Petition as an example of “real-world harms.”

Then defend that choice publicly.

Explain exactly what you believe these patients got wrong.

Explain what evidence you believe they misunderstood.

And explain why patients demanding access to a potentially beneficial treatment deserve to be discussed under “harms,” while the irreversible harm caused by years of waiting receives nowhere near the same rhetorical force.

You put their advocacy into the scientific literature.

Now answer them.

Sunlight works both ways.

@realDonaldTrump @RobertKennedyJr @FDA_KyleD @JDVance @DrJBhattacharya @DrOz @houmanhemmati @HHSGov @FDA @FDACBER

#NurownWorks -Approve with Phase 4 study for 100% fatal #ALS patients
https://t.co/W8gmyjBASN

#EndALS #DoTheRightThing #PleaseAct

[Rain Consulting Group @RainConsulting]

Here's another key stat for ya. 0% cure rate of ALS before Trump and 0% with Trump. A big reason for this is because effective treatments continue to sit on the shelf as patients die waiting. I also saw from a credited source that Trump actually wanted NurOwn approved, but certain Advocacy groups recommended against it last minute. (think about that for a second) Things have changed substantially since then, NurOwn has much more data within its totality of evidence showing it prolongs life. Yet here we are. Still waiting. Patients still dying of this 100% fatal disease. The NurOwn Citizens Petition is still pending. Incredible. @PeterPitts @LoriMills4CA42 @FDA_KyleD

[Craig Reagan @CraigReagan2]

@SecKennedy @FDA_KyleD @BrainstormCell @Cylebo @RobertKennedyJr
ALS IS INFINITELY WORSE THAN THIS! Think about that. APPROVE NUROWN NOW!!

[No More Excuses ALS Watchdogs! @als_now]

YGTBFKM. When patients "disrupted" #ALS (fighting for REAL HOPE (not blend$ of Cipro or supplement$), it was: 1) "divisive" 2) "angry" 3) "counterproductive", etc. Now, some PhD who supported #Relyvrio but hates #NurOwn wants to "disrupt" #ALS? Yep, #ALS hope is FUBAR. 🤪🤡😜

[Shah Minokadeh, M.D. @MinoShah]

.@HeidiOverton @FDADrugs @SecKennedy @FDA_KyleD

Cardiologists don't operate on brains. Yet FDA generalists keep deciding the fate of boys with Duchenne & people with ALS

I wrote this with my eyes as a ventilator-dependent physician with ALS. Melanoma specialists saved patients last month. Rare-disease specialists were ignored

Read this. Then grant accelerated approval to deramiocel, schedule the Huntington’s and ataxia adcomms, and answer the NurOwn Citizens’ Petition that already has eight years of survival data (& is over 9 MONTHS DELAYED by the FDA's own deadline for responding). #DyingWaiting

Specialization is not a luxury in terminal rare disease. It is the difference between life and death.

@realDonaldTrump @RepGuthrie @DrOz @DrOzCMS @SenRonJohnson @ChuckGrassley @RobertKennedyJr @RonJohnsonWI @RepBuddyCarter @DrNealDunnFL2 @LeaderJohnThune @RepDebDingell @RepMMM @SenatorWicker @RepRobinKelly @RepBarragan @RepAngieCraig @RepKimSchrier @RepLoriTrahan @SenatorBaldwin @SenatorHassan @SenTinaSmith @SenMarkey @lisamurkowski @RogerMarshallMD @RepJasonCrow @TeamCalvert @RepBrianFitz @ChrisCoons @RepAndyHarrisMD @DickDurbin @RepMikeQuigley @RepSchakowsky @rosadelauro @RandPaul @RepDonBacon @SenJohnCurtis @PeterPitts @LauraMclinn @FDA @FDACommissioner

[Yani Stavrev @yanistavrev]

I’m sorry, but I don’t share the optimism.

If Marty Makary was removed because his approach damaged trust and failed patients with serious and rare diseases, replacing him with someone widely described as his protégé raises an obvious question: what exactly is supposed to change?

The ALS community cannot afford another experiment in leadership. We cannot wait through confirmation, then wait months for action, only to discover that we have Makary 2.0 and have to start over again.

NurOwn’s citizen petition is already sitting unresolved. Patients do not have the luxury of giving every new FDA Commissioner a year to prove whether they understand urgency.

If Dr. Overton truly represents a break from the Makary era, that needs to be demonstrated immediately and concretely — not through speeches, but through decisions.

At this point, I see no reason to assume that appointing Makary’s protégé represents reform. It looks far more like continuity.

And if this is the strongest candidate they could find to lead the FDA after everything that has happened, that itself is deeply concerning.

[Greg Heydet Sr @GregHeydetSr]

@CraigReagan2 @SecRollins @SecKennedy @MinoShah @Vita_Victoria2 @RainConsulting @Humbl4now @juan23188549 @SecKennedy shows he doesn’t care! Trump signed the Right to Try bill in 2017! Follow Trump’s lead & approve NurOwn! Or I will pray to God that u get ALS! God’s watching u not give patients a chance at life. NurOwn has proven itself 2 many trial patients! Please approve! 🙏🙏🙏

[Rain Consulting Group @RainConsulting]

End the era of terminal. Start the era of treatable. Approve the NurOwn Citizen Petition.

It’s either the beginning of the end of one of humanities worst diseases OR just more guaranteed death.

Will this be the administration that changes the course of one of the worlds deadliest diseases? @SecKennedy @FDACBER @FDA_KyleD @realDonaldTrump

[Yani Stavrev @yanistavrev]

@bsw5020 I deeply doubt that.

[Craig Reagan @CraigReagan2]

@FDA_KyleD @SecKennedy If you have to search this long, and this hard, to say "NO" to the Citizen's Petition for NurOwn (debamestrocel), the only clear answer is "YES"! Think about that. Give us the CHANCE to recover any amount! Why are you making this so hard? We are dying!!

[No More Excuses ALS Watchdogs! @als_now]

@FDACBER Too little too late for @ALS. Thanks for nothing, signed the 29,699 #ALS patients still with no real hope.

[Yani Stavrev @yanistavrev]

This is an important piece, and I agree that rare disease patients need urgency, not bureaucracy.

Right to Try 2.0 is an important step and offers real hope, but it cannot be the final answer. It may help some patients, but a pathway that depends on individual exceptions is not true access. Patients should not have to win a lottery for a chance to try.

It also does not guarantee reimbursement, manufacturing scale, global access, or that a company can or will provide the treatment.

For serious rare diseases with no meaningful options, when a therapy shows a reasonable signal of benefit and an acceptable safety profile, FDA should use accelerated approval and allow broad access while a rigorous confirmatory Phase 4 trial runs.

That is the real goal: broad access, real data, and hope before irreversible decline takes everything away.

[Yani Stavrev @yanistavrev]

Dr. Oz, fighter jets, fireworks, ambassadors, parties and photos may look like a celebration of freedom.

But for Americans with ALS, freedom has a very different meaning.

It means being able to stand, walk, speak, swallow, breathe, hug your family, and live with basic independence — the very things ALS takes away piece by piece.

While some people were celebrating over the National Mall, ALS patients were trapped in bodies that are failing them, and families were watching people they love suffer through a disease that is still 100% fatal.

Strengthening the health of the American people must include ALS patients. Not someday. Not in some future breakthrough. NOW.

Awareness is not a treatment. Fundraising is not a treatment. Another speech is not a treatment.

NurOwn should be granted accelerated approval while a Phase 4 confirmatory study continues.

#ApproveNurOwn #NurOwnWorks #ALS #FDADelaysKill #DyingWaiting

[Yani Stavrev @yanistavrev]

Today, America celebrates 250 years of freedom and independence.

But what exactly are you celebrating @POTUS, while thousands of Americans with ALS cannot even stand for the National Anthem? While healthy people dance, laugh, eat and celebrate, ALS patients are dreaming of being able to take even one single step. While some families celebrate, others are watching someone they love lose every function of the human body.

That is the brutal irony of Independence Day: the people who have lost every ounce of independence are forced to watch everyone else celebrate it.

Is this what you meant @SecKennedy, when you promised to Make America Healthy Again?

Today also marks 87 years since Lou Gehrig’s speech. Eighty-seven years later, people with ALS are still dying the same horrific death.

Decades of awareness. Decades of fundraising. Billions raised in the name of ALS. But let me make something very clear: awareness is not a treatment. A fundraiser is not a treatment. Another speech is not a treatment.

ALS patients today do not need promises for the future. They need treatment now — before they lose their voice, their ability to swallow, their breathing, and their last chance.

@FDA_KyleD, every day of delay can mean a feeding tube, a ventilator, a wheelchair, a funeral.

NurOwn should be granted accelerated approval while a confirmatory trial continues. And while you stay silent, people are living inside a nightmare — and dying.

So while America celebrates 250 years of independence, remember the people with ALS who cannot stand, cannot speak, cannot eat, cannot breathe, and cannot even say “Happy Independence Day.”

America, what the hell are you celebrating?

@realDonaldTrump @RobertKennedyJr @JDVance @DrJBhattacharya @DrOz @houmanhemmati @HHSGov @US_FDA @FDACBER

#NurOwnWorks — Approve NurOwn with a Phase 4 confirmatory study for #ALS, a fatal disease with no time to waste.
https://t.co/W8gmyjBASN

#EndALS #DoTheRightThing #PleaseAct #ApproveNurOwn #MAHA #FDADelaysKill

[Yani Stavrev @yanistavrev]

Dr. Bhattacharya, fireworks over DC are beautiful — but for Americans with ALS, there is no celebration in losing the ability to stand, speak, swallow, breathe, or live with basic independence.

This Independence Day, America celebrates 250 years of freedom while ALS patients are trapped inside bodies that are failing them piece by piece.

87 years after Lou Gehrig’s speech, people with ALS are still dying the same horrific death.

As NIH Director, please help make ALS an urgent national priority. Awareness is not enough. Fundraising is not enough. Promises about future breakthroughs are not enough.

ALS patients today need action now.

Please push for urgent federal focus on NurOwn, the Citizens’ Petition, biomarker evidence, repeated-dosing evidence, EAP/Right-to-Try data, and every serious path that could help patients before they lose their last chance.

NurOwn should be granted accelerated approval while a Phase 4 confirmatory study continues.

#ApproveNurOwn #NurOwnWorks #ALS #FDADelaysKill #DyingWaiting

[Yani Stavrev @yanistavrev]

Today, America celebrates 250 years of freedom and independence.

But what exactly are you celebrating @POTUS, while thousands of Americans with ALS cannot even stand for the National Anthem? While healthy people dance, laugh, eat and celebrate, ALS patients are dreaming of being able to take even one single step. While some families celebrate, others are watching someone they love lose every function of the human body.

That is the brutal irony of Independence Day: the people who have lost every ounce of independence are forced to watch everyone else celebrate it.

Is this what you meant @SecKennedy, when you promised to Make America Healthy Again?

Today also marks 87 years since Lou Gehrig’s speech. Eighty-seven years later, people with ALS are still dying the same horrific death.

Decades of awareness. Decades of fundraising. Billions raised in the name of ALS. But let me make something very clear: awareness is not a treatment. A fundraiser is not a treatment. Another speech is not a treatment.

ALS patients today do not need promises for the future. They need treatment now — before they lose their voice, their ability to swallow, their breathing, and their last chance.

@FDA_KyleD, every day of delay can mean a feeding tube, a ventilator, a wheelchair, a funeral.

NurOwn should be granted accelerated approval while a confirmatory trial continues. And while you stay silent, people are living inside a nightmare — and dying.

So while America celebrates 250 years of independence, remember the people with ALS who cannot stand, cannot speak, cannot eat, cannot breathe, and cannot even say “Happy Independence Day.”

America, what the hell are you celebrating?

@realDonaldTrump @RobertKennedyJr @JDVance @DrJBhattacharya @DrOz @houmanhemmati @HHSGov @US_FDA @FDACBER

#NurOwnWorks — Approve NurOwn with a Phase 4 confirmatory study for #ALS, a fatal disease with no time to waste.
https://t.co/W8gmyjBASN

#EndALS #DoTheRightThing #PleaseAct #ApproveNurOwn #MAHA #FDADelaysKill

[Yani Stavrev @yanistavrev]

HHS, a “healthier future” means nothing to ALS patients who may not live long enough to see it.

This Independence Day, America celebrates 250 years of freedom while thousands of Americans with ALS have lost every ounce of theirs.

They cannot stand, speak, swallow, eat, breathe, or even say “Happy 250th, America.”

87 years after Lou Gehrig’s speech, people with ALS are still dying the same horrific death.

ALS patients do not need promises for the next 250 years. They need action now.

Please urgently review NurOwn and the Citizens’ Petition.

NurOwn should be granted accelerated approval while a Phase 4 confirmatory study continues.

#ApproveNurOwn #NurOwnWorks #ALS #FDADelaysKill #DyingWaiting

https://t.co/T8zd66EKul

[Yani Stavrev @yanistavrev]

Today, America celebrates 250 years of freedom and independence.

But what exactly are you celebrating @POTUS, while thousands of Americans with ALS cannot even stand for the National Anthem? While healthy people dance, laugh, eat and celebrate, ALS patients are dreaming of being able to take even one single step. While some families celebrate, others are watching someone they love lose every function of the human body.

That is the brutal irony of Independence Day: the people who have lost every ounce of independence are forced to watch everyone else celebrate it.

Is this what you meant @SecKennedy, when you promised to Make America Healthy Again?

Today also marks 87 years since Lou Gehrig’s speech. Eighty-seven years later, people with ALS are still dying the same horrific death.

Decades of awareness. Decades of fundraising. Billions raised in the name of ALS. But let me make something very clear: awareness is not a treatment. A fundraiser is not a treatment. Another speech is not a treatment.

ALS patients today do not need promises for the future. They need treatment now — before they lose their voice, their ability to swallow, their breathing, and their last chance.

@FDA_KyleD, every day of delay can mean a feeding tube, a ventilator, a wheelchair, a funeral.

NurOwn should be granted accelerated approval while a confirmatory trial continues. And while you stay silent, people are living inside a nightmare — and dying.

So while America celebrates 250 years of independence, remember the people with ALS who cannot stand, cannot speak, cannot eat, cannot breathe, and cannot even say “Happy Independence Day.”

America, what the hell are you celebrating?

@realDonaldTrump @RobertKennedyJr @JDVance @DrJBhattacharya @DrOz @houmanhemmati @HHSGov @US_FDA @FDACBER

#NurOwnWorks — Approve NurOwn with a Phase 4 confirmatory study for #ALS, a fatal disease with no time to waste.
https://t.co/W8gmyjBASN

#EndALS #DoTheRightThing #PleaseAct #ApproveNurOwn #MAHA #FDADelaysKill