Ajay Sampat, MD

Father, husband, son, and brother. Clinical educator and neurologist. Person living with ALS.

[AZ Latina ☮️🌊🟦 @aVoice4ALS]

The #NurOwn AdCom on Sept 27th was like:

Russian trial of an American citizen... Then
Pharma media was like Russian TV reporting on the Ukraine War.

Pharma media has NOT done Due Diligence. They:
🔹Don't understand #ALS
🔹Don't understand flawed subjective tool used for endpoints
🔹Disregarded the Neurologists' testimony
🔹Disregarded the 2002 Public Comments
🔹Ignored patients in trial who have videos of improvement in function showing them outliving 2-5 yr natural history & clinically documented #RWD and #RealWorldEvidence

Pharma media should have:

1️⃣ Interviewed & quoted AdCom speaker & PI
Dr Tony Windebank of @MayoClinic who has 40+ yrs experience treating people with ALS & testified that NurOwn stabilized loss of function & helped some regain function -- something he's never seen in ALS trials.

2️⃣ Interviewed treating neurologists who actually saw the patients in the trial like @DGeeEss1
https://t.co/ZFycwjYuA5

3️⃣ Interviewed people who have actual experience with #NurOwn like you, @klink52, @nicolecimbura , @pjgreen @stevens_nation, @Smithstrongmom1, @kks69okie

4️⃣ Interviewed MDs with #ALS to help them understand the problems in ALS trials
• Neurologist @Sampat_md
• Former Pharma Exec @BoulderMike29
• Anesthesiologist @MinoSean
• Cardiologist & Clinical Trialist @DrMarcLitt1
https://t.co/CBO0TDntKz
https://t.co/WcU1NEtJ91
https://t.co/WOo66NelUI
https://t.co/jeObyuPLvs

5️⃣ Interviewed neurologist from @TempleUniv who actually treats people with ALS & wrote a Public Comment in support of approval
https://t.co/gQLD4kl2r7

6️⃣ Interviewed PhD ALS researcher @Ozdinler of @NorthwesternMed who submitted Public Comment in support of approval
https://t.co/88HB1YDBv1

7️⃣ Interviewed MD PhD neuroscientist who has family member with ALS &who explained many of the flaws in the trial with his 28-page Public Comment filed August 9th -- 48 days before the AdCom
https://t.co/BfIFNiUWjy

8️⃣ Talked to the many people identified in this summary that makes it easy to read the most relevant Public Comments (that AdCom members obviously didnt read)
https://t.co/MgEmkRx8S8

Please investigate & report complete story about ALS and the NurOwn trial:
@statnews @endpts @PharmaPinkSheet @medpagetoday @drsanjaygupta @maddow @CBSSunday @TheTodayShow @TheProblem @NYTHealth @WillieGeist @chicagotribune

@POTUS @VP @neeratanden @RepAnnaEshoo @ChrisCoons @RepMikeQuigley @RepSchakowsky @SenatorWicker @SenAmyKlobuchar @SenTinaSmith @SenJohnThune @rosadelauro @RepAndyHarrisMD @RepBradWenstrup @DorisMatsui @RepLBR @cathymcmorris

@HouseDemocrats @HouseGOP @HouseCommerce @EnergyCommerce

[AZ Latina ☮️🌊🟦 @aVoice4ALS]

Media reporting on #NurOwn must read response to @MedPageNeuro by @MinoSean, a Johns Hopkins trained anesthesiologist.

Yes, subgroup of 27/48 score was not pre-specified, but for God's sake, people with #ALS are dying. Oncology regularly uses post hoc & CDER used post hoc or other ALS drugs.

Which analysis do you think is more humane?
Post hoc or post-mortem?

And since when does unbiased media IGNORE the opinion of a @MayoClinic #neurologist & trial PI with 40+ years experience ... who has more experience with #ALS than a single person on the 19-person Advisory Committee meeting?

Dr. Tony Windebank testified that NurOwn worked on a "substantial number" that he observed in his clinic and other PIs in the trial had same observations. He added that he witnessed stabilization that he has never seen changes in any ALS trial - ever.

Dr. Windebank's clinical observations corroborate the #RWD & testimony of the only people in the world who have ever had NurOwn in their body, and they filed Public Comments with improvements in function with video & documentary proof #RWE that has never happened in ALS trials... we have PROOF that AdCom members didn't bother to read Public Comments.

The MOA discussion is irrelevant to approval & inappropriate to be discussed at AdCom. There are a lot o drugs on the market that work & scientists don't understand MOA. Patients should not be allowed to die simply because scientists don't understand heterogeneity & pathology of ALS. That happens in #RareDiseases.

Only issues to be discussed at AdCom are:
(1) is it safe
(2) does it improve how people feel, function or survive?
(3) does benefit outweigh the risk?

BLATANTLY INCORRECT FACTS:
1. Dismissing patients' profound RWE, AdCom members said patients' improvement could be "by chance" because people with #ALS routinely regain function. WRONG.

2. FDA repeatedly stated NurOwn didnt meet any endpoints. WRONG. The trial met pre-specified secondary endpoint of ≥35.

DUE PROCESS DENIED:
1. FDA "Lottery" deprived people of Due Process rights to testify about how NurOwn improved their function.

2. Throughout the day, FDA denied Brainstorm chance to rebut incorrect statements & answer questions raised by AdCom members.

Please report these facts!
#NurOwnWorks but the regulatory process didn't

@RepAndyHarrisMD @DrNealDunnFL2 @RepJohnJoyce @RepBradWenstrup @RepJimBaird @michaelcburgess @RepGregMurphy @millermeeks @RepGuthrie @SenBillCassidy @SenJohnBarrasso @SenatorWicker @SenJohnThune @ChrisCoons @SenatorBraun @SenWhitehouse @SenAngusKing @SenAmyKlobuchar @RepAnnaEshoo @RepSchakowsky @RepLBR @rosadelauro @RepJohnCurtis @DorisMatsui @cathymcmorris @SenatorDurbin @SenSanders @bsw5020 @dantate2

@BioCentury @biospace @endpts @medpagetoday

[AZ Latina ☮️🌊🟦 @aVoice4ALS]

Don't you DARE say you thought the patients' testimony was "compelling" or you are "sympathetic" or you "understand" their pain or that you were "moved" by them or "your heart bleeds."

INCREMENTAL changes aren't enough?
You don't get to decide what matters to patients when you have no loss of function.

INCREASED MORTALITY? No kidding.
People have #ALS. They die in 2-5 years.
WTH don't you understand about that?

OPTIMISTIC about the Drug Pipeline? When? Another decade after everyone alive today will be dead?

This is a heterogeneous disease.
No drug is going to work on everyone.

The people in the trial -- and their neurologists -- told you & showed you the difference in their QOL. They showed you SUBSTANTIAL EVIDENCE & you chose to ignore it.

#NurOwnAdCom
#NurOwnWorks but FDA regulatory process doesn't.

Time to change the laws!

[AZ Latina ☮️🌊🟦 @aVoice4ALS]

In all due respect, it seems straightforward to approve #NurOwn, whose benefit to risk ratio is high.

~ Dr Marc Lenaerts, MD
Vice Chair #Neurology at UC Davis with 30+ yrs experience

#NurOwnWorks
#NurOwnPreservesFunction🛟
#ALS #neurotwitter

https://t.co/MmVwTSjE3J

@FDACBER @DrCaliff_FDA @RepGaramendi
@RepAndyHarrisMD @RepJohnJoyce @RepBradWenstrup @RepMarkGreen @millermeeks @michaelcburgess @DrNealDunnFL2 @RepLarryBucshon @RepHarshbarger @DesJarlaisTN04 @RepJimBaird @RepMcCormick

[AZ Latina ☮️🌊🟦 @aVoice4ALS]

WOW! Download & read this powerful statement by @PatriciaManhar1 in support of #NurOwn's approval.

https://t.co/Mv7ewzjGiQ

#NurOwnWorks
#NurOwnPreservesFunction🛟
#ALSclock

@FDACBER
@DrCaliff_FDA
@RepBradWenstrup

[AZ Latina ☮️🌊🟦 @aVoice4ALS]

As a #neurologist with expertise in neurodegenerative diseases, having witnessed serial failures due to inabilities of therapies to cross the BBB, the impact that this successful therapeutic option (#NurOwn) has to halt the progression in #ALS is not one to be ignored.
~ William Benko, MD

#NurOwnPreservesFunction🛟
#NurOwnWorks #neurotwitter

https://t.co/rvlJ66Uae1

[Ajay Sampat, MD @Sampat_md]

"There is an unmet clinical need in ALS that by many measures exceeds that of many malignancies...the clinical data looks very promising, and exceeds the benchmarks which have led to the approval of many drugs in oncology."

-Dr Koshkin, UCSF oncologist.

PLEASE REPOST!!

[Ajay Sampat, MD @Sampat_md]

"Making this product available to patients while validation of this retrospective analysis is carried out seems warranted. Our patients will be the ultimate beneficiaries."

-Dr. Appel, one of the world's preeminent ALS researchers

[Katie Coleman @kaydaustin]

Cancer ghosting, let’s talk about it 👻

I took these two pictures after my first hospital admission then scrolled through my feed and cried. At 29, I found myself using a bedpan while I watched friends skiing down mountainsides. It was the first time I’d recognize how detached my life had become from my peers.

I felt jealous, bitter and threw myself a pity party over chicken broth and graham crackers while the extreme guilt set in over the fact I even had any of these emotions.

When you’re first diagnosed with cancer, your friends, family and even distant acquaintances often rally around you. The messages, phone calls and well wishes pour in, making you feel insanely supported.

But over time the support often dwindles. People go back to their lives, full of events and activities you may no longer be able to participate in. And all the “if you need anything” messages start to feel empty as you look on from afar.

There are often people you thought would be there every step of the way- who you never or rarely hear from. It can feel crushing and make you question if they ever really cared.

Here’s why I don’t fault the people in my life I experienced this with and a few insights I’ve taken away. I thought I’d share in case it’s helpful to others.

I started to feel less hurt by these experiences when I realized we all have busy lives and most people are doing their best to make it through their own day. We live in a world these days where many of us turn to social media to keep tabs on each other. Many of my friends and family saw updates online or were updated through the grapevine. They felt up-to-date on me, even though we never had contact. Others later told me, “Phewf, I’m glad you’re doing okay now. I had to stop reading your posts because it was too hard for me. I kept picturing everything that happened to you, happening to myself or my kids. I emotionally wasn’t strong enough for it. I prayed for you every night but couldn’t read your posts.” Others were at a loss for words and didn’t know what to say.

Look, cancer is hard- really hard. Not just for those of us going through it but for many of those we care about too. I personally believe it’s not about people not caring, I think it’s often people not knowing how to show up or what to say.

I think social media often amplifies the emotions and isolation. When you see others carrying on with their lives while you’re simply trying to survive, it can at times feel like they don’t care.

But what social media doesn’t show are the prayers they send up each night praying for your healing and comfort, the little reminders throughout their day that make them think of you or their compulsive checking of your page at 1am for updates looking reassurance you’re okay.

Social media has created ways for us to feel more connected than we ever have before but it can also create a valley of isolation through the hardest days.

So this is my reminder to anyone who may have loved ones they care about going through a difficult time. Don’t forget to drop the comment, send the message or pick up the phone. If you don’t know what to say, simply let them know you’re thinking about them.

Cancer ghosting I personally believe is often unintentional but a very real experience many face. I’d love for us to create a thread of practical ways to show up for others. So let’s hear your experience below. What are little ways people showed up for you during a difficult time that mean the most?

[Ajay Sampat, MD @Sampat_md]

"Why limit access to a potential therapy, even if it only benefits a subset of patients? Any therapy that can help a patient and their family slow down or reverse the progression of this disease should be left up to those affected by ALS."

#NurownIsSafe #NurownWorks

[AZ Latina ☮️🌊🟦 @aVoice4ALS]

"Your decision has the power to rewrite the narrative for #ALS patients. Please let it be one of hope, compassion & a testament to the value of every human life."
~ 24 yr old's plea for #NurOwn

https://t.co/yBq0wc8L5A

@FDACBER @DrCaliff_FDA help him
@CDCEnvironment count him

[Ajay Sampat, MD @Sampat_md]

"Debamestrocel also made a difference in the actual CSF biomarkers. This is big. This means that, unlike the placebo, it is actually changing the push and pull between the body and the disease and we are actually making progress against the disease itself."

#NurOwnWorks

[Ajay Sampat, MD @Sampat_md]

"FDA’s risk-benefit calculation in ALS must be different than it is for therapies developed for non-life-threatening conditions. There is almost no risk worse than imminent death from ALS."

#NurownIsSafe #NurOwnWorks

[Ajay Sampat, MD @Sampat_md]

"The fact that benefit was most notable in those early in their disease course is uniquely compelling rather than discouraging since earlier interventions have higher chances of modifying the disease course in many neurological diseases."

#NurownIsSafe #NurownWorks

[Ajay Sampat, MD @Sampat_md]

Please review my letter to the @FDACBER and @DrCaliff_FDA in strong support of debamestrocel (“NurOwn”).

https://t.co/PBZMTX4IT8

@RepGuthrie @RepAnnaEshoo  @SenBillCassidy @SenMullin @lisamurkowski @SenatorBraun @RepJasonCrow @TeamCalvert @RepBrianFitz @RepTerriSewell @bsw5020

[Ajay Sampat, MD @Sampat_md]

@WinnaPig @MinoSean @bsw5020 Who doesn't know the famous @bsw5020 !

[Ajay Sampat, MD @Sampat_md]

@PippyOp I hope to have the opportunity to!

[Ajay Sampat, MD @Sampat_md]

@onein300 @brainmatters10 @BartBaker @angelicapeebles @PMelmeyer https://t.co/Q3Ltrz61Pp

[Ajay Sampat, MD @Sampat_md]

@onein300 @brainmatters10 @BartBaker @angelicapeebles @PMelmeyer A phase 4 postmarketing study would allow for a larger pool to be analyzed, something that even another phase 3 trial cannot do. 2/2

[Ajay Sampat, MD @Sampat_md]

@onein300 @brainmatters10 @BartBaker @angelicapeebles @PMelmeyer Agreed, in an ideal world longer trial is optimal. But biomarker evidence shows OBJECTIVE cellular response to treatment at all levels of progression and anecdotal evidence shows some people with less severe disease clearly stabilizing. 1/2