Allow people with ALS access to the drug NurOwn!

Allow people with ALS access to the drug NurOwn!

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No More Excuses ALS started this petition to US FDA US FDA and

IMMEDIATE ACTION NEEDED FOR THE ALS DRUG "NUROWN":

"ALS" (aka Amytrophic Lateral Sclerosis, aka "Lou Gherig's Disease") is an always fatal, catastrophic disease that causes death in 100% of diagnosed cases. Death occurs on average between 2 to 5 years after initial diagnosis. About 6 in every 100,000 people contract the disease across the world, and our military veterans (and even athletes) get ALS at a rate of about 2:1 over civilians.

ALS causes all motor neurons in the human body to die, leaving victims 100% paralyzed and unable to care for themselves. They remain immobile until death, but completely aware of the their situation. For the first time in history, a new biologic compound exists that has shown remarkable progress- NurOwn has slowed and even halted the progression of this evil, insidious disease in some. NurOwn is not a cure, but a single cure for ALS is unlikely due to the heterogeneous nature of the disease. No two cases are the same, but they all end the same way- in death. 

But NurOwn "failed", right?

No. The headlines said that. Although NurOwn did not meet its exceptionally high primary and secondary endpoint (a 125% improvement in the rate of decline), this is not uncommon for ALS in the least. Over 85 new drugs have now "failed" for ALS, even though some experts agree it is likely some of these drugs would have been successful had  ALS' "heterogeneity" problem seen in trials been accounted for properly during analysis of the data. NurOwn's 15 years in development recently ended in a promising Phase 3 trial, where NurOwn was proven to be clinically significant (p=.05) for a group of ALS patients who were above 26 on the ALSFRS-R scale, for whom NurOwn made as much as a 2-3 point difference in progression rate for 7 months during the trial. This kind of difference is huge for ALS patients.

Just recently, a new ALS drug called "AMX0035" was approved using regulatory flexibility from the FDA. AMX0035 (Relyvrio) is actually a combination of two already existing and relatively inexpensive compounds which added about 5 or 6 months of life to the average ALS patient, at best. It should be noted that AMX0035 also "failed" to meet its pre-specified secondary endpoint, as noted in the data. One of the two components of AMX0035 is a widely used supplement sold on Amazon ("TUDCA"), while the other is an already FDA approved drug called "Sodium Phenylbutyrate". Despite some serious hype, "AMX0035" was FDA approved with no Phase 3 trial (against basic FDA rules), using a very small Phase 2 trial sample size (n <140), and with biomarker data that was essentially non-existent. In fact, some of the ~140 pALS in the "CENTAUR" trial for AMX0035 experienced an increase in neurodegeneration (as measured by plasma neurofilament) of about 7% in the treated arm! This is far from the case with NurOwn, as every suspected ALS biomarker showed a beneficial response. Lower neurodegeneration, lower neuroinflammation, and elevated neuroprotective factors was the result of NurOwn, as none of these responses were seen in the placebo arm. It is now clear from the Phase 3 trial that NurOwn WORKS.

Furthermore, partly because of a trial error and the fact that the Phase 2 CENTAUR trial used an inexplicably elevated average ALSFRS-R progression number as a baseline, after the first ADCOM meeting in about September of 2022 an "advisory" panel of medical doctors and experts voted 4 to 6 against granting FDA approval of AMX0035. However, after additional "post-hoc" data analysis conducted by the company, and with the total support of the ALS Association (a major investor and financial stakeholder in AMX0035 which has totally refused to support NurOwn), a simple two ingredient compound is now FDA approved for ALL ALS patients, at any stage of ALS (despite a lack of specific evidence proving it actually helped more progressed patients). What cost about $13,000 just prior to FDA approval now costs over $164,000 a year. It is little wonder many ALS patients have now become skeptical of the motivations behind many in pharma, as well as the FDA's own actions.

Still, despite the number of disappointing actions and questionable motives within ALS over the last few years, ALS patients push on and continue to fight for their own medical justice. Recently, (a second) collection of 30,000 signatures in support of NurOwn were delivered to the FDA's Dr. Peter Marks by iamALS and Dan Tate, aided by the relentless ground work from "No More Excuses'" 14,000 members as ALS patients attempt to force a reversal of whether NurOwn will receive its own "ADCOMM" meeting in January of 2023. Put simply "NurOwn" CAN be legally approved, and it MUST be approved. This therapy is the #1 hope of ALS patients alive from now until at least 2025-2030.

Therefore:

Those who are dying from ALS request urgent action inthe form of an ADCOMM in January of 2023, followed by swift FDA approval of BrainStorm Cell Therapeutic's biologic compound "NurOwn", which can be easily done if the US FDA chooses to use the same appropriate regulatory flexibility it did for Relyvrio in late 2022. There is simply no valid reason not to do so now for NurOwn. The 30,000 people dying from ALS at any given moment cannot wait for another multi-year trial to simply reconfirm what is already known, or waste another two or three decades doing nothing but fundraising- especially when several SAFE and EFFECTIVE new therapies already exist. NurOwn is NOT the only new therapy here now or coming soon- more are on the way that should also be FDA approved. These include "T-REGS", "CuATSM", and "TOFERSEN". To date, only 3 treatments have been known to have stabilized ALS in legitimate clinical trials early in the disease cycle, and NurOwn is one of them. IF NurOwn is approved in 2023, the implications for ALS could be transformative. 

TAKE ACTION:

PLEASE JOIN the "NO MORE EXCUSES ALS WATCHDOGS" group on Facebook if you want to help fight for this life and death effort. For the very time in history a drug exists (and is ready) that has shown the ability to greatly slow (or even stabilize) ALS in some patients. Plus, NurOwn also holds great promise for MS (Phase 2 trial now complete), as well as possibly for Parkinson's and even Alzheimers. Worse, if NurOwn fails now the company will likely be bankrupted and this incredible therapy could be lost forever.

***THOSE WITH ALS DEMAND THE RIGHT TO TRY THIS DRUG IMMEDIATELY, WITHOUT DELAY. Please add your name to this list if you agree, or know somebody with ALS.***

 

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