Treating patients with rare diseases has an impact on your soul. That's why we're moving fast.

University of Virginia president plans to resign as school comes under pressure from Trump administration over its DEI programs, source says cnn.it/3I6EPel

.@DrMakaryFDA @VPrasadMDMPH, as a physician living with ALS, I urge you to grant NurOwn accelerated approval to break a 156-year cycle of despair for a 100% fatal disease. ALS patients, with a median survival of 2–3 years, cannot wait 5 years for Phase 3b trial results, especially now, 15 years after NurOwn's Phase 1 trial began. The 2019 FDA ALS Guidance demands flexibility—act now to save lives. NurOwn, developed by @BrainStormCell, targets ALS’s core drivers: neuroinflammation and neurodegeneration. Its Phase 3 trial (NCT03280056) faced the same challenges plaguing ALS research—small trial sizes, patient heterogeneity, and the flawed ALS Functional Rating Scale-Revised (ALSFRS-R), a 48-point questionnaire insensitive to subtle benefits, especially in advanced patients due to its “floor effect.” Despite not meeting the ALSFRS-R endpoint, NurOwn delivered robust, statistically significant biomarker changes in cerebrospinal fluid (CSF), proving biological efficacy: Neurofilament Light (NfL): Reduced by 20% from baseline to week 20 (p<0.05), signaling slowed neurodegeneration—mirroring the NfL reductions that justified Tofersen’s accelerated approval for SOD1-ALS. Neurotrophic Factors: Increased VEGF and HGF (p<0.01), promoting neuronal survival and repair, with no changes in placebo. Neuroinflammation: Decreased MCP-1 (p<0.05), curbing a key ALS driver. Galectin-1 & LAP/TGF-β1: Enhanced neuroprotective and anti-inflammatory pathways, predictive of better outcomes. These consistent CSF changes, unaffected by ALSFRS-R’s limitations, confirm NurOwn’s plausible mechanism: stem cells deliver neurotrophic factors to protect motor neurons. In a pre-specified subgroup with less-advanced ALS, NurOwn slowed functional decline by 1.5 points on ALSFRS-R slope (p=0.08), with neurologists reporting clinical stability—improved speech, swallowing, and hand function—in some patients. Patient-reported outcomes (PROs) and real-world evidence (RWE) from expanded access programs further validate these benefits, aligning with the 2019 FDA ALS Guidance’s emphasis on PROs and RWE for rare diseases. The Tofersen precedent proves flexibility works. Despite failing its ALSFRS-R endpoint, Tofersen gained accelerated approval in 2023 based on NfL reductions, later confirmed by patient outcomes. NurOwn’s biomarker profile is equally compelling, despite sporadic ALS’s greater heterogeneity. @MartyMakary, you told @megynkelly, “For rare, incurable diseases, we may approve based on plausible mechanisms.” NurOwn meets this standard with biomarker-driven evidence of neuroprotection. @VPrasadMDMPH, your evidence-based leadership can redefine ALS’s hopeless landscape by prioritizing these data over flawed endpoints. A second Phase 3 trial, fifteen years after NurOwn’s Phase 1, is an ethical failure for a disease killing patients in 2–3 years. Peter Marks’ decision to require a Phase 3b trial overlooked that it will face the same intractable limitations—small sample sizes, heterogeneity, and ALSFRS-R’s insensitivity—dooming it to repeat past failures while patients die waiting. Tragically, some who stabilized during trials died after losing access. We propose: Invite @BrainStormCell to apply for accelerated approval, leveraging biomarkers, PROs, and RWE. Implement real-time efficacy monitoring via patient registries and digital tools (e.g., wearables tracking motor function), ensuring safety and validating outcomes post-approval. Meet ALS patients to hear firsthand accounts of NurOwn’s transformative impact—stories of hope extinguished by delays. The 2019 FDA ALS Guidance urges innovative endpoints and surrogate markers for small, heterogeneous trials. NurOwn’s data—NfL, neurotrophic factors, and PROs—meets this call. After 156 years, ALS patients deserve a chance to live. Act decisively to approve NurOwn and restore hope. #ALS #FDAAction @SecKennedy @realDonaldTrump @RepGuthrie @RepLarryBucshon @michaelcburgess @RepGusBilirakis @RepBillJohnson @DrNealDunnFL2 @cathymcmorris @LeaderJohnThune @RepDebDingell @RepMMM @SenatorWicker @RepBarragan @RepLoriTrahan @BernieSanders @PattyMurray @SenBobCasey @SenatorBaldwin @timkaine @SenatorHassan @SenTinaSmith @SenMarkey @SenBillCassidy @SenMullin @lisamurkowski @RogerMarshallMD @RepJasonCrow @TeamCalvert @RepBrianFitz @ChrisCoons @RepAndyHarrisMD @DickDurbin @SenJohnThune @RepDianaDeGette @RepMikeQuigley @RepSchakowsky @rosadelauro @RandPaul @DorisMatsui @RepDonBacon @SenJohnCurtis

This is Marco Rubio explaining how the USA promised to defend Ukraine forever if they got rid of their nuclear arsenal left after the Soviet Union fell. This is why lil marco was sinking into the couch. He was hoping we wouldn’t find it…so don’t RT right now this very second.

We are Americans. We are the most powerful nation on the planet. What defines us is not our power to dominate the weak. What demonstrates our character is not our capacity to traumatize the afflicted. What speaks our truth is not how we taunt, deride or belittle the embattled, broken or battered. We are: The country of the Marshall Plan The nation of The Berlin Airlift The nation whose leader said, “Mr. Gorbachev, tear down this wall.” We are at our best when we are resolute against evil -- not when we kowtow to those who perpetrate it. We tell our truth when we defend the weak -- not when we do it only if we get protection money. We are America when our leaders call the dictators dictators and those who fight to defend democracy against tyranny what they are: heroes. Donald Trump and JD Vance didn't show strength today -- they showed weakness. They belittled a giant and showed just how small they are. They have the most vaunted titles in the world: President and Vice President of The United States of America. But the title doesn't make the man. And today both men brought shame to their titles. A weary warrior was before them. A beleaguered man who has lost thousands of his friends and comrades to the unjust and illegal aggression of an authoritarian leader. They showed no grace; no magnanimity of spirit; no diplomatic acumen. They were small bullies with big titles and they disrespected the history and legacy of the nation they lead. Today, Donald Trump acted more like Russia's Putin than America's President.

This is what a real leader looks like #ISupportZelensky #SlavaUkraïni