What if ALS-one of the most devastating neurological diseases-is closer to being treatable than ever before? What if the cure is not decades away, but within reach in our lifetime?
In this powerful episode of Impossible Dreams, host Aaron Lazar sits down with Dr. Merit Cudkowicz-one of the world's leading ALS researchers and clinicians, the Julieanne Dorn Professor of Neurology at Harvard Medical School, and Chief of Neurology at Massachusetts General Hospital. Dr. Cudkowicz, affectionately known as the "greatest ALS doctor in the world" by countless patients, has dedicated her career to finding better treatments and ultimately a cure for ALS since witnessing the discovery of the first ALS gene in 1993.
This conversation is both deeply scientific and profoundly human. Dr. Cudkowicz walks through the challenging journey of ALS diagnosis-why it typically takes a year and 3-4 doctors before patients get answers-and explains the complexity of a disease that mimics many other conditions. She shares the groundbreaking advances happening right now, from the remarkable success of SOD1 gene therapy (which can halt or even reverse progression in certain patients) to the new wave of therapies targeting TDP-43 protein, the underlying biology present in nearly all ALS cases.
Throughout this episode, Dr. Cudkowicz's message is clear and urgent: "I don't think curing ALS is impossible. I absolutely think it's possible." She explains why staying connected to research centers, being part of the ALS community, maintaining physical and mental well-being, and never giving up hope are essential for patients navigating this disease. This episode is not just for those living with ALS, it’s for anyone who is living through the unknown of a life-changing diagnosis or has ever cared for someone who has. It's about spreading hope and community in the face of what feels impossible
Guest Bio:
Dr. Merit Cudkowicz is the Julieanne Dorn Professor of Neurology at Harvard Medical School and Chief of the Department of Neurology at Massachusetts General Hospital. A chemical engineer from MIT who went on to complete her medical training at Harvard, Dr. Cudkowicz has been at the forefront of ALS research since 1993, when her mentor Dr. Robert Brown discovered the first ALS gene (SOD1). She is the founder and director of the Healey ALS Platform Trial, a revolutionary multi-drug testing system that is dramatically accelerating the pace of ALS drug development. Dr. Cudkowicz has dedicated her career to clinical trials, patient care, and translating laboratory discoveries into treatments that can help people living with ALS.
Learn more about Dr. Merit Cudkowicz:
Harvard Medical School Faculty Profile
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Episode Navigation:
03:15 - I do not think curing ALS is impossible: Dr. Cudkowicz's opening declaration of hope and possibility
03:24 - From MIT chemical engineering to ALS research: The journey that began with the 1993 SOD1 gene discovery
05:05 - Learning the basics at Harvard: Why clinical trial design and epidemiology matter for ALS breakthroughs
05:42 - The brain science frontier: Why neurological diseases like ALS are finally getting the treatments they deserve
07:45 - The year-long diagnosis journey: Why it takes 3-4 doctors and 12 months to get an ALS diagnosis
08:26 - It is a clinical diagnosis: Understanding why there is no single test for ALS and why ruling out mimics is critical
14:24 - The SOD1 breakthrough: How gene therapy can halt or reverse ALS in certain genetic forms and why it is revolutionary
30:43 - The Healey Platform Trial revolution: Testing multiple drugs at once to accelerate the path to treatments
31:55 - Compassionate use and expanded access: How patients can get promising therapies before full FDA approval
34:19 - The cost crisis: Why ALS drugs cost millions and what needs to change to make treatments accessible
38:42 - Why clinical trials are hard: The challenges of running ALS studies and getting drugs to patients faster
40:53 - The TDP-43 breakthrough coming: New therapies targeting the biology present in nearly all ALS cases
41:50 - UNC13A and the upstream approach: Going after the root causes rather than just symptoms
42:20 - VectorY Therapeutics antibody: Using gene therapy to deliver misfolded protein-targeting antibodies
43:23 - The MyMatch precision medicine program: Matching patients to the right drug based on their unique biology
44:56 - Screening with every biomarker: Motor neurons from blood, inflammation imaging, and personalized matching
47:33 - Earlier compassionate access: Why patients do not need to wait for Phase 3 anymore
48:31 - Dr. Cudkowicz's advice for patients: Positive mindset, community, wellness, research participation, and hope
49:17 - It can happen: The unexpected power of the SOD1 results and why we should never give up
