Your Brain On... ALS
Your Brain On
Advancements in Gene Therapy for ALS
This chapter explores recent breakthroughs in gene therapy for SOD1 ALS, including a successful clinical trial indicating potential reversibility of the disease. It highlights promising biomarkers, new drug approvals, and ongoing research into targeting neuroinflammation and prevention strategies for at-risk individuals.
ALS, a nervous system disease, ruled the headlines in the summer of 2014 thanks to the viral Ice Bucket Challenge. How has our knowledge of the disorder evolved a decade later?
Amyotrophic lateral sclerosis, also known as ‘Lou Gehrig's disease’ (named after the iconic baseball player), is a progressive neurological disorder which breaks down a person’s motor neurons. The main symptoms are muscle weakness and impaired physical function — mild, at first, but in the latter stages of the disease’s progression, essential processes like breathing begin to fail.
Most individuals face a life expectancy of two to five years. A small percentage live five to ten years. And an even tinier group survive beyond a decade. Most famously, renowned physicist Stephen Hawking survived more than 50 years past his diagnosis, in part due to the intensive care he was able to afford.
In this episode of the ‘Your Brain On...’ podcast, we discuss:
• What ALS is, and how it affects the brain and the body
• The onset and progression of ALS, from the earliest symptoms to the end-stage impacts
• How ALS patients are diagnosed, treated, and cared for
• Likely causes of ALS, including genetic predispositions and potential environmental risk factors
• How new technologies are rapidly accelerating our understanding of ALS, especially in genetics
Joining us for this installment of the show are two world-class experts on the disease:
• Professor Ammar Al-Chalabi PhD, Professor of Neurology and Complex Disease Genetics at the Maurice Wohl Clinical Neuroscience Institute at King's College.
• Merit Cudkowicz, Director of the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital.
‘Your Brain On’ is hosted by neurologists, scientists and public health advocates Ayesha and Dean Sherzai.
‘Your Brain On... ALS’ • SEASON 4 • EPISODE 3
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LINKS
PROFESSOR AMMAR AL-CHALABI
at King’s College: https://www.kcl.ac.uk/people/ammar-al-chalabi
at Motor Neurone Disease Disease Association: https://www.mndassociation.org/get-involved/cure-finders/professor-ammar-al-chalabi
Project MinE: https://projectmine.com/
MERIT CUDKOWICZ
at Massachusetts General Hospital: https://www.massgeneral.org/doctors/16904/merit-cudkowicz
at Harvard University: https://researchers.mgh.harvard.edu/profile/1520993/Merit-Cudkowicz
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ANNOUNCING: NEURO WORLD RETREAT 2025
We’re so excited to share something very close to our hearts, which we’ve been working on over the past few months: our first ever brain health retreat!
You’re warmly invited to join us in San Diego, California for the inaugural NEURO World Retreat 2025, taking place September 2–5, 2025 at the breathtaking Paradise Point Resort.
For more information, and to book, visit: https://neuroworldretreat.com/
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FOLLOW US
Join the NEURO Academy: NEUROacademy.com
Instagram: @thebraindocs
Website: TheBrainDocs.com
More info and episodes: TheBrainDocs.com/Podcast
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References:
Brown, Robert H., and Ammar Al-Chalabi. "Amyotrophic lateral sclerosis." New England Journal of Medicine 377.2 (2017): 162-172.
Benatar, Michael, et al. "A roadmap to ALS prevention: strategies and priorities." Journal of Neurology, Neurosurgery & Psychiatry 94.5 (2023): 399-402.
Voigtlaender, Sebastian, et al. "Artificial intelligence in neurology: opportunities, challenges, and policy implications." Journal of Neurology 271.5 (2024): 2258-2273.
Zinman, Lorne, and Merit Cudkowicz. "Emerging targets and treatments in amyotrophic lateral sclerosis." The Lancet Neurology 10.5 (2011): 481-490.
Raghav, Yogindra, et al. "Identification of gene fusions associated with amyotrophic lateral sclerosis." Muscle & Nerve 69.4 (2024): 477-489.
Su, Feng-Chiao, et al. "Association of environmental toxins with amyotrophic lateral sclerosis." JAMA neurology 73.7 (2016): 803-811.
Talbott, Evelyn O., et al. "Case-control study of environmental toxins and risk of amyotrophic lateral sclerosis involving the national ALS registry." Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration (2024): 1-10.
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