Scientist whose mother and sisters died of ALS complications hopes experimental treatment will save his life
CBS News • • general
Key Points:
- Jeff Vierstra, whose mother and two sisters died from ALS due to a genetic mutation in the FUS gene, is undergoing an experimental treatment aimed at preventing the disease from developing in him.
- ALS is a progressive neurodegenerative disease affecting motor neurons, with 10-15% of cases linked to genetic mutations; familial forms impact multiple generations.
- Vierstra and his sisters participated in a clinical trial at Columbia University led by Dr. Neil Shneider, involving spinal infusions designed to disable the mutated gene causing ALS.
- While Vierstra’s sisters eventually succumbed to ALS, he has not developed symptoms after three years of treatment, with muscle testing abnormalities normalizing and his condition stabilizing.
- Researchers view this experimental approach as a significant advancement, offering hope that ALS could become a manageable condition rather than a fatal disease.
