Personalized ALS therapy shows early promise in one patient
A therapy designed for a single patient with a rare genetic form of ALS has shown early signs of working, Mayo Clinic researchers report.
The patient carries a mutation in the CHCHD10 gene. Fewer than 1% of people with hereditary ALS have this mutation. The faulty gene damages mitochondria, the energy units of cells, leading to nerve-cell death.
Scientists built an individualized antisense oligonucleotide, or ASO, called nL-CHCHD-001. It blocks the production of the harmful protein. The patient received six spinal injections between April 2024 and April 2025.
Levels of neurofilament light, a blood marker of nerve injury, fell by about 50% and returned to normal. His score on the ALS functional rating scale rose from 33 to 36 over the year. No serious side effects were seen.
The study was published in the journal Med. The ASO is now being tested in eight more CHCHD10 carriers.
Lead author Margot Cousin said such cases build the framework for future individualized therapies. The treatment remains experimental and is not standard care.
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