Stem cell-gene therapy for ALS: Recent trial

Cedars-Sinai investigators have developed an investigational therapy using support cells and a protective protein that can be delivered past the blood-brain barrier. This combined stem cell and gene therapy can potentially protect diseased motor neurons in the spinal cord of patients with amyotrophic lateral sclerosis, a fatal neurological disorder known as ALS or Amyotrophic lateral sclerosis.

In the first trial of its kind, the Cedars-Sinai team showed that delivery of this combined treatment is safe in humans.

The study used stem cells originally designed in Svendsen's laboratory to produce a protein called glial cell line-derived neurotrophic factor (GDNF). This protein can promote the survival of motor neurons, which are the cells that pass signals from the brain or spinal cord to a muscle to enable movement. In patients with ALS, diseased glial cells can become less supportive of motor neurons, and these motor neurons progressively degenerate, causing paralysis.

Ref:

Clive Svendsen et al, Transplantation of human neural progenitor cells secreting GDNF into the spinal cord of patients with ALS: a phase 1/2a trial,Nature Medicine, DOI: 10.1038/s41591-022-01956-3