Cell and gene therapy for Lou Gehrig’s disease

Clinical trial using this therapy shows promising protective outcome in ALS patients.

Artist’s impression of investigational neuroprotective therapy that was safely administered to patients with ALS in a recent trial. | Photo Credit: Journal of American Medical Association (JAMA) Network

Combined cell and gene therapy has been found to be effective in providing protection against degeneration in patients with amyotrophic lateral sclerosis (ALS), or Lou Gehrig’s disease, for up to three-and-a-half years, researchers from Cedars-Sinai Medical Center, Los Angeles, have reported in Nature Medicine. The eminent scientist Stephen Hawking was afflicted with ALS from the age of 21.

In an early clinical trial, human neural progenitor cells genetically engineered to produce a potentially neuroprotective factor were safely transplanted into the spines of patients with ALS. Following the transplantation, the protective factor continued to be produced for 42 months.

The phase 1 and 2a study involved 18 patients injected with cultured donor cells engineered to produce glial cell line-derived neurotrophic factor (GDNF), which can be neuroprotective. The cells were injected only into one side of the lumbar spine to be able to detect differences between treated and untreated neural pathways.

According to the study, treated legs were found to lose strength at a slower rate than untreated legs, but the difference was not statistically significant. One participant showed a markedly preserved function of the treated leg three years after transplant. One individual showed many more motor neurons preserved on the treated side.

The treatment also may have preserved limb function and neurons in some patients, although these differences were not statistically significant overall, the paper noted.