Cell & gene therapy clinical trial for ALS approved to start enrollment
Investigators at Cedars-Sinai Medical Center (Los Angeles, CA, USA) have received approval from the US FDA to conduct a clinical trial for ALS, testing whether a combination stem cell and gene therapy is a safe approach for treating the neurodegenerative disease.
A team from Cedars–Sinai Medical Center in Los Angeles (CA, USA), with support of The ALS Association (Washington, DC, USA) and CIRM (Oakland, CA, USA, has sought and received approval from the US FDA to carry out a clinical trial investigating the safety of a stem cell–gene therapy aimed at slowing the progression of the motor neuron disease amyotrophic lateral sclerosis (ALS). The study will look at muscle strength, to get an initial idea of efficacy outcomes.
ALS is a progressive neurodegenerative disease that currently has no cure, and as there is only a single drug approved by the US FDA that modestly extends survival, there is a significant unmet need for this condition. Previous investigations have demonstrated that when the cells supporting the motor neurons stop functioning correctly and releasing nutrients, the motor neurons die. Patients lose the ability to initiate and control muscle movement, which causes total paralysis and death 2–5 years after diagnosis.
The approach to be used in the upcoming clinical trial was developed by Clive Svendsen (Director of Cedars–Sinai Board of Governors Regenerative Medicine Institute) and involves using engineered neural stem cells injected directly into one side the lumbar region of the spinal cord that controls movement on only one side of the body as a mechanism to deliver the neuroprotective protein GDNF to motor neurons to reduce the rate of their death.
The trial will be carried out over a 1-year period at the ALS clinic at Cedars–Sinai Medical Center, led by Robert Baloh and Peggy Allred, with a neurosurgical team headed by J Patrick Johnson. It may even commence by the end of this year, and will enroll 18 patients who are currently experiencing ALS-caused weakness in their legs.
“This therapy approach holds great promise as a potential ALS therapy based on the preclinical studies and I look forward to seeing the initial safety results of this trial,” explained Lucie Bruijn, Chief Scientist of The ALS Association. If the clinical trial demonstrates that the approach is safe, the trial would be expanded to investigate affects on leg strength on the side of the stem cell injection.