The FDA approved a combination stem cell-gene therapy clinical trial to test the treatment’s ability to slow the progression of amyotrophic lateral sclerosis. It’s the 1st clinical trial to use neural stem cells to deliver a particular protein in the hopes of slowing ALS progression, according to researchers at the ALS clinic at Cedars-Sinai Medical Center.
A progressive neurodegenerative disease, ALS affects nerve cells in the brain and spinal cord and patients eventually lose their ability to control muscle movement. The prognosis is dim – patients are totally paralyzed within 2 to 5 years of diagnosis and the disease is fatal. The ALS Assn. pointed out that there is no cure and only 1 FDA-approved drug that modestly extends survival.
The study at the Cedars-Sinai Medical Center will use stem cells that are engineered to produce glial cell line derived neurotrophic factor, a protein known to protect motor neurons, which are responsible for transferring signals from the brain to muscles to control muscle movement.
Clive Svendsen, the director of Cedars-Sinai Board of Governors Regenerative Medicine Institute, developed this combined gene therapy approach in 2003, with funding support from the ALS Assn.
“Jeff Kaufman, who lost his battle with ALS, was my inspiration to start this work back in 2003 and the ALS Association Wisconsin Chapter provided the initial funding,” Svendsen said in prepared remarks. “Through the Evening of Hope dinners every year, the ALS Assn. tirelessly fought for funding to keep this project going to the point where we could apply to CIRM to take it to the clinic. Jim Barber, who lost his battle with ALS less than a year ago, and his wife Cheryl lobbied for the trial at the CIRM meeting all those years ago.”
“I am delighted to see this study finally move into clinical trials after the many years of our support and the tireless commitment of Dr. Svendsen and his team who continued to move the study forward and overcome the hurdles along the way to develop a product that has the biggest chance of success,” added ALS Assn. chief scientist Lucie Bruijn. “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.”