FDA Clears New ALS Treatment For Human Trials
Q Therapeutics, Inc., a clinical-stage company committed to find new therapies that might address central nervous system (CNS) diseases, has recently announced that the United States Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug Application (IND) so that Q-Cells’ Phase 1/2a trials can be initiated in patients with amyotrophic lateral sclerosis (ALS).
ALS, also known as Lou Gehrig’s disease, is a devastating disease caused by the progressive degeneration of motor neurons, which are the nerve cells in the spinal cord and brain responsible for controlling muscle movement. The disease affects more than 30,000 people in the US and almost half a million people around the world. Currently, there is no cure to address ALS with an 80 percent fatality rate within 5 years post diagnosis.
Q-Cells is Q Therapeutics’ first patented cellular product candidate that uses glial-restricted progenitor cells (GRPs) to address the disease. GRP’s are early descendants of neural stem cells that only produce “glia”, representing 50 percent of the total cells in the brain. Glia are crucial to support, maintain and restore neuron health.
FDA’s clearance addressed to Q Therapeutics’ Investigational New Drug (IND) with Q-Cells in ALS patients in a Phase 1/2a clinical trials confirms that the available pre-clinical data is enough to allow the initiation of the first-ever human trial assessing the use of GRPs in ALS patients. Previous studies in animal models have shown that the delivery of healthy glial cells into the spinal cord and the brain can impact CNS diseases.
Deborah Eppstein, CEO and President of Q Therapeutics, said in a press release: “This is a major milestone not only for Q Therapeutics but also in the quest for a cure for ALS. Our unique approach holds great promise and we look forward to the potential of these trials to establish human safety and efficacy. This achievement builds on a decade of research, nearly $30 million of funding, and the tireless dedication of our scientists and research collaborators. We proudly acknowledge the devotion of all of our scientific co-workers and supporters, especially James Campanelli, Ph.D.; Mahendra Rao, MD, Ph.D.; the National Institutes of Health; the Maryland Stem Cell Research Fund; and Bosarge Life Sciences, in the pursuit of novel therapies for ALS and other debilitating diseases of the brain and spinal cord.”
“Every 90 minutes someone in the world is diagnosed with ALS, and current therapies offer little hope. With this FDA clearance, we are on the cusp of performing groundbreaking human studies with a cellular therapeutic that has the potential to change the outlook for people living with ALS and their loved ones. The FDA’s clearance of our IND for Q-Cells within the original 30-day review period provides regulatory validation of our careful development approach and clinical trial plans,” noted James Campanelli, the Q Therapeutics’ Vice President of Research and Development.
Mahendra Rao, Chief Strategy Officer at Q Therapeutics, added, “GRP cells are really a unique clinical candidate and these will be the first ever human trials. What is special is that their lineage restriction means that after transplantation they only generate oligodendrocytes and astrocytes in vivo – vital new glial cells – and, unlike many other cells, they migrate, survive and respond to cellular signals to produce therapeutic molecules after injury. We are hopeful that these progenitor characteristics of the GRP will prove effective in the treatment of a variety of CNS disorders where repair of the cellular machinery of the brain and spine is required.”