Q Therapeutics, Inc., a company focused on adult stem cell therapies for disorders of the central nervous system, recently announced that its product Q-Cells® has received Investigational New Drug (IND) clearance by the U.S. Food and Drug Administration (FDA) for the start of Phase 1/2a clinical trials in humans to evaluate the safety and tolerability of Q-Cells in patients with amyotrophic lateral sclerosis (ALS).
ALS is a progressive neurodegenerative disease, characterized by the gradual degeneration and atrophy of motor neurons in the brain and spinal cord that are responsible for controlling essential voluntary muscles, such as the ones related to movement, speaking, eating, and even breathing. ALS patients may become totally paralyzed and the majority dies due to respiratory failure within two to five years after diagnosis. It is estimated that more than 300,000 Americans suffer from the disease for which there is currently no cure.
Q-Cells corresponds to a cell-based therapeutic product candidate designed to preserve or restore the normal neural activity. It is based on glial-restricted progenitor cells (GRPs), which are the early descendants of neural stem cells that generate only glia – non-neuronal cells that constitute up to 50% of the brain cells and that provide support and protection for neurons, being also involved in the restoration of neuron health. Pre-clinical data using animal models of ALS and other central nervous system disorders showed that the administration of glial cells into the brain and spinal cord can alter the course of disorders that are currently considered incurable.
“Every 90 minutes someone in the world is diagnosed with ALS, and current therapies offer little hope,” noted the Vice President of Research and Development at Q Therapeutics Dr. James Campanelli in the press release. “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.”
“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.” explained the Chief Strategy Officer at Q Therapeutics Dr. Mahendra Rao. “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.”
“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,” said the President and Chief Executive Officer of Q Therapeutics Dr. Deborah Eppstein. “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.”