Q Therapeutics, Inc., is an emerging biotechnology company developing innovative cell therapy products for the treatment of debilitating diseases of the central nervous system, has announced that its collaborators at The Johns Hopkins University have been awarded $1.3 million in a third year of grant funding from the National Institute of Neurological Disorders and Stroke (NINDS) of the National Institutes of Health (NIH).
The grant will help fund manufacturing and pre-clinical safety studies for Q Therapeutics' lead product, Q-Cells, for the treatment of Amyotrophic Lateral Sclerosis (ALS, or Lou Gehrig's disease).
Approximately 5,600 people in the United States are diagnosed each year with ALS, a progressive neurodegenerative disease that is usually fatal within 2-5 years of diagnosis.Approximately 30,000 Americans suffer from ALS at any given time; and it is responsible for one of every 400 deaths from all causes each year in the United States.
"We are very pleased with the awarding of year three funding of this grant, which will help to support the cell manufacture and remaining preclinical studies necessary for Q Therapeutics to file the IND for clinical testing ofQ-Cells for treatment of patients with ALS," commented Nicholas Maragakis, Associate Professor of Neurology at Johns Hopkins University School of Medicine. "This funding brings us closer to testing the safety and efficacy of this novel cellular therapeutic in patients with ALS."Dr Maragakis is the Principal Investigator on the grant and will be the Principal Investigator for the clinical studies.
"Q Therapeutics has a strong collaboration with Dr. Maragakis and Johns Hopkins on developing ourQ-Cells product for treatment of patients with ALS. We look forward to continued work with Dr Maragakis as we progress toward IND filing and clinical trials in ALS patients," stated Deborah Eppstein, president and CEO of Q Therapeutics. "This award by NIH further validates the results obtained with Q-Cells and provides non-dilutive funding."
Q-Cells are healthy human glial cells. The function of glial cells in the brain and spine is to support and protect neurons, the signal transmission lines of the central nervous system. Glial cells perform many actions including forming an insulating "myelin sheath" around neurons, providing the necessary growth factors needed to maintain a healthy nervous system, and removing compounds that are toxic to neurons. Many neurodegenerative diseases arise when glial cells are damaged or destroyed, causing neurons to malfunction and eventually die. Q-Cells technology aims to treat neurodegenerative diseases by supplementing the damaged or missing glia in the CNS with new, healthy cells that can help maintain and/or restore neuron function to a more robust state.
Q's cell-based ALS therapeutic originates from research at the University of Utah by Mahendra Rao, a co-founder of Q Therapeutics. In research and development since 2004, initialQ-Cell studies and pre-clinical testing have revealed many unique and promising capabilities. Q's first clinical trial will focus on demonstrating safety of Q-Cells in ALS patients. In addition, measures to evaluate therapeutic potential in ALS patients will also be monitored.