StemCells Inc has received clearance from the US Food and Drug Administration (FDA) to begin a Phase I safety and preliminary efficacy trial of the company's proprietary human neural stem cell product, HuCNS-SC to treat Batten disease.
Batten disease is a rare, fatal genetic disorder that affects the central nervous system of children. This is the first-ever FDA-approved clinical trial to use a purified composition of human neural stem cells as a potential therapeutic agent in humans, states a company release.
"This development is a noteworthy milestone not only for our company, but also for the entire field of stem cell therapeutics. Most importantly, it offers hope to the children and families afflicted by this disease for which there is now no cure, and to the clinicians who are seeking a treatment for their patients," said Martin McGlynn, president and chief executive officer of StemCells Inc.
The proposed Phase I trial is designed to evaluate the safety and preliminary efficacy of HuCNS-SC. for the treatment of infantile and late-infantile neuronal ceroid lipofuscinosis (NCL), the most severe forms of a group of disorders commonly referred to as Batten disease. In addition to measuring the safety of HuCNS-SC, the trial will provide initial data on HuCNS-SC's ability to affect the progression of the disease. Potential patients will be tested for eligibility and then evaluated for baseline disease status prior to transplantation of HuCNS-SC. Children enrolled in the study will be evaluated with standardized measures of development, cognition, behavior and language for one year following HuCNS-SC transplantation.
The release further states that the company plans to seek Institutional Review Board (IRB) approval from a number of leading medical institutions, including the Stanford University School of Medicine
"Physicians have been essentially helpless to assist children suffering from Batten disease. This trial opens the possibility that in the future, we may be able to provide relief to our patients and their families from one of the cruelest and most devastating diseases," said Dr. Gregory Enns, director of the biochemical genetics programme, Stanford University School of Medicine.