OHSU Doernbecher Children’s Hospital to conduct second phase of landmark clinical trial for children With batten disease
11/01/10 Portland, Ore.
The OHSU Doernbecher team led the first phase of this groundbreaking trial, becoming the first clinical team in the world to implant human neural stem cells directly into the brains of children with this rare, currently fatal disease.
Oregon Health & Science University Doernbecher Children’s Hospital will lead the next phase of a landmark clinical trial to further assess the safety and preliminary effectiveness of purified human neural stem cells (HuCNS-SC®) as a potential treatment for infantile or late-infantile neuronal ceroid lipofuscinosis (NCL), a rare and currently fatal neurodegenerative disorder that affects infants and children.
The Phase Ib trial, sponsored by StemCells, Inc., is designed to further assess the safety and preliminary effectiveness of StemCells’ HuCNS-SC as a potential treatment for NCL, also referred to as Batten disease. This second trial is expected to enroll six children whose NCL is less advanced than those enrolled in the initial Phase I trial, also carried out at OHSU Doernbecher, and, in addition to safety, is also designed to evaluate the impact of HuCNS-SC on disease progression.
“The hard work of testing neural stem cells for their safety and effectiveness in clinical use began here at OHSU Doernbecher Children’s Hospital. We are very pleased to carry on this important effort with a second major trial. Our great hope is that this work will eventually yield significant benefits for patients who suffer from devastating nervous system diseases,” said Nathan Selden, M.D., Ph.D., FACS, FAAP, Campagna Professor of Pediatric Neurosurgery and head of the Division of Pediatric Neurological Surgery at OHSU Doernbecher Children’s Hospital, OHSU School of Medicine.