StemCells, Inc. has received approval from the Institutional Review Board (IRB) of the Oregon Health & Science University (OHSU) to initiate a Phase I clinical trial of the company's proprietary human neural stem cell product-HuCNS-SC- to treat infantile and late-infantile neuronal ceroid lipofuscinosis (NCL), two forms of a group of disorders often referred to as Batten disease. Batten disease is a rare and fatal neurodegenerative genetic condition affecting infants and children.
StemCells, Inc., in October 2005, received clearance from the Food and Drug Administration (FDA) to initiate a Phase I trial, and this IRB approval means that patient enrolment may now begin at OHSU. StemCells, Inc. believes this will be the first trial using a purified composition of neural stem cells as a potential therapeutic agent in humans.
"IRB approval of this groundbreaking trial removes the only remaining regulatory requirement between us and the initiation of clinical testing in this very needy patient population. It is the culmination of many years of meticulous basic science followed by diligent preclinical research conducted by our scientists and collaborators around the world," said Martin McGlynn, President and CEO of StemCells, Inc.
"Delivering the benefits of stem cell technology directly into the human brain will, in my opinion, be a major step forward in the efforts of scientists and clinicians around the country to find new treatments with the potential to help tens of thousands of patients with degenerative brain diseases," said Dr. Selden.
Batten disease is named after the British paediatrician who first described the juvenile form of neuronal ceroid lipofuscinosis (NCL) in 1903. The name is now often used to encompass the three most common forms of NCL-infantile, late infantile and juvenile onset. All forms have the same basic cause-lack of a lysosomal enzyme-and have similar progression and outcome.