From the Scheaffer Autism Report
RESEARCH
Researchers to Study Effectiveness of Stem Cell Transplant In Human Brain
http://www.eurekalert.org/pub_releases/200...s-drt030906.php First-of-its-kind clinical trial will explore safety, preliminary
efficacy of injecting human stem cells directly into the brain to treat
fatal pediatric neurodegenerative disorder Researchers in Doernbecher
Children's Hospital at Oregon Health & Science University will begin a Phase
I clinical trial using stem cells in infants and children with a rare
neurodegenerative disorder that affects infants and children.
The groundbreaking trial will test whether HuCNS-SC, a proprietary
human central nervous stem cell product developed by StemCells, Inc. is
safe, and whether it can slow the progression of two forms of neuronal
ceroid lipofuscinosis (NCL), a devastating disease that is always fatal. NCL
is part of a group of disorders often referred to as Batten disease.
"NCL is a heartbreaking and devastating diagnosis for children and
their families," said Robert D. Steiner, M.D., F.A.A.P., F.A.C.M.G., vice
chairman of pediatric research, head of the Division of Metabolism and the
study's principal investigator at Doernbecher Children's Hospital, OHSU.
Steiner also is an associate professor of pediatrics, and molecular
and medical genetics in the OHSU School of Medicine. "While the preclinical
research in the laboratory and in animals is promising, it is important to
note that this is a safety trial and, to our knowledge, purified neural stem
cell transplantation has never been done before. It is our hope that stem
cells will provide an important therapeutic advance for these children who
have no other viable options."
NCL is caused by mutations or changes in the genes responsible for
teaching the body how to make certain enzymes. Without these enzymes or
proteins, material builds up inside brain neurons and other brain cells,
causing a rapidly progressive decline in mental and motor function,
blindness, seizures and early death. This study addresses two forms of NCL:
infantile neuronal ceroid lipofuscinosis (INCL) and late-infantile neuronal
ceroid lipofuscinosis (LINCL). Tragically, children with INCL typically die
before age 5 and those with LINCL typically do not live past age 12.
"If delivering stem cells directly into the human brain is safe and
effective, it 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 co-investigator Nathan Selden, M.D., Ph.D., F.A.C.S.,
F.A.A.P. "I am proud that Doernbecher Children's Hospital will be part of
this effort." Selden is Campagna Associate Professor of Pediatric
Neurological Surgery and head of the Division of Pediatric Neurological
Surgery, Doernbecher and OHSU School of Medicine.
Up to six children from Oregon or around the country will undergo
HuCNS-SC transplantation at Doernbecher. Previous studies of mice that are
missing one of the enzymes that causes NCL have shown HuCNS-SC increases the
amount of the missing enzyme, reduces the amount of abnormal material in the
brain and prevents the death of some brain cells. No major side effects have
been reported in animals.
StemCells, Inc. received clearance from the U.S. Food and Drug
Administration to initiate a Phase 1 clinical trial of HuCNS-SC in October
2005. The company believes this will be the first trial using a purified
composition of neural stem cells as a potential therapeutic agent in humans.