Researchers
To Study Effectiveness Of Stem Cell Transplant In Human
Brain
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(TM), 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.
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Wonders of the technology. Hopefully this new technique can help
the young patients. 