HD Lighthouse Contributing Editor's Comment: The potential of stem cell therapy is exciting. At some point, researchers will be able to prevent or stop Huntington's Disease, perhaps through RNA interference or perhaps through a cocktail of drugs and supplements. Research done with mice suggests that stopping the disease will lead to significant improvement in those who are already symptomatic. However, biotechnology which actually restores lost brain cells might mean a full recovery even for those well into the disease's progression.

Although stem cell therapy is promising, it carries with it some risks. There is a concern that cancer may result or the cells may develop or act in unanticipated ways and cause dysfunction. Successful preclinical trials in animal models of neurodegenerative diseases provide some reassurance but the only way to know if this kind of therapy is safe and effective for people is to do clinical trials.

A Phase One clinical trial to transplant fetal stem cells into patients with Batten disease started in November of 2006 (see press release below for details). Batten Disease is a rare genetic disorder which strikes during infancy or childhood and is inevitably fatal. The children lack an enzyme which breaks down fats and proteins for use and recycling in the brain. Seizures result, then loss of the ability to communicate and blindness, then death. If the treatment works the way it is hoped, the stem cells will migrate to where they are needed and supply the missing enzyme.

The FDA approved the Phase One trial in October of 2005. In addition to the preclinical research, the serious nature of the symptoms and the fatality of the disease in childhood were undoubtedly factors in approving the trial. The first patient, a six year old boy, received the fetal stell cell transplant in November, 2006. Five more patients will also receive transplants.

According to a December 18, 2006 Scientific American online article, a month after the transplant surgery, the boy is home, speaking again and no longer experiencing seizures, but of course it is way too early to draw any conclusions. SA: The World's First Neural Stem Cell Transplant

The Lighthouse will continue to follow this trial.

-- Marsha L. Miller, Ph.D.
Posted to the HDL: 22 Dec 2006

StemCells, Inc. Announces First Human Neural Stem Cell Transplant

StemCells, Inc.

PALO ALTO, Calif., (November 15, 2006) –StemCells, Inc. (NASDAQ: STEM) today announced that the first transplantation of the Company’s proprietary human neural stem cell product—HuCNS-SC™— took place yesterday at the Oregon Health & Science University’s (OHSU) Doernbecher Children’s Hospital. This transplant is the first of six that are planned as part of the Company’s Phase I clinical trial designed to evaluate the safety and preliminary efficacy of HuCNS-SC as a treatment for infantile and late infantile neuronal ceroid lipofuscinosis (NCL). NCL, which is often referred to as Batten disease, is a rare and fatal neurodegenerative condition afflicting infants and children.

“Dosing of the first Batten’s patient is a major milestone for StemCells, Inc., and for the entire stem cell field. It is the culmination of many years of careful and diligent preparation on the part of many dedicated people. It is important to remember, however, that this is just the first step in a long and challenging clinical development process ,” said Martin McGlynn, President and CEO of StemCells, Inc . “Our focus at this time is on the health and well-being of the patient. We are grateful for the participation of this family in this landmark trial. Children afflicted by Batten disease are gravely ill, and this family’s courage advances the hope that this trial will lead to a treatment for this devastating disease.”

The Phase I clinical trial is being led by Robert D. Steiner, M.D ., F.A.A.P., F.A.C.M.G., vice chairman of pediatric research and head of the Division of Metabolism at Doernbecher Children’s Hospital, and professor of Pediatrics and Molecular & Medical Genetics at OHSU School of Medicine; Nathan Selden, M.D., Ph.D., F.A.C.S., F.A.A.P., Campagna Associate Professor of Pediatric Neurological Surgery and head of the Division of Pediatric Neurological Surgery, Doernbecher and OHSU School of Medicine; and Thomas K. Koch, M.D., F.A.A.P., F.A.A.N., director of Pediatric Neurology and professor of Pediatrics and Neurology at Doernbecher and OHSU School of Medicine.

About Neuronal Ceroid Lipofuscinosis (Batten Disease)

Neuronal ceroid lipofuscinosis is a rare and fatal neurodegenerative disorder brought on by inherited genetic mutations. The disorder afflicts infants and young children, and the three most common forms of NCL—infantile, late infantile and juvenile onset—are often referred to as Batten disease. All forms have the same basic cause—lack of a lysosomal enzyme—and have similar progression and outcome. Children with NCL suffer seizures, progressive loss of motor skills, sight and mental capacity, eventually becoming blind, bedridden and unable to communicate.

In infantile and late infantile NCL, the disorder is brought on by inherited mutations in the CLN1 gene, which codes for palmitoyl-protein thioesterase 1 (PPT1) or in the CLN2 gene, which codes for tripeptidyl peptidase I (TPP-I), respectively. The consequence of these gene mutations is either a defective or missing enzyme that leads to accumulation of lipofuscin-like fluorescent inclusions in various cell types. These non-degraded lysosomal substrates accumulate to the point of interference with normal cellular and tissue function and ultimately lead to the pathological manifestations of the disease. One way to treat the disease is to provide the brain with a replacement source of functional enzyme that can be taken up by the enzyme-deficient cells.

About HuCNS-SC™

StemCells’ human neural stem cells (HuCNS-SC) are a cell-based therapeutic prepared under controlled conditions. When HuCNS-SC are transplanted into the brain of a mouse model developed to mimic the human form of infantile NCL, the cells spread throughout the brain and produce the missing lysosomal enzyme. The enzyme level increases and continues to do so over time after the transplant. Thus, placement of HuCNS-SC in appropriate places in the brain provides the prospect of long-term delivery of the missing lysosomal enzyme. In laboratory studies, HuCNS-SC also produces the lysosomal enzyme missing in late infantile NCL, the other subtype being studied in the clinical trial. The production of both enzymes by HuCNS-SC provides a scientific rationale for enzyme replacement and cellular rescue in these two subtypes of NCL.

About the Clinical Trial

The Phase I trial is designed to evaluate the safety and preliminary efficacy of HuCNS-SC as a treatment of infantile and late infantile NCL. The trial is open label with two dose cohorts, and is expected to enroll six patients during the next 12 months. Potential patients will be tested for eligibility and then evaluated for baseline disease status prior to transplantation of HuCNS-SC. In addition to measuring the safety of HuCNS-SC, patients enrolled in the study will be evaluated with standardized measures of development, cognition, behavior and language for one year following transplantation.

About StemCells, Inc.

StemCells, Inc. is a clinical-stage biotechnology company focused on the discovery, development and commercialization of cell-based therapeutics to treat diseases of the nervous system, liver and pancreas. The Company’s programs seek to repair or repopulate neural, liver or other tissue that has been damaged or lost as a result of disease or injury. StemCells is the first company to directly identify and isolate human neural stem cells from normal brain tissue. These cells are expandable into cell banks for therapeutic use, which offers the potential of using normal, non-genetically modified cells as cell-based therapies. StemCells has approximately 40 U.S. and 100 non-U.S. patents. Further information about the Company is available on its Web site at www.stemcellsinc.com.

About OHSU

Oregon Health & Science University is Oregon’s only health and research university and its only academic health center. As Portland’s largest employer and the fourth largest employer in Oregon (excluding government), OHSU’s size contributes to its ability to provide many services and community support activities not found anywhere else in the state. It serves more than 184,000 patients, and is a conduit for learning for more than 3,900 students and trainees. OHSU is the source of more than 200 community outreach programs that bring health and education services to each county in the state.

As a leader in research, OHSU earns $274 million annually in research funding. The institution serves as a catalyst for the region’s bioscience industry and is an incubator of discovery, averaging one new breakthrough or innovation every four days. OHSU disclosed 101 inventions in 2005 alone and has helped start 57 new spin-off companies, most of which are based in Oregon.

About Doernbecher Children’s Hospital

Doernbecher Children’s Hospital, an integral part of Oregon Health & Science University, is a world-class academic health center that each year cares for more than 56,000 patients from across the United States. In the most patient- and family-centered environment, children from all corners of the country receive outstanding cancer treatment, specialized neurology care, highly sophisticated heart surgery, and care in many other pediatric specialties. In addition to multiple locations in the Portland metropolitan area, Doernbecher’s pediatric experts travel around Oregon and southwest Washington providing pediatric specialty care through 13 outreach clinics.
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Source: press release

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