At Oregon Health & Science University's (OHSU) Doernbecher Children's Hospital, the first clinical trial determining the safety of stem-cell transplants to cure Batten disease is set to get underway this year.
Batten disease is an uncommon but deadly neurodegenerative disorder. A phase I trial is conducted to determine the safety of a treatment, irrespective of its efficacy.
According to the researchers, the transplants of stem cells at two different doses will be given to 6 children with the symptoms and will be monitored for a year.
The stem cells will be implanted into 3 areas of the brain and drugs to avoid cell rejection will also be given to the childen.
Batten disease is also called as neuronal ceroid lipofuscinosis (NCL) and is a hereditary neurodegenerative disorder. Seizures, progressive loss of motor skills, sight and mental capacity, eventually becoming blind, bedridden and inability to communicate are the characteristics of this disease. The National Institute of Neurological Disorders and Stroke says that at present there is no treatment for the disease and it usually becomes fatal by the late teens or early 20s.
Six months to 10years is the age at which the symptoms develop. The inherited mutated gene determines the age of onset and rate of progression. Around 200-600 kids are affected by this disease in the U.S.
"NCL patients lack an enzyme responsible for breaking down complex fat and protein compounds in the brain," lead researcher Dr. Robert D. Steiner, vice chairman of pediatric research and head of the Division of Metabolism at Doernbecher Children's Hospital, said at a press briefing Friday.
"These materials accumulate and interfere with normal cell and tissue function and ultimately cause cells to die," said Steiner, who's also a professor of pediatrics and molecular and medical genetics at Oregon Health and Science University School of Medicine.
"The hope behind the new research is that the stem cells will evolve into cells that start producing the missing or defective lysosomal enzyme that causes the disease, as was the case in animal studies, " he said.
"Children eligible for the study have clinical symptoms of one of two types of NCL -- infantile or late infantile NCL -- and loss-of-function mutations in either the CLN1 or CLN2 gene. The children will be monitored with standardized measures of development, cognition, behavior and language for one year following transplantation, " the researchers said.
"This potential therapy was developed by StemCells Inc., of Palo Alto, Calif., which is sponsoring the clinical trial with its proprietary human neural stem cells, called HuCNS-SC. Theses stem cells are isolated from normal fetal brain tissue, purified, expanded and then stored in frozen cell banks until they are transplanted," the researchers said.
"We will begin with one patient, but we are not going to continue on with other patients until all of the safety protocols evaluated in the first patient have been met," Dr. Nathan Selden, the Campagna associate professor of pediatric neurological surgery and head of the Division of Pediatric Neurological Surgery at Doernbecher and OHSU School of Medicine, said at the briefing.
"It is our hope that this trial will provide insight into a potential treatment option for this tragic disease," Steiner said. "This trial is just the beginning in a lengthy, ongoing effort to determine a safe and effective means to improve the quality of life of those suffering from NCL," he said.
President George Bush restricted federal funding of Embryonic stem-cell research from Aug. 9, 2001. This had had a serious impact on Embryonic stem-cell research in the United States. These funds are available only for study of stem cell lines derived from embryos that had been destroyed before the limit was set.
Several researchers argue that in spite of the availability of some state, private and university money, including Stanford and Harvard universities, it is not enough on its own.