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.
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