Updated on 1 October 2013
The drug is derived from hydroxylamine, a molecule chemically similar to ammonia.
Singapore: Researchers at the National Institutes of Health (NIH) have identified a potential new drug that could help in the treatment of a form of Batten disease, a fatal childhood disorder. The researchers tested the drug in mice with the disease and found that it slowed the loss of coordination seen in the disorder, and extended the animals' life span.
The drug is derived from hydroxylamine, a molecule chemically similar to ammonia. Hyroxylamine is toxic, but a slight change in the molecule's chemical structure results in a non-toxic molecule, called NtBuHA, short for N-(tert-Butyl-Hydroxylamine).
The term Batten disease refers to a group of disorders resulting in deterioration of the nervous system. These disorders occur in one of every 12,500 births, according to the study authors.
"The NIH researchers have found a promising lead for treating a devastating disease that has defied all attempts to treat it," said Dr Constantine A. Stratakis, director of the Division of Intramural Research at the NIH's Eunice Kennedy Shriver National Institute of Child Health and Human Development.
The researchers hope NtBuHA will be useful for treating a particular subtype of the disease, infantile Batten disease. With infantile Batten disease, children appear normal at birth, but experience a gradual, but steady, loss of brain tissue. By 11 to 18 months, they experience difficulty with physical coordination and begin to lose their vision. By age four, they go blind and have no apparent brain activity. They may live in a vegetative state for several more years before dying.