The March of Dimes has set two main priorities for the coming decades in the fight against birth defects: Gene therapy. In this approach, a genetic disorder might be cured in an individual by supplying a missing gene or repairing a defective one. Still experimental, the technique will be used first on diseases that are caused by a single errant gene. The most likely candidates for gene therapy will be patients with adenosine deaminase (ADA) deficiency, a severe hereditary immune disorder, or those with a Lesch-Nyhan syndrome, a rare form of cerebral palsy. Research with monkeys has been encouraging, but when the technique will be tried on human patients is not known. Prevention of premature labor. Babies born prematurely at a low birth weight are at greatly increased risk of handicaps -- including mental retardation, lung disease, impaired sight and hearing, seizure disorders and behavioral and learning disabilities. About 15 percent of babies weighing less than 3 1/3 pounds, for example, develop cerebral palsy. While more than 95 percent of premature babies survive, thanks to advances in medical care, some have severe brain damage and other disabilities.
What's more, premature births are more common in the United States than in other countries, which helps explain why the U.S. ranks 18th behind other nations in infant survival.
The goal of the nonprofit voluntary health agency is to raise $150 million for research in these two areas. "That will be good for a start," said Charles L. Massey, president of the foundation, at the National Press Club last week.