Children's research facility takes on some of medicine's biggest puzzles

With 272 discoveries in its first 3 years, institute races to help smallest patients

In its first three years, Darby Children's Research Institute has made 272 discoveries, each peeling away another medical mystery.

The seven-story facility opened in 2005 at the Medical University of South Carolina as a place for 11 research programs to tackle some of medicine's most complex problems — cerebral palsy, cancer and congenital heart disease — in some of the smallest patients.

The institute's executive director, Dr. Bernie Maria, said science moves at a breakneck pace, and the still-young institute is stretching into its stride.

Among the institute's most recent breakthroughs is a medicine to help prevent at-risk babies from developing cerebral palsy, a neurological disorder that affects movement and muscle coordination.

Prenatal infections that damage the brain are a primary cause of cerebral palsy. Dr. Inderjit Singh, an expert in childhood brain diseases, discovered that n-acetyl cysteine, a medicine used for treatment of acetaminophen poisoning, seems to protect developing brains of unborn babies exposed to inflammation.

Dr. Doe Jenkins then translated Singh's work into a clinical trial to give the medicine to pregnant women who have fevers before birth.

"That's turning a lot of heads around," Maria said. "This is the first preventive treatment for cerebral palsy."

Maria, a pediatric neuro-oncologist, made the March cover of Clinical Cancer Research with his work on a treatment for particularly hard-to-kill cancer cells.

Those treatment-resistant cells have a coating, Maria said, and he found a way to take a small piece of the coating and displace it, effectively shutting down the cancerous growth by using the body's own resources.

The technology has been patented and licensed to Halozyme Therapeutics for production.

"We have a lot of buzz," Maria said.

Reach Jill Coley at 937-5719 or jcoley@postandcourier.com.