Funds Boost Genetic Research in Woodridge Lab Seeks to Find Cure for Spinal Muscular Atrophy

Category: Research & Development
Date: February 19, 2003
Source: Chicago Daily Herald

Author: Jonathan Wegner Medill News Service

Families of Spinal Muscular Atrophy, a Libertyville-based non- profit organization, have entered into an agreement with Decode Genetics Inc.' s pharmaceuticals group to develop potential treatments for the debilitating genetic disease.
"We're very excited about the lead compounds," Lenna Scott, a spokeswoman for FSMA, said of last week's agreement. "We believe that working with Decode, we will be able to find a drug for the successful treatment of SMA and will be able to move on to human testing."

The agreement, worth an estimated $5.2 million, contracts Decode Genetics' Woodridge lab to investigate promising compounds identified in previous FSMA-funded drug discovery work.

Spinal muscular atrophy is a genetic disorder that can inhibit the functioning of the spinal cord and affect control of muscles in the limbs, neck and chest. According to FSMA, in the United States the disorder affects one in 6,000 babies less than 18 months old. More than 6 million people in the country carry genetic risk factors for it.

Audrey Lewis, executive director of FSMA, said it will retain rights to drugs developed by Decode. She added that any drugs developed through the collaboration will be provided to the families affected by SMA at affordable prices.

"It's really important to not be cost prohibitive," Lewis said.

FSMA, which consists of more than 5,000 families affected by the disease, has spent more than $12 million on SMA research and has pledged an additional $8 million over the next three years.

"One of the remarkable things is they've been able to do it on their own by working with the families," said Edward Farmer, Decode's director of corporate communications. "It's not a disease that's got a lot of attention, and they don't have a celebrity spokesperson. It's just the families."