Oncogene Sciences Inc. announced Thursday that it willcollaborate with Cold Springs Harbor Laboratory and theMedical Research Council to explore a new treatment forDuchenne muscular dystrophy (DMD). Oncogene of Uniondale,N.Y., will contribute its expertise in gene transcription and itshigh-throughput robotic drug-screening technology.
The collaborators will develop a therapeutic approachpioneered by Kay Davies, director of the Medical ResearchCouncil Clinical Sciences Centre at Hammersmith Hospital inLondon. Davies believes the stimulation of utrophin production,a fetal form of dystrophin, will functionally eliminate thegenetic defect that causes DMD.
Muscular dystrophy is caused by a lack of dystrophin, a proteinnecessary for muscle fiber integrity. In healthy people,dystrophin is produced after about 23 weeks of gestation. Untilthen, the body instead produces utrophin. In fetuses with DMD,the gene responsible for the production of dystrophin ismutated; utrophin production drops before the child is bornand the debilitating, progressive genesis of the disease begins.
A body of existing literature, reviewed by Davies and J. Tinsleyin Neuromuscular Disorders last year, suggests that in mice,utrophin can serve the same function as dystrophin withoutany obvious side effects.
Utrophin appears to interact with dystrophin-associatedglycoproteins (DAGs) to prevent damage to muscle cellstructures. However, researchers have not yet been able tostimulate the excess production of utrophin needed to achieveprotection.
Cold Spring Harbor Laboratory in Cold Spring Harbor, N.Y., anon-profit research and educational institution, will providebasic genetic research, while Oncogene will use its ability toscreen more than 100,000 compounds per week to testpotential therapeutic agents. Through gene transcriptions, theresearchers hope to find compounds that "switch on" the generesponsible for producing the utrophin protein.
Oncogene (NASDAQ:ONCS) and Cold Spring Harbor Labs willeach contribute approximately $200,000 to the initial phase ofthe project. If initial drug discovery is successful, an Oncogenespokesman said, the collaborators will seek about $3 million inadditional funding. Royalty arrangements for any productsdeveloped by the collaborators have not been disclosed.
The Oncogene spokesman said that if successful, thecollaborators should overcome the obstacles of current DMDtherapy. DMD patients are currently treated with steroids, genetherapy or myoblast transfer. Steroid treatment is accompaniedby a wide range of side effects and cannot completely controlthe disease, the spokesman said, while gene therapy isincapable of addressing dystrophin production in the brain.Myoblast transfer is impractical because it requires thetransfer of healthy muscle cells at a large number of sites.
-- Karl A. Thiel Associate Editor
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