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CytRx Signs Deal with Mass General to Develop RNAi-based Therapies for ALS

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In line with its strategy to identify and strike RNAi collaborations with key academic researchers, CytRx said last week that it had inked a partnership with Massachusetts General Hospital, one of Harvard Medical School’s teaching hospitals, to develop drugs for amyotrophic lateral sclerosis. The deal comes on the heels of a separate alliance CytRx formed with UMass Medical School to develop a cure for the disease.

The Mass General project, which has an initial term of three years, will be led by Robert Brown, director of the Cecil B. Day Laboratory for Neuromuscular Research and professor of neurology at Harvard Medical School, according to CytRx. Under the arrangement, CytRx will provide Brown’s lab with quarterly research payments of about $70,000 each. CytRx, which will own all commercial rights to discoveries stemming from the deal, will also be responsible for funding all clinical work.

Brown “called us when he read that we were going to do an ALS project in RNAi at the University of Massachusetts Medical School with Dr. [Zuoshang] Xu,” CytRx CEO Steven Kriegsman told RNAi News. “He knew of that project and he felt that … he should team up with us.”

The UMass project, Kriegsman noted, remains separate from the partnership with Mass General, but the two could be combined down the road if the potential for synergies is seen.

Brown’s work is focusing on mutations in the superoxide dismutase 1 (SOD-1) gene. In 1993, Brown and a number of other researchers published a paper in Nature (1993: 362, 59-62) demonstrating a link between SOD-1 and autosomal dominant familial ALS (FALS), a form of the disease which Brown said is typically fatal within a year or less.

“It took about 10 years, [and] involved traditional genetic linkage analysis using Mendelian families,” he told RNAi News. The result of a decade’s work: The researchers identified 11 different SOD-1 mutations in 13 FALS families.

“By and large these are all diseases that are predominantly inherited, and they are typically missense mutations,” Brown said. “The model is that, one way or another, the production of toxic protein is what causes the disease. So clearly, this is a fantastic opportunity to apply RNAi technology to treat a disease.”

Optimistic about the collaboration, Kriegsman said that a large portion of the preclinical work in the project has been completed and that he hopes to see a drug candidate in primate testing soon. Human trials, he added, could start next year. According to Brown, however, the only work that has been done is in a Petri dish, and experiments in mouse models are expected to start shortly.

Brown declined to offer a specific timeline as to when the mouse model testing would be completed, noting that “we’re going to work as expeditiously as we can.” As for Kriegsman’s hopes that phase I testing will start in 2004, Brown said, “I love his enthusiasm and hope we can move this as rapidly as possible to clinical application.”

The path to the clinic will involve assessing a number of possible delivery approaches for the ALS drug, and Brown said the most likely one will involve a viral vector targeting the spinal cord, possibly through direct spinal cord or intramuscular injection.

The use of viral vectors “is almost obligatory until a better strategy evolves,” Brown said. He added that while he is aware of the concerns people have over the use of viral delivery, “if I have a viral system that shuts down a protein that is lethal within a year, I think it is an ideal setting in which to explore the use of those viral systems in the absence of any other treatment.”

—DM

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