Medical school research gets boost with transaction

A little-noticed deal last month brought a promising research discovery at the University of Nevada Medical School closer to commercial development.

At the same time, the complex transaction illustrates the often-long path that researchers and entrepreneurs will trod together as they commercialize university research.

In last month’s transaction, Alexion Pharmaceuticals, a global biopharmaceutical company headquartered at Cheshire, Conn., signed an option to acquire Prothelia Inc., a privately held outfit in Milford, Mass.

The primary attraction: Prothelia’s license with the University of Nevada Medical School for Laminin-111, a patented therapy that holds promise for possible treatment of congenital muscular dystrophy in humans.

Alexion will fund additional research on Laminin-111 at the medical school and will decide whether to exercise its option to acquire Prothelia once the results are known.

The upshot for Dean Burkin, the professor and pharmacological researcher who leads the project: “This is huge. I haven’t been able to sleep.”

And top officials of the university see the transaction as yet another tool to attract world-class research teams to Nevada.

With the involvement of Alexion, Burkin says researchers now see a pathway to bring Laminin-111 to phase-one clinical trials, a critical and expensive step toward winning the FDA approval that’s necessary to bring the therapy to market.

The market isn’t large.

Laminin-111 therapy targets MDC1A, an ultra-rare genetic disease for which doctors currently can provide no treatment.

“These children die,” says Burkin. “It’s a devastating diagnosis for families.”

Alexion specializes in development and production of drugs for ultra-rare diseases that are life-threatening. The Laminin-111 therapy has received “orphan drug” status from the FDA, a designation that provides financial incentives for development of drugs that treat diseases with fewer than 200,000 sufferers in the United States.

The Laminin-111 therapy developed by researchers in Reno relies on a naturally occurring protein. Burkin’s team believes that protein-replacement therapy might compensate for a genetic deficiency in MDC1A patients.

Burkin, a native of New Zealand, published his first research findings in 2001.

He came to the University of Nevada in 2003, drawn in part by a research-friendly environment for himself and his team of post-doctoral students in its Department of Pharmacology.

“They’ve nurtured me and cultured me,” he says. “It’s been fantastic.”

As the research continued, the university’s Technology Transfer Office in 2007 filed a patent covering the work, and the patent was granted in 2008.

In the meantime, Prothelia Inc. licensed Laminim-111 and began chasing down grants to help fund further research. Organizations such as the Muscular Dystrophy Association provided much of the research funding.

Even with the additional financial muscle provided by Alexion, completion of clinical trials are likely to be time-consuming, Burkin says.

The research helps establish Nevada as a center for advanced medical research, says Mridul Gautam, the vice president and research and innovation at the University of Nevada, Reno. That’s one of the sectors that’s been targeted by the Governor’s Office of Economic Development as a cornerstone of Nevada’s economic future.

“This work is a magnet that will attract people and attention,” says Gautam. “Role models are extremely important.”


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