April 10, 2007, 9:20 am

Patient, Fund Thyself
Posted by Jacob Goldstein

A University of Virginia researcher recently asked patients with a degenerative neurological disease to fund their own drug trial, Amy Dockser Marcus writes in this morning’s WSJ. The unusual strategy (experimental drugs are usually free to patients in clinical trials) raises ethical questions and highlights the difficulties in finding funding for rare diseases.

James P. Bennett Jr., a University of Virginia neurologist, believed that a drug called R(+) Pramipexole could help patients with amyotrophic lateral sclerosis, a deadly neurological disorder also known as ALS or Lou Gehrig’s disease. But when he wanted to launch a trial a few years ago, Bennett couldn’t interest manufacturers in the drug, which was patented by the university. This was likely due at least in part to the fact that ALS, which strikes only about 5,600 patients per year, does not present a large market for drug makers.

Bennett (who has no financial interest in the drug) started a trial anyway, paying a manufacturer to make the drug and eventually writing letters to patients asking for money to keep the research going. He assured everyone that they would have access to the drug whether or not they donated money. Still, the move created an thorny ethical situation in which patients might have felt “beholden” to the researcher, one ethicist told the Journal. Some patients paid; others didn’t. “I can’t financially contribute to the trial,” Henry Gardner (pictured above), whose disease forced him to retire from his job selling heavy equipment, told the Journal. “It’s not the place of the patients.”

For now, the financial crisis has passed. A large individual donor gave enough money to keep the trial going through the summer. And Pittsburgh-based Knopp Nuerosciences licensed the drug and is raising money to continue its development.

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