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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Ah Bobby, You knew you would rope me into this one,didn't you?. I have been granted Compassionate Use by the FDA and have taken PPX for over 14 months. Some of my parts are greatly improved, and some keep on slowly progressing. I was never asked to pay for my drugs but was informed of the financial situation. Also I should say here that I am from the generation before entitlements and find it reasonable to pay for what I need..For this partucular drug, I would have sold everything I own to participate in the study, and still would. This drug works. I hope everyone will make a sizeable donation to UVA for further research. In my opinion what good is money when I am dead. Since you only have one trip around, I am asking all of you to write a check to UVA. Your good friend, Susan

Paved With Good Intentions
By Sara Gambrill

The financial model of the Center for the Study of Neurodegenerative Diseases at the University of Virginia leaves much to be desired. Facing shortfalls during a clinical trial studying a drug to treat amyotrophic lateral sclerosis (ALS), James P. Bennett, Jr., director of the center and the physician sponsor of the clinical trial, solicited trial subjects for money to continue to study the drug’s effects on them, according to an article in the Wall Street Journal.

Though Dr. Bennett divested himself of any financial interest in the study drug, the ethical breach is glaring. A small biotech company does have a financial stake, however. It acquired the license for the drug from the university a year ago. As part of the licensing agreement, the company has committed $300,000 to Dr. Bennett’s lab.

Fundamental to what contributed to this situation in the first place is not having the kind of operation that is financially capable of running a clinical trial. This comes at a time when disease foundations are getting savvy about the business end of finding a cure. As this case makes clear, they have to be.

Cystic fibrosis (CF) affects the same number of people as ALS in this country—about 30,000. But the CF Foundation has a well-established, well-run organization to fund clinical trials. The foundation established a Therapeutics Development Program in 1998 with a $20 million grant from the Bill and Melinda Gates Foundation.

It has a 250,000-volunteer grassroots organization raising money for it and its drug discovery and development affiliate, CF Foundation Therapeutics (CFFT), which has committed more than $215 million with biotechnology companies to discover new compounds to create a pipeline for cystic fibrosis of about 30 different products. Dr. Bennett, and the ALS Association, could learn something from CFFT...


Robert J. Beall, Ph.D., president and CEO, CFFT, said, “We knew we would have to establish a unique business model to interest biotechnology and pharmaceutical companies to enter the field of cystic fibrosis. We felt that the best thing we could do was reduce these companies’ financial risk by supporting early stage drug discovery.”

The foundation has $75 million earmarked for this year alone and collaborates with 24 biotechs.

What distinguishes CF from ALS is the discovery of the gene that causes CF and a lack of information about whether genes and/or environmental factors contribute to developing ALS. Compared with a single-gene disease such as cystic fibrosis, the path to success is less clear for ALS, making the risks of investing in a therapy greater. But those risks should be borne by a disease organization or industry, government, academia or some combination—not patients themselves.

Though Dr. Bennett’s intentions to run the clinical trial appear to be good, there simply is no excuse for soliciting money from trial subjects. He should have worked out the financials first. That’s the truly ethical thing to do.

Sara Gambrill, Senior Editor at Thomson CenterWatch.

I have mixed feelingss. As always buyer beware. Examples of scams are plentifull. However, Dr. Bennett's precarious situation only highlights our plight. In the end I must as what is unethical? Dr. Bennett asking for funding or the lack of funding of ALS and other orphan diseases?