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.