NEWS- Drug money: ALS study gets <i>WSJ </i>attention

Should patients with fatal diseases be asked to fund the research that might save them? That's the question  the Wall Street Journal posed in an April 10 article about UVA neurologist Jim Bennett. He's the doctor who came up with that plan in an effort to develop a promising drug to treat ALS, the rare but devastating disease also known as Lou Gehrig's Disease.

Facing a funding shortage for his study– which had shown promise anecdotally– Bennett, the subject of the Hook's February 24, 2005 cover story "She's dying: His drug could save her," told patients in September 2004 that the drug he'd discovered on a German laboratory shelf would probably not continue to be available if he didn't find alternate sources of money. He divested himself of any financial interest, and the money from patients, their friends, and families– hundreds of thousands of dollars– rolled in.

That money, coupled with grants and official sources of funds, has allowed Bennett to continue dosing patients suffering from the fatal neurodegenerative disease, and his studies have expanded to several other university locations around the country.

But some ethicists say patients should not be involved in fundraising. One reason: they might not be objective about the research, according to Dan Brock, director of the division of medical ethics at Harvard Medical School, who was quoted in the Journal.

In Bennett's defense, Brock points out that no patients were coerced or denied treatment if they could not afford to contribute. And Bennett, reached after the Journal article appeared, says the early fundraising efforts were initiated by patients– not at his request– and that patients and their families continue to lead the effort. 

"In a very real sense, they made and continue to make the decision about whether any of this has gone/ will go forward," he says. 

The Hook first reported Bennett's study in that 2004 cover story, detailing the early difficulties he and several of his patients faced when a UVA ethics committee ruled that the research could not continue without further safety testing.

Two of those patients, Mary Jane Gentry and Bev Nicola, were outraged by the decision, asking how the committee could cite safety as a concern when the disease would rob them of their ability to walk, talk, and eventually breathe within two to three years of their diagnosis.

The drug, pramipexole, is the chemical mirror image of the Parkinson's drug Mirapex, and like that drug, it is a powerful free radical scavenger. Bennett hypothesized that free radicals play a role in the nerve damage that occurs in ALS, and he believed pramipexole would not have the same serious side effects as Mirapex. 

Following eight weeks of dosing, Gentry and others provided anecdotal evidence that the drug was lessening– and in some cases reversing– their symptoms. But the UVA Human Investigations Committee, the ethical watchdog for human research at UVA, said such reports could be attributed to the "placebo effect," and that patients in dire circumstances should not be asked to serve as experimental subjects when the effects of the drug were not known. Eventually, the Committee and the FDA approved Bennett's request to continue dosing patients.

Now, two years later, Gentry, Nicola and nearly all of the original 15 subjects have died, but Bennett's study continues– and has expanded.

In May 2005, Bennett connected with Knopp Neurosciences, a small Pennsylvania-based drug company willing to develop the drug commercially, a costly and time-consuming undertaking. 

Knopp spokesperson Tom Petzinger says the company has raised nearly $10 million for the commercial development, and has another $20 million in pledged funds. But the commercial studies, still in preclinical stage, could take several years, and no one who has taken any dose of the drug through Bennett's trials is eligible to participate in the commercial trials.

Fortunately, Knopp has pledged $300,000 to Bennett's study, so that those who cannot participate in commercial trials will still have access to the drug.

While research is ongoing, Bennett says he has submitted several articles to medical journals, but it could be several months before any are published. His articles will show the drug is tolerated well in various doses, he says, but none will reveal efficacy, something that can be shown only through larger scale studies, such as the ones to be conducted by Knopp. 

Neither Gentry's nor Nicola's widowers could be reached by press time, but Nicola's widower, Eli, told the Journal he has no regrets about his late wife's participation in the study or in the fundraising.

"The doctor said he had to raise money somehow," said Nicola, "and Bev put everything in her soul into it."

Dr. Jim Bennett's study on a drug to treat ALS is one of the only hopes for individuals with the devastating disease.


1 comment

It is inhumane, if not criminal, to with hold, much less withdraw, ANY possible treatment from informed and consenting any DYING PATIENTS. I also suffer from bulbar form of ALS and in a few months have lost the ability to speak and swallow. I can not believe it is anything other than the competition for dollars (protection of same) in our immoral, unethical society rather than fear of liability. Where are all the Supposed Christian Conservatives ranting and raving when Terry Schiavo was allowed to die?