Thanks to Efforts of ALS Patients, U.Va. Doctor Can Keep Giving Drug

[Marie_LPN, RN, LPN, RN]

Thanks to Efforts of ALS Patients, U.Va. Doctor Can Keep Giving Drug

BY TAMMIE SMITH

TIMES-DISPATCH STAFF WRITER

Apr 30, 2005

Bev Nicola, diagnosed in late 2003 with amyotrophic lateral sclerosis, also known as Lou Gehrig's disease, can't speak clearly anymore, so she uses a laptoplike device to communicate.

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She types in sentences, and the machine's mechanical voice speaks them.

So it was a big deal last summer when her grandson, Cory, heard Nicola say the words "Hi, honey" and "yes."

"He was thrilled," Nicola said by e-mail, recalling the episode. ALS, a fatal disease that attacks nerve cells controlling muscle movement, has affected muscles in her mouth and throat, making speaking and swallowing difficult.

Nicola thinks being able to say the simple words clearly and other temporary and small improvements in mobility and strength she experienced last summer were the result of an experimental drug she was prescribed by a University of Virginia researcher.

Nicola was one of 15 people in the eight-week study that was intended to test the safety of the drug pramipexole.

"Several of the people spontaneously said they felt themselves getting better in small but meaningful ways," said Dr. James P. Bennett, director of the Center for the Study of Neurodegenerative ALSDiseases at U.Va.

"That was unexpected. That is what propelled going forward" with additional research, said Bennett, who first learned about the drug around 1996 and "decided to do some science to see if it had any potential."

In the months since the eight-week study, Nicola and others with ALS have rallied friends and supporters to raise money for the research, which had been turned down for grants and was being financed with resources Bennett was able to pull together from various sources.

When the ALS patients learned the research might not go forward at all because of university concerns about insufficient animal studies testing the drug's safety, they started complaining.

Months later, their efforts have gotten results.

Bennett has U.S. Food and Drug Administration approval to continue giving the drug to patients on a "compassionate-use" basis. A university committee that oversees research on human subjects, which had at first nixed further study until more animal studies were done, has also given approval with stipulations.

Donors who learned of the research's possible holdup through word-of-mouth, e-mail and a story reported in The Hook, a Charlottesville weekly, have contributed more than $165,000. That money will pay for a full-time study coordinator and for having more of the experimental drug made.

Nicola's friends and supporters have been part of that effort. They raised about $3,800 by selling barbecue and baked goods last weekend at a church in the Richmond area. They are also planning a charity golf tournament.

"Their lives depend on this research being successful. I think it's incredible they took charge like this," said Cathy Easter, regional director for the ALS Association's chapter representing Washington, Maryland and Virginia.

"This was really driven by patients who participated in the study and who were desperate to see the study continue."

Easter said many supporters sent checks directly to Bennett. "I believe the biggest donation was $25,000 from someone out of state. Most of them were a couple of hundred dollars. It was small amounts. Dr. Bennett was telling me he would get his daily mail, and there might be 15 to 25 checks coming from all parts of the country."

There is no guarantee the drug will help the ALS patients get better. The findings from the small study are anecdotal. But with no effective cure or treatment for ALS, patients believe it is worth trying.

"Most patients live three to four years after diagnosis," said Nicola, 66, who worked in community relations for Philip Morris before retiring. "They put you on a boatload of vitamins, creatine and suggest that you get a feeding tube before you are unable to breathe."

Nicola asked her friends and family to donate to the research in the regular e-mail dispatches she sends to about 85 people that update them on how she is coping with ALS. She also asked them to forward the e-mail request to others.

Bennett said the response has been overwhelming.

"It's really been uplifting," he said. "ALS is a rapidly progressive, fatal, essentially untreatable disease. To live under that burden, yet to have such optimism and to be so proactive, is really quite inspiring."

As soon as more of the drug is made and its purity certified, Bennett will begin the treatment study with 40 people. Nicola will be one of them. The drug pramipexole is a chemical cousin and "mirror image" of the drug Mirapex, which mimics the brain chemical dopamine, and is used to treat Parkinson's disease.

Both drugs protect nerve cells from dying, Bennett said. "The rationale behind my study is to use [pramipexole] so that much higher drug levels can be obtained in the central nervous system to protect nerve cells in ALS patients. As far as the FDA is concerned, they are related but separate drugs in terms of having to carry out toxicology studies," Bennett said.

A different arm of the research will enroll about 10 people and study the drug's safety in higher doses than the 30 milligrams per day given to patients last summer, Bennett said.

From: http://www.timesdispatch.com/scripts/isapi_srun.dll/servlet/Satellite?pagename=RTD%2FMGArticle%2FRTD_BasicArticle&c=MGArticle&cid=1031782446114&path=%21news&s=1045855934842&DPL=JvsIDSP7Dg0m5hcQJfsKFjvlCSE%3d&tacodalogin=yes