Featured Research

from universities, journals, and other organizations

Jefferson Scientist Begins Clinical Trial To Study Promising Parkinson’s Disease Drug

Date:
July 22, 1999
Source:
Jefferson Medical College
Summary:
Scientists at Jefferson Medical College, armed with a newly awarded $2.4 million grant from the National Institutes of Health, hope to find out whether a promising, drug, GM1 ganglioside, can improve symptoms, delay disease progression, and in some cases actually restore damaged brain cells in Parkinson’s disease patients.

GM1 ganglioside may improve symptoms, delay disease advance

Related Articles


Scientists at Jefferson Medical College, armed with a newly awarded $2.4 million grant from the National Institutes of Health, hope to find out whether a promising, drug, GM1 ganglioside, can improve symptoms, delay disease progression, and in some cases actually restore damaged brain cells in Parkinson’s disease patients.

Jay S. Schneider, PhD, professor of pathology, cell biology and anatomy and neurology at Thomas Jefferson University in Philadelphia, will lead a five-year clinical trial involving 150 patients. The study will compare the effectiveness of GM1 ganglioside, a naturally occurring substance in the nerve cell’s membrane that plays an important role in cell growth, development, and repair, to standard Parkinson’s disease treatments, which improve symptoms but do not alter the disease process.

"We hope that we will be able to do something no one else has done before – to stimulate remaining brain cells to sprout new nerve endings and rescue other cells that might otherwise die," says Dr. Schneider, who last year published evidence that GM1 may improve symptoms in Parkinson’s disease patients and perhaps even help slow progression of the disease.

"Current therapies for Parkinson’s disease treat only the symptoms but do little to address the underlying disease process," he notes. "Despite the fact that you can alleviate some symptoms for a time, the disease process continues. It’s a progressive neurodegenerative disorder. GM1 has the potential to be a disease-altering therapy.

"If we are able to show that we’ve stimulated repair and regrowth as we and others have done in the laboratory using animal models, we will have evidence for the first time of a therapy that can help restore the part of the nervous system damaged by a neurodegenerative disease in humans," Dr. Schneider says.

He and his team published results of a smaller study in June 1998 in the journal Neurology showing that GM1 treatment can improve symptoms in Parkinson’s patients. "This new study may help explain the mechanisms behind these results and also demonstrate that GM1 helps to slow progression of the disease," he says.

The Jefferson team is collaborating with scientists at the University of Pennsylvania in Philadelphia to perform a special type of imaging called single photon emission computed tomography, or SPECT. SPECT will allow the team to visualize the number of dopamine terminals in the striatum, the part of the brain that receives dopamine from the substantia nigra, which is the brain region that dies in Parkinson’s disease. Comparing patient symptoms and the number of dopamine terminals is a significant advance, notes Dr. Schneider. "This technology will give us important new insights into the relationship between the expression of the symptoms of Parkinson’s disease and the actual amount of dopamine terminals in the brain and will help us better track the progression of the disease."

The study is a randomized, double-blind placebo controlled trial in which neither the researchers nor the patients know who receives the drug. The participants, patients with mild to moderate Parkinson’s disease, will be divided into three groups of 50 each. One group will receive the drug during the first two study phases. Another 50 will receive a placebo during phase one, and will be allowed to get the drug later in the second trial phase. A third control group of 50 will receive neither the study medication nor placebo, but will receive standard Parkinson’s therapy. This latter group will have the option of receiving study medication when the study period ends.

"We’re hoping that in the first six months of the study we’ll see the same kind of symptomatic improvement we’ve seen in previous studies with GM1, as well as a small increase in the number of dopamine terminals shown in subsequent SPECT scans," Dr. Schneider says.

"The second part of the trial will examine whether long-term use of GM1 actually influences the course of Parkinson’s," he says. This study phase will address the question of whether a difference exists between the rate of symptom advancement and loss of dopamine terminals in GM1 versus non-GM1-treated patients.

More than one million people in the United States suffer from Parkinson’s disease. It commonly strikes people over 50. Symptoms include hand tremor, slowness in movement, difficulty initiating movements, difficulty walking, shuffling feet, decrease in speech volume, fatigue, and balance problems.

The GM1 ganglioside, which has also shown promise as a treatment for acute spinal cord injury, is being supplied by the drug’s manufacturer, Fidia SpA of Abano Terme, Italy, under the name Sygen¨. Fidia is Italy’s fourth largest pharmaceutical company with an American subsidiary, Fidia Pharmaceutical Corporation, in Washington, D.C.

For more information about participating in the clinical trial, please call 1-800-JEFF NOW.


Story Source:

The above story is based on materials provided by Jefferson Medical College. Note: Materials may be edited for content and length.


Cite This Page:

Jefferson Medical College. "Jefferson Scientist Begins Clinical Trial To Study Promising Parkinson’s Disease Drug." ScienceDaily. ScienceDaily, 22 July 1999. <www.sciencedaily.com/releases/1999/07/990722064908.htm>.
Jefferson Medical College. (1999, July 22). Jefferson Scientist Begins Clinical Trial To Study Promising Parkinson’s Disease Drug. ScienceDaily. Retrieved November 24, 2014 from www.sciencedaily.com/releases/1999/07/990722064908.htm
Jefferson Medical College. "Jefferson Scientist Begins Clinical Trial To Study Promising Parkinson’s Disease Drug." ScienceDaily. www.sciencedaily.com/releases/1999/07/990722064908.htm (accessed November 24, 2014).

Share This


More From ScienceDaily



More Mind & Brain News

Monday, November 24, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

Could Your Genes Be The Reason You're Single?

Could Your Genes Be The Reason You're Single?

Newsy (Nov. 21, 2014) — Researchers in Beijing discovered a gene called 5-HTA1, and carriers are reportedly 20 percent more likely to be single. Video provided by Newsy
Powered by NewsLook.com
Milestone Birthdays Can Bring Existential Crisis, Study Says

Milestone Birthdays Can Bring Existential Crisis, Study Says

Newsy (Nov. 21, 2014) — Researchers find that as people approach new decades in their lives they make bigger life decisions. Video provided by Newsy
Powered by NewsLook.com
You Don't Have To Be Alcohol Dependent To Need Treatment

You Don't Have To Be Alcohol Dependent To Need Treatment

Newsy (Nov. 21, 2014) — A study by the Centers for Disease Control and Prevention found 9 out of 10 excessive drinkers in the country are not alcohol dependent. Video provided by Newsy
Powered by NewsLook.com
Your Complicated Job Might Keep Your Brain Young

Your Complicated Job Might Keep Your Brain Young

Newsy (Nov. 20, 2014) — Researchers at the University of Edinburgh found the more complex your job is, the sharper your cognitive skills will likely be as you age. Video provided by Newsy
Powered by NewsLook.com

Search ScienceDaily

Number of stories in archives: 140,361

Find with keyword(s):
 
Enter a keyword or phrase to search ScienceDaily for related topics and research stories.

Save/Print:
Share:  

Breaking News:

Strange & Offbeat Stories

 

Health & Medicine

Mind & Brain

Living & Well

In Other News

... from NewsDaily.com

Science News

Health News

Environment News

Technology News



Save/Print:
Share:  

Free Subscriptions


Get the latest science news with ScienceDaily's free email newsletters, updated daily and weekly. Or view hourly updated newsfeeds in your RSS reader:

Get Social & Mobile


Keep up to date with the latest news from ScienceDaily via social networks and mobile apps:

Have Feedback?


Tell us what you think of ScienceDaily -- we welcome both positive and negative comments. Have any problems using the site? Questions?
Mobile iPhone Android Web
Follow Facebook Twitter Google+
Subscribe RSS Feeds Email Newsletters
Latest Headlines Health & Medicine Mind & Brain Space & Time Matter & Energy Computers & Math Plants & Animals Earth & Climate Fossils & Ruins