Note the following news items from the wire services via Compuserve: 06/22 0927 CLINICAL TRIALS SHOW PROMISE FOR NEW PARKINSON'S DRUG PITTSBURGH, June 22 /PRNewswire/ -- A pilot clinical trial shows encouraging evidence that the drug GM1 ganglioside is effective at substantially reducing some of the functional impairments caused by Parkinson's disease. The results are reported in the June issue of Neurology. According to Jon Brillman, M.D., director of neurology at Allegheny General Hospital, these results are the basis for controlled trials being conducted at AGH's medical schools, the Medical College of Pennsylvania and Hahnemann University in Philadelphia. Unlike two controversial Parkinson's procedures recently under public discussion -- fetal cell transplant and pallidotomy surgery -- GM1 ganglioside is non-invasive, self-administered and potentially more accessible to Parkinson's patients. "The drug appears to enhance the brain's ability to produce the key neurotransmitter dopamine," Brillman said. It is the lack of dopamine that leads to the tremors, rigidity and cognitive problems that mark Parkinson's. "The concept of using this drug to help the body produce dopamine shows tremendous promise for the next decade compared to current therapies," Brillman added. According to the study's lead author Jay Schneider, Ph.D., professor of anatomy, neurobiology and neurology, MCPHU, "Though the precise mechanism is not clear, GM1 ganglioside may rescue damaged dopamine neurons, stimulate them to repair themselves, promote growth of new dopamine terminals on the neurons and perhaps even stimulate remaining dopamine neurons to produce more than their normal amount of dopamine." The study reports functional improvements brought about by the drug continued for the duration of the study. Improvements in motor skills and activities-of-daily-living included decreased rigidity and bradykinesia (abnormal slow movement); reduced time required to perform physical tasks; greater ease in walking and dressing; and an increased energy level. Cognitively, the patients were less distractible. Parkinson's disease, a progressive neurological disorder that destroys muscular control, afflicts one percent of all Americans over age 55. "Given a very long life, most people would suffer some effects of the brain's naturally decreasing production of dopamine," Schneider said. Unfortunately, Parkinson's causes a loss of dopamine-producing cells above and beyond that seen in normal aging, and it accelerates the consequences of having too little dopamine in the brain. -0- 6/22/95 /EDITORS' ADVISORY: Call Judy Secreto at 412-359-8604 to arrange an interview with Dr. Brillman or Merrill Meadow at 215-762-8284 to arrange interviews with Dr. Schneider./ /CONTACT: Judy Secreto of Allegheny General Hospital, 412-359-8604/ CO: Allegheny General Hospital; Medical College of Pennsylvania; Hahnemann University ST: Pennsylvania IN: HEA SU: 06/22 0806 CLINICAL TRIALS BEAR OUT PROMISE OF NEW ... /ADVANCE/ PHILADELPHIA, June 22 /PRNewswire/ -- A pilot clinical trial showed encouraging evidence that the drug GM1 ganglioside is effective at substantially reducing some of the functional impairments caused by Parkinson's disease. Those results, reported in the June issue of Neurology, are the basis for controlled trials being conducted at Medical College of Pennsylvania and Hahnemann University (MCPHU), Philadelphia. Unlike two controversial Parkinson's procedures recently under public discussion -- fetal cell transplant and pallidotomy surgery -- GM1 ganglioside is non-invasive, self-administered and potentially more accessible to Parkinson's patients. The drug appears to enhance the brain's ability to produce the key neurotransmitter dopamine; it is the lack of dopamine that leads to the tremors, rigidity and cognitive problems that mark Parkinson's. "Though the precise mechanism is not clear, GM1 ganglioside may rescue damaged dopamine neurons, stimulate them to repair themselves, promote growth of new dopamine terminals on the neurons -- and perhaps even stimulate remaining dopamine neurons to produce more than their normal amount of dopamine," said the study's lead author, Jay Schneider, Ph.D., professor of anatomy, neurobiology and neurology, MCPHU. Dr. Schneider and his colleagues reported that the functional improvements brought about by the drug continued for the duration of the study. Improvements in motor skills and activities-of-daily-living included decreased rigidity and bradykinesia (abnormally slow movement); reduced time required to perform physical tasks; greater ease in walking and dressing, and an increased energy level. Cognitively, the patients were less distractible. Parkinson's disease, a progressive neurological disorder that destroys muscular control, afflicts 1 percent of all Americans over age 55. It is marked by abnormally slow movement, uncontrollable tremors, stooped posture and a shuffling gait. "Given a very long life, most people would suffer some effects of the brain's naturally decreasing production of dopamine," Dr. Schneider explained. "Unfortunately, Parkinson's causes a loss of dopamine-producing cells above and beyond that seen in normal aging, and it accelerates the consequences of having too little dopamine in the brain." Doctors currently treat most Parkinson's patients with L-dopa, which acts as a replacement for their own dopamine. "While L-dopa ameliorates the symptoms, it loses its efficacy within a few years, often causes undesirable side effects, and does not deal with the underlying loss of dopamine-producing neurons," Dr. Schneider noted. Given that GM1 fosters repair and revitalization of remaining neurons, but does not create new neurons, it may prove most effective in early stages of the disease, he said. "However, since laboratory research suggests that only a small portion of the dopamine system needs to be spared or functionally recovered to achieve normal motor and cognitive skills, GM1 could benefit even patients with more established Parkinson's disease -- providing there are enough dopamine neurons remaining for GM1 to act on," Dr. Schneider suggested. The Neurology report involved 10 patients with mild to severe symptoms of the disease, most of whom had been taking L-dopa. At the outset, the patients were given a battery of tests to assess their functional abilities; those tests were repeated four times over an 18-week period. Each patient received a large intravenous dose of GM1 at the trial's beginning, then self-administered 200 mg of the drug each day by injection. Eight patients completed the full 18-week treatment; two patients dropped out early, feeling they were not benefitting from the trial. Among those eight participants, significant improvements in motor skills and ability to perform activities-of-daily-living occurred in the first eight weeks of the trial and stabilized over the remaining 10 weeks. "While the results of this initial study are encouraging, we must be careful not to overstate the significance of these findings," Dr. Schneider observed. "Since the patients in this study all knew they were receiving a new drug, their function may have improved due to psychological factors unrelated to the medication. Yet, since some patients have maintained their original improvement over almost two years now, it is hard to argue these effects are due primarily to psychological factors." The current clinical trial is a double blind, placebo controlled study of 50 patients. If that trial also demonstrates safety and effectiveness in GM1 treatment, the next step would be a multicenter trial that could begin as early as 1996. The big question Dr. Schneider and his colleagues will ultimately try to answer as their research progresses is whether early enough treatment with GM1 or related drugs could alter the course of the underlying disease. GM1 ganglioside is a normal constituent of nerve cell membranes and is known to moderate a number of neuron surface and receptor activities. The type of GM1 used in this study was extracted from cow brain. Dr. Schneider is also studying a semi-synthetic derivative of GM1, called LIGA 20, which could prove even more effective in restoring the function of the dopamine system. The MCPHU research was funded by the F.M. Kirby Foundation, the Marion and Joseph Wesley Foundation, the Alzheimer's Disease Research Center at Hahnemann, and through donations to the Parkinson's Disease Research Fund at Hahnemann. Dr. Schneider's co-authors of the Neurology report were MCPHU colleagues David Rothblat, Ph.D., Jillian Chapas-Crilly, B.S., and Louisa Seraydarian, Ph.D.; David Roeltgen, M.D., of The Health Education and Research Foundation of Williamsport (Pa.) Hospital, and Jayaraman Rao, M.D., of Louisiana State University Medical Center. /delval/ -0- 6/22/95/1700 /CONTACT: Phyllis M. Fisher, Asst. V.P./Communications or Merrill S. Meadow, Media Relations Manager, of Hahnemann University, 215-762-8284/ CO: Medical College of Pennsylvania and Hahnemann University ST: Pennsylvania IN: MTC HEA SU: Bob Cowan <[log in to unmask]>