Date: Sun, 24 Sep 95 02:43:11 -0400 From: Bob Broedel To: als@huey.met.fsu.edu Subject: ALSD216 ALS-ON-LINE =============================================================== == == == ----------- ALS Interest Group ----------- == == ALS Digest (#216, 23 September 1995) == == == == ------ Amyotrophic Lateral Sclerosis (ALS) == == ------ Motor Neurone Disease (MND) == == ------ Lou Gehrig's disease == == ------ maladie de Charcot == == == == This e-mail list has been set up to serve the world-wide == == ALS community. That is, ALS patients, ALS researchers, == == ALS support/discussion groups, ALS clinics, etc. Others == == are welcome (and invited) to join. The ALS Digest is == == published (approximately) weekly. Currently there are == == 1030+ subscribers. == == == == To subscribe, to unsubscribe, to contribute notes, == == etc. to ALS Digest, please send e-mail to: == == bro@huey.met.fsu.edu (Bob Broedel) == == Sorry, but this is *not* a LISTSERV setup. == == == == Bob Broedel; P.O. Box 20049; Tallahassee, FL 32316 USA == =============================================================== == Back issues of the ALS Digest are available on-line at: == == http://http1.brunel.ac.uk:8080/~hssrsdn/alsig/alsig.htm == =============================================================== CONTENTS OF THIS ISSUE: 1 .. Request for Study Participants 2 .. New e-mail address 3 .. ALS - accident related? 4 .. ALS funding 5 .. Whirlpools 6 .. Understanding Needed 7 .. ALS drug approval 8 .. Lou Gehrig's Drug 9 .. Riluzole (1) ===== Request for Study Participants ========== Date : Wed, 13 Sep 1995 21:52:01 EDT >From : TDVT81A@prodigy.com (MR MARSHALL D PINES) Subject: Request for Study Participants We contribute to each other and to those who follow us in several ways. We share information and experiences in dealing with the disease. We participate in drug trials that may lead to effective therapies. We engage in advocacy activity to speed the availability of those therapies. We offer a willing ear to those who wish to articulate their concerns. There's one more thing that some of you may be able to do. It's available primarily to the younger among us. Northwestern University's Dr. Teepu Siddique was instrumental in finding a gene mutation affecting a large number of people with familial ALS. You will recall it as the defective SOD1 issue. Dr. Siddique is expanding the study in an attempt to locate a genetic foundation that may contribute to the development of sporadic ALS. There may be no immediate benefit to patients from participating in this study. However, participation may be helpful in leading to an understanding of sporadic ALS. All clinical and personal information will be kept confidential. The study needs blood samples from patients with sporadic ALS (no family history of ALS) and from their parents, brothers and sisters. The following combinations are appropriate for this study: * Patient and both parents (preferable) or * Patient and one parent only with one or more siblings or * Patient, no parents but two or more siblings The research coordinator for the study is Tony Juneja. He will send you the necessary information. Tony may be reached by: Phone: 312/503-2712 FAX: 312/908-0865 Internet: juneja@merle.acns.nwu.edu ========================= There will be no costs incurred on your part. And maybe, just maybe, you will contribute to solving the puzzle that characterizes this disease. Marshall (2) ===== New e-mail address ========== Date : Tue, 19 Sep 1995 18:04:22 -0400 >From : alssoc@inforamp.net (Jan Rodman) Subject: New e-mail address It's official. Our new address is "alssoc@inforamp.net". =================== Please advise everyone on the ALS Digest list. Thanks. Jan =============================================== Jan Rodman, National Executive Director ALS Society of Canada "We're fighting Lou Gehrig's Disease with everything you give!" phone: 416-362-0269 or toll-free 1-800-267-4ALS (4257) fax: 416-362-0414 e-mail: alssoc@inforamp.net =============================================== (3) ===== ALS - accident related? ========== Date : Fri, 15 Sep 1995 21:28:49 +0100 >From : killeen@iol.ie (Steve Killeen) Subject: ALS ~accident related I developed ALS (I'm 42) after a back injury in 1993 - not a serious one but it nagged at me for months. I know of a young man who had a neck injury and developed ALS right after. Both of us had tetanus vaccinations between injury and diagnosis. Edgar Cayce (US Physic) also reckoned that tonsils & appendix are part of the lymphatic system. I had my appendix out as a kid. My neurologist said that my injury was a "Red Herring". Steve Killeen Dublin, Ireland. Tel 2831971 (4) ===== ALS funding ========== Date : Fri, 22 Sep 1995 16:34:28 -0500 >From : rosen@wadsworth.org (Daniel Rosen) Subject: ALS funding >From a story in "The Scientist", Sept 18, 1995 (5(18):1ff ******************************************** Pressures Wearing Down Researchers by Myrna E. Watanabe The pressures of practicing science in the 1990s are taking their toll on researchers in the United States and throughout the world. Some of the evidence is clear; rising unemployment and underemployment, as well as a ferocious competition for rapidly dwindling resources. Other signs, scientists say, are less obvious -- increased research misconduct, sexual discrimination, disrupted family and personal lives, and the creation of "serial postdocs" with less and less of a chance of ever obtaining a laboratory of their own. Much of their plight is told in cold facts and figures: in reports and surveys conducted by government agencies, society task forces, sociologists, psychologists, and employment analysts. But perhaps the most compelling testimony is given by the scientists themselves, in interviews, over the Internet, and in published accounts. Some of these tales take on an almost soap opera-like quality: (parts deleted) More and more researchers announce that they are abandoning the academic science rat race. Their reasons vary and their committment to this course also varies. (parts deleted) Dan Wetzel, with a Ph.D. in neuroscience from Princeton University and several top postdocs, is looking at unemployment's ugly face. He is collecting unemployment insurance and living on his wife's modest earnings as a home sewer for the Vermont Teddy Bear Co. His research on Alzheimer's disease and amyotrophic lateral sclerosis (ALS) was not funded and he lost his position as a research assistant professor at University of Vermont School of Medicine on June 30. "I have no illusions about staying in science, and [I'm] happy to leave," he firmly states. (parts deleted) Daniel R. Rosen, Ph.D. Wadsworth Center for Laboratories and Research New York State Department of Health (5) ===== Whirlpools ========== Date : Wed, 13 Sep 1995 21:45:32 -0400 (EDT) >From : NAME Subject: Whirlpools I have a specific question regarding the benefits of Whirlpool baths for the treatment of ALS aches and pains. A group of friends are considering a fund raising effort as a gift to an ALS diagnosed friend. If anyone could help answer this question it would be appreciated. (6) ===== Understanding Needed ========== TITLE : LOU GEHRIG -- UNDERSTANDING NEEDED COLUMN: LETTERS TO THE EDITOR DATE : 09/17/95 SOURCE: The Seattle Times Printing John Eisenberg's Baltimore (Sun. Times, Sept. 5) article, "Peeking into the Past" was unfortunate in that the author describes Lou Gehrig's illness as bizarre. This word bizarre is not helpful to persons with amyotrophic lateral sclerosis or ALS, better known as Lou Gehrig's disease. It is a tragic, perhaps sad, maybe unfortunate, but not bizarre. People with this disease lose all motor function and retain all mental faculties. They are misunderstood when they can't smile at your jokes, can't answer your questions, and can't shake your hand. People don't realize they are dealing with a competent mind and a useless body. More understanding is needed, not useless descriptions. Kathleen Wagner, Seattle (7) ===== ALS drug approval ========== Date : Wed, 20 Sep 1995 21:28:00 EDT >From : TASM76A@prodigy.com (MR TED HEINE) Subject: a.l.s. drug approval Report on the FDA Hearing, September 19, 1995 I am very grateful for the oppportunity I had to speak to the FDA Central and Peripheral Nervous System Advisory Panel on behalf of myself and other ALS patients. I regret I did not make a list of all the speakers but the total impact was very emotional, emphasizing the importance of life extension to each of us, and I believe our presence was essential to the panel's positive recommendation, which passed on the very narrow vote of 5-4. The panel used a different procedure from normal. Normal has the drug sponsor make a presentation, followed by an FDA staff presentation. There was only one presentation of data, since RPR and the FDA had agreed on it. The problems lay with the interpretation of data and focussed on three areas: 1. The efficacy of Rilutek is marginal. 2. No efficacy was demonstrated in the North American trials; all positive benefits resulted from the France-Belgium branch of the trial. 3. After the patient population was divided, post hoc, into "low risk" and "high risk" groups, all the efficacy remained with the "high risk" group, and the low risk group showed no benefit. Several members of the panel felt that more data should be collected, perhaps in particular from the on-going trial in Japan, to get a clearer picture of efficacy, before they could recommend positively. Others felt that there was enough to go on, and that delay would only deny present patients an opportunity for life extension. My impression of the discussion is that a critical factor was missed: Rilutek clearly extended the lives of high risk patients in the short run but had no measurable impact on low risk patients. Logically, short run benefit for high risk patients equals long run benefit to low risk patients, because low risk patients turn into high risk patients. Based on my logic, the trial was just too short to measure the benefit to low risk patients. What all of us need to do right now is to write to FDA Commissioner David Kessler and ask him to accept the recommendation of the advisory panel and to give his final approval for drug marketing quickly, speedily, expeditiously, as soon as possible. (You get the idea) The address is: Dr David A. Kessler, Commissioner Food and Drug Administration 5600 Fishers Lane, Rockville, Maryland 20857. Ted Heine in Waverly, Iowa (8) ===== Lou Gehrig's Drug ========== APn 09/19 0115 Lou Gehrig's Drug By LAURAN NEERGAARD, Associated Press Writer ROCKVILLE, Md. (AP) -- The nation's 30,000 sufferers of Lou Gehrig's disease may soon get the first drug to help them live longer -- albeit just a few more months. A divided panel of scientific advisers recommended Monday, on a 5-4 vote, that the Food and Drug Administration approve riluzole, the first drug to ever show effectiveness against the fatal neuromuscular disease. "We recognize it's not a cure," said James Molt, the company's regulatory director. "But Rilutek is an important therapeutic gain." But the FDA panel demanded to know why Rilutek's effect was seen only in Europeans, not the North Americans in the study. "I am just so stunned by that data," said panel chairman Dr. Stanley Fahn of Columbia University, who voted against the drug. "What is the advantage to the American population?" Struggling for an answer, the company separated "high-risk" patients, whose prognosis was worse because of advanced age or rising symptoms, from "low- risk" patients. Only the high-risk group was helped by Rilutek. The company went on to theorize that North Americans were lower risk and received more aggressive treatment for their symptoms, such as feeding tubes and help in breathing. But doctors do not agree whether ALS patients actually can be labeled high- or low-risk. And the company admitted it had not proved its theory about the discrepancy. Still, a majority of the panel voted to recommend FDA approval on grounds that any evidence of even slight help made the drug appropriate. "I will prescribe it to all my patients, and I think other doctors will too," said Dr. Robert Miller of California Pacific Medical Center in San Francisco, who tested the drug. Because the FDA allows dying patients access to experimental drugs, Rilutek already is available for free to more than 2,000 ALS patients in this country through a company-run lottery. (9) ===== Riluzole ========== PR NEWSWIRE Monday September 18, 1995 RILUTEK(R) (RILUZOLE) RECOMMENDED AS TREATMENT FOR LOU GEHRIG'S DISEASE BY ADVISORY COMMITTEE TO FDA Rilutek is First Drug To Treat Disease in 126 Years ROCKVILLE, Md., Sept. 18 /PRNewswire/ -- The Food and Drug Administration's (FDA) Peripheral & Central Nervous System Drugs Advisory Committee today voted to recommend the approval of Rilutek(R) (riluzole) for the treatment of amyotrophic lateral sclerosis (ALS) (also known as Lou Gehrig's disease), Rhone-Poulenc Rorer announced today. There is no currently approved treatment for ALS, a disease first described in 1869. The committee's recommendation, while not binding, will be considered by in its review of the New Drug Application (NDA) for Rilutek (riluzole) submitted by Rhone-Poulenc Rorer (RPR) on June 29, 1995. Rilutek was recommended for approval based on clinical trial data showing that the compound extended survival in people with ALS. "Rilutek is the first drug to demonstrate efficacy in the treatment of ALS," said Dr. Larry Powe, Director of Central Nervous System Research at RPR, in a presentation to the committee. "We have duplicated our results in two clinical trials utilizing survival as the primary endpoint," he added. The recommended dose is 100 mg daily (50 mg twice a day). A total of 959 patients participated in the multi-center, double-blind, placebo-controlled Phase III trial. Efficacy and safety parameters were compared among four randomized treatment groups (placebo, 50 mg, 100 mg and 200 mg of Rilutek). In addition to demonstrating a survival benefit, Rilutek was generally well-tolerated. Side effects reported in the trial included nausea, fatigue and elevated liver enzymes. This data, originally presented at the American Academy of Neurology meeting on May 10, 1995, confirmed the survival results of a Phase II Rilutek trial which appeared in the March 3, 1994, issue of The New England Journal of Medicine. Rilutek (riluzole) Early Access Program In addition to Rilutek clinical trial participants, the compound is being made available to ALS patients in the U.S. through the Rilutek (riluzole) Early Access Program. The Early Access Program has been established by RPR to enable a limited number of people with ALS to receive Rilutek free of charge pending FDA approval and commercial availability. The FDA allowed the Early Access Program treatment protocol to proceed on June 20, 1995. A total of 2,250 patients have been selected to receive Rilutek free-of-charge in the program. The FDA granted orphan drug status to riluzole on March 16, 1993. Orphan drug status is intended for use in rare diseases affecting less than 200,000 people. ALS is a fatal neurodegenerative disease affecting approximately 30,000 people in the United States. It attacks nerve cells in the brain and spinal cord, resulting in muscle paralysis and respiratory failure. Patients generally survive three to five years after diagnosis. Rhone-Poulenc Rorer (NYSE: RPR) is a global pharmaceutical company dedicated to improving human health. CONTACT: Bob Pearson, 610-454-3872, or Rob Partridge, 610-454-3890, both of Rhone-Poulenc Rorer/ === end of als 216 ===