Date: Wed, 28 Jun 95 01:40:15 -0400 From: Bob Broedel To: als@huey.met.fsu.edu Subject: ALSD200 ALS-ON-LINE =============================================================== == == == ----------- ALS Interest Group ----------- == == ALS Digest (#200, 27 June 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 == == 780+ 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 == =============================================================== CONTENTS OF THIS ISSUE: 1 .. ALS patients needed for a concurrent trial 2 .. re: Myotrophin enquiry (from Australia) 3 .. Pain Management in ALS 4 .. DeathNET wins Canadian Internet award 5 .. BDNF 6 .. breathing 7 .. Rilutek 8 .. re: ALERT/ALS/Rilutek 9 .. Epidemiological Survey 10 . Prodigy (1) ===== ALS patients needed for a concurrent trial ========== >From : Jay Meyer Subject: *New non-profit organization web site* Date : Wed, 21 Jun 1995 11:43:05 -0700 (PDT) ------------------------------------------------------------------------- We need ALS patients for a concurrent trial. Please check out the address below to find out how to register and more information. New features: o Online newsletter o Current drug trials o Trial Registration Form (completely anonymous if desired) o On-line Articles (ALS, MS, Alzheimer's & Parkinson's disease; coming soon) CENTER FOR NEUROLOGIC STUDY www : http://www2.connectnet.com/users/cns email: cns@connectnet.com -- Jay Meyer (2) ===== re: Myotrophin enquiry (from Australia) ========== Date : Mon, 19 Jun 95 15:36:01 -0400 >From : "Dr. Kenneth Fischbeck" To : paulf@dali.mel.dbe.csiro.au Cc : bro@huey.met.fsu.edu Subject: RE: Myotrophin enquiry (from Australia) In answer to your question about how to contact Cephalon, Inc. regarding Myotrophin clinical trials, Jason Rubin & Mary Fisher are the contact people listed on the recent press release, and Dr. Michael Murphy is senior vice president for clinical research. The telephone numbers for Cephalon, Inc. are 1-610-344-0200, fax 1-610-344-0065. Additional numbers for information about Myotrophin: 1-800-797-0705 in North America and 1-301-210-0393 elsewhere. K. Fischbeck, M.D. Univ. Pennsylvania, Philadelphia (3) ===== Pain Management in ALS ========== Date : Wed, 21 Jun 1995 01:54:22 -0500 >From : WSCUNNIN@INDYVAX.IUPUI.EDU Subject: Pain mgt in ALS Could someone please post info concerning pain management in ALS patients? My mother-in-law has the illness and has been taking ibuprofen 4 times per day. However, she is well past the point where it is effective as she is in almost constant pain. She is not on a feeding tube, but any meds should be either in liquid form or be available to administer rectally. It would be nice if we could find something that would help her fall asleep as well. Also, as of now, she is not having difficulty breathing. One last thing. Can someone give me a reference to a journal article or articles that provide decent info on patient mgt? In particular, it should cover such things as progression and the appropriate steps to take at each stage of the illness and also provide a good discussion of meds that are used. I have access to an excellent med lib, so any journal will work. Thanks for any assistance. Scott Cunningham wscunnin@indyvax.iupui.edu (4) ===== DeathNET wins Canadian Internet Award ========== Date : Thu, 22 Jun 1995 12:51:11 -0700 >From : jh@islandnet.com (John Hofsess) Subject: DeathNET wins Cdn Internet Award We are very pleased to announce that DeathNET (http://www.islandnet.com/~deathnet) has just been chosen "Best Health/Medical Site" at the 1995 Canadian Internet Awards. The announcement was made last night at the Net95 convention currently taking place in Ottawa, Ontario. The win is especially gratifying considering that most of the other nominees in the same category - Canadian Medical Association Online, Healthnet (the government's own WEB service) and University of McMaster's Medical School, to name a few - are well-heeled "establishment" sites with plenty of influence and power. We like to think that DeathNET is valued for its honesty in matters pertaining to illnesses such as ALS, and for championing the human rights of all individuals to have greater self-determination and freedom of choice. We may not be the voice of official medicine but we represent the "voice of the people" - around the world. john hofsess (5) ===== BDNF ========== UPI BUSINESS & FINANCIAL WIRE Monday June 26, 1995 AMGEN REPORTS PROGRESS ON ALS DRUG THOUSAND OAKS, Calif., June 26 (UPI) -- Biotech giant Amgen Inc. announced Monday it will move into Phase III human trials with partner Regeneron Pharmaceuticals Inc. for its drug for treating people with Lou Gehrig's disease. Amgen said its Phase I and II tests of genetically engineered BDNF treatment for amyotrophic lateral sclerosis (ALS) showed half the deterioration of breathing capacity in comparison to patients taking placebos in a sample of 283 people. Loss of breathing capacity is a major cause from ALS. It said Phase III testing, designed to be the final step to getting regulatory approval, would begin in the third quarter. ``The results of this trial with BDNF suggest that a drug can slow the loss of breathing capacity that is so devastating to patients with ALS,'' said Leonard S. Schleifer, president and chief executive officer of Regeneron. ALS affects the nerve cells which control muscle function, causing progressive weakness and, eventually, paralysis. In most cases, death comes within three to five years from the first appearance of symptoms. Amgen, of Thousand Oaks, Calif., and Regeneron, of Tarrytown, N.Y., said the drug did not affect the degeneration of muscles. It was the third positive development this year among companies trying to find an ALS treatment. Cephalon Inc. reported earlier this month that its Myotrophin drug slowed the loss of function of muscles and earlier this year Rhone-Poulenc Rorer Inc. announced its Rilutek drug increased the survival of patients for an average of three months. < rest of article deleted > (6) ===== breathing ========== Date: Sun, 18 Jun 95 22:26 PDT >From: Tony Oppenheimer Subject: personal request I am interested in staying in touch with and professional as well as lay (public) or patient support groups. My area of interest is ethical issues, decusion making, doctor-patient collaboration, and long-term mechanical ventilation as well as end-of- life care (and palliative treatment). I am a consultant in pulmonary medicine who has considerable experience with ALS and mechanical ventilation (both noninvasive and via tracheostomy). And I chair my medical center's bioethics committee. Edward Anthony Oppenheimer, M.D. Chief, Pulmonary Medicine ************************************************************************* * Edward Anthony Oppenheimer, M.D. * * Pulmonary & Critical Care Medicine * * Southern California Permanente Medical Group (213) 667-7106 (voice) * * 4950 Sunset Boulevard (213) 667-5725 FAX or * * Los Angeles, CA 90027-5822 USA (310) 275-3622 FAX * * Internet e-mail: eig3eao@MVS.OAC.UCLA.EDU * ************************************************************************* (7) ===== Rilutek ========== AP DATASTREAM WASHINGTON NEWS WIRE Thursday June 22, 1995 LOU GEHRIG'S, AIDS PATIENTS TO VIE FOR EXPERIMENTAL DRUGS By LAURAN NEERGAARD Associated Press Writer WASHINGTON (AP) -- Thousands of Americans with two incurable illnesses, Lou Gehrig's disease and AIDS, will vie for free doses of two experimental drugs in government-sanctioned lotteries this summer. Drug maker Rhone-Poulenc Rorer announced today that more than 1,000 Lou Gehrig's sufferers will get free Rilutek, the first drug ever shown to prolong survival in patients with the deadly muscular disease. There is no approved treatment for the 5,000 Americans diagnosed annually with Lou Gehrig's, also called amyotrophic lateral sclerosis or ALS. < parts deleted > Neither company has yet sought Food and Drug Administration approval to sell the drugs. But the FDA said the medicines appear promising enough to give patients outside strict clinical trials before testing is complete. The FDA routinely allows dying patients with no other hope to seek experimental drugs, but companies often are reluctant because they have minimal supplies -- and some have been accused of bias in picking who gets the drug. The ALS and AIDS drugs mark the nation's first lotteries for experimental drugs, the only way the FDA could ensure fair distribution when it knows ``demand exceeds supply,'' said agency spokesman Arthur Whitmore. ``If they had doses for a million people, it might be a completely different situation.'' ``Everybody gets a chance,'' agreed Abbey Meyers of the National Organization for Rare Disorders, which will run the Rilutek lottery. ``I got a promise from the president of Rhone-Poulenc that even if he gets ALS, he is not going to get this drug'' unless chosen at random by the lottery computer. < parts deleted > Rhone-Poulenc will file an FDA application for Rilutek next month, but anyone with ALS can immediately apply for the lottery. NORD will pick the initial 1,000 patients July 24, and several hundred a month thereafter until FDA finishes analyzing Rilutek. < parts deleted > To register for the Rilutek lottery, call 1-800-RX-TRIAL. (8) ===== re: ALERT // ALS // Rilutek ========== Date : Thu, 22 Jun 1995 22:24:10 EDT >From : NATG59A@prodigy.com (MR JIM PAUL) Subject: Re: ALERT // ALS // Rilutek I talked to R-P-R today. Anyone who has previously called them about Rilutek will automatically have their name submitted to NORD, there is no need to call again. The phone wait can be up to an hour, they informed me. Jim Paul (9) ===== Epidemiological Survey ========== Date : Sun, 25 Jun 1995 21:56:25 EDT >From : PDNS06A@prodigy.com ( D L BARBER) Subject: Epidemiological Survey I would like to ask all readers if they have in the past or currently are filling out an epidemiological survey specifically designed to help find the cause of ALS? With all this pomp and circumstance around the recent news on the ALS drug scene, I have forgotten to ask this question. Given the hundreds of years that scientists and Doctors have been working on cures for viruses and diseases, some have been found in the lab, and others in the field. Another path to a cure is an epidemiological study where participants answer a myriad of questions to in order that statisticians can find as much common ground between as many respondents as possible. Questions would cover virtually everything about human life, from diaper rash to bee stings, to types of food intake, to..... you get the idea. While all of us ALS sufferers are sitting and lying down waiting for someone to find a cure, we have heard about recent drug trials and the hopes they give us. What I would like to know more about, is that which is being done in the study of the life experiences of ALS patients, in the context of an epidemiological survey. Can anyone shed some light on this for me,,,,,,, or at least point me in the direction of someone who can?? (10) ===== Prodigy ========== TITLE : Life In Cyberspace: Breeding Hope for Disease Online BYLINE: By Jamie Talan DATE : 06/13/95 SOURCE: Newsday CALL THEM "Magic" and "Bullet." Call them "Zeus" and "Hera." They are some of the dozens of mice being bred with a sole purpose: to bypass a lengthy system of drug development and find a treatment for the fatal disease that killed baseball star Lou Gehrig. Their godmother is Prodigy. The mice were paid for by people who meet in cyberspace daily, people who are weakened by amyotrophic lateral sclerosis, or ALS, who have found no other way to survive than to do it themselves. The research, that is. They've been fighting tooth and nail to become part of the clinical research world, where a handful of drugs are going through the years-long testing process required for federal approval. They don't have the time to wait, they say. The disease, which destroys the body's motor neurons, kills on average in three to five years. So there's "Bob" and "Ray." And "Riluzole" and "Kessler" - the first named for a promising experimental drug that adds a few months to an ALS patient's life; and the second for Dr. David Kessler, the director of the Food and Drug Administration. Kessler has become a symbol of the federal insistence on "blind" tests for ALS drugs. The policy requires that some patients picked at random - even dying ones - take a placebo. In other words, no one in a study knows whether they are getting the real thing. The Prodigy study will not be conducted on humans - yet. Thus, no one has to grapple with the real need to measure a drug response against a placebo. The mice, of course, will be randomized, as in any serious drug trial. So far, there are 106 mice. The Prodigy patients - and their loved ones - responded immediately last December to a note from biochemist and molecular biologist Leslie Johnson. "In the past few months, a lot of exciting things have occurred," she wrote. "The availability of a mouse model for ALS {with a defective SOD1 gene} opens many doors for the rapid examination of potential therapies . . . We can ask the questions . . . We can be the engine . . . We can do the research . . ." Johnson knows the bulletin board well. Her friend Peggy Young, before dying of ALS, spent hours every day fishing the network for any hints of hope. On Prodigy, patients share the details of their triumphs and losses, the drug trials, the feeding tubes, the hard choices many must make about going on a respirator. For Peggy's 59th birthday, which she missed by four months, Johnson wanted some answers. She had been reading about an experimental drug used to boost the body's ability to fight free radicals, scavenger molecules that attack and destroy cells in the body. The drug - alpha-phenyl-tert-butyl nitrone, or simply PBN - is being tested in animals as a weapon against aging and Parkinson's disease. It's a long shot, but Johnson hopes the drug can also stave off the ALS damage to the motor neurons. And she plans to test it using another new discovery. Scientists have recently found a defective gene in families prone to ALS - SOD1, a gene for an enzyme that normally cleans up free radical damage. Now, mice are being bred with this genetic defect; they develop the classic symptoms of muscle-wasting. She offered Prodigy patients a deal: Pay $50 for a mouse, name it and own a piece of the research. She will carry out the mouse study and, even if PBN doesn't work, Johnson will be looking for other unique chemicals that might. The checks and the names, serious and silly, have been coming in. Johnson, who runs a clinical testing and research lab in Ridgewood, N.J., has made an agreement with Jackson Laboratories in Bar Harbor, Maine, to be the first recipient of a batch of ALS mice from the breeding lab. She suspects testing could begin sometime this summer. Her theory about PBN has been published not in scientific medical journals but on the Prodigy network. "No big company is interested in something that won't make money. PBN is a simple synthetic compound. These are easy experiments to do. Either it will slow the symptoms or it won't." Corrine Ostrander will own "The Queen of All Meese." Ostrander, who lives upstate, spends much of her day logging on to the network. The disease has left the former nurse bedridden, unable to move. Last week, Johnson received money for another 13 mice from Kingston High School's Class of 1961, Ostrander's classmates. Janis Dorfman is 48 and developed ALS about 18 months ago. She has been logging on daily from her Syosset home since March, 1994, when she had only a slight inability to work her right arm. Unlike many of her bulletin board comrades who are in wheelchairs, she is able to move about, though rather slowly these days, having lost her ability to balance. Since February, she has fallen 42 times. She waits for a drug treatment to come along. The mouse she paid for was named "Roz," after a sister's friend, who died of ALS long before Dorfman became ill. "Research was just going too slowly," said Jack Norton, manager of the ALS bulletin board on Prodigy. Norton knows everything that goes on in ALS cyberspace, having communicated with 400 people who have logged on. His mouse is "Kessler." "I volunteered him for the placebo group so that he could give his life for future generations," Norton said. "We can do the research," said Johnson, eager to get the first shipment of mice. Johnson owns a blood testing company. She has done postdoctoral work on gene mapping at Columbia University and spent time studying drug mechanisms in the control of leukemia at Sloan Kettering. What if PBN works? "We will contact the FDA and begin to do human studies," said Johnson, who is hoping to get some findings by Oct. 1, the FDA deadline for funding orphan drug research. "A layman came up with Lorenzo's Oil. I have the hope that we can do something along those lines." While Johnson waits for the mice to arrive, several patients who made Prodigy part of their universe have died. Their legacy remains in small white mice who may save someone else's life. ===== Life in Cyberspace runs every other week in the Health & Discovery section. If you'd like to share your experiences in cyberspace, or have a theme you'd like to explore, please mail your idea to Health & Discovery Section, Newsday, 235 Pinelawn Rd., Melville, N.Y. 11747-4250, Att: Liz Bass. ==== = For information about how to join the Prodigy on-line ALS bulletin = board system, call Prodigy Services Co. at 800-776-3449 / 914-448-8000 ==== === end of als 200 ===