Date: Mon, 15 May 95 20:03:17 -0400 From: Bob Broedel To: als@huey.met.fsu.edu Subject: ALSD193 ALS-ON-LINE =============================================================== == == == ----------- ALS Interest Group ----------- == == ALS Digest (#193, 15 May 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 == == 700+ 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: 0 .. ALS Digest back issues sampler diskette 1 .. Impressions of Neuro Conf in Seattle 2 .. Press Release from Rhone-Poulenc Rorer re Riluzole 3 .. Megasystems? 4 .. Cutting edge treatments 5 .. BDNF 6 ..DynaVox 7 .. re: shareholder meeting 8 .. annual meeting / Amgen 9 .. Human Molecular Genetics (0) ===== ALS Digest back issues sampler diskette ========== Because of a promo I am mailing to international ALS-related groups, I have created a MSDOS, 3 inch, 1.44 MEG diskette with the most recent back issues of ALS Digest on it. If any of you want a copy of this diskette mailed to you, please send me your complete mailing address. There is no charge for this. International requests are welcome. The diskette will be mailed via airmail. A few weeks from now I will be able to make the same offer for a MAC- formatted diskette ... but for now, all I have are MSDOS versions. rgds,bro (1) ===== Impressions of Neuro Conf in Seattle ========== Date : Sat, 13 May 1995 00:43:13 -0400 >From : JACKN74940@aol.com Subject: Re: ALSD191 ALS-ON-LINE Impressions Of Neurological Conference in Seattle May 7-11; - I Had the opportunity to attend this scholarly conference and was impressed by the number of sessions covering ALS. I was not in attendance when the session on clinical trials directions and Design was discussed, however I did hear that the Word Prodigy was mentioned several times! That questions on how this could be stopped, Prodigy, sharing of information, were raised. In response it was mentioned that it will never be stopped - I think they are seeing a change in paradigms and sharing of information will be the standard not to be controlled! The pressure to respond to life and death information will not be shuffled to a back burner "Peer review". Prodigy and Internet will never allow that to happen again. If information is hidden or participation requirements in trials forbidding information sharing on public bulletin boards, then We Will Use Private Email! Many neurologists were excited by the RPR announcement. Then there were others! These others are on the wrong end of the paradigm. They believe in Protocol. They believe in "Peer review". They believe in withholding information until all the t's are crossed and all the i's are dotted. They Believe that if it takes six months for a peer review, so be it! They believe that news to patients should come from them and not be reported in the New York Times. They do not want the pressure to commit to new ideas and espoused the Not Invented Here (NIH) Theory. I believe there are many intelligent neurologists and statistians working for the drug companies. I believe they should be only accountable to the FDA. I believe that they own the trial information and can release what ever they wish to whoever they wish whenever they wish. Just like the medical device industry moving their trials overseas, we may see drug trials moving overseas. If the associations are unhappy about how information is released, remember whose information it is. Be careful that biting the hand that feeds you may be hazardous to our health. In fact there is a trend beginning that will allow private labs to conduct trials within the United States, excluding universities and reducing costs. The last thing I recall was a rhetorical question from a key neurologist: now that this early announcement has occurred with RPR what will we do about Cephalon announcement in June? My thought about his concern was - you confuse me with someone who cares about that concern. It is Cephalon' s data. It is between Cephalon and the FDA. If they choose to share the data that is wonderful but not required. Soon the old guard will be consumed by consumerism; and innovation, speed and commitment by the early adapters (including neurologists) of the information age. Jack Norton - the Quest continues! (2) ===== Press Release from Rhone-Poulenc Rorer re Riluzole ========== RHONE-POULENC RORER: RILUTEK(TM) (RILUZOLE) PROLONGS SURVIVAL OF PATIENTS IN LARGEST TRIAL EVER CONDUCTED IN ALS Prolonged Survival Observed in Limb and Bulbar Onset ALS SEATTLE, May 10 /PRNewswire/ -- Results of the largest ever Phase III trial conducted in amyotrophic lateral sclerosis (ALS) demonstrate that Rilutek(TM) (riluzole) is the first compound to prolong survival since the disease was first described in 1869, according to data presented today by investigators from France and the United States at the 47th Annual Meeting of the American Academy of Neurology. The data were presented by Professor Vincent Meininger, of Hopital de la Pitie-Salpetriere in Paris, France, at a session on ALS Clinical Trials chaired by Dr. Robert Miller, of the California Pacific Medical Center in San Francisco. Drs. Theodore Munsat, of Tufts-New England Medical Center in Boston and Larry Powe of Rhone-Poulenc Rorer (NYSE: RPR) also participated in the discussion. The primary statistical test of Rilutek's efficacy was an 18-month intent to treat analysis. The evaluation showed a 12.7 percent increase in the number of patients alive after eighteen months who received a daily 100 mg dose (50 mg tablet twice per day) of Rilutek as compared to placebo. A total of 56.8 percent of patients (134 of 236) receiving Rilutek were alive after eighteen months compared to 50.4 percent of patients (122 of 242) who received placebo. An intent to treat analysis includes all patients who entered the trial to avoid any bias in the statistical evaluation of the results. A 12-month analysis utilized a Kaplan-Meier one-year survival estimate. This evaluation demonstrated a 17.4 percent increase in the number of patients alive after one year who received a daily 100 mg dose of Rilutek as compared to placebo. A total of 73.7 percent of patients (174 of 236) receiving Rilutek (100 mg per day) were alive after one year compared to 62.8 percent of patients (152 of 242) who received placebo. "These results represent the first step in the treatment of ALS," said Manfred Karobath, MD, Senior Vice President and President of R&D at RPR. "We believe this trial s significant for at least three reasons. First, we used survival as a primary endpoint, since it is a definitive measure of efficacy. Second, we demonstrated a statistically significant increase in survival in both bulbar and limb onset ALS. Finally, we confirmed the findings of our previous Phase II clinical trial, which will enable us to file a registration dossier with health authorities beginning in July, 1995." The Phase III clinical trial was a multinational, double-blind, placebo-controlled study conducted at 31 investigator sites in Europe and North America. This study, involving 959 patients, was conducted in collaboration with a Steering Committee and an independent Data Safety Monitoring Board. Enrollment began in December, 1992 and the study was completed in December, 1994. The U.S. investigators participating in the trial were Dr. Yadollah Harati of Baylor College of Medicine and Veterans Affairs Medical Center (Houston), Dr. Jeffrey Rothstein of Johns Hopkins University (Baltimore), Dr. Robert Sufit of Northwestern University (Chicago), Dr. Robert G. Miller of California Pacific Medical Center (San Francisco) and Dr. Theodore L. Munsat of Tufts-New England Medical Center (Boston). All U.S. centers are certified by the Muscular Dystrophy Association. Efficacy and safety parameters were compared among four randomized groups. Three different doses of Rilutek were studied, a 50 mg per day, 100 mg per day, and 200 mg dose per day versus placebo. A total of 75 percent of patients received Rilutek. The treatment duration was up to 18 months, after which all patients were given the opportunity to receive Rilutek 100 mg/day on an open-label basis. Eligible patients were between 18-75 years of age and had a probable or definite ALS diagnosis within the last five years. For the 18 month intent to treat analysis, the increase in survival was statistically validated by stratified Logrank and stratified Wilcoxon tests. The stratified Logrank test between the 100 mg Rilutek and placebo groups, resulted in a two-sided p value of p0.076. The stratified Wilcoxon test is also significant (p0.05). When all three doses were combined, Rilutek continued to differ significantly from placebo on both stratified Logrank (p0.048) and Wilcoxon tests (p0.038). In a separate analysis of a surrogate endpoint, Rilutek did not exhibit a statistically significant effect on the rate of muscle function deterioration for the overall ALS population. Rilutek was generally well-tolerated. Side effects reported in the trial included nausea, asthenia (fatigue) and elevated liver enzymes. Since there is no approved treatment for ALS, RPR has begun discussions with local regulatory authorities to initiate locally approved programs that would allow broader access to Rilutek before marketing approvals are potentially granted by regulatory authorities. Worldwide, approximately 70,000 people have ALS, which is also known as Lou Gehrig's disease, maladie de Charcot, or Motor Neuron Disease. Dr. Charcot is credited for the clinical description of ALS in 1869. He was the first physician to link symptoms of ALS to a group of nerves specifically affected by the disease. Generally, patients survive three to five years after diagnosis. Death usually occurs due to respiratory failure. For more information, patients, caregivers or health care providers can call 1-800 RX TRIAL. Rhone-Poulenc Rorer is a global pharmaceutical company dedicated to improving human health. CONTACT: Bob Pearson, 610-454-3872, or Guy Esnouf, 610-454-5048, both of Rhone-Poulenc Rorer. (3) ===== Megasystems? ========== >From : bernie@rbdc.rbdc.com Date : Tue, 9 May 1995 08:46:52 +0000 Subject: ALS-ON-LINE Does anybody have any information about an Alex Durante and a book "Megasystems"? He advocates alternative healing methods. Please email me at bernie@rbdc.rbdc.com Thanks (4) ===== Cutting edge treatments ========== Date : Wed, 10 May 95 13:10:29 -0800 Subject: Cutting edge treatments >From : cschind@mindlink.bc.ca (Chris Schindelhauer) I am a new subscriber to this group, and have a very good friend with ALS. Could anyone give me any information on new treatments for ALS? Any info appreciated. Thanks. (5) ===== BDNF ========== Date : Thu, 11 May 1995 22:36:21 -0400 (EDT) >From : WECK0820@duq3.cc.duq.edu Subject: Re: ALSD191 ALS-ON-LINE Are there any ALS patients out there that have gone through a BDNF drug trial and found that the drug actually improved their condition? If so, what kind of improvements occurred? I would appreciate any responses to my question. They may be sent to e-mail address: Weck0820@duq3.cc.duq.edu Thank you, Brian M. Weck (6) ===== DynaVox ========== Date : Thu, 11 May 1995 20:19:59 -0400 Sender : CNET - Speech Language Diagnostics Dilemmas : >From : "Janice L. Singer-Wagner" Subject: DynaVox A colleague of mine is working with a cognitively intact ALS patient with an aug com system, specifically, the DynaVox. The problem being experienced by this patient is that of fatigue since the scanning process involves three steps to reach a word/concept. Does anyone know of a more efficient and expeditious method of scanning to alleviate the fatigue factor? Any resources or readings that we can refer to re: this? Janice Singer-Wagner Date : Fri, 12 May 1995 08:04:00 CST Sender : CNET - Speech Language Diagnostics Dilemmas : >From : DROBIN@WJSHC-PO.SHC.UIOWA.EDU Subject: Re[2]: DynaVox For an excellent system that we have used with many persons with ALS contact Richard Hurtig at richard-hurtig@uiowa.edu DAR (7) ===== re: shareholder meeting ========== Date : Thu, 11 May 1995 13:13:29 -0700 (PDT) >From : Leigh Redding Subject: Re: shareholder mtg. statement & ALSD191 ALS-ON-LINE Joe Snyder, ... > SHAREHOLDER MEETING. AMGEN HAS NOW DECIDED TO TEST GDNF ON ALS IN HUMANS > THIS YEAR. THIS IS A BIG WIN FOR ALL OF US WHO HAVE BEEN PUSHING AMGEN FOR ... Go Joe, way to go. I wish I had Amgen stock so I could put my oar in the way you did! I too am a PALS, I was diagnosed in 7/93. I am a 55 year old male with sporadic ALS, Bulbar onset. In 4/94 I ended up with a trach and on a vent for 70 - 80% of the time. What little voice I had left was really finished with the trach installation. This spring I got a synthasizer from the words+ folk in California. It sure has brought back the joy of being able to "talk" again. We, my wife Shawn is my primary caregiver, are new grandparents, one in 5/94 & twins in 7/94. I would like to think that I could have a chance to watch them grow. People like us with a death sentence look at life differently than those who get bad news on a life threatening disease. One offers so much more hope than the other when you look at it from our, a PALS' eyes! Enough of this self serving pap, you know what I'm talking about. What I would like to ask you is what can we do to help you to move this process along. We have a small close support group made up of family and friends. They can help by writing and calling whoever can help us with the FDA, Amgen, our Congressmen whoever can help? That is a question. Joe, thank you for your time. We as others in the ALS community need to say another thank you to you for the efforts you already have made. Thank you so very much! Regards Leigh & Shawn Redding (8) ===== annual meeting / Amgen ========== CREDIT: Los Angeles Daily News DATE : 05/11/95 SOURCE: The San Francisco Chronicle Victims of Lou Gehrig's disease pleaded Tuesday with Amgen Inc., the Thousand Oaks (Ventura County) biotechnology company, to speed up testing on possible cures for the deadly ailment. Through letters and a presentation Tuesday at Amgen's annual meeting, the company was asked by victims of the disease, formally known as amyotrophic lateral sclerosis or ALS, to proceed with testing on a drug that combines two agents, which have been effective on laboratory mice. "I don't want to be on a respirator, totally paralyzed and have them say, `Oh yes, here it is,' " said Don Altier, 35, an ALS patient who spoke to Amgen executives from a wheelchair Tuesday. Altier also read a letter from an ALS patient and Amgen shareholder who now is unable to speak. < parts deleted > If it turns out that the BDNF proves to be more effective, the company will push for further testing and approval of that drug, said N. Kirby Alton, vice president of development. Amgen is looking at a third agent, gleail-derived neurotropic factor or GDNF, as another possible cure, he said. "The better hope is GDNF or BDNF, not a combination," he said. (9) ===== Human Molecular Genetics ========== Date : Sun, 30 Apr 1995 12:47:09 -0400 Sender : Human Molecular Genetics >From : "Frank S. Zollmann" Subject: Introduction of WWW for HUM-MOLGENeticists PREVIEW OF THE HUM-MOLGEN WWW SITE To improve the quality of HUM-MOLGEN communication, we are very happy to introduce to you: the HUM-MOLGEN WWW site! HUM-MOLGEN now combines list- communication with a brand-new, exclusive WWW-site at the address http://www.informatik.uni-rostock.de/HUM-MOLGEN/ (via NETSCAPE; capitals in the adress are important) The conventional E-mail service and the WWW-site are set up as one interactive unit: The E-mail service will further focus on condensed, summarized active information and communication of your key-interest. The WWW-site will focus on much more extensive passive information and communication, which can also be selected by choice. In general, subscribers to HUM-MOLGEN will have the advantage when it comes to pre-information, new features, etc. Both list- and WWW communication on HUM-MOLGEN are equally divided in TOPICS, such as you are used to (NEWS, CALLs, ANNOuncements, COMPuters science in genetics and Internet highlights, molecular DIAGnostics/clinical research, LITErature,BIOTechnology/molecular biology,ETHIcal/social) Communication on HUM-MOLGEN remains entirely FREE and very FAST. Subscribers can easily ANNOunce meetings, events or positions, CALL for collaboration, and look for, send or receive all sorts of summarized information of HUM-MOLGEN interest. From the LITErature, previews of journals, articles of hard-copies can be sent or received on HUM-MOLGEN. COMP specializes in communication in Internet highlights and computational genetics. The DIAG section is especially suited for communication between clinicians and biologists. BIOT will a.o. provide communication about new products and information about new techniques as well as commercialisation of Human Genome research. The NEW HUM-MOLGEN WWW site is: -A Medical, Clinical Genetics and DNA diagnostic Service on-line. -a one point entry, and easy acces for clinicians and molecular biologists to Internet highlights in the field of Genetics -An Interactive forum for collaboration and digital meeting place for clinicians, molecular biologists and geneticist, as well as other individuals interested in the field. -a host for Literature and preview information -a host for Biotechnology and molecular biology compagnies, publishers, DNA diagnostic laboratories, hospitals and clinical genetic centres -a host for new products, techniques and developments; -provide technical information, protocols and methods-on-line -provide grant information. -etc. The WWW-site is still under construction; new features and new information will be added in the next few weeks. We are looking for your critical comments and support! Frank S. Zollmann Arthur Bergen Zollmann.1@osu.edu Bergen@amc.uva.nl === end of als 193 ===