Date: Thu, 20 Apr 95 04:27:08 -0400 From: Bob Broedel To: als@huey.met.fsu.edu Subject: ALSD187 ALS-ON-LINE =============================================================== == == == ----------- ALS Interest Group ----------- == == ALS Digest (#187, 20 April 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 == == 650+ 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 .. Editorial 1 .. Riluzole/Rilutek availability 2 .. AMGEN ANNUAL REPORT (corrected) 3 .. ALS antioxidant? 4 .. question on Parkinson's 5 .. Availability of experimental drugs 6 .. enquiries 7 .. ALS research 8 .. Myotrophin (0) ===== Editorial ========== For the next few days I will be in St. Louis at an ALS conference. I expect to be on-line while there but serious work will need to wait till I return to Tallahassee on Monday. As always, I thank everyone of you for joining and helping with our ALS e-mail network. rgds,bro (1) ===== Riluzole/Rilutek availability ========== Date : Tue, 11 Apr 1995 19:10:23 -0500 (CDT) >From : HEINET@wartburg.edu Subject: Riluzole/Rilutek availability I don't think it's been made entirely clear in press releases and discussions of Riluzole/Rilutek, but anyone who wishes to be considered for "expanded access" to the drug (that means to receive it) while it is undergoing regulative review (FDA aproval) MUST call Rhone-Poulenc Rorer at 800-798-7425 to get on RPR's list. Ted Heine, ALS patient in Waverly, Iowa (2) ===== AMGEN ANNUAL REPORT (corrected) ========== Date : Tue, 11 Apr 1995 12:11:52 EDT >From : UKRY13A@prodigy.com (MR JOSEPH L SNYDER) Subject: AMGEN ANNUAL REPORT BOB-COULD YOU PLEASE RERUN THIS AMGEN NOTE, THE FIRST SUBMISSION I FORGOT TO PUT 'ALS HUMAN TRIALS' THANKS JOE ...ALSO IF ANYONE WANTS TO ATTEND AMGEN MTG. SEND ME EMAIL. Amgen's annual report released last week has the following quote on page 20 "Synergen's pipeline includes glial derived neurotrophic factor(GDNF) which is in preclinical development for Parkinson's disease and is expected to enter human clinical trials this year". Several highly respected research journals have indicated in the past year that GDNF SHOWS GREAT PROMISE AS ALS TREATMENT. Also note Amgen purchased Synergen in Dec 1994. WE NEED TO LET AMGEN KNOW THAT GDNF ALS HUMAN TRIALS SHOULD BEGIN IN 1995 ALSO. Write Gordon Binder CEO OF AMGEN at 1840 DeHavilland Drive, Thousands Oaks, California 91320-1789, phone #805-447-1000. Better yet attend the AMGEN annual stockholders meeting on Tuesday, MAY 9TH,1995 at 10:30 a.m. PDT at the Regent Beverly Wilshire, 9500 Wilshire Boulevard, Los Angeles,California. I assume that animal toxicity studies of GDNF must be going well for AMGEN to expect human trials in 1995. Comments ? Joe Snyder Louisville Ky (3) ===== ALS antioxidant? ========== >From : bernie@rbdc.rbdc.com Date : Fri, 14 Apr 1995 23:07:58 +0000 Subject: ALS antioxidant ? Does anybody have any information about an antioxidant named pycnogenol relating to the treatment of ALS? please email me if you do. (4) ===== question on Parkinson's ========== Date : Mon, 10 Apr 1995 12:17:21 -0400 >From : BDominion@aol.com Subject: ?'s on Parkinson's I am trying to look into a new drug being tested on Parkinson's disease. It is called "Ropinirole" and I believe it is under the development of SmithKline. If you know of a Parkinson's newsgroup like this one for ALS, please let me know, I would greatly appreciate this information. Thank you, Barbara Estep (5) ===== Availability of experimental drugs ========== >From : sallitt@netcom.com (Dan Sallitt) Subject: Availability of experimental drugs Date : Wed, 19 Apr 1995 15:50:18 -0400 Does anyone know if the drugs riluzole and myotrophin are available in any country? A recent posting here about riluzole made it seem as if there wasn't a lot of it around anywhere, but I don't know anything about the availability of myotrophin. Is anyone taking these drugs other than the people in the experimental trials? Thanks. - Dan Sallitt (sallitt@netcom.com) (6) ===== enquiries ========== Date : Sat, 15 Apr 1995 20:52:25 -0700 >From : vjhuber@halcyon.com (Virginia Huber) Subject: enquiries for als digest I am a counselor who facilitates a support group in the Southend of Seattle for the Muscular Dystrophy Assn. Participants are ALS patients, caregivers and friends. Two questions have recently been raised: One about women with ALS. Does anyone have information about women's experiences with ALS that I could share with members? The second question is about vitamin therapies. The person interested is a man in his 70's who is interested in supplementing his medical treatment with vitamins. Thanks, Virginia Huber (7) ===== ALS Research ========== Date : Thu, 13 Apr 95 3:42:04 EDT >From : Cathleen Bowley Subject: ALS Research I am a graduate student at Rutgers University. I am in the process of doing extensive research on ALS and I came across your address. I read that you host a list for ALS patients, support/discussion groups, clinics and researchers. I am writing to ask for some help with my research. Please do not go to too much trouble, but if you have lists of clinics, researchers, associations, or a contact of some one or some group that may be able to supply info for further research (latest findings, who is working on what...) I would greatly appreciate it. Thank you so much for your help. Cathleen Bowley (8) ===== Myotrophin ========== WALL STREET JOURNAL 04/19/95 Moment of Truth: Biotech Firm's Hopes Ride on the Results Of First Drug's Trial --- Payday or a Problem Awaits As Cephalon Tests Drug For Lou Gehrig's Disease --- The Secrets of `the Crypt' ---- By Elyse Tanouye, Staff Reporter of The Wall Street Journal WEST CHESTER, Pa. -- Within weeks, a group of statisticians will emerge from a soundproof, windowless room at the heart of a young biotechnology company here. The news they deliver may bring the company fabulous success -- or miserable disappointment with damaging financial repercussions. Cephalon Inc., like other biotechnology concerns before it, is living through its most stressful, nerve-racking time: the period just before the company finds out if its first drug will work. Cephalon's researchers are feverishly analyzing the results of a large human trial of its drug, Myotrophin, being developed as possibly the first treatment against amyotrophic lateral sclerosis, or ALS, a deadly nerve disorder known better as Lou Gehrig's disease. < parts deleted > But Myotrophin's chances of working against ALS are between 10% to 20%, guesses Joseph "Skip" Klein, a health-care analyst at T. Rowe Price Associates in Baltimore. < parts deleted > The company, whose initial research was done in an employee's master bathroom, began looking for new drugs by studying small pieces of neurotrophic factors, human proteins that help motor neurons grow. Motor neurons are large nerve cells that carry the brain's messages to muscles. When they die, as they do for unknown reasons in ALS, the brain can't communicate with muscles. Weakness and ultimately paralysis follow. Cephalon discovered that a natural nerve growth factor, which it dubbed Myotrophin, kept damaged neurons alive in test tubes longer than usual, and sometimes, to researchers' astonishment, it prevented cell death altogether. Researchers theorized that the factor might help ALS sufferers. In other central-nervous-system diseases, drugs like Myotrophin can't reach important sites in the brain and spinal cord, which are protected by a natural barrier that keeps out proteins. But the neurons affected by ALS reach out past the barrier to connect to muscles. Cephalon researchers thought Myotrophin, injected into the bloodstream, could travel to and protect neurons affected by the disease. In animals, it consistently worked. But because the animal disorders differed significantly, the only way to find out whether Myotrophin was effective in humans was to administer it to ALS patients. In 1992, Cephalon began giving it to healthy people and to ALS patients to test its safety. Usually, drug companies next do a study or two in a small number of patients to get a preliminary indication of the drug's effect on the disease. But Cephalon decided instead to launch two large, fullscale trials -- in 266 patients in the U.S. and 183 in Europe -- to determine definitively whether the drug worked. Many biotechnology companies have failed by following the same strategy: tackling diseases about which little is known, using animal tests that aren't reliable and rushing through early testing. Mr. Klein of T. Rowe Price, which owns Cephalon shares, says those weren't mistakes but calculated, high-risk business decisions that are necessary in innovative research. Cephalon decided that ALS progresses too slowly to draw any meaningful conclusions from a short, small study, Dr. Baldino says. < parts deleted > Cephalon, investors, and patients will soon find out if Myotrophin works. A few weeks ago, Cephalon closed the U.S. Myotrophin study and began data analysis. Statisticians Stephen Hardiman and Kathryn Miller entered "the crypt," a newly built 16-by-17-foot room with an alarm, soundproofing, no windows and a special ceiling to prevent break-ins from above. The statisticians, forbidden to discuss their work with anyone except Drs. Baldino and Murphy, will be racing to complete in a few weeks an analysis that normally takes four to six months at a larger drug company. "These measures wouldn't be necessary at a big pharmaceutical company, but for this clinical trial at this point in our company's history, we need to take these precautions," says Barbara Schilberg, general counsel. Dr. Baldino argues vigorously that Myotrophin's failure wouldn't break Cephalon. It has more than $100 million in cash, which can sustain it for two years. In that time, it hopes to finish testing and gain approval for another drug, for the sleeping disorder narcolepsy. Revenue also is beginning to flow from an arrangement with Bristol-Myers Squibb Co. to sell a painkiller to neurologists. And the research labs have turned out a host of other compounds that Dr. Baldino is itching to move into human testing. Among them is a one that mimics the effects of drugs like Myotrophin but is smaller and more likely to reach key areas in the central nervous system. And Myotrophin might be useful against other ills, such as numbness in hands and feet caused by cancer treatments or diabetes. "At each step of the way, we diversified and balanced our risk," Dr. Baldino says. < parts deleted > But there are lots of places to find money, he adds. Since 1989, Cephalon has struck partnership deals with large pharmaceutical companies, such as SmithKline Beecham PLC and Schering-Plough that could eventually produce more than $120 million in research funding. Indeed, Chiron Corp., another biotechnology company, paid for much of the Myotrophin development costs in return for half the profits, if there are any. < parts deleted > === end of als 187 ===