Date: Mon, 13 Mar 95 01:47:58 -0500 From: Bob Broedel To: als@huey.met.fsu.edu Subject: ALSD#179 ALS-ON-LINE =============================================================== == == == ----------- ALS Interest Group ----------- == == ALS Digest (#179, 12 March 1995) == == == == ------ Amyotrophic Lateral Sclerosis (ALS) == == ------ Motor Neurone Disease (MND) == == ------ Lou Gehrig's disease == == ------ Charcot's Disease == == == == 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 == == 550+ 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 .. Announcements 2 .. ALS Digest on Floppy Disks 3 .. als@huey 4 .. Ask the Experts on April 30 5 .. re: stiffness 6 .. re: Managing Swallowing Problems 7 .. some questions 8 .. powerlessness/power/control 9 .. Riluzole 10 . CNTF & BDNF & Neurontin & more. 11 . BDNF and GDNF 12 . Patients suffer while FDA dithers 13 . Battelle looks at the future (1) ===== Announcements ========== If you don't see your note in this issue, it should be in the next one. The next issue will be here soon. Maybe we will need to start publishing two times per week. But we need to keep each issue short so it will make it to all destinations. The new e-mail group has been set up. It will be called "als-talk". It will be open to all, it will be unmoderated, free-of-charge, etc. Though it is now in operation, it still needs some work before going public. Full details about how to join, how to use, etc. will be in the next issue. rgds,bro (2) ===== ALS Digest on Floppy Disks ========== Date : Sun, 12 Mar 1995 01:25:13 -0500 (EST) >From : WECK0820@duq3.cc.duq.edu Subject: ALS Digests on Floppy Disks Bob: I joined the ALS Digest and I have found it very informative. My mother, who was diagnosed with possible ALS, is doing fine so far, except for that she gets depressed often. Anyway, I downloaded every issue of the digest from Brunel, although it took me a very long time. I then proceeded to download the digest files from the mainframe at my college to my home, which also took a long time. I was wondering if anyone is sending these old issues to other people by way of floppy disk. Some people do not have internet access, but they could still get all of these informative issues on a floppy and pull them up as ASCII in their word processor and then print them out. Also, people who, for some reason or another, do not feel like spending time downloading all of these. For convenience purposes, I am willing to send a floppy disk, through the mail, full of these digests to anyone that wants them (I would only ask for the postage and the cost of the floppy to be paid for by whoever is interested). I am fairly new to the Internet and the digest and there probably is someone already doing this, or something close to it, but if you think anyone would be interested just tell them to send me a message: (weck0820@duq3.cc.duq.edu) Brian Weck ===== = Brian, I have been sending diskettes with back issues to various people = (mostly to international contacts) but I haven't talked about it much = because I was not sure I would be able to find the time to actually = service such requests. My biggest priority is to produce new issues of = the Digest, with everything else taking second priority. Grant Nicholas = has been helping a lot by sending diskettes, by creating indexes of back = issues, etc. His address is: gnicholas@lanl.gov = If I ever get caught up with day-to-day production activity, I will create = the ALS-ON-LINE-FAQ document (where FAQ means requently sked = uestions). The FAQ will tell all interested folk how to get diskettes = with back issues from people like you & Grant. It will also tell people = about other ALS on-line activity, etc. Probably I will not be able to = do the FAQ until after the ALS conference in St. Louis (late April). = But, yes Brian, I know that some people would prefer to get the back = issues on diskette. They will be very happy to see your offer. Thank you = for doing this. rgds,bro ===== (3) ===== als@huey ========== Date : 11 Mar 95 23:40:58 EST >From : wayne <70303.173@compuserve.com> Subject: using als@huey Bob, Could you post a note in the digest asking people not to send messages to als@huey? I'm sure I'm not the only one paying twice to read or download the same message, once from als@huey and once in the digest. Maybe you could put a reminder in the digest header. Thanks again for your service with this excellent resource. Wayne ===== = Dear Wayne, = Now that people can retrieve back issues they can read that at one = time sending to als@huey was the "preferred" way of doing things. = Some time ago we started losing international subscribers and important = ALS researchers because there was too much traffic to sort through. = Because of that loss, the "preferred" way became to send to bro@huey, = and to make it a weekly broadcast. = New people are signing up every day and because I never got around to = writing rules, to writing the FAQ, etc. some people don't know. Most = new subscribers don't even get a proper "Welcome" message from me. = Hopefully I will get caught up soon and will be able to make this a = better network. We don't have any bad people here. At this point in = time most of our problems are because of poor administration on my = part. In the near future the ALS Digest will be run from an entirely = different piece of software. It will still look the same though. Also, = I am committed to keep the ALS Digest going for at least the next four = years, or until someone comes up with something better. Until we get = set up on the new software, I will deactivate the als@huey feature. = I think this will be a solution to the problem. rgds,bro ===== (4) ===== Ask the Experts on April 30 ========== Date : Wed, 8 Mar 1995 15:02:25 -0500 >From : BillB67399@aol.com Subject: Ask the Experts on April 30 >From : Debra Bulkeley, Mass. Chapter, ALSA A distinguished panel of ALS experts will discuss the latest in ALS research on Sunday, April 30, from 1 to 4 p.m. at the Cecil B. Day Laboratory for Neuromuscular Research, Massachusetts General Hospital East, Charlestown, Mass. Sponsored by the Massachusetts Chapter of the ALS Association, the conference is free to all patients, families, caregivers, health care professional, and anyone interested in ALS research. The panel members are: Dr. Robert H. Brown Jr. of Mass. General Hospital, Boston Dr. Jeremy Shefner of Brigham & Women's Hospital, Boston Dr. Theordore Munsat of New England Medical Center, Boston Dr. Richard Gan of Boston University Medical Center Among the topics panelists will discuss include current clinical trials as well as drugs under development for treating ALS. A major portion of the afternoon will be a Question and Answer session. Please call the Mass. Chapter office to register and for detailed directions to the conference site. Telephone: 617-245-2133; 1-800-258-3323. (5) ===== re: stiffness ========== Date : Wed, 08 Mar 95 20:59:01 -0500 >From : "Dr. Kenneth Fischbeck" Subject: RE: stiffness Leg stiffness (spasticity) in ALS can sometimes respond to treatment with baclofen or low dose Valium. It might be worthwhile for your mother to check with her neurologist about this. K. Fischbeck, M.D. Neurology Dept., Univ. Pennsylvania (fischbeck@a1.mscf.upenn.edu) (6) ===== re: Managing Swallowing Problams ========== Date : Fri, 10 Mar 95 06:25:56 PST >From : andrea@tc.fluke.COM (Carey Denise Pickens) Subject: Managing Swallowing Problems. ALS Digest 178 Thank you Robert Macdonald for your contribution on handling swallowing problems. I was diagnosed with (F)ALS in Sept. '94 a month after my brother was diagnosed with the same. We are both in our late 30's. Of eleven known cases in our family since my Great-grandmother, four of us are currently living with ALS. At least two others are bing checked as the symptoms begin to show. I would like to add a additional suggestion for help with choking. Please understand that this is something that may sound like snake oil to most and I won't blame anyone for saying so. But I have seen it work and believe ALS patients should at least know about it as they can make their own decision. A relative of mine has had the disease for a little more than twenty years. She has had a trach for some time and for the most part is wheelchair bound, but is active as she can be with ALS. Before and after the trach she had experienced some very bad bouts of choking.It was during one of these terrible choking episodes, out of desperation and frustration that she had her husband make a martini. That's right, a martini, (on the rocks). She stumbled on a treatment that provides her with instant relief from choking. Granted, it's not 100% effective but I would venture to say at least 80%. Something with the cold alcohol seem to relax the throat and reduce the choking. This is not scientific and to mention it to your doctor would probably result in a chuckle if not a cycnical sneer. And of coarse, not everyone that chokes should try this. In fact if a non-drinker like myself tried it, would probably choke worse on the gin than from the ALS induced choking. But this has been a blessing for her, and allowed her to eat and function more normally. She only consumes a sip when the choking begins or when she feels she might start choking. The relief seems almost instantaneous. It certainly has given more control to her gag reflex. As far fetched as this all sounds, I hope it helps at least one person with this affliction. And if anymore is using a similar method please let us all know about it. Sincerely, Al Pickens (7) ===== some questions ========== Date : Fri, 6 Jul 1990 16:21:19 +1030 >From : Mark Walter Subject: Re: ALSD#177 ALS-ON-LINE Do most patients with lower motor neuron disease later develop upper motor neuron signs? And at what point if they have not (1 year , 2 years etc.) would you think they will not? Also can anyone tell me is adult Progressive Muscular Atrophy now considered to be a variation of ALS or a separate diagnosable disorder? Thanks Mark Walter (8) ===== powerlessness/power/control ========== Date : Fri, 10 Mar 95 20:42:01 EST >From : "DESSUREAULT,JULIE,MS" Subject: Interested in conversing on ALS I am doing a study on ALS patients at the Montreal Neurological Institute. I would appreciate obtaining information on any recent articles pertaining to the issue of powerlessness/power/control. I thank you for your time and cooperation. Hope to hear from you soon. Julie Dessureault B.Sc (neuropsychology) M.Sc.(A) nursing BRRE@MUSICB.MCGILL.CA (9) ===== Riluzole ========== Marketletter 02/20/95 Rhone-Poulenc Rorer Looks To New Products For Growth < most of article deleted > Riluzole, an N-methyl-D-aspartate (NMDA) glutamate receptor antagonist, was also highlighted at the meeting as an agent with great potential, even though its first indications are in small patient populations. The first indications are for amyotrophic lateral sclerosis and Charcot-Marie-Tooth syndrome (progressive peripheral neurodegenerative diseases) affecting around 60,000 people worldwide. A global Phase III trial was closed at the end of last year, and analysis of the data is expected to be completed in April. Riluzole is classified as an orphan drug in the USA and Japan. A second Phase III trial is still ongoing in Japan. R-PR hopes to file for approval of riluzole this year if the pivotal trial results are positive. Simultaneous filings will be made in the USA and Europe, with a filing in Japan following on somewhat later. The company hopes that riluzole will eventually turn out to be useful in a number of other diseases - notably Huntington's and Parkinson's. Evidence for efficacy in the latter disease has come from animal studies, which suggest that the compound can protect against damage to dopaminergic neurons caused by the neurotoxins MPP+ and 6-hydroxydopamine. < rest of article deleted > (10) ===== CNTF & BDNF & Neurontin & more. ========== BIOTECHNOLOGY NEWSWATCH Monday February 20, 1995 LAST CLINICAL TRIAL OF CNTF TO TREAT LOU GEHRIG'S DISEASE DISBANDED By: Marjorie Shaffer A joint venture of Syntex and Synergen discontinued the only remaining clinical trial of CNTF for the treatment of Lou Gehrig's disease after the neurotrophic growth factor was ineffective in slowing progression of the neuromuscular disease. < parts deleted > Last year, Regeneron Inc., based in Tarrytown, N.Y. had to stop its clinical trial of CNTF because of intolerable side effects in ALS patients. The Syntex-Synergen trial of CNTF used lower doses than the Regeneron test, so researchers were hopeful that the growth factor would prove less toxic. ``People were really hopeful, and this latest failure has been a huge disappointment,'' said Sydell Chaiet, a spokeswoman for the ALS Association, a patient support and educational group based in Woodland Hills, Calif. < parts deleted > Currently, there is only one large ongoing study of an experimental treatment for ALS. Amgen is testing brain-derived neurotrophic factor in a multicenter study, but results have not yet been released. In animal studies, BDFN, like ciliary neurotrophic factor, delays progression of ALS. When both factors are injected, the axons of motor neurons actually regenerate, according to Hiroshi Mitsumoto of the Cleveland Clinic Foundation, who is testing Amgen's BDNF. Some researchers have speculated that a cocktail of nerve growth factors could be the next step in clinical tests. A small study of megadoses of anti-oxidant vitamins in ALS patients is underway at Massachusetts General Hospital in Boston, but so far there have been no dramatic results, said Didier Cros, co-director of the hospital's Neuromuscular Disease Clinic, who is directing the study. Two years ago, researchers led by Robert Brown of Massachusetts General Hospital and Teepu Siddique of Northwestern Medical School discovered mutations in the gene for the enzyme superoxide dismutase were linked to inherited cases of ALS. The enzyme sops up free radicals, and there is some preliminary evidence that some damage to motor neurons was caused by the accumulation of free radicals. The drug Neurontin, which is used to treat epilepsy, is also being tested in ALS patients and that study is just getting started. And researchers at Ohio State University, Columbus, are studying the effects of androgen hormones on the progression of ALS in men only. ===== = For some reason, the clinical trial of Riluzole was not mentioned in = this article. Also, does anyone know what happened with Cephalon's IGF-1 = (Myrotrophin)? = bro ===== (11) ===== BDNF and GDNF ========== WALL STREET JOURNAL 02/28/95 Corporate Focus: Amgen Turns Bullish on Possible Lab Breakthroughs --- Biotech Firm, After Drought of New Products, Has `Burgeoning' Pipeline ---- By Rhonda L. Rundle Staff Reporter of The Wall Street Journal THOUSAND OAKS, Calif. -- Amgen Inc. is back in Wall Street's good graces after a year of strong drug sales, a smart acquisition and a 50% gain in its stock price -- even after backtracking from loftier levels spurred by takeover speculation. But it's the science percolating through the laboratories at Amgen's sprawling complex here that is sending pulses racing inside what has become the world's leading biotechnology company. After a long, fallow period without new products, Amgen researchers and executives are fired up by what they hope will turn out to be a number of true "breakthrough" drugs in development. < most of article deleted > Amgen's Pipeline Selected drugs being tested: PRODUCT THERAPEUTIC USE STATUS < parts deleted > Brain-derived Amyotrophic Lateral Phase I/II neurotrophic Sclerosis (Lou Gehrig's factor (BDNF) disease) Glial cell-line Parkinson's disease Preclinical derived neurotrophic factor (GDNF) < rest of article deleted > (12) ===== Patients suffer while FDA dithers ========== Source: THE DES MOINES REGISTER 02/27/95 Titlt : The Register's Readers Say Credit: Readers Patients suffer while FDA dithers Your editorial on Feb. 15 defended the FDA as essential to protect the public from bad drugs and charlatans. As a result of its action in preventing distribution of Thalidomide 30 years ago, the FDA has benefited from an undeserved reputation as a protector of patients. Actually, thousands die while the FDA dithers in approving life-saving drugs. In the two years it took to approve TPA, some 200,000 people died of heart attacks; not that a large number of them might have survived if TPA were available, but they did not even have a chance to get the drug. In my own case, as a victim of amyotrophic lateral sclerosis (Lou Gehrig's disease), a new drug for this disease was withdrawn from clinical trials recently because it did not meet FDA standards of efficacy. This efficacy standard is too strict when dealing with potential life-saving drugs for people with terminal illnesses. In essence, the FDA says that there must be a 95 percent probability (or close to it) that any beneficial effect is the result of the drug and not due to some random factor. I would be happy to take any drug that might have even a 50-50 chance of improving my survival, but I am denied the opportunity to judge for myself whether I want to take that risk. Morning sickness is not terminal or life-threatening. A 95 percent probability of effectiveness and ample data on safety are essential for drugs in that category, but patients with terminal diseases need the opportunity, now denied by the FDA, to take risks and take experimental therapies. A recent New York Times article on the political pressures being applied to the FDA pointed out that the drug companies do not want to abolish the FDA, only make it more efficient and consume less time in its deliberations. That is a reasonable goal, toward which the FDA is slowly moving, having reduced its average approval time for new drugs 20 percent in 1994 compared to 1993, to 19 months! We need safe drugs to deal with routine illnesses, but we also need to let patients take informed risks when they want. The FDA should realign its thinking to let patients make decisions, not make decisions for them. - Ted Heine, 503 Third Ave. N.E., Waverly. (13) ===== Battelle looks at the future ========== Title: Battelle looks at the future COLUMBUS, Ohio, Feb. 22 (UPI) -- The song says "the future's not ours to see," but researchers at Battelle Memorial Institute have compiled a list of 10 technologies they see becoming realities by 2005. Included in the worldwide business and technology institute's list are personal computers with vast storage capabilities that we will carry in our pockets, anti-aging products and services that really work, and medical treatments delivered through sensors to specific parts of our body. < parts deleted > The researchers predictions, in order of perceived importance, are: 1. Mapping of the human genome for genetic-based personal identification and diagnostics. Researchers will look for ways to treat diseases before they occur. For example, if a person carries the gene that gives them a 90 percent probability of contracting Lou Gehrig's disease, treatment could start in hopes of preventing the occurrence of the disease. Genome mapping might also lead to cures for specific cancers. < rest of article deleted > === end of als 179 ===