Date: Fri, 15 Dec 95 18:44:27 EST From: Bob Broedel To: als@huey.met.fsu.edu Subject: ALSD229 ALS-ON-LINE =============================================================== == == == ----------- ALS Interest Group ----------- == == ALS Digest (#229, 15 December 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 == == 1320+ 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 == =============================================================== == A full set of back issues (on MSDOS 3.5 INCH HD diskette) == == are available by sending me your full mailing address. == == They are free-of-charge. International requests welcome. == =============================================================== CONTENTS OF THIS ISSUE: 1 .. Editorial 2 .. Rilutek/Riluzole #1 3 .. Rilutek/Riluzole #2 4 .. Rilutek/Riluzole #3 5 .. Alaska Doctors 6 .. Request for ALS treatments 7 .. Identical twins and ALS 8 .. re: Indentical twins and ALS 9 .. CytoTherapeutics / CNTF / ALS 10 . Fund Drive Press Release 11 . Myotrophin 12 . Amylodosis (1) ===== Editorial ========== Sorry ... I got behind again. I will try to make up for lost time by sending one issue per day until all notes are published. rgds,bro (2) ===== Rilutek/Riluzole #1 ========== RILUTEK(R) (RILUZOLE) APPROVED BY U.S. FOOD AND DRUG ADMINISTRATION FOR TREATMENT OF LOU GEHRIG'S DISEASE First Drug to Effectively Treat Disease Since it was First Described in 1869 COLLEGEVILLE, Pa. Dec. 12 /PRNewswire/ -- Rhone-Poulenc Rorer Inc. (NYSE: RPR) announced today that it has received clearance from the U.S. Food and Drug Administration (FDA) to market Rilutek(R) (riluzole), the first effective drug for the treatment of amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease). No treatment was previously available for this fatal, neurodegenerative disease since it was first described 126 years ago. Rilutek is the first drug shown to extend survival in people with ALS. The New Drug Application (NDA) received approval from the U.S. FDA less than six months after RPR submitted it on June 29, 1995. "Rilutek is the first drug to demonstrate efficacy in the treatment of ALS," said Gary T. Shearman, Senior Vice President, Drug Development, RPR. "It represents a critical first step and important therapeutic gain in ALS treatment. Our number one goal is to make the drug available to ALS patients as quickly as possible." Rilutek will be available by prescription in six weeks as a 50 mg tablet to be administered twice daily. An official launch news conference will be held on January 10, 1996, to discuss drug availability and patient support services (a separate announcement will identify the exact time and location). A toll-free telephone number will be available for people who cannot attend the conference. People who have questions about Rilutek before January 10th can call 1-800-RX-TRIAL (1-800-798-7425) for more information. Clinical Trial Results In the largest ALS clinical trial ever, Rilutek was shown to extend the survival of ALS patients. A total of 959 patients participated in a multi-center, double-blind, placebo-controlled Phase III trial, in which survival served as the primary endpoint. In addition to demonstrating a survival benefit, Rilutek was generally well-tolerated. Side effects reported in the trial included nausea, asthenia (fatigue) and elevated liver enzymes. The Phase III trial was conducted at 31 sites in Europe and North America. In the United States, all centers were certified and funded by the Muscular Dystrophy Association. The participating centers were Baylor College of Medicine, Veterans Affairs Medical Center (Houston); Johns Hopkins University (Baltimore); Northwestern University (Chicago); California Pacific Medical Center (San Francisco); and Tufts-New England Medical Center (Boston). ALS is a fatal, neuromuscular disease affecting approximately 25,000 to 30,000 people in the United States and 70,000 people worldwide. 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. The outward signs of ALS are progressive weakness and deterioration of the muscles (amyotrophic), beginning in limbs, usually on one side of the body (lateral). Inside the body, the nerves controlling motor function die off after their cell bodies become hardened and shriveled (sclerosis), leaving the patient increasingly helpless. The FDA allowed an Early Access Program (EAP) treatment protocol to proceed on June 20, 1995. The EAP was established by RPR to allow a limited number of people with ALS to receive Rilutek free-of-charge pending FDA approval and commercial availability. A total of 3,000 patients were selected to participate in the program. Rhone-Poulenc Rorer is a global pharmaceutical company dedicated to improving human health. (3) ===== Rilutek/Riluzole #2 ========== FDA APPROVES FIRST DRUG FOR LOU GEHRIG'S DISEASE WASHINGTON, Dec. 12 /PRNewswire/ -- The Food and Drug Administration today announced the approval of riluzole, the first drug that has been shown to prolong the survival of patients with amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease. ALS, which results in progressive muscular weakness and paralysis, has been without cure since it was first identified in 1869. FDA carried out the review of riluzole in five-and-a-half months. Commenting on the approval, Secretary of Health and Human Services Donna E. Shalala noted that when tested in two placebo-controlled studies on more than 1,000 patients, riluzole prolonged survival on average by about three months. "This is a modest effect, but before riluzole there was no therapy for ALS at all," the Secretary said. "Today's approval is an important milestone in the decades-long search for treatment for this devastating disease." FDA Commissioner David A. Kessler, M.D., pointed out that before today's approval, riluzole had been made available to more than 3,000 patients -- about one-tenth of the 30,000 Americans with ALS -- under an early access program granted by FDA and administered by the National Organization for Rare Disorders. "This is the first drug we have ever had that seems to make a difference in the course of ALS -- a dreadful disease," Kessler said. "We hope this is just a first step." ALS is a progressive disease that affects nerve cells in the brain and spinal cord and is usually fatal within five years after diagnosis. Adverse effects of riluzole observed in clinical tests include weakness, nausea, vomiting and elevated liver enzymes. FDA's Peripheral and Central Nervous System Drugs Advisory Committee reviewed the data from trials on riluzole on September 18, and recommended its approval for treatment of ALS. Riluzole is manufactured by Rhone-Poulenc Rorer Inc. of Collegeville, Pa., and is distributed under the trade name Rilutek. FDA ON THE INTERNET: http://www.fda.gov/ /CONTACT: Susan Cruzan of the Food and Drug Administration, 301-443-3285 or, home, 301-834-7332/ (4) ===== Rilutek/Riluzole #3 ========== This section is a reprint from ALS Digest #190. ===== International numbers for Rhone-Poulenc Rorer ========== COUNTRY MAIN TELEPHONE MAIN TELEFAX =================================================== Australia 61 3 854 8000 61 3 854 8001 Austria 43 1 45 35 05 41 1 45 77 73 Belgium 32 2 529 46 11 32 2 529 46 98 Canada 1 514 384 8220 1 514 385 7480 Denmark ....... 54 42 81 28 88 .... 45 45 82 12 50 Finland 358 0 757 26 22 358 0 757 32 80 France 33 1 40 91 61 23 33 1 47 02 50 14 Germany 49 221 509 01 49 221 509 27 11 Greece 30 1 65 44 962 30 1 65 44 964 Holland ....... 31 20 547 3922 .... 31 20 645 3816 Ireland 353 1 289 6586 353 1 289 4984 Israel 972 9 851 222 972 9 851 444 Italy 39 2 9610 1 39 2 967 30535 Japan 81 3 3532 6111 81 3 3532 9605 South Korea ... 82 2 362 2388 ..... 82 2 393 1400 Mexico 52 5 559 4988 52 5 575 1785 New Zealand 64 4 567 86 21 64 4 567 55 38 Norway 47 67 14 99 92 47 67 14 85 66 Poland 48 2 614 57 15 48 2 614 58 11 Portugal ...... 351 1 886 61 34/9 . 351 1 87 88 37 South Africa 27 41 33 4450 27 41 33 1598 Spain 34 1 641 60 13 34 1 641 67 61 Sweden 46 42 25 34 00 46 42 15 73 00 Switzerland 41 1 723 53 11 41 1 723 53 10 United Kingdom 44 1323 534 000 ... 44 1323 534 080 USA 610 454 8000 610 454 3813 (5) ===== Alaska Doctors ========== Date : 01 Dec 95 02:01:00 GMT >From : JAMITCHE@health.state.ak.us (Mitchel, Judy A) Subject: Alaska Doctors I am looking for an Anchorage doctor that has experience in diagnosing ALS, any help would be greatly appreciated. (6) ===== Request for ALS treatments ========== Date : Thu, 30 Nov 1995 20:00:18 -0500 >From : HHEMP@aol.com Subject: Request for ALS treatments My Aunt has recently been (tentatively) diagnosed with ALS. While I have had little contact with her directly in regards to this diagnosis, I was wondering what typical treatments (sustaining) exist at this time. What resources exist, and how can they be contacted? Also, what European drugs (not FDA approved) are currently being employed? Can anyone out there point me in the right direction? Please E-mail to: HHemp@AOL.com Thank you very much, Harlan Hemple (7) ===== Identical twins and ALS ========== Date : Wed, 13 Dec 1995 00:27:39 -0500 >From : MLUTERMAN@aol.com Subject: identical twins and als Does anyone know of a study looking at ALS and identical twins? I have a patient whose identical twin has ALS and he is freaking? Would be grateful for help.... Thanks Dr. L MLuterman@AOL.com (8) ===== re: Identical twins and ALS ========== Date : Wed, 13 Dec 1995 11:18:40 >From : Stuart.Neilson@brunel.ac.uk (hssrsdn) Subject: Re: identical twins and als Dear Dr Luterman, The largest examination of MND/ALS amongst twins was conducted by Dr Hawkes at the Department of Clinical Neurology, Ipswich Hospital (England). The abstract to the first report records "Results. The search produced: 54 living co-twins; 31 who died as adults; 29 infant deaths; 5 emigrated; 3 incorrectly diagnosed index twins; and 9 untraceable co-twins. Among the adult deaths two concordant pairs were identified. This has created the largest twin population sample worldwide for MND." [3]. Two more reports have appeared, but both are abstracts to meetings rather than full papers [1,2] and more methodological than analytical. The number of cases in which MND/ALS has been recorded in both of twins is so small (just 2 in this study) that it is not possible to conclude that the twin of a person with MND is at exceptional risk, or to exclude that possibility. However, shared environment and shared exposure to possible risk factors (eg: influenza during childhood, or of the mother during pregancy [4]) should be considered in addition to genetic similarity of twins. Yours sincerely, Stuart Neilson (stuart.neilson@brunel.ac.uk), CSHSD, Brunel University (WWW: http://http2.brunel.ac.uk:8080/~hssrsdn/) [1] HAWKES, CH; GRAHAM, A; MACDONALD, A (1995). BRITISH MOTOR-NEURON DISEASE TWIN STUDY. NEUROLOGY, 45;4(S4):A381-A381 [2] GRAHAM, AJ; HAWKES, CH (1995). TWIN STUDY USING MORTALITY DATA - A NEW SAMPLING METHOD. INTERNATIONAL JOURNAL OF EPIDEMIOLOGY, 24;4:758-762 [3] HAWKES, CH; GRAHAM, AJ (1992). NATIONAL UK MOTOR-NEURON DISEASE TWIN STUDY USING THE DEATH DISCORDANT APPROACH. ANNALS OF NEUROLOGY, 32;2:272-273 [4] CURRIER, RD; CONWILL, DE (1988). IS AMYOTROPHIC LATERAL SCLEROSIS CAUSED BY INFLUENZA AND PHYSICAL ACTIVITY - RESULTS OF A TWIN STUDY. ANNALS OF NEUROLOGY, 24;1:148-148 (9) ===== CytoTherapeutics / CNTF / ALS ========== CytoTherapeutics has positive Phase I data NEW YORK, Nov 9 (Reuter) - CytoTherapeutics Inc said a Phase I study of its cell-containing implant for treating ALS has shown the treatment well-tolerated by patients and effective in delivering a growth factor. CytoTherapeutics Chief Executive Officer Seth Rudnick told Reuters that in the Phase I study conducted in Switzerland, ten patients with ALS received the implants containing cells genetically engineered to deliver the growth factor CNTF. The study showed the cells lived three months and produced CNTF in measurable quantities in the cerebral spinal fluid space, Rudnick said. CNTF has been shown to combat ALS by sustaining motor neurons by ability, he said. < parts deleted > Rudnick noted attempts by other biotechnology companies to administer CNTF intraveneously failed due to severe side effects, not noted in the CytoTherapeutic study. < parts deleted > (10) ===== Fund Drive Press Release ========== Date : Sun, 10 Dec 1995 21:00:48 -0800 >From : jphilbro@nwlink.com (John Philbrook) Subject: Fund Drive Press Release PRESS RELEASE**PRESS RELEASE**PRESS RELEASE**PRESS RELEASE**PRESS RELEASE ------------------------------------------------------------------ Robert Codomo, a young man of 25, has quite normal plans for taking advantage of the latest technological developments in the near future: getting on the information super-highway, creating web pages, sending e-mail, playing the latest multimedia games, maybe even publishing a book or taking a course on the Internet. His immediate problem now seems more pressing, however. He is trying to speak the words, "Please move my legs." His Nurse obligingly accommodates him, having learned to interpret Robert's barely audible whispers. Robert has ALS (Amyotrophic Lateral Sclerosis), Lou Gehrig's disease, which he developed from it's onslaught in less than a year. He cannot communicate effectively, for he is connected to a respirator; he can move only his mouth and his eyes--he will probably not lose the movement of his eyes. Most would say Robert has little if anything to live for. He thinks they are wrong. "I want to live," is Robert's emphatic reply. Just another tragic story on a list of cruel realities in life? No. Modern computer technology can give Robert a "virtual" life to live: an infrared computer control interface, "Eyegaze," has been developed for ALS victims, enabling them to speak and utilize phone communication through voice synthesis, to control their environment through computer switch controls, and most importantly, to control a computer for navigation of the worldwide Internet. Robert has the promise of coming to life again with this marvel of modern technology. This is to announce the organization of a fund drive for Mr. Robert Codomo, toward the acquisition of an Eyegaze System for his use. It is being organized by his close friends who have investigated the possibilities of giving Robert access to the outside world through computer technology. The fund drive will begin the week of December 10, and will continue until approximately $30,000 are raised to purchase the System and peripherals. The fund drive will focus on individual contributions, encompassing the entire Puget Sound region, and will have, as it's basis, information supplied by a Web site (http://nwlink.com/robertfund/ available beginning the week of the 10th) and local media. All news media are asked to participate at any level in publicizing Robert's need for funding of his equipment. The equipment will be demonstrated for Robert the afternoon of the 11th, and the morning of the 12th by Nancy Cleveland RN, of LC Technologies, Fairfax, VA. at: Rainier Vista Care Center 920 12th Ave. SE Puyallup, WA 98372 (206) 841-3422 Robert's friends, Stan Gazaway and John Philbrook are coordinating the drive. John Philbrook 206-746-2693 jphilbro@nwlink.com Stan Gazaway 206-865-8714, 206-703-0389 gazaway@decwet.ENET.dec.com Technical questions can be addressed via e-mail to: Catherine W. Britell, M.D. britell@u.washington.edu Or to LC Technologies: 9455 Silver King Court Fairfax, VA 22031-4713 (800) EYEGAZE (703) 385-7133 We believe this has the potential to be of great interest to the citizens of Western Washington, having the elements of biotechnology, computer technology, Internet accessibility, advancement of the handicapped, and human interest in the face of overwhelming obstacles. Robert is a wonderful young man, sure to win the hearts of all he comes in contact with. We welcome and urge any interest and participation. (11) ===== Myotrophin ========== Date : Wed, 6 Dec 95 09:18:29 est >From : "Shalom J. Wind 914 945 2670" Subject: Myotrophin Has there been any word yet on a recommendation by the FDA committee on Myotrophin? My mother had heard a rumor that such a decision was to be deferred an extremely long time (16 - 18 months) in order to reconcile results of European and North American trials. We are, of course, hoping that this is not the case. Dr. S.J. Wind Nanostructures and Exploratory Devices IBM T.J. Watson Research Center P.O. Box 218 Yorktown Heights, NY 10598 (12) ===== Amylodosis ========== Date : Sun, 10 Dec 1995 15:18:59 -0500 >From: JOzBear@aol.com My father has been diagnosed as having Amylodosis NOT ALS though very similar. I am having a difficult time finding any information or research being done in association with this disease. Can anyone help? === end of als 229 ===