Date: Tue, 8 Nov 94 09:35:45 -0500 From: Bob Broedel To: Stuart.Neilson@brunel.ac.uk Subject: Re: ALS DIGEST BACK ISSUES =============================================================== == == == ----------- ALS Interest Group ----------- == == ALS Digest (#136, 19 October 1994) == == == == ------ Amyotrophic Lateral Sclerosis (ALS) == == ------ Motor Neurone Disease (MND) == == ------ Lou Gehrig'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 == == 360+ 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 survivors 2 .. neurontin 3 .. Charlie Wedemeyer 4 .. long lived ALS patients 5 .. MDA Forum on CompuServe 6 .. Longevity with ALS 7 .. ATTN: Scientists who want to write 8 .. Attention PHYSICIANS -- Please read 9 .. The HopeLine 10 . rare diseases (1) ===== ALS survivors ========== Date : Tue, 18 Oct 94 08:40:47 CDT >From : optimg!philw@uunet.uu.net (Phil Walsh) Subject: ALS survivors RE: the current thread about "longest surviving ALS patient": I believe Stephen Hawking, the British physicist, contracted ALS around 1961. He's still alive or was as of just a year or so ago. For those that haven't read it or seen the movie, I highly recommend "A Brief History of Time," which is about Hawking and his accomplishments in the field of theoretical physics. Some call him "the new Einstein" (others don't...:-) ) but the movie is very good, and it is especially captivating to anyone with a personal interest in ALS. --philw (2) ===== neurontin ========== Date : Sat, 15 Oct 1994 21:51:11 EDT >From : CVVW06A@prodigy.com (MR ROBERT A HESTON) Subject: Re: ALSD135 ALS-ON-LINE My best friend was dxed with ALS almost 2 years ago and has been reading this digest with a lot of interest. He raised an interesting point about Neutontin. He is maybe in line to start a new drug test on Neurontin in January. His question was this: Does it make sense to start a double-blind study and take a chance on receiving a placebo or to go to another MD and just have him write an Rx for the real thing? He is currently supposed to be off a CNTF study in January and them nothing except for a chance to be in this new trial . He doesn't want to do anything to jeapordize his place in the CNTF study, but as he is afflicted with Bulbar ALS and Neurontin seems to help this he is rather confused. Any help from other readers would be appreciated. Thanks Rob (3) ===== Charlie Wedemeyer ========== Date : 15 Oct 94 19:29:15 EDT >From : wayne <70303.173@compuserve.com> Subject: Charlie Wedemeyer Hi all; A friend gave us an inspirational book, "Charlie's Victory," by Charlie and Lucy Wedemeyer, published by Zondervan. I want to share it with you. Charlie has had ALS for 18+ years. He's a high school football coach living in the San Francisco bay area. You may have seen one of the movies about him made by PBS and CBS. A new movie is in the works now. Enjoy, Wayne Phillips 70303.173.compuserve.com ===== = Does anyone have ordering info ... address of publisher, price, etc. ? ===== (4) ===== long lived ALS patients ========== Date : 15 Oct 94 18:28:33 EDT >From : Wayne Phillips <70303.173@compuserve.com> Subject: long lived ALS patients Stephen Hawking, the British physicist; onset at age 20, lived 30 years with it so far Lu Medel, of Carpenteria, Calif.; passed away August; originaly diagnosed with Parkinson's in 1974 at 53, later changed to ALS; never on a feeding tube or ventilator Charlie Wedemeyer, the San Francisco area football coach; onset 1977 (maybe '74?), still going Ron Leeb of L.A. (reads this digest); now 37, has had ALS for 15 years Dr. Lance Meagher of Oregon, passed away several years ago, after 12+ years with ALS, onset at age 28 (5) ===== MDA Forum on CompuServe ========== MDA Forum Administrators (Sysops) have a wide range of backgrounds, but all have one thing in common: they have firsthand, personal experience living with a neuromuscular disease. They are ready help you get the most out of the MDA Forum. If you have questions about the Forum or suggestions on how it can be improved, send any one of the Sysops an e-mail. The MDA SYSOPS are: * Jim Tillman [74431,2312] ......... 74431.2312@compuserve.com * Chris Rosa [74431,2310] .......... 74431.2310@compuserve.com * Ken Jasch [74431,2306] ........... 74431.2306@compuserve.com * Lori Hinderer [74431,2311] ....... 74431.2311@compuserve.com * Mark Hathaway [74431,2307] ....... 74431.2307@compuserve.com * Barry Goldberg [74431,2305] ...... 74431.2305@compuserve.com * Tom Bush [74431,2513] ............ 74431.2513@compuserve.com The MDA Forum is managed by the Muscular Dystrophy Association. Please address all inquiries to: Tom Bush, Primary Sysop Compuserve's MDA Forum MDA National Headquarters 3300 East Sunrise Drive Tucson, AZ 85718-3208 E-mail: 74431.2513@compuserve.com ===== = I want to do something like this for the Prodigy ALS group. = Could someone provide a brief overview and list the Internet = formatted e-mail addresses of the sysop, some key people, etc. = Also please give info about how to join. rgds,bro ===== (6) ===== Longevity with ALS ========== Date : Tue, 18 Oct 1994 00:06:37 -0500 (CDT) >From : Clive David Subject: Longevity with ALS Adding to the discussion about "records": A 1991 biography on Stephen Hawking by Kitty Ferguson gives the following chronology: Born: 1942 Diagnosed: "... in his early twenties ..." (say, 1965?) BY 1980: age 38; still teaching & doing research, but no longer walking, writing, feeding himself, or able to raise his head; virtually no intelligible speech As far as I know, Hawking is still alive today (of course, he has had supports that may not be available/offerred to others?). From first diagnosis, he has lasted just about 29-30 years. ============================ |Clive David | |E-mail: cdavid@worf.uwsp.edu| ============================ (7) ===== ATTN: Scientists who want to write ========== Subject : ATTN: Scientists who want to write Newsgroups: bionet.neuroscience Date : 9:21 PM Oct 3, 1994 >From : Robert Coontz WANTED: Scientists who want to write about their research or fields for intelligent non-specialists. That's the stock in trade of "The Sciences," an unusually handsome and literate science magazine published by the New York Academy of Sciences. Specifically, we are looking for articles and essays, 3,000 to 4,500 words long, dealing non-technically with all scientific disciplines and issues. You don't have to be the next Asimov, Gould or Trefil (though nothing would please us more), as long as you have something worth saying and a knack for telling the tale. Our editorial team can help you to burnish your prose to a high gloss. If you are interested, get in touch with me by E-mail, phone, or letter. And please feel free to post this notice anywhere appropriate. I look forward to hearing from you. -- Robert Coontz Senior Editor, "The Sciences" 622 Broadway, New York, NY 10012 Phone: 212-460-9027, ext. 29 E-mail: rcoontz@igc.apc.org or 100010.3053@compuserve.com (8) ===== Attention PHYSICIANS -- Please read ========== Date : Wed, 12 Oct 1994 03:07:21 GMT Sender : "Clinical WANS: Design & Usage" : >From : "Richard E. Gans M.D." Subject: Attention PHYSICIANS -- Please read I am the Medical Director of the National Public Telecomputing Network (NPTN), a network of educational, community-based, computer bulletin board systems. I am now seeking the assistance of other physicians in all specialties, world- wide, who might be willing to donate a small amount of time on a regular basis to serve as medical consultants to this network. Consultants would answer questions posted, anonymously, by local subscribers. If desired, they could compose static information files to enhance public knowledge of their particular field of interest. All newsgroups are moderated to ensure the presence of appropriate postings. All information posted is cybercast to affiliate systems throughout the world. NPTN is the parent organization of the "FreeNet" systems, springing up in cities throughout the world. The first freenet was Cleveland Freenet in Cleveland, Ohio USA. There are now 37 active systems online. In addition, there are 124 organizing committees in cities planning to come online in the future. All totalled, there is involvement in 41 of the 50 United States and in ten countries. If you are interested in additional information, please contact me via email at: aa014@nptn.org Richard E. Gans, M. D. (9) ===== The HopeLine ========== Of course everyone should subscribe to LINK as a way of keeping informed about ALS, but for those seeking more info, some of the monthly newsletters of ALS Association chapters and/or support groups can be useful also. For example the monthly THE HOPELINE newsletter of the ALSA, SE Wisconsin Chapter (3070 North 51st Street, Suite 107; Milwaukee WI 53210; TEL 414-445- 7257, FAX 414-445-1723) is excellent. It is edited by Pat Vermilyea RN, and is $10 per year for non-members. (10) ===== rare diseases ========== Citation: The Lancet, June 18, 1994 v343 n8912 p1560(2) --------------------------------------------------------------------- Title: Rare diseases and orphan drugs. (international symposium hosted by the Mario Negri Institute at Villa Camozzi, Italy, June 8-9, 1994) Authors: Daina, Erica --------------------------------------------------------------------- Rare diseases are little-known and generally misunderstood, poorly investigated, and left with no remedy. A clinical centre devoted to develop research projects on rare diseases was recently opened by the Mario Negri Institute at Villa Camozzi, near Bergamo, Italy. On June 8-0, the centre held an international symposium on rare diseases and orphan drugs. Rare diseases are often called the "health orphans," and researchers, physicians, and health authorities recognise that the knowledge about causes and pathogenesis, the effort in experimental and clinical research, and the prospect of a therapeutic breakthrough are scarce or negligible for too many of them. If seems that the needs of patients with rare diseases are unlikely to be met even in developed countries. The barrier to prevention, diagnosis, and treatment of rare diseases is lack of knowledge. Basic science researchers and clinical investigators acknowledged that rare-disease projects are not appealing because they are unlikely to be funded. This happens despite the lesson from the history of medicine that many advances have been achieved as the result of the study of rare diseases. The availability of drugs to treat rare disorders is limited by their weak economic incentive and lack of commercial value. Yet rare conditions such as amyotrophic lateral sclerosis, Gaucher's ============================= disease, and Huntington's chorea affect thousands of people world wide. Dr Marlene Haffner, director of the Orphan Products Development Office at the US Food and Drug Administration, emphasised the importance of the Orphan Drug Act. Under the law, signed in 1983 by President Reagan, a designated orphan product - ie, a product used to treat a condition that affects fewer than 200000 people - has seven years of marketing exclusivity. Other encouragements are protocol assistance from the FDA, tax incentives for clinical development of the product, and availability of open protocols for using orphan products during the clinical testing phase. Haffner reported that 103 orphan drugs received FDA approval in the 11 years since the Act was passed, and she estimated that more than 3.5 million people with rare diseases have benefited. 20% of these drugs are for childhood diseases, 15 drugs are indicated for treatment of rare cancers, and the geriatric population has also gained. The Act has also helped in the development of drugs for disease that are rare in North America but common in developing countries. Since 1983, amendments have been approved and have ameliorated the original bill, thanks to the continued efforts of rare-disease voluntary organisations. Ms Abbey Meyers is the leader of one of the most strenuous advocates for rare-disease patients, the National Organization of Rare Disorders (NORD). She underlined that one of the most important barriers to advancements in the identification, understanding, and treatment of rare diseases is the paucity of information available to patients and professionals. Thus patients are often not diagnosed or misdiagnosed for long periods, physicians do not provide timely diagnosis and appropriate treatment, scientists have difficulty locating subjects for clinical trials, and organisations ignore research and development of treatments because of the small patient population. NORD's most important task is the dissemination of information. As a patient said: "Orphan diseases are not important unless you happen to have one". What about in Europe? William Gunnarsson of Orphan Europe, SARL, addressed the question of how European Community rules applicable to orphan products compare with the Orphan Drug Acts of USA and Japan. In Europe there are two different regulatory systems: the European Union has one regulatory system and each country has its own. The harmonisation between the different structures is not always ideal. Moreover, although there are specific European directives that can be applied to some aspects of orphan drug development, the companies in Europe have no specific programmes for developing these products. The most common explanation for this lack of activity is that there is no European legal definition of orphan drug. It is very difficult, Gunnarsson said, for the European pharmaceutical industry to motivate investment into research and development for orphan drugs without specific legislation in the European Union. The speakers and the attenders at the symposium were strongly convinced of the need for continuing the battle for rare diseases. ============================================================================== === end of als 136 ===