Date: Fri, 1 Sep 95 23:36:22 -0400 From: Bob Broedel To: als@huey.met.fsu.edu Subject: ALSD212 ALS-ON-LINE =============================================================== == == == ----------- ALS Interest Group ----------- == == ALS Digest (#212, 02 September 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 == == 960+ 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 == =============================================================== CONTENTS OF THIS ISSUE: 1 .. re: ALS heredity 2 .. IGF-1 3 .. Should my wife have a tracheostomy? 4 .. Questions on throat irritation 5 .. Riluzole 6 .. CLINICAL-TRIAL-FINDER list started 7 .. Listing of clinical trials on the Internet (1) ===== re: ALS heredity ========== Date : Wed, 30 Aug 95 09:10:01 -0400 >From : "Dr. Kenneth Fischbeck" To : docdavy@aol.com Subject: RE: ALS heredity In answer to the question you posted on 25 Aug 1995, only a small proportion of ALS is familial (about 5-10%). Genetic testing may be available on a research basis, but unless multiple family members are affected the yield is very low. K. Fischbeck, M.D. Univ. Pennsylvania Med. School (2) ===== IGF-1 ========== Date : Wed, 30 Aug 95 17:28:52 BST >From : a-furley@nimr.mrc.ac.uk (Andy Furley) Subject: IGF-1 Cc : 72124.2314@compuserve.com In response to Scott Veggeberg, ALS208 ALS-ON-LINE > >As most of you know, IGF-1 has also been the subject of successful phase >III clincial trials by Cephalon, Inc. of West Chester, PA, as a new >treatment for ALS. In fact, I would think a number of subscribers have >already been treated with IGF-1 as part of these trials. > I think that there is serious reason to be cautious about the "success" of the IGF-1 trials. I have heard, from both a respected UK ALS/MND clinician and from a company of financial analysts specialising in biotech pharmaceuticals (Smith Barney, New York) that there are some serious questions about the way the trial was set up and about how the results were reported. The information from Smith Barney came in the form of a report entitled: "Biotechnology: Overview of recent ALS clinical trials" dated July 7, 1995. I am not qualified to go into the details here but Smith Barney conclude that "IGF-1 (Myotrophin) will have difficulty getting approval from the FDA". The report was written by Toni Claudio, Ph.D. [(212) 816 8835] and Reijer Lenstra, M.D., Ph.D., M.B.A. [(212) 816 8834]. For further information I would suggest contacting the authors at the above phone numbers. I hope that this information merely reflects the caution of Smith Barney rather than a real deficit in the efficacy of IGF-1. However, it seems possible that Cephalon presented their results in a more rosy light than the data justifies and so I feel it only fair that the subscribers to ALS-ON-LINE be aware of these criticisms. I should make it clear that this information personal to me and does not reflect the views of the Medical Research Council of the UK. Andrew J.W. Furley, Ph.D. Developmental Neurobiology National Institute for Medical Research The Ridgeway, Mill Hill LONDON, UK-NW7 1AA Tel. +44 181 959 3666 x2252 Fax. +44 181 913 8536 or 906 4477 (3) ===== Should my wife have a tracheostomy? ========== Date : Thur., 31 Aug 1995 >From : dick.tauber@turner.com Subject: Should my wife have a tracheostomy? My wife, Marjorie Hirschberg is now bedridden with ALS. She lost the use of her legs last January, and after being stable for several months, began loosing use of her right arm along with stressful breathing at the end of May. Today she has totally lost the use of her right arm and hand and has just minimal use of her left arm. She cannot write or feed herself. On the 4th of July we went out to the Mayo Clinic where ALS was confirmed. Before that her neurologist had only diagnosed her as having atypical ALS. This left us some hope that it might be something else, but it also cut her off from any of the ALS trial drug programs. Since returning to Atlanta in the middle of July, her condition has worsened. She has been on a B-Stat P forced air machine since 7-21-95. At first she only used it during the night, and on occassion during the day. She can no longer breath without the machine. She can still talk while wearing the nose mask (she switches off with a full nose and mouth mask) to get her air, and can be understood 85% of the time. A pulmonologist has offered the possibility of a tracheostomy that would make her lungs work, and keep them working no matter how advanced her condition becomes. He has also suggested that a feeding tube be inserted at the same time to insure proper nourishment. BEFORE PROCEEDING WITH THE TRACHEOSTOMY OR THE FEEDING TUBE, WE WANT TO FIND OUT MORE ABOUT WHAT THIS WILL MEAN FOR HER AND WHAT HER LIFE WILL BE LIKE. But, before putting forward a number of questions we have, here is a bit more of her history: Marjorie was seeing a podiatrist in the Spring of 1994 for a painful heel spur in her LEFT foot. After exercises and finally shots failed to give her relief, the doctor suggested minor surgery that worked 80% of the time and 100% if a second cutting was necessary. My wife sought a second opinion from an orthopedist. He said: "No, don't have surgery! This can be cured with special streching exercises. Give it a try! You can always have the procedure done later." While doing the exercises, Marjorie felt discomfort in her legs that was more than just aches from the workout. While attending a yoga class in August '94 she had trouble balancing and could not stand on her toes. She had also started limping on her RIGHT foot. She called the orthopedist and he said she should see a neurologist right away. During the fall of 1994 the limp became drop-foot and she started needing a cain, then a walker. The neurologist did a bunch of nerve-muscle reaction tests; an MRI; a spinal tap and in October, a nerve and muscle tissue biopsy from her right leg. All tests were negative. In December she started using a wheel chair. During this entire period we held a strong belief that she could be suffering from Lyme disease, but the neurologist we were seeing did not (would not) pursue this. When he failed to ask for any Lyme work-up from the spinal tap or the biopsy, we changed neurologists. In late January we visited a Lyme disease specialist in New Jersey. The blood tests done were negative for Lyme except for some unaccounted for antibodies. In February 1995 Marjorie started a seven week course of Rocephin which failed to produce any positive results. Since the advancement of the disease quickened starting in late-May, early- June things have progressed rapidly. She will likely need the trach done in the next couple to three weeks. If Marjorie does not have the tracheostomy she will probably die from respatory failure in the next few months. Here is were we need some help and as soon as possible: 1. Is there anyone familiar with supporting a patient with a trach who is also as far along as Marjorie? What can we expect things to be like in the future? 2. I've heard that there is much discomfort with a trach. Is this so? Or does it become so? Or does it truly better the life of the patient. 3. I've heard that a feeding tube can only be in for a limited period of time. Is this the case? 4. While some patients can effect talking by controling the venting of the air flow, Marjorie will not be able to do this. Is there any other chance of talking, or at least communicating while the trach is in place? 5. Since ALS effects the voluntary muscles, are there any muscles or muscle groups that are spared, such as neck or facial muscles. How about the eyes or eyebrows? Are there other ways of indicating "yes" or "no"? 6. How have patients with advanced ALS done while on a trach? 7. If all muscles fail, how can an advanced patient let you know they have decided it's time to turn off the machines? Marjorie is 52 years old. Her birthday is at the end of November. We have two children -- a 17 year son, a senior in high school; an 11 year old daughter in sixth grade. We've been married 22 years. I hope some folks out there can offer some helpful advise to the questions above, and to those questions Marjorie and I may not have thought of yet. Thanks for any help you can give us. dick.tauber@turner.com (4) ===== Questions on throat irritation ========== Date : Fri, 1 Sep 1995 10:01:55 -0600 (CST) >From : "Kathy Martin" Subject: Questions on throat irritation I want to thank you for your wonderful service in providing the ALS Digest. It is a tremendous source of information and encouragement to me as I search for help for my 77-year-old mother who was diagnosed with bulbar-onset ALS in Oct. 94. She had a G-tube put in in May and a tracheostomy in July. So it's been a rough summer for her, but happily, she has adjusted by now to both and is doing better. I would be interested in corresponding with anyone who has information and experience in managing a trach with ALS. Coughing spasms seem to be her biggest problem right now, and also accumulation of mucuous and secretions in her mouth and throat. She uses the suction machine to remove excess throughout the day and night, but she still copes with a chronic problem of a prickling irritation at the back of her throat which initiates a coughing spasm. And this much coughing causes soreness around her G-tube. Cough medicines by G-tube may help some, but not much. She can't take anything by mouth. We would appreciate any suggestions or advice from others for these problems. Again, my deep apppreciation for your work with the ALS Digest in memory of your friend, Carmen Avila. Sincerely, Kathy Martin kcmartin@uiuc.edu University of Illinois Office of Women in International Development 320 International Studies Building 910 South Fifth Street Champaign, IL 61820 Phone: 217-333-1994 Fax: 217-333-6270 (5) ===== Riluzole ========== PR NEWSWIRE (Friday August 25, 1995) 1,000 NEW PEOPLE WITH ALS SELECTED TO RECEIVE RILUTEK(R) (RILUZOLE) VIA EARLY ACCESS PROGRAM; PROGRAM DOUBLES NUMBER OF PARTICIPATING CENTERS NEW FAIRFIELD, Conn., Aug. 25 /PRNewswire/ -- The National Organization for Rare Disorders (NORD) announced today that an additional 1,000 people with amyotrophic lateral sclerosis (ALS), were selected to receive Rilutek(R) (riluzole) in the first ever early access program for ALS patients. A total of 2,250 people with ALS have now been selected to receive Rilutek free of charge prior to its commercial distribution in the United States. Rilutek is the first compound shown to extend survival in ALS patients. Due to the response from both the patient and medical community, the number of participating sites was increased from 66 to over 120 centers during the last four weeks. "Our top priority is to meet the needs of the ALS community," said Abbey S. Meyers, President of NORD. "In this case, we received a clear signal that there needed to be more centers, so we nearly doubled the number of centers, many of which are associated with the Muscular Dystrophy Association or ALS Association." "We are pleased with the manner in which NORD and RPR have accelerated the development and implementation of this access program," said Michael Havlicek, President and Chief Executive Officer of The ALS Association. "We hope that other companies will begin similar programs in the near future to help meet the needs of the ALS community." A Program Update Patients who were not selected on July 24th were automatically re-entered into the August 24 computerized random selection process supervised by NORD. Patients not selected in previous drawings, as well as newly diagnosed patients, may participate in a third selection scheduled for September 25th. Previously submitted enrollment forms will be automatically re-entered into the upcoming selection. First time callers may obtain an official enrollment package by calling 1-800 RX TRIAL (1-800-798-7425). How the Program Works Patients are selected from a computerized random selection process administered by NORD. All participants are notified by mail if they have been selected or if their name will be made available for the next random selection process. Selected patients must have a confirmed ALS diagnosis from a designated Diagnostic Confirmation Center (DCC). Participating centers will be available to assist selected patients in cities throughout the U.S. and Puerto Rico. The New Drug Application (NDA) for Rilutek was submitted to the Food and Drug Administration (FDA) on June 29, 1995. An advisory committee to the FDA is scheduled to review Rilutek on September 18th. ALS is a fatal neuromuscular disease affecting approximately 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. NORD is a federation of voluntary health organizations dedicated to serving people with rare (orphan) diseases and assisting the organizations that serve them. NORD is committed to the identification, treatment and cure of rare disorders through programs of education, advocacy, research and service. CONTACT: Abbey S. Meyers of the National Organization for Rare Disorders, 203-746-6518 (6) ===== CLINICAL-TRIAL-FINDER list started ========== Date : Mon, 28 Aug 1995 15:55:40 -0400 >From : Marshall Kragen Subject: CLINICAL-TRIAL-FINDER list started I am pleased to state that the Clinical-Trial-Finder mailing list is now live. The purpose of this list is to help those seeking clinical trials for any problem to communicate with hospitals and other medical providers offering the same. The list will be moderated and I will be the moderator, initially. Medical facilities are urged to subscribe to the list and post detailed descriptions of trials they are conducting or intend to conduct. The more details given the better. Also, the trial's approval by an appropriate authority should be shown. Patients or caretakers should post as much information as they are comfortable describing as to medical problems for which they are seeking a medical trial. Discussion in general of trials and medical problems should not be posted to the list. The idea is to keep the bandwidth at a minimum so that all interested will keep watch for postings without feeling overburdened by too much mail. The postings will be moderated and I will also attempt to post back to the list trials already submitted matching later posts by patients. Once a trial is completed or closed I should be notified so it can be removed from the archive. Later it is hoped to have the postings also put into a companion Web page. Many thanks are given to my employer Garcia Consulting, Inc., an Internet service provider, for making this list available via their facilities. To subscribe send to listserv@garcia.com the message subscribe ctf **************************************************************************** Marshall Kragen /\ Internet liaison for National Coalition mkragen@access.digex.net \/ for Cancer Survivorship (NCCS), 1010 Wayne gwrepmk@aol.com \/ Avenue, Silver Spring, MD 20910 (301)650-8868 (7) ===== Listing of clinical trials on the Internet ========== Date : Mon, 28 Aug 1995 15:48:50 -0400 >From : Rwhitak@aol.com Subject: Listing of clinical trials on the Internet Recently there was a discussion on this board about the need for a dedicated site on the Web that would provide a comprehensive, nationwide listing of clinical trials. I am currently involved with the development of such a site through my company CenterWatch (we publish a newsletter that covers the clinical trials industry.) This site is being established as a dedicated forum for this purpose. It has been organized so that patients will be able to quickly search for trials by therapeutic area and by geographical region. The Internet address is: http:\\www.centerwatch.com, and will be up and running on September 15. Sponsors, CROs and researchers may post trials on this site. We also have designed this service so that research centers, if they so choose, may place their own pages on this site. Once a patient finds a trial of interest, the patient will be directed to the center's own page. The patient will be able to send a confidential E-mail message directly to the research center from this page. Anyone interested in posting trials on this CenterWatch site, or to learn more about it, can contact me, Robert Whitaker, at: CenterWatch, 617-247-2493. (Or at Rwhitak@aol.com). We'll send you information about how the site is organized and a form for posting trials, which can then be sent to us by E-mail or by fax. (617-247-2535). _________________________________________________________________________ Distributed by: The Clinical Trials Mailing List Paul Bleicher, MD PhD (Moderator) Voice: 508 798-6900 E-Mail: bleicher@world.std.com Send Submissions to: clinical_trials@world.std.com ALL POSTINGS MUST HAVE YOUR REAL NAME AND AFFILIATION Subscribe by sending a message to: majordomo@world.std.com body of message: subscribe clinical_trials your@email.addr Unsubscribe by sending a message to: majordomo@world.std.com body of message: unsubscribe clinical_trials your@email.addr ________________________________________________________________________ === end of als 212 ===