Date: Mon, 12 Jun 95 11:38:24 -0400 From: Bob Broedel To: als@huey.met.fsu.edu Subject: ALSD198 ALS-ON-LINE =============================================================== == == == ----------- ALS Interest Group ----------- == == ALS Digest (#198, 12 June 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 == == 730+ 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 .. Speech Assist 2 .. ALS/MND publications, 1992-1995 3 .. Myotrophin (1) ===== Speech Assist ========== Date : Thu, 8 Jun 1995 01:31:18 -0400 >From : DanB5974@aol.com Subject: Speech Assist This is a report on my experience on speech assist equipment, for the ALS Digest and for others. I first tried setting up the Words+ equipment. That company was the one my friend Robert wanted to go to, because their equipment was used by Steve Hawking in his video. The software was Scanning WSKY, which was said to be more advanced than what was used in the video. For the hardware end of the deal, instead of DECTalk, I chose Vocalite, which cost less and I felt (correctly) that the vocal quality was adequate. Their Scanning WSKY was a DOS program, very rigid and inflexible. (Quite frankly, they did a horrible job on it. Additionally, their documentation was next to unuseable; this spoken as a tech. writer.) My friend Robert would have been controlling it with an eyebrow switch. The way Scanning was set up would have been much too difficult for controlling with the eyebrow switch, in my book. Further, the Vocalite (and DECTalk) is an external thing, not internal to the PC. It contains a speaker, although it has a micro headphone jack that one could plug in a computer or other speaker. The speaker being built into it made it really a bad deal for Robert. There are surely others in the same situation: in a nursing home, with very limited (shared hospital room-sized) space. The computer and equipment could not be placed at bedside. The Words+ people were wonderful to work with, but the software and equipment wasn't. Unfortunately, their software could not be utilized with a PC soundcard. (They are supposed to release a Windows version of their software in June; that's got to be so very much more flexible and usable. So I looked on the ALS society's list of resources, and saw MicroSystems Inc in Mass which sounded good and is good. They do have both DOS and Windows programs, that are much more flexible and usable, plus a magnifier program which will magnify what you see on the screen. I very highly recommend them. Their software is HandiKey, with other useful auxillary stuff. The Windows version worked particularly well, and could be stretched to display large on the screen. It was very highly configurable; choose number of matrix columns and rows to have, edit cells to contain messages, etc. MicroSystems makes software only. You have to get the switch or control elsewhere. Although the software is designed mainly for DECTalk, they don't sell the DECTalk hardware. With our needing to use a PC Soundblaster card instead of external, HandiKey was the choice. However, there are snags here. Neither MicroSystems nor Creative Labs (Soundblaster) had it all together; it took many phone calls, faxes, weeks, and hours to get it all together. Now I know, and now you can know. To work with a Soundblaster card, their software needs a Monologue for Windows program. Monologue didn't come with the soundcard in my PC, so I called Creative Labs. The sad part of it is, they thought they didn't even have it. Only after looking at various Soundblaster boxes did I see that Monologue for Windows comes with most Soundblaster cards, even the $99 one. The people running Creative's forum on Compuserve didn't even know that. So - Microsystem's software, plus any Soundblaster card whose box says it has Monolog, and a switch or control from Words+ or elsewhere, provides the ability to compose and talk. One more piece to mention. The higher level Soundblaster cards (professional, AWE32 Value, or SCSI) also have Text'Ole and Text Assist. Take note: this is the same stuff that DECTalk has; Creative Labs bought or licensed the rights. The ability to play a (Windows notebook) text file to you. Words don't come out always perfect. The benefit of this is that a patient who doesn't have the ability to hold reading material can listen (over and over) to a letter someone has typed on floppy, to articles downloaded from Internet services, to the ALS digest, or to computerized scriptures. Contacts: MicroSystems, 1-800-828-2600, 600 Worcester Road, Framingham, MA 01701. They will send a Demo disk. Words+: 800-869-8521; 40015 Sierra Hwy, Bldg B-145, Palmdale CA 93550. They will send a loaner system. Must mention another company that I never made connection with enough to really try out: ASYST, in Illinois, 708-816-8580. My impression was that their software was the same vintage as Words+ DOS Scanning WSKY. The system didn't get in place in time to help Robert. He deteriorated too far. He will soon be looking for that situation spoken of in the scriptures where there is no more sickness, bodies are restored, etc. He has made descisions and we have said goodbye. (2) ===== ALS/MND publications, 1992-1995 ========== Date : Fri, 09 Jun 1995 10:24:03 >From : Stuart.Neilson@brunel.ac.uk (hssrsdn) Subject: ALS/MND publications, 1992-1995 Dear Members, I have just updated the CSHSD bibliography of articles on MND/ALS, which are available from the WWW archive at the address below. Due to the number of entries (about 500 per year) they are stored in several files: Comprehensive, annual lists contain ALL papers that refer to ALS/MND in each year. These are MND92.TXT, MND93.TXT, MND94.TXT and MND95.TXT. Each file is around 100 Kbytes in plain ASCII format. Papers relating to epidemiology and some general issues are in PAP-MND.HTM (a hypertext markup language file) - this includes selected papers over the last several decades and represents a broad overview of research into MND/ALS. Any comments about the files, questions about access or requests for an EMail copy of any of the files are welcome to: Stuart Neilson (stuart.neilson@brunel.ac.uk) http://http1.brunel.ac.uk:8080/~hssrsdn/ (home page) http://http1.brunel.ac.uk:8080/~hssrsdn/alsig/als_rsrc.htm (ALS resources) (3) ===== Myotrophin ========== CEPHALON REPORTS MYOTROPHIN (RHIGF-1) SLOWS PROGRESSION OF LOU GEHRIG'S DISEASE; CLINICAL STUDY DEMONSTRATES POSITIVE IMPACT ON DISABILITY WEST CHESTER, Pa., June 12 /PRNewswire/ -- Cephalon, Inc. (Nasdaq: CEPH) announced today that in a Phase III clinical study of 266 patients with amyotrophic lateral sclerosis (ALS, or Lou Gehrig's disease), patients who received Myotrophin(TM) (recombinant human insulin-like growth factor-1, or rhIGF-1) experienced less disease severity, slower progression of disease, and better functional ability compared to patients who received placebo. The effects from Myotrophin administration were dose-related and consistent across primary and secondary measures using intent-to-treat analysis. Demonstration of these effects did not require subgroup analysis. Benefits of Myotrophin were evident as early as the first few months of drug therapy. "This study demonstrated that IGF-1 had highly statistically significant effects on clinically important measurements for patients with ALS, for whom no approved therapies currently exist," s (???) P clinical research. "We are enormously gratified to see positive results in such a devastating disease." Dr. Murphy presented the findings on Saturday, June 10 at a session of the World Federation of Neurology Committee on Motor Neuron Diseases in Talloires, France. The double-blind Phase III study was conducted at eight medical centers in the United States and Canada to evaluate Myotrophin's potential to treat ALS. In this study, after an evaluation period of up to three months, eligible patients with ALS were randomized to receive placebo, 0.05 or 0.10 milligrams per kilogram per day of Myotrophin by subcutaneous injection for up to nine months. After nine months of therapy, patients who received 0.10 mg/kg per day of Myotrophin showed approximately 25 percent less deterioration than patients receiving placebo, based on their scores on the Appel ALS Rating Scale in analyses of both rate of change (p less than or equal to 0.01) and functional severity of disease (p less than or equal to 0.01). Positive effects were observed in the 0.05 mg/kg dose group without statistical significance. In both Myotrophin dose groups, patients experienced a slower decline in lifestyle, as measured by twenty-point changes on the Appel scale (Logrank analysis: p 0.002 for 0.10 mg/kg and p 0.1 for 0.05 mg/kg; Wilcoxon: p 0.001 for 0.10 mg/kg and p 0.2 for 0.05 mg/kg). The slower decline in lifestyle in patients receiving the 0.10 mg/kg dose was confirmed by an independent assessment of sickness and disability (p 0.01), compared to patients receiving placebo. "Review of the data at this stage indicates a significant treatment effect of IGF-1 on both the loss of strength and the patient's quality of life. This is a most important new development in the struggle to find effective treatment for ALS," said Theodore L. Munsat, M.D., professor of neurology and pharmacology at Tufts University School of Medicine and a member of the organizing committee for the Talloires meeting. Myotrophin was well-tolerated. Generally mild injection site inflammation, changes in hair growth or texture, sweating and mild knee pain were observed in all treatment groups but occurred more frequently in Myotrophin-treated patients. The Appel ALS Rating Scale is a validated index of disease severity developed in 1982 to provide a quantitative estimate of the clinical condition and disease progression of patients with ALS. It includes assessment of swallowing, speech and respiratory function, as well as muscle strength in upper and lower extremities, and function of upper and lower extremities. Each twenty-point increase in the Appel scale represents a major increase in a patient's disability or dysfunction. An Appel score of 30 points is considered normal for a healthy individual. A patient with ALS who can still function independently averages 52 points on the Appel scale; one who is still able to work but needs some assistance averages 75 points; one who usually can no longer work averages 99 points; one who is home-bound averages 119 points; and one who is completely bed- bound averages 135 points. On average, the group receiving placebo achieved a twenty-point deterioration by the sixth month of treatment, while the group receiving 0.10 mg/kg/day of Myotrophin never reached this threshold for the duration of the study. "We are excited about this accomplishment and grateful to the patients with ALS who courageously chose to participate in this landmark study," said Frank Baldino, Jr., Ph.D., president and CEO of Cephalon. "This is the first demonstration that the use of Myotrophin can alter the course of a neurodegenerative disease. These findings represent an important validation of the company's mission, and we are encouraged about future prospects for treating chronic neurodegenerative conditions." Cephalon is conducting a second Phase III clinical study comparing 0.10 mg/kg per day of Myotrophin to placebo in 183 patients with ALS (also called motor neurone disease, or MND) in Europe. That study is expected to conclude this summer. The company has also initiated a Phase II clinical program to evaluate Myotrophin's use in treating a variety of peripheral neuropathies, including chemotherapy-induced peripheral neuropathy, post-polio syndrome and diabetic neuropathy. Cephalon is developing Myotrophin in North America and Europe for use in ALS in collaboration with Chiron Corporation (Nasdaq: CHIR), and on behalf of Cephalon Clinical Partners, L.P. The limited partnership was formed in 1992 to fund development and clinical testing of Myotrophin. Cephalon is developing Myotrophin in Japan for use in ALS and other neurological disorders in collaboration with Kyowa Hakko Kogyo Co., Ltd. Cephalon will continue to provide its limited supply of Myotrophin on an open-label basis to all patients who have participated in the North American and European Phase III clinical trials, using drug supplied from its Beltsville, Maryland manufacturing facility. Cephalon and Chiron are committed to expanding patient access to Myotrophin by applying their combined resources and expertise to expand manufacturing capacity. In October 1991, the U.S. Food and Drug Administration designated Myotrophin an orphan drug for the treatment of ALS. Patients, caregivers and health care professionals may call 1-800-797-0705 in North America or 1-301-210-0393 from outside North America for more information about Myotrophin. Background on IGF-1 and ALS IGF-1 is a naturally occuring protein found in muscle and other tissue which mediates regeneration of the peripheral nervous system and its recovery from injury. In preclinical studies, IGF-1 has been shown to support the survival of motor neurons and accelerate the regeneration of damaged neurons. Preclinical studies have also shown that IGF-1 promotes sprouting and function of peripheral nerves, and induces skeletal muscle hypertrophy, or enlargement of muscle cells, in the presence of neurodegenerative conditions. Neuronal sprouting is the natural process by which neurons generate additional branches, enabling them to establish functional contacts with muscle fibers whose original nerve contacts have been lost as a result of neuronal death. These multiple actions of IGF-1 may explain the effects of Myotrophin demonstrated in this Phase III study. ALS is a fatal neuromuscular disease characterized by the chronic, progressive degeneration of motor neurons. The term "amyotrophic" refers to muscle wasting or atrophy; "Lateral sclerosis" refers to the scarring or degeneration of motor neurons which project from the lateral columns of the spinal cord to the muscle tissue. It is the loss of these motor neurons that leads to muscle weakness, muscle atrophy, and eventually death from respiratory failure. According to the ALS Association, approximately 5,000 people in the United States are diagnosed with ALS each year, and more than 30,000 Americans and more than one out of every 100,000 people worldwide have the disease at any given time. The disease usually afflicts men and women between the ages of 40 and 70. ALS was first described in 1869 by the French neurologist, Jean- Martin Charcot. There is currently no cure for the disease. Chiron Corporation, headquartered in Emeryville, CA applies biotechnology and other techniques of modern biology and chemistry to develop products intended to improve the quality of life by diagnosing, preventing and treating human disease. Separate from its collaboration with Cephalon, Chiron has been researching and developing IGF-1 for more than a decade, and currently is sponsoring a Phase II clinical trial studying IGF-1 in acute kidney failure. Cephalon discovers, develops and markets products to treat neurological disorders. The company's principal focus is on neurological disorders such as ALS, narcolepsy, peripheral neuropathies, Alzheimer's disease, head and spinal injury, and stroke. CONTACT: Jason Rubin or Mary Fisher of Cephalon, Inc., 610-344-0200, or Larry Kurtz of Chiron Corporation, 510-601-2476 === end of als 198 ===