WELCOME TO THE ALS NEWSLETTER
...from Robert Ross, Executive Director, MDA
With this newsletter, MDA inaugurates a new service for those affected by amyotrophic lateral sclerosis (ALS) and their families. It's fitting that we launch a new ALS newsletter in January 1996, the same month that pharmacies will, for the first time ever, have on their shelves a drug that slows the relentless progress of ALS (see story about Rilutek on this page).
The ALS Newsletter is designed to provide you with up-to-date, useful information about ALS research, MDA's extensive ALS service program and other relevant topics. As you may be aware, MDA has led the worldwide fight against ALS for more than four decades, providing the most comprehensive array of services for those with Lou Gehrig's disease of any voluntary health agency in the country, and investing more in ALS research than any U.S. organization outside the federal government.
The ALS Newsletter joins a variety of information services by which MDA keeps you up to date on new developments in treatment or understanding of the disease. We'll publish the newsletter every other month. In between those issues, when our rapidly progressing research program yields other significant findings, we'll immediately send you a special issue of the newsletter explaining how the new development affects you.
You can read additional in-depth stories about ALS support groups, research and other programs in MDA's quarterly magazine, Quest, which all those served by MDA receive at no cost. Other print materials include our updated "Facts about ALS" and "MDA: First in ALS Services and Research" brochures, as well as several books on living with ALS.
We've also produced a new video containing ALS news (see article below). If you're a CompuServe user, you probably know about the MDA Forum (type "GO MDA") with its popular "Ask the Experts" and "ALS Support" message sections. Our World Wide Web page will soon put MDA online with all Internet users, giving access to the MDA Forum and its MDA libraries.
I hope you'll find The ALS Newsletter helpful and timely. Please send your suggestions for topics you'd like to see covered to: Editor, The ALS Newsletter, MDA, 3300 East Sunrise Drive, Tucson, AZ 85718, e-mail MDA at 74431.2513@compuserve.com.
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 RILUTEK HITS PHARMACIES
At a Jan. 10 (1966) press conference in New York, the drug company Rhone-Poulenc Rorer (RPR) announced that its new ALS drug, Rilutek, is now available at pharmacies for those with a doctor's prescription. RPR is charging pharmacies $590 for a month's supply of Rilutek (at a recommended dose of 50 mg every 12 hours), but the company estimates consumers will pay $730 per month or more.
The drug's development was made possible by MDA-funded basic research into the nervous system chemical glutamate and by the participation of MDA clinics in Rilutek drug trials. These trials showed Rilutek extended survival an average of three months. Taking part in the news conference were Dr. Leon M. Charash, chairman of MDA's Medical Advisory Committee; MDA grantee Dr. Jeffrey Rothstein of Johns Hopkins University; Dr. Robert Miller, director of the Forbes Norris MDA/ALS Research Center at California Pacific Medical Center in San Francisco; and Jim Brown, MDA's director of Public Affairs.
"Rilutek is a step on the road to a more effective treatment or a cure. Continued MDA research is the key," Charash said.
RPR has set up three toll-free phone lines: general information about Rilutek, (800) RX-TRIAL; obtaining the drug or financial assistance, (800) 790-RTEC; Rilutek Early Access Phase- Out Program, (800) 459-7599.
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MDA CLINICS, PATIENTS PLAY ROLE IN DRUG DEVELOPMENT
What does it take to develop a drug for ALS?
In the case of Rilutek, the first ALS drug to receive Food and Drug Administration approval, it took a great investment of time and money by the global drug manufacturer Rhone-Poulenc Rorer (RPR). But it also required the cooperation of MDA-funded researchers, clinic directors and individuals with ALS who attend MDA clinics.
Drawing upon MDA-funded research that suggested that the nervous system chemical glutamate plays a role in the death of motor neurons in ALS, RPR developed a glutamate-inhibiting substance called riluzole under the trade name Rilutek.
Phase I clinical trials for Rilutek were begun in the late 1980s on healthy human subjects to see how the drug is used by the body. Phase II trials, conducted in Europe, tested the drug in patients with ALS for the first time. Results suggested that Rilutek could prolong survival in ALS, but more comprehensive data was needed.
Some 960 ALS patients participated in Phase III trials at 31 centers in seven countries, including five centers in the United States. All the U.S. sites were MDA clinical care centers: Baylor College of Medicine in Houston, California Pacific Medical Center in San Francisco, Johns Hopkins University in Baltimore, Northwestern University in Chicago and Tufts-New England Medical Center in Boston.
The data gathered from these trials, which compared patients on Rilutek with patients taking a placebo, allowed RPR to make a case to the FDA that Rilutek extended survival in ALS. Dr. Robert G. Miller, director of the MDA ALS Center in San Francisco that participated in the trials, testified at an FDA advisory hearing last September that Rilutek should be approved. On Dec. 12, 1995, RPR announced that it had received formal approval from the FDA to market Rilutek for treatment of ALS.
MDA also played an important role in Rilutek's development by allowing the use of more than 55 MDA clinics for RPR's early access program. This program allowed over 3,000 Americans to receive Rilutek at no charge through a random selection process prior to the drug's formal approval. RPR intends to keep the program running until the drug is commercially available.
"Our experience with Rilutek has been one that teaches the importance of building partnerships such as working with MDA," says Rob Partridge, senior manager of product communications at RPR.
MDA clinics have also served as testing sites for the neurotrophic factor IGF-1, an ALS drug being developed under the trade name Myotrophin by another company, Cephalon. Cephalon is waiting for approval from the FDA to administer an early access program for the drug and hopes to be ready to apply for marketing approval for Myotrophin in the early months of 1996. Cephalon's Myotrophin Product Information Line is (800) 797-0705.
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MDA SEEKS MORE ALS GENES
MDA has funded a three-year grant to researchers at three institutions to look for genes that may make people susceptible to ALS.
The lead investigators are Margaret Pericak-Vance at Duke University in Durham, N.C., Teepu Siddique at Northwestern University Medical School in Chicago, and Robert Brown and Jonathan Haines, both at Massachusetts General Hospital in Boston.
All four were MDA-supported members of a team that found the SOD1 gene for ALS in 1993. Only a small percentage of people with ALS have defects in their SOD1 genes, Pericak-Vance said. The rest of ALS is usually called "sporadic," or non-genetic, ALS. But this may not be true, Pericak-Vance said. There may be as-yet-unidentified defects in other genes that either directly cause the disease or predispose people to getting it.
When a gene doesn't directly cause a disease, it's hard to trace a family history, because only some family members with the gene defect will become ill, while others may have the defect without showing symptoms.
Genes that make people susceptible to a disease may need an environmental trigger to start the disease process, Pericak-Vance said.
The methods used by the multicenter team have already been used to successfully uncover genes involved in diabetes, Alzheimer's disease, high blood pressure and the eye condition macular degeneration. They're now being used to look at genes that may influence multiple sclerosis.
The SOD1 gene discovery opened a new line of research in ALS. The researchers hope similar gene discoveries will lead to other approaches to ALS treatment.
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MDA TAKES FAST LANE
Internet users who want information about ALS or want to gain access to the MDA Forum on CompuServe will soon be able to use MDA's World Wide Web page.
The Web site will eventually direct users to subjects such as ongoing ALS research; MDA/ALS support services; nationwide clinical care; MDA Forum on CompuServe; local MDA chapters and regional offices; and much more. If you're interested in traveling the information superhighway, take advantage of a one-month free trial membership with CompuServe and credits to sample the MDA Forum. More details about this free offer are available by calling CompuServe at (800) 621-1253, or go to CompuServe's Website, http://www.compuserve.com.
CompuServe's fixed monthly fee was recently lowered to $9.95, with the first five hours of any connect time (such as Forum or e-mail) free of charge.
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NEW MDA/ALS VIDEO COMING SOON
MDA has produced a videotape, "With Strength and Courage: Understanding and Living with ALS.
" This 24-minute video gives an overview of the disease and touches on everything from recent research advances to aids for daily living. Actor Ed Fry, chairperson of MDA's ALS Division, is the video's host, and Dr. Stanley H. Appel offers information about possible causes of and potential treatments for the disease. The video was shot at Appel's MDA/ALS Research and Clinical Center at Baylor College of Medicine in Houston, and is geared primarily for newly identified patients.
The drug company Rhone-Poulenc Rorer, maker of Rilutek, provided financial assistance to make the video. "With Strength and Courage" will be made available Feb. 1 through your local MDA office for viewing on an individual basis or in support groups.
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PUBLICATIONS OFFER HELP WITH ALS
A new edition of MDA's "First in ALS Services and Research" is now available. The eight-page brochure describes the Association's $5 million-a-year ALS program, briefly summarizes current avenues of research and outlines MDA's services for those affected by ALS. The brochure contains a list of some two dozen institutions where MDA is currently funding ALS research.
For free brochures and pamphlets about ALS, contact your local MDA office or call the Program Services Department at MDA National Headquarters, (800) 572-1717, or e-mail MDA at 74431.2513@compuserve.com giving your name and land address. Ask for these titles:
* "MDA - First in ALS Services and Research" *
"Facts About Amyotrophic Lateral Sclerosis"
Other publications, prepared by the MDA/ALS Center medical team at Baylor College of Medicine in Houston (some at nominal charge), are also available at local MDA offices. They are:
* "Amyotrophic Lateral Sclerosis" This (free) 28-page publication offers detailed information about ALS, methods of diagnosis and medical management, as well as current research strategies aimed at finding an effective treatment.
* "ALS: Maintaining Mobility" This 149-page book was written specifically for individuals with ALS to assist in maintaining mobility, prolonging muscle function and enhancing independence. Cost: $6.00.
* "Meals for Easy Swallowing" This 125-page book is useful to people with ALS, who often have swallowing difficulties. It provides a collection of recipes for easy-to-swallow foods and beverages, as well as suggestions on food preparation and service. Cost: $6.00.
* "ALS: Maintaining
Nutrition" This 130-page book was designed primarily for use by health professionals who care for those affected by ALS. Topics include swallowing, diet, alternative feeding methods and tube feeding. Cost: $6.00.
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MDA FUNDS 8 ALS CENTERS
In addition to its 230 comprehensive neuromuscular disease clinics, MDA funds eight centers dedicated to research and medical care in ALS. The centers and their directors are:
BOSTON: MDA/ALS RESEARCH AND TREATMENT CENTER (617) 636-5855 Theodore L. Munsat, M.D. New England Medical Center Hospitals Neuromuscular Research Unit 750 Washington Street, Box 273 Boston, MA 02111
CHICAGO: THE MDA/ALS CENTER UNIVERSITY OF CHICAGO (312) 702-6390 Raymond Roos, M.D. Department of Neurology/MC2030 5841 South Maryland Avenue Chicago, IL 60637
HOUSTON: MDA/ALS RESEARCH AND CLINICAL CENTER (713) 798-4072 Stanley H. Appel, M.D. Baylor College of Medicine Neurosensory Center 6501 Fannin Street, NB302 Houston, TX 77030
LOS ANGELES: JERRY LEWIS MDA/ALS CLINICAL AND RESEARCH CENTER (213) 743-1611 W. King Engel, M.D. University of Southern California School of Medicine 637 South Lucas Avenue Los Angeles CA 90017
MDA/ALS CENTER AT UCLA (310) 825-7266 Michael C. Graves, M.D. UCLA Neurological Services, Suite B200 300 UCLA Medical Plaza Los Angeles, CA 90024-6975
NEW YORK: THE ELEANOR AND LOU GEHRIG MDA/ALS CENTER (212) 305-8551 Lewis P. Rowland, M.D. College of Physicians & Surgeons of Columbia University Neurological Institute Department of Neurology 710 West 168th Street Room 1406, 14th Floor New York, NY 10032-3784
MADISON, WI: MDA/ALS MIDWEST REGIONAL RESEARCH PROGRAM (608) 263-9057 Benjamin R. Brooks, M.D. University of Wisconsin-Madison J6/504 Clinical Science Center 600 Highland Avenue Madison, WI 53792-5132
SAN FRANCISCO: FORBES NORRIS MDA/ALS RESEARCH CENTER (415) 923-3604 Robert G. Miller, M.D. California Pacific Medical Center 2324 Sacramento Street, Suite 150 San Francisco, CA 94115
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THE ALS NEWSLETTER
Muscular Dystrophy Association
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Tucson, Arizona 85718-3208
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