MDA/MDAC GROUP FORMALIZES ALS AGENDA
The newly formed MDA/MDAC Physicians' Group on ALS, created jointly by MDA and the Muscular Dystrophy Association of Canada, as a follow-up to its initial meeting in May (1996), has issued the following official statement of its goals:
MDA and MDAC have organized the MDA/MDAC Physicians' Group on ALS to provide advocacy on behalf of patients to accelerate progress in clinical trials of experimental drugs and to provide impartiality in the analysis and reporting of clinical trial results.
The mission of the MDA/MDAC Physicians' Group on ALS is to promote and facilitate efforts of drug companies and independent investigators to advance testing of experimental drugs in the treatment of ALS, to provide an objective forum from which patients and the public may obtain information about the likely or proven efficacy of experimental drugs in the treatment of ALS, and to foster cooperation between otherwise competing companies in testing the efficacy of experimental drugs in combination as treatments for ALS.
The first objectives of the MDA/MDAC Physicians' Group on ALS are:
* To improve understanding by patients of what constitutes clinically significant improvement demonstrated by a clinical trial by providing unambiguous standards by which to evaluate claims of clinical efficacy.
* To expand new treatment initiatives by providing independent investigators with resources to conduct pilot studies of experimental drugs previously untested as possible treatments for ALS.
* To accelerate cooperation between drug companies in the testing of proprietary drugs by providing an objective and impartial oversight body for the evaluation and reporting of clinical results.
* To deepen understanding by the public of the need for expanded support of ALS clinical trials by providing an impartial and knowledgeable resource for the assessment of progress and the cost in human suffering of delayed action in the effort to develop an effective treatment for ALS.
Noted ALS experts who belong to the Physicians' Group and who are currently working to achieve its goals include Chairman Donald Wood, MDA director of science technology; Dr. Michael Brooke, president of the MDAC; Dr. Leon Charash, chairman of the MDA Medical Advisory Committee; Dr. Rodney Howell, chairman of the MDA Scientific Advisory Committee; Dr. Robert Brown of Massachusetts General Hospital in Boston; Dr. Hiroshi Mitsumoto of the Cleveland Clinic Foundation; Dr. Steven Ringel of the University of Colorado Health Sciences Center in Denver; Dr. Jeffrey Rothstein of Johns Hopkins University in Baltimore; and Dr. Teepu Siddique of Northwestern University Medical School in Chicago.
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 FDA APPROVES EARLY ACCESS PROGRAM FOR GROWTH FACTOR DRUG MYOTROPHIN
On June 24 (1966), the U.S. Food and Drug Administration granted Treatment Investigational New Drug (T-IND) status and an early access program for the ALS drug Myotrophin.
Myotrophin was developed by Cephalon (West Chester, Pa.) and Chiron (Emeryville, Calif.). The drug is derived from the natural substance insulin-like growth factor 1, or IGF-1, and belongs to a biochemical group known as neurotrophic factors, which appear to protect nerve cells from damage in a number of adverse conditions. It has to be injected.
T-IND status allows a limited number of patients access to a drug, without charge, prior to its full approval by the FDA. A drug with a T-IND is usually distributed by random selection. Rilutek, fully approved as an ALS treatment by the FDA in December, was granted a T-IND prior to its full approval. On June 7, a panel of experts convened by the FDA unanimously recommended T-IND status for Myotrophin. However, a cautionary note was sounded by most of the panel members, who agreed with the FDA's own assessment of the drug companies' two clinical trials, one conducted in North America and the other in Europe. The FDA said one of the two trials failed to show that Myotrophin works. (In addition, industry analysts have voiced serious safety concerns about Myotrophin.)
Panel members strongly urged the companies to conduct a third clinical trial before seeking full approval for Myotrophin. Cephalon spokesperson Kori Beer said the companies will seek approval this summer on the basis of the two completed studies.
"For ethical reasons, it's difficult to envision another phase 3, placebo-controlled trial," Beer said. She said it would be difficult to put patients in a placebo-controlled trial while Myotrophin is available through the early access program.
Beer said Cephalon will conduct the early access program through a computerized, random selection process. The program will be administered through NORD, the National Organization for Rare Disorders, which administered Rilutek's early access program.
Although they haven't provided an estimate of the number of patients who will be able to get Myotrophin through an early access program, Cephalon officials have said that the amount of Myotrophin available will be small.
The company encourages people to keep in touch with Myotrophin developments through Cephalon's Myotrophin Information Line at (800) 797-0705. The company plans to contact people who leave their names and addresses with this information line as Cephalon gets ready for the early access program.
MDA has supported research in neurotrophic factors for several years, and MDA clinics were six of the eight North American sites used for the Myotrophin clinical trials.
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MDA FORUM OFFERS ONLINE CONFERENCE ON VENTILATORS
An expert on use of ventilation in ALS will offer authoritative advice during MDA's Online Ventilator/ Respiratory Conference on Aug. 20 (1966).
The interactive conference will be held on the MDA Forum on CompuServe at 8 p.m. EDT on Tuesday, Aug. 20.
Pamela Cazzolli, R.N., of Canton, Ohio, an ALS home care consultant for 12 years, will be the primary participant. Cazzolli, who has worked with nearly 1,500 people with ALS, specializes in respiratory care and mechanical ventilation, and provides educational programs on managing ALS and other neuromuscular diseases.
The conference will be moderated by Lori Hinderer of St. Louis, an MDA vice president who has had a tracheostomy and used full- time ventilation for six years. Hinderer is an MDA Forum systems operator and specializes in domestic and international air travel information for ventilator users.
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HELP FIGHT ALS TODAY AND TOMORROW
Many people who know the devastating effects of ALS are providing lasting support for MDA's battle against the disease. Through your will, you can designate a gift to MDA earmarked to support ALS research or services.
Bequests to MDA can be made with cash, securities, real estate, or other property. You can bequeath a percentage of the entire estate to MDA or make a bequest of the residue, donating property remaining after all bequests to family and others have been satisfied. You may also name a memorial gift in honor of a family or individual.
To give what remains of your estate after other bequests have been satisfied, just include the following language in your will:
"I give, devise and bequeath all (or a specified fraction of) the rest, residue and remainder of my estate, whether real or personal, of every kind and description, and wherever situated, to Muscular Dystrophy Association Inc., a New York not-for-profit corporation having its principal office at 3300 East Sunrise Drive, Tucson, Arizona, 85718-3208, for its program of research and services related to amyotrophic lateral sclerosis."
To give a dollar amount or percentage of your estate:
"I give, devise and bequeath the sum of $________ (or ________ percent of my estate) to Muscular Dystrophy Association Inc., a New York not-for-profit corporation having its principal office at 3300 East Sunrise Drive, Tucson, Arizona, 85718-3208, for its program of research and services related to amyotrophic lateral sclerosis."
Your attorney or financial adviser can help you work out the details of a bequest to MDA's ALS program. For more information, call MDA's Planned Giving Department at (800) 572-1717.
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REEDY SHARES TIPS ON ADJUSTING TO ALS
Use your head... Make life adjustments... Then move forward. Those are the ideas that characterize Joel Reedy's view of living with ALS.
The 47-year-old marketing instructor at the University of South Florida has been moving forward with his life since his ALS diagnosis 16 years ago. He now has no use of his limbs and relies on full-time ventilation assistance. Yet his description of the disease is understated: It's been quite an inconvenience.
Reedy, of Tampa, Fla., is sharing his thoughts on living with ALS in a book he's writing called Living with Chronic Illness: A Solutions Guide for Managing Chronic Health Conditions. He believes that, with the right attitude, people can often live for years and continue to do business or teach or do research or basically stay contributing and earn their own keep, despite life-threatening medical conditions. The observations in the book are based on his own experiences and interviews with others who've had success in life while dealing with long-term health problems.
The secret to making the most of ALS or other serious medical disorders, Reedy said, is, "Using your head to think through changes and figuring out alternatives and picking the best solution. What has worked for me is, I'm the boss and It's up to me to fix myself. Nobody else can do it for you."
Reedy has mastered many personal and professional adjustments. He worked for 20 years in advertising agencies until the late 1980s when mobility became more difficult. He earned a master's of business administration degree in 1990 and got a college teaching position. He's also a consultant for businesses and government agencies that want to market products and services to consumers with disabilities.
There have been adjustments in his family life, too. His daughter, Chanel, was 4 and his son, Chandler, an infant when their dad received his ALS diagnosis. They've become high-achieving students while seeing the changes their father has faced.
"I'm proud of them, and evidently they do have some inner strength. They've learned that things aren't always easy and you have to work around it," he said. Reedy's wife, Shirley, is an elementary school teacher.
To Reedy, the progressive nature of ALS has to be taken in stride. "I think in life we're dealt a set of cards and we've got to play them as best we can. I'm just trying to get through life as positively as possible. I really don't think of myself as doing anything unusual. I try to be the best teacher, I try to be the best advertising person, I try to be the best volunteer that my abilities allow."
Reedy has received several teaching awards and been nominated as Volunteer of the Year by the Tampa Mayor's Alliance for Persons with Disabilities. He's also active with MDA, appearing on the local Telethon broadcast and earning the Association's Central Florida Personal Achievement Award last year.
In 1993, his first book, Marketing to Consumers with Disabilities, was published by Probus Publishing. Reedy had hoped that passage of the Americans with Disabilities Act would generate attention from marketers of various products, but has found change to be slow.
"I was hoping that more marketers would embrace people with disabilities as a market potential and make special efforts, or at least make their messages or their copy or graphics more sensitive to their conditions to woo them better," he said.
"But from what I read, corporations are still waiting to see what the court decisions are going to be, how hard they're really going to have to work to accommodate. Many times it takes almost a generation to readapt or alter their perceptions and their attitudes toward things."
Reedy uses voice-activated computer software to dictate his writing and interface with the Internet, as well as E-mail and other electronic communication assistance.
He participated in a trial of Rilutek for about six months but discontinued using it because of skin problems and other reactions.
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DIRECT SPINAL FLUID DELIVERY OF CNTF SHOWS PROMISE
CytoTherapeutics (Providence, R.I.) has been testing a new way to deliver neurotrophic factors for ALS which the company hopes will maximize effectiveness while minimizing side effects.
As reported in the April issue of The ALS Newsletter (Vol. 1, No. 4), the company has been conducting a small trial in Switzerland in which 10 patients have been given hamster kidney cells that make ciliary neurotrophic factor (CNTF). The cells are encapsulated in a membrane and implanted at the base of the patient's spinal cord.
In the June issue of Nature Medicine, CytoTherapeutics reported that six patients (three more than previous reports indicated) have been studied at either 14 weeks or 17 weeks after receiving their CNTF implants.
At the time they were studied, all six had CNTF in their spinal fluid and no CNTF in their blood, which was the desired result. Two patients experienced a cough as a possible side effect of CNTF. Serious side effects didn't occur.
The treatment didn't have any effect on disease progression as measured by a standardized rating scale, but effectiveness wasn't expected during this trial.
Two earlier trials of CNTF given by injection under the skin showed severe side effects and no effect on disease progression. A lack of CNTF in the central nervous system and too much CNTF in other tissues may have contributed to these negative results.
Future applications of the delivery method may include using cells that are genetically engineered to continuously produce more than one neurotrophic factor.
In the June issue of Nature Medicine, the researchers wrote: "The encapsulated, cell-based approach has the potential of overcoming many of the problems associated with systemic administration of single or combination growth factor therapies. Encapsulated cell therapy may demonstrate unique advantages for the continuous delivery of pharmacological levels of bioactive proteins across the bloodbrain barrier."
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MDA/ALS ON WORLD WIDE WEB
For Internet users who have ALS, there's an excellent online source of information about positive approaches to living with the disease. It's the MDA/ALS Clinic World Wide Web Site maintained by health professionals at the MDA/ALS Research and Clinical Center at Baylor College of Medicine in Houston.
The MDA/ALS Clinic World Wide Web Site contains information about the various kinds of specialists who assist people with ALS at the Baylor center. The site also features a news section and an area where special publications about many aspects of ALS can be ordered. (See Vol. 1, No. 1, of The ALS Newsletter for a list of these publications.)
The site also enables visitors to leave questions about ALS via E-mail to be answered by the Baylor staff. The Baylor center, one of eight such MDA/ALS centers at major medical institutions across the country, operates under the direction of MDA-funded researcher and clinician Dr. Stanley H. Appel.
The web address for the center at Baylor is http://www.bcm.tmc.edu/neurol/struct/als/als1.html
MDA's own official World Wide Web address is http://www.mda.org.
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THE ALS NEWSLETTER
Muscular Dystrophy Association
National Headquarters
3300 East Sunrise Drive
Tucson, Arizona 85718-3208
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