=============================================================== == == == ----------- ALS INTEREST GROUP ----------- == == ALS Digest (#66, 10 November 1993) == == == == ----- 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. Currently there are == == 160+ subscribers. == == == == To subscribe, to unsubscribe, to contribute notes, == == etc. to ALS Digest, please send e-mail to: == == bro@huey.met.fsu.edu (Bob Broedel) == == == == All interested people may "broadcast" messages to == == ALS Digest subscribers by sending to: == == als@huey.met.fsu.edu == == == == Bob Broedel; P.O. Box 20049; Tallahassee, FL 32310 USA == =============================================================== == Back issues are available via e-mail from: == == LISTSERV@mailer.fsu.edu == == send an e-mail message that says INDEX ALS == == Also available via anonymous FTP at mailer.fsu.edu == == directory ftp/pub/als == =============================================================== (1) ===== INTRODUCTION - IAN PHILLIPS ========== >From: ian FRIENDS.IN.TIME phillips Date : Sat, 30 Oct 1993 20:04:35 -0600 (MDT) My name is Ian Phillips, am near 43, and have had ALS for three years. I grew up in Albuquerque, went to the University of Iowa on a tennis scholarship, and worked as a tennis teaching professional and university coach at different towns around the country. Since I was diagnosed I have tried many things including: spiritual surgery in Brazil, replacing my silver fillings, acupuncture, Reike, Ayerveda (East Indian medicine), psychic healers, faith healers, and many other things. Right now I am trying apitherapy (bee stings), meditation, and Reike. I think something is helping because my breathing has improved slightly since July when a Neurologist told my mother I wouldn't live til September. That is when I began researching home care for people on a vent. Here in New Mexico medicaid won't cover any home care for people on a vent, but other states have good coverage. Washington is good, Colorado sounds good from what their medicaid office said, and I think Oregon is pretty good. I believe that ALS can be beat, even though I haven't won many battles. There are two people I have spoken with who have recovered from ALS, and I have heard of two others. One I spoke with is a surgeon in Australia and the other is the woman who was cured by the psychic Dean Kraft as seen on national TV. The surgeon is a very positive, enthusiastic, and caring man. He says that a change in his thinking was the key to his recovery. There is also a woman here in Albuquerque who was diagnosed six years ago and she is in near normal health. Plus we have two men who have had ALS more than twelve years and both are doing well (by ALS standards). I like to pass along this information because it seems most are told there is no hope. Ian Phillips Albuquerque (2) ===== ALS CareGiver ========== Date : 09 Nov 1993 16:44:53 -0400 (EDT) >From : JDEMURO@CENTER.COLGATE.EDU Subject: ALS caregiver I took care of my mother for the last nine months of her life. She died March 92. I kept her at my home. It was difficult. I wish I could have had a support group at that time. Hospice people helped but were not familiar enough with the disease to give much support in the emotional area or what to expect. If I can be of help to anyone, feel free to write. My e-mail address is: jdemuro@center.colgate.edu My mother was 73 at the time of her death. We had great difficulty trying to get a diagnosis when my mother became ill approximately 2 years before her death. She even underwent open-heart surgery thinking that was the reason she was having difficulty walking. Judy DeMuro, RD 2 Box 120B, Earlville NY 13332 (3) ===== ALS Target Senator Harkin -- model letter -- ========== X-Mailer: America Online Mailer Sender : "jackn74940" Date : Tue, 09 Nov 93 22:27:36 EST Please ask for assistance from Senator Harkin In getting NIH to change protocol testing from Placebos in terminally ALS to Parallel or varying drug doses as well as insisting drug companies like Regeneron provide compassionate access to drugs like CNTF & BDNF. Senator Harkin is the Chair of Appropriations for NIH Honorable Senator Tom Harkin, I am writing you because of your courageous stand against bureaucracy and open apathy that "our" NIH and FDA takes in dealing with the issuance of compassionate drugs. In October of 1992, I was diagnosed with ALS, a terminal illness that usually is fatal within 3 - 6 years. I am married and a father of three young daughters and although I reside in Minnesota, my brother Greg Norton is a resident of your State. I know he will be writing also. I know of your stand because of my research in trying to understand why drugs that have passed safety trials could not be given to terminally ill patients. The article in Science 11 December 1992, "Gene Therapy Harkin Seeks Compassionate Use of Unproven Treatments" was quite clear that your efforts were seen to be an affront to established methodologies. You were 100% correct in your efforts. Why in diseases that are terminal must there be Placebo testing? In letters that I have read from others that had written their congressmen, the FDA and NIH are adamant about Placebos, the responsibility of the drug companies to issue the drugs and that all of this was mandated by Congress. What needs to be changed is the direction of health administration. We do not need the FDA or NIH to Protect to us to Death! Normally, safety trials only take a few months, efficacy trials can take years. For the terminally ill, that is a death sentence! You know it and so do thousands of others. The arrogance of this body of science is outrageous. They have lost their humanity. It is time to give the choice of access to drugs back to patients and their doctors. I submit to you the following items for action: 1) For drug trials involving the terminally ill, no placebos should be given - only differences in dose levels. If the trials need to run for more than a year so be it. The drug company should only be required to monitor a significant sample size. 2) The drug once it has passed safety trials - not efficacy - will be made available to those terminally ill at their cost and sanctioned by the FDA for Health Insurance coverage. If manufacturing levels are low, then lotteries should be sponsored by the drug companies until manufacturing capacities are sufficient. Access to safe drugs should be the patient's choice not a researcher's. Effectiveness will be measured by those who are still around to Vote! We are now waging the same battle all over again with the FDA, NIH and the drug companies. The facade called Fast Track is broken! We are dying! Please call Leonard Schleifer, CEO Regeneron (914-347-7000) to urge him to make the drugs CNTF and BDNF available on a compassionate basis. You want this access to CNTF outside the parameters of the double-blind, placebo control trial through a Treatment IND, as per FDA regulation 21 CFR 312.34. Inform him you will be modifying the FDA/NIH regulations for the terminally ill. Also call Robert E. Cawthorn, CEO of Rhone-Poulenc Rorer to make Riluzole available (215-454-8000), it too has shown promise and has passed safety trials. No one drug may stop this disease. We pray God Speed for you in our hour of need. We must have action not in years or months, but in weeks and days! The Quest continues. John & Cindy Norton cc: The PROD! Jack Norton (4) ===== re: CNTF ========== >From : stuart@onyx.cuug.ab.ca (Stuart Lory) Subject: CNTF Date : Wed, 10 Nov 1993 00:24:21 -0700 (MST) Bob, I received the following information regarding CNTF, I am enclosing it for your consideration to use in a future ALS DIGEST. I'm sure somebody else out there would find it interesting to read. Regards, -- Stuart Lory stuart@onyx.cuug.ab.ca Calgary, Alberta, Canada. FAX: (403) 283-1376 INFORMATION CONCERNING THE CLINICAL TRIAL OF RECOMBINANT HUMAN CILIARY NEUROTROPHIC FACTOR (rhCNTF) FOR AMYOTROPHIC LATERAL SCLEROSIS WHAT IS rhCNTF? Neurotrophic factors are naturally occurring proteins that promote the survival and functional activities of nerve cells. Ciliary neurotrophic factor, or CNTF, is one specific type of neurotrophic factor. Although its exact role is not yet known, CNTF appears to play a part in protecting nerve cells from damage when the nervous system is injured. Neurotrophic factors have been shown to protect nerve cells from experimental damage. We think then that neurotrophic factors like CNTF may help to protect nerve cells that become sick and die in progressive diseases like amyotrophic lateral sclerosis. HOW WILL CNTF BE STUDIED? The clinical trial of CNTF will be a phase II, prospective, placebo-controlled, double-blind study. This means: Phase II: The stage of a study where we try to establish efficacy or effectiveness of a test agent (ie CNTF) on a disease. Prospective: A study when the results are examined from a point in time forward to a point in time in the future. This is different from a retrospective study, which looks at results from a past population. Placebo controlled: A study where a placebo, or inactive substance, is give to one group of patients and the drug being tested is given to the other group of patients. We compare the results of the two groups. Double-blind: Neither the investigator or the patient knows whether the patient is taking the test drug or placebo. WHO WILL BE ELIGIBLE FOR THE STUDY? 1. Patients who have ALS. 2. Males or females between the ages of 21-85 years. 3. A patient whose general physical condition is acceptable to the investigator, and who is expected to survive for at least 12 months. 4. Patients who have understood, signed and given informed consent. WHO IS NOT ELIGIBLE FOR THE STUDY? 1. Patients with familial ALS or "pure" motor system diseases other than ALS. 2. Patients who are quadriplegic. 3. Patients who are in a condition too poor to realistically participate. 4. Patients whose breathing capacity is less than 50% of the normal predicted value. 5. Patients with a major neurological disease in addition to ALS. 6. Patients with a history of recent drug or alcohol abuse. HOW WILL THIS STUDY BE CARRIED OUT? Once patients have met the general eligibility criteria just listed, they will be evaluated monthly using a method known as TQNE. This stands for Tufts Quantitative Neuromuscular Evaluation, which consists of a series of tests to objectively assess muscle function and strength. The entire study will be broken down into 3 parts: 1. Admission and Screening: Written, informed consent is obtained from the patient. A medical history is obtained from the patient and a complete neurological/physical examination is performed. Patients must meet all inclusion and exclusion criteria at this point. One visit is required for this part of the study. 2. Evaluation: This portion of the study is also called the Natural History. Patients will be evaluated monthly by TQNE testing*, which was defined earlier. Prior to each TQNE evaluation, vital signs such as temperature and blood pressure will be measured. Each monthly visit will last 1-2 hours. The evaluation/natural history portion of the study will last from 3-6 months. *The Neuromuscular Diseases Unit is one of only a few centres involved in this drug study that specialize in EMG, also known as electromyography. Most patients being considered for this trial have had EMG testing at one time or other. A small series of EMG tests may be carried out monthly along with the TQNE testing, but this has yet to be confirmed. 3. Treatment: If the patient is still eligible*, treatment with either CNTF or placebo will start. Neither the patient nor the investigators will know which the patient is taking. During the treatment portion of the study, patients will continue to be evaluated with TQNE testing and a small number of EMG tests. The treatment portion of the study will probably last for 6 months. *It is possible that during the evaluation/natural history portion of the study a patient may be found ineligible to proceed with to treatment. The criteria for this transition are still being decided. It is crucial that patients proceeding to treatment meet rigid criteria in order to objectively and scientifically determine whether CNTF is effective in retarding ALS. WHO WILL GET CNTF AND WHO WILL GET PLACEBO? In a double-blind, placebo-controlled study such as this one, only half of the patients who are entered into the study will get CNTF. This is not decided by the physicians in charge, but rather by a computer that mathematically randomizes patients to receive either placebo or CNTF. The patient, physicians, technologists and nurses involved do not know what the patient is receiving. This ensures that evaluation of patients at all levels is not biased. As in many drug studies, this is one of the only ways to effectively study whether CNTF is effective in treatment of ALS. WHY CAN'T MY DOCTOR SIMPLY GIVE ME CNTF? CNTF is an investigational agent, and has not yet been approved for use. Once enough data on the safety and efficacy of CNTF has been gathered, it either will or will not be approved for use and subsequently, available through your doctor. HOW IS CNTF ADMINISTERED? CNTF is given by injection. A final decision on frequency of and site of injections has not yet been made. WHAT ARE THE SIDE EFFECTS OF CNTF? Side effects to CNTF in humans MAY be weight loss, irritation of the skin at the injection site, and fever blisters. IS THERE ANY COST TO PARTICIPATE? While CNTF is still under investigation, there is no cost to either the patient or the investigating physicians. == end of als 66 ==