Date: Tue, 7 Nov 95 10:22:08 EST From: Bob Broedel To: als@huey.met.fsu.edu Subject: ALSD223 ALS-ON-LINE =============================================================== == == == ----------- ALS Interest Group ----------- == == ALS Digest (#223, 07 November 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 == == 1180+ 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 == =============================================================== == A full set of back issues (on MSDOS 3.5 INCH HD diskette) == == are available by sending me your full mailing address. == == They are free-of-charge. International requests welcome. == =============================================================== CONTENTS OF THIS ISSUE: 1 .. Bid For A Cure 2 .. FDA Reform/Abuse 3 .. BDNF study in Tampa, Florida 4 .. BDNF trial in Southern California 5 .. ALS and dental surgery 6 .. ALS and loss of hearing 7 .. Mevacor 8 .. TENS 9 .. West Michigan ALS Association (1) ===== Bid For A Cure ========== >From: vitale@dectlk.ENET.dec.com Date : Sat, 28 Oct 95 22:05:11 EDT You are cordially invited to attend the Second Annual "Bid For A Cure" Dinner Auction honoring Anthony J. Vitale, Ph.D. Senior Consulting Engineer Linguistics & Speech Technology Assistive Technology Group Digital Equipment Corporation Thursday, November 16, 1995 6:00pm Westboro, Marriott $25.00 per person Program Schedule - 6:00pm - Cocktail hour and silent auction 6:30pm - Food stations open 7:00pm - Silent auction closes 7:15pm - Guest Speakers - Dr. Jeremy Shefner MDA Clinic Director and ALS Researcher Brigham & Women's Hospital - Karen Regan Demand Generation Manager, Embedded and Realtime Products Group Digital Equipment Corp. - Dr. Tony Vitale Honoree, 1995 MDA "Bid For A Cure" 8:00pm - Live Auction 9:30pm - Closing remarks/coffee All proceeds will benefit the neuromuscular research and patient service programs of the Central Massachusetts Chapter of the Muscular Dystrophy Association. For Tickets, call Kevin Gould at MDA (508) 898-3375 (2) ===== FDA Reform/Abuse ========== Date : Thu, 2 Nov 1995 11:30:38 -0500 >From : JACKN74940@aol.com To : FDAabuse@hr.house.gov Cc : tellbill@hr.house.gov, mail_grams@grams.senate.gov, : senator@wellstone.senate.govalex_clyde@wellstone.senate.gov, : David_Zlatchin@wellstone.senate.gov, : Connie_Lewis@wellstone.senate.gov, broedel@geomag.gly.fsu.edu Subject: FDA Reform/abuse Jack & Cindy Norton III 3344 139TH Lane NW Andover, MN 55304 2 November, 1995 FDAabuse@HR.House.Gov House Commerce Committee Chairman Joe Barton Dear Representative Barton, My name is Jack Norton, I am married and have three young daughters. I am a victim of the terminal illness, called ALS or Lou Gehrig's disease. I am also a victim of a failed FDA drug approval process that has claimed thousands of American lives. How were these lives lost? Through Apathy and Lazy Science. How is the FDA apathetic? When ALS drug trials are being conducted, thousands of Americans are excluded from these trials. FDA regulations allow for compassionate access to experimental life saving drugs, when trials are fully enrolled. However, when the terminally ill requested compassionate access they were told that FDA could not "Compel" drug sponsors to provide the drug, even if the victims were willing to pay for it. The FDA goes on to say that their authority comes from the Congress. We have asked the FDA for recommendations on how to "Fix" the broken compassionate access program. They never responded. They must believe that access to life saving drugs, while drug trials are underway, works. Nothing could be further from the truth. It is quite clear that the FDA has time to recommend to Congress that cigarettes should be treated as a drug delivery system, but has no interest in giving the terminally ill the "Choice" to take experimental life saving drugs. These acts of omission cost lives and because they are hidden no one knows that a chance at life was promised, but extinguished by an unworkable compassionate access flaws in the FDA regulation. What does Lazy Science have to do with the FDA? The FDA authorizes drug trial protocols and will allow for parallel track, history controls, dose variances and placebo control studies. Of these forms of drug trials, the use of placebos borders on "crimes against humanity". In a disease such as ALS, there has never been a therapy or cure/remission. For over 125 years, physicians have measured and observed the deaths of hundreds of thousands suffering from this horrific disease. In cases were there is no alternative treatment, the FDA has an "Institutional bias" toward the use of Placebos, their "Gold standard". The use of placebos in the terminally ill is the same as saying these people are "expendable". Measuring how effective a drug is against a" body count" is lazy science. Why allow for other forms of testing and yet through their known bias give credit only to placebo controlled trials? Claiming that placebos allow drug trials to proceed faster, cheaper and provide meaningful measurements ignores the humanity of those given "False Hope" by this research methodology. The FDA asserts that it is not their responsibility to find alternative ways of measuring effectiveness. I believe their leadership in finding and encouraging alternative surrogate endpoints, as opposed to body counts, is Apathetic and Lazy! The approval process is extremely bureaucratic! Drug sponsors spend thousands of dollars for user fees and wait months for FDA Advisory panels to meet and recommend approval. Then months pass again until final FDA approval is given. A case in point includes the drug Rilutek. The last week of June the drug sponsor,RPR, submitted their request for approval. Two and 1/2 months later, Sept. 18, a panel was convened to review the drug. It received panel recommendation for approval that day. Over 45 days later, the FDA has still not given final approval. Lives are being lost. Any drug that extends life, must receive the highest priority within the FDA! User fees from drug sponsors of life saving experimental drugs must be used to accelerate the approval process. If the FDA needs more personnel to assess effectiveness of life saving drugs, then get the personnel hired or divert existing personnel to this desperate battle for survival. Since the FDA has no recommendation or interest in dealing with issues of the terminally ill, I respectfully submit a list of policy changes that should be included in the FDA Reform act and hope you will cosponsor them. Compassionate Access or Expedited access to products (drugs or therapy) for individuals diagnosed with a terminal illness. for which no clinical treatment exists for the remission or cure of the terminal illness, during human drug trials, Phases 1-3. 1. Drugs that have successfully completed Phase 1 safety trials, or any level past Phase 1, can be made available to patients provided the Drug Sponsor has sufficient supply; that the patient, attending physician, and Drug Sponsor reach a financial agreement for payment and treatment protocol in accordance with Drug Sponsor Charges (Point 6, below). 2. During Phase 2 trials, experimental drugs may be made available by the Drug Sponsor to those patients not able to qualify for the trials after the trials are full; the drug will be offered only for its intended use and intended population and the Drug Sponsor may charge for the drug in accordance with Drug Sponsor Charges (Point 6, below). 3. The FDA shall require that Drug Sponsors submitting protocols for Phase 3 clinical trials, must make the drug trial protocol available to all patients to whom the Drug Sponsor intends to market the approved drug. After the trial is full, the Drug Sponsor may charge for the drug in accordance with Drug Sponsor Charges (Point 6, below). 4. The FDA shall identify Investigational New Drugs as "Non-FDA" approved when the Drug Sponsor initiates Phase 3 clinical trials, to allow physicians to prescribe and to require insurance carriers to pay for experimental life saving drugs. 5.The FDA will encourage alternative drug trial designs that incorporate history controls, dose variances, and other humane methodologies. It will discourage the use of placebos on terminally ill patients and encourage innovative surrogate endpoints. 6. Drug Sponsor Charges defined: Until the FDA has approved a New Drug Application, the Drug Sponsor can only recover the cost of manufacture (including allocation of overhead), research, development, distribution and handling costs, plus an escrow account amount. The purpose of the escrow account is to allow distribution to patients who are unable to pay the established price. Escrow accounts are recommended to be administered, for Orphan Diseases, jointly by the National Organization for Rare Disorders (NORD) and the Drug Sponsor. Drug costs for each category will be disclosed only to the FDA to ensure proprietary trade confidentiality and are exempt from the Freedom of Information Act. 7. The attending physician is prohibited from accepting any additional compensation from the Drug Sponsor. 8. Drug Sponsor defined: Any company, corporation, educational institution, research laboratory or foundation, charitable organization defined under Section 401 of the Internal Revenue Code, foreign or domestic, that submits drug trial or therapy protocols to the FDA for approval. I am sure that the substance of this letter is clear: make experimental drugs available to patients suffering from terminal illnesses and allow drug companies to recover direct costs. I hope that you will consider adding this amendment to H.R. 2019. Don't "Protect Us to Death!" Offer the Choice. The Quest Continues!... Your response will be posted on the ALS Medical Support bulletin board on Prodigy where hundreds participate in this battle for survival. Let me know if you will conduct further FDA oversight hearings. I know there are many victims of this horrific disease that are willing to testify. Respectfully, Jack Norton cc: tellbill@hr.house.gov Rep. Bill Luther Mail_Grams@Grams.Senate.gov Sen. Rod Grams Senator@Wellstone.Sen.Gov Sen. Paul Wellstone ALS Medical support group The PROD! (3) ===== BDNF study in Tampa, Florida ========== Date : Sat, 28 Oct 1995 21:46:14 -0400 >From : ChuckBoo@aol.com Subject: Re: BDNF study in Tampa, Florida The BDNF study mentioned by Dr. Raul Mandler is also taking place in Tampa, Florida. I assume the qualifications for participation are the same as they are in New Mexico. That is, patients who have a diagnosis of ALS, who have good pulmonary function, a moderate degree of ability in daily living activities, and who are under 80 years of age may be eligible. I know of this because my wife was just accepted into the program yesterday. We thought this was a good study to be in because it does not prevent you from using other treatments concurrently. For more information please call Cathy Wayand, R.N. at (813) 870-3287. (4) ===== BDNF trial in Southern California ========== Date : Mon, 30 Oct 1995 11:29:52 -0800 >From : cns@connectnet.com (Jay Meyer) Subject: Re: ALSD221 ALS-ON-LINE Dear Bob: Please add our clinical trial site to your list for patients interested in BDNF in Southern California. Contact Dr. Richard Smith or Jonathan Licht at CNS, 11211 Sorrento Valley Rd., San Diego, 92121. Richard Smith, M.D. (5) ===== ALS and dental surgery ========== Date : Sat, 04 Nov 1995 17:06:22 +0500 >From : cbang@vt.edu (Chulho Bang) Subject: Want information about the relationship between ALS and dental : surgery My brother has been patient of ALS for 1 year. Recently I am interested in the relationship between ALS and dental surgery. My brother got his molar removed in July, 1994. He felt so terrible pain that he almost lost his senses because the doctor did not use an anesthetic. About 3 months later in Oct., he felt the symptoms and was diagnosed the limb ALS in Dec. Now, he can not use his all limb and voice. He and I have doubt that the surgery might affected nerve system, which could induced the heinous disease. In my opinion, this issue is something different from amalgam poisoning. Because any of filling material was not used. I want any information about the relationship between ALS and dental surgery. (6) ===== ALS and loss of hearing ========== Date : Wed, 1 Nov 1995 13:07:30 PST >From : "John Patterson (604) 666-0524" Subject: ALS and Loss of hearing My wife experienced profound hearing loss - particularly in her left ear - at the same time she started to experience the ALS symtoms (Sporadic ALS). The ear specialist had difficulty determining that she was in fact hard-of- hearing because she can hear sounds fine. The problem is that she can no longer understand them without watching the speakers lips. The specialist said her loss was consistant with the type of ear nerve damage attributable to working in an industrial plant - something which she has never been exposed to. Our Neurologist said that the two nerve conditions (ALS and hearing loss) are not related. However, they manifested symptoms at the same time. It is my hope that the ALS Digest subscribers may have had some similar experience or PALS and medical practitioners may be able to suggest some course of action to futher investigate this hearing loss issue. Hopeful in Life, John H. Patterson (7) ===== Mevacor ========== Date : Sun, 5 Nov 1995 20:25:02 -0500 >From : Stirfry2@aol.com Subject: RE: ALS I HAVE ALS AND MY DAUGHTER JUST SENT ME SOME OF YOUR NEWSLETTERS. I AM INTERESTED IN FINDING OUT IF YOU HAVE ANY SUBSCRIBERS WHO HAVE TAKEN A DRUG CALLED MEVACOR/LOVISTATIN THAT THEY BELIEVE HAS CAUSED THEIR ALS. THE SYMPTOMS STARTED WHEN I WAS TAKING MEVACOR. THE DRUG DIDN'T HAVE ANY WARNING SIGNS WHEN IT CAME OUT EXCEPT FOR LIVER DAMAGE. THEN IN 1994 THE WARNINGS BEGAN TO COME OUT IN PRINT IN TIME MAGAZINE. I HAVE THE SYMPTOMS THAT THE ADS REFERRED TO. I AM ON A FAST TRACK TO DYING, WHICH IS OK WITH ME BECAUSE I AM NOT AFRAID TO DIE. IF YOU HAVE ANY PEOPLE THAT BELIEVE THEIR ALS IS CAUSED BY MEDICINES/DRUGS WOULD YOU REFER THEM TO ME? THANK YOU VERY MUCH, ROSE MARY WALKER-SAMTER (8) ===== TENS ========== Date : Sat, 4 Nov 1995 13:58:55 -0500 >From : Terry Byles Subject: TENS I would like to know if anyone has any information or experience on using TENS (Transcutaneous Electrical Neuromuscular Stimulation) for strengthing atrophied muscles. I know there was rhis study in Japan with good results. Title: A Clinical trial of theraputic electrical stimulation for als Source: Tohoku Journal of Expirimental Medicine(VTF),1995 feb; 175(2):123-134 I would love to get a copy of this article!!!!! I have been using a TENS unit for about two weeks, and believe my atrophied right hand is increasing in strength. It is probably to soon to tell and it may only be psychological and this time but it feels good just to know I'm attempting to do something. Terry in NC (9) ===== West Michigan ALS Association ========== DATE : 10/29/95 SOURCE: The Grand Rapids Press When Lou Gehrig stood before thousands of fans in Yankee Stadium 56 years ago, tears filling his eyes and weakened from the early symptoms of ALS, not much was known about the disease from which he suffered. Today, the cause, cure and means of controlling Amyotrophic Lateral Sclerosis (ALS), often referred to as Lou Gehrig's Disease, remains somewhat elusive, according to information from the West Michigan ALS Association. First described in detail by noted French physician Jean-Martin Charcot in 1869, ALS is a progressive, fatal, neurological disease that attacks nerve cells in the brain and spinal cord. Characterized by a deterioration of motor cells in the spinal cord and brain, the disease leads to muscle weakness which may affect hands, feet, legs, arms, tongue and other muscles. Symptoms may include general weakness, twitches and cramps in the muscles of the hands and feet at the early stages to severe impairment of the use of the arms and legs in later stages. Muscles of the palate, pharynx, tongue, neck and shoulders are commonly affected in advanced stages leading to thick speech and difficulty in swallowing. At no time, however, are a patient's mental faculties affected. ALS can affect anyone, however, first symptoms most often appear between the ages of 40 and 70 years. Upon diagnosis with ALS, the average life expectancy is three to fours years, but some individuals remain active for 10, 15 or more years with short periods of apparent respite from the disease. For more information, contact the West Michigan ALS Association at 616-365-0303. === end of als 223 ===