Date: Mon, 8 Jan 96 23:28:56 EST From: Bob Broedel To: als@huey.met.fsu.edu Subject: ALSD235 ALS-ON-LINE =============================================================== == == == ----------- ALS Interest Group ----------- == == ALS Digest (#235, 08 January 1996) == == == == ------ 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 == == 1350+ 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 .. Prod! Points Presented to FDA. "Compassionate Access" 2 .. FDA/Patient Meeting Recap 3 .. Thanks for the Christmas Cards 4 .. Question re: DHEA 5 .. New ALS Web Page 6 .. Alternative Treatments (1) ===== Prod! Points Presented to FDA. "Compassionate Access" ========== Date : Sat, 6 Jan 1996 15:59:26 -0500 >From : JACKN74940@aol.com (Jack Norton) Subject: Re: ALSD233 ALS-ON-LINE Bob, I wanted to make sure Internet readers saw the recap of the patient meeting with Dr. Kessler, commissioner of the FDA. Also I attached the compassionate access points we brought up in that meeting. Let's hope the changes we proposed are considered. Now we look forward to distribution of Rilutek and formation of an ALS drug company consortium. Prod! Points Presented To FDA. "Compassionate Access" Definition : 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, may 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 physically qualify for the trial, after the trials are full; and the drug will be offered only for its intended use and intended population. 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 placebo 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 to be administered 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 beyond normal and reasonable fees. 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. 9. FDA will encourage drug sponsors to file a "Early Access Program (EAP)" for drugs which fall within the definition of point 4 above and will act upon the EAP within 60 days of application (2) ===== FDA/Patient Meeting Recap ========== FDA/Patient Meeting Meeting Recap On December 14, 1995, Tim and Kevin Kolquist as well as Jack and Cindy Norton met with the commissioner of the FDA, Dr. David Kessler. This meeting was held at the office of Senator Paul Wellstone of Minnesota in Washington DC and focused on Compassionate access to life saving experimental drugs with some discussion on Combination trials, and input on how to encourage future early access programs. There were nine points presented that suggested changes in how clinical trials should be conducted so that a wider population of ALS patients are included. These nine points were developed on the Prodigy Bulletin board by Larry Sing, Ted Hein, and the rest of us that participated in this discussion since August. I will attempt to recount the meeting to you and look to Larry or Kevin to assist in clarifying or correcting my report to you. When Cindy, Kevin and Tim Kolquist, and I arrived at Senator Wellstone's office we were uncertain as to the length of time Dr. Kessler would allow us. Initially, we thought it would only be about 10 minutes, so we had to have the points typed and ready to give him in case we couldn't get through them all. We arrived early so we could discuss these points with the Senator and his staff. We also discussed Senator Kassebaum's pending legislation on FDA reform. Dr. Kessler arrived with two additional doctors/staff at 4:30 p.m.. We immediately took some pictures of the occasion, uncertain that at the end of the discussion any time or cordiality would still exist < g >. After all folks I have been writing for three years advocating compassionate access to the FDA and was not certain I would not become extremely confrontational. We congratulated the FDA in approving Rilutek within 5 1/2 months. He said that he was happy that for the first time ever a drug had shown effectiveness, even if it was small, it was important. He was proud that the United States was the first country to approve this drug. So on that positive note we began covering the compassionate access points. While Senator Wellstone was present, we recited the requirements for compassionate access as stated in the FDA regulation. We also noted that hundreds of us had received letters from the FDA stating that they could not compel the drug sponsors to offer compassionate access. They had no such authority from Congress and he clarified that to Senator Wellstone. ( It is unlikely that such authority would ever get passed by Congress. My note) Our first several points discussed expanding access to not only "Healthy" ALSers but to those that have failed to meet acceptance criteria into the trial. Although he and his staff appeared genuinely interested, trial design usually is in the purview of the drug sponsors. We hoped the FDA would incorporate some of these points when they review new protocols, we didn't get a yes or no, but finally we felt we were heard. There was some discussion about reimbursement benefits for new drugs and he believed once the FDA approves a drug the HMOs will certainly pick up the drug. Our concern would be how long until the HMOs add it to their list of insured drugs. We had the discussion concerning placebos and how in our view we wouldn't expect to see them used ever again in ALS. At one point Dr. Kessler indicated that the "Tension for us" is how to keep the trials going if expanded access is offered. Many of us believe that once placebos are gone, few would drop out of a trial, even if compassionate access was offered. While in the trial the drugs are free, under compassionate access patients may be charged. Even though Rilutek was approved there still may be some skeptical researchers that for instance may be testing one aspect of ALS that they don't believe Rilutek would impact on and decide to use placebos. Our view was that now there is an approved drug and placebos should not be used in the terminally ill if alternative treatment is available. Rilutek is the best standard of care now available to ALS victims. It appeared that the use of placebos was an issue between the investigator and patients and that the FDA was not sure they could tell them what design criteria to use. If we thought about it we'd probably would have pursued this further with them. Dr. Kessler believes that incentives should be offered drugs sponsors. He believes that the speed in which Rilutek was approved should be an incentive for others to move their drugs into testing. We talked about new delivery methods for drug administration. We asked why Cytotherapeutics was testing in Switzerland instead of the United States. No specific response was given. We stated that many of us don't believe subcutaneous delivery methods are as effective as intrathecal or Cytotheraupics new method. They acknowledged that Cytotherapeutics was very active and that there needed to be other methodologies of delivering drugs looked into. It is apparent that this is a function of the pharmaceutical companies but we asked the FDA to allow these companies to take a risk on new techniques. We asked if the FDA was holding up combination testing. They implied that combination testing was also a function of the pharmaceutical companies and in fact such trials have been conducted before. The more difficult task is getting the companies to work together. The question about why does it take an advisory panel to approve new drugs came up. We were concerned that if a drug company submitted their request for marketing approval and a panel wasn't available they may have to wait several months until getting scheduled. Dr. Kessler believes that the advisory panel allows for public discussion of the drug request and prefers this methodology to accelerate drug reviews rather than just internal FDA. He did mention that it was really up to the FDA divisions and drug companies submitting the necessary data before consideration was given to moving up a panel. This would probably only be done on an exception basis where the data was very compelling. In the FDA regulations compassionate access is only given when no alternative therapies were available. We were concerned that there were no new drugs that were either a cure or put the disease into remission and they might be construed to be the alternative therapy and prevent future compassionate access. Dr. Kessler has approved six anti viral drugs for another disease and has not precluded compassionate access. Dr. Kessler believed that point 9 sort of wrapped up all the other points. That it was thoughtful and well written, especially asking that the FDA "Encourage drug sponsors to file an early access program". He took the points and felt it was time well spent. That was 1 hour and 15 minutes later. At the end of the meeting I informed Dr. Kessler that I had named a mouse after him and it was in the placebo group Patient sponsored research trial. Then I also told him that I had this quote on the Wall in my office "When people are suffering and dying from a devastating disease we cannot wait for all the evidence to come in -for all the i's to be dotted and t's crossed. We must be prepared to accept greater risks from a drug when greater benefits are possible." We asked that his entire organization embraced this quote from him and that all advisory panels read it. Senator Wellstone indicated that his staff had been taking notes and they will be following up. We also wanted Senator Wellstone's to continue to consider Senator Kassebaum's Bill in light of this discussion and the points presented. Legislative pressure as well as FDA policy changes continue to be necessary to achieve compassionate access. The Quest continues! Jack Norton (3) ===== Thanks for the Christmas Cards ========== Date : Thu, 4 Jan 96 23:59 EST >From : chubsche@explorer.csc.com (Carl Hubscher) Subject: Thanks for the Christmas Cards I sincerely thank everyone who responded to my request that was posted in an ALS Digest Newsletter to mail my brother-in-law a Christmas Card. When I spoke to my sister in North Carolina she said that her and my brother-in- law and their family had only received a few Christmas Cards. But they lied, when I walked into their home situated in the woods of rural North Carolina, they were two large rows of Christmas Cards strung across and down the entryway between their living room and dining room, plus another 50 or so Christmas Cards that decorated the rest of the house. Last count, I heard that they had received over 140 Christmas Cards from my request. The returned addresses on the Christmas Cards ranged from every part of the US, plus some from Canada and Belgium. The lovely notes that were enclosed in many of the cards has given him encouragement and meant a great deal to my brother-in-law and his wife. Both of them wish you God bless and hope you and yours had a Merry Christmas and will have a Happy New Year! Carl Hubscher, Manassas, VA. (4) ===== Question re: DHEA ========== >From : Maryellen Rudolph Date : Sun, 31 Dec 1995 13:22:05 -0500 (EST) Subject: Question RE:DHEA I am feeling like a first time caller on a radio talk show. I am not familiar with the procedures so please forgive me. I am connected with the ALS Regional Center in Albany NY. I manage the Speech Pathology department at St. Peter's Hospital. I have been working in the area of ALS for several years. I have a few questions, and was hoping someone may be able to guide me. Our ALS team is looking for any journal references for info on "Steroid Sex Hormones and Neuro degenerative diseases, more specifically DHEA Estrogen. My second request is for information from persons on line who are using augmentative communication or adaptive single switches to communicate on their computers. I am looking for info for PCs as well as Macs. We have received a grant and are looking for the most practical and functional systems to purchase. Thank You all for your information. As I have stated many times to several patients. "I am the professional but you are the experts." Thank you for your help. You may contact me through the Digest or individually at: Rudolphm@capital.net (5) ===== New ALS Web Page ========== Date : Sat, 30 Dec 1995 23:52:37 -0500 >From : srdawson@interlog.com (Scott Robert Dawson) Subject: New ALS Web Page Hi All! I've just put up a web page about ALS. I intend to concentrate on info about ALS in the Greater Toronto Area, but I've got links to the ALS Digest, Brunel University, Canadian societies and MedHelp International. Much more coming! The URL is: . Hope this proves useful. Please send comments, contributions, etc to: Thanks! Scott Robert Dawson --------------------------------------------------------------------------- | Genetics is fun, but Scott Robert Dawson | _my_ family is defined by love... | srdawson@interlog.com http://www.interlog.com/~srdawson/scothmpg.htm --------------------------------------------------------------------------- (6) ===== Alternative Treatments ========== Date : Sat, 30 Dec 1995 14:00:07 -0500 >From : Terry Byles Subject: Alternative Treatments There does not seem to be a comprehensive study on the alternative therapies for ALS. I'm tring to research the following; 1. Interferon and sandostatin pulse therapy - Trying to locate people who have tried the interferon and sadostatin pulse therapy protocol used by the Medenica Clinic. So far the only information I can locate comes only from the clinic and their published articles. I would like to talk to any patients or cargivers who have used this protocol. Also, any persons who have investigated this protocol please share your findings. 2. Live cell therapy - Are there any persons who have tried this therapy. Past issues of the digest mention only two patients. If you have investigated this therapy please share your findings. 3. Antibiotic therapy - Has anyone used long term antibiotic therapy such as the types that have had some sucess with MS? 4. Ayurvedic Medicine - Is there any als patients experimenting with this. They have many claimes but again this always seems to come from people who would profit from the therapy. If anyone would care to comment on any of these therapies, please do so. 'greetings for the new year' Terry Byles === end of als235 === ALS Association (National Office) 21021 Ventura Boulevard, Suite 321 Woodland Hills, CA 91364 USA TEL 818-340-7500 FAX 818-340-2060 TEL 800-782-4747 (Patient Hot Line) E-M eajc27b@prodigy.com ALS Society of Canada 220 - 6 Adelaide Street East Toronto, Ontario, M5C 1H6 CANADA TEL 416-362-0269 TEL 800-267-4ALS (toll-free in Canada) FAX 416-362-0414 E-M alssoc@inforamp.net