Date: Wed, 16 Nov 94 09:54:44 -0500 From: Bob Broedel To: als@huey.met.fsu.edu Subject: ALSD#148 ALS-ON-LINE =============================================================== == == == ----------- ALS Interest Group ----------- == == ALS Digest (#148, 16 November 1994) == == == == ------ 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. The ALS Digest is == == published (approximately) weekly. Currently there are == == 390+ 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 == =============================================================== CONTENTS OF THIS ISSUE: 1 .. ALSA/PATIENT MTG 2 .. travel (1) ===== ALSA/PATIENT MTG ========== Date : Wed, 16 Nov 1994 01:09:59 -0500 >From : JACKN74940@aol.com Subject: ALSA/PATIENT MTG Bob, FYI. ALSA/PROD! meeting NOV 5TH. I want to thank ALSA and the Advocacy committee for their willingness to listen to OUR goals and concerns. The discussions were quite frank and positive in tone. I can tell you that we all were there to identify the bottlenecks and set before the committee patients perspectives. It has always been my goal to work within the existing organizations and to change their priorities via dialogue such as this meeting and patient input through the PROD!. I presented to Ellyn the names of all PROD! members that voted for the goals. Topics of discussion included Combination Trials, Compassionate Access, Cessation of Placebos, Research funding, Patient Registry, FDA Advisory Panel and Communication. There were other areas discussed such as Patient representation on the ALSA boards and upcoming meetings with the FDA and NINDS. Those present at the meeting included Linda Mcknight, Ted Heine, Stephen Pie, James Rather, Jack Norton from the PROD! Unfortunately, Mark Malconian was ill and was unable to attend. ALSA had the majority of the Advocacy committee present to include Ellyn Phillips -Chair, Joshua Javits, Dara Alexander, and Lynn Klein. Also in attendance was Dr Hiroshi Mitsumoto - published the CNTF/BDNF Wobbler mouse study. Absent was Robert Abendroth who I believe was at the Joint Conference ALSA and Human Genome project, Pam Berry and Jim Copeland. All the Advocacy committee members have had someone a friend or family member with ALS. Ellyn got the meeting going by identifying the above mentioned topics and then we agreed on a prioritized set of topics to discuss in the few hours we had available. 1) Combination Drug Trials - this was a good learning experience for all present and I will try to get you a flavor of the discussion. Dr Mitsumoto and others within a physicians consortium - name I have forgotten - have agreed that combination studies must begin for ALS. I was surprised that although Dr Mitsumoto had shown positive efficacy results in the Wobbler mouse there was no safety data generated to provide the FDA. Apparently mice are much hardier than us and can live even in the most inhospitable bioenvironment - not so for us were sepsis can take us out. To move combo trials ahead the FDA will need safety data that all other systems are not damaged because of the combination treatment. Funding has to be obtained, toxicologists, pathologists brought on board and animal models procured with approval of animal testing boards at the trial centers, as well as gaining approval of the yet unwritten protocol. These obstacles are manageable but access to the various drugs are dependent on the drug sponsors. Which brings us to the discussion of what we have espoused for months on this BB, that a Drug company consortium for ALS be established. All the above areas of concern can be worked on but a more integrated effort of drug sponsors, ALSA and principal investigators must be initiated. With several drug companies getting ready to evaluate their data in the next few months, the timing of a Drug sponsored ALS Consortium must be set to late January or Feb. (If effectiveness is say 20-40% for any one drug in slowing the disease than drug sponsors should demonstrate that their drug and someone elses might offer a 80 to 90% slowing down of the disease and they may find that different combos work for different people or forms of ALS - my opinion). We need all variations of drugs processed through animal safety trials as soon as the drug sponsors will make them available. (I don't believe for animal testing we have to wait for human efficacy in each drug) I believe ALSA and Dr Mitsumoto's efforts will begin immediately in getting safety trials setup and forming the drug company sponsored consortium. ( there may be a Nobel prize in the offering ) I regret that we can not go immediately to combos because of FDA animal safety data, but within a few months this can be accomplished and perhaps by April or May the FDA could approve safety trials for humans - timelines need to be established my opinion....it really depends on how fast investigators, clinic boards, ALSA ,FDA and drug sponsors can work on this opportunity. Combos and Consortium discussion was exciting, I hope the momentum continues - I hope ALSA can successfully orchestrate this for our Quest! Topic 2) Compassionate Access - not to be confused with FDA's term of expanded Access which applies in this case to extended participation of current trial patients. The FDA does not use the term Compassionate - which they should, it has a ring of humanity to it. The concern that came out was that if patients are given compassionate access why would they participate in trials? The regs say that extended/ compassionate access may occur when clinical trials are proceeding with the patients they need, is offered to the intended population and can be offered no earlier then Phase II. The drug can be offered when it may be effective not proven effective. The FDA has told all of us that they can not compel a drug sponsor to offer a drug. If it was offered after all the above criteria was met the question again arises as to why would you participate in a trial? Our answer would be that participation offers the drug for free and all the attendant tests. If offered on a patient or single IND the company can charge for the drug but only for shipping, research, handling etc , no profit. I would also assume that these patients would have to pay for Dr visits and testing, because they need to be monitored. The Question then arose, how do they pay for it. The answer is those that want the CHOICE will find the way, but we can't even get there yet because not one of these drug companies offered the drug during their phase III testing. The FDA needs to be appraised of this and either the regulations must compel compassionate access to the drug sponsors capacity or they should be confronted by the FDA when they submit their protocol. ALSA will include this clarification to FDA in their upcoming meeting. I think the Advocacy committee now knows what a total lack of Choice patients outside the trials have. Discussion about who may get Compassionate access started when the how criteria would be set to select those willing to take the chance. Again we have not even got the drug sponsors to attempt this. The Lottery idea for MS was said to have been flawed. I believe flawed or not it was a viable attempt to offer a drug and should not be discounted. What is also hopeful is that Ellyn Phillips is trying to get on the FDA Advisory Panel and if successful should be able to address these issues as part of the approval process - not to stop the process but to enlighten drug sponsors and FDA experts on how ALSA perceives the issues surrounding the drug and its trial - that is what I would expect an ALS representative to do - Lynn or others correct me if this is an overstatement of how you see this position filled. 3) Cessation of Placebos and tying in a Patient registry with natural history data from trials as well as just population data. The use of placebos remains currently accepted method of the scientific community. I do believe that there are scientists that want to see this changed. The idea of a fully populated patient registry would allow investigators to at least consider it for a control or for comparison of new drugs to documented patient progression. We pointed out that once one drug was shown to be effective it becomes the standard treatment and there should never be a placebo used in ALS again! We mentioned that PAACT had a bill before the Senate and the House to get the NIH to have a National Registry of Prostate Patients. It was thought that their success was uncertain. It was unclear from my recollection of who would be a creditable organization to collect and manage the data. I felt that this point was not thoroughly fleshed out, but was recognized as a goal. Further work is needed here. 4) Research Funding - it was explained how ALSA has been able to get special report language from Congress identifying ALS as a priority for the NIH to accept. This report language is something few other diseases have accomplished and should tell the NIH the sense of the Congress. ALSA has been told that $11 million will be spent on ALS in Fiscal year 95 and ALSA wants proof it isn't being spent on diseases that are related but not ALS. They have gotten report language that tells NINDS to conduct more intramural research - I assumed this meant internal research. We were also told that sometimes even if money is allocated NIH may redirect because researchers have not submitted any requests for grants - so tell your researchers to get grant requests in! I believe after three years of getting report language in it must be complemented with other functions. There has been a set of diseases that have had aggressive in your face patient lobbying as well as full time lobbying organization. I believe that we should look at those successful organizations and add their methodology to the expertise of our congressional efforts. I think ALSA is evaluating their current efforts and what else can be done. There was concern about funding. This is no small matter - yet a full range of patient participation, paid lobbyists and ALSA advocacy may get us more research. Finally, Communication, we asked ALSA to have more of a presence on Prodigy. The on-line access to data means information is spread across the country immediately. The Link , a quarterly update has information the majority of ALS victims have known about for months. ALSA's absence on Prodigy creates a vacuum that is filled by an appearance of little or no activity. ALSA then begins to get some negative comments tossed at them. There has been activity going on and yet because we are unaware of it we are unconvinced they are concerned about us. We feel that if they have a meeting with the FDA, NIH or drug sponsors we would like to know how it went. Good or bad news. If they need our help they should use us. We don't expect that they answer every message on Prodigy, but more frequent updates would position ALSA closer to the patients. Again I think we learned quite a lot and got the messages we could get in across to ALSA advocacy, now lets see how their National board reacts. James Rather, I want to thank you for your efforts in attending the meeting. You have courage and a will that your fellow Prodders so deeply appreciated. Ellyn, I believe the meeting went well and your ability to keep us on track allowed us to cover a lot of ground. If you need the PROD! to push Advocacy let us know. We wish you success at the Board meeting next week and hope to get measurable results. The Quest Continues! Jack Norton (2) ===== travel ========== Date : 15 Nov 94 23:25:04 EST >From : wayne phillips <70303.173@compuserve.com> Subject: travel In the fall of 1992 we went on a Carribean cruise on Royal Carribean's Majesty of the Seas. It's maiden voyage was April '92. It was designed to be wheelchair accessable; only a couple of small upper sun decks are stairs-only, but there are plenty of accessable sunning areas. I had my power chair and got around fine. I used my walker to transfer and wasn't on a vent or feeding tube. The handicaped rooms are double wide. We moved the twin beds together so I had my wife on one side and a wall on the other. I used my walker to get into the small bathroom and transfer to the toilet and a small wall mounted seat in the shower. Transferring was especially tricky due to the ship's motion, the slick tile bathroom floor, and a ridge around the shower area to contain water. I'd recommend bringing a potty chair and planning on bed baths. They made a special note about the cabins where the handicapped tourists were in case of an emergency. The crew took excellent care of us, letting us go to the front of the lines, and providing my food chopped so my wife had less work to do. We were told before the trip (by phone) that (1) they had manual wheelchairs on board, but only for emergencies, and (2) ports served by tender (small boats) rather than docks would be inaccessable. But once on board the maiter d' told us to ask the medical people about getting a manual chair, and they were glad to lend one for the whole week, which we used for going ashore. The maiter d' also encouraged us to ask the crew to get me (in the borrowed manual chair) onto the tender and from the tender to the dock. They carried me on and off the tenders without being asked or objecting. Actually, maiter d' said that if the medical staff or crew didn't help us, we should tell him and he'd take care of it. Perhaps the fact that I only weighed 120 lbs. and we had a non-combative approach helped. Cozumel: docks We took my power chair ashore but couldn't go far. The town is about three miles from the docks with many large bumps in the sidewalk at driveways, one of which I couldn't climb, even going backwards. But there were tourist shops and interesting scenery within range. It was that evening that the maiter d' suggested the manual chair. If we'd had one we could have transferred me to a taxi, put the chair in the trunk, and made it to town. Grand Cayman: tender The town is next to the docks, with much to see, somewhat accessable. The museum is interesting and accessable. We took a taxi several miles to the turtle farm (accessable) and saw good scenery along the way. Jamaica: docks Anything interesting requires a taxi drive; the drivers are highly competitive and willing to take you somewhere, then tell you the price! The people were very friendly and helpful. This was the only port where the people were not intimidated by my illness and willing to talk much with me. Royal Carribean private island in the Bahamas(instead of the embargoed Haiti): tender Only sunning, snorkelling, scuba, and snacking available here. No pavement; wheelchair had to be dragged through the sand. BUT...the on board video the previous night said, "You can snorkel even if you can't swim!" I had my wife call the dive master, describe my condition, and ask if there was a way for them to take me snorkelling. I'd been a strong swimmer and been snorkelling before my illness, but their safety review was good. I had a life vest on, hung my arms over a float, and put my mask in the water while an instructor pulled me around the protected lagoon. I'm sure their lawyers would be appalled! This isn't for everyone, but I've heard of some people with severe disabilities even going scuba diving here in the States. This was the highlight of my trip, a real blessing. See digest #115 for info on our airline flight LAX->Miami->LAX. Good luck, adventurous ones! Wayne === end of als 148 ===