Date: Tue, 28 Mar 95 01:13:25 -0500 From: Bob Broedel To: als@huey.met.fsu.edu Subject: ALSD#182 ALS-ON-LINE =============================================================== == == == ----------- ALS Interest Group ----------- == == ALS Digest (#182, 27 March 1995) == == == == ------ Amyotrophic Lateral Sclerosis (ALS) == == ------ Motor Neurone Disease (MND) == == ------ Lou Gehrig's disease == == ------ Charcot'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 == == 580+ 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 .. re: Hereditary Defect 2 .. re: Hereditary Defect (cont.) 3 .. Remission and Reversal of ALS? 4 .. re: feeding tubes 5 .. letter to Dr. Hall 6 .. re: Managing Coughing Problems 7 .. Keyboard and electrical stimulation 8 .. re: Phoenix 9 .. Cytotherapeutics begins ALS trials (1) ===== re: Hereditary Defect ========== Date : Wed, 22 Mar 95 09:04:01 -0500 >From : "Dr. Kenneth Fischbeck" Subject: RE: Hereditary Defect > >Are there any studies on ALS as an hereditary defect? > Much work has been published on this topic recently. About 10% of ALS is familial, and about 15-20% of familial ALS is associated with mutations in a superoxide dismutase (SOD) gene on chromosome 21. Transgenic mice that express the mutant human SOD gene have motor neuron degeneration resembling human ALS, and thus can serve as an animal model for the disease. A few references: Rosen DR et al. Nature 362:59-62, 1993 Gurney ME et al. Science 264:1772-1775, 1994 Rothstein JD et al. Proc Natl Acad Sci USA 91:4155-4159, 1994 Beckman JS et al. Nature 364:584, 1993 K. Fischbeck, M.D. Neurology Dept, Univ Pennsylvania (2) ===== re: Hereditary Defect (cont.) ========== Date : Thu, 23 Mar 95 13:07:01 -0500 >From : "Dr. Kenneth Fischbeck" Subject: RE: Hereditary Defect (cont.) I also just noticed a very good review of familial ALS by Robert Brown in the 10 March 1995 issue of the journal Cell. K. Fischbeck (3) ===== Remission and Reversal of ALS? ========== Date : Mon, 27 Mar 1995 16:27:01 -0500 (EST) >From : WECK0820@duq3.cc.duq.edu Subject: Remission and Reversal of ALS? I am currently reading a book by Hiroshi Mitsumoto and Forbes Norris called "Amyotrophic Lateral Sclerosis : A Comprehensive Guide to Managment". In chapter two, page 25 of this book, Dr. Walter Bradley explains that he tells newly diagnosed ALS patients that the "condition can "burn itself out" or arrest at a certain stage". He also tells patients that he has seen three cases himself and others in "literature, who clearly had a syndrome of ALS but who spontaneuosly went into remisson". Is there any other documented cases of ALS going into remission or even reversing? If so, where can I find this literature? Please send responses to : Weck0820@duq3.cc.duq.edu I will appreciate any info that anyone can give very much. Thanks! (4) ===== re: feeding tubes ========== Date : 23 Mar 95 18:35:24 EST >From : Mike Ward <72567.400@compuserve.com> Subject: Re: ALSD#181 ALS-ON-LINE In response to Jerry Creedy's question on feeding tubes. I feel very strongly that feeding tubes are best when done through the neck as opposed to a standard g tube. The surgery is done on an outpatient basis and involves only a local anesthetic. A g tube affects your breatheing ability, a neck tube doesn't. I have had my tube 5 years now and there have been 0 problems. It is easy to use and I was being fed while writing this note. An ENT does the surgery not a general surgeon. I encourage people to get a feeding tube as soon as you can't maintain your weight or eating a full meal gets too tiring. Then you can eat what you can and blend and tube the rest. I have found it works better to suck out all the stomach air before feeding. This reduces the gas in the intestines. IF you need further detail contact me at 72567.400@compuserve.com Regards, Mike Ward (5) ===== letter to Dr. Hall ========== Date : Tue, 14 Mar 1995 18:32:10 EST >From : TASM76A@prodigy.com (MR TED HEINE) Subject: a.l.s. After consulting with Ellyn Phillips, Chair of the ALSA Advocacy Committee, and Lynn Klein, ALSA VP for Patient Services, I sent the following letter to Dr Zach Hall, Director of NINDS, on March 9. The letter is basically an outgrowth from the ALSA Advocacy Committee meeting in Philadelphia, last November 5, and an effort on my part to focus effort on developing a data bank of ALS patients who received placebo in clinical trials. Dr. Zach Hall, Director National Institute of Neurological Disorders and Stroke National Institutes of Health 9000 Rockville Pike Bethesda, Maryland 20892 Dear Dr. Hall: At your meeting on December 9, 1994, with representatives of the Amyotrophic Lateral Sclerosis Association, one of the topics discussed was the possibility of establishing a registry of placebo controls from recent drug trials involving ALS patients. Dr. Hiroshi Mitsumoto of the Cleveland Clinic, who advises ALSA, had suggested that placebo controls might be eliminated in future ALS drug trials by making use of placebo data collected in recent and on-going trials. This possibility is of great interest to ALS patients. It is important that some agency proceed with dispatch in securing necessary approvals, drug company agreement, and finding a site to locate the placebo patient registry. I believe that your assistance will enable the ALS community to move forward aggressively to achieve this desirable objective. NINDS seems to me to be the logical agency to host such a registry of placebo-patient data. To facilitate working on this project, your identification of an individual within your organization who would be able to focus on this endeavor and serve as a point of contact for interested parties and the drug companies that have recently conducted or are planning to conduct clinical trials, would be beneficial. The article on the front page of the February 27, Wall Street Journal, highlighted the concern of the medical community that future clinical trials may be contaminated by inter-patient contact. That concern can be obviated by use of information on placebo controls in prior clinical trials, where the contamination factor is minimized. It would also enable all patients in a future clinical trial to receive active drug (albeit in varied doses), which should certainly increase their willingness to participate in a clinical trial. An additional benefit is that the cost of conducting a clinical trial would be reduced, since drug companies would not be obliged to enlist as large a number of patients as is necessary when one third or one fourth receive placebo, but must still be evaluated as carefully (and as expensively) as patients who receive active drug. This factor should encourage drug companies to make past data available to the agency hosting the registry. Those of us who have been diagnosed with amyotrophic lateral sclerosis are eager to see the scientific community take vigorous, timely, and focused action to find effective treatments for this vicious, debilitating, and terminal disease. I hope that you are able to respond positively to this request to host a placebo patient registry. Your presence at the meeting of the Amyotrophic Lateral Sclerosis Association in Chicago on May 19-20 would be a particularly propitious opportunity to announce a preliminary commitment to host the patient registry. I look forward to your response. Sincerely yours, Ted Heine ALS patient 319-352-3899 (home) 319-352-8416 (office) 319-352-8581 (fax) e-mail: heinet@wartburg.edu TASM76A@prodigy.com (6) ===== re: Managing Coughing Problems ========== Date : Wed, 15 Mar 1995 09:59:15 -0500 >From : rbm@hookup.net (Robert Macdonald) Subject: Managing Coughing Problems Thanks to Al Pickens for his suggestion. I have had very annoying coughing problems lately, and welcomed your story about martinis. Since my diagnosis I had given up alcohol. I thought it might have a negative impact on my immune system. However, yesterday I tested your suggestion. I tried a weak mixture of scotch (my old favourite) and water in my feeding tube. It worked! My coughing was greatly reduced. I presume the alcohol relaxes the throat muscles. That's the greatest prescription I've had so far! Robert Macdonald Robert Macdonald rbm@hookup.net (7) ===== Keyboard and electrical stimulation ========== Date : Thu, 23 Mar 95 09:05:45 MST >From : sth@piglet.lanl.gov (Steve Hildebrand) Subject: Keyboard and electrical stimulation ALS has weakened my arms and hands. Now, I type into my Mac with my left hand on top of my right hand; but that is getting more difficult. I'm still capable of using the mouse and a lot of my work is working with computer code and math equations. Does anybody out there have a recommendation for hardware/software to input info into a Mac? Can ALS patient's muscles be stimulated by external electrical sources? Could this method be used to keep the muscles from atrophying? Steve Hildebrand (8) ===== re: Phoenix ========== In ALS Digest 173 we mentioned that a journal named Phoenix was being published by ALS patient Jerry Weber. In the current issue of the ALS Association's Southeast Wisconsin Chapter newsletter we learn that one can order the Phoenic as follows: === = Phoenix = S90-W22905 Rose Avenue = Big Bend WI 53103-9717 === (9) ===== Cytotherapeutics begins ALS trials ========== Trial Designed to Deliver Targeted Quantities of CNTF Across Blood-Brain Barrier PROVIDENCE, R.I. March 23 /PRNewswire/ -- CytoTherapeutics, Inc. (Nasdaq: CTII) today announced that the first human clinical trial for a cell-containing implant to deliver a recombinant protein within the central nervous system (CNS) is underway in Europe. The implants contain cells from a non-human cell line genetically engineered to release ciliary neurotrophic factor (CNTF), a growth factor under investigation to treat amyotrophic lateral sclerosis (ALS). The cells are encapsulated in a membrane which protects them from the patient's immune system, and offers the ability to retrieve the cells, if this is desirable. The study is designed to test the safety of the implants in up to 10 patients with ALS over a period of six months. The trial is being conducted by Patrick Aebischer, M.D., Ph.D., a founding scientist of CytoTherapeutics, at the Centre Hospitalier Universitaire Vaudois (CHUV) in Lausanne, Switzerland where he is Director of the Gene Therapy Center. "This trial marks an important step in the Company's strategy," said Seth A. Rudnick, M.D., CytoTherapeutics' Chairman and Chief Executive Officer. "For the first time, it tests the ability of our proprietary encapsulated-cell technology to deliver potent, genetically engineered growth factors, such as CNTF, across the blood-brain barrier. Demonstrating safe delivery of CNTF via our implants would validate the ability of our unique cell and gene therapy approach to treat devastating neurodegerative disorders." CNTF is one several substances produced naturally by the body that is thought to provide a protective effect on the nerve cells affected in ALS. Human trials of CNTF conducted to date utilized systemic administration of the factor. Those trials were halted reportedly due to side-effects and unclear efficacy. Systemic administration generally requires that large quantities of a substance be delivered to assure that a small amount may pass across the blood-brain barrier. However, high systemic doses may be associated with side-effects. By contrast, the implants used in this trial are designed to deliver approximately 500 times less CNTF than that delivered systemically in previous trials, and to deliver the factor directly to the portion of the CNS where it is required. Dr. Aebischer commented, "The selection of CNTF as the first of several potential growth factors to deliver in humans was based on the profound unmet need in ALS treatment, and our belief that our delivery approach may overcome the difficulties CNTF has encountered when delivered by conventional approaches." The implants used in the trial contain CNTF-producing cells and measure approximately 5 cm in length and less than half a millimeter in diameter. The implants are surgically placed at the base of the spine in a common, outpatient procedure similar to a spinal tap. The implant design and surgical procedure are similar to those currently used in the Company's CereCRIB(TM) clinical trials for the treatment of severe pain. After implantation within the spinal fluid, the implants are designed to deliver small quantities of biologically active CNTF, synthesized by the cells, directly to the neuronal cell bodies located within the spinal cord and the lower areas of the brain. This is the first trial to utilize a membrane developed in conjunction with Akzo Nobel Faser AG in Wuppertal, Germany, the Company's membrane partner. CytoTherapeutics, Inc. is a leader in the development of cell-containing, biocompatible implants designed to deliver therapeutic substances for the treatment of central nervous system disorders. The Company is currently developing products for the treatment of chronic pain, Parkinson's disease, Alzheimer's disease and ALS, with research efforts directed to a variety of other CNS disorders. CONTACT: Elizabeth Razee, Manager, Corporate Communications of CytoTherapeutics, 401-272-3310, ext. 2132/ (CTII) === end of als 182 ===