=============================================================== == == == ----------- ALS Interest Group ----------- == == ALS Digest #829 (30 March 2001) == == == == ------ 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 == == 4700+ subscribers in 70+ countries. Please be advised, == == the editor is not a medical doctor and the Digest is == == not peer reviewed. This newsletter is not intended to == == provide medical advice on individual health matters. == == Any such advice should be obtained personally from a == == physician. == == To subscribe, to unsubscribe, to contribute notes, == == etc. to ALS Digest, please send e-mail to: == == bro@met.fsu.edu (Bob Broedel) == == == == Bob Broedel; P.O. Box 20049; Tallahassee, FL 32316 USA == =============================================================== == Back issues of the ALS Digest are available on-line at: == == http://www.glnicholas.com/ == == http://www.alslinks.com == == http://www.alssurvivalguide.com == == http://cc4144-a.ensch1.ov.nl.home.com/~digest == == http://health.oldeman.net == =============================================================== CONTENTS OF THIS ISSUE: 1 .. Refusing to Die. 2 .. Catheter for advanced ALS 3 .. topiramate? 4 .. re: call button/alarm 5 .. 1999 Van For Sale 6 .. Perlmutter Center? (1) ===== Refusing to Die. ========== >From : Projectals@aol.com Date : Fri, 30 Mar 2001 Subject: MEDIA RELEASE Below is an article from the 4/16/01 issue of Forbes magazine about Jenifer Estess and Project A.L.S. which hit newsstands today. Thank you - Julianne Hoffenberg Co-Founder, Project A.L.S. Refusing to Die Told she had Lou Gehrig's disease, Jenifer Estess cursed the lack of research aimed at curing the fatal malady. Then she decided to do something about it. by Robert Langreth, Forbes Magazine, 04.16.01 Jenifer Estess received her death warrant four years ago. Just 35 years old at the time, she learned she had Lou Gehrig's disease, or ALS, a progressive and fatal paralysis in which the nerves that operate the muscles slowly waste away, but the mind remains cruelly intact. There is no cure. Most people die within two to five years after symptoms surface, when they can no longer raise their chest muscles to take in a breath. Praying her New York doctor had erred, she sought a second opinion at Johns Hopkins University from neurologist Jeffrey Rothstein, a world-renowned expert in ALS (amyotrophic lateral sclerosis). She hoped her symptoms, strange muscle spasms and unexplained falls, were caused by some thing else. After a careful exam, Rothstein confirmed the diagnosis. Sensing Jenifer wanted to hear it straight, he was brutally frank: Her motor neurons were dying and would not be replaced. Experimental drugs had largely flopped. Jenifer couldn't believe what she was hearing. "We can transplant hearts and livers," she told Rothstein, why not replace dying nerve cells with new ones? "I'm really sorry, but that's science fiction," the neurologist replied. And it was. She grilled him for two hours, then left. They would meet again. Jenifer walked away determined to turn science fiction into reality. Aided by two of her sisters and friend Julianne Hoffenberg, she formed her own charity, Project A.L.S., to stoke new research in a field long neglected by academic scientists and drugmakers. Ultimately the women quit their jobs to commit full time to this research race. Jenifer, now 39, and Julianne, 35, were New York theater producers; Valerie Estess, 41, was an advertising copywriter; Meredith Estess, 37, worked in fashion. The vow: to find a cure for Jenifer and the more than 25,000 Americans with ALS before their time runs out. "ALS used to be an academic exercise for doctors. It was stamped as unfixable. We're saying that's not good enough," Jenifer says. Until the Estess sisters came along, ALS research fell through the cracks. The National Institutes of Health spends only about $20 million annually on ALS, compared with $80 million for cystic fibrosis, which afflicts a similar number of people. Worse, ALS sufferers constitute too small a market to draw much attention from drug giants; therefore little money is spent trying to translate lab findings into treatments. Even small biotechs have shied away from the disease recently, after several ALS drugs failed in trials. Operating like a brash biotech boutique, the sisters have raised more than $7 million from corporate donors and have recruited an all-star team of eminent researchers from Harvard Medical School, Johns Hopkins and Columbia University. Their pitch is appealingly simple: We will hand you fistfuls of cash, quickly and with fewer hassles than government grants entail, if you agree to work with your rivals, share your results and brief us regularly on your progress. Normally the researchers would have gone their separate ways, for science is often a competitive and insular pursuit. But because of Project ALS, they are making progress faster than even the wildest optimists would have predicted. Within two years, if all goes well, Project ALS plans to begin the first human trial of a breakthrough therapy to treat ALS. It is likely to be one of the first human trials aimed at attacking a brain disease with stem cells, primordial cells that give rise to the specialized cells that form the blood, bones and organs of the body. "They have compressed five years of research into one," says Harvard neurologist Evan Snyder, a collaborator. Adds Andrew Jarecki, chairman of AOL Moviefone and a Project ALS donor: "It's the first charity to move at Internet speed." The Estess family lived a comfortable life in Harrison, N.Y. until their parents separated when they were teenagers. Suddenly their mother had to support five children (Jenifer's oldest sister and a younger brother aren't heavily involved with Project ALS). In the 1980s the sisters settled in the New York area to pursue careers. ALS most often strikes in late middle age, but Jenifer started getting symptoms in 1996, when she was just 34. Her first signs were muscle twitches. Then she started feeling out of breath after climbing upstairs, and she began falling for no apparent reason. Her doctors dismissed it as stress, before diagnosing her in March 1997. Doctors explained that her motor neurons, responsible for coordinating movement, would shrivel and die over several years. Why, nobody knows, but one theory is that the cells are poisoned by excess levels of a brain chemical, glutamate. The only approved drug, Rilutek, prolongs life only several months. ALS patients cope with their disease in various ways. Some try to ignore it; others commit suicide. The Estess sisters attacked it head-on. Since Western medicine had little to offer, Jenifer first tried alternative medicine. She bought something called a "wet cell," which was supposed to cure ALS with electric currents; it didn't work. Twice a week, Valerie and Meredith drove Jenifer to a healer who claimed she could treat ALS with massage and visualization. By November 1997 Jenifer was out $20,000 and getting sicker. She could no longer walk without help. Over Thanksgiving the three sisters and Hoffenberg brainstormed what to do next. The conversation turned to their disgust at the lack of research for a cure. "Why can't we create a Manhattan Project for ALS?" Valerie blurted out. Everyone liked the idea. Within minutes Jenifer was on the phone to her actor friends to solicit their help. The division of labor came naturally. Jenifer, the de facto chief executive, had numerous contacts in show business, as did Hoffenberg. Meredith was savvy about financial and organizational details. Project ALS' first benefit, in June 1998 in New York, was hosted by Jenifer's friend Ben Stiller, and it raised $800,000. Katie Couric also hosted a benefit. The sisters thought about joining forces with an established medical charity but quickly rejected it as too bureaucratic. They wanted to operate more aggressively, gambling on a few big projects, cutting checks to scientists in weeks not months, and following up to make sure they were making progress. Valerie, rekindling a childhood interest in science, spent hours surfing the Web for research on ALS. Two names kept popping up: Rothstein, the neurologist who had confirmed Jenifer's diagnosis in the beginning, and Robert Brown, an ALS expert at Massachusetts General Hospital. Both agreed to become medical advisers and got grants. Government grants require a 50-page application and take at least nine months to be issued; heads of big labs often spend months each year on the paperwork. Project ALS eliminated the hassle. It asked for only a four-page application, and it could cut a check in several weeks. "It's a great selling point to say we are able to fund you now," Valerie explains. The catch was that scientists funded by Project ALS must swallow their egos and work as a team. Valerie figured that scientists needed help networking. She organized several joint sessions with Rothstein, Brown and other top neuroscientists. This yielded several new projects. Among others, Project ALS funded Rothstein and Brown to screen drugs already approved for other ailments to see if any of them might slow ALS. Soon Rothstein's lab found that Celebrex, the hot new arthritis drug from Pharmacia, prolongs the life of mice with ALS by about 25%, better than any drug previously tested. Rothstein and Brown hope to begin a trial of Celebrex in ALS patients in a couple of months. Still, Valerie wanted to go further. In early 1999 a neurologist friend suggested she call Harvard University stem-cell expert Evan Snyder. She did not know much about stem cells, but was intrigued by what she had read. In recent years researchers have learned how to grow these elusive cells in the lab, though such research is controversial because stem cells are often derived from fetal tissue. Her timing was ideal. Dr. Snyder had just isolated brain stem cells in the lab for the first time. In theory these cells could transform into any type of brain cell. Snyder hadn't given much thought to ALS treatment, but he took a liking to the Estess sisters. Meeting Jenifer in New York, he was touched by her courage. By then she was confined to a wheelchair and no longer had the strength to pick up a glass of water. But Johns Hopkins' Rothstein was skeptical about stem-cell therapy, and for good reason. ALS is about the hardest possible disease to consider treating with cell transplants, as the damage is spread across the entire spine. It would, Rothstein figured, require surgically filleting a patient's entire backbone to implant the new cells. That didn't seem feasible anytime soon. Still, Valerie prevailed on Rothstein to meet with her and Snyder at a scientific conference in Toronto. There, Snyder whipped out a set of slides and described his results. Rothstein was stunned by what he heard. Like most scientists, he had assumed that stem cells stayed in one place. But Snyder described experiments in which he took mice with injuries to one section of the spine and injected stem cells into another section. The stem cells, as if equipped with homing devices, migrated to the site of damage, took root, and transformed themselves into cells that, under a microscope, resembled neurons. Few had seen this data because Snyder had not published it. The surprising finding changed Rothstein's mind. It indicated you didn't have to worry about delivering stem cells to exactly the right place; they would target themselves. A few months later Project ALS gave Snyder and Rothstein $500,000 grants to begin testing Snyder's stem cells in paralyzed mice. Meanwhile, the sisters worked on expanding the team. There are numerous kinds of stem cells, so Valerie figured it made sense to test as many as possible. She recruited John Gearhart, a Johns Hopkins researcher who had isolated an even more primitive type, called embryonic stem cells. To figure out how to coax stem cells into new motor neurons, she signed up Thomas Jessell, a top cell biologist at Columbia University. By last summer, Rothstein, Snyder and Brown were testing no fewer than nine types of stem cells. Initially nothing worked. Jenifer, meanwhile, was getting worse and needed round-the-clock care. Then last fall a colleague of Rothstein, Douglas Kerr, showed that stem cell injections helped mice paralyzed by a virus recover partial use of their hind limbs. Unexpectedly, the stem cells did this by secreting growth-promoting proteins that helped the remaining motor neurons work better. This is promising because it means that stem cells may slow ALS even if they don't become motor neurons. Now the Project A.L.S. researchers are putting stem cells through a much more realistic round of tests in mice genetically engineered to develop ALS. They are also completing safety tests on monkeys, which the FDA requires before human trials. The heads of all nine labs involved in the stem cell project meet with Valerie and Meredith in New York every few months to strategize. The hope is to develop a therapy that shows early promise and then entice a biotech firm to take over. Private funds may become more important, as President Bush may limit or end federal funding for fetal stem cell research. Big hurdles remain. The researchers must figure out where to inject the cells, how to coax them into forming motor neurons and how to stop the immune system from killing them. Still, everyone agress the sisters have energized the field. "We have made a ton of progress because of them," Rothstein says. The scientists are motivated by Jenifer's decline. Evan Snyder sometimes stays up late imagining what he would do if he could implant her with stem cells tomorrow. "My goal is a cure, but I would accept anything that prolongs life," he says. Jenifer leaves her Manhattan apartment only occassionally, since going anywhere has become a major production requiring several people to move her. She needs 24-hour nursing to help her eat, dress and perform other basic functions. She requires a ventilator to assist in breathing. Nevertheless, she remains surprisingly energetic, working from her bed, propped up into a sitting position with pillows and talking into her headset. Since the Project A.L.S. headquarters is her apartment, small meetings can come to her. She is planning a made-for-television movie about her quest and a televised benefit next fall. One day not too far in the future Jenifer may literally get a dose of her own medicine. She hopes to be one of the first to try it. But even with the world's best and brightest working on a cure, she knows that a breakthrough may not happen soon enough for her. She is braced for the consequences. "It would be great if it comes in time for me, but I don't think about it on a day-to-day basis. I think about the work," she says. "People say this disease is about letting go. That's nonsense. It's about fighting." forbes april 16, 2001 (2) ===== Catheter for advanced ALS ========== >From : "John McCulloch" Subject: Catheter for advanced ALS Date : Thu, 29 Mar 2001 My wife is incontinent, on a G-Tube, can't talk or move any part of her body. She stays in bed all the time, except when I get her on the shower-chair/comode to take her into the shower or to change her and the bed and clean her. Hospice has asked me if I would like to put her on a catheter. My question: What experiences have others had with a catheter. I fear she will stay in bed more and won't have the benefit of the little movement and change of position she gets now. I use "Attends" and "Blue Pads". The catheter would, of course be a saving. She has no skin break-down. I want the best for her. Any advice? (3) ===== topiramate? ========== Date : Wed, 28 Mar 2001 >From : Redhots1@netscape.net Subject: Does any one know what is happing at Baylor U. on the test that is going on with topiramate (topamax)? How much are they taking Mgs per day? How long is the study? Are they allowed to say how they feel to out side people? If any one knows some one in this study I sure would like to hear from them to see how they are doing. Thank you for your time. My e-mail address: Redhots1@netscape.net (4) ===== re: call button/alarm ========== Date : Fri, 02 Mar 2001 >From : James Ferrel Subject: Doorbell Radio Shack sells a wireless door chime for about $24 that is excellent. The transmitter is about the size of a very small cell phone. The chime unit is about 4"x 6" and can easily fit into a pants pocket. Jim F. (5) ===== 1999 Van For Sale ========== >From : LanaVan@aol.com Date : Wed, 28 Mar 2001 Subject: 1999 Van for sale Hello fellow PALS, We have a 1999 Ford E250 Hi-Top Conversion Van for sale. This is a great van! It has raised side doors to accommodate a taller wheelchair; a Van Gator II Electric Lift (this lift folds back into thirds, allowing people to enter the side doors when it's folded); Qstraint 4 point tie downs with lap and shoulder belts; plus all the really nice features in a conversion van: TV/VCR; CD player; AM/FM cassette; Captain's chairs in the front (plus 2 Captain's chairs that were removed for wheelchair access); sofa in back that converts into a bed; power windows, locks, etc. The technical aspects are: automatic, 5.4 Liter, 8 cylinder (V8), 4 spd, EFI, 2 wheel drive., only 7,000 miles!!! Brand new condition-only taken on a few trips to out of town hospitals, and very well cared for. It's Oxford White w/Beige stripes. Cost $40,863 plus $6,275 worth of modifications. Asking $32,000. Located in South Austin, Texas area. Email LanaVan@aol.com for more information or to make an offer. Thanks! (6) ===== Perlmutter Center? ========== >From : "alaninglisa" Subject: Perlmutter Heath Center Date : Thu, 29 Mar 2001 I would appreciate hearing from anyone who has had experience with the treatment offered by the Perlmutter Health Center. Thank you, Alan === end of alsd 829 ===