Scientists Move Closer to Stem Cell Therapy for Neurodegenerative Diseases
Jeffrey Rothstein |
Soon after scientists identified neural stem cells — primitive cells that can generate new nerve cells (neurons) — they recognized that the cells held potential for treating neurodegenerative diseases like ALS and spinal muscular atrophy (SMA).
Now, MDA-funded scientists have made the breakthrough discovery that neural stem cells can repair neuronal damage in rodents that have a condition similar to ALS or SMA.
ALS and SMA are both paralyzing diseases, caused by the destruction of muscle-controlling neurons (motor neurons) in the spinal cord.
"The [new] research may lead to improved treatments for patients with paralyzing motor neuron diseases," said Jeffrey Rothstein, leader of the MDA-funded research team and co-director of the MDA/ALS Center at Johns Hopkins University in Baltimore.
Rothstein, along with MDA grantee Douglas Kerr of Johns Hopkins and Evan Snyder of Harvard University in Boston, presented the research on stem cells at the Society for Neuroscience meeting in New Orleans last month.
To create an acute condition that resembles the end stages of chronic motor neuron diseases, the researchers infected rodents (mice and rats) with the Sinbis virus. Though harmless to humans, the virus quickly paralyzes rodents by destroying their motor neurons.
The researchers then treated some of the paralyzed mice with neural stem cells derived from primordial germ cells (more primitive stem cells that normally produce eggs or sperm). They delivered the cells by injection into the cerebrospinal fluid (CSF), which fills spaces within the spinal cord and brain. A control group of rodents received a "sham" injection with no stem cells.
About eight weeks after the injection, all of the control rodents remained completely paralyzed, while roughly half of the treated rodents recovered some movement in their hind limbs. When the researchers examined the spinal cords of rodents that didn't benefit from treatment, they found that, in some cases, the injection had missed its mark and the stem cells hadn't entered the CSF, Rothstein said.
In the rodents that did benefit from treatment, the researchers found that the stem cells had dispersed to damaged areas of the spinal cord. Rather than replacing dead neurons, the stem cells appeared to help nurture dying ones back to health.
These two findings are important advances in stem cell research, said Rothstein, because they show that "stem cells delivered to the CSF can have widespread invasion into the nervous system... and have some effect in offsetting neural degeneration."
Nonetheless, Rothstein emphasized that improved mobility in the rodents was small. Scientists are hopeful that stem cell therapy could at least lessen the most life-threatening effects of motor neuron diseases, such as weakening of the muscles that control the lungs.
Also, much research needs to be done before the stem cell treatment can be tested in people with ALS or SMA, Rothstein said.
"There are different labs coming up with different stem cells," he said. "But the question is: Are they equivalent? We'll test different sources of stem cells and make comparisons about their efficacy."
"Clinical trials [of stem cell treatment] are probably several years away," he said, "but we're working as fast as we can." 
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Avanir Launches Trial of Drug to Control Crying and Laughing
In ALS, lower motor neurons in the brainstem that control muscles in the mouth, throat and face can be lost, resulting in abnormalities in speaking, chewing, and swallowing, and sometimes in the physical expression of emotion. These symptoms are often called bulbar palsy (because part of the brainstem can be called the bulb and palsy is a word for paralysis).
ALS also involves loss of upper motor neurons, those in the upper part of the brain, which exert some control over the lower motor neurons. The term pseudobulbar palsy is used to describe what's thought to be uncontrolled activity of the lower motor neurons of the bulbar (brainstem) region, a phenomenon that seems to cause laughing and crying sounds not necessarily associated with emotions. When upper motor neurons are lost, it's as if the upper motor neuron "brake" has been removed from the brainstem neurons that usually control these physical manifestations of emotion.
Uncontrolled spells of laughing and crying can be embarrassing and even frightening to people with ALS and their families. To lessen these symptoms, Avanir Pharmaceuticals of San Diego, Calif., has developed a drug that combines dextromethorphan and quinidine, two drugs already on the market for other uses. Dextromethorphan is a cough remedy, and quinidine is used to treat heartbeat abnormalities.
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| A current theory says that when upper motor neurons that control bulbar lower motor neurons are lost, a neurological "brake" on laughing and crying can be lost. The makers of AVP-923 hope the drug can restore some of the lost control. |
James Berg, vice president of Clinical and Regulatory Affairs at Avanir, says dextromethorphan has long been known to inhibit glutamate, a nervous system chemical implicated in ALS. He adds that the drug is very quickly metabolized by most people.
Some years ago, Richard Smith, a California neurologist, tried treating ALS patients with dextromethorphan, hoping to slow the progression of the disease. According to Berg, Smith and his patients "serendipitously found that it did not retard ALS but that patients came in and said their spontaneous outbursts of laughing or crying were suddenly not there anymore."
In 1995, Smith went on to conduct a pilot study of dextromethorphan hooked to quinidine to see if he could get a longer dextromethorphan effect. Results were promising enough to convince Avanir to go further.
The company plans a 96-person, 30-day trial of the compound, known as AVP-923, at the centers listed below.
"I think we can get a read on its usefulness in this indication quickly," Berg said.
After applying for inclusion in the trial, patients will be assessed for their degree of pseudobulbar palsy by clinical observations, questions and a short survey called the Central Nervous System Lability Scale.
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RESEARCHER EXPLORES GENETIC INFLUENCES ON ALS RISK
The cause of ALS is often a mystery, but an MDA-funded researcher is tracking down genes that might modify a person's risk of developing the devastating neuromuscular disease. Her work might lead to a better understanding of disease susceptibility, and is expected to reveal "protective" genes that might hold clues to effective treatments.
Some 90 percent of all ALS cases (sporadic ALS, or SALS) have no obvious origin, leading researchers to speculate about a daunting combination of genetic and environmental causes. But familial ALS (FALS) accounts for the remaining 10 percent of ALS cases, clearly pointing to strong genetic factors in at least some cases of the disease.
SOD1 Mutations
The genetic defects underlying FALS are mostly unknown, but scientists are doggedly pursuing the culprit genes. In 1993, they discovered that about 25 percent of FALS cases are caused by mutations in the gene that produces copper/zinc superoxide dismutase (SOD1) — a protein that helps keep free radicals in check.
Though scientists don't understand why, mutated SOD1 protein doesn't simply lose its normal activity. Instead, it appears to gain an activity that's toxic to muscle-controlling nerve cells (motor neurons) in the spinal cord and brain, leading to paralysis and death. Rarely, people who have disease-causing SOD1 mutations don't develop ALS, suggesting that other genes might modify the destructive effects of the renegade SOD1.
Identification of these so-called genetic modifiers probably would shed light on the disease process and open up therapeutic possibilities. But finding them presents a major challenge to researchers.
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| Catherine Kunst |
"SOD1 mutations only cause about 2 percent of all ALS, so we're talking about maybe four or five people in the world who have one of those mutations but didn't get ALS," says MDA grantee Catherine Kunst. "So, finding whether or not they have a common [genetic modifier] is really hard."
In her lab at the Eleanor Roosevelt Institute in Denver, Colo., Kunst is tackling this obstacle by studying a mouse model of ALS. Her recent progress toward identifying genetic modifiers of ALS in the mice is described in the Dec. 1 issue of Genomics.
Preventive Genes
Scientists created several different lines of mice with ALS for research soon after finding a link between SOD1 and FALS. To do so, they constructed human and mouse SOD1 genes containing the disease-causing mutations, and then incorporated them into the genetic makeup of mice. Time and again, they found that the mutated SOD1 caused these transgenic mice to develop ALS.
But Kunst's latest research points to genes that can delay, and sometimes even prevent, the onset of ALS in one line of SOD1 transgenic mice.
She discovered evidence for these genes by crossbreeding the line of SOD1 transgenic mice to a separate line of inbred mice. That mating blended the genes from the two parent lines, creating SOD1 transgenic mice with a new (and variable) genetic makeup.
This mixed genetic makeup stalled the onset of ALS in the SOD1 transgenic mice. In the original SOD1 transgenic mice, disease onset oc-curred uniformly at about 100 days of age. But in the "mixed" SOD1 transgenic mice, the disease onset times were much later, and highly variable — from 143 days to two years of age.
A small percentage of the mice remained disease-free after two years (the average life span of a healthy mouse), echoing the few cases in which people with SOD1 mutations have escaped ALS.
"If we can find the specific [genes] helping the mice, we should be able to help people with the specific SOD1 mutation [the mice have]," says Kunst, who plans to examine more lines of SOD1 transgenic mice to determine whether the as-yet-unknown genes protect against other SOD1 mutations.
So far, she's localized candidate protective genes to discrete regions of chromosomes 1, 13 and 18 in the mice.
The SMA Connection
Kunst is focusing her attention on the region of mouse chromosome 13, which contains 23 genes, she says. That piece of chromosome, she's observed, is responsible for about 90 percent of the improved health of mixed SOD1 transgenic mice.
Kunst says she's especially excited about this region because it contains the genes for the survival motor neuron protein (SMN) and neuronal apoptosis inhibitory protein (NAIP).
Both SMN and NAIP are often missing in people with spinal muscular atrophy (SMA), a disease that destroys a subset of the motor neurons killed in ALS. Though the role of NAIP in SMA is unclear, loss of SMN is sufficient to cause SMA.
SMN and SOD1 are "both obviously important to the survival of motor neurons," says Kunst, so it's intriguing to speculate that they interact to modify the onset of ALS (at least in mice).
While it's possible that these mouse genes are identical to the genes suspected to protect some people from ALS, Kunst isn't jumping to conclusions. "If we find a protective gene in the mouse, we can look for it in humans," she says, "but the chances of finding it are small."
Genetic Modifiers
Kunst is also quick to point out that calling these genes protective is rather arbitrary.
"You could turn it around and say that in the [original SOD1 transgenic] mice, these genes are susceptibility genes, because they cause those mice to get ALS sooner." Similarly, depending on the precise activity of certain genes — which probably differs among individuals —- people might either be predisposed to, or protected from, ALS. That's why scientists often refer to such genes as genetic modifiers.
If Kunst finds identical genetic modifiers of ALS in mice and humans, scientists could have a way to evaluate the risks of developing ALS — maybe even SALS. Kunst might even find some genetic modifiers that directly cause the types of human FALS that aren't caused by SOD1 mutations.
Even if they're never found in humans, identifying genetic modifiers of ALS in mice will still benefit people with the disease, says Kunst. Figuring out how genetic modifiers protect mice from mutant SOD1 might finally reveal how mutant SOD1 becomes toxic and causes FALS, she explains. Even for people who have sporadic ALS — for which the cause is unknown — such insights might reveal the general process that leads to motor neuron death.
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Tuesdays With Jean-Martin Charcot
Every Tuesday, famed nineteenth-century French neurologist Jean-Martin Charcot would lecture his students at the Salpêtriére hospital in Paris, discussing disorders affecting muscle strength and mobility.
While Charcot's "Leçons du Mardi" (Tuesday Lessons) probably weren't as warm and uplifting as Mitch Albom's "Tuesdays with Morrie" as recounted in his best-selling book about Morrie Schwartz, they were undoubtedly of greater scientific interest.
Charcot, a charismatic speaker and teacher and one of the most famous neurologists of his day, wasn't the only medical thinker of the time to focus on ALS, but he was the first to give a name and a full and accurate description to the disease.
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Jean-Martin Charcot,
1825-1893 |
Charcot was born in Paris in 1825, the son of a carriage maker. He earned his M.D. at the University of Paris in 1853.
In 1862, he was appointed senior physician at Salpêtriére, a hospital, insane asylum and shelter for women. The setting provided Charcot with an unending supply of patients with a variety of chronic conditions that he could study and treat over the years.
In fact, Charcot's hospital became a kind of mecca for aspiring neurologists, drawing students from across Europe. He and his students were concerned with the anatomy and functioning of the brain and spinal column, with trying to sort out the differences between physiological and psychological disturbances, and with the clinical observation of neuromuscular abnormalities.
Charcot employed the "anatomo-clinical" method of studying symptoms of living patients and coordinating that meticulously gathered information (including photographs, drawings and footprints) against post-mortem examinations of the same individuals. He was particularly interested in learning how lesions found in the nervous system might be matched up against the symptoms of degenerative neuromuscular conditions.
Closing in on ALS
ALS had no definite name when Charcot began studying it. Other medical thinkers, such as Sir Charles Bell and François Aran, had studied conditions closely allied to ALS.
Guillaume-Benjamin-Amand Duchenne, the provincial French doctor who became a friend of the more worldly Charcot and whose name would become linked with the most severe form of muscular dystrophy, also contributed to understanding of motor neuron disorders and employed the anatomo-clinical method before Charcot.
In 1865, Charcot wrote about a woman he at first thought was suffering from hysteria, a mental disturbance which Charcot attributed to physiological causes. Her symptoms included extreme muscle weakness with no intellectual, sensory or bladder impairment.
Subsequent post-mortem examination of the woman revealed "sclerosis" — hardening — on areas of the spinal column.
It was Charcot who, in a series of lectures in 1874, provided a definitive name for the disorder, amyotrophic lateral sclerosis, based on his observation of 20 patients and five autopsy studies. He also gave a clinical description of the condition that's still largely accurate.
In fact, ALS is still referred to as Charcot's disease in many places, although in the United States the link with baseball legend Lou Gehrig has superceded the Charcot connection. Charcot's role in identifying numerous other disorders — such as Charcot-Marie-Tooth disease — may also have blurred the importance of his connection to ALS in some minds.
"Charcot did not give the first description of ALS, nor did Shakespeare originate the plots of his plays; the elements of the story were known, but it remained for the master to produce a masterpiece," said neurologist and author David Goldblatt.
Charcot was a real celebrity in his day, renowned as much for his showmanship and lecturing skill as for his medical insights. He treated royalty and other elite patients in his private practice.
Unconventional Methods
Despite his solid scientific achievements, Charcot's often remembered for his fascination with hypnotism — then called mesmerism — as a tool in diagnosing and treating hysteria. A young Sigmund Freud witnessed these demonstrations and was greatly influenced by them.
Other unconventional treatment methods tried by Charcot included hydrotherapy, electrotherapy — and even suspending a patient in the air using a harness and pulleys in an attempt to stretch the spinal cord.
In Charcot's later years, a special position was created for him at Salpêtriére as professor of Diseases of the Nervous System. His interests remained wide-ranging. He differentiated multiple sclerosis from Parkinson's disease, and worked with his student Gilles de la Tourette in studying tic disorders.
It could be said that his greatest legacy was the next generation of neurology students, who carried forward his work to better understand, classify and treat neurological disorders.
Charcot died in 1893, while on holiday with some of his students.
Once, after presenting his students with the case of a man with ALS, Charcot commented: "Let us keep looking in spite of everything. Let us keep searching. It is indeed the best method of finding, and perhaps thanks to our efforts, the verdict we will give such a patient tomorrow will not be the same we must give this man today."
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PATI MILEWSKI: A LOT ON HER PLATE
When Pati Milewski cares about somebody or something, she isn't content just to say so. She does something about it.
That's why, when she attends meetings of her MDA ALS support group in her home city of Olympia, Wash., she usually doesn't come empty-handed, but arrives with an armload of gifts for the group members. The bamboo back scratchers she provided proved to be particularly popular.
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| Pati Milewski attended an MDA summer camp this summer on the same day the Harley-Davidson riders visited. |
And even though she faces her own tough daily battle against ALS, she's always seeking other ways to make a positive difference — including her distinctive decorative plates which she auctions off to raise money to send kids to MDA summer camp.
Varied Career
Milewski, 45, was born in Connecticut and raised in Pennsylvania, but moved to the state of Washington as a young woman.
While working at Woolworth's for 15 years, she studied at night school and secured a job working for the state as a computer programmer.
"I created the system that the public disclosure commission uses for campaign finance," she says with pride. She worked for the state for seven years.
In her free time, she successfully completed a six-month course training as a volunteer reserve police officer. While undergoing her last weapons recertification, she first became aware of muscular weakness.
"I was having trouble with my index finger on the trigger, which is critical," she says.
After ordering an EMG, her doctor suspected a neurological impairment, and referred her to the MDA clinic at the University of Washington Medical Center in Seattle, where Milewski received a diagnosis of ALS in 1999.
Support from MDA
"The first thing they did was have me fill out MDA papers," she says. "I didn't know why but they said that, after my insurance, I wouldn't have any bills from there. That's really a wonderful thing. It helps me and my family. Since I'm not married, my father worries who will help take care of these things. So I told him, MDA is there for me."
Milewski's ALS progression has so far been fairly slow. She still manages to walk with a cane but sometimes uses a manual wheelchair.
As appreciative as Milewski is for the help she's received from the MDA clinic, she is perhaps most grateful for the friendship and understanding she gets through her ALS support group.
At her first meeting, one of the other attendees set the tone when he said: "Pati, we all have ALS and we don't have time to waste 'ho-humming,' so ask anything you want to know from anyone here and we'll tell you like it is."
"We care about one another unconditionally," Milewski says. "We ask each other the tough questions, and get straight answers."
The group is attended both by people with ALS and their caregivers. Milewski is apprehensive about the notion of needing a caregiver someday, but she knows she can count on the group for providing guidance and reassurance.
She says she sometimes suffers from "survivor's guilt" from having seen other members of the group lose their lives to ALS.
"We continue to celebrate each person who joins our group, and we never let them be forgotten," she says.
Too Pretty to Eat From
A favorite pursuit of Milewski's is making decorative plates which use names and images of members of her support group to pay tribute to this important aspect of her life.
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| Milewski with one of the plates she made and donated to the MDA for an auction. |
It started out as an activity within the group, but it took on another life after Milewski was asked to contribute some of her plates to MDA auctions.
At one auction, when bids on a plate were coming in too slowly, Milewski stood up and made a speech about the invaluable help and friendship she had received from MDA. Bidding accelerated, and the plate brought in more than enough to send one child with a neuromuscular disease to MDA summer camp, a contribution that's become very important to Milewski.
This year, Milewski visited the MDA camp session at Camp Waskowitz, located on the Snoqualmie River near North Bend, Wash. She was greatly touched by the youngsters and the volunteers who make the camp's magic possible.
"Nothing but good comes out of camp," she says.
MDA local staff members are very grateful to have such a caring MDA volunteer and persuasive public speaker.
Although the progression of ALS hasn't made the plate-decorating process any easier, Milewski remains determined to keep producing her unique plates — and making a big difference for the MDA youngsters that she has come to care about so very much.
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Texas ALS Cluster Could Be 'Incredibly Important,' Experts Say
Renowned ALS researcher and clinician Hiroshi Mitsumoto says a recently identified cluster of 39 cases of ALS at Kelly Air Force Base in San Antonio, Texas, could be "incredibly important" in the quest to identify potential environmental and genetic factors underlying ALS.
Mitsumoto, who directs the Eleanor and Lou Gehrig MDA/ALS Center at Columbia-Presbyterian Medical Center in New York (see The ALS Newsletter vol. 5, no. 5), said that, while other ALS clusters have been identified over the years, "this is certainly, to my knowledge, a huge number, which is most unusual and which we cannot ignore.
"We need to investigate and not let this opportunity go by without finding out the cause. First, every effort should be made to see if those 39 patients are truly ALS or if other diseases have been included," he emphasized.
If the 39 current and former Kelly employees who are thought to have ALS do have the disease, Mitsumoto said, "My great hope is that the Defense Department will convene some kind of investigation team." (That effort is under way at Brooks Air Force Base in San Antonio, which houses the Air Force Institute for Environmen-tal Safety and Occupational Health Analysis.)
Mitsumoto stressed that, even if an environmental factor at Kelly that triggers ALS is identified, that doesn't mean environmental factors are the only triggers in the disease.
ALS, he says, doesn't have just one cause, "but multiple causes, all of which can lead to cell death in motor neurons." Environmental factors, he said, could include viral infection, an accelerated aging process or aberrant actions of the immune system, as well as internal or external toxins.
"We know so much about the cell death process," he said, "but as to the mechanisms that trigger it, we don't know."
Mitsumoto notes that only a limited number of people at Kelly got the disease.
"There are some genetic factors which make one vulnerable to certain exposures, while other people are very resistant to the same factors," he said. (For more on ALS-modifying genetic factors, see "Researcher Explores Genetic Influences.")
Neurologist and MDA research grantee Stanley Appel, who directs the Ronny and Linda Finger MDA/ALS Center in Houston, said he's also concerned about the cluster in Texas but doesn't think answers will come quickly.
"The bottom line is that we've had many other situations where clusters of ALS patients have occurred, and we have not been successful in pinning down any single, specific cause," Appel said.
"My own theory is that there could be many different environmental hazards that could trigger ALS in a 'susceptible' individual, but until we understand what causes the increased susceptibility, it will be difficult to implicate the specific environmental factors involved."
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Alcohol and Anti-Arthritics: New Treatments, or Shots in the Dark?
The search for ALS treatments has led to a variety of prospects, some promising (stem cells), some disappointing (nerve growth factors) and some completely unsubstantiated (snake venoms).
Two recent studies in the Annals of Neurology have identified two more possible ALS treatments that, at this point in time, may prove to fit into any one of those categories.
An October study suggests that red wine — previously reported to help prevent heart disease — might also defend against ALS.
In the study, powdered red wine was mixed into the drinking water of mice carrying a mutant SOD1 gene that causes ALS. The elixir allowed those mice to live about 6 percent longer than those receiving normal drinking water.
The study's authors, led by Italian researcher Domenico Rotilio, suggest that red wine might exert a beneficial effect on ALS by acting as an antioxidant, or by blocking the action of caspases, enzymes that naturally trigger cell death.
A November study suggests that a class of prescription drugs commonly used to treat arthritis might also be useful for treating ALS. These drugs — called COX-2 inhibitors — block the formation of prostaglandins, fatty acid compounds that both promote inflammation and stimulate the release of glutamate from astrocytes (support cells in the brain and spinal cord).
Given evidence that excess glutamate exposure might be the cause of nerve cell death in ALS, Daniel Drachman and Jeffrey Rothstein of the MDA/ALS Center at Johns Hopkins University in Baltimore tested whether a COX-2 inhibitor could protect damaged nerve cells in a petri dish. The COX-2 inhibitor, they found, significantly increased survival of the cells.
Will these treatments prove useful to people with ALS? Only time (and more research) will tell.
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Book Offers Laughs and Genuine Inspiration for Living with ALS
"This is the day the Lord has made. Get up, go to the ballpark, and do your very best."
These are the words that Joe Martin, a Charlotte, N.C., man who has ALS, has chosen to live by. It's a simple phrase that mixes wisdom from the Psalms of King David and a famous quote by baseball player Cal Ripken Jr.
But how Martin arrived at this philosophy is not so simple. He chronicles his journey in On Any Given Day, an autobiography that's a hopeful, entertaining and yet honest look at life with ALS.
Martin teams with writer Ross Yockey to tell a story that begins with his diagnosis in 1994 and ends with him still living "like this": unable to talk or walk but fighting to live his life to the greatest extent possible. In between, he continued to build his career as an executive at one of the nation's biggest banks, energized and strengthened race relations in his city, and helped found the clinic that's now the MDA/ALS Center at Carolinas Medical Center in Charlotte.
The book features a fun-to-read, light tone and delightful humor that makes the reader smile and laugh at some of the most unexpected moments.
Martin doesn't dwell on his losses of physical abilities as the disease progresses, but he doesn't ignore problems such as the depression, anger and other common realities of the disease. Readers will appreciate Martin's detailed accounts of coping with the disease, whether it's explaining his diagnosis to friends and family members, creating adaptive equipment or finding ideal personal caregivers.
Along the way, Martin finds strength in the loved ones around him and in his religious faith, thanks to his son-of-a-Presbyterian-minister upbringing.
Martin's writing is intertwined with comments and personal observations by co-author Yockey. Although this italicized text can be a bit intrusive when it interrupts Martin's storytelling, the commentary allows the reader to learn more about Martin and his family that he either omits or is too humble to write about himself.
Absent in Martin's journey with the disease are financial strains that often plague families dealing with ALS. After all, this is a man who hobnobbed with Bill Clinton before and during his presidency, added an elevator to one of his homes, and regularly flew to Houston's MDA/ALS center for treatment.
He's keenly aware that his comfortable financial standing sets him apart from other people with ALS, and even jokes that some might think his story could be titled "Little Lord Fauntleroy Copes With Trouble." But Martin readily offers low- or no-cost ideas for finding help and support to readers who aren't as financially fortunate.
Overall, Martin's genuine story and remarkable attitude are nothing short of inspirational. There is something to be gained from this book for any person touched by ALS.
On Any Given Day, published by John F. Blair Publisher, is available in most bookstores and over the Internet; it's in hardcover for $21.95.
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22nd ALS Center Opens in Philadelphia
MDA has announced the opening of the MDA/ALS Center of Hope at MCP Hahnemann University in Philadelphia. The facility is the Association's 22nd designated MDA/ALS research and clinical center.
Terry Heiman-Patterson, an ALS specialist for 18 years, is director of the center. She is professor of neurology and director of the university's Division of Neuromuscular Diseases.
Heiman-Patterson heads a team that offers a multidisciplinary approach to ALS treatment. The center also conducts research on ALS, including clinical trials of promising new drugs and basic research.
To schedule an appointment, call the center at (215) 762-5035 or call MDA's Philadelphia office at (215) 683-9270 in Horsham, Pa. You can learn more about the center by visiting www.alshopefoundation.org/clinic/frames.html.
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Are You Receiving Quest?
Anyone who has ALS and is registered with the Muscular Dystrophy Association should be receiving The ALS Newsletter and Quest, MDA's national magazine. Both are published bimonthly and sent to registered clients with ALS at no charge.
Quest contains feature stories about many aspects of living with a disability; reports on the status of MDA research; ads and items about products that can be helpful to people with ALS; detailed articles about medical and daily care for people with neuromuscular diseases; and other information on MDA programs.
If you're not receiving Quest please contact MDA's department of Public Information at (800) 572-1717, or publications@mdausa.org. You can see back issues of Quest and The ALS Newsletter at our Web site, www.mda.org.
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Speech Devices to be Covered by Medicare
Starting Jan. 2, augmentative and alternative communication (AAC) devices that generate speech for people with limited vocal ability will be considered durable medical equipment eligible for coverage by Medicare.
Under previous guidelines, speech generators and synthesizers and their accessories have been considered convenience items. A speech system can cost up to several thousand dollars.
Currently, local Medicare providers are operating under an interim measure approved this spring by the U.S. Department of Health and Human Services through its Health Care Financing Administration, which allows providers to decide whether to cover speech generators and synthesizers on a case-by-case basis.
"The scope of this policy change is enormous. This is the biggest change in regards to access to medical treatment in a decade," says attorney Lewis Golinker, director of the Assistive Technology Law Center in Ithaca, N.Y., of the planned change. "People with ALS are among the primary beneficiaries. This is giving them the opportunity to communicate with their families."
Drafted this fall, the new policy extending Medicare coverage to AAC devices is expected to be finalized by June. In the meantime, cases submitted on or after Jan. 2 will be individually reviewed, with the expectation that they'll be approved.
Under the new rules, Medicare will cover at least five classifications of AAC tools and accessories, including digitized and synthesized speech generators; speech-generating software programs; and mounts to attach the equipment to wheelchairs.
Details of the new policy still being worked out include the types and numbers of AAC items to be covered and the amount of reimbursement that will be provided by Medicare. Questions about coverage of specific items should be directed to local or regional Medicare providers.
Interested parties are invited to express opinions on the new regulations before Dec. 19. To view a draft of the proposal, visit www.augcomin.com/whatsnew/policy.htm.
Comments may be sent to:
Lewis Golinker, Director
Assistive Technology Law Center
202 E. State St., Suite 507
Ithaca, NY 14850
lgolinker@aol.com
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HELP FIGHT ALS
Many people who know the devastating effects of ALS are providing lasting support for MDA's battle against the disease. Through your will, you can designate a gift to MDA earmarked to support ALS research or services.
Bequests to MDA can be made with cash, securities, real estate, or other property. You can bequeath a percentage of the entire estate to MDA or make a bequest of the residue, donating property remaining after all bequests to family and others have been satisfied. You may also name a memorial gift in honor of a family or individual.
Your attorney or financial adviser can help you work out the details of a bequest to MDA's ALS program. For more information, call MDA's Planned Giving Department at (800) 572-1717.
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