MYOTROPHIN 'POTENTIALLY APPROVABLE'
Companies in Talks With FDA
Myotrophin, an as-yet-unapproved, experimental treatment for ALS, received a yellow light May 12 from the Food and Drug Administration. The FDA's statement followed a May 1997 disapproval of Myotrophin by an agency subcommittee and postponement of a hearing scheduled for April 9 of this year that could have overruled that decision.
According to a May 12 press release from the drug companies Cephalon of West Chester, Pa., and Chiron of Emeryville, Calif., makers of Myotrophin, the FDA said the drug is "potentially approvable, subject to submission of additional information from ongoing studies which demonstrates that Myotrophin is effective in the treatment of ALS."
A committee appointed by the FDA to review material on Myotrophin last year raised questions about both safety and effectiveness. In a large trial of the medication in Europe, the percentage of deaths in the Myotrophin-treated patients was higher than that in patients not taking the drug (placebo group). In a similar trial in North America, however, the Myotrophin-treated patients showed a slower progression of disease than the untreated patients.
Myotrophin is based on a natural substance, insulin-like growth factor 1. It has to be injected.
The May 12 release notes that the companies plan to meet with the FDA "to clarify the conditions for approval and to determine whether these conditions can be satisfied." The release says "there can be no assurance that the FDA's conditions for approval of Myotrophin can be satisfied by the companies at all or in a reasonable time frame." In mid-June, Cephalon spokesman Jason Rubin said representatives of his company and Chiron are in discussions with the FDA regarding requests for additional information.
Myotrophin is still available through Cephalon to a limited number of people with ALS, Rubin said. This "expanded access program," a mechanism that allows people to use an experimental drug, has been in place for Myotrophin since mid-1996. According to Cephalon's Web site -- www.cephalon.com -- in early July, no new patients were being selected to receive Myotrophin through the expanded access program because of the uncertainty of Myotrophin's future. However, the company is still taking names for the program, according to information received through its 800 number.
To follow developments regarding Myotrophin, check the Web site or call Cephalon
at (800) 896-5855.
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 COMPANIES: BDNF IN SPINAL FLUID SAFE
Next Study Will Revisit Sub-Cu Delivery
The experimental ALS drug BDNF (brain-derived neurotrophic factor) was shown last year to be ineffective for ALS in a large, multicenter trial in which the drug was administered by subcutaneous (just under the skin) injection.
In the system developed by Medtronic of Minneapolis, BDNF enters the spinal fluid via an access port implanted under the skin in the abdomen. |
The drug companies Amgen (Thousand Oaks, Calif.) and Regeneron (Tarrytown, N.Y.), with the medical equipment company Medtronic (Minneapolis, Minn.), have been testing to see whether BDNF given directly into the fluid surrounding the spinal cord would be safe. Such a delivery route might prove more effective than subcutaneous delivery of BDNF.
Results released this April showed that BDNF delivered in this way was safe and mostly well tolerated in a trial of 25 patients with definite or probable ALS. After a 12-week, placebo-controlled trial, all participants received BDNF for at least a year.
Side effects were minimal except at high dosage levels. Two people at the highest dosage level and one person at the next highest level had their dosages reduced because of side effects (dry mouth, insomnia and agitation).
Three trial participants died during the study; the trial summary, published in Supplement 4 of the April issue of the journal Neurology, says they died of ALS, not of BDNF complications.
Jeanne Flynn, senior manager of professional services at Amgen, says the Amgen-Regeneron partnership will be conducting a trial to evaluate the effectiveness of BDNF delivered into the spinal fluid (known as "intrathecal" delivery) starting later this year.
Murray Goldberg, spokesman for Regeneron, says the companies are planning some very small, preliminary trials of subcutaneously delivered BDNF, despite the earlier failure of that delivery method, because later analysis suggested some patients may have benefited. They're not looking for participants for these trials now.
Amgen will coordinate the intrathecal trials, and Regeneron the subcutaneous trials.
Further developments regarding BDNF will be reported in this newsletter and through Amgen's information line at (800) 772-6436. You can also check the following Web sites, which periodically update information on BDNF: www.mda.org, wwwext.amgen.com and www.regeneron.com.
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MAJOR GIFT TO BAYLOR CENTER WILL SPEED ALS RESEARCH
The MDA/ALS research and clinical center at Baylor College of Medicine in Houston was recently the recipient of a substantial monetary gift from Ronny and Linda Finger. The Fingers have requested that the contribution be used to speed the Baylor center's research effort seeking effective treatments and ultimately a cure for ALS.
In acknowledgment of this outstanding voluntary initiative to help MDA vanquish ALS, the center, which operates under the direction of leading ALS clinician and researcher Dr. Stanley Appel, will henceforth be called the Ronny and Linda Finger MDA/ALS Center.
This gift of much-needed funding follows in the tradition of actions taken by the Kessenich family in Florida earlier this year. Through a large personal donation and the solicitation of further contributions from friends and associates, the Kessenich family amassed sufficient funds to launch the first MDA/ALS center in Florida, the Kessenich Family MDA/ALS Center at the University of Miami.
Dr. Appel's team at Baylor has been involved in trials relating to the development of IGF-1 (Myotrophin) and other agents of potential value in the treatment of ALS.
"I'm genuinely heartened and inspired by Ronny and Linda Finger's decision to make such a considerable personal investment in our Association's ALS research program," said MDA Senior Vice President and Executive Director Robert Ross. "Their generous support of Dr. Appel's work will most certainly hasten the day when the destruction caused by ALS will be halted once and for all."
The Ronny and Linda Finger MDA/ALS Center will complement the activities of the Vicki Appel MDA Neuromuscular Clinic at Baylor, which was named in honor of Dr. Appel's wife and professional colleague who died last year of cancer. Vicki Appel was a clinic coordinator, nurse and acknowledged authority on ALS whose name became virtually synonymous with the concept of quality care for people with ALS.
The Baylor center is one of 17 MDA/ALS centers across the country that combine clinical care and scientific research targeting ALS in one multidisciplinary setting. To view a list of MDA/ALS centers, check the Clinics section of MDA's World Wide Web site at www.mda.org.
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LONG ISLAND TEACHER RIDES FOR LIFE
Chris Pendergast has bigger things on his mind than the weather. So encountering rain on every one of the 14 days of his ALS Ride for Life from New York to Washington was no big deal.
The East Northport, N.Y., elementary school teacher, now in his fifth year with ALS, began his 325-mile journey on May 1 at the Lou Gehrig Memorial in Yankee Stadium in New York. Riding his electric scooter for eight to 10 hours a day on his way to Capitol Hill, he delivered his message directly to others -- curious onlookers, one of the nation's leading ALS researchers, members of Congress, state and city officials, and the president of the United States.
His message: People with ALS need more: more assistance from their government, more funds for research, more time.
Pendergast, who was featured on the 1997 national broadcast of the Jerry Lewis MDA Labor Day Telethon, pronounced the ride "extraordinary. It was an odyssey, it was an adventure, it was a dream."
On a personal level, the ride was "a statement that, with support, almost anything is achievable," he said. "And it's a testament not only for disabled people that while we are limited in certain ways, we are also limitless with our imagination and our desire to make a difference."
Pendergast, 48, was accompanied for much of the journey by Fernando Tedesco, another New Yorker who has ALS. During the trip, they were joined by others with ALS in the communities they passed through.
The motivation for the trip was the fact that at least 80 percent of medical research funding comes from the federal government, and Pender-gast's group wants to see an increase in the share of federal funds devoted to ALS. MDA, as the nation's largest private supporter of ALS research, funnels over $5 million a year into ALS research and services.
Pendergast's right hand on the journey was another teacher from his school district, Charlie Catania. With the backing of their district and other volunteer support, the two spent five months planning the trip. Catania and other friends, family members and co-workers of all ages rode bicycles, and the whole gang was followed by a van and a truck with a camper.
Everywhere they went, media coverage was extensive and police escorts were generous. Pendergast calls the police assistance in each jurisdiction "extraordinary."
"After meeting us, every one of them had a heart of gold, without exception," he said. "We ran into several officers who had family members or very close friends who in one way or another had been affected by ALS. If I had to pick one particular thing that made the trip as successful as it was, it was the cooperation of the police."
Pendergast made a point of visiting state capitals on the route: Trenton, N.J.; Dover, Del.; Annapolis, Md., and joining local residents to create ALS awareness. Other highlights were a visit to Rutgers University's chapter of Phi Delta Theta, Lou Gehrig's fraternity; an appearance at a Philadelphia Phillies game at Veterans Stadium; a stop at Chandler Hall in Newton, Pa., believed to be the only U.S. nursing home with an ALS wing; and celebration of Chris' daughter Melissa's 21st birthday.
At Dover Air Force Base, Pendergast met with President Clinton and spoke with him about ALS and had photos taken with him. Detouring from Annapolis to Baltimore, the group visited the MDA/ALS Center at Johns Hopkins University and met at length with codirector Jeffrey D. Rothstein.
After his arrival in Washington on May 13, there were meetings and a news conference with the Long Island congressional delegation, with three members enthusiastically endorsing H.R. 2009 (see story on page 5). He also participated in a march encouraging FDA approval of Myotrophin (see story on page 1).
The journey raised about $30,000, mostly from corporate donations and T-shirt sales. When figures are tallied, he plans to distribute funds to researchers, including Rothstein.
Meanwhile, Pendergast is planning the 1999 Ride for Life and maybe a 120-mile Montauk-to-Manhattan scooter marathon this summer. He'll also assist MDA with an October golfing event at Montauk Downs.
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"TASTE OF FAME" EVENT HONORS MAN WITH ALS WHILE RAISING $92,000
Bill Quinn, a Massachusetts resident who died of ALS last December, received a special tribute a few months before his death when family members put together a fund-raising event in his honor, the First Annual ALS Taste of Fame food and wine event on Oct. 4 in Springfield.
The sports-themed event, held at Springfield's Basketball Hall of Fame, raised $92,000 to support MDA's ongoing efforts to help people with ALS through research and services.
Quinn was an athlete and sports enthusiast who worked as vice president of W.J. Quinn Co. Inc., a family contracting, building and construction business.
Quinn's interests included golf, skiing, weight lifting, reading, political analysis and travel. Quinn was in his early 30s when he received a diagnosis of ALS in 1991.
A special note from Quinn's family was included in the ALS Taste of Fame program:
"This event is held in honor of our brother, Bill, in recognition of the courageous way he has fought this disease while maintaining his humor, positive attitude and love for those around him."
Although Quinn wasn't able to attend the event, he viewed videotapes of the remarkably successful fund-raiser before he passed away on Dec. 30.
Quinn's family, including his sisters, Jodi, Marianne, Kathy and Dianne, and his cousin Dan hit upon the idea of creating the Taste of Fame event early in 1997. More than 1,000 people attended, and the event also received support from MDA "Love Network" station WGGB in Springfield.
Twenty-five local restaurants provided hors d'oeuvres, while six beverage companies provided wine and microbrewery beer.
More than 100 items were bid on through silent and live auctions. The items included a photograph signed by baseball great Roger Maris, a football autographed by All-Pro quarterback Joe Theisman as well as art signed by "Peanuts" creator Charles Schulz.
After the successful event, the Quinn family wrote a letter to MDA, saying: "What began for us as a personal way of showing our love and support for our brother turned into much more. Before long our motivation began to extend far beyond our own family. If every family affected by ALS did what they could to raise funds and awareness for this disease, we would be that much closer to finding a cure."
Plans are underway for a Second Annual ALS Taste of Fame event to benefit MDA on Oct. 17 in Springfield. For more information, call MDA at (413) 567-5500.
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ALS TO BE FEATURED ON MDA TELETHON
MDA's 33rd annual Jerry Lewis Labor Day Telethon will again bring special attention to ALS.
One segment will be a visit with Dr. Stanley Appel, director of the Ronny and Linda Finger MDA/ALS Center at Baylor College of Medicine in Houston. Dr. Appel will help bring viewers up to date on MDA's ALS research program.
Telethon viewers will also see a profile of Ted Monoyos of Bethpage, Long Island, N.Y. Monoyos, 32, has ALS, and the profile will show the impact of the disease on himself, his wife and their young baby.
Many of the 200 stations on MDA's "Love Network" will also feature segments on ALS in local segments of the Telethon.
The Telethon can be seen across the United States and Canada on Sept. 6-7, beginning at 9 p.m. EDT on Sunday. Watch local television listings for exact times and stations in your area.
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 NEW LEADS IN SPORADIC ALS
When MDA-funded researchers found a gene defect associated with ALS in 1993, there was much rejoicing in the ALS community, and with good reason. The identification of the gene for SOD1 -- the enzyme superoxide dismutase -- led quickly to the first reliable animal models of the disorder and to the best hypotheses about motor neuron degeneration to date.
However, it's worth remembering that only about 2.5 percent of ALS cases are directly related to flaws in the SOD1 gene -- about a quarter of the 10 percent of ALS that's considered familial, the rest being considered "sporadic," occurring with no previous history.
For almost everyone with ALS, the underlying cause of the disease remains unknown, although motor neuron degeneration in all forms of ALS seems so far to be the same as that created in laboratory models using abnormal SOD1 genes and proteins.
RNA Abnormalities
In the last issue of The ALS Newsletter (vol. 3, no. 2, April 1998), we reported on a key finding by a team headed by MDA grantee Jeffrey Rothstein at Johns Hopkins University in Baltimore.
That group found that a protein that helps clear away glutamate from the area around motor neurons is damaged not because of a gene defect but because of what happens to the genetic code for this protein down the line. The problem isn't in the DNA for the glutamate-clearing protein; it's in how the DNA's chemical successor, RNA, is processed. This finding may help explain many cases of sporadic ALS, researchers say.
Mitochondrial Malfunctions
Recently, a group from Italy and the Netherlands reported on a patient with ALS who was found to have a mutation in a mitochondrial gene.
Mitochondria are the cell's "power plants." They resemble miniature cells but can only exist inside other cells. They have their own DNA, but their functions are also heavily influenced by DNA from the cell's nucleus.
Salvatore DiMauro, an MDA grantee in the Neurology Department at New York's Columbia University, says that, although DNA mutations in mitochondria may in fact be a rare cause of ALS, the phenomenon of mitochondrial involvement is well worth studying because other aspects of mitochondrial malfunction could result in motor neuron degeneration of the type seen in ALS.
SOD1 Protein Problems
MDA-supported researcher Dale Bredesen at the Program on Aging at the Burnham Institute in La Jolla, Calif., notes that changes in the SOD1 protein may occur with aging so that, even without a genetic mutation, SOD1 might conceivably become toxic later in life.
All these studies provide avenues for new animal models and new pathways to ALS treatments.
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SUPPORT GROWING FOR ALS BILL
The Amyotrophic Lateral Sclerosis Research, Treatment and Assistance Act, introduced in June 1997, now has about 130 sponsors in the House of Representatives. But committee hearings, which are required before the bill can be voted on, hadn't been scheduled as of press time.
Randolph Harrison, a spokesman for the bill's primary sponsor, Rep. Lois Capps, D-Calif., said Capps is working with members of the Commerce and the Ways and Means committees to schedule hearings. She's also asked the Appropriations Committee to consider increasing federal funding for ALS research.
The major provision of H.R. 2009 would waive the 24-month waiting period for Medicare eligibility on the basis of disability for those with ALS. The bill would also provide Medicare coverage for outpatient drugs and therapies for ALS. In addition, it would double federal funding for ALS research.
Hearings have yet to be scheduled for two other bills of concern to families affected by ALS.
The Family Caregiver Enumeration Act of 1997 (H.R. 2081/S. 1206) would require the Census Bureau to gather information on family caregivers in the census of 2000. Family caregivers provide two-thirds of all home care services in this country, and counting them during the census would be an important step toward forming policies to support and recognize their contributions.
The Medicaid Community Attendant Services Act, also known as "CASA" (H.R. 2020), introduced by House Speaker Newt Gingrich last June, would provide for Medicaid coverage of qualified attendant care services for individuals in their homes or in community-based settings such as schools, workplaces and recreation or religious facilities. As of press time, CASA had 68 cosponsors. There are several other bills on this topic in Congress.
Harrison said that, between now and adjournment in early October, members of Congress will be largely focused on the appropriations process and the November elections. Capps will introduce H.R. 2009 in the next congressional session if action isn't completed this year.
In the meantime, the bills need additional cosponsors. A letter, phone call or visit explaining what ALS is, and how the law would help you, is the best way to convince your congressional representative to sign on as a cosponsor.
You can write to: (Name of Representative), House of Representatives, Washington, DC 20515; or (Name of Senator), U.S. Senate, Washington, D.C. 20510; or The White House, Washington, D.C. 20500. You can also write to your representative's local office, or get the exact office address and phone number from either of two congressional Web sites: www.house.gov or thomas.loc.gov.
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ALS CLAIMS KAY POSTON
by MDA Senior Vice President and Executive Director Robert Ross
As we were going to press with this issue, we learned the terrible news that Kay Poston, wife of beloved television actor Tom Poston of "Newhart" fame, passed away.
Kay had ALS. She and Tom appeared on our Telethon first in 1995, when Kay was still able to walk with the use of a cane. They returned in 1997 to talk about the further impact that Kay's ALS had had on their lives, by which time the disease progression had made the use of a wheelchair necessary.
One thing was for certain. ALS never altered the deep, abiding affection that Kay and Tom had for each other and for their two grown children, daughter Hudson and son Jason.
"She's very much loved," Tom said in the special video profile that preceded his live appearance with Kay on last year's Telethon broadcast. "I want people to know that in spite of this terrible disease, it's possible to be a person with a very great spirit, because my wife is a champion. We're all very proud of her." Hudson and Jason both appeared in the profile to testify to their mother's courage in the face of ALS and to express their wish for a way to stop the disease progression in time to save Kay's life.
At all times, Kay herself was a model of dignity, honesty and true eloquence. In the video, she recalled her years as a professional dancer and expressed her amazement that, due to ALS, she was no longer master of her own body. She spoke of the incredible frustration she felt as she willed with all her might just to move her right foot, without success.
Yet, during the live remarks that she and Tom made to Jerry Lewis following the video, Kay acknowledged the positive feelings that came from knowing that so many people wanted to help MDA defeat "this catastrophic disease."
"You begin to see such goodness in human beings," Kay said. "I am so struck by the kindness and the generosity that I have been witness to, given my situation. That's the gift that I have received."
All of us at MDA extend our deepest sympathies to Tom and his family. We'll remember Kay fondly for her loving nature, her grace, her humor and her willingness to share her story and help MDA increase awareness of the need to stop the destruction caused by ALS once and for all.
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BOOKS
Tuesdays With Morrie by Mitch Albom, 1997. 192 pages, $19.95. Doubleday, available in bookstores.
This nonfiction book describes the relationship between a sociology professor with ALS and one of his former students.
Rather than primarily a story about ALS, it's an intimate look at a man's reflections on life as he faces his illness and death, and the impact those thoughts have on Albom, the sports reporter and former student he shares them with. Morrie Schwartz, a Brandeis University professor who was featured on ABC's "Nightline" with Ted Koppel three times before his death in 1996, was a remarkable man who continued to love life even as ALS took it from him piece by piece.
Schwartz helps Albom, and readers, understand how much our lives are affected by our attitudes toward death, regret, family, aging, money, love and forgiveness. Schwartz's own book, published in 1996 by Walker & Co., is Letting Go: Morrie's Reflections on Living While Dying.
Albom gives a realistic picture of the physical deterioration brought on by ALS, while showing that the disease concentrated Schwartz's spirit rather than diminishing it.
The Diving Bell and the Butterfly by Jean-Dominique Bauby, 1997. 132 pages, $20. Alfred K. Knopf, available in bookstores, www.randomhouse.com.
Another exploration of the mind and spirit during illness is found in The Diving Bell and the Butterfly. The author, a French magazine editor, woke after a rare brainstem stroke to find himself "locked in" -- mentally intact but unable to move or speak -- a condition similar to that of some advanced ALS patients.
By blinking his left eye in response to an assistant's reading of an alphabet board, Bauby wrote this memoir. He muses on practical concerns -- learning to swallow excess saliva, communicating with hospital staff, combating rumors that he's become a "vegetable" -- and reflects on the experiences and people who've given meaning to his life. Most interestingly, Bauby uses his imagination to relive sensory experiences -- the smells, sights and sounds of his worldwide travels; the tastes of a fine meal; sleeping beside the woman he loves.
Bauby died shortly after his book's initial publication in French. But he, like Schwartz and others forced into immobility, left readers the gift of his insight into the inner life: "I can listen to the butterflies that flutter inside my head. To hear them, one must be calm and pay close attention, for their wingbeats are barely audible."
Making Moments Count: Leisure Activities for Caregiving Relationships by Joanne Ardolf Decker, Ph.D., 1997. 192 pages, $14.95 paperback, $29.95 hardcover. Johns Hopkins University Press, 2715 N. Charles St., Baltimore, MD 21218-4319.
Decker, a therapeutic recreation specialist, offers detailed strategies for building leisure into the caregiving experience. Separate chapters cover physical, intellectual, emotional and expressive, social, and spiritual activity, and each contains many examples and suggestions for specific activities. Practical checklists help caregivers determine realistic leisure ideas. The book can be used by professional, family or volunteer caregivers.
Decker emphasizes the importance of sharing personal moments such as reviewing a snapshot album and simple pleasures like an evening breeze and a sunset. Such moments, she points out, become memorable and meaningful breaks from the demands of caregiving, both for the giver and the receiver.
Are You Tired Again? I Understand by Marilyn Weisberg Deutsch, Ph.D., 1998. 42 pages, $14.95 (discounts for bulk orders). Western Psychological Services, (800) 846-8857.
With appealing illustrations by Gary Cangemi, this workbook helps children understand certain disabilities that may affect their parents or other adults close to them. The workbook may be used by individual children or in support groups. It includes opportunities for children to color pages, discuss feelings and ask questions about their parents' illnesses.
The text reassures youngsters that they didn't cause the problem and suggests ways they can help. The author has lupus, but the book could also be informative to children of those with ALS.
The Patient's Voice: Experiences of Illness by Jeanine Young-Mason, Ed.D., R.N., 1997. 240 pages, $21.95. F.A. Davis Company, 1915 Arch St., Philadelphia, PA 19103, (800) 323-3555.
Young-Mason, a professor at the University of Massachusetts School of Nursing, has gathered personal accounts from children and adults with a variety of serious disorders. These frank stories give much insight into the experience of being hospitalized, the effects of drugs, changing relationships with friends and family, and dealing with the medical community.
There's no account of ALS, but powerful stories by people with muscular dystrophy, Parkinson's disease and cancer shed light on caregiving, spiritual growth, altered self-concept and other aspects of chronic illness. Readers affected by serious diseases will find they aren't alone in their intense responses to their experiences.
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HELP FIGHT ALS TODAY AND TOMORROW
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.
To give what remains of your estate after other bequests have been satisfied, just include the following language in your will:
"I give, devise and bequeath all (or a specified fraction of) the rest, residue and remainder of my estate, whether real or personal, of every kind and description, and wherever situated, to Muscular Dystrophy Association Inc., a New York not-for-profit corporation having its principal office at 3300 East Sunrise Drive, Tucson, Arizona, 85718-3208, for its program of research and services related to amyotrophic lateral sclerosis."
To give a dollar amount or percentage of your estate: "I give, devise and bequeath the sum of $________ (or ________ percent of my estate) to Muscular Dystrophy Association Inc., a New York not-for-profit corporation having its principal office at 3300 East Sunrise Drive, Tucson, Arizona, 85718-3208, for its program of research and services related to amyotrophic lateral sclerosis." 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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The Association welcomes gifts for ALS research honoring significant occasions of achievement. These gifts may be made in tribute to special people or to mark such events as anniversaries, birthdays, weddings, graduations or retirements.
THE ALS NEWSLETTER
Muscular Dystrophy Association
National Headquarters
3300 East Sunrise Drive
Tucson, Arizona 85718-3208
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