Telethon 2007: Fueling the Fight Against ALS

For more than four decades, Jerry Lewis has worked overtime on Labor Day weekend to raise funds to defeat neuromuscular diseases. In that time, the Telethon has brought in more than $2 billion for MDA’s programs of help and hope.

Entertainment Tonight's Jann Carl will again serve as co-host of the Jerry Lewis MDA Telethon.

Broadcasting from Las Vegas, Lewis and longtime anchor and friend Ed McMahon will orchestrate a marathon event that alternates between premier entertainment and the serious business of raising awareness of deadly illnesses like ALS.

Prominent again among this year’s spokespersons will be Augie and Lynne Nieto of Corona del Mar, Calif., co-chairs of MDA’s ALS Division.  Another familiar figure will be R. Rodney Howell, M.D., new chairman of MDA’s Board of Directors. Howell will update viewers on the status of MDA research.

New Telethon faces include 34-year-old Scott Stafne of Minneapolis. Stafne, who faces ALS with spirit and determination, will share his story, accompanied by his wife, Kristen, and 2-year-old daughter, Eva.

The 2007 Jerry Lewis MDA Telethon begins at 9 p.m. EDT on Sept. 2 and runs non-stop for 21½ hours. To find your local station, visit www.mda.org/telethon/findyourstation.html. The show also is available online by way of streaming video provided by RealNetworks.

Wind Beneath MDA's 'Wings'

The seventh annual Wings Over Wall Street gala will be held Sept. 20 at the New York Marriott Marquis in Times Square. Supported by celebrities and financial leaders, the event has raised more than $6 million to benefit MDA’s ALS research program.

The funds assist the research teams of Hiroshi Mitsumoto, co-director of the Eleanor and Lou Gehrig MDA/ALS Research Center at Columbia University in New York City, and Jeffrey Rothstein, director of the MDA/ALS Center at Johns Hopkins University in Baltimore.

Four awards also will be given to leaders in the fight against ALS.

Neuroscientist Don Cleveland, head of the ALS research laboratory at the University of California, San Diego, will receive the Diamond Award, named for the event’s co-founder, Toni Diamond, who died of ALS in November 2004.

Dorothy and David Deutsch of West Sayville, N.Y., will receive the Spirit Award for their commitment to raising ALS awareness and funds for research. David, 39, the father of two young children, received an ALS diagnosis in 2004.

Lisa Marie Utasi, director and senior equity trader at ClearBridge Advisors in New York City, will receive the Michael P. Beier Award, which honors a person who motivates others to help find a cure for ALS.

Larry Schiffer of East Rockaway, N.Y., a partner in the international law firm of LeBoeuf, Lamb, Green & MacRae LLP, will receive the Humanitarian Award. Schiffer’s late sister-in-law, Toni Diamond, and brother, Warren Schiffer, founded the Wings event.

For more information, visit www.wingsoverwallstreet.org, call (212) 689-9040, or e-mail
specialeventwings@mdausa.org.

ALS TDI Says Tighter Controls Needed in Mouse Studies

by Margaret Wahl

Failure to recognize differences among mice with mutated SOD1 genes may be the key to understanding why so many medications that looked good in mouse studies later failed to benefit humans with ALS, say researchers at the MDA-supported ALS Therapy Development Institute (ALS TDI) in Cambridge, Mass.

Another possible explanation for why mouse-study results have consistently failed to pan out in human trials may be that ALS caused by an SOD1 gene mutation isn’t the same as non-SOD1 ALS. (See “Are SOD1 Mice Good Models of Human ALS?” in the July-August 2007 issue.)

ALS TDI president Sean Scott says overlooking factors such as mouse gender, litter of origin, and number of mutated SOD1 genes per cell, as well as not putting enough mice into a study and including mice with infections or other conditions, may have affected study results and caused investigators to misinterpret “noise” in the system as an actual survival benefit conferred by an experimental medication.

When the ALS TDI repeated trials using mice with SOD1 mutations, using more mice and tighter controls, they found that the drugs that failed to benefit human patients also failed to show benefit in the animals. They repeated trials of minocycline, creatine, Celebrex and others, and found none of them effective in the mice.

“I’m of the camp that believes everything we have seen in the mice clinically, molecularly and cellularly matches up to humans,” says John Lincecum, associate director of research biology at ALS TDI.

He says the mouse model predicts the human response very well, albeit negatively, which is “a very different conclusion from saying the mouse model doesn’t work.”

“We’ve become more sophisticated, and we have better tools,” Lincecum says. “We have a lot more resources available than we did 13 years ago, when this mouse model became available.” In his view, “the bar needs to be set a little higher before people jump from a mouse model to a human trial.”

Not everyone agrees with the TDI findings, including MDA grantee M. Flint Beal at Weill Medical College of Cornell University in New York. “I believe that they are very well-intentioned,” Beal said of the TDI group. “The fact, however, that they cannot replicate the data of any other academic laboratory within the United States gives one great concern.”

Sharon Hesterlee, MDA vice president of translational research, says, “this is obviously a very touchy subject, but it bears further investigation.

“The information that academic laboratories have given us by studying smaller numbers of SOD1 mice over the last several years has produced several good leads, but we need to make absolutely sure that the results from mouse studies are airtight before we go to humans. Every time you launch a large, multicenter human clinical trial, there are ‘opportunity costs’ in the form of other research projects that can’t be funded or other trials that may not have enough participants. We owe it to those we serve to make wise choices.”

Life in the Fast Lane

by Amy Labbe

Darlene Bates of Lee’s Summit, Mo., has a thing for checkers. Not the board game, but the black-and-white painted squares that mark the beginning and end of a stock car race.

Bates, who received a diagnosis of ALS in November 2001, is a NASCAR fan who watches the races on television every weekend, rooting for her favorite driver, Jimmie Johnson, 2006 NASCAR Nextel Cup Champion and driver of the Lowe’s-sponsored No. 48 Chevrolet. (Lowe’s also is an MDA national sponsor.)

Despite being unable to move her arms and legs, this June, Bates took her passion for NASCAR one step further than most, with a 180-mph ride in a stock car at Kansas Speedway in Kansas City.

The Need for Speed

Bates uses a chin-controlled joystick to control her wheelchair.

Lyn Rebel, Bates’ hospice social worker at the time, made it all happen. After hearing Bates comment once that she wished she had gone sky diving, and knowing of her love of NASCAR, Rebel decided to try to secure her the ride of a lifetime.

Rebel checked first to make sure Bates, who uses a power wheelchair and Mercury communication device, was physically up to the experience. She then spoke with Bates’ daughter and caregiver, Jane Boyd, to see if she thought it was something her mom would enjoy.

“Jane told me to go for it,” Rebel says.

Rebel next contacted The Racing Experience, a stock car racing school that gives people the opportunity to either pilot a stock car in near-racing conditions, or to see how the professionals do it as viewed from the passenger seat. President and CEO Kelly Bussey happily agreed to donate a racing experience to Bates.

“He also said his crew would do everything possible to get Darlene into a race car so she could experience the thrill of riding,” Rebel says.

With the offer in place, the next thing to do was tell Bates, which Rebel did on her next home visit.

“I’ll never forget Darlene’s reaction,” Rebel says. “She just laughed and laughed until tears were coming down her cheeks.”

At the Drop of the Green Flag

Bates was originally scheduled for a ride in May, but her NASCAR adventure had to be postponed twice due to rain. June 15, however, dawned sunny and bright, and the temperature climbed to a sweltering 91 degrees.

Bates, in firesuit and helmet, is strapped in place ready to go. Her ride was a 600-horsepower Ford Fusion.

Bates spent nearly two hours at the track, accompanied by her hospice nurse, Brian Ghafari-Naraghi; hospice volunteer, Sarah Glavin; friend Jim Wilson; daughter Jane; granddaughter Shae Boyd; and Kathy Peters, Bates’ friend and MDA ALS health care service coordinator.

After checking in at 9 a.m., Bates spent time soaking up the ambiance of the speedway. Not one to miss out on anything, she included a trip through the garage area, where she was treated to an up-close look at some of the cars.

“The track smelled like hot tires and gas,” she says.  “They had a bulletin board where they would write your name. Once your name went on the board, you had to get into a firesuit and wait to be called.”

When her turn finally came, Bates made her way to the car surrounded by friends and family. Getting into the car — through the window, the way NASCAR drivers do — wasn’t exactly easy.

“Brian, the hospice nurse, had made Velcro straps,” Peters says. “We strapped her legs and thighs together with those, then three strong young men picked her up and set her in the window with her feet in the car and her body still out. Another man crawled in headfirst over the driver with his feet sticking out the driver’s window and helped pull her legs in and get them positioned comfortably.”

The men made sure Bates’ neck and head were supported, as everyone tugged and pulled to get her comfortable, and then the helmet went on.

“When we added the helmet it was too heavy, and her head fell so that she couldn’t see where she was going,” Peters says. “Out came the Velcro. Her head was Velcroed in place, she was belted in and, with a huge smile, she was off.

“By the time we got the wheelchair and all of us off the infield, the driver radioed back from Turn 4 saying she was smiling from ear to ear.”

Bates estimates she spent 10 “awesome” minutes in the car, the high point of which were the three laps she sped around the track.

“It was loud when my driver first started it, but we took off and it was great,” she says. “He kept asking me on the ride if I was OK. I was laughing and having the time of my life.

“I just loved it when he changed gears and I knew we were going to go faster,” she adds.

Bates says the experience has given her a greater appreciation for her Sunday heroes.

“It was sure hot,” she says. “I certainly have a new admiration for the NASCAR drivers who spend hours in those cars. I don’t know anyone else who endures such extreme conditions.”

The Checkered Flag Waves

Bates' friend and caregiver, Jim Wilson, shared her special day at the track.

Bates’ NASCAR ride may have come and gone, but life at high speed — even with ALS — continues to be her style. She attends her grandchildren’s ballgames, goes to church every Sunday and never misses an opportunity to enjoy life.

“What a fabulous woman,” Peters says of Bates. “She truly does epitomize living a full life no matter the circumstances.”

Bates tells everyone those few minutes in the car left her “exhilarated, thrilled and happy — never scared.” And though she topped out at a lightning 180 mph, it’s a testament to her determination to get the most out of every minute that she adds, “Yes, indeed, I would have gone faster.”

Thinking Outside the Ventilation Box

by Margaret Wahl

New York author Philip Carlo first started showing the symptoms that eventually would be diagnosed as ALS in February 2005.

“I was coming back from doing an interview of Richard the Ice Man at Trenton [N.J.] State Prison,” recalls the 58-year-old Carlo, who has published five books of true and fictional crime and has a sixth one (on the Mafia) under way.

“I was in Penn Station, and my left foot was flopping on the marble floor. I was in excellent physical condition, and I thought I had some problem attributable to running too much.”

For the dedicated cyclist and runner, ALS was “a very hard pill to swallow,” he says, and he soon started searching “outside of the box” for help.

Carlo’s search led him to Raymond Onders, a general surgeon  at University Hospitals of Cleveland who’s dedicated to developing an alternative to existing methods of assisted ventilation.

If It Contracts, You Breathe

Augie Nieto, whose ALS was diagnosed in 2005, says he can now breathe while lying flat.

Rather than pumping air into the lungs, which is how ventilators work, Onders has pursued a different strategy to assist breathing: stimulating the diaphragm, the flat, pancake-like muscle that sits just under the lungs and moves up and down to allow the lungs to fill and empty. (When you take a breath, the diaphragm contracts and moves down. When you exhale, it relaxes and moves up.) The use of this device in ALS is somewhat controversial. (See “Stimulating the Diaphragm,” January 2007.)

A few years ago, Onders began surgically implanting electrodes in the diaphragm muscles of patients with spinal cord injuries who couldn’t breathe on their own. When the electrodes are stimulated through wires outside the skin, the diaphragm contracts. The strength and rate of the contractions can be regulated to suit individual needs.

“If you look at recovering arm function, it’s complex,” Onders says. “But with the diaphragm, if it contracts, you breathe.”

He soon began implanting diaphragm stimulators (also called pacers) in people with ALS who were losing respiratory function. “Patients with ALS develop nighttime sleep dysfunction,” he says. “The diaphragm works differently in the daytime and in the nighttime. There are [centers] in the brainstem that control night breathing, and that part of the brainstem, which controls the diaphragm, is affected in ALS.

“BiPAP [bilevel positive airway pressure] will help patients when they don’t breathe at night, but when you stimulate the diaphragm, that’s natural breathing.” People don’t notice anything different from normal breathing, he says.

Carlo got his diaphragm stimulator in Cleveland in March. “Overall, I highly recommend it,” he says. “I find it helps me sleep better and has put more power in my cough. I can actually cough now, where before I had this little kitten cough.”

Decision was a ‘No-Brainer’

For Augie Nieto of Corona del Mar., Calif., a leader in the fitness industry, ALS became a reality in March 2005. The 49-year-old entrepreneur and his wife, Lynne, have since become co-chairpersons of MDA’s ALS Division and helped launch MDA’s Augie’s Quest, an ALS research initiative.

In May, Nieto went to see Onders in Cleveland and get a diaphragm stimulator. “Every time I make a decision, I think about what the risk is to my quality of life and measure it with the possible benefit,” Nieto says. The diaphragm stimulator decision, he says, was a “no-brainer.”

“There are so many charlatans out there preying on ALS victims, whether it’s inserting stem cells in China or some other exotic treatment,” Nieto says. “This is for me the best investment I’ve ever made.” (His insurance plan didn’t cover the procedure.)

Nieto says he now can lie flat on his back and breathe without difficulty, something he couldn’t do before getting the stimulator. However, he says he continues to use his BiPAP device during the night because he believes it helps his lungs stay well inflated and not become brittle.

“Augie was one of the first patients to have his diaphragm electromyogram [record of muscle activity] serially followed,” Onders says. “There has been significant improvement in Augie’s diaphragm EMG.”

Study Remains Open

Onders says he thinks diaphragm stimulation works in at least two ways: It causes the person to breathe at a rate and depth that’s comfortable, and it also appears to condition the diaphragm, keeping it from deteriorating and perhaps actually strengthening it.

Onders believes the device can extend survival and delay ventilator use in ALS. “They may get an extra year or so not on a ventilator,” Onders says. “But they can also use their device along with a volume ventilator or BiPAP.”

For now, if you have ALS and want a diaphragm pacer in the United States, the Food and Drug Administration requires you to be part of Onders’ study. It’s open to people who meet the criteria in Cleveland, Baltimore and Stanford, Calif., with an anticipated additional seven centers to open soon.

As of mid-July, Onders’ team in Cleveland had implanted pacers in 26 people with ALS and scheduled another 25 implants.

Some insurance plans cover the procedure, which has received “category B” approval from the FDA. Medicare considers category B devices, which are deemed essentially safe but still under investigation, “eligible for coverage,” but all the agency’s criteria must be met.

For those without third-party coverage, the cost of the procedure is about $5,000.

For details of Onders’ study, contact MaryJo Elmo at (216) 844-8594 or maryjo.elmo@uhhs.com; or see “Diaphragm Conditioning with Motor Point Stimulation”.

A Possible Biomarker for Clinical Trials

In addition to its possible therapeutic effects, Onders is enthusiastic about using the diaphragm pacer as a “biomarker” for ALS clinical trials.

With its ability to detect natural diaphragmatic muscle contraction when the electrodes are not stimulated, it could provide a reliable indicator of whether a therapy that’s supposed to stabilize or improve muscle strength is working, he says.

Social Workers Offer a Wealth of Resources

by Kathy Wechsler

Social workers can play many important roles during your journey with ALS.

Clinic Team Member

Multidisciplinary clinic teams at MDA/ALS Centers often include social workers as part of their vital services.

Sheilah Blansit, a licensed clinical social worker (L.C.S.W.) at the MDA/ALS Center at Texas Neurology in Dallas, works with local MDA ALS Health Care Services Coordinator Carolyn Minnerly to get newly diagnosed individuals into the center’s ALS clinic quickly to address immediate concerns.

“What ALS clinics do best is get patients and families together who would otherwise never cross paths,” said Blansit, who’s been a social worker since 1988. “They get to know and care about each other, and a bond develops that is indescribable. I think it’s that bond that helps more than anything else.”

Starting at the time of diagnosis, social workers follow people with ALS throughout the course of the disease, assessing needs and making recommendations at clinic visits, over the phone and on home visits.

Working in conjunction with physicians, nutritionists, counselors, and occupational, physical and speech therapists, social workers are tuned into the whole person, said Dave Burkett, an L.C.S.W. with the University of Kansas Medical Center MDA/ALS Center in Kansas City.

Resource & Case Management

Social workers have many resources to offer those affected by ALS.

“I like to think we’re experts in the community services that are available to individuals, how to direct people in obtaining those services, and some of the specific problems in accessing services,” said Burkett, who has almost 30 years of experience as a social worker.

Social workers make people aware of what’s available to help them better function at home and meet their future needs. They explain and help people with creating advance directives and applying for Social Security Disability Insurance, Medicare and Medicaid benefits.

Hospice social workers, who work in homes, hospitals or hospice facilities, deal with end-of-life issues, answering questions about hospice and helping with the transition to a hospice.

John Hickman and his wife, Edna, attend a clinic at the MDA/ALS Center at Methodist Hospital Neurological Institute in Houston, where they see Rebecca Axline, an L.C.S.W. on the clinic team. The Hickmans, of New Carney, Texas, have nothing but good things to say about their social worker.

“She has so much knowledge — she can help us with insurance, home health and hospice,” said Hickman, 70, who received a diagnosis of ALS in 2005. “If we ever have to go to hospice, she knows all about that. She has been a terrific outgoing person, and she volunteers all that information.”

John, who has weakness in his legs, arms and shoulders, uses a power wheelchair. His speech also is affected.

Edna said that Axline is the first expert she would call if she ever needed anything for her husband.

“When John was in the hospital in April to get a feeding tube, she would come by the hospital to visit with him and be sure that we had everything we needed,” said Edna. “She also helped with finding a home health agency that would come out until I got adjusted to feeding him with the feeding tube.”

Comfort & Support

Burkett, who specializes in mental health, offers solid support to individuals with ALS and their families who are struggling to cope with the psychological aspects of having a life-threatening disease and the many changes and losses due to disease progression.

As part of his mental health role, Burkett helps people manage their emotional concerns, using depression scales to determine if they have clinical depression and should see a psychiatrist for medication.

He also eases transitional concerns related to a new diagnosis of ALS. People come to clinic frustrated with the physical and emotional changes going on in their lives, such as no longer being able to work, or needing help getting dressed, or trying to cope with the loss of independence.

“Our role is to help them with that transition so their personal needs are met and we can begin the process of care planning for their future needs,” said Burkett.

Social workers with an L.C.S.W. designation also sometimes provide private counseling outside of the clinic setting. These private sessions are covered by Medicare and some insurance providers.

Many social workers also serve as MDA Support Group facilitators.

Once a month, the Hickmans attend the MDA/ALS caregiver support group, where their social worker, Axline, is a facilitator. Hickman’s wife, Edna, says that Axline has been an emotional resource.

“She’s a tremendous help with knowing what the caregivers need,” Edna said. “And she encourages us to get the help we need and to take time for ourselves.”