Our mission: mission Leading the fight to treat and cure ALS through global research and nationwide advocacy
In My View:
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By Susan Woolner, Executive Director
while also empowering people with Lou Gehrig’s Disease and their families to live fuller lives by providing them with compassionate care and support.
Our Vision: Create a world without ALS. Staff
Board of Directors
Susan Woolner Executive Director ed@alsa-michigan.org
John Crosby President
Kristen Munyan, RN, MSN-Ed Care Services Manager, East kristen@alsa-michigan.org
Matt Wey* Past President, and voting member, National ALSA Board of Representatives
Robbie Banfill Care Services Coordinator, West rbanfill@alsa-michigan.org Paula Morning Development Director, East pmorning@alsa-michigan.org Vicki Bouwkamp Development Manager, West vicki@alsa-michigan.org Cathryn Rybicki Accountant crybicki@alsa-michigan.org Julia Bauer “Michigan Matters” editor news@alsa-michigan.org Website: www.alsa-michigan.org Email: fightals@alsamichigan.org
West Michigan Office 678 Front St., Suite 410 Grand Rapids, MI 49504 Phone: 616-459-1900 Toll-free: 800-387-7121 Fax: 616-459-4522 Skype: alsawestmichigan East Michigan Office 675 E. Big Beaver Rd., Suite 207 Troy, MI 48083 Phone: 248-680-6540 Toll-free: 866-927-CURE Fax: 248-680-6541 Skype: alsamichganeast
Ed. Note: Have news or compelling story? E-mail your ideas to news@alsamichigan.org
J.D. Concepcion Chip Hurley Pat Moran Todd Noeske Jeffrey Swain Craig VanSplunter Susan Woolner Advisory Board Member Daniel Newman, M.D. Honorary Board Members Pamela Bouma Lew Chamberlain Ed Dobson, Ed.D. Randy Ernst Eva Feldman, M.D., Ph.D. Mike Melcher, former board president
Michigan ALS Centers Harry J. Hoenselaar Clinic Henry Ford Hospital 2799 West Grand Blvd K11-Neurology Detroit, MI 48202 313-916-2835 Dr. Daniel S. Newman www.henryford.com/neuro
U. of M. Health System 1914/0316 Taubman Center 1500 E. Medical Center Drive Ann Arbor, MI 48109 734-936-9020 Dr. Kirsten Gruis, Director Michigan ALS Clinic
The MSU at Mary Free Bed Rehabilitation Hospital 360 Lafayette Suite 308 Grand Rapids, MI 49503 616-493-9727 Dr. Deborah F. Gelinas Medical Director
For many readers, this may be my first contact with you as the new Executive Director of The ALS Association Michigan Chapter. I’ve been involved with the ALS community in Michigan for more than 17 years and have served the Chapter as a board member for better than a year. This summer I stepped in as temporary executive director, to fill in for Stacey Orsted during her leave of absence. After Stacey notified the board in mid-August that she would not be returning to the role, I accepted the executive director position full-time. Because of my personal connection to ALS (my father-inlaw died of ALS in 1994), I know first-hand the value of The ALS Association, our involvement with local ALS Clinics, our leadership role in patient care, advocacy and ground breaking research. I’m grateful for the opportunity to serve the extended ALS Community in Michigan and look forward to meeting many of you face-to-face at one of our five upcoming Fall Walks to Defeat ALS. As you will read in “Michigan Matters” research updates, investigators have identified a new gene linked to familial ALS. Indeed, this is a promising time for ALS research. You have the power to take our research and the fight against Lou Gehrig's Disease to the next level, and fortunately, there is an easy way to get there by giving through payroll deductions. You can contribute to The ALS Association through Community Health Charities (CHC), a federation of premier health organizations that have joined together to raise charitable contributions in the workplace. Your gift will not only fund The Association's global research endeavors, but also our Chapter's care services programs and public policy efforts. To find out how you can participate in the CHC workplace giving program, please visit our website. Your gift – no matter how large or small – will have a profound impact on people living with this disease. Susan Woolner
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Inside Information Walkers stomp on ALS ...................... 4 Iron Horse, Gears & Beers ................. 6 Meet Don and Annette ...................... 8 Hello, Susan Woolner ...................... 10 ALSA-Michigan news ........................ 11 Breakthrough: cause of ALS found?. .... 12 Connecting the dots: ALS research...... 14 Didja Know? Briefs of interest ........... 19 We remember.................................19 Events, Support Groups abound ......... 20
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Walkers, rollers prepare to launch all across Michigan. The aim? Stomp on ALS! Half of life is showing up! Here’s where:: Sept. 11 — Jackson Check-in 9 a.m., Walk 10 a.m. Ella Sharp Park, 2800 4th St., Walk 1 mile
Sept. 17 — Detroit Check-in 9 a.m., Walk 10 a.m. Detroit RiverWalk, on the Riverfront, 1340 Atwater St.; Walk 1.4 miles
Sept. 18 Grand Rapids Check-in 1 p.m., Walk 2 p.m.; Ah-Nab-Awen Park 303 Pearl St. NW Walk 2 miles
Sept. 24 — Portage Check-in 1 p.m., Walk 2 p.m. Celery Flats, 7335 Garden Ln.; Walk 3 miles
Oct. 1 — Traverse City ,Check-in 10 a.m., Walk 11 a.m. Civic Center Park, 1213 W. Civic Center Drive; Walk 3 miles
All month — Michigan Virtual Walks
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Top 10 reasons to hit the pavement for Michigan’s Walk to Defeat ALS this month: (apologies to David Letterman) 10. It’s the last month you won’t have to wear snow boots outdoors. 9. It’s the biggest single fundraiser for the ENTIRE year for ALSA Michigan! 8. You’ll get out of your rut and onto the sidewalk, with a bunch of friendly people. 7. Your team’s donation will help resupply the ALSA loan closet, with things like wheelchairs, bath seats, even talking computers. 6. The research grant your donation supports could find a cure for ALS. 5. Money from your trek will help patients coming to Michigan’s ALS centers in Detroit and Ann Arbor, and its ALS clinic in Grand Rapids. 4. Your co-workers who don’t walk will be sorry when they hear how much fun — and money — your team raised to defeat ALS. 3. When you cross the finish line, you’ll find great munchies (thanks, sponsors!) to reward your effort. 2. Walking is good for you, and on this day, it’s good for ALL ALS patients and caregivers in Michigan, too; and finally, the top reason to join the 2011 Walk to Defeat ALS? 1. Because you can!!!!
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Iron Horse Riders logged more than 7,100 miles in two days of hill-anddale courses in June, and teamwork made all the difference. By the time the last team pedaled in, the dual effort marked plenty of firsts:
First time event expanded to two locations: original has been in Ann Arbor since 2007; Lowell was new this year. First time PALS have biked in the event (two made history) Best fund-raising in history of the event: nearly $45,000 to date
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Great teamwork, great weather, and a great pyramid of sponsors created two sweet days of bicycling, first in Ann Arbor, then in Lowell in June. Thanks to ALL who rode, cheered, and contributed to our Iron Horse Ride, and the refreshing Gears and Beers afterparty!
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By Don Barnowski, Howell The neurologist paused. "… or it could be something called ALS. Have you heard of that? Yeah, it only seems to hit the good guys...". That's when the reality of what we were dealing with really hit. Confirmation four weeks later actually brought a strange kind of relief since months of uncertainty was now over. That numbing day in July 2010 changed our family forever. But at least we knew what we were fighting. The Barnowski Family: Don, kids Emily and Nick, and wife Maggie
And fight we do, using all the arrows in the quiver. Our family's core values encompass faith, family & friends, school and sports. Since they could walk, I played and coached hockey and baseball with our teenage kids, with just about every spare minute spent playing catch, tennis, golf, basketball, Ping-Pong, pool, and on our backyard rink, hockey and curling. The kids always look forward to a slim but certain victory against Dad, a.k.a. “The Threat.”
Four of the five Barnowski brothers: Tim, Don, Doug & Mike
Sports create lasting memories, generate new friends, and teach important life Emily is goalie in a home hockey game; lessons. That becomes ingrained, and ALS Don and son Nick join friends on the ice. can never take that away. And though ALS is taking my physical strength, my faith and spirit intensifies; rather than our family becoming weaker, our core values are actually strengthening. Being as proactive as possible, we are taking advantage of the chance to participate in clinical trials, and are looking forward to beginning the NP-001 phase 2 trial in October.
ALS is a terrible disease, yet its spawns so many positives. Most humbling to us – by far – is how this has touched the generosity of others. A wise person once said, "when darkness falls during your journey, that's when the stars come out." We are inspired to give back by supporting efforts to help families living with ALS and to find a cure. Last year, in addition to money raised by family and friends at the Virtual Walk to Defeat ALS, our son's Howell High School "Play for the Cure" tennis tournament raised $3,100 for the ALSA Michigan chapter. Our goal for this year's tournament, on September 24, is $10,000. So take a look at the event's Detroit walk page, and help us to spread the word about this tournament that gives 125 student athletes, from eight schools, an opportunity to directly participate in raising awareness and funding for a good cause. Some day soon, each of those kids can be proud that they played a part in curing ALS.
L. to R.: Mark Oglesby, Howell High varsity tennis coach; Maggie Barnowski; Joe Kulwicki, ALSA Michigan; Nick and Don Barnowski; former ALSA-Michigan executive director Stacey Orsted; and Don’s brother Mike, at 2010 “Play for the Cure.”
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First, Annette Farmer noticed she was tripping occasionally in 2009, as she worked her shift as a nurse in Florida. A few months later, her foot wouldn’t raise as she jogged along the beach. As a nurse, she could rationalize the muscle weakness to a herniated disc years before. Then her voice started to change, too. Her patients picked up on that first. “As I was working with patients, I could see, at first they thought I had an accent,” Annette said. “Then I could tell it was getting a little bit more difficult to form words. I decided, even as a nurse, something must be going on.” Her family doctor urged her to see a neurologist. Early tests indicated ALS, but her friends urged her to go to Mayo Clinic, for a second opinion… “Sure enough, it was confirmed,” she said. She and her exhusband, a physician, have two adult children, and have just welcomed their first grandson. He’s not the only excitement. On Sept. 4, 2010, just weeks after Mayo confirmed her diagnosis, Annette married anesthesiologist Richard Hackim. They celebrated their first anniversary at their Cascade Township home last week.
Both dog lovers, Annette and
Richard and Annette love their Richard dote on their pups, Jake time on the family farm in Ohio. The couple met in a Florida hospital operating room — she was and Chloe. The feeling is mutual. the nurse, he was the new anesthesiologist.
“It was love at first sight,” Annette said, smiling broadly. Two years ago, the new couple invested in an Ohio farm, and spend nearly every weekend at the 300-acre farm, a four-hour drive from Grand Rapids. In the meantime, Annette has struggled to cope with her growing infirmities. The former ballerina and snow skier now wears an ankle brace and leans on a cane — for any distance, she resorts to a wheelchair. “I’m working to be independent, as far as eating and dressing. I can still drive.” But the biggest blow is in her speech. “The hardest thing has been not being able to communicate. I’ve had lots of friends and enjoy talking to people. It seems to be the most frustrating,” Annette said. And after a year and a half of living in France, she appreciated red wine… until now. “I can’t even take a sip without it really affecting my speech,” she said. At 52, she struggled to adjust to the vagaries of the disease. “For the longest time — I think it was partly denial — I wouldn’t read any of the literature,” Annette said. “Every time I did, I would break down.” Gradually, though, her mind and attitude made an adjustment. She’s entered a Phase 3 clinical trial, and praises the care she has received at the ALS Clinic in Grand Rapids.
Nothing beats Mackinac Island!!
“Now, it’s just strange. All of a sudden, my mind has changed,” she said. “I’m learning to go out there and try to help other people.” Talking with friends is her biggest hurdle, Annette said.
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rmer s go to fo e h is w t s s and be ted! Our thank tacey Ors S r o t c e ir d executive makes me a little bit more flexible,” she said. Her career includes her current role with CBS Radio, and previous stints with Walt Disney and as director of marketing for Quicken Loans, a major supporter of ALSA Michigan.
Susan Woolner
Susan Woolner, ALSA Michigan’s new executive director, had her first brush with ALS in 1992, when her father-in-law was diagnosed with the terminal illness.
“The board has a really strong commitment toward patient education and patient care,” Susan said. “Whatever PALS need.”
“By the time it was diagnosed, it was fairly quick-moving,” Susan said. He passed away two years later, in 1994.
“It can increase donations from various groups, including younger people, under 25, who interface with technology in a different way and want to donate in a different way.”
Now 48, she’s taken the helm of the state organization whose aim is to eradicate ALS. No small task.
One of her passions is using technology to raise money for the cause.
She expects to split her work week between the Troy office Mon-Wed, and the Grand Rapids office on Thurs.Fri. Paula Morning, Morning our new development director based out of ALSA Michigan’s Troy office, is a U of M grad and joined our organization in early June. She previously worked with the state’s Colitis Association and the Washtenaw County Republican Party.
Susan took the temporary role of executive director this summer, to cover while executive director Stacey Orsted took a personal leave. Stacey had just lost her father to Alzheimers, the latest challenge her family has faced. By summer’s end, Stacey decided to step away from the post. At that point, Susan felt ready to step in. She had served on the ALSA Michigan board for the past year. Based in Canton, Susan started her career in marketing and media. Most recently, she’s been developing smart-phone applications with her own small business. She and her husband have three sons. The eldest is a puppeteer in California, and has created clips for the Muppet movie. Her 16-year-old twin sons are sophomores in high school, and have been the motivation for her biggest non-profit cause — autism. Both boys are autistic, and Susan has worked tirelessly to advocate for her sons, and other families touched by autism. Since she was in college, Susan has worked with cuttingedge technology, first to handle data in natural resources projects. She is a grad of U. of Illinois-Champaign Urbana. “It’s the exhilaration of discovering, of solving problems. It
Paula Morning
“My mission at ALSA is to develop strong and lasting relationships with donors, the community and the corporate leadership of Michigan to assist in the building of a stronger Michigan Chapter. The goal is better care and resources for all patient and families touched by ALS,” she said. Vicki Bouwkamp, Bouwkamp a newlywed and grad of Aquinas College, is our new development manager for Michigan’s West side, based out of the Grand Rapids office. She previously was student outreach coordinator for new nurses at Aquinas. That role gave her a good foundation in development, and a wealth of contacts in the Grand Rapids area.
Vicki Bouwkamp
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CORE VALUES AT ALSA MICHIGAN People with ALS and their families come first in everything we do. Scientific credibility and innovation are the hallmarks of our research program. Commitment to excellence and professionalism are key tenets at all levels of our organization. What better accessory than fuzzy dice? ALSA drove home its message at August's Metro Cruise in Grand Rapids
We are one team with one vision and one mission working together.
Here’s your chance to wear your cause. Check out this link to order red wristbands and help win the battle (price averages $2 per); $22 for 10; $206 for 100!
Collaboration and partnership within our organization and with others who share our vision and values are key to sustained success in the fight against ALS.
Financial strength enables us to accomplish our goals.
Integrity, honesty and ethical behavior guide all our endeavors. Mutual respect is the cornerstone for all our working relationships. Diversity of ideas, cultures, ethnicities and backgrounds strengthen our efforts.
A new payroll deduction program for charity could help ALSA expand research as it broadens its care services and public policy efforts. The federation, Community Health Charities , joins health organizations under the new program. Check it out, and make your paychecks start serving our shared goal — beating ALS in our lifetimes.
ALSA-Michigan kicked off its season of Walks to Defeat ALS at an August 25 gathering at the Grand Rapids Art Van Furniture store. Our thanks go to keynote speaker and chef Rod Pierce, with Calvary Church. He owned Hot Tamale in East Grand Rapids and was diagnosed with ALS in March. His wife, Dr. Eileen Pierce, is with the City of Grand Rapids, Water Department.
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No matter how you phrase it, the findings are at the peak of a flood of hopeful research developments this summer. So, what’s this “ubiquilin 2”?
Stunning news made headlines around the world in August, when Northwestern University researchers said they had uncovered a single gene and related protein wrecking havoc in both familial and sporadic victims of ALS.
In essence, the problem amounts to poor housekeeping at the molecular level. When the protein ubiquilin 2 works properly, it helps to sweep out misfolded and damaged proteins, enabling nerves to stay healthy. But when UBQ2 fails to take the protein debris to the recycling center, havoc ensues. Kelly Johnstone, a writer with the Motor Neuron Disease Association, described the process as a defunct recycling system:
“Imagine a world where all recycling collectors are on strike. Every Wednesday at 7am you place a box of recycling on your driveway ready to be collected, but it’s still there in the “NU Team: Cause of ALS Found” — Chicago evening. The next week you put out more Tribune recycling, and that isn’t collected either. After “New Gene Finding Suggests Recycling is Key weeks of putting your recycling outside, you to All Forms of MND” — Motor Neuron Disease notice that the pile is mounting and still isn’t Association. being collected. This doesn’t bother you too much as you can still step over it, albeit in a “New Research Points to Common Cause of slightly ALS” — ALSA.ORG slower And the actual headline for the NU study, as manner. printed in the well-respected journal Nature, on A few August 21, 2011? months “Mutations in UBQLN2 cause dominant X- pass and you can linked juvenile and adult-onset ALS and no longer ALS/dementia.” get out of your driveway as it’s covered by recycling. Now you can’t get rid of your rubbish, you can’t get to work and you can’t even leave “Possible Culprit is Found for Lou Gehrig’s Disease” — New York Times
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waste-proteins, without the genetic defects found in the families haunted by ALS. If you’re a knitter, think of a tangle of yarn where smoothly knit stockinette stitches should be. Or for fishermen, imagine the knotty twist of line that bedevils a reel. Such tangles in the spinal cord are chief suspects in the nerve atrophy troubling people with ALS. your house all because of the pile up of recycling. The same thing is happening to everybody all over your town. This is what happens in MND. “This build up of recycling causes pandemonium in cells, as vital movement of nutrients around the cell cannot easily pass to where they need to get to, causing an additional burden to the motor neurons. Eventually, the motor neurons start to degenerate because of this.”
Similar ubiquilin 2 protein tangles were found in the brains of those who suffered from both ALS and dementia, or FTLD — frontal temporal lobar dementia. So, the good news is knowing the identity of a primary suspect, whose fingerprints were found in ALS patients whether of the sporadic , familial, or dementia variety. Such a trail could lead researchers to more viable drug therapies and (dare we say) eventually a cure?
Not all types of ALS have the same culprit, the NU researchers said. Mutations in the UBQLN2 gene were found in some of the relatively rare familial ALS cases, but another surprise came after autopsies of sporadic ALS victims. They, too, had the tell-tale twisted cluster of UBQ2
The team of 23 researchers was led by Prof. Teepu Siddique at Northwestern University Feinberg School of Medicine in Chicago. Their report in Nature describes how problems with the “rubbish recycling” system in motor neurons appear to be integral to the degeneration seen in MND.
Another gene, Sigma R1, tied to young victims of familial ALS
New correlation links long ataxin 2 genes and proteins to higher risk of ALS
Researchers in Riyadh, Saudi Arabia, report that a mutation in the sigma R1 gene is the culprit behind some cases of young people who have familial ALS. The sigma R1 gene carries the genetic instructions for a protein that is known to have properties that protect the motor neurons, or nerves. Average age of onset of ALS in the U.S. and Europe is 56 to 63 years, while a relatively rare form of juvenile ALS can start in child, teens, or early 20s. About the new findings: The Saudi research team performed genetic testing on four out of six people with juvenile-onset ALS, all members of an extended family. All four young people shared a single mutation in the sigma R1 gene on chromosome 9.
In a 2010 study, scientists working in the United States and Germany found that small polyglutamine ("polyQ") expansions of DNA in the ataxin 2 gene significantly increase the risk for developing ALS. The number of glutamine molecules in normal ataxin 2 protein typically is 22 or 23. Expanded sections consisting of more than 34 glutamines in this protein are associated with a neurological disease called spinocerebellar ataxia 2 (SCA2). But mid-range expansion, in the 24– to 34-glutamine range, is the sore spot for ALS risk. Three new studies link long ataxin 2 to ALS.
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Connecting the Dots…………………………………….
Stem Trials Advance BrainStorm Cell Therapeutics
The Israel-based stem cell trial is expanding to two U.S. sites: Massachusetts General Hospital in Boston and the University of Massachusetts Medical School in Worcester, Mass. Now in Phase 1 and 2, the trial uses BrainStorm’s NurOwn stem cell technology, Neurologist Merit Cudkowicz will lead the Massachusetts General Hospital team, while Prof. Robert Brown will head the team at the University of Massachusetts Medical School.
NEURALSTEM
Using techniques developed at the University of Michigan, Maryland-based Neuralstem is seeking FDA permission to inject fetal nerve stem cells into the spinal cord-neck region of three ALS patients.
The new trials require FDA approval, but in February, NurOwn gained orphan drug status, providing incentives to develop drugs for rare diseases. To date, BrainStorm has tested 12 people with advanced ALS, and 12 in the early stages.
The first dozen study participants have all received neural stem cells in their lower spine.
NurOwn uses mesenchymal stem cells, taken from the bone marrow of the trial participant.
The Phase 1 study must prove the safety of the procedure, and does not focus on its efficacy. Drs. Eva Feldman and Jonathan Glass are the neurologists overseeing the trial, conducted at Emory University, Atlanta. The stem cells were derived from a single donor fetus and cultured to become neural cells before being injected. Stem cells injected into the neck region will target breathing and swallowing functions that trouble ALS patients. Participants’ functions are tracked as part of the trial. Eligibility criteria can be found at the Emory ALS Center trial website.
Early trial participants received injections into their arms; later participants will get them by lower back puncture, into the cerebrospinal fluid. To find out more about the ongoing trial in Israel, view Autologous Cultured Mesenchymal Bone Marrow Stromal Cells Secreting Neurotrophic Factors (MSC-NTF) in ALS Patients at clinicaltrials.gov.
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Who Ya Gonna Call?
Curious about ALS clinical trials? A new central info service started last month, so those eager to learn about trials can reach an expert. Phone (877) 458-0631 9 a.m. to 5 p.m. Monday through Friday (Eastern); e-mail alstrials@partners.org. Rather check a web site? Click http:// www.alsa.org/news/archive/als-trial-expertavailable.html to read the full announcement.
NP001 Trial in Phase 2 Neuraltus Pharmaceuticals is nearly threefourths of the way to enrolling 105 ALS patients for its Phase 2 trial at 15 sites across the U.S. The closest to Michigan residents is at the Cleveland Clinic in Ohio. Participants receive injections with either a low dose or high dose of NP001, or a placebo. Results are not expected until mid-to-late 2012. The ALS Association encourages all eligible participants to enroll in this trial. For study details, visit http:// clinicaltrials.gov/ NP001 is a small molecule regulator of macrophage activity. NP001 is designed to restore the normal functioning of macrophages within the central nervous system. In addition to ALS, NP001 is being developed for the treatment of Parkinson's disease, Alzheimer's disease and Multiple Sclerosis.
Lighting up the role of MCT-1 in neuron degeneration Youngjin Lee of Johns Hopkins University has won a $180,000 grant from MDA to study a Youngjin Lee, Â transporter protein called MCT1. His theory? Glial cells may not provide enough energy components called substrates.
Dexpramipexole in Phase 3 BY THE END OF THIS MONTH, the Phase 3 clinical trial of Dexpramipexole (structure shown above) should be fully enrolled. Biogen Idec and Knopp Biosciences said in late August they were no longer seeking participants. The Grand Rapids ALS Clinic is the only Michigan site for the trial. Earlier trial phases showed good promise, slowing progression and restoring strength for some ALS clinical trial participants. The drug is taken orally.
Update on Ceftriaxone At 2 p.m. Monday, Sept. 26, an Internet conference hour is set for an update on the Ceftriaxone study. Clinical trial participants, caregivers, and others involved in the Ceftriaxone study are welcome to join the conversation. It will include Dr. Merit Cudkowicz, principal investigator for the study, and Dr. Jeremy Shefner. Webinar Instructions: 1. To register visit: https:// www1.gotomeeting.com/register/151881345 2. Register a day or two ahead, if possible. Dialin info will be e-mailed to you. Rather than enter both first and last name, please use either first initial and last name, or the reverse, for the sake of privacy.
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Can familial ALS troublemakers work together to keep neurons strong?
Cogane, a drug in clinical trial for Parkinson’s Disease, has been granted orphan drug status for potential treatment of ALS. The drug is in a Phase 2 trial for Parkinson’s, but is in pre-trial status for ALS. The orphan drug status granted News arrived this month that two recently by the FDA gives drug companies incentives to discovered ALS familial genes, FUS/TLS and TDP develop cures for diseases affecting fewer than -43, can actually work together to help keep 200,000 people in the U.S. motor neurons healthy. “The drug is in very early stages of development The study by Columbia University Medical for ALS; however, important information will be Center showed the genes work in tandem to gleaned from the Parkinson’s trial, which could support the long-term survival of motor expedite a clinical trial for ALS if the current neurons. Results were published in the Sept. 1 study testing the compound in the ALS mouse edition of the Journal of Clinical Investigation. model shows some promise,” said ALSA chief scientist Lucie Bruijn. “Any therapy based on this discovery is probably Curious to know more? a long way off. Nonetheless, it’s an important step toward piecing together the various factors For more information, visit http:// www.businessweekly.co.uk/biomedtech-/12443 that contribute to ALS,” says the study’s lead -fda-backs-new-treatment-for-motor-neuroneauthor Brian McCabe. disease to read an article in Business Weekly “The two genes make proteins with similar form and function, which suggested to us that they Blocking AMPA could work together, and that disruptions of either gene would affect neuronal survival,” Dr. receptors that cause toxicity and motor McCabe said. The other view? That gene abnormalities create a toxic combination to neuron death motor neurons, regardless of their normal role. A three-year, $294,000 grant has Researchers used the venerable fruit fly to sort out the good from the bad theories. The hope? That a strong normal gene could help out a weaker, mutant one, and keep the motor neuron stable. Is mitochondrial dysfunction a cause or consequence of motor neuron degeneration? Studying the inherited form of ALS, SOD1, researcher Don Cleveland has won a three-year, $430,000 MDA grant at the Ludwig Institute for Don Cleveland Cancer Research in La Jolla, Calif. He is studying mice to figure out the impact of mitochondria, and its role in the ALS disease process.
Vasanthi Jayaraman gone to associate professor Vasanthi Jayaraman, at the University of Texas Health Science Center in Houston, for her study of a glutamate receptor known as a calcium-permeable AMPA receptor.
AMPA receptors cause toxicity and may be a major trigger for selective motor neuron death and loss of muscle control in ALS. Better neuron health and survival time has already been proven in ALS mice, but they have yet to be proven out at the clinic. Jayaraman plans to develop AMPA receptor antagonists that prevent activation of the calciumpermeable AMPA receptors, without causing harmful side effects or affecting the function of other subtypes of glutamate receptors.
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For the first time, human astrocytes show they’re toxic to motor neurons. Although researchers have already tested the toxicity of astrocytes (the connection between neurons and other tissue), all of the tests have been with ALS mice. Now, scientists have launched the first study using astrocytes from people who died from either sporadic or familial ALS. Lots of attention centers on SOD1, the first familial genetic glitch identified in the early 1990s.Researchers were able to create a new model of the disease, and confirmed that the astrocytes become toxic to motor neuron cells. They showed the cells secreted toxic factors that caused the motor neurons to degenerate. So, when they lowered the levels of SOD1 in the human astrocytes, both the familial and sporadic forms had lower toxic impact on their motor neurons. That’s good news, because the results impacted both types of ALS. “"The mouse models capture a type of familial ALS that accounts for only 2 percent of all cases. The field has begged for new disease models that can provide a clear window into sporadic ALS," said senior author Brian Kaspar. “Studies are currently underway in mouse models of ALS to determine whether astrocyte replacement would be a feasible therapeutic approach,” said ALSA Chief Scientist Lucie Bruijn.
“Reporter” mice give real-time inside look at ALS disease Jasna Kriz, associate professor at Laval University, Quebec City, Canada, has won a three-year $445,086 MDA grant to generate ALS imaging reporter mice. Noninvasive, three-dimensional images reveal early neuronal stress in live bioluminescent “reporter” mice. Signals imaged from these animals can be used as biomarkers to isolate distinct elements of the ALS disease process, Kriz said, including indications representative of presymptomatic stages of the disease.
Previously, Kriz and colleagues developed mouse models to visualize ALS-related events such as neuroinflammation and neuronal damage in the brains and spinal cords of living mice. They even were able to detect distinct and disease-specific signals linked to presymptomatic stages of the disease. Now Kriz plans to use the previously generated models to create the ALS imaging reporter mice, which will enable scientists to visualize, in live mice, different elements of ALS.
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Wrapping up Research Notes: For sorting out the good and the bad among CD4+T cells in immune-system dysfunction: $180,000 over three years to Junping Xin, Loyola University, Chicago; Astrocytes in the central nervous system, magnified 100 times in photo. Neurons appear round and red. The astrocytes (motor neuron support cells) are yellow and activated, signifying an inflammatory response in the nervous system. Following nervecell injury, moderate inflammation is helpful for motor neuron regeneration, but uncontrolled inflammation may result in the death of nerve cell body instead.
Deciphering signals between cells that provoke disruption of mitochondrial function
Above: (A) Normal muscle fibers; (B,C) Muscle fibers with perturbed mitochondria. Dashes indicate muscle diameter, and asterisks illustrate the space between individual muscle fibers. Normal muscle fibers adjoin each other and are longer in diameter than muscle fibers with perturbed mitochondria. In contrast, muscle fibers with disruption in mitochondrial function separate from each other and appear either split (B) or angulated (C). Edward Owusu-Ansah, Harvard Medical School, won $180,000 over three years from MDA for his research in mitochondria’s molecular mechanisms.
Testing a possible ALS combination drug therapy in mice One process suspected to be heavily involved in ALS disease progression is inflammation, which can create a toxic environment and kill motor neurons. Using the SOD1 ALS research mouse model, Raymond Grill’s research team will test the hypothesis that a drug called Licofelone will enhance the ability of riluzole (Rilutek) to better penetrate the nervous system. He won $202,500 in a three-year grant from MDA, for his work at the University of Texas. The photo shows a microscope scan of microglial cells that support nerve cells, taken from the lower spinal cord of a mouse.
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Medic-Alert bracelet costs about $40/year
New Medic Alert bracelet, flash drive help ALS PALS, CALS When an emergency strikes, first responders can have trouble quickly assessing the situation, or what’s happening to you. For people with ALS, key information is particularly critical because signs and symptoms, such as movement or communication limitations, could easily be misconstrued and lead to a misdiagnosis. While you want Medic Alert bracelets and information systems to be easily noticed, they no longer have to look clunky or unattractive. And if they’re linked to a medical-information service or include a wellmarked USB flash drive, they can include a wide A MedInfoChip is $50/year range of information, including all diseases or conditions, blood type, allergies, disabilities and family and friends to contact. American Medical ID sells a flash drive that looks like a dog-tag necklace, updated by computer, for about $10 a year. Links: Medic-Alert: www.medicalert.org MedInfoChip: www.medinfochip.com American Medical ID: www.americanmedical-id.com American Ambulance Association: www.invisiblebracelet.org
http://wwwn.cdc.gov/als/
Remember to register! If you have ALS, especially if you’ve been recently diagnosed, don’t lose a minute before signing onto the National ALS Registry. Started just a year ago, the site is already expanding its scope in hopes of breaking ALS in our lifetime. Beyond logging on, people with ALS will also be able to register biological samples (yep, another blood draw). The registry also plans to seek surveys to answer questions about risk factors, and implement a system to notify PALS of clinical trials as they open up.
We Remember Louis Kolenbrander James Sampsel Lynn Cooper Lucille Turchetta Margaret Brechting Thomas Wiegand Nancy Gardner Patrice Steinhour Bob Barker Jeannine Barrera Sandra Simon Claire Wangler Nicholas Ritter Shirley Holzimmer Jean Baker Sandra Bayes James Cole William Avery Jackie Music Pitsch Colleen Cottrell Thomas Tepper
Frank Ruth Sandra Robert
Parker Morgan Adams Goick
We send heartfelt condolences to all the families and friends of those who’ve passed away in recent months. If we’ve overlooked anyone lost since June 1, 2011, please let us know at fightals@alsamichigan.
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EVENTS SEPTEMBER: Walks to Defeat ALS (details, P. 4) Sept. 11: Jackson Sept. 17: Detroit Sept. 18: Grand Rapids Sept. 24: Portage Oct. 1: Traverse City Anytime: Virtual Walk to Defeat ALS
Finding support: Here’s a line-up of support groups this fall. Watch for new groups in Traverse City and St. Joe/Benton Harbor! Check for updates at www.alsamichigan.org or phone 616-459-1900 (West Michigan) or 248-680-6540 (Eastern Michigan) Second Sundays: Grand Rapids: 2-4 p.m., First Evangelical Covenant Church, 1933 Tremont Blvd. NW; 616-453-6346 Second Wednesdays: Kalamazoo: 7-8:30 p.m., First United Methodist Church, 212 S. Park St.; 269-381-6340 Second Thursdays: Troy: 6:30-8 p.m., Big Beaver United Methodist Church, 3753 John R Rd. Statewide call-in: 2-3 p.m., Dial toll-free 877-643-6951; passcode 31723015# to join the group Third Mondays: Bay City: Zion Evangelical Lutheran Church, 545 7th St., Freeland Third Tuesdays: Statewide call-in: 6:30-7:30 p.m., Dial toll-free 877-643-6951; passcode 31723015# to join the group Fourth Mondays: Ann Arbor: 6:30-8 p.m., University of Michigan Medical Center, Dept. of Neurology, First Floor Taubman Center Flint: 6:30-8 p.m., Fenton United Methodist Church, 119 S. Leroy St., Fenton (New Family Center Building, East Silver Lake Rd/Main St. and S. Leroy) Fourth Wednesdays: Grand Rapids: 6-7:30 p.m., Mary Free Bed Rehabilitation Hospital Fourth Thursdays: Gaylord: 2:30-4:30 p.m., University Center, 80 Livingston Blvd. Wyoming: 11:30-1 p.m., Survivors Lunch, Arnie’s Restaurant, 77 54th St.; 616-532-5662