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Our missionOur mission: Leading the fight to treat and cure ALS

through global research and nationwide advocacy

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: Our Vision: Create a world without ALS.

Staff

Susan Woolner Executive Director ed@alsa-michigan.org Kristen Munyan, RN, MSN-Ed Care Services Manager, East kristen@alsa-michigan.org 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@alsa-michigan.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 com-pelling story? E-mail your ideas to news@alsa-michigan.org

Board of Directors John Crosby President Matt Wey* Past President, and voting member, National ALSA Board of Representatives 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

In My View: By Susan Woolner, Executive Director

Michigan ALS CentersMichigan 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-in-law 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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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

Inside Information

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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: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-and-dale 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 after-party!

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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. 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.”

Sports create lasting memories, generate new friends, and teach important life lessons. That becomes ingrained, and ALS 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.”

Emily is goalie in a home hockey game; Don and son Nick join friends on the ice.

The Barnowski Family: Don, kids Emily and Nick, and wife Maggie

Four of the five Barnowski brothers: Tim, Don, Doug & Mike

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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 ex-husband, 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.

The couple met in a Florida hospital operating room — she was 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.

“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. 

Richard and Annette love their 

time on the family farm in Ohio. 

Both dog lovers, Annette and 

Richard dote on their pups, Jake 

and Chloe. The feeling is mutual. 

Nothing beats Mackinac Island!! 

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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.

“By the time it was diagnosed, it was fairly quick-moving,” Susan said. He passed away two years later, in 1994.

Now 48, she’s taken the helm of the state organization whose aim is to eradicate ALS. No small task.

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 cutting-edge 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

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.

“The board has a really strong commitment toward patient education and patient care,” Susan said. “Whatever PALS need.”

One of her passions is using technology to raise money for the cause.

“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.”

She expects to split her work week between the Troy office Mon-Wed, and the Grand Rapids office on Thurs.-Fri.

Vicki BouwkampVicki 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.

Paula MorningPaula 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 previ-ously worked with the state’s Colitis As-sociation and the Washtenaw County Re-publican Party.

“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 bet-ter care and resources for all patient and families touched by ALS,” she said.

Paula Morning 

Vicki Bouwkamp 

Susan Woolner 

Our thanks and best wishes go to former

executive director Stacey Orsted!

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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!

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.

 

 

 

 

 

 

So here is

What better accessory than fuzzy dice? ALSA drove home 

its message at August's  Metro Cruise in Grand Rapids 

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.

We are one team with one vision and one mission working together.

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.

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.

Financial strength enables us to accomplish our goals.

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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.

“Possible Culprit is Found for Lou Gehrig’s Disease” — New York Times

“NU Team: Cause of ALS Found” — Chicago Tribune

“New Gene Finding Suggests Recycling is Key to All Forms of MND” — Motor Neuron Disease Association.

“New Research Points to Common Cause of ALS” — ALSA.ORG

And the actual headline for the NU study, as printed in the well-respected journal Nature, on August 21, 2011?

“Mutations in UBQLN2 cause dominant X-linked juvenile and adult-onset ALS and ALS/dementia.”

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”?

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 evening. The next week you put out more recycling, and that isn’t collected either. After weeks of putting your recycling outside, you notice that the pile is mounting and still isn’t being collected. This doesn’t bother you too much as you can still step over it, albeit in a slightly slower manner. A few months pass and you can no longer 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

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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.”

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

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.

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?

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 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.

New correlation links long ataxin 2 genes and proteins to higher risk of ALS In a 2010 study, scientists working

in the United States and Germany found that small poly-glutamine ("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 consist-ing of more than 34 glutamines in this protein are asso-ciated 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

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 first dozen study participants have all received neural stem cells in their lower spine. 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.

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. 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. NurOwn uses mesenchymal stem cells, taken from the bone marrow of the trial participant. 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-expert-available.html to read the full announcement.

NP001 Trial in Phase 2

Neuraltus Pharmaceuticals is nearly three-fourths 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 transporter protein called MCT-1. 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. Dial-in 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.

Youngjin Lee,  

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Can familial ALS troublemakers work together to keep neurons strong? News arrived this month that two recently discovered ALS familial genes, FUS/TLS and TDP-43, can actually work together to help keep motor neurons healthy. The study by Columbia University Medical Center showed the genes work in tandem to support the long-term survival of motor neurons. Results were published in the Sept. 1 edition of the Journal of Clinical Investigation. “Any therapy based on this discovery is probably a long way off. Nonetheless, it’s an important step toward piecing together the various factors that contribute to ALS,” says the study’s lead author Brian McCabe. “The two genes make proteins with similar form and function, which suggested to us that they could work together, and that disruptions of either gene would affect neuronal survival,” Dr. McCabe said. The other view? That gene abnormalities create a toxic combination to motor neurons, regardless of their normal role. 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.

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 by the FDA gives drug companies incentives to develop cures for diseases affecting fewer than 200,000 people in the U.S. “The drug is in very early stages of development for ALS; however, important information will be gleaned from the Parkinson’s trial, which could expedite a clinical trial for ALS if the current study testing the compound in the ALS mouse model shows some promise,” said ALSA chief scientist Lucie Bruijn. Curious to know more? For more information, visit http://www.businessweekly.co.uk/biomedtech-/12443-fda-backs-new-treatment-for-motor-neurone-disease to read an article in Business Weekly

Is mitochondrial dysfunction a cause or consequence of motor neuron degeneration?

Studying the inherited form of ALS, SOD1, re-searcher Don Cleveland has won a three-year, $430,000 MDA grant at the Ludwig Institute for Cancer Research in La Jolla, Calif. He is study-ing mice to figure out the impact of mitochon-

dria, and its role in the ALS disease process.

Blocking AMPA receptors that cause toxicity and motor neuron death A three-year, $294,000 grant has 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 calcium-permeable AMPA receptors, without causing harmful side effects or affecting the function of other subtypes of glutamate receptors.

Vasanthi Jayaraman 

Don Cleveland 

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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 bio-luminescent “reporter” mice. Signals imaged from these animals can be used as biomarkers to isolate distinct elements of the ALS disease process, Kriz said, including indica-tions representative of presymptomatic stages of the disease.

Previously, Kriz and colleagues de-veloped mouse models to visualize ALS-related events such as neuroin-flammation and neuronal damage in the brains and spinal cords of living mice. They even were able to de-tect 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:Wrapping up Research Notes:

For sorting out the good and the bad among CD4+T cells in im-mune-system dysfunc-tion: $180,000 over three years to Junping Xin, Loyola Universi-ty, Chicago; Astrocytes in the

central nervous system, magnified 100 times in photo. Neurons appear round and red. The as-trocytes (motor neuron support cells) are yellow and activated, signifying an inflammatory re-sponse in the nervous system. Following nerve-cell injury, moderate inflammation is helpful for motor neuron regeneration, but uncon-trolled 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 in-dicate 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 per-turbed mitochondria. In contrast, muscle fibers with disruption in mitochondrial function sepa-rate 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 mo-lecular mechanisms.

Testing a possible ALS combination drug therapy in mice One process suspected to be heavily involved in ALS disease progres-sion is inflammation, which can create a toxic environment and kill motor neurons. Using the SOD1 ALS research mouse model, Ray-mond Grill’s research team will test the hypoth-esis that a drug called Licofelone will enhance the ability of riluzole (Rilutek) to better pene-trate 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 microgli-al cells that support nerve cells, taken from the lower spinal cord of a mouse.

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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.

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 well-marked USB flash drive, they can include a wide 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

Medic-Alert bracelet costs about $40/year

A MedInfoChip is $50/year 

http://wwwn.cdc.gov/als/

We We We RememberRememberRemember

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 Parker Ruth Morgan Sandra Adams Robert Goick

We send heartfelt condo-lences to all the families and friends of those who’ve passed away in recent months. If we’ve over-looked anyone lost since June 1, 2011, please let us know at fightals@alsa-michigan.

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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.alsa-michigan.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