Sept 2011News Magazine

8/3/2019 Sept 2011News Magazine

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Our missionOur mission: Leading t he fi ght t o t reat and cure ALS

through global research and nationwide advocacy

while also empowering people

with Lou Gehrigs Disease and their families

t o live fuller l ives by providing t hem wit h

compassionate care and support .

Our Vision:Our Vision: Create a world without ALS.

Staff

Susan Woolner

Executive Director

[email protected]

Kristen Munyan, RN, MSN-Ed

Care Services Manager, East

[email protected]

Robbie Banfill

Care Services Coordinator, West

[email protected]

Paula Morning

Development Director, [email protected]

Vicki Bouwkamp

Development Manager, West

[email protected]

Cathryn Rybicki

Accountant

[email protected]

Julia Bauer

Michigan Matters editor

[email protected]

Website: www.alsa-michigan.org

Email: [email protected]

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@al sa-

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 CentersHarry 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 asthe new Executive Director of The ALS AssociationMichigan Chapter. Ive been involved with the ALScommunity in Michigan for more than 17 years and haveserved the Chapter as a board member for better than ayear.

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 shewould not be returning to the role, I accepted theexecutive 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 ALSClinics, our leadership role in patient care, advocacy andground breaking research.

Im grateful for the opportunity to serve the extended ALSCommunity in Michigan and look forward to meeting manyof you face-to-face at one of our five upcoming Fall Walksto 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 fightagainst Lou Gehrig's Disease to the next level, andfortunately, there is an easy way to get there by givingthrough payroll deductions.

You can contribute to The ALS Association throughCommunity Health Charities (CHC), a federation ofpremier health organizations that have joined together toraise charitable contributions in the workplace. Your gift

will not only fund The Association's global researchendeavors, but also our Chapter's care services programsand 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


3/20

Walkers st omp on ALS ... . .. .. .. . .. .. .. . .. .. 4

Iron Horse, Gears & Beers .. . . . . . . . . . . . . . . . 6

Meet Don and Annet t e .. .. .. .. .. .. .. .. .. .. .. 8

Hel lo, Susan Woolner .. . .. .. .. . .. .. .. . .. .. . 10

ALSA-Michigan news .. .. . . . . . . . . . . . . . . . . . . . . 11

Breakt hrough: cause of ALS found?. . . . . 12

Connect ing t he dot s: ALS research ... . . . 14

Didj a Know? Br ief s of int erest . . .. . .. . .. . 19

We remember.................................19

Event s, Suppor t Groups abound ... . . . . . . 20

Inside Informat ion


4/20

Walkers, rol lers prepare t o

launch all across Michigan.

The aim? St omp on ALS!Half of l i f e is showing up! Heres where:Half of l i f e is showing up! Heres where::

Sept . 11 Jackson

Check-in 9 a.m., Walk 10 a.m.

Ell a Sharp Park, 2800 4th St ., Walk 1 mil e

Sept . 17 Det roit

Check-in 9 a.m., Walk 10 a.m.

Detr oit RiverWalk,

on the Riverf ront ,

1340 At water St .; Walk 1.4 mil es

Sept . 18Grand Rapids

Check-in 1 p.m., Walk 2 p.m.;

Ah-Nab-Awen Park

303 Pearl St . NW

Walk 2 mil es

Sept . 24 Port ageCheck-in 1 p.m., Walk 2 p.m. Celery Flats, 7335 Garden Ln.; Walk 3 miles

Oct . 1 Traverse Ci t y ,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


5/20

Top 10 reasons to hit the pavement for Michigans Walk to Defeat ALS

this month: (apologies to David Letterman)

10. Its the last month you wont have to wear snow boots outdoors.

9. Its the biggest single fundraiser for the ENTIRE year for ALSA Michigan!

8. Youll get out of your rut and onto the sidewalk, with a bunch of friendly people.

7. Your teams 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 Michigans ALS centers in Detroit and

Ann Arbor, and its ALS clinic in Grand Rapids.

4. Your co-workers who dont 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, youll find great munchies (thanks,

sponsors!) to reward your effort.

2. Walking is good for you, and on this day, its 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!! !!


6/20

Iron Horse Riders logged more than

7,100 mi les in t wo days of hi l l -and-

dale courses in June, and t eamwork

made all t he di ff erence.

By t he t ime t he last t eam pedaled in, t he dual eff ort

marked plent y of f i rst s:

First t ime event expanded to t wo locat ions: or iginal has

been in Ann Arbor since 2007; Lowell was new t his year.

First t ime PALS have biked in t he event (t wo made hist ory)

Best fund-raising in hist ory of t he

event: nearly

$45,000 t o

date


7/20

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, andcontributed to our Iron Horse Ride, andthe refreshing Gears and Beers after-party!


8/20

By Don Barnowski, Howell

The neurologist paused. " or it could be something called ALS. Haveyou heard of that? Yeah, it only seems to hit the good guys...". That'swhen the reality of what we were dealing with really hit.

Confirmation four weeks later actually brought a strange kind of reliefsince months of uncertainty was now over. That numbing day in July2010 changed our family forever. But at least we knew what we werefighting.

And fight we do, using all the arrows inthe 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 minutespent playing catch, tennis, golf, basketball, Ping-Pong, pool, and on ourbackyard rink, hockey and curling. The kids always look forward to a slimbut certain victory against Dad, a.k.a. The Threat.

Sports create lasting memories, generatenew friends, and teach important lifelessons. That becomes ingrained, and ALScan never take that away. And though ALSis taking my physical strength, my faith and spirit intensifies; rather thanour 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-001phase 2 trial in October.

ALS is a terrible disease, yet its spawns so many positives. Most humbling tous by far is how this has touched the generosity of others. A wise person once said, "when darknessfalls during your journey, that's when the stars come out." We are inspired to give back by supportingefforts to help families living with ALS and to find a cure.

Last year, in addition to money raised byfamily and friends at the Virtual Walk to DefeatALS, our son's Howell High School "Play for theCure" tennis tournament raised $3,100 for the

ALSA Michigan chapter. Our goal for this year'stournament, on September 24, is $10,000.

So take a look at the event's Detroit walkpage, and help us to spread the word about thistournament that gives 125 student athletes,from eight schools, an opportunity to directlyparticipate in raising awareness and funding fora good cause.

Some day soon, each of those kids can beproud that they played a part in curing ALS.

L. to R.: Mark Oglesby, Howell High varsity tennis coach; MaggieBarnowski; Joe Kulwicki, ALSA Michigan; Nick and Don Barnowski;former ALSA-Michigan executive director Stacey Orsted; and Donsbrother 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


9/20

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 wouldnt 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. Hes 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. Im 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. Ive 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 cant even take a sip without it really affecting my speech,

she said. At 52, she struggled to adjust to the vagaries of thedisease.

For the longest time I think it was partly denial I wouldnt

read any of the literature, Annette said. Every time I did, I

would break down. Gradually, though, her mind and attitude

made an adjustment. Shes entered a Phase 3 clinical trial, and praises the care she has received at the

ALS Clinic in Grand Rapids.

Now, its just strange. All of a sudden, my mind has changed, she said. Im learning to go out

there and try to help other people.Talkingwithfriendsisherbiggesthurdle,Annettesaid.

RichardandAnnettelovetheirtimeonthefamilyfarminOhio.

Bothdoglovers,AnnetteandRicharddoteontheirpups,JakeandChloe.Thefeelingismutual.

NothingbeatsMackinacIsland!!


10/20

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Susan Woolner, ALSA Michigans

new executive director, had her

first brush with ALS in 1992, when

her father-in-law was diagnosedwith 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, shes 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 Orstedtook a personal leave. Stacey had just lost her father to

Alzheimers, the latest challenge her family has faced. By

summers 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, shes 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.

Its 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 towardpatient education and patient care, Susan said.Whatever PALS need.

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

It can increase donations from various groups, includingyounger people, under 25, who interface with technologyin a different way and want to donate in a different way.

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

Vicki BouwkampVicki Bouwkamp, a newlywed and gradof Aquinas College, is our newdevelopment manager for MichigansWest side, based out of the GrandRapids office.

She previously was student outreachcoordinator for new nurses at Aquinas.That role gave her a good foundation indevelopment, and a wealth of contactsin the Grand Rapids area.

Paula Morni ngPaula Morni ng, our new development

director based out of ALSA Michigans

Troy office, is a U of M grad and joined

our organization in early June. She previ-

ously worked with the states 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.

PaulaMorning

VickiBouwkamp

SusanWoolner

Ourthanksandbestwishesgot

oformer

executivedi rectorS

taceyOrsted!


11/20

1

Heres 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 atan 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

Whatbetteraccessorythanfuzzydice?ALSAdrovehomeitsmessageatAugust's MetroCruiseinGrandRapids

CORE VALUES AT ALSA MICHIGAN

People wit h ALS and t heir f amili es come fir st in everyt hing we

do.

Scienti f ic credibi li t y and innovat ion are the hallmarks of our

research program.

Commit ment t o excellence and professionali sm are key t enets

at all levels of our organizat ion.

We are one team with one vision and one mission working

together.

Collaborat ion and part nership wit hin our organizat ion and wit h

others who share our vision and values are key to sustained

success in the f ight against ALS.

Int egri t y, honest y and ethical behavior guide all our endeavors.

Mutual r espect is the cornerstone for all our working

relationships. Diversit y of ideas, cult ures, et hnicit ies and backgrounds

st rengt hen our effor t s.

Financial st rengt h enables us t o accompli sh our goals.


12/20

1

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 Culpr i t is Found for Lou Gehr igs

Disease New York Times

NU Team: Cause of ALS Found Chicago

Tribune

New Gene Finding Suggest s Recycl ing is Key

t o Al l Forms of MND Motor Neuron Disease

Association.

New Research Point s to Common Cause of

ALS ALSA.ORG

And the actual headline for the NU study, asprinted in the well-respected journal Nature, on

August 21, 2011?

Mutat ions in UBQLN2 cause dominant X-

l inked juveni le and adult -onset ALS and

ALS/dement ia.

No matter how you phrase it, the findings are at

the peak of a flood of hopeful research

developments this summer. So, whats 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 UBQ2fails 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 readyto be collected, but its still there in the

evening. The next week you put out more

recycling, and that isnt collected either. After

weeks of putting your recycling outside, you

notice that the pile is mounting and still isnt

being collected. This doesnt 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 its covered by

recycling. Now you cant get rid of your rubbish,

you cant get to work and you cant even leave


13/20

1

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 pandemoniumin 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 cameafter 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 youre 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 thespinal 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 foundin 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 neuronsappear to be integral to the degeneration seen

in MND.

Another gene, Sigma R1,tied to young victims of

familial ALSResearchers in Riyadh, SaudiArabia, report that a mutation in

the sigma R1 gene is the culprit behind some cases ofyoung people who have familial ALS.

The sigma R1 gene carries the genetic instructions for aprotein that is known to have properties that protectthe motor neurons, or nerves.

Average age of onset of ALS in the U.S. and Europe is 56to 63 years, while a relatively rare form of juvenile ALScan start in child, teens, or early 20s.About the new findings: The Saudi research teamperformed genetic testing on four out of six people withjuvenile-onset ALS, all members of an extended family.All four young people shared a single mutation in thesigma R1 gene on chromosome 9.

New corr elat ion li nks longat axi n 2 genes and prot einst o higher r isk of ALS

In a 2010 study, scientists workingin the United States and Germany found that small poly-glutamine ("polyQ") expansions of DNA in the ataxin 2gene significantly increase the risk for developing ALS.

The number of glutamine molecules in normal ataxin 2protein 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 spinocerebellarataxia 2 (SCA2).

But mid-range expansion, in the 24 to 34-glutaminerange, is the sore spot for ALS risk.

Three new studies link long ataxin 2 to ALS.


14/20

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Connecting the Dots.

Stem Trials Advance

NEURALSTEMUsing techniques developed at the Universityof Michigan, Maryland-based Neuralstem isseeking FDA permission to inject fetal nervestem cells into the spinal cord-neck region ofthree ALS patients.

The first dozen study participants have allreceived neural stem cells in their lowerspine.

The Phase 1 study must prove the safety ofthe procedure, and does not focus on itsefficacy. Drs. Eva Feldman and Jonathan Glassare the neurologists overseeing the trial,conducted at Emory University, Atlanta.

The stem cells were derived from a single

donor fetus and cultured to become neuralcells before being injected.

Stem cells injected into the neck region willtarget breathing and swallowing functions thattrouble ALS patients. Participants functionsare tracked as part of the trial. Eligibilitycriteria can be found at the Emory ALS Centertrial website.

BrainStorm Cell

TherapeuticsThe Israel-based stem cell trial is expanding totwo U.S. sites: Massachusetts General Hospitalin Boston and the University of MassachusettsMedical School in Worcester, Mass.

Now in Phase 1 and 2, the trial usesBrainStorms NurOwn stem cell technology,

Neurologist Merit Cudkowicz will lead theMassachusetts General Hospital team, whileProf. Robert Brown will head the team at theUniversity of Massachusetts Medical School.

The new trials require FDA approval, but inFebruary, NurOwn gained orphan drug status,providing incentives to develop drugs for rarediseases. To date, BrainStorm has tested 12people with advanced ALS, and 12 in the earlystages.

NurOwn uses mesenchymal stem cells, takenfrom the bone marrow of the trial participant.

Early trialparticipantsreceivedinjectionsinto theirarms; laterparticipantswill get

them by lower back puncture, into thecerebrospinal fluid.

To find out more about the ongoing trial inIsrael, view Autologous Cultured MesenchymalBone Marrow Stromal Cells SecretingNeurotrophic Factors (MSC-NTF) in ALSPatients at clinicaltrials.gov.


15/20

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Who Ya Gonna Call?Curious about ALS clinical trials? A new centralinfo service started last month, so those eagerto learn about trials can reach an expert. Phone(877) 458-0631 9 a.m. to 5 p.m. Monday throughFriday (Eastern); e-mail [email protected].

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 2Neuraltus Pharmaceuticals is nearly three-fourths of the way to enrolling 105 ALS patients

for its Phase 2 trial at 15sites across the U.S. Theclosest to Michiganresidents is at theCleveland Clinic in Ohio.Participants receiveinjections with either alow dose or high dose ofNP001, or a placebo.Results are not expecteduntil mid-to-late 2012. The ALS Association

encourages all eligible participants to enroll inthis trial. For study details, visit http://clinicaltrials.gov/NP001 is a small molecule regulator ofmacrophage activity. NP001 is designed torestore the normal functioning of macrophageswithin the central nervous system. In additionto ALS, NP001 is being developed for thetreatment of Parkinson's disease, Alzheimer'sdisease and Multiple Sclerosis.

Lighting up the role ofMCT-1 in neurondegenerationYoungjin Lee of Johns HopkinsUniversity has won a $180,000grant from MDA to study atransporter protein called MCT-1.His theory? Glial cells may not provide enough energycomponents called substrates.

Dexpramipexole in Phase 3

BY THE END OF THIS MONTH, the Phase 3clinical trial of Dexpramipexole (structureshown above) should be fully enrolled. BiogenIdec and Knopp Biosciences said in late Augustthey were no longer seeking participants.

The Grand Rapids ALS Clinic is the only Michigansite for the trial. Earlier trial phases showed

good promise, slowing progression and restoringstrength for some ALS clinical trial participants.The drug is taken orally.

Update onCeftriaxone

At 2 p.m. Monday, Sept.26, an Internetconference hour is setfor an update on theCeftriaxone study.

Clinical trial participants, caregivers, and othersinvolved in the Ceftriaxone study are welcometo join the conversation. It will include Dr. MeritCudkowicz, 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 thanenter both first and last name, please use eitherfirst initial and last name, or the reverse, forthe sake of privacy.

Youngjin Lee,


16/20

1

Can familial ALStroublemakers worktogether to keep

neurons strong?News arrived this month that two recentlydiscovered ALS familial genes, FUS/TLS and TDP-43, can actually work together to help keepmotor neurons healthy.

The study by Columbia University MedicalCenter showed the genes work in tandem tosupport the long-term survival of motor

neurons. Results were published in the Sept. 1edition of the Journal of Clinical Investigation.

Any therapy based on this discovery is probablya long way off. Nonetheless, its an importantstep toward piecing together the various factorsthat contribute to ALS, says the studys leadauthor Brian McCabe.

The two genes make proteins with similar formand function, which suggested to us that they

could work together, and that disruptions ofeither gene would affect neuronal survival, Dr.McCabe said. The other view? That geneabnormalities create a toxic combination tomotor neurons, regardless of their normal role.

Researchers used the venerable fruit fly to sortout the good from the bad theories. The hope?That a strong normal gene could help out aweaker, mutant one, and keep the motor

neuron stable.

Cogane, a drug in clinical trial for ParkinsonsDisease, has been granted orphan drug status

for potential treatment of ALS. The drug is in aPhase 2 trial for Parkinsons, but is in pre-trialstatus for ALS. The orphan drug status grantedby the FDA gives drug companies incentives todevelop cures for diseases affecting fewer than200,000 people in the U.S.

The drug is in very early stages of developmentfor ALS; however, important information will begleaned from the Parkinsons trial, which couldexpedite a clinical trial for ALS if the current

study testing the compound in the ALS mousemodel shows some promise, said ALSA chiefscientist 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 acause 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 causetoxicity and motorneuron deathA 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 majortrigger 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.

VasanthiJayaraman

DonCleveland


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For the first time, humanastrocytes show theyre toxic tomotor neurons.

Although researchers have already tested thetoxicity of astrocytes (the connection betweenneurons and other tissue), all of the tests havebeen with ALS mice. Now, scientists havelaunched the first study using astrocytes frompeople who died from either sporadic or familial

ALS.

Lots of attention centers on SOD1, the first familial genetic glitch identified in the early1990s.Researchers were able to create a new model of the disease, and confirmed that theastrocytes become toxic to motor neuron cells. They showed the cells secreted toxic factors thatcaused the motor neurons to degenerate.

So, when they lowered the levels of SOD1 in the human astrocytes, both the familial and sporadicforms had lower toxic impact on their motor neurons. Thats good news, because the resultsimpacted 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 astrocytereplacement would be a feasible therapeutic approach, said ALSA Chief Scientist Lucie Bruijn.

Reporter mice give real-timeinside look at ALS diseaseJasna Kriz, associate professor at LavalUniversity, Quebec City, Canada, haswon a three-year $445,086 MDA grantto generate ALS imaging reporter mice.Noninvasive, three-dimensional imagesreveal early neuronal stress in live bio-luminescent reporter mice. Signalsimaged from these animals can be usedas biomarkers to isolate distinct elements of theALS disease process, Kriz said, including indica-tions representative of presymptomatic stages ofthe disease.

Previously, Kriz and colleagues de-veloped mouse models to visualizeALS-related events such as neuroin-flammation and neuronal damage in

the brains and spinal cords of livingmice. They even were able to de-tect distinct and disease-specificsignals linked to presymptomaticstages of the disease.

Now Kriz plans to use the previously generatedmodels to create the ALS imaging reportermice, 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 badamong CD4+T cells in im-mune-system dysfunc-

tion:$180,000 over three years toJunping Xin, Loyola Universi-ty, Chicago; Astrocytes in the

central nervous system, magnified 100 times inphoto. Neurons appear round and red. The as-trocytes (motor neuron support cells) are yellowand activated, signifying an inflammatory re-sponse in the nervous system. Following nerve-cell injury, moderate inflammation is helpfulfor motor neuron regeneration, but uncon-

trolled inflammation may result in the death ofnerve cell body instead.

Deciphering signals between cells thatprovoke disruption of mitochondrial

function

Above: (A) Normal muscle fibers; (B,C) Musclefibers with perturbed mitochondria. Dashes in-dicate muscle diameter, and asterisks illustratethe space between individual muscle fibers.Normal muscle fibers adjoin each other and arelonger in diameter than muscle fibers with per-

turbed mitochondria. In contrast, muscle fiberswith disruption in mitochondrial function sepa-rate from each other and appear either split (B)or angulated (C). Edward Owusu-Ansah, HarvardMedical School, won $180,000 over three yearsfrom MDA for his research in mitochondrias mo-lecular mechanisms.

Test ing a possible ALS combinat ion

drug t herapy in mice

One process suspectedto be heavily involvedin ALS disease progres-

sion is inflammation,which can create atoxic environment andkill motor neurons.

Using the SOD1 ALS research mouse model, Ray-mond Grills research team will test the hypoth-esis that a drug called Licofelone will enhancethe 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 theUniversity of Texas.

The photo shows a microscope scan of microgli-al cells that support nerve cells, taken from thelower spinal cord of a mouse.


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Remember t o regist er!If you have ALS, especially if youve been

recently diagnosed, dont lose a minute before

signing onto the National ALS Registry. Started

just a year ago, the site is already expanding itsscope 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, flashdrive help ALS PALS, CALS

When an emergency strikes, first responders canhave trouble quickly assessing the situation, or

whats happening to you.

For people with ALS, key information is particularlycritical because signs and symptoms, such asmovement or communication limitations, couldeasily be misconstrued and lead to a misdiagnosis.

While you want MedicAlert bracelets andinformation systems to beeasily noticed, they no

longer have to look clunkyor unattractive. And iftheyre linked to amedical-informationservice or include a well-marked USB flash drive,they can include a widerange of information,including all diseases or

conditions, blood type, allergies, disabilities andfamily and friends to contact.American Medical ID sells a flash drive that looks

like a dog-tag necklace, updated by computer, forabout $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

AMedInfoChipis$50/year

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

WeWeWe

RememberRememberRemember

Louis KolenbranderJames SampselLynn CooperLucille TurchettaMargaret BrechtingThomas WiegandNancy GardnerPatrice SteinhourBob BarkerJeannine BarreraSandra SimonClaire WanglerNicholas RitterShirley HolzimmerJean BakerSandra BayesJames ColeWilliam AveryJackie Music PitschColleen CottrellThomas Tepper

Frank ParkerRuth MorganSandra AdamsRobert Goick

We send heartfelt condo-

lences to all the families

and friends of those whove

passed away in recent

months. If weve 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:

Heres 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.

St atewide call-in:2-3 p.m., Dial toll-free 877-643-6951; passcode

31723015# to join the group

Third Mondays:

Bay Cit y:Zion Evangelical Lutheran Church, 545 7th St., Freeland

Third Tuesdays:

St atewide call-in:6:30-7:30 p.m., Dial toll-free 877-643-6951; passcode31723015# 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, Arnies Restaurant, 77 54th St.

616 532 5662

Documents

Sept 2011News Magazine