sept 2011news magazine
TRANSCRIPT
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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
Kristen Munyan, RN, MSN-Ed
Care Services Manager, East
Robbie Banfill
Care Services Coordinator, West
Paula Morning
Development Director, [email protected]
Vicki Bouwkamp
Development Manager, West
Cathryn Rybicki
Accountant
Julia Bauer
Michigan Matters editor
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
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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
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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
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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!! !!
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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
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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, andcontributed to our Iron Horse Ride, andthe refreshing Gears and Beers after-party!
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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
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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 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!!
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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!
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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.
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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 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
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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 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.
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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.
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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,
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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