The landscape of MS therapies andapproaches to care is rapidly changing.An emphasis on symptomatic relief, aswell as controlling disease progressionare two major growths in MS research.

Improved insights in MS pathogenesisare leading to new hypotheses and sci-entific studies.

The European Committee of Research InMultiple Sclerosis meeting in Septemberwas attended by thousands of physi-cians and scientists devoted to theunderstanding and care of MS. Many ofthe presentations involved novelresearch developments and advances inMS therapy. Here are some of the meeting’s highlights:

Update on therapy

A new oral medication called Fampridine, which is designed to improvewalking and strength, is a novel approach to the management of MS andhas been recommended for approval to the Food and Drug Administration(FDA). Fampridine is designed to improve nerve conduction by blockingpotassium channels on the membrane of myelinated axons. In doing so,the electrical impulses can travel more efficiently to their targets. In two dif-ferent studies, approximately one third of individuals taking the medica-tion had a consistent improvement on 3 of 4 tests of walking speed. This“responder” group also had improved strength and felt better overall.Responders included people who had more advanced MS, but were stillable to ambulate, albeit in some cases with assistance. It is estimated thatthis medication will become available in early 2010.

More options are now available to treat individuals with their first attack ofMS. The PreCise study, comparing Copaxone versus placebo for individualswith their first MS attack, showed that for the Copaxone-treated group, therisk of developing clinically definite MS (i.e having the second MS definingattack) was reduced by 45% compared to placebo, and the time to having asecond attack was delayed by 386 days more than in the placebo group.

Pediatric MSWinter 2009 The National Pediatric MS Center at Stony Brook University Medical Center

Progress Report:News in Research

www.pediatricmscenter.org SUPPORTED BY THE NATIONAL MS SOCIETY (631) 444-7802 1

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[ÉÄ|wtç ZÜxxà|Çzá4At this special time of year morethan ever, our thoughts turngratefully to those who havemade our progress possible.

We thank you and wish you a joy-ous holiday season and a newyear of peace and happiness.

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Dr. Lauren Krupp

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Please forward youremail address to:

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2 www.pediatricmscenter.org SUPPORTED BY THE NATIONAL MS SOCIETY (631) 444-7802

In a post-study analysis based on all patients who completed two years ofthe study without developing MS, there was a significant reduction in new"T2" lesions (43% during the first year and 52% over the entire two years) inthose who took Copaxone versus those who took placebo.

Based on these results, the FDA has now added Copaxone to Avonex andBetaseron as medications approved for individuals with Clinically IsolatedSyndrome (the first MS attack).

Positive data continues to accumulate on new oral therapies. The medica-tions fingolimod and cladribine have beneficial effects on relapse rate andMRI burden. More information is leading to a better understanding of theirsafety.

Other new findings may point to novel pathogeneic mechanisms in MS. In arecent study examining the blood outflow from major veins draining fromthe brain to the heart, clinical researchers detected abnormalities of veindrainage. The investigators found significant evidence of slowed andobstructed drainage in the veins draining the brain in many of those withMS. The investigators called this venous obstruction “chronic cerebrospinalvenous insufficiency.” They speculate that the reverse flow of blood into thebrain might set off the inflammation and immune-mediated damage thathas been well described in MS. If confirmed, these findings may open upnew research avenues into the underlying pathology of MS. Many questionsremain about how and when this phenomenon might play a role in nervoussystem damage seen in MS, and at the present time there is insufficient evi-dence to suggest this phenomenon is the cause of MS.

Research continued from page 1

About the NationalPediatric MS Center

The National Pediatric MS Center isa unique multidisciplinary clinicaland research program locatedwithin Stony Brook UniversityMedical Center, one of the world’sleading research institutions. TheCenter was the first of its kind inthe United States exclusively com-mitted to the care of children andadolescents with MS. It is a desig-nated Center of Excellence by theNational Multiple Sclerosis Society.

Our mission: We are committedto improving the lives of childrenwith multiple sclerosis andadvancing a research programthat will benefit all individuals withMS.

Director: Lauren Krupp M.D. Co-Director: Anita Belman M.D.Phone: (631) 444-7802Web: www.pediatricmscenter.orgMail: Stony Brook Univ. MedicalCenter, Dept. of NeurologyHSC T12 Rm 020Stony Brook, NY 11794-8121

What a nightto remember!Gaily dressedrevelers in“denim chic”filled the ball-room at the

Crest Hollow Country Club onceagain and helped raise funds to sup-

port the Center’s research efforts andTeen Adventure Camp.

Feet were stompin’ and dancers wereswinging to the fabulous music ofBig Shot, a Billy Joel tribute bandthat was the hit of the evening.

But it was the night’s honorees,Cynthia and George Marks, whoput it in proper prospective byreminding us all of the special reasonto support this event:

“Children get MS too, and we needto do all we can to help,” Cynthia saidas she accepted the PhilanthropistAward from Dr. Lauren Krupp.

Summer Soiree Exceeds Goal and Raises $190,000

Above: Hospital President Dr. StevenStrongwater (in back) stops to chat withhonoree, Cynthia Marks (seated center)and Merry Slone & guests at the Soiree

Left: The fabulous Summer SoireeCommittee

www.pediatricmscenter.org SUPPORTED BY THE NATIONAL MS SOCIETY (631) 444-7802 3

Updates in Pediatric MSResearchMany research studies focused on children with MS are underway. Children,in general, have a greater relapse rate early in their disease course. As part ofa network of six Pediatric MS Centers of Excellence, we found that most ofthe disease modifying therapies in adults can be used in children.

However, about one-third of patients continue to have relapses despitetreatment and go on to second-line therapies including Tysabri.

We have been part of another study that found that the youngest children(under 12) show marked differences in their clinical presentation on MRI andCSF findings relative to teenagers. Whether this younger group will providethe most information regarding the causes of MS remains a distinct researchgoal for our Pediatric MS Center and others.

As part of the network of Pediatric MS Centers, we are currently seeking NIHfunding to better understand the environmental and genetic interactionsthat lead to MS, including certain HLA genetic markers and Vitamin D levels,an environmental factor which is increasing in importance

Since our last newsletter, two papers from our group have been publishedon the children we have evaluated and another paper has just been accept-ed for publication. One of our collaborators, a group to which we sendblood samples from the children, has identified an antibody in the blood ofchildren directed against one of the components of myelin, myelin oligo-dendrocyte glycoprotein. This antibody is specific to this protein and bindsto brain glial cells which include oligodendrocytes, the cells that makemyelin within the central nervous system. Remarkably, this antibody wasmost often present in the very youngest children with MS (those less than10 years of age).

This finding might explain in part why the youngest children with MS haveclinical presentations that differ most from adults. Identifying antibody pro-teins which could be integral to the pathogenesis of the disease is one steptowards identifying interventions which could halt MS.

Almost all of the children we have evaluated for MS have continued to par-ticipate in our research studies and donate blood for various studies. It is thecourage of both these children and their parents which underlies all ourefforts to change for the better the course of all individuals with MS. Wethank them and all the friends for support in the crusade against MS.

FAQPediatric MSWhat are the types of MS in chil-dren?Virtually all children start with arelapsing-remitting course, character-ized by clearly defined attacks(relapses) of symptoms that subside(remit) on their own or with treat-ment. During the periods of remis-sion, there are no new symptoms orprogression of the disease. Otherforms of MS are rarely seen in chil-dren.

Is MS in children different than MSin adults? How?The symptoms of MS are similar inboth children and adults. However,some young persons with MS havesymptoms which adults rarely experi-ence. In a rare number of cases, thesesymptoms may include irritability andaltered mental status. It is not yetknown what the impact of the dis-ease is on the developing centralnervous system of a young personwith MS. The early onset of MS mayhave an impact on learning ability.Children with MS may experience dif-ficulty with school performance, par-ticularly with respect to concentra-tion and short-term memory.

Taken in part from KIDS GET MSTOO: Questions and Answers – apublication of the MS Society ofCanada and the National MS Society.

Have a question? Please submit to:johanna.biederman@stonybrook.edu

new ways to

G I V EG I V EMake your donations to the NPMS Center online via credit card, by simplygoing to Stonybrook.edu/pediatricmsgiving

We also offer memorial and honor donation envelopes; for extras contact631-444-8096 or johanna.biederman@stonybrook.edu

Our sixth year of summer campbrought a new model, with twooverlapping sessions.

Forty campers quickly got caught upin the excitement of the TeenAdventure Program. First timecampers settled in and by the timereturning campers arrived two dayslater, camp was running at fullswing.

This year’s camp also includedfour Peer Mentors, camp alumni who

are now in college and bring lifeexperience to the group.

This year’s campers included teensfrom Jordan and Panama, as well asa group from California, and ouralumnus from Alaska. Staff includedthe team from the National PediatricMS Center at Stony Brook University,a staff member from the LI chapterof the NMSS, as well as a number ofvolunteers.

Access-to-Adventure once againorganized the activities for thecamp, including the high ropescourse, kayaking, and a day sailing inNewport Harbor.

To close the session, Campersshowed off their music, dance andartwork skills in an innovative per-formance of “Where the Wild ThingsAre”.

The Campers are planning a mid-winter reunion. For more informa-tion, contact Maria Milazzo atMaria.milazzo@stonybrook.edu.

New Model for Camp MORE INTERNATIONAL CAMPERS JOIN THE FUN

National Pediatric MS CenterStony Brook University Medical CenterDepartment of NeurologyHSC T12 Rm 020Stony Brook, NY 11794-8121

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