ACCELERATING the CURE

WINTER 2006 NEWSLETTER

GENE THERAPY: THE NEXT GENERATION NEWS FROM THE $4.2 MILLION LEAPS AWARD WILL JUMP-START PRESIDENT AND CEO DEVELOPMENT OF REGULATABLE GENE THERAPY The thread that runs through all of the he Michael J. Fox Foundation for The project will begin with a focus on two Foundation’s work is Parkinson’s Research (MJFF) has com- genes. One produces small molecule GDNF, the question we ask mitted $4.2 million to a team led by a potent neurotrophic factor; the other pro- T about every prospec- RheoGene Inc. to develop what could be the duces large molecule AADC, an enzyme tive research invest- next generation of gene therapy: a delivery involved in dopamine synthesis. Both genes may ment: What is the system that would provide clinicians a “switch” have therapeutic merit, and different technolo- potential of this tool, to control a gene implanted in a patient’s brain. gies may be required to regulate each. By work- study or trial to shorten patients’ wait for In collaboration with several academic institu- ing on both, the research team can widen the improved Parkinson’s treatments and, ultimately, tions, RheoGene will develop, optimize and test variety of genes to which the regulation switch a cure? Our driving goal is to propel scientific its “RheoSwitch® Therapeutic System” (RTS) they develop could ultimately be applicable. Like discoveries beyond academia, through the technology through Phase I clinical trials within all LEAPS awards, the project hinges on specific translational gap where promising ideas too often four years. While Parkinson’s disease is the first milestones that will determine whether work languish, and into the clinic for translation into application, the work will have broad applicabil- on one or both of the genes ultimately goes forward. meaningful therapies and interventions. ity to the safety and efficacy of gene therapies for many other diseases as well, and for their The RheoGene award is also a reflection of the This issue highlights some major recent Foundation’s increasing interest in neurotrophic accelerated advancement into the clinic. expressions of the Foundation’s commitment factor research, to which MJFF’s funding com- to translational research. They include a LEAPS “This project has potential to revolutionize the mitment to date totals about $8.5 million. award that could spawn the next generation clinical application of gene therapy,” said Neurotrophic factors (also known as trophic or of gene therapy, a validation study of genes Deborah W. Brooks, MJFF president and CEO. growth factors) have long been considered one implicated in PD, and a project that builds on “It is a natural fit with the Foundation’s commit- of the most promising avenues of Parkinson’s a promising Alzheimer’s therapy to drive a ment to drive innovative technology that will research, as they promote survival and improve potential new treatment for Parkinson’s. have a significant impact on patients’ lives.” function of neurons. (For more information on trophic factors, see box, page 2.) You’ll also see, as a special insert, our 2005 Gene therapy has been touted for years as Progress Report. As we enter our sixth year, a prospective cure-all for a wide range of health “We are very excited that the Fox Foundation we assess our progress and accomplishments ailments. But its development as a widespread has recognized RheoGene’s ability to create to date, including driving the first dopaminer- therapeutic technique has been hampered by leading-edge technology,” said Thomas Tillett, gic human stem cell line and the first large- the lack of any way to time or finely adjust doses CEO of RheoGene. “This grant will enable us to scale genetic map of Parkinson’s — tools with or to “turn off” a gene once it has begun continue to build on our successes with RTS potential to transform the way Parkinson’s is expressing a protein in the brain. The to date in creating innovative solutions that pro- diagnosed and treated. RheoGene-led team will work to establish RTS vide safer and more effective gene therapies.” as a safe and effective means to regulate both We take pride in what we’ve achieved so far, but the level (dose) and timing of gene expression The award is made under the Foundation’s we won’t rest until we reach our goal: a world using an orally administered activator drug, or LEAPS (Linked Efforts to Accelerate Parkinson’s where Parkinson’s disease is only a memory. “on switch.” The advent of RTS would furnish an Solutions) initiative. LEAPS are multi-year, multi- unprecedented safety mechanism by allowing million, multi-disciplinary projects to address Warm regards, gene expression to be completely shut off in the questions that will have significant practical event of adverse side effects, simply through impact on the understanding and treatment of withdrawal of the activator drug. Parkinson’s disease. Deborah W. Brooks President and CEO See Page 4 for Members of the RheoGene-led LEAPS Team

PAGE 2 FOUNDATION SUPPORTS CEREGENE INC. PHASE 1 CLINICAL TRIAL IN THIS ISSUE PAGE 3 MJFF-FUNDED RESEARCHERS PUBLISH FIRST WHOLE-GENOME MAP OF PARKINSON’S PAGE 4 $3.1 MILLION FOR POTENTIAL NEW THERAPY TO STOP PD PROGRESSION Research News

BOARD OF DIRECTORS FOUNDATION SUPPORTS CEREGENE, INC.

Michael J. Fox PHASE I GENE THERAPY CLINICAL TRIAL Holly S. Andersen, MD Eva Andersson-Dubin, MD he Michael J. Fox Foundation has com- for more routine procedures such as blood tests). Mitchell Blutt, MD Barry Cohen mitted $740,000 over three years to Assuming successful results, measurements from Donny Deutsch T Ceregene, Inc., a San Diego-based the Phase I study will lead to more efficient plan- David Einhorn biotechnology company focused on the develop- ning of a larger Phase II study that will gather Karen Finerman ment of gene therapies for neurodegenerative more detailed data on both safety and efficacy. Nelle Fortenberry Al Glickman disorders. The grant, announced in November, David Golub will help fund Ceregene’s Phase I clinical study of “Extensive studies in animal models, including the John Griffin CERE-120, a new gene therapy product that has most widely accepted models of Parkinson’s Rev. Msgr. Thomas J. Hartman shown potential in pre-clinical testing to slow or disease, have consistently demonstrated that Jeffrey Katzenberg Kathleen Kennedy stop the progression of Parkinson’s disease by CERE-120 is safe and well tolerated in animals Morton Kondracke using a viral vector to deliver neurturin, a potent even at doses hundreds of times higher than the Edwin Levy nervous system growth factor. equivalent doses being tested in humans. These Nora McAniff studies also demonstrate that CERE-120 may be Kenneth Olden, PhD Douglas I. Ostrover “The Phase I trial of CERE-120 brings several able to improve symptoms as well as slow the Tracy Pollan Foundation priorities to bear,” said Deborah W. progression of Parkinson’s disease,” said Raymond George Prescott Brooks, MJFF president and CEO. “These include T. Bartus, PhD, Ceregene’s COO and principal Michael Price Lily Safra investigating the neurorestorative properties of investigator on the grant. Curtis Schenker neurotrophic factors [see box], advancing transla- Donna Shalala, PhD tional research, and shortening the time it takes The Foundation funding supplements Ceregene’s Daniel Spitzer, MD to turn basic research advances into meaningful own multi-million dollar investment in the study, Fred Weiss therapies for patients.” which is under way at the University of California, President and CEO San Francisco Medical Center and Rush Deborah W. Brooks While the primary goal of any Phase I clinical University Medical Center in Chicago. study is to demonstrate safety, Ceregene will also SCIENTIFIC measure the efficacy of CERE-120 through brain “We’re pleased to have Fox Foundation support ADVISORY BOARD imaging studies and standardized Parkinson’s to optimize our clinical tests of CERE-120,” said tests. MJFF support will significantly enhance the Jeffrey M. Ostrove, PhD, president and CEO of Alberto Ascherio, MD, PhD speed and depth of this data collection, allowing Ceregene. “This funding will allow us to gather, Erwan Bezard, PhD Anders Bjorklund, MD, PhD* for more regular testing of a wider range of neu- in the shortest time possible, the data needed to Susan Bressman, MD rological functions than would otherwise be pos- know if we are on to a safe therapy that might David J. Brooks, MD sible. Each patient will undergo a PET scan and a slow or stop the progression of the disease — Robert E. Burke, MD Marie-Francoise Chesselet, MD, PhD* full battery of neurological tests every three something no treatment on today’s market can do.” P. Jeffrey Conn, PhD months (in addition to regular visits to the clinic Continued on Page 8 Mark Cookson, PhD David Eidleberg, MD Matt Farrer, PhD Chip Gerfen, PhD Fred Goldberg, PhD Why trophic factors? Tim Greenamyre, MD, PhD* Oleh Hornyklewicz, MD “Neurotrophic factors work in the brain the required for normal bodily movement and that Ole Isacson, MD (Dr. MD Sci) way fertilizer works in a field,” says Todd degenerate in people with Parkinson’s. Joseph Jankovic, MD Sherer, PhD, MJFF associate director of Gene Johnson, PhD* research programs. “As the fertilizer helps Other MJFF investments in neurotrophic fac- Jennifer Johnston, PhD* Jeffrey H. Kordower, PhD crops to thrive, neurotrophic factors promote tors to date include two current LEAPS (Linked J. William Langston, MD* survival and improve function of neurons.” Also Efforts to Accelerate Parkinson’s Solutions) Olle Lindvall, MD, PhD known as trophic or growth factors, these projects and two Community Fast Track 2004 Andres Lozano, MD, PhD* molecules have long been considered one of Kenneth Marek, MD projects. Of the LEAPS projects, one seeks to Eldad Melamed, MD the most promising avenues for Parkinson’s develop a regulatable gene therapy delivery Kalpana Merchant, PhD therapies and are a priority for MJFF,which has system (see page 1) and one investigates C. Warren Olanow, MD funded approximately $8.5 million in growth encapsulated cell technology as a delivery Theo Palmer, PhD Ira Shoulson, MD factor research to date. mechanism for GDNF. Under Community Fast Clifford W. Shults, MD Track, one team evaluated two proteins from David Standaert, MD, PhD Neurturin, the growth factor in the Ceregene the neuregulin family of growth factors as Dennis A. Steindler, PhD trial, is a member of the same protein family as potential therapeutic agents in animal models, Clive Svendsen, PhD Caroline Tanner, MD, PhD GDNF (glial-derived neurotrophic factor), and another examined a molecule called G. Frederick Wooten, MD which has previously been tested in people pleiotrophin as a potential neuroprotective/ Michael Zigmond, PhD with Parkinson’s. Both maintain survival of the restorative agent for dopamine neurons in *Executive Committee dopamine-producing nerve cells that are animal models.

2 WINTER 2006 ACCELERATING the CURE Research News

FOUNDATION DRIVES FIRST WHOLE-GENOME Validating the MAP OF PARKINSON’S DISEASE results of the Perlegen/Mayo study FINDINGS HIGHLIGHT 12 SUSCEPTIBILITY GENES BUT NO “SMOKING GUN”; VALIDATION STUDY UNDER WAY To determine whether the initial Perlegen/ Mayo findings hold in the broader population n fall 2005, researchers at the Mayo Clinic association of about 200,000 single-letter varia- and to home in on which if any of the SNPs and Perlegen Sciences, Inc., funded under The tions in the genome known as single nucleotide may merit additional scrutiny, the Foundation Michael J. Fox Foundation’s LEAPS (Linked polymorphisms, or “SNPs” (pronounced “snips”), I has quickly pushed forward with a validation Efforts to Accelerate Parkinson’s Soliutions) initia- in people with Parkinson’s disease. The study study of major speed, size and scope. tive, produced the first large-scale whole-genome examined DNA from 1,550 people — 775 with, map of Parkinson’s disease. The research, and 775 without, Parkinson’s disease. Although 13 Using genetic resources available through the published in October in the American Journal of SNPs — found within 12 genes — were statisti- Edmond J. Safra Global Genetics Consortia — Human Genetics, highlights changes in 12 genes cally more common in PD patients than in healthy MJFF’s network of geneticists committed to that may increase the risk for Parkinson’s disease individuals, the size of any single SNP’s effect was sharing their population data on Parkinson’s in some people. However, the comprehensive small. This indicates that these single gene disease — the Foundation assembled three study found no genetic “smoking gun” — no variants contribute only slightly to whether some- large consortia of research teams that strong single genetic determinant of Parkinson’s. one has PD or not.These results may support the worked in tandem to validate the initial find- theory that rather than single genes, combinations ings. The consortia aggregated DNA samples As the field of “genomic medicine” expands at a of genes or gene-environment interactions may from some 10,000 Parkinson’s patients and rapid pace, the Perlegen/Mayo study represents the be necessary to develop most common forms of control subjects. For each DNA sample, the first large-scale attempt to assess the comprehen- Parkinson’s disease. researchers determined the SNP variant (also sive role of genes in Parkinson’s disease. known as a genotype) for all 13 SNPs identi- Other noteworthy findings include confirmation fied in the Perlegen/Mayo study. The resulting “If validated,” said MJFF president and CEO that variation in two previously known regions of data sets are now being analyzed. Once Deborah W. Brooks, “the discovery of these 12 the genome, PARK10 and PARK11, are likely asso- results of the analysis are available, later this potential susceptibility genes — genes that don’t ciated with Parkinson’s disease susceptibility. spring, the Foundation will coordinate with cause a disease outright, but might make a person Some of the other SNPs found to be associated the investigators to publish the findings in a more or less likely to develop it — could provide with susceptibility were in or near genes with peer-reviewed journal. new insights into what causes Parkinson’s. (See box, direct biological relevance to the disease. right, for more information on validation.) “Our goals for the validation study were “This is something we’ve wanted to do for years, twofold,” said Brian Fiske, PhD, MJFF associate In one of the most comprehensive genetic studies and now we finally had the technology and fund- director of research programs. “Naturally, it of any disease to date, researchers studied the Continued on Page 8 was of primary importance to better under- stand the initial findings and whether they hold in a larger set of patient populations EPIDEMIOLOGICAL STUDIES OF PARKINSON’S: worldwide. Our secondary goal was to pro- WHAT DO THEY MEAN FOR YOU? ceed in a way that would help unify the field of Parkinson’s genetics by clearly outlining and reliminary results of epidemiological stud- “Epi studies play a crucial role in the research prioritizing the next stages of this work.” ies conducted by MJFF scientific advisor cycle,” said Katie Hood, MJFF vice president of P Alberto Ascherio, MD, PhD, recently led research programs, “by pointing up promising The research consortia for the validation to media reports that the regular use of ibuprofen directions for future research.” She noted that study were led by Lorene Nelson, PhD, of may delay or prevent the onset of Parkinson’s other epi studies have demonstrated a link to Stanford University; Haydeh Payami, PhD, of disease. But while the study is important and reduced Parkinson’s risk for behaviors including the Wadsworth Center/New York State intriguing, researchers emphasize that it is too soon increasing caffeine intake and smoking — generally Department of Health; and Alexis Elbaz, PhD, for people with Parkinson’s to draw the conclu- considered a bête noire of human health. of INSERM in France. Meta-analysis of the sion that they should begin an ibuprofen regimen. resulting genotype data they generated is “A smaller risk of Parkinson’s is not a good trade- being conducted by John Ioannidis, PhD, of the “It would be premature for people with Parkinson’s off for severely compromised cardiovascular University of Ioannina (Greece), an expert in to start taking ibuprofen or other anti-inflamma- health and a vastly increased risk of cancer,” said large-scale studies of this kind. tory drugs,” Dr.Ascherio cautioned. “A single epi- Ms. Hood. “No physician can in good conscience demiological study’s results do not directly trans- recommend that her patients take up smoking.” “The consortium approach brings a consis- late into prevention or treatment actions.” But further research into potentially neuropro- tency that would not be possible if multiple tective compounds found in cigarettes could groups tried to validate findings on their own,” To validate substances associated with increased eventually lead to a new Parkinson’s therapy. concluded Dr. Fiske. “And our compressed risk, he explained, multiple epidemiological (or timeline allows the validation results to follow epi) studies must be done. To validate the treat- As always, Ms. Hood added, the best way for people closely on the preliminary findings, maximizing ment potential of substances associated with with PD to optimize their treatment is to establish their relevance and utility to the field.” decreased risk (as in the case of ibuprofen), ran- and maintain a good relationship with their primary domized trials are needed. care physician and movement disorder specialist.

ACCELERATING the CURE WINTER 2006 3 Research News

MEMBERS OF PROTEOTECH AWARDED $3.1 MILLION FOR THE PROTEOTECH- DEVELOPMENT OF NEW THERAPY WITH LED LEAPS TEAM POTENTIAL TO STOP PARKINSON’S PROGRESSION

ProteoTech, Inc. he Michael J. Fox Foundation for alpha-synuclein in 2005 — one targets cellular Alan Snow, PhD Parkinson’s Research (MJFF) has commit- pathways hypothesized to block the toxic effects T ted a LEAPS award of $3.1 million over of a form of alpha-synuclein, and the other two Boston College three years to a team led by ProteoTech Inc. to look at ways to lower alpha-synuclein levels or Daniel Kirschner, PhD develop a treatment for Parkinson’s that can prevent its ability to aggregate. The LEAPS project, Professor of Biology disrupt or inhibit clumping of the protein alpha- however, aims to potentially leapfrog over the synuclein.This clumping is associated with the loss harmful/helpful debate about alpha-synuclein by University of California, San Diego of dopamine-producing cells in the brains of peo- testing the hypothesis that protein clumps in Eliezer Masliah, MD ple with Parkinson’s. The researchers theorize Parkinson’s are harmful through the development Professor, Neurosciences and Pathology that blocking it could prevent further cell loss and of a therapy to disrupt and prevent their formation. stop Parkinson’s disease progression. Consultant ProteoTech is a leader in research and develop- Manfred Weigele, PhD Compounds already shown by ProteoTech to be ment of new therapeutics derived from proteo- Former Director of Chemistry, effective in the test tube will be tested in cellular glycan and amyloid technologies for the treatment Hoffman-LaRoche U.S. and animal models of Parkinson’s disease. By the of major human diseases. This project leverages end of the three-year project, the team hopes to the company’s prior experience with protein Advanced Pharmaceutical Research, Inc. identify a compound and perform the preclinical clumping in Alzheimer’s disease. In the past five Anil Kumar, PhD testing needed to file an application with the FDA years Proteo-Tech has developed a small mole- CEO and Lead Chemist for a Phase I clinical trial. cule compound, Exebryl™-1, that in preclinical testing has been shown to markedly reduce brain Boston University “Researchers have focused a great deal of atten- beta-amyloid deposits in animal models of Benjamin Wolozin, MD, PhD tion on alpha-synuclein, but many questions about Alzheimer’s disease as well as to result in notable Professor of Pharmacology its role in Parkinson’s remain unresolved,” said improvements in and reversal of memory impair- Deborah W. Brooks, MJFF president and CEO. ments in these animals. “A chief goal of this project is to move the debate out of the lab and into the clinic. If successful, the “We’re excited that The Michael J.Fox Foundation work could speed the discovery of a ground- has recognized ProteoTech’s work in Alzheimer’s MEMBERS OF breaking therapy to slow or stop the progression as a significant base to build on in Parkinson’s dis- of Parkinson’s disease.” ease,” said Alan Snow, PhD,president and chief sci- THE RHEOGENE-LED entific officer of ProteoTech. “I look forward LEAPS TEAM Many neurodegenerative diseases share the trait working with this stellar team of researchers to of clumping of various proteins, although there is develop a disease-modifying small molecule ther- RheoGene Inc. considerable debate over whether the protein apy that we anticipate will help slow or even, (a wholly owned affiliate of the clumps in PD are a cause or effect, damaging or reverse the progression of Parkinson’s disease. University of Pittsburgh Medical Center) protective. Validating alpha-synuclein is therefore Dean Cress, PhD a high priority for the field, and the Foundation For information on the other award under this round of Mark Braughler, PhD funded three Target Validation projects targeting LEAPS funding, see page 1.

University of California, San Francisco Krys Bankiewicz, MD, PhD Professor, Neurological Surgery and CLINICAL DISCOVERY TO BE Principal Investigator, Movement Disorders ANNUAL INITIATIVE Research Program JFF has announced that it will make The Foundation launched Clinical Discovery in Northwestern University Clinical Discovery an annual initia- 2004 to significant response from the scientific Martha C. Bohn, PhD M tive, earmarking up to $3 million community. Four grants were awarded totaling Medical Research Council Professor and for the program in 2006.The program supports about $2 million: an investigation in China of Director, Neurobiology Program, Children’s small-to-medium clinical research projects that the potential neuroprotective effects of green Memorial Research Center apply cutting-edge Parkinson’s science directly tea; a trial of a novel strength training tech- to patients and patient care. nique for dysphagia, which occurs when the For more information, see story, page 1 muscles involved in swallowing weaken or do “Our goal is to drive novel therapies to patients,” not work properly; and two investigations of said Deborah W. Brooks, MJFF president and novel uses of Positron Emission Tomography CEO. “The lack of adequate funding for small- (PET) imaging to quantify changes in the brain to-medium clinical research projects sets up a associated with Parkinson’s onset. major roadblock to new treatment options.”

4 WINTER 2006 ACCELERATING the CURE Foundation Events

LAUGHTER IS THE BEST MEDICINE “FUNNY THING” RAISES MILLIONS FOR PARKINSON’S RESEARCH

he Michael J. Fox Foundation raised and a citrus medley garnished with gold leaf nearly $4 million at the fifth install- chocolate and passionfruit, appetites for laughs T ment of its gala event, “A Funny Thing were sated with rollicking stand-up perform- Happened on the Way to Cure Parkinson’s,” ances by Colin Quinn, Sarah Silverman and held Saturday, November 19, 2005, at the Wanda Sykes. In between stand-up sets, guests (L-R) Taylor Phinney, Connie Carpenter and Kelsey Phinney with Michael J. Fox. They were among more Waldorf-Astoria hotel in New York City. rocked out to music performed by the versa- than 130 Foundation supporters from across the country who came to New York City on Saturday, The evening’s costs were entirely covered by tile and talented all-star house band under November 19, 2005, for a special Research Roundtable and brunch at the Waldorf-Astoria hotel. Guests met the Foundation’s Board of Directors, meaning leader Simon Kirke. Band members included Michael J. Fox and MJFF president and CEO Debi Brooks, and learned about the latest advances in that every penny raised goes straight to cut- Rob Arthur,Tommy Burns, John Conte, Domino Parkinson's research from three of the Foundation's scientific advisors. Alberto Ascherio, MD, PhD, spoke ting-edge Parkinson’s research. Kirke, Paulette McDaniel and Mark Rivera. on the importance of epidemiology studies in under- standing PD; Anders Björklund, MD, PhD, talked about current strategies for restoring and replacing dopamine neurons; and Susan Bressman, MD, The event was As the evening drew toward its close, Michael discussed how to optimize current PD treatment options. The Roundtable was made possible by a hosted by comic J. Fox took the stage to thank everyone who generous gift from GE Healthcare. Jimmy Kimmel, contributed to the evening’s success — includ- who welcomed ing event co-chairs Karen Finerman and Barry the evening’s Cohen, the Foundation’s Board of Directors GOLFERS TEE UP FOR approximately 750 (with a shout-out to his “favorite Board mem- guests — includ- ber,” wife Tracy Pollan), and especially the loyal PARKINSON’S ing supermodels supporters whose generosity and continued Elaine Irwin commitment have made the Foundation a lead- Wanda Sykes Mellencamp and ing funder of Parkinson’s research. Maggie Rizer, candy impresario Dylan Lauren, business leader Ronald O. Perelman and adver- tising mogul Donny Deutsch — as they entered the Waldorf-Astoria’s striking Grand Ballroom and took their seats.The crowd was then warmly welcomed once again by actress

Amanda Peet, who told guests that she was Michael J. Fox’s fivesome: (L-R) Vincent Tese, Jimmy Cayne, Michael J. Fox, Ed Levy, and Lee Mikles personally grateful for their support of the Foundation because her own family has been he BREAKING PARkinson’s Fifth Annual touched by Parkinson’s. John Mellencamp Golf Outing to benefit The Michael J. T Fox Foundation for Parkinson’s Research, held on September 19, 2005, raised With that, Fox introduced the evening’s closing more than $1.7 million for Parkinson’s research. act: a musical set by American icon John The Foundation’s guests and 98 golfers — includ- Mellencamp, who thrilled the crowd with some ing Michael J. Fox, whose five-some finished in sec- of the biggest hits of his 30-year career, includ- ond place — enjoyed a fantastic day of sport and ing “Little Pink Houses,” “Small Town” and camaraderie, followed by cocktails, dinner and an “Scarecrow.” (L-R) Jimmy Kimmel, Sarah Silverman, auction. Colin Quinn, Michael J. Fox, Sam Fox and Tracy Pollan The Foundation joins Michael J. Fox in thanking Guests bid on an array of rare and wonderful As guests dined on sumptuous courses of everyone whose immense contributions made donated prizes that included a ride in the grilled shrimp with mango-cilantro salad, the event such a success. Goodyear Blimp, a summer internship at Deutsch tournedos of beef with roasted garlic confit, Advertising and, of course, more golf, including rounds on two spectacular courses: the esteemed National Golf Links of America in Southampton, NY, and Merion Golf Club in Ardmore, PA.

FOR INFORMATION ABOUT Held in honor of Robert Klein, the outing was co- chaired by Foundation Board members Holly UPCOMING FOUNDATION EVENTS, Andersen and Edwin A. Levy with Gene Gurkoff, PLEASE VISIT US AT Robert R. Greenberg, R.J. Nemer and Eric Rothfeld. An anonymous donor matched all 2005 WWW.MICHAELJFOX.ORG donations in honor of Stanko Stojkovic.The event took place at Deepdale Golf Club in Manhasset, New York.

ACCELERATING the CURE WINTER 2006 5 Team Fox & Community Events

FLUX CAPACITATOR FUTURE HOLLYWOOD SHOWS PROMISE AS FUNDRAISING DEVICE LEADERS RAISE THOUSANDS FOR PARKINSON’S

or the third year running, the Junior Hollywood Radio and Television Society F (JHRTS) made its annual holiday party a benefit for The Michael J. Fox Foundation. This year’s event raised close to $45,000 for Parkinson’s research, more than doubling the proceeds from the previous year’s event.

Uday Sehgal (right) and his brother Niraj pose in front of Doc Brown’s DeLorean The idea to dedicate the annual event to rais- ing funds for the Foundation came from JHRTS Jacob Fenton and Kiersten Robinson, JHRTS co-presidents Board members with a driving desire to find a n November 5, 1955 — as fans of cure. One had lost her father to PD; the other, to the assistant through coordinator levels Michael J. Fox and his iconic turn as Justin Sternberg, worked for Michael J. Fox at from any area in the entertainment industry — Marty McFly in the “Back to the O his production company, Lottery Hill, for especially assistants from television studios, Future” trilogy are aware — Doc Brown (played almost two years in New York during the “Spin networks, agencies and production companies. by the vibrant Christopher Lloyd) fell and hit his City” years. “It seemed like a natural choice,” head. When he came to, he had the idea for said Andrea Kavoosi, JHRTS vice president,“to “It is amazing,” concluded Ms. Kavoosi, “that history’s greatest invention: the flux capacitor,that raise money to cure a disease that affects most of the group organizing this event don’t marvel of modern science that would make time millions of people, including one of young make $40,000 a year themselves, but come travel a reality (in the movies, at least). Hollywood’s idols — Michael himself.” together once a year to raise that much to help the millions who live with Parkinson’s. Our One great idea often begets another. And so it The party, attended by about 800 society mem- success, as always, was a result of the energy was that Uday Sehgal of Los Angeles and his cre- bers and guests, was held at Privilege, a West and motivation of our committee and board ative group of friends were inspired with another Hollywood hotspot.Tickets cost $25 in advance who worked tirelessly. The majority of mem- stroke of genius. Realizing that November 5, 2005, and $40 at the door,and a raffle included prizes bers and board members of JHRTS are in the marked 50 years since the invention of time from Dell Computer and Coach. television business and Michael is as close to travel, this merry band set out to celebrate in a the hearts of people in the television industry manner befitting such an epic anniversary: with a JHRTS is a membership organization exclusive as you can get. We love him.” screening of “Back to the Future,” drinks and dinner to benefit The Michael J. Fox Foundation.

The “50-Year Flux Party” was a great success. Tickets were $50 to attend the screening, dinner The 2005 VFW Poker Run raised a celebration-worthy $17,000 for and party, or $20 just for the party. The event The Michael J. Fox Foundation. The annual event is sponsored by VFW raised more than $4,000 for Parkinson’s research Post 9362, Victory Riders Association (VRA) (Sun Prairie Chapter) and the from ticket sales, donations and a raffle. Proceeds Hanley Company. This year's total matched the proceeds from last year's event – were donated in honor of Daniel and Michele “I guess there are worse ruts to be in!” quipped Dan Clavette of VFW Walker and family in memory of their father, who Post 9362, who snapped this shot of poker sharks (L-R) Tom Gannon, suffered from Parkinson’s disease. Commander VFWPost 9362; Phil Gerg, Quartermaster VFWPost 9362; Tammy Coker, Manager VFWPost 9362; Larry Danielson Past VFW State Says Uday:“We had a wonderful time and felt very Quartermaster/Adjutant and “Parkinson’s Poster Boy”; Sue Manthe, Secretary, VRA; good about the results, both monetarily and with Jean Manthe, Treasurer, VRA; and Don Klein, President, VRA. regard to advocacy for the cause.”

INTERESTED Michael J. Fox at the 2006 AARP The Magazine's Impact Awards IN ORGANIZING Luncheon, held in New York City in December, with (L-R) host Paula Zahn and fellow honorees A FUNDRAISER Jane Kaczmarek and Harry Belafonte. The Impact Awards are given FOR MJFF? annually to 10 individuals whose “innovative thinking, wisdom, and leadership have improved LEARN MORE ABOUT OUR the world we live in.” Fox was also featured on the cover GUIDELINES AND GET SOME of the January/February issue of the AARP The Magazine, which profiled IDEAS BY VISITING all 10 honorees. WWW.TEAMFOX.ORG

6 WINTER 2006 ACCELERATING the CURE Team Fox & Community Events

ROSEMARY BEACH TEAM FOX MARATHONERS 5K RAISES RAISE MORE THAN $70,000 $14K FOR MJFF FOR PARKINSON’S RESEARCH The second Jim DelMauro Rose- Every Team Fox member finished the 26.2-mile mary Run, a 5K race, including New York’s Abbi Gleason, the organized by the 37th woman to finish (in 2:57:07). And of Rosemary Beach course, in addition to their impressive athletic (FL) Property Own- and fundraising feats, the Team Fox marathon- ers Association ers helped raise awareness of Parkinson’s in honor of Jim disease and the ongoing need for research dollars. DelMauro, raised nearly $14,000 Team Fox provides community fundraisers for Parkinson’s research on October 8, 2005. (Front row, L–R:) Susie Rosenthal, Chris Busbee, Dorrie Harris (Middle row, L–R:) Jennifer Abrams, with helpful resources,Web-based tools, and a Heather Allerdice-Gerow, Martha Ruest, logo to “brand” events as helping to find a cure The annual run/walk was inaugurated in 2004 and Natalia Daniel, Pam Presser (Back row, L–R:) Mike Kloepfer, Seth Degarmo, Heath Tohara, for Parkinson’s disease. Anyone can join the in its first year raised about $7,900. It honors Jim Eric Birnbaum (Not pictured:) David Sack, Dean Spignola, Becky Decker, Jennifer Shaw, team, and there are innumerable creative ways DelMauro, first president of the Rosemary Beach Ryan Roelle, Ray Camono, Derek Yan, Poonam Khanna, Abbi Gleeson, to have a great time while supporting the Property Owners Association.The event not only Jerry Costello, Colleen Reagan Foundation’s mission to find a cure for raises money for MJFF but is a natural tribute to eam Fox, the Fox Foundation’s new Parkinson’s. Supporters have organized golf the active lifestyle of DelMauro, a former grassroots community fundraising ini- tournaments, dedicated birthday and anniver- marathoner now living with Parkinson’s. T tiative, hit the ground running on sary celebrations to finding a cure, competed in Sunday, November 6 — the date of the 2005 walks, runs and triathlons, held tag sales and About 140 runners and walkers — including ING New York City Marathon. Some 22 block parties, flipped pancakes, and found so DelMauro — completed the 2005 race, whose runners came from as far away as California many other ways to involve their friends, family course goes through the center of town. Event and Florida and ran one of the nation’s most and community in this meaningful cause. organizers Lori Bradley and Kathy Kemp raised challenging courses to raise an astounding funds by approaching local businesses and individ- $70,000-plus for The Michael J.Fox Foundation. For more information on how you can get in uals for sponsorships, garnering a total of 40 spon- Team Fox supporters joined spectators from the game, visit www.teamfox.org or contact sors whose logos were featured on the official the Parkinson’s Disease Society of the United Amanda McDorman, the Foundation’s special event T-shirt. Runners also paid entry fees of $15 Kingdom along the route, cheering on all the gifts officer, at [email protected]. in advance or $20 on event day. athletes racing for the fight to end Parkinson’s. In spite of a nearly 100-percent fundraising increase over their first time out, the organizers are hardly resting on their laurels. They are working to continue building the annual event into a time- honored Rosemary Beach tradition, aiming to How can I get involved? increase the total amount raised by 20 percent each year. MJFF is deeply grateful for their com- Everyone is invited to join Team Fox, and there are so many ways you and your community can make mitment, which is crucial if the Foundation is to a difference in the fight against Parkinson’s. An initial contribution entitles Team Fox members to cross its own finish line and find a cure for download materials that make it fun to plan and stage an event.These include: Parkinson’s.

• The Team Fox logo; • “How to”guides to event planning; • Customizable form letters for enlisting • Sample press releases to publicize sponsors and thanking donors; the event and the cause.

Team Fox members are also invited to attend an annual spring recognition event in New York City.

For more information on how you can get in the game, visit www.teamfox.org or contact Amanda McDorman, the Foundation’s special gifts officer, at [email protected]

Jim DelMauro (center) crosses the finish line

ACCELERATING the CURE WINTER 2006 7 NON PROFIT US POSTAGE Paid PERMIT NO. 453 CINNAMINSON, NJ

Grand Central Station, P.O. Box 4777 New York, New York 10163 www.michaeljfox.org

WINTER 2006 NEWSLETTER

FOUNDATION ANNOUNCES NEW RFAS FOR DRUG DISCOVERY, MAMMALIAN MODELS OF PD

JFF is currently reviewing proposals ies determine whether altering the biological func- Parkinson’s disease — crucial research tools for submitted in response to two new tion of a target or pathway provides a beneficial testing neuroprotective and neuroregenerative M Requests for Applications (RFAs): effect in a relevant PD model.This essential step is therapeutic strategies in clinical studies. one to which neither academia nor industry has “De-risking” pharma investment in consistently devoted the necessary resources. While existing models can be useful for studying Parkinson’s — PD Drug Discovery and the pathology of specific mechanisms and path- Development is designed to validate the therapeu- The initiative is part of the Foundation’s commit- ways implicated in Parkinson’s, it is generally tic potential of scientific discoveries and push them ment to draw increased industry attention to recognized that no validated model in standard use one step closer to the clinic. MJFF will provide up Parkinson’s disease and ‘de-risk’ the investment of currently mimics the disorder’s gradual progres- to $1.5 million in funding to validate therapeutic R&D dollars for new PD therapies. sion in humans. targets involved in aspects of Parkinson’s including both motor and non-motor symptoms. An ideal model would reproduce as many features Building a better mouse model of PD — of Parkinson’s disease as possible, including The translation of basic science findings into ther- The $2-million Progressive Predictive Animal Models progressive dopaminergic degeneration and apeutic interventions requires additional applied of Parkinson’s initiative aims to catalyze the creation dysfunction and evidence for relevant protein work in the form of validation studies. These stud- of progressive, predictive mammalian models of aggregation.

“PHASE 1 TRIAL” CONT’D “GENETIC MAP OF PARKINSON’S” CONT’D

The Ceregene grant was funded in its entirety by the ing to make it happen,” said the study’s first The Mayo Clinic/Perlegen Sciences study was Pioneer Fund, a private family foundation that supports author, Mayo Clinic neurologist Demetrius funded by a one-year,$2.8 million LEAPS grant, endeavors including medical research, under the Maraganore, MD. with funding provided by prominent Foundation’s Clinical Discovery program. Clinical Wisconsin businessman George Prescott and Discovery is designed to stimulate well-designed clinical “In one year, The Michael J. Fox Foundation his family and matched by the Foundation’s research projects focused on potentially high-impact and the Mayo Clinic have generated results Board of Directors. The work also benefited approaches to Parkinson’s disease. “We committed to that will greatly focus future research efforts from longstanding funding from the National funding the CERE-120 trial because of its great poten- in Parkinson’s disease,” added David Cox, MD, Institute of Environmental Health Sciences tial benefit for people with Parkinson’s,” said Scott PhD, chief scientific officer and co-founder of (NIEHS), which supports the study of humans. Hamilton, Olympic gold medalist and Pioneer Fund Perlegen Sciences.“If replication of only one of Board member.“We are pleased to work together with these findings leads to a better understanding The Michael J. Fox Foundation to drive the kind of cut- of the causes of the disease or improvements ting-edge science that can lead to meaningful advances in early detection or treatment, we will have for the millions touched by this disease.” made significant progress.”

THE MICHAEL J. FOX FOUNDATION FOR PARKINSON’S RESEARCH IS DEDICATED TO ACCELERATING THE DEVELOPMENT OF A CURE FOR PARKINSON’S DISEASE THROUGH AN AGGRESSIVELY FUNDED RESEARCH AGENDA.

For questions or comments regarding the MJFF newsletter or Web site, please contact editor Holly Barkhymer at [email protected]