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Final Layout 1/03 SUMMER 2005 FSHA publication of the FacioscapulohumeralW Muscular Dystrophyatch Society Index FSH Society Facilitates Myostatin Inhibitor Research Myostatin Research . .Front Cover, 4, 5 By Daniel Paul Perez President’s Letter . .2, 3 Guide to Acronyms . .3 I want to illustrate how the FSH Soci- the studies with which she was involved. National Websites . .4 ety works, which attests to our unique I recommended that Dr. Whittemore In Memoriam: Karen Johnsen . .5 ability and networking efforts on behalf of talk with Dr. Tawil, who serves on the FSH Karen’s Dream for a Cure . .5, 6 FSHD. This story highlights our Her- Society’s Scientific Advisory Board and has In Memoriam: William T. Michael . .6 culean efforts to move FSHD into the done clinical trials on FSHD. He also is a Younger Research Grant . .6 Gala Benefit . .7-10 member of the multi-center Muscle Study mainstream for research. After years of FSH Society Funded Grants . .11-15 neglect in FSHD research, the FSH Soci- Group (MSG) that is exclusively designed FSH Society Grant Application Process . .15 ety has made great strides behind the and structured to do clinical trials on mus- First FSH Society Roberts Grant . .16, 17 scenes. Much of the work that we do is cle disease. Additionally, Dr. Tawil runs Second FSH Society Roberts Grant . .16 confidential. However, when the work the NIH FSHD National Registry (see 2005 Bronfman Grant . .17 results in this kind of study, it is time for article, page 36) at the University of 10th Bronfman Fellowship . .17 7th & 8th Delta Railroad Grant . .17 Rochester. us to blow our own horn and let you Tides Foundation Grant . .17 know what our organization, with your On March 11, 2003, Dr. Kathryn Wag- MDCRC Fellowships Available . .18 support, is ner, M.D., Ph.D., NY Community Trust Grant . .18 accomplishing There have been very few clinical trials in FSHD a neurologist and NIH Program Announcements . .18-20 every day. Since and [we have] helped Dr. Wagner position her researcher at Congressional Testimony . .20-23 the Society does Johns Hopkins CRCs Open . .23 Funding Arrays . .24-27 work on myostatin inhibition at Johns Hopkins Hospital in Balti- not have a pub- Tracking MD Funding . .24 lic relations and with the pharmaceutical industry which repre- more, requested NHGRI Funding . .24-26 marketing team, sents, for the first time since the discovery of my assistance. Dr. History of the MD-CARE Act . .28-30 this is our Wagner was col- What is CRISP? . .30 FSHD in 1886, a serious candidate therapy to opportunity to laborating with Combined Federal Campaign . .30 credit ourselves treat, ameliorate and reverse the effects of FSHD! Se-Jin Lee who Porter Named NIH MD Director . .30, 31 discovered and NIH MD Program Staff . .31 where credit is Albuterol Trial Stalled . .31-33 due. This is the real story behind the has done much of the subsequent work on van der Maarel & Frantz Article . .32 myostatin-inhibitor trials and the years myostatin, an inhibitor of muscle growth. First FSHD Medical Textbook . .33, 34 invested into seeing it come to pass. Their work appeared in the December Chromatin Structure . .34 In the autumn of 2001, Lisa-Anne 2002 issue of “Annals of Neurology” on Toronto FSHD IRC Workshop . .34, 35 Whittemore, Ph.D., from the Wyeth the effects of inhibiting myostastin in a National DM & FSHD Registry . .36 mouse model of muscular dystrophy. Respiratory & Breathing Issues . .36 Ayerst Genetics Institute in Cambridge, Non-affected Family Members . .36 MA became a professional member of the Dr. Wagner wanted to talk with the Early Onset FSHD & IFSHD . .37 FSH Society. I contacted her to see if the Society about applying this method to Pregnancy & Delivery Study . .37 FSH Society could be of further assistance, FSHD. I, of course, wrote a lengthy Brain & Tissue Banks . .37 and to inquire about her areas of interest. response outlining why FSHD would be an Prenatal Genetic Testing in US . .38 At that time, Dr. Whittemore had ques- excellent candidate. Around this time, Dr. Mission Statement of Society . .38 Tawil said he and his colleagues at the Making a Bequest . .39 tions regarding muscle satellite pool cell Bulletin Board & Chat . .39 exhaustion in FSHD and the current state MSG were interested in myostatin- Society Welcomes LaPlante . .39 of clinical trials on FSHD. We referred her inhibitor research. I had Drs. Wagner and First Hand: Aubrie Lee . .39, 40 to the top three researchers in molecular Tawil contact one another. First Hand: Justin Cohen . .40 genetics and cell biology: Drs. Winokur, Dr. Wagner asked if we could provide Dardonville Raises Funds . .40 Figlewicz and Frants. Also, we referred the number of FSHD patients within one End of Tax Season Bar Crawl . .40 hour driving distance of Johns Hopkins. The Third Choice . .41, 42 her to the top clinicians in research trials: Scooting Across the Sea . .42 Drs. Tawil, Kissel and van der Kooi. I Thanks to Karen Johnsen and her Mid- Visit us @ www.fshsociety.org . .43 indicated the senior people who work Atlantic Support Group, as well as Carol Read-a-Thon in MD . .43 with Tawil, Kissel and van der Kooi as Perez with the national list of FSHD Memorial Weekend Garage Sale . .43 Drs. Griggs, Mendell and Padberg. Due to patients, within 24 hours, we were able to Acknowledgements . .43-47 the confidential nature of her work, Dr. provide the total number of affected Adapted Van For Sale . .43 FSHD patients listed with the Society, not- Thank You! . .47 Whittemore asked that we not disclose FSH Society Fact Sheet . .47 her name to FSHD researchers regarding continued on page 4 Membership Application/Donation . .48 2 FSH WATCH NEWSLETTER I SUMMER 2005 Board of Directors Letter from the President Stephen J. Jacobsen, Ph.D., Chairman Choosing & Maintaining Direction in the Face of Gain & Loss Daniel P. Perez, President & CEO William R. Lewis, M.D., Vice-Chairman By Daniel Paul Perez, President and CEO, FSH Society, Inc. Richard A. Lefebvre, M.B.A., FHFMA, Dear Reader & Friend of the FSH Society, over the past eight years speaks brilliantly Secretary There has been remarkable progress in for itself. William Michael, C.P.A., Treasurer the past few years! The FSH Society is I More countries are joining the E. Ann Biggs-Williams growing and becoming even more produc- fight against FSHD – foreign funding Howard L. Chabner, J.D. tive thanks to you who have, throughout agencies, volunteer health organizations, William E. Hall Jr., J.D.* the years, been generous and forthcoming patient support and network groups are Louis M. Kunkel, Ph.D. with much needed help. organizing. C. Larry Laurello, P.E. In the past year, we have lost more I During this past season, we sub- William R. Lewis III, M.D. than our fair share of dear friends, family mitted testimonies before the U.S. Con- Theodore L. Munsat, M.D.* members and loved ones. Balancing these gress regarding the need for projects and Paul Schultz, M.D. * losses are the gains we have made in funding on FSHD (see pages 20-23). Robert F. Smith, J.D. FSHD research and community building. I The Society continues to work Z. John Stekly, Sc.D. These are our accomplishments in 2004 closely with the NIH, a federal research through to the present: agency responsible for muscular dystrophy Scientific Advisory Board I A promising new drug trial is being research. FSHD research programs are David E. Housman, Ph.D., Chair conducted with FSHD patients using slow in coming but the foundation for the Michael R. Altherr, Ph.D. Wyeth-Ayerst myostatin-inhibitor MYO- research programs through the NIH is Robert H. Brown Jr., M.D./D.Phil.* 029 (See front cover). secure. Rune Frants, Ph.D. I A study on pregnancy and preg- I The FSH Society represented by Robert C. Griggs, M.D. nancy outcomes in FSHD was initiated myself and our legal counsel, Morgan Stephen J. Jacobsen, Ph.D. and exclusively funded by the Society (see Downey, changed the complexion of U.S. Louis M. Kunkel, Ph.D. page 37). muscular dystrophy research forever by re- William R. Lewis, M.D. I A study was initiated by the Society writing the MD-CARE Act 2001 to William R. Lewis III, M.D. on defining infantile and early onset include all dystrophies and not just DMD. George W. A. M. Padberg, M.D. FSHD (see page 37). Muscular dystrophy research is now get- Rabi Tawil, M.D. I The Society is initiating a study on ting its long overdue and needed federal breathing and respiratory issues in FSHD funding. Though FSHD still has a long * Board Member Emeritus (see page 36). way to go, our concerns are being heard I Cutting edge research, clinical about the disproportionate funding within General Counsel projects and fellowships continue to push the nine dystrophies (see pages 28-30 for Morgan Downey the envelop establishing new ground for History of the MD CARE Act). Suite 300, 1250 24th St. N.W. high risk and new innovative research I I continue to serve on the U.S. Washington, D.C. 20037 directions for FSHD (see pages 11-15). federal government MD-CARE Act 2001 (202) 466-0580 We have invested more than $1.2 million Coordinating Committee to help oversee (202) 776-7712 Fax since the inception of the research pro- and devise the research and education gram in 1997. plan for all MD research. FSHD is promi- Executive Director I We organize and co-chair the nently and well represented in the new Carol A. Perez, M.Ed.
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