21St Century Cures Act Passes the House
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UMDF Quarterly publication connect Volume 19, Issue 3, Summer 2015 21st Century Cures Act Passes the House t is now up to the United States Senate to the testing and approval of new treatments will vote on the 21st Century Cures Act. During be provided to the Food and Drug Administration the UMDF’s “Day on the Hill” in June, we (FDA). encouraged participants to ask their House and ISenate members to support the measure. Also Under the measure, clinical research trials, vital in known as H.R. 6, the bill passed the U.S. House on providing new medications and therapies, will be July 10, 2015, with bi-partisan support. expanded. The 21st Century Cures Act was introduced in the The 21st Century Cures Act has received House because health care innovation is moving widespread praise from an array of doctors, very fast. From the mapping of the human genome researchers and experts in medical innovation. to personalized medicines linked to advances in molecular Dr. Francis Collins, Director of the National Institutes of Health, medicine, breakthroughs continue that are changing the way says he has “never seen a more exciting time” in transforming disease is treated, managed and cured. Those who proposed biomedical research. the legislation say federal drug and device approvals lag far behind the advances in medical science. That is why the Life sciences research drives resources and dollars into our legislation, led by House Energy and Commerce Committee hospitals and educational and scientific institutions, helping them Chairman, Fred Upton (R-MI-06), was introduced. prepare the next generation of scientists and doctors. The bill will help new scientists begin careers in research and apply their The 21st Century Cures Act helps to modernize the research skills to tackling disease. It enables scientists and caregivers to infrastructure and streamlines the bureaucracy between patients more effectively communicate, collaborate and share data on the and cures for conditions like mitochondrial disease. The diseases they study. National Institutes of Health (NIH), whose budget has been stagnant over the past few years, will see an increase of $10 The measure now rests within the U.S. Senate. If approved billion over the next five years. More resources to speed along there, it will be sent to the President for his signature. With generous support from From the Chairman Contents UMDF News - pages 5 - 7 UMDF Member Stories - pages 9 - 11 UMDF Education - pages 12 - 13 UMDF Symposium - page 14- 23 Adult Corner - page 25 UMDF Development - page 26 - 27 e are coming off another a clinical trial and one supports a UMDF Events - pages 8 - 30 very successful UMDF potential treatment. You can read WSymposium, and I am more about these projects in this UMDF Connect - page 31 always amazed by the number of newsletter, but we are very excited people who travel from near and far about the potential these research to attend. No matter how much projects could bring to patients and BOARD OF TRUSTEES we know about mitochondrial families in the not so distant future. Patrick Kelley - Chairman disease, it is our job as patients and W. Dan Wright - Vice Chairman caregivers to continually expand As you read this, the calendar is John A. DiCecco - Treasurer/Secretary that knowledge. It should be rapidly approaching the month of Bruce H. Cohen, MD our personal mission to stay on September. It’s a busy month. Sumit Parikh, MD - SMAB Chair top of the latest information and Awareness week will begin on Brent Fields developments, because they seem September 20th. Awareness Week Hooper Hardison to be coming at a very rapid pace. I is sandwiched in between our 17 John Kieffer always come away from the UMDF Energy for Life Walkathons that Richard Leach Symposium energized after hearing will continue into November. I Sharon Shaw Reeder - Member Liaison the presentations and sessions that encourage you to do your part in Tyler Reimschisel, MD clearly point to the fact that we are raising awareness. Anything that moving forward toward potential helps us get the word out about Debra Schindler-Boultinghouse treatments and cures. If you were mitochondrial disease and its impact Charles A. Mohan, Jr. - Chair Emeritus able to be with us in Washington, is considered a success. Check out DC, in June, you no doubt heard the fall Energy for Life schedule at SCIENTIFIC & MEDICAL ADVISORY BOARD about the many clinical trials that are www.energyforlifewalk.org. Find Sumit Parikh, MD - Chair underway or about to start. These a walk near you and sign up a William Craigen, MD, PhD trials are critical to the affected team. Encourage family and friends Marni Falk, MD community, because they are paving to exercise their mitochondria by Amy Goldstein, MD the way towards better treatments participating in a walk. Andrea L. Gropman, MD, FAAP, FACMG and potential cures. Richard H. Haas, MB, BChir The path to a cure is made possible Marcia Haigis, PhD I am also delighted to report that through awareness and the power of Carla Koehler, PhD UMDF awarded nearly $500,000 the Energy for Life Walkathon! Dwight Koerbel, MD, PhD towards research. If successful, Mark Korson, MD two of these research projects could result in a faster diagnosis; Giovanni Manfredi, MD, PhD an additional two could result in Robert McFarland, MBBS, PhD potential therapies - one supports Patrick Kelley, UMDF Chairman Gerard Vockley, MD, PhD Kendall Wallace, PhD Richard Youle, PhD UMDF Connect | Summer 2015 3 advocacy DAY ON THE HILL ore than 230 UMDF to stimulate mitochondrial research; members and their families make the pre-clinical development Dino Scanio and his son, Giovanni, talk about how took part in the third “Day of therapeutics for the treatment of mitochondrial disease impacts their family with on the Hill” on June 18, mitochondrial diseases a focus area Rep. Gus Bilirakis of Florida. Dino’s son, Gianluca, M2015. After being bussed to the Hill in for the Therapeutics for Rare and has mitochondrial disease. the morning, members fanned out across Neglected Diseases (TRND) program the United States Capitol for scheduled within the National Center for Advancing meetings with their Congressmen and two Translational Sciences (NCATS); work U.S. Senators. to develop novel, safe and effective nutritional interventions for mitochondrial UMDF Members were educated on the disease; and The Office of the Director issues that are affecting the mitochondrial should create a coordinator for disease community and were instructed mitochondrial research who is empowered to provide three requests of their House to hold the various institutes accountable Members and one for each U.S. Senator. for their commitment and progress. Among the “asks” for Congress was Congress members were also asked a request for each Member to join the to support the 21st Century Cures Act, Congressional Mitochondrial Disease which does several things to help with the Caucus. Currently, 20 members of clinical trial process and the review and Congress are active on the caucus. approval of new drugs and biologicals. The goal for our “Day on the Hill” Micheal and Peggy Wichert of Husdon, Ohio, meet participants was to double the amount of For their meetings with U.S. Senators, with U.S. Senator Sherrod Brown. Congressmen sitting on the caucus and UMDF members asked each to sign participating in the numerous briefings onto a similar letter to Dr. Collins. That held each year to educate elected officials letter was written and signed by Senator about mitochondrial disease and its Barbara Boxer (D-CA). Numerous impact on individuals and families. Senators said they would review the letter and consider adding their name. UMDF members were also instructed to ask their Congressmen to sign onto If you were unable to participate in a letter, written by Rep. Jim McGovern Washington, DC, you can add your voice (D-MA-02) and Rep. Tim Murphy (R-PA- from home. UMDF invites you to visit the 18) to the NIH Director Francis Collins, UMDF Legisilative Initiatives page and Melissa Lane and her son, Parker, meet with MD, PhD. The letter to Dr. Collins asks the make appointments in your community Congressman Evan H. Jenkins of West Virginia. NIH to ensure that each of the relevant with your Congressman and U.S. NIH institutes are issuing mitochondrial- Senators. A complete outline of talking specific Requests for Applications and points, “asks”, and materials for your Program Announcements in an effort meeting can be viewed and downloaded to bolster inclusion of such research in at www.umdf.org/legislation. Our fourth “Day their various research portfolios; utilize on the Hill” will be held in Washington, DC, a portion of Common Fund resources in June 2017. UMDF Connect | Summer 2015 5 Stealth BioTherapeutics is committed to the development of therapies for mitochondrial disease and proudly supports the advocacy efforts of the UMDF To learn more about our work, please visit StealthBT.com or follow us on social media: @StealthBT Stealth BioTherapeutics news New Congressional Caucus New Therapeutic Approach Members Revealed uman Molecular Genetics recently published a study At the time the newsletter went to press, eight congressmen revealing a novel, promising therapeutic approach for joined the Congressional Mitochondrial Caucus as a direct result Hhuman mitochondrial disease based on the findings in of “Day on the Hill”. They are: human cells and animal models. Rep. Hank Johnson (D-GA-04) The study is entitled “Inhibiting cytosolic translation and Rep. David P. Joyce (R-OH-16) autophagy improves health in mitochondrial disease” and Rep. Ryan Costello (R-PA-06) was conducted by researchers at The Children’s Hospital of Rep. Jared Polis (D-CO-02) Philadelphia and Perelman School of Medicine at University of Rep.