The path may not be smooth, and the climb may be steep, but, together, we can climb mountains. Please join me in the fight against IBM.
Kevin Dooley, MD
Chairman, Cure IBM
Million Dollar Bike Ride
Cure IBM participated in the Million Dollar Bike Ride, a rare disease fundraising event sponsored by the Orphan Disease Center at the University of Pennsylvania. Team Cure IBM rode on June 8, 2019 in Philadelphia to raise money for an inclusion body myositis research grant.
Donations can still be made until the end of June, 2019. All donations are collected by the University of Pennsylvania, which will double the money, up to $30,000. All of the funds, 100%, will be distributed to fund IBM research. Scientists from all over the world are eligible to receive the grants. We’ve already raised enough for a grant of over $85,000. Help us get to $100,000!
Click on the large blue button below to donate by credit card!Donate to Team Cure IBM in the Million Dollar Bike Ride!
The Cure IBM Research Fund at Washington University in St. Louis, MO
Started with a personal donation of $10,000 from Dr. Kevin Dooley, the Chairman of Cure IBM, the new Cure IBM Research Fund will support inclusion body myositis research at Washington University, home to one of the top medical schools in the United States. Conrad Weihl, MD, PhD, a neuromuscular specialist and leading IBM researcher at Washington University, writes:
Our research group has made novel mechanistic insights into the pathogenesis of hereditary inclusion body myopathy (HIBM) and sporadic inclusion body myositis (sIBM). Hereditary causes of IBM, often due to a single genetic mutation, are rare but inform us about disease pathogenesis that may be common in sIBM. One example are disease mutations in two proteins associated with HIBM, VCP and SQSTM1, in which disease variants are also found in ~2-3% of patients with sIBM. These proteins participate in the degradation of intracellular proteins via a process known as autophagy or “self-eating.” Our lab is interested in understanding how disturbances in autophagy lead to sIBM.
Our more recent studies have harnessed the power of large data sets generated on patient tissues and patient DNA. By overlapping these datasets, we have identified risk alleles that may stratify patients or treatment strategies. Specifically, we identified variants in a novel risk allele, FYCO1 that is overrepresented in sIBM patients. FYCO1 functions similarly to that of VCP and SQSTM1 and further unifies autophagic degradation as a therapeutic target in sIBM.
Our current and future studies will: 1) Further define manipulation of autophagy as a therapeutic target in sIBM. 2) Develop proteomic signatures of sIBM pathology that may help to predict treatment responsiveness. And 3) Generate tractable models of FYCO1 associated sIBM.
Take me to the Washington University webpage for donations to the Cure IBM Research Fund
Your selection of the “Cure IBM Research Fund” is already entered in the second box, “I prefer to enter my own designation,” so you do not need to complete the first box. If you choose to send a check by mail, you will need to click the “Give by Mail” button on the right, and enter “Cure IBM Research Fund” on the Giving Form.
The IBM Research Fund at Johns Hopkins Medical Center
Started with a personal donation of $10,000 from Dr. Kevin Dooley, the Chairman and author of Cure IBM, the new IBM Research Fund will support inclusion body myositis research at Johns Hopkins, a leading medical center. Thomas Lloyd, MD, PhD, the co-director of the Johns Hopkins Myositis Center, writes:
For the last 10 years at Johns Hopkins, I have devoted myself to two goals: understanding the cause of IBM and defining better treatment options. In truth, it is the former endeavor that provides the latter options. Essential to that discovery process is understanding the root cause(s). With this in mind, I believe that research in genetics and animal model development holds great promise to change the way that we approach and treat IBM for the future. Along these lines, we are beginning two major research endeavors: (1) whole genome sequencing in sporadic IBM – this is the first such research effort to truly understand IBM at the genomic level, and (2) development of a novel mouse “xenograft” model of IBM using patient muscle biopsy tissue – our early data suggests this is very promising, and we are testing new IBM therapies in this mouse model to determine if they have benefit in human IBM diseased muscle.
Take me to the Johns Hopkins webpage for donations to the IBM Research Fund
If you would like to send a check by mail, please call the number listed near the top of the Johns Hopkins webpage to receive instructions.
The Myositis Association
In 2018, The Myositis Association (TMA) will celebrate its 25th anniversary. TMA improves the lives of patients with all types of myositis, including inclusion body myositis, polymyositis, dermatomyositis, and juvenile myositis. The Myositis Association provides extensive education for patients and physicians. The Annual Patient Conference is highly regarded, and regional KIT (Keep in Touch) groups provide additional opportunities for patients to receive support from one another.
When you contribute to The Myositis Association, your donation can support the broad spectrum of TMA activities, or you can specify that you would like your donation to be restricted to supporting inclusion body myositis research.
During the past five years TMA has provided IBM research funds for studies of genetic sequencing, hand function and quality of life, characteristics of patients who test positive for the NT5C1A antibody, and a mouse xenograft model of IBM. Most recently, in 2017, TMA awarded a grant of $50,000 to Tahseen Mozaffar, MD, University of California, Irvine, for a small pilot study of the drug dalazatide, a novel compound derived from sea anemone venom.
Take me to The Myositis Association website
by Kevin Dooley, MD
Revised November 5, 2018