The Myositis Association (TMA) is excited to announce the groundbreaking development of the first humanized mouse model for sporadic inclusion body myositis (sIBM) achieved through TMA’s innovative Pilot Research Funding Program.
With the generous funding of The Myositis Association’s Pilot Research Grant, Dr. Thomas Lloyd, a member of TMA’s Medical Advisory Board, and his research team have successfully transplanted human muscle tissue from sIBM patients into mice in order to create an animal model that mimics the disease. This model can be used experimentally to understand sIBM better and to test new treatments.
sIBM is a severe muscle-wasting disease that causes weakness in the legs, forearms, and fingers. This rare disease affects an estimated 20,000 people in the United States, is twice as common in men than women, and is the most common myopathy in those over the age of 50. sIBM is characterized by damage and inflammation of the skeletal muscles and often results in increased risk of falls and ultimately loss of mobility.
As a result of the support from TMA, the team of researchers, led by neurologist and codirector of the Myositis Center at Johns Hopkins University School of Medicine Thomas Lloyd, MD, PhD, have successfully developed the first mouse model for sIBM, which will open the door for development of new molecular-based targeted treatments.
Kyla Britson, a PhD candidate and member of Dr. Lloyd’s research team, successfully transplanted human muscle tissue from sIBM patients into mice without a functioning immune system in order to mimic the disease in the animal model. After completing 300 xenografts, as the transplanted tissue is called, from patients with both sIBM and patients with other forms of myositis used as controls, the researchers have confirmed that, not only does the graft regenerate in the mouse, but it demonstrates similar characteristics to the human disease.
Researchers in Lloyd’s lab are now using RNA sequencing to determine if the xenografts still have the genetic characteristics as the original muscle biopsy. They are also using RNA sequencing to identify specific molecular pathways that are changed in sIBM biopsies as compared to controls.
The research team is hopeful that their model will provide better insights not only into sIBM, but also into the many other forms of myositis and the over 50 million individuals suffering from autoimune disease from some type of autoimmune disease across the US.
“Each year, TMA’s Research Program provides a number of small grants to fund novel pilot projects like this,” says Conrad C. Weihl, MD, PhD, chair of TMA’s Research Committee. “These are high risk but high reward research projects that would not otherwise attract traditional funding mechanisms from the NIH, for example, because they lack supporting data.”
With the preliminary data generated through the TMA award, Dr. Lloyd has already received additional funding from other organizations to continue this important work through the Peter and Carmen Lucia Buck foundation and the Catalyze-a-Cure foundation.
“The Myositis Association is hopeful that our funding of a mouse model will be the catalyst to jumpstart the creation of more novel research and therapy development in sIBM,” says TMA executive director Mary McGowan. “This project epitomizes the type of research that the Myositis Association’s Innovative Pilot Research Grants aim to fund. TMA is proud to collaborate with our researchers and fellows in our quest for world without myositis.”