Skip to contents
In This Issue:
BWH researchers from The BWH Center for LAM Research and Clinical Care have been awarded a grant from the U.S. Department of Defense Office of the Congressionally Directed Medical Research Programs to fund their work on lymphangioleiomyomatosis (LAM).
LAM is a rare lung disease that affects women almost exclusively. In LAM, the normal lung tissue is progressively destroyed. This disease can also occur in women with a genetic syndrome called tuberous sclerosis complex (TSC), which causes tumors in different parts of the body such as the skin, brain and kidneys.
LAM usually affects healthy, non-smoking, young women, according to Elizabeth Henske, MD, director of The BWH Center for LAM Research and Clinical Care. Symptoms, such as shortness of breath, often begin in the late 20s, and can worsen with pregnancy. LAM can lead to lung collapse, oxygen dependency, the need for lung transplantation and even death in young adulthood.
The researchers will use the $1 million-plus grant to study a combination of two drugs to treat LAM. The drugs they will test are hydroxychloroquine and sirolimus. Patients will be given different doses of the medications to find out which are safe and effective. The study will also evaluate the drugs’ effects on lung function, exercise capacity and quality of life. Independently, hydroxychloroquine and sirolimus treat different conditions. Sirolimus is an immunosuppression drug used after organ transplantation, while hydroxychloroquine is used to treat rheumatoid arthritis and lupus. This will be the first time the two drugs will be combined to test women with LAM.
“We will learn whether the combination of these two drugs is safe in women with LAM,” said Henske. “In our work in the laboratory, we have seen that this combination of drugs is more effective than either drug alone in models of LAM.”
This work builds on research by Andrey Parkhitko, MS, and Jane Yu, PhD, BWH Pulmonary Division, published in July in the Proceedings of the National Academy of Sciences. The grant, which spans four years, will open the doors to further collaboration between researchers and clinicians to learn more about LAM with the goal of providing other treatment options for the debilitating disease.
“This project brings together the scientific discoveries of laboratory-based investigators with clinical research driven by pulmonary physicians who care passionately about preserving lung function in women with LAM,” said Henske.
Souheil El-Chemaly, MD, BWH pulmonologist, adds, “It feels great to get funding to do this clinical trial. It is especially more rewarding since it was such a total team effort from the LAM and ILD (interstitial lung disease) team at BWH.”
The LAM Treatment Alliance is the founding collaborative partner and a major funding source of The BWH Center for LAM Research and Clinical Care.
Comments
Email Address:
Subject:
Comment: {Please limit your charaters to 300}