The NHLBI is part of the U.S. Department of Health and Human Services’ National Institutes of Health (NIH)—the nation’s biomedical research agency that makes important scientific discovery to improve health and save lives. We are committed to advancing science and translating discoveries into clinical practice to promote the prevention and treatment of heart, lung, blood, and sleep disorders including LAM. Learn about the current and future NHLBI efforts to improve health throughand scientific discovery.
Improving health with current research
Learn about some of the ways we continue to translate current research into improved health for people with LAM.
- Building on past NIH-sponsored research on sirolimus (rapamycin) as a treatment for LAM. In 2011, the NIH funded Multicenter International LAM Efficacy of Sirolimus (MILES) trial published key findings. NHLBI researchers supported the MILES trial, which showed sirolimus to be a safe and effective treatment for LAM. Read about how NHLBI continues to perform and fund research on sirolimus as a treatment of LAM. Read the NIH Media Availability: Sirolimus Therapy Alleviates Symptoms of Lung Disease LAM or Lessons from the MILES trial to learn about the NIH-supported scientific discovery that led to the current use of sirolimus to treat LAM.
- Trans-NIH funding of the Rare Lung Diseases Consortium (RLDC). NHLBI co-funds some RLDC activities with NIH’s the National Center for Advancing Translational Science (NCATS). Through funding the RLDC, NHLBI is supporting new clinical trials for LAM. Learn more about the RLDC, including its role with the Rare Lung Disease-Clinic Network and the annual international LAM research conferences it helps organize.
Learn about some of the research contributions we have made over the years to help improve clinical care and increased the life expectancy for people LAM.
- NHLBI Working Group on LAM. In 1995, the NHLBI organized a working group on LAM to determine what was needed to help find the cause and possible treatments for LAM. A critical recommendation from this group was to create a national LAM registry to store tissue and other biological specimens from LAM patients as a way to support basic and clinical research.
- NHLBI support for the LAM Registry and National Disease Research Interchange. In 1997, the NHLBI co-funded this national registry to obtain clinical data and blood and tissue samples from LAM patients. In 2007, management of the data and materials form this registry were transferred to the National Disease Research Interchange (NDRI). NHLBI helps fund NDRI activities, which include the collection and distribution of fresh LAM tissue to qualified researchers.
In support of our mission, we are committed to advancing LAM research in part through the following ways.
- We perform research. Our Division of Intramural Research, specifically its Cardiovascular and Pulmonary Branch, has an active Translational Research Section and Lymphangioleiomyomatosis (LAM) Research Program that was the Nation’s first research program focused on LAM. Read Participate in NHLBI Clinical Trials to learn more about any open and enrolling LAM clinical trials that our researchers are leading, such as our Multicenter International Durability and Safety of Sirolimus in LAM trial called MIDAS.
- We fund research. The research we fund today will help improve our future health. Our Division of Lung Diseases (DLD) oversees the LAM research that we fund. Watch a video of how past NHLBI funding has supported genetic research in LAM. Search the NIH Research Portfolio Online Reporting Tools (RePORT) to learn about research NHLBI is funding on LAM, including RLDC Multicenter Interventional Lymphangiomyomatosis Early Disease (MILED) trials that are studying whether early sirolimus use can prevent LAM from worsening. Read Participate in NHLBI Clinical Trials to learn more about any open and enrolling LAM clinical trials that we fund.
- We stimulate high-impact research. The NHLBI Trans-Omics for Precision Medicine (TOPMed) Program has the potential to help advance LAM research by helping to define lung health across the lifespan so we can more precisely determine when disease begins. Through TOPMed and other collaborations, we hope to advance science so health professionals will be able to better predict who will develop lung diseases, identify subtypes of disease, and deliver personalized interventions to preempt, prevent, and treat disease. Our Strategic Research Priorities highlight ways we may support research over the next decade, including new efforts that may be relevant to LAM.
Learn about other exciting ways we are advancing research to help improve the lives of people with LAM, as well as their clinical care. This includes identifying better treatments and biomarkers for severe disease and to find out how well patients will respond to treatments.
- Exploring the molecular role of estrogen in LAM to find new treatments. Because LAM primarily affects women, estrogen is thought to play a role in this condition. NHLBI-funded research has discovered that known signaling pathways are activated by estrogen in LAM. These pathways may be possible targets for new therapies in LAM, and NHLBI is funding research in this area.
- Biomarkers to predict which LAM patients will respond to sirolimus (rapamycin) treatment. Researchers from the NHLBI-funded MILES trial are trying to see whether VEGF-D can be a biomarker for LAM severity and help doctors predict which patients will respond well to sirolimus treatment. Early findings suggest that patients who show decreased VEGF-D levels over the course of sirolimus treatment are more likely to have improved lung function than patients whose VEGF-D levels are unaffected by sirolimus treatment.
- Participate in NHLBI Clinical Trials will discuss some of our open and enrolling LAM clinical trials.