The Lucy Engles TSC/LAM Medical Research Program

A message from Elizabeth Henske, MD, Program Leader, Lucy Engles TSC/LAM Medical Research Program; Founding Director, BWH Center for LAM Research and Clinical Care; Director, Brigham Research Institute.

2017 Report: Advances in Research

Thanks to your remarkable vision and generosity, the Lucy Engles TSC/LAM Medical Research Program supports innovative research efforts aimed at improving our understanding of tuberous sclerosis complex (TSC) and lymphangioleiomyomatosis (LAM). Led by Elizabeth (Lisa) Henske, MD, a dedicated, multidisciplinary research team is driving vital lines of study aimed at unraveling the mysteries of these complex diseases and paving the way toward improved methods of diagnosis and treatment.

The group continues to work diligently across three key program areas: pre-clinical research (cell- and mouse-based studies); clinical research and biomarker development; and clinical trials. This comprehensive strategy results in a robust translational research effort: discoveries made at the bench can be advanced to the clinic, where lessons learned in patient care then go back to the bench for further refinement. This powerful feedback loop helps to accelerate the pace of discovery and the evaluation of potential new treatments, offering new hope to patients and their families.

Dr. Henske and her team have leveraged this approach to remarkably productive results. Since the fall of 2015, they have published or contributed to 18 peer-reviewed articles, including several key review and policy recommendation papers that are shaping how we care for patients with TSC and LAM. We have provided you with an abstract of each paper via the links below, as well as a summary from Dr. Henske’s team explaining the key significance of each publication. As you read, we hope you take great pride in knowing that your support has been essential to each of these critical advances.

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Pre-Clinical Research

Through this basic science work, the team is creating new cellular and animal models of disease to gain insight into TSC and LAM disease processes and test potential new therapies.
Read bibliography.

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Clinical Research and Biomarker Development

This area of the program is focused on identifying and validating new disease markers, which are urgently needed to improve diagnosis, monitor disease progression, and identify better treatment options for patients with TSC and LAM.
Read bibliography.

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Clinical Trials

Through the development and assessment of potential new therapies, the team is working deliver improved treatment options to patients, including their ultimate goal: a cure.
Read bibliography.

On behalf of our patients, families, and staff, please accept our profound gratitude for everything you do to move this important work forward. Together, we are changing the future of medicine.