Tag Archives: lymphangioleiomyomatosis

Episode 37: The MILED Trial and the Future of LAM Research

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In this episode of TSC Now, host Dan Klein celebrates Worldwide LAM Awareness Month (#WWLAM) and raises awareness of lymphanhgioleiomyomatosis (LAM) and a pivotal clinical trial in LAM that is enrolling right now.

Dan interviews Frank McCormack, MD, Professor and Director of the Division of Pulmonary, Critical Care and Sleep Medicine at University of Cincinnati and former Scientific Director of The LAM Foundation for 25 years. Dr. McCormack is the lead investigator for the Multicenter Interventional Lymphangioleiomyomatosis Early Disease (MILED) Trial, designed to answer the question of whether we should be starting sirolimus at low doses earlier in the course of LAM, before symptoms develop and while lung function is still normal, similar to the manner in which we treat diabetes and high blood pressure early to prevent future heart and kidney complications..

Dr. McCormack shares how the previous MILES trial which led to the approval of sirolimus for the treatment of LAM in 2015 gave women a safe and effective treatment and created the foundation for the current trial looking at whether earlier intervention may potentially prevent serious loss of lung function for women with LAM. He shares the eligibility criteria for the trial, the risks and benefits of participation and how those interested in participating can enroll. He also discusses the future of LAM research and how new tests, preclinical models and drug candidates are needed to accelerate therapies that don’t just impede the growth of LAM cells, but actually kills the cells to help restore lost lung function.

Researchers are currently seeking 10 additional participants for the MILED Trial.

You may be eligible if you:

  • Are an adult woman with LAM
  • Have an FEV1 greater than 70% predicted
  • Are not currently taking sirolimus

During the study, participants will:

  • Attend 8 study visits over 2 years (about one visit every 4 months)
  • Complete blood tests, a physical exam, and pulmonary function tests at visits
  • Answer questions about breathing, fatigue and quality of life
  • Take one capsule every day (1 mg sirolimus or a sugar pill) throughout the study
  • Record their pill taking and any side effects in an electronic diary

Participants will receive:

  • Physical exams, pulmonary function tests, a chest x-ray, and laboratory tests free of charge
  • Study drug (either 1 mg sirolimus or placebo) throughout the study
  • Reimbursement for travel expenses to attend each study visit

To get more information or to see if you may be eligible, please contact Susan McMahan Sellers, BSN, RN at SUSAN.MCMAHAN@UC.EDU or by calling 513-558-4376.

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Episode 31: The Present and Future of TSC & LAM Research

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In the final episode of TSC Now in 2021, host Dan Klein recaps the 2021 Virtual International TSC & LAM Research Conference: Driving Discoveries Beyond Boundaries, presented by Greenwich Biosciences and the Rothberg Institute of Childhood Diseases and co-hosted by the TSC Alliance® and The LAM Foundation. The conference brought together 179 people from 18 countries including researchers with a wide array of specialties and representing everything from basic to clinical science, who took part in plenary sessions, oral presentations, posters, and discussions around cross-cutting topics, including big data and clinical translation.

Dan chats with conference co-chairs Nishant Gupta, MD, Assistant Professor of Medicine at the University of Cincinnati, Director of the LAM Clinic Network, and Scientific Director of the LAM Foundation, and Rebecca Ihrie, PHD, Associate Professor of Cell and  Developmental Biology and Neurosurgery at Vanderbilt University about their impressions of the conference overall, what topics they found most interesting and how the TSC and LAM research community can build off the momentum of the conference and foster the next generation of TSC and LAM researchers. Dan also asks them what areas of TSC and LAM research they find most exciting and what they think the future holds for the field.

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Episode 25: Lymphangioleiomyomatosis & Worldwide LAM Awareness Month (#WWLAM)

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In Episode 25 of TSC Now, host Dan Klein recognizes Worldwide LAM Awareness Month (#WWLAM) by focusing on lymphangioleiomyomatosis (LAM), a rare lung disease affecting women that is characterized by an abnormal growth of smooth muscle cells, especially in the lungs, lymphatic system and kidneys. LAM is caused by mutations in the same gene(s) as TSC and is a common manifestation for women with TSC but can also occur sporadically with no other TSC manifestations. To help raise awareness of LAM on the last day of Worldwide LAM Awareness Day, this episode will focus exclusively on LAM, research being done to develop new treatments and ultimately a cure, and the organization that is leading the charge to improve the lives of women with LAM in the US.

First, Dan talks to Sue Sherman, MHA, Chief Executive Officer of The LAM Foundation (02:18). The LAM Foundation was founded in 1995 by a mother of a woman with LAM with a mission to urgently seeks safe and effective treatments, and ultimately a cure, for lymphangioleiomyomatosis through advocacy and the funding of promising research. They are dedicated to serving the scientific, medical and patient communities by offering information, resources and a worldwide network of hope and support. They discuss how The LAM Foundation has changed over the years, how their investment in research has helped pave the way for breakthroughs, how the organization pivoted during the COVID-19 pandemic, and how they are raising awareness of LAM during Worldwide LAM Awareness month.

Next, Dan talks to Hilaire Lam, PhD (14:22), Research Associate in the Division of Pulmonary and Critical Care Medicine at Brigham and Women’s Hospital and an instructor in medicine at Harvard Medical School. The ultimate goal of Dr. Lam’s research is to leverage mTORC1-driven mitochondrial alterations to uncover therapeutic targets for TSC and LAM. She shares more about the aims of her research, how she first became interested in LAM, what gaps exist in our understanding of LAM and how researchers are currently looking to address those outstanding questions to develop new therapies. She also discusses how she is motivated by women living with LAM and how their relentlessness inspires her to help find a cure.

Earlier this month in recognition of Worldwide LAM Awareness Month, the TSC Alliance hosted an e-webinar titled “LAM in TSC: Surveillance, Treatment and Research” presented by Souheil Y. El-Chemaly, MD, Clinical Director of the Center for LAM Research and Clinical Care at Brigham and Women’s Hospital. The webinar covered surveillance, management and treatment recommendations based on the latest consensus guidelines, clinical trials currently taking place and a look to the future of research in LAM. A recording of the presentation will be available on our e-webinars website soon at tscalliance.org/e-webinars.

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