Our Recent Work - Not a Breath Wasted

Introduction

In just over three years, the LAM Treatment Alliance (LTA) has become the largest LAM research organization in the world and the driving force in advancing the cure for LAM. Whether we are introducing first-of-their-kind Global LAM Summits and monthly seminars for scientists, clinicians and patient-collaborators; catalyzing and funding ground-breaking collaboration among scientists on a rolling and peer-review basis; or forming high-performance partnerships with industry, top academic labs and clinical centers; the LAM Treatment Alliance is at the forefront of finding a cure for LAM.

Summits, Seminar Series, and Ad Hoc Meetings

Now widely recognized in the LAM and LAM-relevant scientific and clinical communities, the LTA has sponsored 13 international summits and more than 30 monthly LAM/TSC Seminars, or research meetings, to help facilitate and advance the cure. The Seminar Series is now heading into its fourth consecutive year and has been attended by more than 170 MDs and PhDs since its inception, with as many as 85 attendees at each meeting. Additionally, recognizing the need to move quickly to most effectively drive discovery in LAM science, the LTA convenes more than 85 ad hoc research collaboration facilitation meetings each year.

These efforts – combined with direct, large-grant funding, infrastructure development, fast-turnaround training support and pilot grants, tissue program support, quality control and facilitation, mutational analysis for the LAM community, and LAM cell validation "services" – have emboldened the LTA to, among other things:

• Rigorously pursue a field-wide revisiting of validation criteria for defining the LAM cell;
• Spearhead a fresh comprehensive look at strategic approaches to modeling LAM;
• Direct a revisiting of the influence of hormones in the pathophysiology of the disease;
• Foster and coordinate more than 35 strategic collaborations to address gaps and barriers to timely tissue availability for researchers;
• Revisit the cell-of-origin question as a key to understanding how to effectively treat LAM; and
• Create partnerships poised to undertake the first unbiased drug screen of FDA-approved drugs, which will lead to next steps for moving towards multiple treatment options for LAM and for actively working through our close partnership with the MIT Media Lab to improve hypothesis-generating tools available to researchers through the creation of a global LAM informatics system.

In sum, the LTA continues to rigorously and quickly facilitate problem-solving that will effectively overcome the most difficult challenges to finding a cure. Through fostering practical interventions, infrastructure development, aggressive treatment-focused funding of work that would not move forward otherwise but for the infusion of funds, we have spurred collaboration, recruited new highest-caliber junior and senior researchers to the field, created regular venues for pooling knowledge, and driven the process of mapping out and aggressive advancing of novel therapeutic approaches to treating and curing LAM.

Driving Critical Science

Based in Boston, MA, one of the world's medical meccas, the LTA takes advantage of direct and ongoing in-person access to world leaders in LAM research and thought leaders in scientific domains with a wide reach of global institutional ties. This access has resulted in close and active year-round trans-institutional working collaboration with partners including Lisa Henske, MD and David Kwiatkowski, MD, PhD of Harvard Medical School, Joel Moss, MD, PhD, (National Institutes of Health - Heart, Lung and Blood Institute), and Chris Austin, MD at the National Institutes of Health, National Chemical Genomics Center/TRND, and Scientific Advisory Board (SAB) membership including Bob Langer, ScD (Massachusetts Institute of Technology) and Sten Lindahl, MD, PhD (Karolinska Institute/University Hospital and Chair of the Nobel Assembly).

The LTA Collaborations

Thanks to a strong funding profile, the LTA is poised to grow its role in funding science aggressively focused on clinical trials. That said, the LTA has a unique ability to spur and inspire collaboration and participation from scientists who are already working in parallel or have compatible LAM-related work. This has allowed LTA to fund efficiently, often inspiring scientists to seek their own funding sources as fuel for pilot work. For example, there are major projects occurring in some of the world's leading institutions and labs as a result of the LTA's facilitation and partnership. These include pilot programs at the Karolinska Institute and the University of Helsinki, as well as research projects with the National Institutes of Health, Children's Hospital Boston, Brigham and Women's Hospital, MIT, Johns Hopkins University and other institutions.

The LTA's Role in Driving Treatment

The LTA has been a driver in creating the Brigham and Women's Hospital LAM Center for Research and Clinical Care. Based on investment and highest-level commitment, the Center is poised to become the foremost dedicated LAM center of its kind connected to strategically selected clinical partners across the United States and throughout the world.