Overview of Accomplishments:
Not a Breath Wasted
Introduction
In just over two years, the LAM Treatment Alliance (LTA) has become a leading 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; facilitating 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.
The LTA Summits, Seminar Series, and “As needed” Meetings
Now widely recognized in the LAM and LAM-relevant scientific and clinical communities, the LTA has sponsored seven international summits and over 20 monthly LAM/TSC research meetings to help facilitate and advance the cure. The seminar series is now in its third consecutive year and has been attended by over 170 MD and PhDs since inception with 35-85 attendees at each meeting. Additionally, recognizing the need to move quickly to meet the rapid changes in the science, the LTA convenes over 85 “As Needed” research meetings each year.
These efforts, combined with direct, large-grant funding, training support, tissue access facilitation, and LAM cell validation “services,” has enabled the LTA and its collaborators to, among other things:
- Rigorously pursue a field-wide revisiting of validation criteria for what constitutes 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 over 25 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;
- Create partnerships poised to undertake the first ever unbiased drug screen of FDA approved drugs, which will lead next steps for moving towards multiple treatment options for LAM and 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;
- Convene the first ever Industry Roundtable/ Summit focused on treating LAM (Roundtable/ Summit planning now underway)
- Rethink the “staging” system in LAM and the mechanism of lung destruction (Summit planning now underway)
In sum, the LTA continues to rigorously and quickly facilitate problem-solving that will effectively overcome some of the most difficult challenges to finding a cure. Through fostering practical interventions and funding those that would be radically accelerated or which 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 facilitated the mapping out of novel therapeutic approaches to treating and curing LAM.
LTA’s Access to Critical Science
Based in Boston, Massachusetts, the heart of one of the world’s medical meccas, the LTA has regular in-person access to scientific leaders with institutional connections globally. This access has resulted in the active SAB membership of, among others, Sten Lindahl who has been chair and a member of the Nobel Prize Committee in Sweden and who in 2009 will be Chair of the entire Nobel Assembly.
The LTA Collaborations
Thanks to a strong funding profile, LTA is poised to grow its role in funding translational science. 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 Institute of Health, Boston Children's Hospital, Brigham and Women's Hospital, MIT, Johns Hopkins and others.
The LTA's Role in Driving Treatment
The LTA has been a driver in creating the Brigham and Women's LAM Center of Excellence which, based on investment and highest level commitment, will become the foremost dedicated LAM Center of its kind connected to strategically selected clinical partners throughout the world and across the United States.
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