The LAM Treatment Alliance is a 501(c)(3) non-profit organization driving an aggressively funded, high-impact
research strategy and fostering innovative patient partnerships in order to effectively treat and then eradicate
LAM in time for patients living with the Disease today. The LTA works through direct research funding, creation of
community research resources/infrastructure, forging of strategic collaborations with patients, academia, clinical
centers, the pharmaceutical industry, government agencies and other private individual and community/corporate
The LAM Treatment Alliance’s (LTA) mission is to fast-track research focused on Lymphangioleiomyomatosis (LAM) by aggressively and strategically driving, fostering and funding collaborative, high-impact research and patient partnerships dedicated to finding an effective treatment for and then eradication of LAM for women living with the disease today.
Lymphangioleiomyomatosis (lim-FAN-jee-o-LYE-o-MY-o-ma-TOE-sis) is a rare, fatal, multi-system disease affecting
women in their childbearing years. LAM causes destruction throughout the body, often in the lungs, kidneys and
lymphatics. This destruction is attributed to migration, clustering, cell signaling and cell-other cell-cell “LAM
cell” abnormalities. Over time, patients may progress to respiratory failure as cysts and nodules take over normal
lung. There is no known cure or treatment, and the mechanisms of destruction of lung and other tissues in LAM are
poorly understood. One gene defect (TSC2) has been identified in lung tissue, kidney lesions (i.e., angiomyolipomas
or AMLs) and circulating cells. Work to identify other mutations and modifier genes is now underway.
Early symptoms of LAM may include shortness of breath, collapsed lung(s), chest pain, abdominal discomfort, and frequent coughing. Over time, women with LAM may experience complications due to leaky lymphatics (chylous or pleural effusions), become less active and require supplemental oxygen full time. Not only is LAM commonly mis-diagnosed as asthma, emphysema or bronchitis, it is also under-diagnosed, as a LAM diagnosis requires a high-resolution CT scan. People with respiratory issues are not routinely scanned due to cost and other factors. There is increasing thought that otherwise healthy, non-smoking women with one or more pneumothoraces (i.e., lung collapses) should have a CT scan to rule out LAM. Progression is variable in LAM. Women with LAM can progress to respiratory failure in less than two years or more than 20 years. We cannot predict who will progress quickly very accurately at this time.
LAM affects women of all races, nations and economic backgrounds. Between 30,000 and 50,000 women worldwide are estimated to have sporadic pulmonary LAM. Approximately 250,000 women worldwide have the form of LAM linked to a genetic disorder called Tuberous Sclerosis Complex (TSC). LAM is understood to be more sex-specific than breast cancer.
Upon her diagnosis with LAM in April 2005, Amy Farber worked closely with the Cincinnati-based LAM Foundation to advance LAM research, advocacy and fundraising efforts. Through dedicated grassroots community-building efforts, she and her family raised within a few months more than $220,000 dedicated solely to research. In October 2005, Amy, her husband, Michael Nurok and established LAM investigator David Kwiatkowski, MD, PhD, launched the monthly LAM/TSC Seminar Series at Harvard Medical School (now entering its 4th year). Meanwhile, Farber and Nurok began to mobilize intellectual resources and Nobel-caliber researchers throughout the globe to build on the work of established LAM investigators to help think about how to streamline collective forward movement. By late 2005, Amy and a diverse support base had established a donor-advised fund at a community foundation in Boston so that funds raised to advance a fast-track vision would also qualify for tax deductions. The LAM Treatment Alliance was incorporated and received its federal tax-exempt status in February 2006. Since then, the LTA has maintained a rigorous focus on identifying and overcoming key obstacles holding back the field and taking advantage of next-generation opportunities and technologies at the leading edge of cancer therapeutics.
Even with a PhD in medical anthropology and having studied medicine and health care delivery from a sociological
perspective in her doctoral work and in law school, Amy was stumped by her LAM diagnosis. She had focused her own
research on the social, political and economic factors affecting patient outcomes for diseases that have cures but
where access to existing treatments is stymied by logistical and financial constraints. With LAM, Amy confronted
the challenges of ensuring not only that the best and the brightest multi-disciplinary experts in the world were
working on or thinking about finding an effective treatment for LAM, but also that the necessary infrastructure to
open up the field, overcome key obstacles, seize unique opportunities to leverage leading-edge work in other
fields, and support the collaborations of diverse experts – all required to drive the cure for a complex but
relatively rare biological problem – was in place. This wasn’t a challenge of distributing a treatment more
effectively but rather, a challenge of engineering a solution to a complex problem where no life-saving treatment
The challenges were sociological, economic, political, scientific and personal. Solutions would require thinking not only outside of the box, but also through and beyond norms and orthodoxies. Solutions would require year-round driving and ongoing management of an aggressive research portfolio tackling diverse research streams that had the potential to achieve the greatest impact. This required new types of partnerships to advance an aggressive research agenda and to actively help create the leading-edge infrastructure to support global collaboration, global access to de-identified global patient data, and cultivation of a fertile physical and virtual environment in which discovery could flourish quickly. Solutions would require the development of novel state-of-the-art tools and systems to enable research to take place more efficiently and with greater impact. Fast-tracking would require validating and building on existing knowledge in the field, focusing on bridging disciplinary and institutional silos, and learning from and improving upon best practices within and beyond the non-profit and academic sectors – in other words, re-thinking business models and business-as-usual approaches to medical research on rare diseases and innovative approaches to leveraging technology, know-how and funds in a fresh way.
With the strong support of scientists, colleagues, other patients, institutions, family and friends, Amy Farber founded the LAM Treatment Alliance to accomplish all of the above. In doing so, the Organization built upon the lessons and achievements of predecessors and mentor foundations – including the Cystic Fibrosis Foundation, the Multiple Myeloma Research Foundation, the Michael J. Fox Foundation, the ALS Therapy Development Institute, Project ALS, and the Adenoid Cystic Carcinoma Research Foundation – and embarked upon an historic case study in aggressively fast-tracking disease research that would also benefit more common serious linked diseases (e.g., breast cancer, ovarian cancer, lung cancer, prostate cancer, melanoma, diabetes).
Today the LAM Treatment Alliance is the largest LAM research organization in the world and solely focused on treating and then eradicating LAM. Our partnerships include academic institutions, clinical centers, foundations, industry, biotech, government agencies, patients and private donors worldwide. Our portfolio encompasses global research and focuses on overcoming the key scientific barriers and taking advantage of key high-impact opportunities. With the creation of its 2009 strategic scientific plan, the LTA has turned its focus to moving science into the clinic as its key priority. Staffing, infrastructure and organizational priorities and commitments being advanced at the LAM Treatment Alliance reflect this strategic thrust.
Since its start, the LAM Treatment Alliance has focused on identifying and overcoming key obstacles slowing down the Field while taking advantage of next-generation opportunities and technologies at the leading edge of cancer therapeutics. Our portfolio and top priorities span the following key areas:
Excellence, Collaboration, Access and Impact
are our core values. Our commitment is to staying lean without sacrificing the quality of the research we drive or
the integrity of our scientific peer review process. We are determined to keep science cumulative and accessible by
forging partnerships with foundations, patient groups and institutions throughout the world. We work to ensure that
all funds invested in LAM research have the greatest impact in the shortest time.
The LAM Treatment Alliance is a 501(c)(3) non-profit organization.