Who We Are and Our Mission

What is the LAM Treatment Alliance (LTA)?

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 supporters.

Mission Statement:

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.

What is Lymphangioleiomyomatosis or LAM? What is the possible disease course and prognosis of someone diagnosed with LAM?

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.

Who is affected by LAM?

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.

When was the LAM Treatment Alliance (LTA) established?

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.

Why was the LAM Treatment Alliance (LTA) created?

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 existed.

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.

How does the LAM Treatment Alliance fast-track LAM research?

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:

• Infrastructure development/facilitation of collaboration and discovery: Development and support for international LAM tissue program; LAMsight patient-researcher collaboration platform accessible to networked patients, researchers and clinicians globally; the International LAM Registry, a clinician-entered global patient data platform accessible to researchers globally; patient staging and stratification projects to learn how segments of the LAM patient population may differ and how treatments might differentially affect different women; problem-solving summits and monthly research/clinically focused seminars accessible globally via webcast and video download; community-accessible deep sequencing data about LAM tissue samples; LOH validation for any LAM sample of interest to any researcher working on LAM anywhere in the world; projects and programs focused on developing quality control best practices and validation techniques to ensure that scarce resources are not based on “garbage-in/garbage-out” phenomenon; creation of the international regulatory and networking infrastructure to support faster and smaller trials or, a trial for every patient;
• Basic science: Genomics program; deep sequencing of TSC1/2/rheb; high throughput cell capture and purification projects to enable closer characterization of LAM biology/pathophysiology and biomarkers for diagnosis and disease progression; development of improved models; leveraging stem cell technologies to figure out the cell/organ of origin in LAM;
• Validation: Use of current best-surrogate models for screening FDA-approved and other drug libraries; validation of potential single and combined therapeutic agents in best-surrogate models of LAM; validation of potential LAM biomarkers of diagnosis and disease progression (urine/blood/imaging);
• Clinical Studies and Trials: Center for LAM Research and Clinical Care as tertiary level hub, incubator and catalyst for moving all LAM research to the bedside more quickly; cultivation of pharmaceutical industry interest, research and trials; focus on innovative small trial design; again, creation of the international regulatory and networking infrastructure to support faster and smaller trials or, a trial for every patient; and sponsorship of clinical trials.
  
  

We drive these priority research streams using the following programmatic vehicles and approaches:

• Investigator-driven quarterly peer-reviewed programs including competitive $10K pilot grants; $50K/2 years grants reviewed quarterly; RFP fellowship in collaboration with the American Thoracic Society; RFP fellowship in collaboration with Harvard Medical School and affiliated institutions;
• LTA-driven projects in collaboration with fee-for-service (or CRO) partners/contract research organizations; academic labs and institutions, clinical care centers, pharmaceutical and biotech companies, partner foundations, the NIH and other governmental bodies; driving a process that matches the highest-priority projects with those partners in industry, biotech, the NIH, academic labs and clinical care institutions best equipped to carry out that research;
• Building global community expertise and interdisciplinary collaboration and strengthening expert commitment in fields relevant to treating and eradicating LAM by sponsoring seminars, brainstorming, problem-solving, state-of-the-science and prioritizing Summits for the scientific, medical, ancillary support and patient communities throughout the year and throughout the globe; recruiting leading-edge thinkers, creators, industry partners, clinicians, scientists and institutions on an ongoing basis to work on strategic gaps and research opportunities;
• Driving, managing and funding accelerated development of community resources leveraging next-generation technology, expertise within the LAM field, domain-specific expertise beyond the LAM community in order to overcome key rate-limiting barriers to accelerating the pace of LAM research;
• Raising awareness within communities: medical, scientific, and patient; rare disease; legislative; research and trial-focused regulatory institutions; general public;
• Creating and sustaining the infrastructure to make high-impact global researcher, clinician and patient collaboration possible;
• Supporting patients with LAM and those affected by it through direct patient contact and by helping to build and fund top-caliber multi-disciplinary care, management and referral networks;
• Keeping LAM science cumulative and partnering with other foundations and institutions so all funds generated in the name of research have the greatest impact.
  
  

What are the LAM Treatment Alliance’s core values?

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.