In 2017, The LAM Foundation hosted a landmark event in Los Angeles—the Patient Benefit Conference—bringing together patients and researchers in a first of its kind effort to identify and fund proposals for new products or services that address the mental and physical health issues that LAM patients want solved in the short term. This event represented more than just a gathering; it was a turning point in patient-driven research and collaborative scientific discovery.

 

 

What We Set Out to Accomplish

For over two decades, The LAM Foundation has worked to bridge the gap between patients and researchers, fostering a community grounded in shared goals and mutual respect. The Patient Benefit Conference was designed as an innovative step forward in this mission.

With a unique and inclusive format, the weekend focused on the following areas:

• Biomarkers and imaging for LAM
• Supplemental oxygen and LAM
• Exercise and LAM
• Fatigue
• Coping and mental wellness
• The LAM Clinic experience and patient-reported data

The goal was simple yet bold: to give patients a powerful voice in determining research priorities that could have an immediate impact on their lives.

What We Achieved Together

The conference did more than foster dialogue—it sparked action. With participation from individuals with LAM across the country, the community not only shared invaluable insight but also raised over $190,000 for Patient Benefit Research Grants.

These funds planted seeds for a host of impactful studies focused on critical areas, targeted at making a difference in the quality of LAM patients’ lives.

Here are just a few of the remarkable accomplishments made possible by the 2017 Patient Benefit Grants:

1. Single‐Cell RNA Sequencing for Identifying Differential Responses to Sirolimus Therapy in LAM
   Principal Investigator: Dr. Jane Yu, University of Cincinnati
   This project, related to the focus area of biomarkers and imaging for LAM, used single-cell RNA sequencing to identify molecular mechanisms responsible for variable responses to sirolimus treatment. The research generated compelling data that supported three successful NIH grant applications totaling approximately $8 million. According to Dr. Yu, “Our research identified novel signaling pathways and potential new molecular targets responsible for LAM progression.”
2. Impact of Menstrual Cycle-Related Variation in Lung Function on Disease Progression in LAM
   Principal Investigator: Dr. Nishant Gupta, University of Cincinnati Medical Center
   This clinical study explored how hormonal fluctuations across the menstrual cycle influence lung function and disease progression in LAM patients. The findings deepened understanding of estrogen’s role in LAM and laid the groundwork for more personalized treatment strategies for an increased quality of life.
3. Mobile Health to Increase Patient Accessibility to Exercise and Elucidate Exercise & Fatigue in LAM
   Principal Investigator: Dr. Mary Beth Brown, University of Washington
   This project, related to the focus area of exercise and fatigue in LAM, evaluated the use of mobile health technologies in monitoring a home-based exercise program called LAMFit. The study demonstrated that remote support significantly improved exercise tolerance and quality of life, making rehabilitation more accessible for LAM patients.
4. Feasibility Study of [11C]acetate PET as an Indicator of Early Response to Rapamycin in LAM Patients
   Principal Investigator: Dr. Carmen Priolo, Brigham and Women’s Hospital, Boston
   This cutting-edge imaging study showed that [11C]acetate PET scans could detect even small renal angiomyolipomas in LAM patients and monitor their response to rapamycin. Dr. Priolo stated, “Our findings could improve LAM clinical trials and, ultimately, bring us closer to personalized patient care. This pioneering, multidisciplinary study in the LAM field would not have been possible without the generosity and trust of LAM women and donors.”
5. LAM Patient Needs Assessment and Research Priorities Survey (LAM-PREP)
   Principal Investigator: Dr. Marina, New York Medical College, Valhalla
   With insights directly from the LAM community to better understand and rank research and health priorities, the early findings confirmed that while developing new treatments remains a central goal, other top concerns include:

• The role of hormones in LAM disease and treatment.
• The causes of LAM initiation and progression.
• Mental health, oxygen management, and awareness of LAM in the broader medical community.
• Access to comprehensive care and clearer understanding of symptoms.

These findings are now helping to shape The LAM Foundation’s strategic planning process and guide future research funding decisions to align with patient-identified priorities.

Looking Ahead

The success of the 2017 Patient Benefit Conference was made possible by the commitment, courage, and generosity of the LAM community. It showed the world that when patients are empowered, research becomes more impactful, more targeted, and more urgent.

The LAM Foundation remains deeply grateful to each woman who participated, contributed, and believed in the mission. This milestone conference reaffirmed a powerful truth: the path to a cure is one we walk together.

As we continue this journey, the spirit of the 2017 conference will guide us—patient voices leading the way, science responding with hope.