https://globalgenes.org/raresymposium/ #GGPennRareSymposium #PennMedMDBR2016

Organizational Growth:

Building Community & Longevity

Sue Sherman, MHA

Executive Director, LAM Foundation

Proudly supported by:

Organizational Growth:Three Case Studies in Building

Community & Longevity

Sue Sherman, MHA

Executive Director, The LAM Foundation

Rare Patient Advocacy Symposium

May 6, 2016

“The future is not something we enter. It is something we create.”

~ Leonard I. Sweet

1995 2016

May 2015: FDA Approval of Rapamune for treatment of LAM

NCT00414648

20 Years of Effort

The LAM Foundation Model of Success

1. Continuously engaging the hearts and trust of LAM patients.

2. Research is the core of the mission.

3. Access to consistent and passionate scientific leadership with a goal of nurturing and distributing expertise.

4. Joint research and patient donferences.

5. Collaboration, innovation and gratitude.

1. LAM Biomarker Innovation Summit2. LAM Clinic and Research Network3. Annual LAM Research Conference &

LAMposium

Three Case Studies

LAM Biomarker Innovation Summit

• 2 Day Scientific Meeting

• 50 Scientists; diverse expertise

• Patient Survey

• Rapid presentations followed by discussion groups

• Major donor plus LAM community funding: match/mail/social media

• 6 BIG Grants awarded (Biomarker Innovation Grants: $185,000

LBIS: November 2014

BIG: Biomarker Innovation Grant Awards

Brian Bartholmai,

MD

Mayo Clinic -

Rochester

Elizabeth Henske, MD

Brigham & Women’s

Hospital

Simon Johnson, MD

Nottingham University

BIG: Biomarker Innovation Grant Awards

David Kwiatkowski, MD,

PhD

Brigham & Women’s

Hospital

Raymond Yeung, MD

University of WashingtonCarmen Priolo, MD, PhD

Harvard Medical School

Access to Clinical Expertise and Research

Network

U.S. LAM Clinics

LAM patientLAM Clinic

Data center

AustraliaBrazilCanadaChinaCroatia

FranceIsraelJapanMexicoRussia

South KoreaSpainSwitzerlandThe NetherlandsUnited Kingdom

InternationalLAM Clinics

New Disease CommunitiesLAM 3280Alpha 1 AT 2054Sjogren’s cystic 1281Langerhan’s 749Alveolar proteinosis 706Birt-Hogg-Dube 434Lymphangiomatosis 136PAM 49PCH 25PVOD 53LCDD 22

NCATS Rare Lung Disease Consortium (RLDC)

Hermansky Pudlak Syndrome 50Dif. idiopathic neuroendocrine cell hyperplasia 83Castleman’s Disease 176Benign metastasizing leiomyoma 74Erdheim-Chester 34IgG4 Lung Disease 333Cryptic cause cystic lung disease 390Plastic bronchitis 21Yellow nail syndrome 30Nontuberculous mycobacteria 6342Pleuropulmonary fibroelastosis 42

Joint Research and Patient Conferences

2016 Rare Lung Disease Research Conference & LAMposium

Cincinnati Marriott RiverCenterCovington, KY

September 22-25, 2016

• 1:1 Patient to Scientist-Clinician Ratio

• 118 LAM patients; 120 Friends/Family; 108 Scientists

• Remembrance Room

• $217K raised for research at Fund A Cure

• 59,667,276 Media impressions

• Scientific Presentations, Awards and Posters

• Research Room

• Board Meetings, Video production

2015 LAMposium Chicago

LAM Liaison Network

• 35 Volunteers – all women with LAM• 21 Regions; Quarterly calls; LAMposium scholarship• 2015: 31 Meetings; 17 LAM Clinics; 500 educated• Support for newly diagnosed and families

MAP here

Regional Map

LAM/TSC Joint Conferences

“Never doubt that a small group of thoughtful, committed citizens can

change the world; indeed, it's the only thing that ever has.”

~Margaret Mead