Research Conferences








2017 International Research Conference on TSC and LAM: Innovating Through Partnerships
June 22-24, 2017
Hyatt Regency Washington on Capitol Hill
Washington, DC

Conference Registration

Please register online. Registration prices are as follows:

Type Until May 25 May 26 and After
Professionals – Full Conference Package $545 $595
Professionals – Conference Only $395 $445
Trainees – Full Conference Package $475 $525
Trainees – Conference Only $325 $375

Conference only package: The conference only package includes all research sessions and meals.

Full conference package: The full conference package includes all of the above, plus The LAM Foundation’s Breath of Hope Gala on the evening of Saturday, June 24 and the TS Alliance’s Step Forward to Cure TSC National Walk on the Mall on the morning of Sunday, June 25.

Trainee rates: The trainee rates are reserved for graduate students and postdoctoral scholars.

Conference Agenda

Tentative conference agenda (times and speakers subject to change):

Day 1, Thursday

Welcome and Plenary Session 1 (2pm-6:30pm)

Time Presenter
2:00-2:15 TSC/LAM patient or family member
2:15-2:45 Brendan Manning, PhD, Harvard T.H. Chan School of Public Health – Targeting anabolic balance downstream of mTORC1 to treat neoplastic lesions in TSC and LAM
2:45-3:15 Maikel Peppelenbosh, PhD, Erasmus Medical Center – Kinome profiling identifies PAK2 as a novel targets in Tuberous Sclerosis Complex treatment
3:15-3:30 Rebecca Ihrie, PhD, Vanderbilt University School of Medicine – Differential mTORc1 Activity in Neural Stem Cell Subdomains Leads to Location Specific Tumor Development in Models of TSC
3:30-4:00 Kimberly Raab-Graham, PhD, Wake Forest School of Medicine – Parkinson protein 7 links aberrant calcium dynamics and glutamate receptor trafficking to a model of TSC
4:00-4:30 Break
4:30-5:00 Emily Osterweil, PhD, University of Edinburgh – mRNA and translation at synapses regulated by mTOR
5:00-5:15 Sungho Won, PhD, Seoul National University – A LAM Genome Wide Association Study (GWAS) implicates NR2F2 in LAM pathogenesis
5:15-5:30 Emmanuelle Logette, PhD, Association sclérose tubéreuse de Bourneville – The TSC burdens in France for patients and caregivers
5:30-5:45 Yun-Jung Chiang, PhD, Academia Sinica – CBAP critically regulates Akt-dependent inhibition of Rheb-GAP activity of TSC2 to promote Rheb-mTORC1 signaling axis and tumorigenesis
5:45-6:00 Jane Yu, PhD, University of Cincinnati College of Medicine – Tuberin negatively regulates the expression and function of prostaglandin receptors via Rheb
6:00-6:15 Helen Bateup, PhD, University of California, Berkeley – Second hit mutations generate cortical tuber-like cells in human neuronal models of Tuberous Sclerosis
6:15-6:30 Chris Ankney, Loyola University Chicago – TCR transgenic, gp100 reactive T cells to target lymphangioleiomyomatosis


Dinner and Keynote Speaker (7pm-9pm)

Time Presenter
7:00-7:10 Opening Remarks by Kari Luther Rosbeck and Sue Sherman
7:10-7:25 Dr. Walter Koroshetz, Director, National Institute of Neurological Disorders and Stroke
7:25-7:40 Dr. James Kiley, Director, Division of Lung Diseases, National Heart, Lung, and Blood Institute
8:00-9:00 Keynote Address by David Sabatini, MD PhD, Massachusetts Institute of Technology – impact of basic research on advances in TSC and LAM clinical care

Day 2, Friday

Plenary Session 2 and Breakouts (8:30am-12pm)

Time Presenter
8:30-8:45 TSC/LAM patient or family member
8:45-9:15 Elizabeth Thiele, MD PhD, Harvard Medical School – CBD effects on epilepsy in TSC and Lennox-Gastaut Syndrome
9:15-9:45 Vera Krymskaya, PhD MBA, University of Pennsylvania – Pulmonary Lymphangioleiomyomatosis (LAM): from Hallmarks of Monogenic Malignancy towards Mechanism-Driven Hallmark-Targeting Therapies
9:45-10:00 Andrew Tee, PhD, Cardiff University – Identifying novel mechanisms and drug targets that induce a selective cytotoxic effect in model systems of Tuberous Sclerosis
10:00-10:15 Hilaire Lam, PhD, Brigham and Women’s Hospital – miR-21 inhibits the tumor suppressor argininosuccinate synthase 1 (ASS1) to ensure aspartate availability for pyrimidine biosynthesis in mTORC1 hyperactive cells
10:15-10:45 Break
10:45-11:15 Jeanine D’Armiento, MD PhD, Columbia University – HMGA2 an absolute requirement for TSC tumorigenesis: an alternative therapy for tuberous sclerosis through an mTOR independent pathway
11:15-11:45 Darcy Krueger, MD PhD, Cincinnati Children’s Hospital Medical Center – Targeting mTOR to improve neurocognition in patients with Tuberous Sclerosis
11:45-12:00 Loren Leclezio, University of Cape Town – Multivariate data analysis identifies natural clusters of Tuberous Sclerosis Complex Associated Neuropsychiatric Disorders (TAND)


Lunch and Breakouts (1pm-2:30pm)

Time Event
12:00-1:00 Lunch provided
1:00-2:30 Breakout working groups


Plenary Session 3 (3pm-5pm)

Time Presenter
3:00-3:30 Mustafa Sahin, MD PhD, Harvard Medical School – TACERN biomarker studies
3:30-4:00 Shafali Jeste, UCLA David Geffen School of Medicine – Timing and mechanisms of atypical development in Tuberous Sclerosis Complex
4:00-4:30 Elizabeth Henske, MD, Harvard Medical School – Achilles’ Heel: Therapeutic Targeting of Metabolic Vulnerabilities in Lymphangioleiomyomatosis (LAM)
4:30-5:00 Yonghao Yu, PhD, University of Texas Southwestern Medical Center – Navigating Downstream of mTORC1: A Quantitative Proteomic Approach

Day 3, Saturday

Plenary Session 4 and Lunch (8:30am-1pm)

Time Presenter
8:30-8:45 TSC/LAM patient or family member
8:45-9:15 Ravi Jagasia, PhD, F. Hoffmann-La Roche –mTORC1 controls neurodevelopment and synaptic plasticity in a time-dependent manner in cell models of tuberous sclerosis
9:15-9:45 Martina Bebin, MD MPA, University of Alabama Birmingham –The Role of EEG as a Biomarker to Predict Impending Epilepsy in Seizure-Naïve Tuberous Sclerosis Complex Infants
9:45-10:15 Michael Gambello, MD PhD, Emory University School of Medicine – Metabolomic studies of TSC mouse model brains uncover major changes in the methylation pathway – implications for neuropathology and treatment.
10:15-10:30 You Feng, PhD, Brigham and Women’s Hospital – Targeting Secreted Lysophospholipase D/Autotaxin in Tuberous Sclerosis Complex (TSC) and Lymphangioleiomyomatosis (LAM)
10:30-10:45 Break
10:45-11:15 David Kwiatkowski, MD PhD, Harvard Medical School – Molecular studies in EPISTOP
11:15-11:45 Sergiusz Jóźwiak, MD PhD, Children’s Memorial Health Institute – EPISTOP project – data from current status
11:45-12:00 Caroline LePoole, PhD, Loyola University Chicago – Opportunities to treat TSC-deficient tumors by GD3 CAR-T cells
12:00-1:00 Lunch provided


Plenary Session 5 (1pm-3pm)

Time Presenter
1:00-1:30 Cheryl Walker, PhD, Baylor College of Medicine
1:30-2:00 John Blenis, PhD, Weill Cornell Medical College – mTORC1/S6K1: Regulation of cell metabolism
2:00-2:15 Charilaos Filippakis, PhD, Brigham and Women’s Hospital – Lysosomal regulation of cholesterol homeostasis in Tuberous Sclerosis Complex is mediated via NPC1 and LDL-R
2:15-2:30 David Neal Franz, MD, Cincinnati Children’s Hospital – Sustained seizure reduction with adjunctive everolimus for treatment-refractory seizures associated with tuberous sclerosis complex (TSC): Long-term results from the phase 3 EXIST-3 study
2:30-3:00 Break


Joint Session with LAMposium and Regional TSC Conference (3pm-5pm)

Time Event
3:00-4:30 Joint session with individuals and families affected by TSC and/or LAM covering reports from Friday’s breakout sessions and discussion of important research topics that are relevant and understandable to a non-technical audience
4:30-5:00 Conference close

Additional Events

Before the conference: Junior investigators are invited to register for the 2nd Early-Stage Tuberous Sclerosis Complex Researcher Meeting on Wednesday, June 21, organized by Angela Peron, MD; Brian Siroky, PhD; Nicola Alesi, MD; Charilaos Filippakis, PhD; and Krinio Giannikou, PhD. Space is limited, so please register early.

After the conference: All participants are invited to The LAM Foundation’s Breath of Hope Gala on the evening of Saturday, June 24 and the TS Alliance’s Step Forward to Cure TSC National Walk on the Mall on the morning of Sunday, June 25. Both events are included with the “full conference package,” but you may also register for these events separately.

Hotel Information

Conference attendees can book a Double or King room at a discounted rate of $199 per night (not including the 14.5% District of Columbia accommodations tax and any applicable hotel fees). Book online or by calling +1 (800) 233-1234 and mentioning the “International Research Conference on TSC and LAM.” To receive this discounted room rate, you must make your reservation before Thursday, June 1, 2017.


For more information, please contact Katie Smith.

Past Research Conference Program Books