The goal of this international conference is to stimulate collaborative research to address unmet medical needs of those affected by tuberous sclerosis complex (TSC) and related disorders, including autism, epilepsy, lymphangioleiomyomatosis (LAM), cancer, and rare diseases with overlapping phenotypes. Because of the wide variety of symptoms associated with TSC, research into TSC will increase knowledge relevant to similar symptoms that occur in individuals without TSC. Likewise, advances in the broader fields of autism, epilepsy, and cancer can improve the understanding and, therefore, the treatment of TSC. This conference is important and timely in part because the TSC Clinical Consensus Conference held in June 2012 updated the recommendations for diagnosis, surveillance, and management of TSC. These recommendations reveal gaps in knowledge that need to be addressed by additional research. Based on previous, similar conferences, the 2013 research conference will attract >200 attendees, including approximately 20 invited speakers, 10 oral presentations selected from submitted abstracts, and 30 posters. The conference will bring together individuals who may otherwise not have an opportunity to meet. In addition to inviting speakers from outside the TSC field as noted above, from submitted abstracts the organizing committee will select 15 trainees or junior investigators to receive trave awards to offset transportation, hotel, and registration costs. On Saturday morning during the conference, the TS Alliance will host the second annual National Walk on the Mall in Washington, DC, providing a unique opportunity for researchers to engage directly with individuals affected by TSC. This conference maintains the structural and logistical features of previously successful conferences hosted by the TS Alliance while changing the topics and invited participants in important ways. To ensure that concepts presented differ greatly from previous conferences, 72% of invited speakers for 2013 were not speakers at the 2011 conference, and 44% did not attend the 2011 conference at all. To ensure new knowledge and ideas are presented, 22% of invited speakers have no or only one publication on "tuberous sclerosis" in PubMed. Most sessions include non-clinical and clinical presentations to encourage thinking and interaction along the continuum of translational science. There are no simultaneous platform sessions, so all attendees can hear and discuss all oral presentations. Breaks of 30 minutes are designed to encourage networking. During two mealtimes, panel discussions will enable interactive question-and-answer sessions with senior researchers from pharmaceutical and biotech companies of varying size on one day and with senior leaders of NCATS on another day. Working groups will discuss and develop actions to address key problems. Success of the 2013 conference will be measured by the impact of implementing recommendations of working groups and the acceleration of research through collaborative projects discussed during the conference.

Public Health Relevance

This conference will bring together basic and clinical researchers interested in tuberous sclerosis complex (TSC) and related disorders, including autism, epilepsy, lymphangioleiomyomatosis (LAM), cancer, and rare diseases with related features. This is important because research into TSC increases knowledge relevant to similar symptoms that occur in individuals without TSC, and because advances in the broader fields of autism, epilepsy, and cancer can improve the understanding and, therefore, the treatment of TSC. Through communication and collaboration of researchers working on a variety of related disorders, the conference will stimulate and accelerate basic and clinical research to improve health care and quality of life for individuals with TSC and multiple related disorders.

Agency
National Institute of Health (NIH)
Institute
National Institute of Neurological Disorders and Stroke (NINDS)
Type
Conference (R13)
Project #
1R13NS083351-01
Application #
8529364
Study Section
National Institute of Neurological Disorders and Stroke Initial Review Group (NSD)
Program Officer
Mamounas, Laura
Project Start
2013-04-15
Project End
2014-04-14
Budget Start
2013-04-15
Budget End
2014-04-14
Support Year
1
Fiscal Year
2013
Total Cost
$25,500
Indirect Cost
Name
Tuberous Sclerosis Alliance
Department
Type
DUNS #
103180691
City
Silver Spring
State
MD
Country
United States
Zip Code
20910