FAQs

What is our hope for the future?

The TS Alliance envisions a world where no one has to endure the devastating effects of tuberous sclerosis complex.

What do we do?

We provide information, access to resources and a place for hope for those affected by tuberous sclerosis complex. Over the years, we have expanded our mission to improve quality of life for individuals and families affected by TSC through:

  • the stimulation and sponsorship of research;
  • the development of programs, support services and resource information; and
  • the development and implementation of public and professional education programs designed to heighten awareness of TSC.

What is our mission?

The Tuberous Sclerosis Alliance (TS Alliance) is dedicated to finding a cure for tuberous sclerosis complex (TSC) while improving the lives of those affected.

What are our guiding principles?

TS Alliance is committed to operate and to deliver its programs using the following guiding principles:

  1. all actions must reflect the highest standard of integrity, candor and common decency;
  2. all decisions will be based on the merits, a sense of urgency and enthusiasm; and
  3. all opportunities for collaboration and inclusion should be considered.

What does it mean to be a member of the TS Alliance?

The TS Alliance is a membership organization.  Anyone can elect annually to become a member by informing the TS Alliance of their desire to do so and to participate in the corporate governance of the organization (by voting for the board of directors).  This can be done by completing an online membership application, by writing the TS Alliance or calling the Development Department at (800) 225-6872.  A membership fee is not required but we do rely on the generous support of our members to assist in carrying out our important programs.

Why are there Community Alliances rather than chapters?

The TS Alliance started Community Alliances in 2001 with the goal of providing a network of local connections to better serve parents, teens and adults affected by TSC.  We chose volunteer branches rather than state-incorporated chapters because we wanted to work with the amazing strength of our grassroots community volunteers, and provide a structure to succeed without incurring unnecessary overhead costs.  We had also seen chapter systems divide rather than unite communities.

The words community and alliance mean, “joining together for a common purpose.” A community alliance is an informal, yet structured, group of empowered and caring volunteers who work closely with the TS Alliance to facilitate local connections for individuals and families affected by tuberous sclerosis complex, raise revenue, and increase awareness while supporting the mission of the organization. The Community Alliance program fosters stronger links between the TS Alliance, communities and individuals across the country.

What research should we fund?

In our quest to find a cure, funds provided by the TS Alliance since 1984 have contributed to a number of significant scientific breakthroughs.  TS Alliance-sponsored research helped support the identification of the two genes that cause TSC, developed a genetic test and initiated the first clinical trials in TSC.  Today, thanks to decades of basic research to help better understand this disease, there are currently two FDA-approved indications for TSC — one for the treatment of brain tumors and the other for the treatment of kidney tumors common in TSC.

Finding a “cure” for TSC will be a significant challenge due to the genetic nature of the disease and the fact that so many individuals with TSC have spontaneous mutations. Gene therapy is not likely to be a meaningful approach to treating TSC because the most significant manifestations of the disease, those affecting the central nervous system, are in place at birth. However, improvements in diagnosis of TSC leading to earlier diagnosis and recognition of those individuals who are at risk for epilepsy, neurodevelopmental and cognitive delays would lead to earlier treatment and therapies that may prevent the impact of the disease. The TS Alliance has committed to building a research program to accelerate the development of treatments that normalize the lives of individuals with TSC so they can lead healthy and productive lives.

In 2011, the TS Alliance initiated the Unlock the Cure campaign, a vital step toward implementing the next generation research program needed to move potential treatments along the path from the lab (pre-clinical) to the TSC community (clinical trials) more rapidly and efficiently. In 2015, the TS Alliance entered phase two of this campaign, Unlock the Cure ACT (Accelerate, Collaborate, Target) Now!

TS Alliance Research Grants

Since 1984, the TS Alliance has awarded more than $18.8 million to further basic, translational, and clinical research. The funding has supported research on the genetics of TSC, basic mechanisms of both tumor growth and central nervous system manifestations of TSC, translational research, and clinical trials as well as the development of research tools such as biomarkers, animal models, and a database of TSC1 and TSC2 genetic variants. TS Alliance-funded research continues to play a vital role by encouraging and fostering collaborations between basic and clinical researchers.

Supporting small, short-term research projects enables researchers to produce data on which to build an application for funding of a much larger project from other sources. This seed funding combined with a dedicated Government Advocacy Team advocating for Federal research funding has leveraged an additional $271 million in research grants in the past 14 years from the National Institutes of Health (NIH) and the Department of Defense’s Tuberous Sclerosis Complex Research Program (TSCRP).

Drug Screening Program

The TS Alliance established the TSC Drug Screening Program to accelerate progress in the development of potential new treatments for individuals with TSC with a focus on existing FDA-approved drugs that could be rapidly repurposed for use in TSC. This program supported the identification and pre-clinical testing of new drug therapies for TSC. In the first three years of the campaign, 14 drugs or combinations were identified with interesting activity for TSC, prompting the need for a TSC Preclinical Consortium — with input from academic, regulatory, and industry stakeholders — to prioritize candidate treatments based on head-to-head data using consistent animal models and testing procedures.

Clinical Research Network and Biomarkers

The execution of clinical studies requires extensive planning, cooperation, and collaboration to protect the safety of participants and the integrity of the valuable data. To initiate and implement a clinical study as quickly and effectively as possible, the infrastructure must be in place to support the needs of TSC clinical researchers. Clinical researchers from five TSC clinics formed the nucleus of a TSC Clinical Research Network, or consortium. The TS Alliance provided vital seed funding to support the creation of the core data center and to ensure that study coordinators were able to focus full time on TSC. In 2013, the consortium received NIH grants to conduct two clinical studies to (1) determine what early signs or tests can identify infants with TSC at highest risk of developing autism by age three, and (2) measure the ability of EEG and brain imaging to assess the risk of newly diagnosed infants with TSC to develop infantile spasms. In a 2015 interim analysis of the latter study, researchers identified an EEG abnormality that is a positive predictor for seizures in infants with TSC. Discovery of this biomarker has laid the foundation for the first ever preventative clinical trial in TSC and epilepsy.

TSC Natural History Database

Implemented in 2006, the TSC Natural History Database captures clinical data to document the impact of the disease on a person’s health over his or her lifetime. As of June 2016, 2,000 people with tuberous sclerosis complex were enrolled in the project from among 18 U.S.-based sites. The TS Alliance provides funding to participating clinics to perform data entry, monitors the integrity of the database, and makes data available to investigators to answer specific research questions and identify potential participants for clinical trials and studies. To date, six articles utilizing data from the Natural History Database have been published in scientific journals, including Molecular Psychiatry, Epilepsy Research, Ophthalmology and Epilepsia.

Preclinical Consortium

To accelerate the drug discovery process for TSC and standardize models and methods of testing, the TS Alliance engaged researchers from academia and industry in a Preclinical Consortium tasked with evaluating data on each of the available animal models of TSC and recommending the most appropriate use for each model, evaluating various experimental methods for evaluating epilepsy, cognitive, and tumor-related endpoints in animal models relevant to TSC, and evaluating existing data on various candidate drugs to generate a prioritized list of drugs for testing. These efforts ensure a consistent method for testing candidate drugs to treat TSC.  Importantly, this Consortium is built under the leadership of the TS Alliance and the TSC community with input from a variety of sources.  This approach enables us to compare candidate treatments in the best possible way, so we can make the best decisions as a community about which treatments to move forward rapidly. Additionally, this infrastructure enables the TS Alliance to contract with industry to test drugs through the Consortium that companies would be interested in moving into clinical trials if the drugs show positive results in preclinical studies.

Biosample Repository

In 2015, the TS Alliance established a TSC Biosample Repository to house biosamples such as blood, DNA, and tissues collected in a consistent manner at TSC Clinics and linked to detailed clinical data in the TSC Natural History Database. High-quality biosamples linked to clinical data will enable researchers to discover biomarkers, establish human cell lines or tissue arrays for drug testing, and search for clues to understand why TSC is so different from person to person.

By investing in the infrastructure of an integrated research platform that is systematic, highly collaborative and leverages Federal and private funding to accelerate drug discovery and uncover the mysteries of TSC, you can help us alter the course of the disease and drastically improve the quality of life of those affected by TSC with breakthroughs that include:

  • Preventing the onset of infantile spasms
  • Preventing or decreasing the development of autism
  • Preventing or eliminating tumors, angiomyolipomas, and LAM – beyond shrinkage and stabilization
  • Personalized medicine – the ideal therapy for each individual

Why do we sponsor a research conference?

Researchers funded by the TS Alliance are strongly urged to collaborate with each other. Sharing information with each other is important to us for a variety of reasons.  It helps the researchers to expand on ideas and collaborate, and we avoid funding duplicate research. Conferences also arm the TS Alliance with the most updated findings in research so we continue to nurture progress toward a cure.

Additionally, in June 2012 the TS Alliance brought together more than 60 health care professionals, each having expertise treating one or more aspects of TSC, to update consensus recommendations for the diagnosis, surveillance, and management of TSC.  The prior guidelines were based on a 1998 consensus conference, and the TSC field has made tremendous advancements in the meantime.  To ensure that the resulting recommendations would benefit from diverse perspectives and be internationally applicable, experts from 14 different countries were included. Consensus guidelines can be found here.

Why have we helped set up TSC Clinics?

Service to those affected by TSC across the lifespan is part of our mission, so it was natural for the TS Alliance to encourage healthcare providers to create TSC Clinics across the country.  The cornerstone of the program is to encourage TSC Clinics to provide full service to all affected by TSC.  TSC Clinic designation is given to a clinic after an application has been received and peer reviewed by the Clinics sub-committee, then reviewed and recommended by the Science and Medical Committee and ultimately voted on for approval by the TS Alliance Board of Directors.

Why was the name changed from National Tuberous Sclerosis Association (NTSA)?

The name was changed to the Tuberous Sclerosis Alliance to reflect the growing organization and our commitment to forming “alliances” with TSC grassroots volunteers across the country and around the world, TSC Clinics and healthcare providers, researchers and scientists, other nonprofits, and corporations.

Why are we referred to as the TS Alliance and not TSA?

The Tuberous Sclerosis Association was formed in Great Britain in 1977 when we were known as the NTSA. The organization in Great Britain is known as TSA, and not to confuse the two, we are known as the TS Alliance.

Why is the TS Alliance a California Corporation (legally known as the National Tuberous Sclerosis Association)?

The Tuberous Sclerosis Alliance was founded by four mothers in Southern California in 1974 and incorporated in the state of California on March 15, 1975 as the National Tuberous Sclerosis Association.  We remain a California corporation, the National Tuberous Sclerosis Association, doing business as the Tuberous Sclerosis Alliance.

The Tuberous Sclerosis Alliance Endowment Fund was founded after the TS Alliance had moved its headquarters just outside of Washington, DC.  It was incorporated in the state of Maryland on April 3, 1995.

What does the TS Alliance logo symbolize?

The intertwining arcs represent an abstract of the double helix or the structure of DNA. The spiral also shows the TS Alliance’s commitment to family and patient support, education and research are interdependent.

The lower arc signifies shelter and a place for people to congregate. It symbolizes the bridge from where we’ve been to where we want to go. Its structure reassures us that it will always be here, for as long as we need it. The upper arc shows arms reaching out to people, to knowledge and to progress.

What is the role of the Board of Directors?

The TS Alliance is responsible for the financial health and success of the TS Alliance. The board establishes policies and procedures to guide the organization, monitor the financial performance, develop and examine short- and long-range planning goals, and lend their professional expertise to support the mission of the TS Alliance.  Each board member (Director) is elected by the TS Alliance membership.  A Director serves a one-time renewable three-year term.   Board officers include a Chair, Vice Chair, Secretary, Treasurer and Immediate Past Chair.

What role does the TS Alliance Endowment Fund play?

The TS Alliance Endowment Fund was established as a nonprofit supporting entity of the TS Alliance.  The TS Alliance Endowment Fund was specifically chartered to receive gifts that are invested to generate an income stream that will help fulfill the mission of the TS Alliance.  The Endowment Fund ensures the TS Alliance has an ongoing source of funding to better serve families touched by TSC through research, family services and education.  The TS Alliance Endowment Fund is governed by a separate Board of Directors and annually makes an operating gift from the investment proceeds of the organization to the TS Alliance.

What is the Professional Advisory Board (PAB)?

The PAB was formed to provide clinical expertise and advice to the TS Alliance staff, Board of Directors and constituents as requested; respond to requests for assistance with specific clinical questions and/or clinical situations within the individual’s area of expertise; prepare articles for the TS Alliance publications as needed; prepare TSC Information Sheets on specific clinical issues in TSC and/or review TSC Information Sheets prepared by other PAB members; bring new clinical information, publications, conferences, etc. to the attention of the TS Alliance staff; give presentations on specific issues in TSC at TS Alliance regional and/or national conferences, as part of the Meet the Expert series, or to the TS Alliance Community Alliances nationwide; actively educate professional and lay audiences about TSC; and foster partnerships and collaborations within advisor’s community of expertise.

The members of the Professional Advisory Board serve a renewable three-year term beginning on January 1 of each year.

What is the International Scientific Advisory Board (ISAB)?

The ISAB was formed to provide scientific advice to the TS Alliance staff and Board of Directors as needed, and to serve as expert reviewers of grant applications submitted to the TS Alliance.  In addition, the ISAB members bring new scientific information, publications, conferences, etc. to the attention of the TS Alliance staff; give presentations on specific issues in TSC research at TS Alliance regional and/or national conferences, as part of the Meet the Expert series, or to the TS Alliance Community Alliances nationwide; actively educate professional and lay audiences about TSC research; and foster partnerships and collaborations within advisor’s community of expertise. The members of the International Scientific Advisory Board serve a renewable three-year term beginning on January 1 of each year.  Individuals must be nominated by a colleague for a position on the ISAB.

What is the Corporate Advisory Board (CAB)?

The is a group of dedicated professionals from a variety of industries that have come together to provide guidance, strategic support and act as advisors to the President and CEO.  The CAB will be called upon to offer assistance in the areas of business planning and operational growth, marketing and public relations, development and fundraising, infrastructure, technology and human resources.