Spotlight on TS Alliance

Groundbreaking PREVeNT Clinical Trial Recruiting The TSC Clinical Research Consortium is making history with its latest clinical study. The PREVeNT trial (Preventing epilepsy using vigabatrin in infants with tuberous sclerosis complex) is the first preventative trial in the United States for any form of epilepsy and began enrollment December 2016. The PREVeNT study is a national, multi-site study funded by the National Institute for Neurological Disorders and Stroke of the National Institutes of Health.

Previous studies by the TSC Clinical Consortium identified electroencephalography, or EEG, biomarkers of abnormal brain activity in infants with TSC. These biomarkers typically are detectable within the first four to six months of the infant’s life, and predate the onset of seizures by two to three months.

There is a small window, perhaps two to three months in duration, between the first detectable signs of abnormal brain activity and the onset of seizures in infants with TSC. Those detectable signs can be discovered through EEG before any symptoms are present. This window gives us an opportunity for preventive therapeutic intervention to delay or prevent the onset of seizures.

The researchers are hoping to determine whether early intervention will have a positive effect on developmental outcomes and delay or prevent the onset of seizures.

The study will recruit 80 infants with a genetic or clinical diagnosis of TSC who are less than 6 months of age with no history of seizures or infantile spasms. The aim of the study is to determine the impact of preventive treatment with vigabatrin on the developmental outcome of children at 2 years of age. Dr. Bebin said, “Traditionally, most children with TSC don’t see a neurologist until seizures begin. We want to examine the effects of early intervention, prior to onset of seizures. If the intervention is successful, it could have a significant impact on clinical practice.”

Anyone interested in participating can email the PREVeNT study, call them at 205-975-2890, or email Jo Anne Nakagawa at the TS Alliance.

About the TS Alliance

Our Story

The challenge…the promise… and the critical role you can play: A message from TS Alliance President and CEO Kari Luther Rosbeck.

Our Mission

The TS Alliance is dedicated to finding a cure for tuberous sclerosis complex, while improving the lives of those affected.

Our story is one of determination and devotion—an unyielding determination to help bring about better treatments and a cure for tuberous sclerosis complex (TSC) and an intense devotion to all those who share this battle with us. Individuals and families. Health care professionals. Medical researchers. Friends and family. Everyone who has a stake in this fight can multiply his or her impact by linking arms and working together to bring this disease to the brink of a breakthrough.

The Tuberous Sclerosis Alliance (TS Alliance) was founded on the core belief that community is a sustaining strength in the face of difficult challenges.  In 1974 four mothers who shared the common bond of tuberous sclerosis complex came together to provide fellowship, generate awareness, pursue more knowledge and offer hope to each other. That was the birth of the Tuberous Sclerosis Alliance, and the spirit and intention of those founders permeate everything we do to this day.

Tuberous Sclerosis Alliance

Historic Timeline

1974: The first meeting of National Tuberous Sclerosis Association’s (NTSA’s) founders — Debbie Castruita, Adrianne Cohen, Susan (Diaz) McBrine and Verna Morris — is held in Southern California. Susan had previously sent a letter to Exceptional Parent magazine seeking other parents of children with TSC.

1983: Ann Landers writes a column about TSC, which triggers 1,500 phone calls and 650 letters to NTSA.

1984: NTSA celebrates 10th anniversary and funds first research grant of $5,800 to Dr. Ken Dumars, University of California, Irvine.

1986: First TSC clinic opens at Wyler’s Children’s Hospital in Chicago.

1987: TSC gene localized to chromosome 9.

1988: NTSA relocates to Washington, D.C. area. President Ronald Reagan signs resolution declaring May 8-15 as National Tuberous Sclerosis Awareness Week.

1992: Second TSC gene is linked to a region on chromosome 16. NTSA testifies for the first time before Congress about increased research funding for TSC.

1993: TSC2 gene on chromosome 16 is identified by European consortium.

1997: NTSA launches first website. TSC1 gene on chromosome 9 is identified by International Consortium.

1998: NTSA introduces formal research program and funds $886,741 in research grant awards. Historic TSC Consensus Conference held in Annapolis, MD.

1999: NTSA holds first National Family Conference in Washington, DC.

2000: NTSA adopts a new logo and begins doing business as the Tuberous Sclerosis Alliance (TS Alliance).

2001:TS Alliance obtains support from Congress to start TSC Program in Department of Defense Congressionally Directed Medical Research Program.

2002: TS Alliance partners with NIH to hold a scientific research conference, “New Perspectives in Tuberous Sclerosis Research,” to produce a comprehensive research plan for TSC.  TS Alliance awards $1 million in funding to laboratories within Harvard Medical School, Brigham and Women’s Hospital and Massachusetts General Hospital.

2005: The TS Alliance amends its Articles of Incorporation to be inclusive of people with TSC across their lifespan and continues to expand services for adults with TSC.  Organization also begins developing the TSC Natural History Database.

2008: Rapamycin (Sirolimus) is shown to be effective in preventing and treating seizures in an animal model of TSC. Publication in the New England Journal of Medicine reports the results of the single site trial for the use of rapamycin (Sirolimus) to treat renal angiomyolipomas in individuals with TSC and/or LAM.

2009: During an FDA Advisory Panel Hearing, TS Alliance constituents advocate for approval of vigabatrin (Sabril), which is eventually approved by the FDA to treat infantile spasms.

2010: FDA approves the first drug (called Afinitor) designated to specifically treat SEGAs associated with TSC. TSC Natural History Database enrollment reaches more than 1,000 participants at 15 U.S.-based TSC Clinics.

2012: TS Alliance sponsors the first TSC Clinical Consensus Conference since 1998 to update the recommendations for diagnosis, surveillance and management of TSC. The conference includes participants from 14 countries.  TS Alliance leads and participates in first world-wide TSC Global Awareness Day, held May 15.

2014: TS Alliance initiates a TSC Biosample Repository housed at Van Andel Institute in Grand Rapids, MI, to collect high-quality biosamples such as blood, DNA, and tissues linked to detailed clinical data to help researchers to understand why TSC is so different from person to person. The 2014 World TSC Conference, held in Washington, DC, attracts almost 700 participants from 44 states and 20 countries.

2015: The National Institutes of Health and TS Alliance sponsored a workshop in March entitled “Unlocking Treatments for TSC: 2015 Strategic Plan,” bringing together 82 participants to update research recommendations and priorities for the TSC field for the next 5 to 10 years.

2016: The TS Alliance and The LAM Foundation jointly hosted Regional Conferences in Nashville, Seattle and Cleveland. The first-ever preventative epilepsy clinical trial in the United States launched, and the TS Alliance introduced the TSC Preclinical Consortium with members from industry and academia to test the efficacy of candidate therapeutic drugs and advance the best to the clinical stage. The TS Alliance announced new global partnerships with TS Canada ST, the TS Alliance of Mexico and the TS Alliance Foundation in Thailand. In addition, the Engles Family Foundation on behalf of Engles Collaborative Research Fund made a significant $1.5 million gift to support the Biosample Repository and Preclinical Consortium.