New Target in the War on Cancer Ready to Download to Your Computer Largest volunteer computer-based drug design project allows you to

contribute to the search for drugs for Tuberous Sclerosis and other

childhood diseases

'We're excited to have a target that anyone can download to their personal

computer that will allow them to participate in the development of drugs

for both tuberous sclerosis and cancer,' says Wolfgang Hinz, head of

Computational Chemistry at The Rothberg Institute



    GUILFORD, Conn., April 13 /PRNewswire/ -- The Rothberg Institute for
 Childhood Diseases (TRI), a non-profit research institute devoted to
 discovering and developing drugs to treat childhood diseases, today
 announced the release of a new target that may be key to both fighting
 tuberous sclerosis and cancer. Tuberous sclerosis is a genetic disorder
 that may serve as a Rosetta stone for understanding cancer. Individuals can
 download free software to their personal computers that allows them to
 contribute to the largest search ever for drugs for childhood diseases. The
 CommunityTSC project is comprised of 40,000 volunteers and their computers
 in 93 countries working to identify new drugs to fight TSC and other
 childhood disorders.
     The CommunityTSC project uses software developed at The Rothberg
 Institute. This software models potential drug targets and computationally
 tests the binding of drug candidates to these targets in order to identify
 promising potential drugs. The process is akin to searching through a
 collection of keys (drug candidates) to find the one that will fit a
 specific lock (target protein). Each user that downloads the software, gets
 one target at a time, and a set of 20 to 100 drug candidates. Using the
 idle time on their computer, the software tests one drug candidate at a
 time against the target, and sends back to central servers at The Rothberg
 Institute the drug candidate that has the best chance of working against
 the target. Results from the over 40,000 volunteers are then ranked, with
 the best candidates being selected for further evaluation. CommunityTSC's
 top candidates are studied in leading academic centers working with The
 Rothberg Institute (TRI), including Harvard, Yale, and Fox Chase Cancer
 Center.
     The introduction of the latest TSC target, Ras homolog enriched in
 brain (Rheb), is an exciting advance in TRI's dedicated efforts in fighting
 TSC. The overexpression of Rheb has been shown to result in unusual
 overgrowth of various tissues, and is believed to be central to the growth
 processes underlying tumorgenesis. The continued identification and sharing
 of target proteins associated with TSC allows the CommunityTSC project to
 most effectively leverage the massive computer resources of its dedicated
 user community in finding new drug treatment options for this
 life-threatening disease.
     Background on TSC
     Tuberous sclerosis complex (TSC) is a genetic disorder characterized by
 the presence of benign tumors, known as hamartomas, which occur in many
 tissues and organs, including the brain, eyes, kidney, heart, lungs, and
 skin. During the first few years, the severity of TSC can range from mild
 skin abnormalities to severe epilepsy, mental retardation, autism, or
 attention deficit-hyperactivity disorder. In recent years significant
 advances in the understanding of the underlying cause of TSC has been made.
 In particular, the mutable genes (TSC1 and TSC2) responsible for the
 condition were identified, and the role of their respective gene products
 (harmatin and tuberin) explored. Interestingly, the animal models first
 used to formulate our modern understanding of cancer (Knudson's two hit
 hypothesis) were later identified as a mutation in one of the two TSC
 genes. The same genes that lead to TSC in children. In addition, the first
 description of autism was in a TSC patient. Hamartomas are lumps of
 disorganized, but differentiated, cells, which in the case of TSC, can but
 rarely progress to malignancy. Three common and life- threatening
 manifestations of the disease are renal angiomyolypomas (AMLs), which can
 lead to kidney failure; lymphangioleiomyomatosis (LAM), a growth that
 occurs only in women and can require a lung transplant; and subependymal
 giant cell astrocytomas (SEGAs), growths that can require brain surgery.
     Background on CommunityTSC
     The CommunityTSC project uses TSC-relevant proteins identified by TRI
 and sponsored collaborators at Harvard Medical School, Yale Medical School,
 Fox Chase Cancer Center, and other leading institutions as therapeutic
 targets for computational screening. The targets are screened against all
 commercially available drug-like chemical entities (an estimated 2.5
 million potential therapeutics) to prioritize the compounds to be tested in
 the laboratory both at TRI and collaborating academic institutions
 worldwide. To date, six targets have been identified and are currently
 screened by a user-community in excess of 40,000 members, in 93 countries.
     About the Rothberg Institute for Childhood Diseases
     The Rothberg Institute for Childhood Diseases is a private, non-profit
 research institution dedicated to discovering and developing therapeutics
 for tuberous sclerosis complex (TSC) and other childhood diseases. TSC is a
 genetic disorder as well as a Rosetta stone for understanding cancer and
 causes benign tumors in the brain, eyes, heart, kidney, skin, and lungs.
 The Rothberg Institute operates at the intersection of molecular biology,
 chemistry, nanotechnology, and computer science. The Rothberg Institute
 collaborates with academic laboratories at Yale, Harvard, and the Fox Chase
 Cancer Institute through the Rothberg Award for Courage in Research. For
 more information on TSC and the Rothberg Courage Award see
 http://www.childhooddiseases.org. The Rothberg Institute is located in
 Guilford, CT. To help make drugs for TSC and other childhood cancers
 download free software at http://www.childhooddiseases.org
 
 

SOURCE The Rothberg Institute for Childhood Diseases

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