In the news release, New Target in the War on Cancer Ready to Download to Your Computer, issued earlier today by The Rothberg Institute for Childhood Diseases over PR Newswire, we are advised by the company that the subhead, should read “‘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.” rather than “‘It’s only fitting that a distributed computer platform allows global participation to potentially prevent a global pandemic,’ says Wolfgang Hinz, head of Computational Chemistry at The Rothberg Institute” as originally issued inadvertently.
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’It’s only fitting that a distributed computer platform allows global participation to potentially prevent a global pandemic,’ 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
