The Rothberg Institute For Childhood Diseases (TRI) supports initiatives and research to cure Tuberous Sclerosis Complex and improve the lives of children and their families affected by the disease. Through a network of world-class scientists and physicians, state of the art technology, and support from a generous community of volunteers, significant research has and continues to be done.

Three presentations are available to describe the research that is taking place at TRI.

Overview Of The Rothberg Institute and Goals. (MS Powerpoint) 9/22/2002

Overview of Virtual Screening with CommunityTSC. (MS Powerpoint) 9/22/2002

Overview of Cell Based Drug screens at TSC. (MS Powerpoint) 9/22/2002

Updates describing progress of the research that is taking place at TRI

Progress and Overview of Virtual Screening with CommunityTSC. (MS Powerpoint) 5/22/2003

Overview of Cell Based Drug screens at TSC. (MS Powerpoint) 5/22/2003

View the latest presentations given by TRI Staff at the Second Annual Courage Fund Meeting

Overview - Presented by John Chant. (MS Powerpoint) 10/10/2003

LAM and AML Cell Immortilization - Presented by Rachel Squillace. (MS Powerpoint) 10/10/2003

D2OL and the Docking Result Analysis System - Presented by Wolfgang Hinz. (MS Powerpoint) 10/10/2003

Drug Screens at the Rothberg Institute

TRI is conducting screens for potential drugs that will either kill cells that are mutant for TSC (we each have two copies of each gene and we will only kill cells that have two bad copies of the TSC genes, the rest of the cells that don't cause problem with people with TSC only have one bad copy), or make these cells more like normal cells(revert the phenotype).

These screens are done on automated robotic stations, testing each drug against the cell lines and assays we develop to determine if the drugs will have potential beneficial affect. These laboratory screens are also conducted with the best candidate molecules that come out of the computer based screens we are doing in the CommunityTSC project. The candidates that are promising will be further developed as potential drugs and tested in animal models of the disease prior to us working with the TS alliance and the national cancer institute to test these drugs in people with TSC.

Read more about drug screening strategies used at TRI. (PDF). We are lucky to have the corresponding author, Bert Volgelstein, one of the most respected cancer researchers in the world, and someone that has given great insight into the progression of cancer provide us with some of the tools he developed to aid us in our drug screens.

In our search for drugs to treat TSC we will be combining the most advanced molecular biology, modern robotic screening technologies (see our collaboration with Harvard Medical School and ICCB), the most sophisticated computer science (see Shakhnovich), and the oldest source of drugs; the plant kingdom.

In addition to the chemical collections or libraries of synthetic compounds we will get from commercial sources and directly screen in our cell assays, and the best drug candidates from the CommunityTSC projected (selected from the available 2 plus million chemical compounds), we are also going to be screening chemicals systematically derived from the plants in the Amazon basin.

The amazing diversity of products from living plants is well beyond anything chemists have been able to make, and have led from the worlds first drug (Ancient Egypt's discovery of the popular medication to treat heart failure digoxin from foxglove) to the most potent drugs and anti-cancer compounds known to man such as Taxol (from Taxus brevifolia the Pacific Yew), the miracle drug to treat breast and ovarian cancer.

The modern identification of drugs from plants will take into consideration the importance of conservation planning, harness technologies from Landsat satellites and high throughput extraction of minute samples to allow the identification and cataloging of 100,000's of new samples and millions of new potential natural drug candidates.

Read more about how we can work with these wonderful sources of potential products while supporting both conservation efforts and local economies.

Coming soon more on collaborations to ecologically harness the rainforests to make drugs to treat people with Tuberous Sclerosis...

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