*NEW PROJECT*: Targeting the Ref1/STAT3 axis to treat Tuberous Sclerosis

Grant holder: Dr Andrew Tee
Cardiff University 

Project status: New project

Rapamycin and Everolimus are known to reduce the size of tumours in patients affected by Tuberous Sclerosis (TS). However, not all tumours respond to the drugs and even when effective, the tumours regrow when the treatment is stopped. We need to understand what limits the effectiveness of Rapamycin and Everolimus and we also need better treatments. After 5 years of testing the tumour vulnerabilities, we believe that we now have a better treatment. We think we may be able robustly to block tumour formation when we target two proteins within the tumour called Ref-1 and STAT3. These proteins are not affected by Rapamycin and Everolimus and this might explain why current treatments are not fully effective. 

As well as promoting tumour growth, Ref-1 and STAT3 might also be involved in causing seizures in TS patients. We have a series of new drugs in clinical development that target Ref-1 and STAT3 (we are working with our partners, ApeX Therapeutics). These new drugs will be tested in this work by a Ph.D student within our international TS research team. By working closely with industry and our international collaborators our intention is to fast-track this research to the clinic. To move our recent discovery towards a treatment, we will examine how TS tumour cells respond to these new drug treatments and will compare them to existing treatments. 

This study will not only improve our current knowledge of how these new drugs work to prevent tumour growth but will also help guide future research to find an effective, better cure for patients with TS.

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