Project title: Evaluating a novel protein methyltransferase inhibitor for poor-prognosis rhabdomyosarcoma therapy
Lead investigator: Dr Karim Malik, University of Bristol
Funded by CCLG and CCLG Special Named Funds including Team Jake, The Jenni Clarke Fund, Angus's Door and Ollie's Star
Awarded January 2019
Award: £99,956.00
Rhabdomyosarcoma (RMS) represents a set of malignancies within the broader group of soft tissue sarcomas. It is the most common soft tissue sarcoma in children and young people. Although the majority of RMS patients have a good response to multimodal therapies currently used in the clinic, they can still suffer from quite severe side-effects. Regrettably, there is another form of RMS known as fusion-positive alveolar RMS, for which patient survival rates are very poor. Together this underlines the need for development of new potent and specific drugs to improve the standard of care for RMS patients.
We are currently assessing drugs which inhibit a protein known as PRMT5 for therapy of neuroblastoma, another childhood cancer with poor prognosis. We believe that this protein is also involved in RMS, and herein we present strong supportive evidence for this hypothesis. Most importantly, we show that drugs inhibiting PRMT5 are efficient in suppressing the growth and killing RMS cells. This project will further establish PRMT5 inhibition as an effective alternative/addition to current therapies. Unfortunately, the pharmaceutical industry does not always prioritise development of drugs for childhood cancers, but in the case of PRMT5 inhibitors, large pharmaceutical companies are invested in developing clinically utilisable PRMT5 inhibitors, including GlaxoSmithKline. In fact the lead compound we will evaluate originates from GlaxoSmithKline, who we are collaborating with on the neuroblastoma project.
In summary, we believe that our studies have the potential to translate to real therapeutic value for rhabdomyosarcoma patients in the near future.
Project updates
Download the PDF here.