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The Foundation has awarded a grant of $130,000 to the Paediatric Brain Tumour Consortium for a Phase 1 Clinical Trial of Panobinostat in Children with Diffuse Intrinsic Pontine Glioma.

The Foundation made a final stage grant of $130,000 for a Phase 1 Clinical trial of Panobinostat, a deacetylase inhibitor that has shown anti-tumour activity in pre-clinical models and cancer patients.

Many clinical trials over the past three decades have explored the use of various chemotherapeutic agents for DIPG but none have ever shown significant efficacy. 

Pre-clinical evidence for panobinostat shows: 1) effects against DIPG cell lines to histone deacetylase inhibitors in in vitro cell culture studies and 2) a tumour inhibitory effect of panobinostat in in vivo efficacy studies performed using orthotopic xenograft models of DIPG. Panobinostat has yet to be tested against DIPG in humans.
This trial will use panobinostat to assess safety, tolerability and pharmacokinetics as well as initial observations of any clinical effect in paediatric patients with DIPG.
The trial is multi-institutional. Panobinostat will be supplied by Novartis who have given final approval.