‘Drug repurposing’ is the process whereby researchers and clinicians look at drugs that are already proven to be safe and are approved for human use for other diseases to see if a drug shows sufficient activity against a new disease (that it is not approved for) such as EHE. This is a growing area of drug development research and is a hugely important part of any cancer research programme. Using drug repurposing it is possible to identify drugs that can provide effective treatments in a far shorter time frame than developing new or novel compounds.
Repurposing is therefore a core part of the EHE Group’s ongoing research programme and future EHE strategy. The EHE Group is currently working hard, for example, to secure an approval from the European Medicines Agency (EMA) for a label extension of sirolimus for the treatment of EHE. Expert clinicians and advocates believe the application to the EMA is justified given a growing body of evidence demonstrating that sirolimus is safe and effective for the treatment of EHE. The evidence in consideration is based on small data sets and evidence reported from clinical practice over the last ten years, initially based on the work promoted by Dr Silvia Stacchiotti and her team at INT in Milan, Italy.
The process of seeking such an approval from the EMA is being undertaken by a group comprising the EHE Rare Cancer Charity UK, The EHE Foundation, sarcoma clinical experts both in Europe and the US, and The AntiCancer Fund (ACF), a specialist not-for-profit organisation focused on drug repurposing. Hugh Leonard and Denise Robinson are both working members of this EHE repurposing group, representing the EHE global patient community. Hugh Leonard outlined the history and current status of this application:
“The ACF submitted ‘sirolimus-for-the-treatment-of-EHE’ to the new EMA repurposing pilot process in 2022. The EMA accepted the proposal and the ACF then reached out to Dr Silvia Stacchiotti and the EHE Rare Cancer Charity to bring clinical expertise and patient advocacy into the EHE repurposing group. We were of course very pleased and hugely grateful to the ACF for including us. The pilot process was established by the EMA to try and facilitate and accelerate repurposing of drugs for ultra-rare cancers. We have so far passed through two rounds of the EMA’s Scientific Advice programme. This has allowed us to gain a better understanding of what the EMA require in terms of the data they ultimately want to see. Overall, the EMA have been helpful and constructive, but the process takes a long time.”
The EMA recognise that their process is slow and in January the EHE repurposing group participated in an EMA public workshop in Amsterdam, reported earlier on our blog.
At the end of March an additional informal meeting was held with the EMA to review two new study proposals submitted by the EHE repurposing group, and to answer questions the group have with regard to the Scientific Advice being sought. Hugh Leonard said:
“While we would love the process to be faster, we are pleased that the dialogue is continuing and we hope that we can report further progress in the near future.”