International call for research proposals on rare cancer drug development
Five European anti-cancer charities joined forces to stimulate international research on the treatment of rare cancers. We are excited to announce that the ATTRACT-call, the first international call to accelerate drug development for rare cancers through cross-border clinical academic research, has funded 4 projects with a total budget of 21 million euros.
Rare cancers account for as many as 20% of new cancer cases. Yet, for most of them, there are hardly any specific, effective drugs available, leaving patients with limited or no treatment options. Main hurdle for rare cancer drug development is the small patient populations, resulting in limited interest from the industry and difficulties in setting up clinical trials with adequate statistical power. International collaborations are necessary, and require appropriate funding.
Multinational, multicenter clinical trials
Therefore, 5 European anti-cancer charities joined forces to give an impulse to the field of rare cancer research: Spanish Association Against Cancer Scientific Foundation (Spain), Anticancer Fund (Belgium), Fondation ARC (France), Kom op tegen Kanker (Belgium) and KWF Dutch Cancer Society (the Netherlands), jointly set up this call to stimulate international research on rare cancer drug development.
4 awarded projects:
- Interfant-21: International collaborative treatment protocol for infants under one year with KMT2A-rearranged acute lymphoblastic leukemia or mixed phenotype acute leukemia.
Main applicant: dr. Janine Stutterheim
Institute: Princess Maxima Center for pediatric oncology (The Netherlands)
Infants under 1 year with newly diagnosed KMT2A-rearranged acute lymphoblastic leukemia or mixed phenotype acute leukemia are treated in this open-label, non-randomized, phase 3 clinical trial with blinatumomab. Blinatumomab is already approved for older children with relapse CD19 positive ALL. The results of the Interfant-21 study are compared with the historical results of the Interfant06 protocol.
- CARTALLEU: Phase 2 clinical trial to evaluate the efficacy and safety of varnimcabtagene autoleucel for relapsed/refractory adult acute lymphoblastic leukaemia.
Main applicant: dr. Julio Delgado
Institute: Oncoimmunotherapy, FRCB-IDIBAPS/HCB, Barcelona (Spain)
Patients with relapsed/refractory adult acute lymphoblastic leukaemia (R/R ALL) are treated in this phase 2 single-arm trial with a Chimeric antigen receptor (CAR) T cell product, varnimcabtagene autoleucel (var-cel). Var-cel has been developed in academia and approved in Spain for the treatment of patients older than 25 years with R/R ALL, under the Hospital Exemption Clause. This confirmatory EU-based clinical trial in academic centres from 5 different EU countries is necessary for centralised approval via the European Medicines Agency (EMA).
- FOSTER-CabOs: European randomised first line phase-3 trial, evaluating Cabozantinib against placebo as maintenance treatment after the end of standard treatment, in newly diagnosed osteosarcoma patients.
Main applicant: dr. Nathalie Gaspar
Institute: Institut Gustave Roussy (IGR). Dpt of Oncology for child and adolescent (France)
Newly diagnosed osteosarcoma patients older than 2 years with no upper age limit are treated in this randomized controlled phase 3 clinical trial with Cabozantinib against placebo as maintenance treatment after the end of standard treatment. As most of the relapses in osteosarcoma patients occur in the first year after the end of chemotherapy, introducing a maintenance treatment after the end of chemotherapy with cabozantinib might prevent relapse.
- ALL-TARGET: A precision medicine randomized trial for patients with relapsed or refractory T-cell Acute Lymphoblastic Leukemia based on a functional approach.
Main applicant: prof. dr. Philippe Rousselot
Institute: Centre Hospitalier de Versailles, Hematology (France)
The aim of the ALL-TARGET project is to validate the added-value of a precision medicine strategy for patients with relapsed or refractory T-cell acute lymphoblastic leukemia (T-ALL) by running a randomized prospective pragmatic trial in 6 European countries. T-ALL patients (older than 15 years) are treated in this phase 2 clinical trial with a combination of drugs versus standard of care. The targeted therapeutic options (TTOs) are selected by using a functional companion drug testing assay. By using combinations of venetoclax plus tofacitinib, venetoclax plus temsirolimus and erwinase and venetoclax plus 5-azacytidine, the three most frequently altered signaling pathways in T-ALL are targeted.