Description of the tasks:

The goal of WP5 is the testing of compounds from all consortium members (academic and industrial) in a limited number of approved and validated testing sites by a quality-assured methodology, including single drugs with readouts of ‘biological efficacy’. The main drug testing component on PDX will be performed in a standardized fashion exclusively at the SMEs and at two EFPIA partners (Charles River and PharmaMar) to ensure quality and comparability of the PD data. The proof-of-concept drug testing in organoids will be exclusively done at the PMC (where the organoids also will be established); that for leukemia PDX in the three academic centres Newcastle, Zuerich and PMC.

The respective disease entity groups will select a panel of SOC and investigational drugs. Three standard-of-care drugs and five selected targeted therapy will be evaluated for their anti-tumor activity in vivo. Exploratory combination studies will be performed based on the results of WP 2 and 5. Combinations may include small molecules/targeted therapies/new drugs with each other or with chemotherapy or radiotherapy (e.g. to disrupt the blood-brain-barrier).
All data will be rapidly distributed to the consortium. Response evaluation is expected to provide guidance for clinical tumor treatment, to support the identification of suitable combination partners and to uncover potential resistance mechanism through correlation with molecular data generated in WP3.