AcSé

The objective of AcSé Immunotherapy is to evaluate the efficacy and toxicity of treatments with anti-PD-1 monoclonal antibodies for patients with rare diseases.

This programme is completed by the ancillary research AcSé CIBLE, whose objective is to identify biomarkers that will allow the identification of patients who will not benefit from anti-PD-1 immunotherapy

AcSé Immunothérapy

In 2017, under the aegis of the INCa ‘secured access’ (AcSé) programme, Unicancer launched AcSé Immunotherapy – twin trials aimed at evaluating anti-PD1 therapies (nivolumab and pembrolizumab) in 650 patients with specific rare cancers

Scientific coordination is provided by Dr. Aurélien Marabelle and Pr. Christophe Massard of the Drug Development Department (DITEP) at Gustave Roussy, with the support of the INCa rare cancer network groups. The French National Cancer Institute (INCa), the “Ligue Nationale contre le cancer“ (French League against cancer) and our industrial partners (Bristol-Myers Squibb and MSD France) provide treatments and financial support.

These phase II “basket” studies provide patients with specific rare metastatic or locally advanced cancers for which no other standard or experimental treatment options are available a secure access to immune checkpoints inhibitors. The study objectives are to assess the treatment efficacy and safety in each cohort and to identify predictive factors of response.

AcSé nivolumab

• Non-clear cell renal cell carcinoma
• Rare head and neck cancer
• Rare skin cancer
• Non-colorectal cancers with microsatellite instability
• Penile cancer
• Cancers with POLE mutation

• Rare sarcoma
• Rare ovarian cancer
• Primary central nervous system lymphomas
• Rare thyroid cancer
• Rare malignant neuroendocrine cancer
• Germ-cell cancer
• NK/T-cell lymphoma.

End of inclusions in December 2020.

The first results of Sarcoma and POLE cohorts have been presented at ESMO congress in 2020 (oral communications).

AcSé CIBLE : Ancillary program

This is an ancillary program which proposes to take a more pragmatic approach to the question of biomarkers for immunotherapy by concentrating on factors that may contraindicate treatment with PD-1 antagonists, permitting clinicians to identify patients for whom treatment may have an absence of effect or even a deleterious effect (e.g toxicity without efficacy, paradoxical hyperprogression, …).

A multi-parametric analysis of the clinical, biological and radiological characteristics of the AcSé patients treated by anti-PD-1 (nivolumab or pembrolizumab) will be performed in order to identify combination of factors which strongly correlate with early failure of nivolumab or pembrolizumab therapy. 200 Patients from the two AcSé trials will be pooled and analysed together (“discovery cohort”), regardless of the tumor type, in order to develop a signature or a combination of markers that is predictive of treatment failure. The reliability of the signature in predicting clinical outcome will be tested in the 300 additional patients in the AcSé programme on top of the first 200 patients (“validation set”).

This ancillary project will provide practical clinical tools to select the prescription of these expensive treatments to patients with expected benefits while preventing patients without any hope of immunotherapy efficacy to be exposed to immune related adverse events. Moreover, the results of this collaborative work will highlight new biological pathways responsible for primary resistance to anti-PD1 and identify potential novel therapeutic targets for cancer immunotherapy.