New blueprint outlines a regulatory framework for novel “actively personalized” vaccines (“APVACs)

Mainz, October 17, 2013 – The Regulatory Research Group of the Association for Cancer Immunotherapy (CIMT-RRG) has published a blueprint for the development of “actively personalized” vaccines (APVACs). The proposal, published in the October issue of Nature Biotechnology, outlines how APVACs may be pursued within the existing regulatory framework of the European Union (EU).

Personalization of cancer treatment has been proposed as a solution to increase the efficacy of novel therapies to treat cancer. The availability of whole genome sequencing enables therapeutic concepts that actively use whole genome data to design tailored vaccines that target the unique genomic alterations found in an individual patient’s tumor. Such “actively personalized vaccines (APVACs)” have not yet been tested in patients. APVAC concepts that lead to unique medicines for every individual patient change the paradigm of how drugs are developed and clinically tested and thus pose a series of regulatory challenges. The CIMT Regulatory Research Group has analyzed the existing EU regulatory framework and has come to the conclusion that APVACs may be pursued within established principles. A meeting with the Innovation Task Force of the European Medicines Agency (EMA) confirmed the possible path for the development of APVACs as proposed by the CIMT Regulatory Research Group.

“Our publication shows that the specific concepts already in use for cellular therapies may be applicable for individualized therapies,” says Ulrich Kalinke, director of TWINCORE and corresponding author of the paper in Nature Biotechnology. “Even if challenges remain, the first hurdle for clinical testing of this new class of therapies is cleared.”

“This is a milestone publication for the clinical translation of the APVAC concept,” says Cedrik Britten (Ribological GmbH, Mainz), one of the authors of the paper. “Where there had previously not been any regulatory guidance for such actively personalized therapies, we have been able to describe a possible path that will facilitate human testing of individually tailored medicine.”

“I am very pleased that this was an international effort as it would not have been possible for single individuals or institutions,” says Harpreet Singh (Immatics Biotechnologies, Tübingen), also a co-author of the paper. “The results of the Regulatory Research Group pave the way for the future of personalized immunotherapies from which researchers and developers worldwide will benefit.”

Britten CM, Singh-Jasuja H, Flamion B, Hoos A, Huber C, Kallen K-J, Khleif SN, Kreiter S, Nielsen M, Rammensee H-G, Sahin U, Hinz T, Kalinke U (2013): The regulatory landscape for actively personalized cancer immunotherapies. Nature Biotechnology 31 (10), 880-882. doi 10.1038/nbt.2708.