Brussels, December 3, 2019 – Highly specific detection and destruction of cancer cells even in patients who do not respond to established therapies: that's what CAR T cell therapy promises. The first clinical therapeutic successes of this immunotherapy, which has been approved for certain types of leukemia in the US since 2017 and in Europe since 2018, give cause for hope. But regulatory hurdles and significant challenges regarding their reimbursement aggravate the advancement of new CAR T therapies in EU.
At the invitation of BIO.NRW and Miltenyi Biotec,, scientists, physicians, healthcare economics experts, and members of European regulatory bodies met in Brussels to discuss the latest research results and strategies to promote the advancement of CAR T therapies at the Advanced Therapy Medicinal Products (“ATMP”) Workshop. The event took place under the patronage of the Ministry of Economic Affairs, Innovation, Digitalization, and Energy of the German Federal State of North Rhine Westphalia (NRW).
In CAR T cell therapy, the patient’s own T-cells - white blood cells that are part of the immune system - are genetically engineered with receptors that allow detection of tumor cells via surface markers. These receptors are called CARs, "chimeric antigen receptors", explains Prof. Dr. med. Claudia Rössig from the University Hospital Münster, Germany. She has already treated children suffering from cancer with CAR T cell therapies and is convinced of the potential of immunotherapy. “Our task now is to identify antigens and develop strategies that allow to successfully apply CAR T cell therapy also to solid tumors.”
Personalized therapies require automated manufacture
But making personalized therapies available to patients not only imposes high requirements on their development it also depends on complex manufacturing and logistics.
“Clinical grade manufacturing of gene-modified T-cell therapies remains a challenge. Therapies that are approved to date require very large clean room facilities, multiple sophisticated biotechnological procedures and complex logistics to get a patient’s T- cells from the clinic to the manufacturing site and back”, explains Dr. Andrew Kaiser of Miltenyi Biotec who is the coordinator of the CARAT consortium. “Right now we see that capacities remain very limited and costs high, and the procedure is very difficult to spread to less specialized, local clinical centres. Hence, the number of patients that can be treated is limited. To address that we need automated, safe and effective solutions, that can be applied also at the point of care.”
One of the objectives of the CARAT initiative is to develop a more efficient and safer technology platform for advanced cellular therapies, namely to manufacture CAR T cell products for personalized treatment of cancer patients.
ATMP development requires adequate reimbursement
Not only the manufacture of cellular products requires new concepts, the technologies are new to European regulatory bodies as well. Established approval procedures for common drug products need to be adapted to meet the needs of personalized cell and gene therapies.
Although the experts at the ATMP workshop agreed that immunotherapy might become part of potential future standard cancer treatment regimens, the issue of reimbursement by health insurance providers is far from being solved. “Development of novel cellular therapies is a long and expensive process”, according to Prof. Dr. Christian Dierks, Dierks+Company "We see the high price tags at which the therapies need to be sold at the moment. These prices will go down significantly, especially when these treatments will be available to many patients. However, if these treatments will not be adequately reimbursed, their advancement will be slowed down or even cease." To fuel the process of their development, novel EU funding programs will be substantial to enhance the competitiveness of Europe in the development of advanced cell and gene therapies.
"Cellular immunotherapy is a fantastic step forward”, sums up Dr. Andrew Kaiser. “Now it is time to pool expertise from different disciplines, collaborate and develop strategies to overcome challenges and make CAR T therapies an accessible standard of care in an ever growing number of indications.”
About Miltenyi Biotec
Miltenyi Biotec is a global provider of products and services that advance biomedical research and cellular therapy. Our innovative tools support research at every level, from basic research to translational research to clinical application. Used by scientists and clinicians around the world, our technologies cover techniques of sample preparation, cell isolation, cell sorting, flow cytometry, and cell culture. Our 30 years of expertise spans research areas including immunology, stem cell biology, neuroscience, and cancer. Today, Miltenyi Biotec has about 3,000 employees in 28 countries – all dedicated to helping researchers and clinicians make a greater impact on science and health.
About BIO.NRW, The Home of Biotech
BIO.NRW is a central catalyst for the sustainable development of the state’s biotech sector. It activates cooperation between business, research, investors and policy-makers. BIO.NRW also promotes the strengths and achievements of biotechnology in the state.
To support young as well as already established biotech companies, BIO.NRW offers the following core competencies: Individual matchmaking for collaborations and partners; Overview on all up-to-date R&D activities in industry and academia; Profound knowledge on financing possibilities; International promotion and marketing for NRW as biotech location; Direct contacts to decision makers.
CARAT is an EU-funded collaborative research project involving 8 partners from 4 different European countries (France, Germany, Italy & United Kingdom).
CARAT's goal is to develop an efficient and safe technology platform for advanced cellular therapies, namely to manufacture CAR T-cell products for personalized treatment of cancer patients.
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