Cell & gene therapies are now used to treat both rare and common diseases. Gene therapy involves the transfer of genetic material, usually within a viral vector for uptake into diseased cells. Cell therapy involves the transfer of genetically engineered cells into a patient. For oncology treatments, CAR-T, or Chimeric Antigen Receptor T Cell, is a type of cell therapy which involves the introduction of T cells that have been genetically modified to recognize specific antigens that are expressed on tumor cells.
CAR-T therapeutic development is now commonplace following recent approvals of drugs in CD19 expressing disease. These CAR-T products have been approved by the US FDA for certain types of leukemia and lymphoma, and both pharmaceutical and biotech companies are investing in this approach to treating cancer, with an estimate of more than 500 active clinical trials globally.
CAR-T clinical development and associated laboratory analysis for safety and efficacy are different to traditional small molecule and antibody therapeutic approaches, due to genetically modified cells being used. Accordingly, specific laboratory approaches are required to derive pharmacokinetic profiles utilizing innovative flow cytometry and molecular assays. For safety, immunogenicity testing is required for the measurement of humoral antibodies and cellular host response against autologous CAR-T constructs. Additionally, assays or a panel of assays covering cytokines, pro-inflammatory markers and chemokines are required to safety events due to cytokine storms.
As a laboratory services organization supporting CAR-T clinical development programs, we are able to provide Flow Cytometry, Cytokine, Genomic, BioAnalytical & Immunohistochemistry (IHC) testing for different intended uses including Expansion/Persistence (Pseudo PK), Efficacy, Prediction, Resistance, Safety, and Proof of Mechanism/Pharmacodynamics