In regenerative medicine, a major inflection point has been reached in chimeric antigen receptor T-cell (CAR-T) therapy, a personalized immune-based approach to treating various hematological cancers. CAR-T therapy involves the extraction of autologous T cells, which areare then genetically engineered to express CAR and reintroduced back into the patient, where the cells are expected to target cancer cells. The US Food and Drug Administration approved the first such CAR-T product, Novartis AG's Kymriah (tisagenlecleucel-t), on August 30, 2017, and the next therapy soon followed with the approval of Kite Pharma Inc.'s Yescarta (axicabtagene ciloleucel; axi-cel) on October 18, 2017. Being the first, Kymriah's approval clearly marks a milestone, but as David DiGiusto, PhD, executive director of stem cells and cellular therapeutics operations in pediatric transplantation and regenerative medicine at Stanford University, expressed at the September 2017 KNect365 Cell & Gene Therapy (CGT) Bioprocessing & Commercialization meeting in Boston, this is only really the dawn of discovery, and is the start of a long road in effectively and profitably marketing these types of advanced therapies. At the CGT meeting in September 2017 and at the Alliance for Regenerative Medicine (ARM) Meeting on the Mesa in San Diego in October 2017, both Novartis and Kite, as well as other stakeholders, discussed various aspects of manufacturing and commercialization that will impact their success in the CAR-T market.
Kymriah's approval is revolutionary for many reasons. According to DiGiusto, it showed that cell therapies can become viable medicines, especially...
