The treatment is not without severe side effects for some patients, as discovered by Juno Therapeutics. Researchers are learning more about what creates the potentially harmful "cytokine release syndrome" and how they can avoid severe side effects.
The project is based on the initial joint work done with UPENN researchers on the CD19 protein target. Challenges in the production include the actual manufacturing platform in order to bring the therapy to the largest number of patients possible. The second goal is to expand the program into blood cancers targeting other proteins. For example, multiple myeloma patients have few CD19 cells on the surface of their myeloma cells but many BCMA proteins. Novartis will also test the CAR T Cell technology in diseases like multiple myeloma with other standard myeloma therapies (proteasome inhibitors, IMiDs, checkpoint inhibitors, etc.) which is very common for myeloma treatment.
Future work will be performed on solid tumors, the grandest challenge of all. Part of the process rests on the approval speed performed by the FDA which works hard to get these types of therapies priority approval. Narasimhan says:
I would say, in general, that the FDA does an extraordinary job having worked with them for many years, including on the H1N1 pandemic, and through various cycles of innovation. But, I think they need more time and money to invest in their people, so that they are up to speed on the latest science so that they can make measured decisions.The Myeloma Crowd Research Initiative is funding CAR T Cell research going after two targets (BCMA and CS1) from the University of Wurzburg in Germany. You can help accelerate this important work by donating here: Donate to CAR T Cell Myeloma Research