By Janice Frias, Ph.D., Bio-Techne Scientist
The CAR-TCR Summit in Boston from September 5-8 could not have had a better venue than the Seaport World Trade Center. The space was the perfect size for a group like this. It was able to accommodate all the concurrent talks and expo, yet keep the attendees in still a rather intimate setting to encourage interaction. And a glance out the window at the Boston Harbor in between talks was a nice perk.
The summit was very timely in that the week prior, the Novartis CAR T therapy, Kymriah, received FDA approval to fight leukemia in children and young adults. This was a hot topic, sparking speculation about who is next to follow and how the pricing of Kymriah was determined. Novartis described the considerations that went into the pricing determination: access of the treatment to patients, the value of the treatment, and the return to investors. He also mentioned two financial assistance programs through Novartis that are already in place.
Another highlight of the Summit was hearing about the progress of the non-viral Sleeping Beauty technology. This technology is unique in that ex vivo expansion is not required. ZIOPHARM licenses Sleeping Beauty platform from MD Andersen, and is one of the main players of this approach. Dr. Partow Kabriaei from MD Andersen walked us through promising pre-clinical data on their 3rd generation of CAR-T Sleeping Beauty, showing a higher level of disease-free survival maintained with treatment compared to without. These cells are manufactured as point-of-care in less than 2 days, have a signal additional to CAR, and include a safety switch.
Finally, Nicole Gularte shared her moving experiences as a CAR T patient with gratitude for the conference attendees working hard to progress the field. She was celebrating her 1 year anniversary post treatment and was feeling better than she ever thought she would. Some of the adverse effects of standard cancer treatments had actually reversed after having the CAR T therapy in clinical trials, such as regaining her color vision. Stories from the successful patient are rarely what a bench scientist is exposed to while they are doing their research, but it is very powerful.