New Paradigms in moving toward Point-of-Care Cell Therapy Services in the Hospital
The video presenter, Robert Kruse, MD, PhD, is at Johns Hopkins and working on immunotherapy of hepatitis B virus using CAR-T cells. He discusses current CAR-T manufacturing methodologies requiring long wait times for product to be manufactured, expensive and remote GMP facilities, and other limitations on the current methods versus efficient, cost-effective in-hospital point-of-care models (or in Ziopharm's case, now "Rapid Personalized Manufacturing" (RPM) in the context of CAR-T (and not mentioned in this video but as Dr. Cooper mentioned at Jefferies yesterday, in the future a Ziopharm RPM TCR-T platform in solid tumors). He points out that many patients are not able to survive the wait for their infusion product under the current manufacturing practices.
Dr. Kruse discusses Ziopharm in particular here as an innovator in solving for this "point of care" solution. He appears to have no personal or professional connection to the company. I have transcribed his comments and provided links to the Ziopharm sections of his presentation, but in this case the whole presentation is worth watching. Dr. Kruse does a nice job of explaining the current landscape. The three YouTube links below point to each particular section noted in the screenshot.
“So, if you think about this, this is an image from Ziopharm which is trying to propose an on-site manufacturing strategy. They made the comparison: they have a model flying cells all over the country which can really take two days, occasionally more or you can model where you have a manufacturing in the hospital - patients are in the hospital at the same time receiving their products in a very short time frame.”
“Ziopharm Oncology is pursuing as I mentioned before a point-of-care-like trial and a different device that's not using the Prodigy. So, they are piloting a process where you take the apheresis product and you electroporate it. So, electroporation you can think about like zapping with electric field cells that has temporary holes in it - you can slip in DNA inside the cell. So, their protocol outline would be culturing the cells really for like one to two days after electroporation, then infusing back into the patient and they're using an IL-15 gene to drive expansion persistence of the cells from smaller starting numbers.”
“And just to explain a bit more about the Ziopharm platform, again they're making this comparison on the left side which is the “traditional” model of flying CAR-T cells into the tumor - or sorry, of flying CAR-T cells into this transportation site and then to the company, manufacturing by the company and then transportation back to the hospital; as opposed to the Ziopharm model where you have an apheresis product, then you have the modification with the gene therapy. Here, they're using a transposon called Sleeping Beauty to integrate inside cells, returning it to the patient and then the patient immediately has the CAR-T cell product for infusion.”