Wednesday, July 12, 2017

Precision Medicine A New Dawn: First CAR T Cell Recommended for FDA Approval

One small step for a new drug,  one giant leap for precision medicine !

A new dawn is breaking in the field of hematologic malignancies, as the first product based on chimeric antigen receptor (CAR) T cells was scrutinized today by a panel of experts and unanimously recommended for approval.

The product is tisagenlecleucel-T (previously known as CTL019; developed by Novartis), and the "yes" vote was in answer to this question: "Considering the efficacy and safety results of Study B2202, is the benefit-risk profile of tisagenlecleucel favorable for treatment of pediatric and young adult patients (age 3-25 years) with relapsed (second or later relapse) or refractory (failed to achieve remission to initial induction or reinduction chemotherapy) B cell precursor acute lymphoblastic leukemia (ALL)? "

Similar Products Not Far Behind 

This product is made individually for each patient.
Blood is collected from the patient, and then autologous T cells are separated out and genetically engineered. The process involves inserting a CAR that targets CD19, an antigen expressed on B cells and tumors derived from B cells.  These CAR T cells are then infused back into the patient, who has undergone chemotherapy, and in the body the product homes in on B-cell leukemic cells and destroys them. 
Several similar products are in late stages of development. Coming up soon for review by the FDA is Kite Pharma's axicabtagene ciloleucel (KTE-C19) for lymphoma, while further back are CAR T-cell products from Juno.

There is much rivalry between pharma companies dedicating enormous resources in the field of precision medicine. So much so that,  "I am amused by the horse-race metaphors that are used to frame the various companies developing CAR-T therapies," Dr Belldegrun wrote in a blog post. "Today is not about competition," he continued. "Today is about advancing an exciting technology that has the potential to transform cancer."

A New Dawn: First CAR T Cell Recommended for FDA Approval
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