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Re: JSwy17 post# 20583

Tuesday, 10/07/2014 12:20:13 PM

Tuesday, October 07, 2014 12:20:13 PM

Post# of 827644
The cost and side effects for Car-T therapies seem somewhat daunting. Still, I wish Pfizer, Juno, Kite, Celgene, Bluebird and Novartis the best of luck in their efforts.

CAR T-cell therapy can cause several worrisome side effects, perhaps the most troublesome being cytokine-release syndrome. -- National cancer Institute -- www.cancer.gov/cancertopics/research-updates/2013/CAR-T-Cells




"While most analysts think it is too early to estimate potential revenue or price, Citigroup believes CART therapies could cost in excess of $500,000 per patient, which it notes is roughly in line with the cost of a stem cell transplant. " -- JSwy17



Both trials were in small numbers of patients: 22 children in the Novartis trial and 16 adults in the Juno trial. The patients had acute lymphoblastic leukemia—the most common childhood cancer—and had exhausted standard treatments. Both companies are now conducting larger trials.... -- JSwy17



The deaths of two patients in a Juno-backed Sloan-Kettering trial in March caused a temporary halt in the study because of worries over these immune responses. -- JSwy17



2 immediate deaths out of 16 patients treated means Juno needs to back off their potency (or dose level), find immune response controls, and/or restrict it to patients that can tolerate the treatment.

I hope they can get past "several" worrisome side effect issues. Particularly Runaway cytokine release syndrome in such a high percentage of patients. Next, they will need to get the cost down from $500,000.00 per patient -- if Citibank's estimate is correct.

Finally, it will be interesting what response they achieve in solid tumor applications.

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