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December 13, 2012

Gene therapy shows promise in eradicating some blood cancers

Out of 10 patients, only two patients have not gotten better on the T cell therapy, even though all had stopped responding to conventional treatments. Four patients have had a complete response - their leukemia was eradicated - with the longest so far lasting 28 months. In four other patients, leukemia diminished dramatically.

This is the same treatment that was described in a Nextbigfuture article yesterday. Novartis has the rights to this and is developing it.

"We believe we know why the two patients didn't respond," said Penn physician David L. Porter, a cancer and blood specialist. "If we're right, it's something we can fix."

The therapy, culminating 20 years of research, involves genetically engineering T cells - the immune system's big guns - to attack B cells, the blood component that turns malignant in chronic lymphocytic leukemia and acute lymphoblastic leukemia.

Interfering with the immune system, however, is dicey. From the start, it was clear the designer T cells had side effects that caused flulike symptoms.


The modified T cells can attack the patients B cells and set off a self-destructive chain reaction.

The T cells flood the body with signaling chemicals called cytokines, particularly interleukin 6. The interleukin, in turn, stimulated cells called macrophages that normally consume cellular debris.

The macrophages can go on a rampage and consume the patients red blood cells.

A drug tocilizumab (Actemra), a new rheumatoid arthritis drug can stop the dangerous side effects.

Doctors now know how to manage the side effects and they are determining when to provide Actemra to reverse the T cells.

SOURCE - Philly Inquirer


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