Open Trial for Brain Cancer Treatment

Glioblastoma stem cell transplant may offer protection

/ Author:  / Reviewed by: Joseph V. Madia, MD

(RxWiki News) Glioblastoma is a very aggressive grade of brain cancer, and attempts at treatments so far have met with limited success. But as researchers proved, sometimes you have to cheat to win.

Treatment with chemotherapy strong enough to make significant progress against the cancer sometimes weakens the patient too much. With a little genetic engineering, researchers may have found a way around that.

"Ask your doctor about local clinical trials available to you."

Similar to storing some of your blood a few weeks before your surgery, doctors in one small cancer trial took samples of the stem cells in the bones of patients, where blood and immune cells are produced, and enhanced them.

While similar ideas have been tried before, this time doctors enhanced the stem cells to be extra resistant to chemotherapy.

While there were only three patients in the trial, two lived nearly 22 months, double the average survival time. The third patient is still alive three years after the treatment.

Chemotherapy is based on the idea of giving a strong enough dose of a drug to kill the cancer, without killing the patient. In this version of chemotherapy, the transplanted cells are enhanced by including the antidote, giving transplanted cells a copy of a gene called P140K to protect the immune system from the drugs used in chemotherapy.

The type of glioblastoma studied in this clinical trial was a specific mutated form where the gene MGMT is mutated, and the toxic drug benzylguanine has to be included in chemotherapy.

Necessary to treat this form of glioblastoma, including the benzylguanine in the chemotherapy is the last straw for an already weakened immune system.

"This therapy is analogous to firing at both tumor cells and bone marrow cells, but giving the bone marrow cells protective shields while the tumor cells are unshielded," said Jennifer Adair, Ph.D.,

The study was published May 9, 2012 in the journal Science Translational Medicine. No conflicts of interest were disclosed by researchers.

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Review Date: 
May 9, 2012
Last Updated:
May 11, 2012