Experimental Gene Therapy Show Success against Grim Form of Leukemia

By Makini Brice | Update Date: Mar 21, 2013 12:10 PM EDT

An experimental new treatment may prove helpful as a last resort for adults with a grim form of cancer. In a small study, the treatment helped four out of the five patients enter remission.

According to the New York Times, the treatment was performed in adults with B-cell acute lymphoblastic leukemia. The disease is grim; patients have a 40 percent chance of survival after chemotherapy and a round of stem cell therapy. Often, the disease can claim victims in just weeks.

Developed by doctors at the University of Pennsylvania, as Businessweek reports, the treatment removes T-cells from the body. These cells are important to the immune system, and are used to fight viruses and other invaders. Those T-cells were modified with a virus to attack a protein called CD19.

That protein is singled out because it is carried by B-cells, another type of cell in the immune system. Those immune cells are affected by this form of cancer. The treatment causes T-cells to attack healthy B-cells, as well as the cancerous ones, but that effect is treatable.

The resulting war can be uncomfortable for the person affected, provoking immune system responses like a fever, elevated blood pressure and a skyrocketing heart rate. However, if it works as theorized, it is able to cause people to go into remission. As the Los Angeles Times reports, that can add as many as 24 months to a person's life - which is far better than nothing.

When the phase-II trial was performed on the five patients, the results were ultimately positive. One person did not go into remission. Another patient died of a blood clot; researchers believe that death occurred because the steroids, intended to treat the immune system response to the fight between the T-cells and B-cells, may have attacked the T-cells before they had completed their jobs.

In the other patients, though, the treatment was overwhelmingly positive. One patient saw his cancer disappear in just eight days - a staggering feat. The three remaining patients had bone marrow transplants as well, which is intended to be curative.

The study was published in the journal Science Translational Medicine.

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