First cancer-fighting CRISPR study in the US has researchers optimistic

CRISPR used to treat Angelman in mice, has applications for autism.

The first cancer-fighting CRISPR trial in the U.S. that involves humans has started to show promising results, The New York Times reports. While still in the safety testing stages, it’s a step in the right direction for utilizing CRISPR to boost the immune systems of cancer patients. The treatment allows the body to fight the disease on its own without using invasive treatments.

Early results have indicated that the procedure is safe. Yet, the question remains: Does the technique actually work to help fight cancer? Three patients have received treatment so far. All three are in their 60s and have advanced cancers that defy conventional treatments like surgery, radiation, and chemotherapy.

“The good news is that all of them are alive,” Dr. Edward A. Stadtmauer said. He is the section chief of hematologic malignancies at the University of Pennsylvania Abramson Cancer Center. “The best response we’ve seen so far is stabilization of their disease,” he added. Two of the patients have a form of blood cancer called multiple myeloma. The third has a rare connective tissue cancer called sarcoma.

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Soldiers of the Immune System

To test the CRISPR method, researchers pulled T-cells from the patients’ blood. Think of T-cells as the soldiers of the immune system. The scientists then used CRISPR to edit three genes that could hinder the cells’ cancer-fighting abilities or that could cause adverse side effects.

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The research team used the conventional method of programming the gene-edited cells with a virus. The carrier then guides the T-cells to attack a protein called NY-ESO-1, which is found on cancer cells but rarely on healthy tissue. Since they didn’t want any unintended genetic consequences, the researchers scrubbed the T-cells of any CRISPR editing info. They then put the cells back into the patients’ bodies.

“Once we infused these cells, there was anywhere from a 10,000-fold to 100,000-fold increase in the amount of cells growing in the patients, which is exactly what we hoped for,” Dr. Stadtmauer said. “It’s good to have these cells hanging around doing serial surveillance for tumor,” he said. “We’re happy that up to nine months later we’re still seeing the cells.”

Moving Forward

The next step in the study is to discern whether or not the cells can stick around and also to see if they actively seek and destroy cancer cells. To do this, the team will first mix edited T-cells with cancerous ones in a petri dish to see if the immune system’s soldiers can emerge victorious before actually testing the process in the human body. Dr. Stadtmauer expects to see the results of that testing in a month.

Furthermore, later stages of the study will involve more patients. This should give researchers a better idea of whether or not their current approach is effective. If not, it’s back to the drawing board. However, Dr. Stadtmauer sounded confident that CRISPR is the way to go.

“It really just opens up a whole world of this type of manipulation of cells to be directed to whatever the imagination can think of.”