Cancer research in the past decade alone has led to some pretty incredible discoveries, and immunotherapy is at the forefront of them. Immunotherapy is the process of harvesting the body’s immune system to recognize and attack cancer cells. Now, scientists at the University of Pennsylvania are testing out a gene editing tool called CRISPR in the fight against cancer. If all goes according to plan, researchers will recruit a number of participants for a very small trial that tests out the therapy. But for now — the testing started with just two.
UPenn confirmed last month that they treated two people with cancer using CRISPR combined with another innovative cancer treatment called CAR-T therapy. CAR-T therapy involves removing T-cells from the body and editing those cells outside of the body to recognize and attack cancerous cells — the cells are then put back into the body and sent to battle. There’s been no word yet on whether UPenn’s new approach, using CRISPR and CAR-T together, has benefitted either patient — but the potential here is really exciting.
Read MoreThe Technique
Even though the innovation here is the introduction of CRISPR into the cancer fight, CAR-T therapy is the most important part of this approach. CAR-T has made major headway when it comes to treating blood cancers like non-Hodgkin’s lymphoma and leukemia — and doctors and researchers are really excited about the future of this technique. The first type of CAR-T therapy to get approval from the Food and Drug Administration (FDA) — Kymriah by Novartis — was used to treat certain blood cancers that were not responding to other treatments. Kymriah got the official FDA OK in 2017. When discussing a blood cancer that affects plasma cells called multiple myeloma with SurvivorNet, Dr. Nina Shah, a hematologist at UCSF Medical Center, said that researchers really hope — with all the clinical trials currently going on — that these treatments will soon get full approval from the FDA.“[T cells] are the cells that usually are responsible for killing viruses and some bacteria, but what they’re also responsible for is killing tumors,” Dr. Shah said. “Unfortunately for people with cancer — any type — a lot of times their T-cells just don’t function well. So we have to change how the T-cells work and reprogram them with new genes, so that those T-cells are able to find myeloma cells specifically and attack those myeloma cells. These T-cells can actually kill myeloma, so much so that people who may have had their bone marrow packed with myeloma actually are able to see the bone marrow clean.”
The Trial
In UPenn’s new trial, the two participants have two different types of cancer — one has multiple myeloma and the other has sarcoma, which affects connective tissues. Both relapsed following standard treatment. In preparation for the experimental treatment, both patients had their natural white blood cells depleted using chemotherapy — making way for the edited T-cells to take their place. According to the trial’s documents, safety is the most important factor here. Researchers are monitoring for signs of toxicity after the altered T-cell infusion.
Eventually, the UPenn team hopes to recruit 18 people to participate in the CRISPR trial. It’s a small sample size, but again, safety is key here. The therapy may very well not work, but it will open a whole lot of doors if it does.
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