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First US Cancer Patients Are Undergoing CRISPR In New Gene-Editing Trial


CRISPR  consists of key molecules that target specific DNA, acting as molecular scissors that "cut" and remove strands of DNA at a specific location. Alpha Tauri 3D Graphics/Shutterstock

A CRISPR clinical trial is currently underway at the University of Pennsylvania, where researchers are testing the efficacy of gene-editing technologies in treating cancer patients.

“To date, two patients with relapsed cancers – one with multiple myeloma and one with sarcoma – have been treated as part of this trial,” wrote Penn Medicine spokesperson in an email sent to IFLScience. “Findings from this research study will be shared at an appropriate time via a medical meeting presentation or peer-reviewed publication.”


CRISPR works by targeting certain genes responsible for certain functions or traits. When found, an enzyme called Cas9 binds to the DNA, “cuts” it, and shuts off that targeted gene. Beginning last fall, the team has begun the process of removing cells from the patients’ immune systems and modifying them in the lab, before introducing them back into the patients. Hopefully, those modified cells will target and destroy cancer cells, NPR first reported. No information is available about how well the procedure is working on the two patients, but researchers plan to eventually treat 18 patients with various forms of cancer by the close of the trial in 2033.

An illustration shows the use of CRISPR to enhance CAR T cell-mediated killing of tumor cells through disruption of PD-1 protein. Targeted cells are extracted from the cancer patient, edited in the lab, and reinfused back into the body in hopes they will attack cancer cells. Meletios Verras/Shutterstock

If successful, the trial is one of many that could revolutionize how diseases are prevented and treated.

Earlier this year, concerns over the use of CRISPR were raised after Chinese researcher He Jiankui used the technology to modify the DNA of twin girls in an attempt to give them resistance to the human immunodeficiency virus (HIV) before the two were born, prompting a moratorium from the research community on such practices. This contentious research differs dramatically from the clinical trial involving cancer patients in that it was conducted on human embryos and created changes in heritable traits that could be passed down for generations to come. Rather, UPenn researchers are providing a medical treatment that only modifies the DNA of the participating individuals and, to their knowledge, would not result in potentially passing down altered genes to their children.

As NPR notes, other human trials involving CRISPR are set to begin in the US, Canada, and Europe in order to test how well it can treat a variety of diseases, including two other US-based CRISPR studies designed to treat the genetic blood disorders sickle cell disease and beta thalassemia


 [H/T: NPR]

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