First U.S. Patients Are Treated With CRISPR as Echoes of Scandal in China Still Linger

illustration of DNA

The first clinical trials using CRISPR technology are expected to get underway in the United States, even as echoes of a scandal involving the use of CRISPR on embryos in China are still being felt across the globe.

NPR reported that the University of Pennsylvania, along with the Parker Institute for Cancer Immunotherapy and Tmunity Therapeutics, has been cleared to use CRISPR technology on two cancer patients. One of the patients had multiple myeloma and one had sarcoma. Both had relapsed after undergoing standard treatment, NPR reported. The team conducting the trial redirected autologous T-cells with CRISPR edited endogenous TCR and PD-1.

Other clinical trials using CRISPR in the U.S. are expected to follow. As NPR noted, CRISPR Therapeutics and Vertex Pharmaceuticals are using the gene editing technology to tackle blood disorders like sickle cell disease and beta thalassemia. In February, CRISPR and Vertex dosed the first beta-thalassemia patient in Germany. The first patient was treated with CTX001 in a Phase I/II clinical study of patients with transfusion-dependent beta thalassemia (TDT), the companies said in a joint statement. That dosing marked the first company-sponsored use of a CRISPR/Cas9 therapy in a clinical trial, Vertex and CRISPR said.

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A sickle cell trial using CRISPR is expected to begin later this year in the U.S., NPR said. Also, Editas Medicine, another gene-editing company, plans to initiate clinical trials using CRISPR to treat a genetic form of blindness known as Leber congenital amaurosis. As NPR notes, the Editas trial is significant as the study looks to edit the genes while still in the body. The other trials involved removal of cells from the body, editing them and then reinserting them back into the patient.

As the CRISPR path moves forward, the CRISPR baby scandal in China continues to raise some concerns regarding the practice. On Monday, BioSpace reported that another researcher from Stanford University had been brought in for questioning about the scandal involving the research conducted by He Jiankui. A researcher in Shenzhen, China, Jiankui used CRISPR-Cas9 gene editing to alter the DNA of embryos to protect them from the HIV virus. Following the announcement, researchers across the world responded that Jiankui’s experiment was unethical and “monstrous,” particularly since it is unknown how those edits could be passed down to future generations of children. Stanford researcher Stephen Quake had been in contact with Jiankui, who had worked in his lab nearly a decade ago. There were some questions about whether or not Quake had provided some kind of guidance or assistance to Jiankui. However, Quake denied this.

This morning, STAT News reported that Stanford cleared Quake and other Stanford employees who had communicated with Jiankui from any wrongdoing. The prestigious university said the researchers “were not participants in [He Jiankui’s] research regarding genome editing of human embryos for intended implantation and birth and that they had no research, financial or organizational ties to this research,” according to STAT. Additionally, Stanford said its investigation into the matter showed that Quake and others expressed concerns about the work being conducted by Jiankui and encouraged him to follow proper scientific practices.

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