Penn Medicine researchers have used the gene-editing technology CRISPR to treat two cancer patients in the first ever human trial with CRISPR in the United States, a University spokesperson confirmed to NPR on April 15.
The treatments were part of a clinical study at the Abramson Cancer Center that applied CRISPR to cancer immunotherapy. One of the patients is being treated for myeloma, a cancer of white blood cells, while the other is being treated for sarcoma, a cancer found in bones and soft tissue, NPR reported. Both patients had relapsed after going through standard cancer treatments.
The scientists plan to use CRISPR to modify the genes of these patients’ immune cells and retrain them to fight cancer, NPR reported. Another immunotherapy that Penn Medicine has developed in the past, CAR-T treatment, also modifies immune cells to target cancer.
CRISPR is a technique for genetic engineering developed in recent years that allows scientists to edit genes with almost exact precision. It uses a special enzyme complex derived from bacteria that can identify specific genetic sequences and replace them with new sequences. The potential ability of this technique to rewrite the human genome has caused many ethical controversies within the scientific community. In November, a scientist in China said he used the technique to modify the genes of two human embryos to make them immune to HIV.
Unlike the Chinese study, the Penn Medicine study is performed on adult non-reproductive cells, which evades the ethical concerns that CRISPR can create changes to the human genome that will be passed down to future generations. The study was initially approved in 2016, and it intends to target around 18 patients in total.
While all previously published human CRISPR studies have taken place in China and have mainly focused on cancer, there are multiple human CRISPR studies that are projected to take place in the United States, Canada, and Europe, applying the technique to diseases like genetic blood disorders and blindness.
Penn Medicine has also previously worked with CRISPR to treat lethal diseases in prenatal animals, which may also eventually translate to treatment in humans.