China Takes Lead in CRISPR Race, Will Test on a Human for the First Time

November 16, 2016
By Alex Keown, BioSpace.com Breaking News Staff

BEIJING – China has taken the lead in CRISPR research, as an oncology team in that country became the first to inject cells that contained edited genes using CRISPR-Cas9 technology, Nature reported Monday.

At the end of October, an oncology team at Sichuan University in Chengdu dosed a patient with an aggressive form of lung cancer with the edited genes. The Chinese team beat U.S. companies to first human clinical trials using the cutting-edge technology that could become a game changer in oncology treatments.

According to Nature, the Chinese team, helmed by oncologist Lu You, removed immune cells from the lung cancer patient’s blood and disabled a gene using the CRISPR-Cas9 technology. The gene codes for the PD-1 protein, which functions as an immune checkpoint. The cells were edited and re-injected back into the patient without the PD-1 protein in hopes that the cells will attack and destroy the cancer.

Currently details of the patient and early data on his progress have not been released. Lu told Nature that the procedure went well, but did not want to provide too much information to preserve patient confidentiality. Lu did say his team has plans to begin testing nine other patients. The patients will be monitored for six months “to determine whether the injections are causing serious adverse effects,” Nature reported. Additionally, the oncology team in China will observe the patients to gather efficacy data, according to the report.

"CRISPR" refers to Clustered Regularly Interspaced Short Palindromic Repeats that occur in the genome of certain bacteria, from which the system was discovered. Cas9 is a CRISPR-associated endonuclease (an enzyme) known to act as the "molecular scissors" that cut and edit, or correct, disease-associated DNA in a cell. Gene therapy essentially transforms cells inside a patient to harness their immune system to fight an invading disease on its own. CRISPR-Cas9 is considered revolutionary technology, and as such is likely, at some level, to be used by many companies and institutions.

The technology was discovered by UC Berkeley professor Jennifer Doudna and Emmanuelle Charpentier. Doudna went on to found Caribou Biosciences, which later formed Intellia Therapeutics. Charpentier sold her part of the rights of the CRISPR-Cas9 platform to CRISPR Therapeutics, a company making big moves in the gene therapy field.

While it is still early to determine the efficacy of technology on the Chinese patient, Nature said a group at Peking University in Beijing has hopes to start CRISPR-Cas9 human trials in early 2017 targeting bladder, prostate and renal-cell cancers. Although, Nature said those trials have not yet received funding or approval.

The first CRISPR trial in the U.S. is slated for next year, backed by The Parker Institute for Cancer Immunotherapy. The gene editing technology will be used to alter a patient’s blood cells, particularly the T cells, in order to kill the cancer cells. T cells are the soldiers of the body’s immune system. The proposed use of this particular trial would be to combat myeloma, melanoma, and sarcomas. The trial is likely to be small and test for safety, as opposed to efficacy.