Proven preclinical results for CRISPR technology in the treatment of blood cancers

A proof-of-concept study carried out by researchers at the Walter and Eliza Hall Institute (Melbourne, Australia) and published recently in Cell Reports has proven that a newly developed technology could potentially be used for the direct treatment of blood cancers. The CRISPR technology, which is designed to edit genomes, can be used to target and delete specific genes essential for cancer cell survival.

Using one of the most advanced CRISPR laboratories in Australia, established at the Walter and Eliza Hall Institute by Marco Herold (Walter and Eliza Hall Institute), the research team adapted the CRISPR technology to specifically mimic and study hematologic malignancies through direct manipulation of disease-related genes.

“Using preclinical models, we were able to kill human Burkitt lymphoma cells by deleting MCL-1, a gene that has been shown to keep cancer cells alive. Our study showed that the CRISPR technology can directly kill cancer cells by targeting factors that are essential for their survival and growth. As a clinician, it is very exciting to see the prospect of new technology that could in the future provide new treatment options for cancer patients,” explained researcher Brandon Aubrey (Walter and Eliza Hall Institute and The Royal Melbourne Hospital).

The CRISPR/Cas9 system functions by locating and targeting specific genes of interest within the entire genome, either introducing mutations that render the gene nonfunctional or that result in mutated genes functioning normally again.

Although the study is the first to explicitly demonstrate that CRISPR technology can be utilized in cancer therapy, pharmaceutical companies worldwide have already invested millions in the development of CRISPR as a tool for treating genetic diseases.

“There is a lot of excitement and a significant amount of resources being invested worldwide to use CRISPR technology for treating patients. The technology can directly target any gene in the person’s genome, therefore overcoming many common drug development problems,” commented Herold.

Herold also believes that CRISPR has a range of advantages over existing tools: “CRISPR is a rapid, easy and efficient technology with the best results for genome editing. The technology dramatically shortens the time frame for fundamental research, allowing us to speed up the discoveries that could be translated to better diagnostics and treatments for the community.”

“In addition to its very exciting potential for disease treatment, we have shown that it has the potential to identify novel mutations in cancer-causing genes and genes that suppress cancer development, which will help us to identify how they initiate or accelerate the development of cancer,” Herold concluded.

Source: Walter and Eliza Hall Institute of Medical Research press release