CRISPER's First Human Trial

By Khanh Nugyen

The newly developed gene editing technology CRISPR-Cas9 has become more and more popular as it has become relatively cheaper and more accessible. This fast way of moving genes around and cleanly removing them had essentially revolutionized the way doctors and scientists look at and treat diseases. Scientists have used this CRISPR technique to fix mutations that cause blindness and were able to even stunt the growth of cancer cells. Simply put, this new editing technology holds promise for new treatment of virulent genetic diseases.

CRISPR is an acronym for Clustered Regularly Interspaced Short Palindromic Repeats, which are segments of DNA that contains short and repetitive base sequences. It works to protect bacteria from a repeated viral attack, and the specificity of CRISPR-based in destroying invading viruses is not just useful for bacteria but also in areas like basic research and medicine. The introduction of CRISPR will probably spark a race for the development of the best biotechnology across the world.

Editing genes in a lab is one achievable thing, actually attempting it in a human genome is a challenge that researchers and scientists are still trying to overcome. It is so difficult to get it to work in a human body because Cas9 is bulky and the journey to our genes is much more difficult than we think. Its main problem is the drug delivery.

Recently, however, scientists have proposed to bring CRISPR into our genes to battle cancer. Scientists at the University of Pennsylvania are hoping to use this technology to edit genes in a patient’s own immune cells to attempt to attack any malignant tumors at the any first signs of growth. The trial actually, however, will test the safety of CRISPR before it moves on to effectiveness so it will need to be approved by the FDA and the university’s ethics board.

Gene editing has been attempted before, but what is different this time is that CRISPR will attempt to edit three different genes at a time, which is very difficult to accomplish. Scientists can now have the power to improve other genes that lengthen the lifespan of T cells while simultaneously giving them better targeting power with CRISPR. The new trial hopes to solve the issue that surfaced in a previous cell therapy trial.

The plan will require 18 patients with different types of cancer that have stopped responding to their treatments. Scientists will use CRISPR instead of T cells and attempt to simultaneously edit three genes at a time and will continue to monitor the status of the cancer. However, some complications with using CRISPR include editing the wrong segments of DNA, creating new mutations for diseases or even other cancers, and getting this human trial approved. Since the university had some complications with gene therapy in the past with Jesse Gelsinger, a patient who died during a clinical trial, it will definitely be difficult to get this approved by the board.

If approved, this editing technology might be our cure to cancer including a lot of other genetic diseases such as cystic fibrosis and muscular dystrophy. Although the world is still waiting to see the results of this trail, CRISPR has undoubtedly become one of the most valuable tool in research.