CRISPR Clinical Trial

In a monumental development for the field of gene-editing, a pharmaceutical company has applied to run the first CRISPR clinical trial. CRISPR Therapeutics hopes to begin industry-sponsored, clinical human trials with a CRISPR therapy in 2018.

Officially submitted to European regulatory authorities, the application outlines a test of CTX001, a CRISPR treatment designed for patients with sickle cell disease and β-thalassemia. In an interview with Wired, Samarth Kulkarni, CEO of CRISPR Therapeutics, said, “I think it’s a momentous occasion for us, but also for the field in general. Just three years ago we were talking about CRISPR-based treatments as sci-fi fantasy, but here we are.”

CRISPR, which stands for Clustered Regularly Interspaced Short Palindromic Repeats, is a relatively new gene-editing technique based off of a bacteria defense system. Generally speaking, it is a “programmable” system that targets and edits specific pieces of DNA. This technique can also be used as a diagnostic tool.

At first, the concept of “gene-editing” could seem like part of a dystopian sci-fi nightmare in which we have an ethically murky amount of control over our biology. But as the technology has progressed and the incredible potential of gene-editing has been further explored, CRISPR is seen much more as an innovative tool that has the potential to save lives.

This is apparent in this clinical trial, as both patients with sickle cell disease or β-thalassemia have specific genetic mutations that adversely affect a subunit of hemoglobin, which is integral to the red blood cell’s ability to carry oxygen to the body. The mutations can cause symptoms ranging from fatigue to jaundice, severe pain, and potentially death.

Advancing Gene Editing

The treatment that will be tested in these trials, CTX001, works by cutting out a gene that represses the production of fetal hemoglobin. This fetal form of hemoglobin gets switched off when a baby begins making the adult form, but since the adult form is mutated in patients with sickle cell disease or β-thalassemia, turning fetal hemoglobin back on could allow the red blood cells to carry oxygen efficiently again.

According to data collected through cell and animal experiments, as presented by the company at the American Society of Hematology meeting in Atlanta, CTX001 is highly efficient in editing these genes and so far shows no signs of affecting other genes.

However, Stuart Orkin, a hematologist-oncologist at Boston Children’s Hospital, said to Chemical & Engineering News, “It is important that they do this very carefully. Because if there is a mistake or bad effect [from CRISPR], it will have repercussions beyond a single patient.”

Orkin is right. If there are any ill-effects of this treatment, not only could it be potentially disastrous for the patient receiving treatment, it could be a major detriment to the future of CRISPR-based therapies. If this CRISPR clinical trial goes badly in any way, not only could other companies be put off from starting other clinical trials, but governing organizations might not approve future testing.

However, if this clinical trial proves to be a success, or at least does not produce any harmful complications, it could spell a long future of innovative and boundary-pushing CRISPR treatments. From antibiotic resistance to disease reversal and eradication, gene-editing could become a new staple in the medicine of tomorrow.