Recently, the first attempt in the United States to use the gene editing tool CRISPR to combat cancer appears to have gone well, according to the initial results of a small human trial to determine safety for the approach.
Gene editing is a way to permanently change DNA in order to potentially cure a disease by attacking the root causes. CRISPR is a tool that can cut DNA at a specific spot, allowing genes to be removed or replaced or new genes to be inserted. CRISPR and other similar gene editing tools have long been used in the lab and are finally, after many years, starting to reach human trials for cancer and other diseases.
The approach involved doctors harvesting immune T cells from three cancer patients’ bloodstreams and modifying those cells with CRISPR to make them better able to detect and destroy cancer. Two of the patients have multiple myeloma, and the third has a sarcoma. Essentially, this therapy uses the body’s own immune cells to fight the disease rather than going with the traditional route of using drugs to disrupt the growth and spread of cancer.
This method also has the advantage of having minimal and manageable side effects, unlike traditional drugs, which can often have multiple off-target effects that can often be unpleasant or dangerous in their own right.
The treatment used CRISPR gene editing to delete three harmful genes that are believed to impair the ability of the T cells to spot and attack cancer, and it added a new feature to the cells to help them become more effective.
Two to three months down the line, one patient’s condition has continued to deteriorate, but the second patient has stabilized. The outcome of the third patient given this treatment remains to be seen, as the treatment was too recent to reach a conclusion. While the trial was very small, it is a demonstration that gene editing in humans can be done safely if appropriate steps are taken.
So far, the modified T cells have survived and are increasing in number, as anticipated, to form a cancer-fighting army, essentially a living drug. Given that this is a new approach to cancer treatment, it is as yet unclear if or when any anti-cancer effects might be seen, so the researchers will be following this initial data with further testing.
The researchers plan to enroll an additional 15 patients to further assess the safety of the therapy as part of this phase 1 clinical trial. If this technique is shown to be safe in these additional patients, it is highly likely that the technique will move to phase 2 testing, where efficacy of the approach will be the focus.
This interesting development is somewhat behind the times, given that Chinese scientists previously tested this approach in cancer patients a while ago. It has taken over two years for the regulatory system in the United States to approve testing, which highlights the urgent need for reform in the approval system lest the U.S. fall further behind in developing new medical innovations and therapies.
That said, the arrival of CRISPR gene editing approaches in humans in the U.S. is very welcome, as they have the potential to be an effective solution to cancer. Not only that, this trial may also open the door for CRISPR therapies against other age-related diseases as well.