Healing gene therapy | A turning point in medicine

By Sascha Karberg
For the first time worldwide, two patients, one of them in Regensburg, have been cured of their severe genetic blood diseases using the gene scissors “CRISPR/Cas9”. This became known this week.
One can now go and – typically German – highlight the imponderables of this news: The study is far from complete. The results have only been confirmed by the treating physicians in Regensburg and Nashville, not by independent experts. They only became public because the publicly traded company Crispr Therapeutics, which is developing the therapy, wanted it that way and profited from the good news. Gene therapies – of a different kind – have already cured the hereditary blood diseases beta-thalassemia and sickle cell anemia, just not with the help of the Crispr gene scissors. And nine months after the intervention into the genetic material of the defective blood-forming cells of the patients, can one already speak of a “cure” and the absence of side effects?
These are all justified objections. But they fail to recognize the historic moment, the turning point for medicine, which will accompany the first successful use of the Crispr technique, from which researchers and doctors have high hopes.
There is a good reason why scientific circles use terms such as breakthrough, milestone or sensation sparingly. Too often, for example, supposed breakthroughs in cancer treatment later turn out to be therapeutic dead ends. But it is a sensation that Selim Corbacioglu, a physician in Regensburg, can describe his patient as “cured for the moment” despite the necessary restraint – even if it is unclear whether this will still be the case in ten or 20 years.
A major milestone has been reached because the “momentary cure” was achieved with a technology that was only discovered in the first place in 2011, just eight years ago. That’s a breathtaking pace for drug development, where it can sometimes take decades for a new compound to go through the meticulous testing processes and reach patients.
It is also a breakthrough for “genome editing” that the Crispr gene scissors can now modify human cells as desired. To be sure, gene therapies that introduce curative replacement genes into cells already exist. There have also been clinical success stories from other gene scissors, “zinc fingers” or “TALENs”, which were used, for example, to successfully make the immune cells of Emily Whitehead, a cancer patient, fit to fight her blood cancer. But the Crispr gene scissors open up a completely new dimension of genetic intervention in medicine. The technology is simpler, more precise and cheaper than ever before. It literally makes the term “gene therapy” come true: the patient’s own disease-causing genes are treated. If this cannot be called a turning point in medicine, what can?
Without question, it will be years before Crispr-based therapies cure not just two, but many patients. But now, at this moment, one may pause and simply rejoice – so not at all German – about the medical progress and with the cured patients.