For the first time worldwide, two patients, one of whom is in Regensburg, have been cured of their severe genetic blood diseases by the "CRISPR / Cas9" gene scissors. That became known this week.
Now you can go and – typically German – emphasize the impossible in this message: The study is far from over. The results were only confirmed by doctors in Regensburg and Nashville, but not by independent experts. They were only published because the said Crispr Therapeutics company, which is developing the therapy, wanted it that way and benefited from the good news.
Even before – various – gene therapies, hereditary blood diseases of beta-thalassemia and sickle cell anemia were cured, just not with the help of the Crispr scissor gene. And is it possible to talk about a "cure" and the absence of side effects all nine months after intervention in the genome of defective, circulatory cells of patients?
These are all legitimate objections. But they miss a historic moment, a milestone in medicine, which goes hand in hand with the first successful use of Crispr technology, which is promised by researchers and doctors.
In scientific terms, terms such as breakthroughs, milestones or sensations are rarely used. Too often, the supposed shifts in cancer treatment later turn out to be therapeutic dead steps. But the feeling is that Regensburg-based physician Selim Corbacioglu can describe his patient as "cured in a moment," despite the need for restraint – even if it's unclear whether this will still be the case in ten or 20 years.
It has reached an important milestone since the "current cure" was achieved with technology first discovered in 2011, just eight years ago. This is an incredible pace of drug development, where it can sometimes take decades for a new drug to undergo a careful examination process and reach patients.
Technology is simpler, more accurate, less expensive
It is also an advancement in genome editing in such a way that the scissors of the Crispr gene can now modify human cells as desired. Although gene therapies already exist, beneficial replacement genes are found in cells. There are also clinical reports of the success of other gene scissors, "zinc fingers" or "TALEN", which, for example, have successfully treated cancer patients' immune cells with Emily Whitehead for blood cancer.
But with Crispr gene scissors, medicine is opening up a whole new dimension to genome invasion. Because technology is simpler, more accurate and less expensive than ever before. With it, the term "gene therapy" becomes literally true: it treats the patient's pathogenic genes. If you can't call it a turning point in medicine, then what?
It will no doubt take years for Crispri-based therapies to cure not only two, but many patients. But now, at this point, it can stop and simply – not at all German – be happy about medical progress and cured patients.