A one-time shot of a gene-editing treatment has shown to be remarkably effective in patients suffering from genetic angioedema, a mysterious, sometimes horrifying, inherited disease. In a phase 3 trial, the treatment decreased attack rates by 87 percent relative to placebo, and more than half of treated patients experienced no attacks for six months following.
Hereditary angioedema is characterized by the unpredictable onset of episodes of swelling (angioedema) which can be severe and occur in the face, extremities or in the abdomen or worse still in the airway. These attacks can be very painful and while that drug treatment can be life saving when the airway is affected the need to keep administering injections or infusions of other medications which either prevent or treat the episodes have not been ideal.
This new method operates differently. It involves using a gene-editing technique that permanently alters the patient’s liver cells to produce less of a protein responsible for excessive swelling. This permanent edit means that it will need only be administered once to be effective on an ongoing basis.
Patients in the study experienced a quite a bit reduced number of attacks, with several requiring no extra medication throughout the study period. For patients and caregivers who have existed for years with the shadow of the attack hanging over them, the data felt surreal. One injection replacing a lifetime of therapies is just the sort of progress which until now was confined to fiction. The physicians involved in the trial stated that these are some of the most encouraging results they have seen with this disease. The safety profile appeared to be promising as well, with no major unexpected side effects seen during the trial. The science underlying the treatment is the result of years of progress in CRISPR-based gene editing and other editing tools.
The delivery mechanisms have been perfected so that when the machinery enters the body, it is able to reach the desired cells (when it comes to this treatment, liver cells) with a very high efficiency. That a single dose might cause such a dramatic and sustained drop in attacks indicates that this technology is evolving fast. Similar technologies are in trials for a range of genetic disorders, from high cholesterol to some blood diseases. Still, more critical questions need to be addressed. Longer follow-up will be required to determine that that this will be a long-term solution, and not only a shorts-term cure of hemophilia. Larger number and more diverse patients population need to be involved.
As the mature of every new medicine will have to go through gene editing manufacturing and regulation pathway, and those operations are both complicated and costly, whether it will delay the use of the medicine in patients, is also worthy of consideration. Pricing and access will also be need to consider if its approved. Even taking all of those reservations into account, the findings represent a genuine milestone.
Hereditary angioedema has been an affliction of perpetual alert for so long. Patients have been conditioned to look for an early warning of an attack, to carry medications with them at all times, to plan their lives around a potential attack. A treatment that can take all of that away with just one dose could forever enhance the lives of tens of thousands of people around the world.
