A parents worse nightmare number one is to hear that your child has an incurable disease and will likely not make it passed 20. This is reality for children with Duchenne ( a muscle distrophy desease). There is no cure yet, but gene therapy might be the solution, and scientist are working on it.
Gene therapy might sound like science fiction, but the first medicine was approved in August 2017. This is a huge milestone, as 19 years ago, gene therapy was thought to be a dead end.
Gene therapy was hot over 20 years ago, many pharmaceutical companies were absolutely over the moon by the potential of it.
However, in 1999 in one of the human trials of a novel medicine using gene therapy, a volunteer of 18 years old died, and this set back the field completely. Luckily universities continued the research and pharma picked it up again, now 19 years later the first medicine was launched on the market.
The new medicine was approved in August 2017 to treat children and young adults with acute lymphoblastic leukemia (ALL). While this is a treatable cancer of the bone marrow and blood, conventional drug treatment regimens can be brutal and not always successful. In August the FDA announced the approval of a new gene therapy to treat this desease:
”we’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” said FDA Commissioner Scott Gottlieb, M.D.
The medicine approved is known as Kymriah and was developed by Novartis. Indeed a new era is approaching, as we can see that more than 2,500 gene therapy products are in early and later stage trials. Gene therapy is now the second largest class of drugs being developed, bigger than antibodies and just behind New Chemical Entities (NCEs). This is not coincidence. The gene therapy market is a green field for pharmaceutical companies, who are desperately looking to get new blockbusters to the market.
Of course pharmaceutical companies are in it to make a profit. The pharmaceutical companies have had it hard getting new medicine to the market, and this first gene therapy medicine is priced at $450k ( while they could have asked double and only needs to be paid if the medicine works). Still Novartis got a lot of criticism, because of the large sum this medicine costs. It’s the pharmaceutical paradox. On one hand it’s unethical to charge a lot, on the other hand it costs an incredible amount to develop new medicine, while it’s still a business that needs to make a profit. If this price is justifiably, I want to go into another time.
In any case, there is now hope for people with incurable diseases. Also hope for chronic diseases that are treatable with daily medicine to get a one time treatment.
I’m amazed at the milestone humanity has achieved, by being able to edit genes of very ill people and cure them.
( I’m a PhD in chemistry, my expertise is in medicinal chemistry, which will for ever have my love and fascination)