Two of the main reasons why gene therapy is commanding so much attention nowadays is because of its promise to provide cures for terrible conditions and its seven figure price tag. But there are a few investigators at the University of Pennsylvania that have raised concerns over this weekend that their blindness cure was not going to work for more than 3 years. This shocking discovery could spell trouble for many biotech’s.
This team was led by Samuel G. Jacobson. They followed up with 3 long-term patients of gene therapy who received treatments where a virus was used to produce healthy copies of the RPE65 gene. The mutations within this gene are linked to close to 10 % of all Leber congenital amaurosis cases which is an inherited disease.
There were multiple patients within the 15 person group that showed remarkable improvements in vision as well as three patients that were studied over a long period of time. This was just a small study although it did go a long way in reviving expectations for gene therapy. But instead of this leading to permanent cures like everyone was expecting, their vision peaked before eventually declining.
But this minor setback is definitely not going to spell the end for them. Jacobson said that all it took was a follow-up treatment for patients who wanted to maintain their vision and several combination therapies could also be developed to help amp up efficacy. They could then further evaluate patients in order to determine which procedure was the most beneficial for the patients.
Spark Therapeutics’ lead therapy helps in delivering a fully functional copy of RPE65 gene to LCE patients. These treatments are still in the 3rd phase of study and have 28 patients.
There has been a lot of speculation about how anyone is going to be able to afford gene therapies that could easily cost more than a million dollars. And the difficulty of pricing a therapy which doesn’t really guarantee any cure can be quite complicated right?