I was not very optimistic about the future of gene therapy 3-4 years ago. Well, I was wrong about it. I was obsessed by cell therapy so much that underestimate the progress in gene therapy. Both fields have matured and so did I. Last two years brought us tremendous development in both fields. I realized how much gene and cell therapy could be bind together. Today I’d like to share some links and opinions on why gene therapy is ready for prime time.
First of all, I’d like to highlight the recent notable advances in gene therapy. Last two years there were many of them. I’d mention a few:
- Long-term survival and recovery of T-cell immunity in children with SCID – UCL study 1/2
- French study on SCID – 10-years follow up, one death out of 8, but disease is corrected
- Results of Phase 2 trial in Parkinson’s Disease
- Results of Phase 1/2 trial study of cell gene therapy for the Wiskott–Aldrich Syndrome
- Few cases of treatment chronic lymphoid leukemia with gene-modified T-cells – reports 1/2
- AVV-mediated gene transfer in hemophilia B – results of Phase 1 trial
- A new gene therapy drug was approved on the market
But not everything went so smoothly this year. There were some setbacks. I’d mention two:
- Gene therapy of critical limb ischemia trial (sponsored by Sanofi-Aventis) failed in Phase 3.
- The EMA rejected Glybera (AMT’s gene product) to market in Europe.
Now, I’d like to share an opinion of two important figures in the field.
We now passed the stage which we’re trying to prove something to somebody and trying to overcome our history of maybe promising too much and being criticized for that. And now we’re at the point when we can begin to deliver real treatment for real people.
Gene therapy is ready for prime time and will revolutionize practice of many aspects of medicine and soon to follow will be stem cells.
So, what does gene therapy readiness mean?
In the recent poll of Genetic Engineering News magazine 61.4% of professionals were positive about first FDA or EMA approval of gene product on the market in the next 2 years. There are more than one and half thousand of currently ongoing gene therapy clinical trials. 60 of them on a Phase 3. There are 3 commercially available gene therapy drugs on a market.
Based on all clinical data that gene therapy has accumulated in the last decade, we can say that it works! It could cure a disease or provide better quality of life at some indications. We are ready for expansion and advance of clinical trials. We are ready for marketing approval of few more gene products in the next one-two years. We are ready for post-marketing analysis and commercial manufacturing of the products. So, let field to explode!