A recent article in The New York Times, headlined “After Setbacks, Small Successes for Gene Therapy,” quoted IU School of Medicine’s Kenneth Cornetta, MD, chair of the Department of Medical and Molecular Genetics and director of the IU Vector Production Facility. The Times article reported on three recent small successful trials involving gene therapy, suggesting that the field is “on the edge of a resurgence.” I asked Dr. Cornetta to expand on his comments:

Q: In your comments to the New York Times, you called the recent developments “exciting.” Do these recent trials reflect a resurgence of interest by researchers in gene therapy?

A: Gene therapy has always been there, and if you look at the estimated accruals (of patients into gene therapy trials) over the past decade – at least reviewing data from the Office of Biotechnology Activities, which oversees gene transfer research at the National Institutes of Health – patient accrual has not changed significantly – or has increased. Some re-tooling has been done as we learned what approaches are likely to work or not work.

Q: What is it about gene therapy that has made successes so difficult to achieve?

A: Considering how long scientists have been working on gene therapy, I think we are doing extremely well. We are seeing success in less time than it took to successfully perform bone marrow transplants. Gene therapy is moving at the expected pace for any new therapeutic.

Clearly we have the proof of principle to treat disease, and as was reported in the New England Journal of Medicine earlier this year (in a trial to treat children withsevere combined immunodeficiency) we have patients with long term benefits – with one patient now out over eight years.

Q: How does the IU School of Medicine benefit from the presence of the IU Vector Production Facility and the National Gene Vector Biorepository?

A: The recent Science article shows the arrest of disease progression in ALD utilized lentiviral vectors which are the type of vectors produced at the IUSM. We are one of the few centers in the world capable of making these vectors for clinical use. While we were not involved in that trial, we are involved with a number of trials that are just starting and we will continue to be involved as these trials move into the clinic.