First the update. The first patient that was treated at Sloan Kettering with gene therapy to attempt to cure thalassemia is home and doing well physically. While we won't really know about gene expression for 6-8 months, I can report that the patient has done quite well and has recovered from the chemo regimen used in the procedure. A second patient began during the third week of February and a third will begin in May. In addition, the National Institute of Health has also now come on board and is now involved with SK in this project. That was long sought and is great news.
I want to talk about an issue that is at the heart of this and any other attempt to cure thalassemia via gene therapy, and I want to point out some of the differences between this project and that from another company that is also planning on beginning trials in the US. First, only Sloan Kettering has access to the actual beta globin gene. The patent for this gene has long been held by Errant Gene, and it is now controlled through an arrangement through EG and SK. No other company can use this gene unless it is licensed to them and at this point in time, that seems rather unlikely, so mutated beta globin genes must be used by any other group. Secondly, and to me this is very important. Dr Sadelain, EG and SK decided to use myelosuppression of the bone marrow rather than myeloablation. With myelosuppression chemotherapy, the bone marrow is suppressed but not entirely wiped out, as it is with myeloablation. The thinking is why give the patient more chemo than needed and possibly cause many side effects, including death, if it is not needed? Why not use the approach of a milder chemotherapy to prepare the patient?
This is not a race, although one company may view it that way. Dr Sadelain's team approach is to think about the long term effects on the patient of both the gene therapy and the preparation for the procedure. I totally agree with this approach. If the myelosuppression used is not adequate, then it can be enhanced, but why do this unless necessary. Having had an employee die 4 years after a BMT directly from the damage done to his lungs and kidneys by the preparation for the BMT, was a very sobering experience for me. I don't think we should be approaching gene therapy in the same way as a BMT. I think the lowered chemo regimen is the best approach, until it is proven to not be sufficient. As things stand now, everything looks very good for the first patient and the recovery has gone quickly and gone well. I just cannot see how using a more severe approach of myeloablation is the best decision when we are first starting on this road to a gene therapy cure. I realize that this is also a subject of much disagreement in the world of BMT, as Dr Krishnamurti has been a pioneer of the "kinder, gentler" preparation for a BMT. The big difference here is that we are just starting on this road. Using the milder approach is not an afterthought. As I said, this is not a race. We want to see gene therapy done right and cause as little side effects to patients as possible.
As always, I agree with Dr Sadelain's well thought out methods and I see no reason to use any other approach unless further down the road, we see that it has not been sufficient. My thoughts are always with the patients first and I cannot agree with a more intensive preparation unless it is absolutely necessary. At this point, we do not see that and we should all wait and see what happens as time passes over the next 6 months. I know the other group wants to rush and that they are well financed, but I am not convinced that their methods are what we should be utilizing. Let's stay patient and see how thse first few patients do over the next year.