next »

[Sunny2701]

First nail is in. FDA approves gene therapy for leukemia.

https://www.cnbc.com/2017/08/31/f-d-a-approves-first-gene-altering-leukemia-treatment-costing-475000.html

[Pratik]

First nail is in. FDA approves gene therapy for leukemia.

https://www.cnbc.com/2017/08/31/f-d-a-approves-first-gene-altering-leukemia-treatment-costing-475000.html

Excellent news!

[Pratik]

Is this also for Thalassemia Major @Andy and Sunny?

[Pratik]

Is this going to be an oral drug or injection? Will chemo be required?

[Andy Battaglia]

No, this is gene therapy for leukemia. The importance is that gene therapy is approved, which will mean the FDA already has experience approving gene therapy and this will make it easier for gene therapy for thalassemia to be approved. This requires chemotherapy conditioning before the process is done. However, it does not pose the risks that bone marrow transplant does. Gene therapy for thals is in trials and doing well. I will point out that there is now a great discussion about developing gene therapy for thalassemia in India. Dr Ramanan is one of the doctors currently talking about the need to get it moving, so it can be made affordable to Indians.