Questions and Answers with Prof. G.J. Ossenkoppele
Which are the today most important unmet clinical needs in AML?
- Improving CR rate in elderly AML.
- Preventing relapse in elderly and younger AML.
- Approval of new promising drugs.
- Determine who is fit for intensive treatment.
Which are the most promising new drugs for the treatment of AML?
- FLT3-ITD inhibitors (midostaurin, quizartib).
- Hypomethylating agents (HMAs) and new “second generation” HMAs.
- Vosaroxin (topoisomerase II inhibitor).
- Bcl-2 inhibitors (venetoclax).
- CRM 1 inhibition (selinexor).
Which were the most exciting news from the recent ASH Meeting ?
- Positive outcome in the Phase III RATIFY study in FLT3-ITD/TKD mutated patients of midostaurin added to standard therapy.
- Promising combinations in addition to HMAs (i.e. azacytidine plus pracinostat).
How far is a really targeted therapy for AML (apart APL) ?
- Not so close because of the heterogeneity of AML; FLT3ITD is the first example although midostaurin is not very specific.
Has immunotherapy a future in AML ?
- Absolutely, allogeneic stem cell transplantation has already been an example for many years. But new modalities are emerging:
- monoclonal antibodies (anti-CD33, anti-CD123, and anti-CD47)
- BiTEs (anti-CD33, anti-CLEC12A)
- CARTs will soon be explored in AML
- Immune checkpoint inhibitors.
Gert J.Ossenkoppele, M.D., PhD., is Professor of Hematology at the VU University Medical Center, Amsterdam, The Netherlands.