Acute Myeloid Leukemia (AML) is challenging disease to treat and quite distinctly different from its cousin, acute lymphoblastic leukemia (ALL). The first is more common in adults, while the second is more prevalent in children. Success rates with pediatric ALL have far outstripped what we have achieved with adults in AML to date, partly due to the elderly nature of the disease making for poorer outcomes with stem cell transplants (SCT), as well as increased clonal heterogeneity and cytogenetic complexity with age.
Quite a few FLT3 inhibitors have come and gone over the years – many keen observers will remember Cephalon’s (now Teva) TKI called CEP-701, which was tested in relapsed/refractory disease and Elderly AML, for example, and slid off largely unnoticed to dog drug heaven.
How much does clinical trial design impact a drug’s success or failure?
Sometimes quite a bit, as this story with midostaurin demonstrates; limited activity in advanced disease but much more dramatic results in the upfront setting. Clearly, sometimes testing drugs in later disease does not predict their future performance elsewhere!
To put more colour on the data presented at ASH, we interviewed a thought leader in adult AML for his perspective on the FLT3 R&D developments.
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ASH 2015 — taken before 7am!
Orlando – a presentation in the plenary session at #ASH15, the 2015 annual meeting of the American Society of Hematology, is the pinnacle of any doctor or researcher working in the hematology field.
Yesterday, we had the privilege to interview Dr Richard Stone (Dana-Farber Cancer Institute) ahead of his plenary presentation at ASH:
Abstract 6: The Multi-Kinase Inhibitor Midostaurin (M) Prolongs Survival Compared with Placebo (P) in Combination with Daunorubicin (D)/Cytarabine (C) Induction (ind), High-Dose C Consolidation (consol), and As Maintenance (maint) Therapy in Newly Diagnosed Acute Myeloid Leukemia (AML) Patients (pts) Age 18-60 with FLT3 Mutations (muts): An International Prospective Randomized (rand) P-Controlled Double-Blind Trial (CALGB 10603/RATIFY [Alliance])
Anyone who has been to an ASH education session on AML knows how hard a nut it is to crack, so it’s wonderful to see some positive data, in what is commonly considered to be a “graveyard” disease.
The trial has taken a long time to come to fruition, so all credit to Dr Stone and colleagues. We’ll be writing up more about the data in our post meeting coverage. For additional background, you can also check out our FLT3 preview in AML, which details some of the history and context for this study. The data from the phase 3 study is likely to form the backbone of a registration filing for Novartis with this compound in the near future based on successfully meeting the trial endpoints.
We also kick off today’s highlights with quick reflections on some of the hot topics that emerged yesterday including Bluebird Bio’s lentiglobin, Bellicum’s pipeline and .
During the day, as the opportunity presents, we’ll also be providing commentary on sessions we attend.
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