Biotech Strategy Blog

Commentary on Science, Innovation & New Products with a focus on Oncology, Hematology & Cancer Immunotherapy

Posts tagged ‘Acute Myeloid Leukemia’

Post 2016 US Election, we move on and get back to business with an in-depth review of some new science and clinical data.

ash-2015Yes, it’s time for another Bushidō – “Way of the Warrior” – guide to the key ASH abstracts!

Here we focus on acute myeloid leukemia (AML), a difficult and challenging disease to treat with a high unmet medical need for new effective therapies.

In this Preview we look at key companies in the AML space, as well as a look at what’s happening in classic targets and also some new ones that are receiving notable attention, both preclinically and also in the clinic.

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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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San Francisco F line Trolley originally from MilanSan Francisco – Acute Myeloid Leukemia (AML) is largely a disease of the elderly since it is uncommon before the age of 45. It generally has a much poorer prognosis compared to other leukemias such as CML and even ALL. There are two main treatment options – high chemotherapy (ara-C is the main bedrock) or a stem cell transplant in those patients who are considered eligible. With the average age at diagnosis being ~66yo, many patients may be elderly and frail, making a SCT not a viable option.

Ara-C (cytarabine) has been around for many years and despite numerous clinical trials, it has yet to be displaced. There’s plenty of room for improvement though, and a high unmet medical need still exists. The good news is that despite the challenge of finding a highly effective yet well tolerated therapy, there’s a lot of R&D activity in this space.

In this preview of the data to be presented at the American Society of Hematology (ASH) annual meeting in San Francisco, I highlight my top 10 AML abstracts that are worth checking out.

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