Biotech Strategy Blog

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

Posts tagged ‘AML Drug Targets’

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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ENA2014_Banner_400x250The annual Symposium on Molecular Targets and Cancer Therapeutics jointly run by the EORTC, NCI and AACR (aka “the Triple meeting”), starts tomorrow in Barcelona (Twitter hashtag #ENA2014).

This makes it a particularly busy week on the conference calendar as we segue from immunotherapy at SITC to Molecular Targets, not to mention the start of our previews on hematologic malignancies at the American Society of Hematology (ASH) coming up fast!

The Triple symposium alternates between the US and Europe. In case you missed it, here’s a link to our extensive coverage from last year’s Boston Molecular Targets Symposium that we attended.

This year in Barcelona, one of the highlights is the presentation of the latest clinical data for the phase 1 trial of AG-120 (Agios), an inhibitor of the IDH1 enzyme.

As the abstract that will be presented tomorrow by Daniel Pollyea, MD (University of Colorado, Denver) notes, “Cancer metabolism represents an emerging field of novel cancer target discovery.” Along with epigenetics, it’s an exciting area that we are actively following. For years metabolism has been promising to breakthrough with new ideas that move the needle in clinical research but few have lived up to the lofty expectations, with one exception.

We first wrote Agios back in 2012, when they showed that that mutations of the metabolic gene IDH1 were consistent with that of a cancer causing oncogene in glioblastoma.

Then at ASH 2013, we heard the preclinical data for AG-221 using IDH2 mutant acute myeloid leukemia (AML) xenografts and interviewed the Agios CEO, Dr David Schenkein. The story for AG-221 continued in a positive vein with early clinical data at AACR and ASCO earlier this year. You can read more in the related posts we’ve highlighted at  the end of this article.

Tomorrow at EORTC-NCI-AACR Molecular Targets in Barcelona we will hear about the preliminary results for the phase 1 trial of AG-120 in patients with advanced hematological malignancies including those with relapsed or refractory AML, myelodysplastic syndromes (MDS) and elderly untreated AML that harbor an IDH1 mutation.

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