Biotech Strategy Blog

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

Posts tagged ‘PDGFRA’

Paths to success in cancer research

It’s time to pull together some notes, ideas, and clinical data on various biomarkers based on data available from clinical studies in oncology R&D and see how much progress we are making.

Are biomarkers a good path to success in cancer research or are they a gloomy red herring to the road less travelled?

Both answers can be equally true, but how do we tell the difference?  Are there any clues that we can use ahead of time to avoid later disappointment?

There have been several early studies that we’ve been following lately with readouts available from numerous cancer conferences, both positive and negative.

Can we learn from the failures and successes of the past to better interpret outcomes from future trials?

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Palace guard in Stockholm

Stockholm, Sweden – The annual meeting of the European Hematology Association (EHA) is in full swing with updated data from two blue companies, Blueprint Medicines and bluebird bio of interest to BSB readers.

There is often beauty and simplicity to be found in nature that also applies to oncology R&D.

One of those aspects can be found in the concept of targeting particular aberrations or molecular rearrangements that driven oncogenic activity.  Once you connect the dots to arrive at these key targets, you can develop therapeutics that inhibit the activity, resulting in cessation or reduction in proliferation.

In our latest post, we focus on an update on Blueprint Medicine and take a look at their various programs in early clinical development, as they have quite a lot going on with multiple targeted compounds in different areas, including hematology.

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Sarcoma is something we call one disease but actually represents 50-70 different histologies, which poses challenges for drug development.  Not only do you have to identify what’s the unique target, but it’s hard to accrue patients into trials, when a major center may only see a few of each sub-type.

Soft tissue sarcoma is an area of unmet medical need, and one I have been interested in since launching Gleevec in GIST (way back when) when I was fortunate to get to know many of the leading sarcoma experts.

Dr George Demetri

George D. Demetri, MD. Photo Credit: DFCI

One of these is Dr George Demetri, who is Director, Center for Sarcoma and Bone Oncology at the Dana-Farber Cancer Institute and a Professor of Medicine at Harvard Medical School.

At the recent European Cancer Congress in Vienna, I had the privilege to talk with Dr Demetri about some of the latest research in soft tissue sarcoma.

We spoke about cancer immunotherapy, new small molecules and monoclonal antibodies, and the potential of targeting the epigenetic machinery.

A lot of what Dr Demetri is doing is currently “under the radar” and while he didn’t give any secrets away, he did give some sense of where some breakthroughs may occur in the not too distant future.  He also talked about how sarcomas with a specific target can be used for proof of concept clinical trials of novel agents.

Given the pressure that many companies are under to speed up their path to market strategies, accelerated approval in a rare tumour subset is one approach that can be considered.

It’s an exciting time in the field with the potential for several agents in development to move the needle and make a difference. I hope you enjoy this post, it was a real pleasure to talk with Dr Demetri again.

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