Oncology R&D is very much a tale of two cities. At one end you have all big pharmas and biotechs with significant resources in the form of very large budgets, (hopefully) an extensive pipeline, plus many hands on deck to efficiently spread the workload, while at the other end you have what I call the ‘baby’ biotechs with completely the opposite situation coupled with a much greater need for prudence in how those scarcer resources are managed.
A failed drug development may not affect big pharmas very much, it’s written in to the strategic plans after all, and a 90% failure rate is very much de rigeur so you’re looking for the rare gems that will shine and carry the rest. In small biotechland, such inherent risks are much more prominent – and drastic – because a failed program can wipe out the stock overnight such that future endeavours to raise money are greatly hampered, putting the very life of the company at risk of not only delisting (if publicly traded) from stock exchanges such as NASDAQ, but also the ultimate doom.
The constraint that both bookends have in common, however, is familiar to many readers – how to get the best shots on goal given the time, energy, and resources available?
At BSB we don’t write just about big Pharma – we also try to highlight the roller coaster experienced at the other end of the spectrum and showcase some cool science in the process. Given our interest in stapled proteins as well as the various challenges associated with both tumour suppressors and MDM2, it seemed like a good idea to catch up with the folks at Aileron Therapeutics (NASDAQ: ALRN) and learn more about their progress since they combine all three elements in one go…. it’s time for some gems from the ESMO19 poster hall.
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Much of the focus in multiple myeloma over the last decade has focused on two key drug classes – proteasome inhibitors and IMiDs – with some recent approvals for monoclonal antibodies targeting key proteins on the surface of malignant myeloma cells such as CD38.
#ASH16 in San Diego
Combinations of these core therapies have lead to a noticeable improvement in outcomes for people living with the disease – from 3-4 years over a decade ago to now approaching 10 years post diagnosis.
If we want to continuously beat the status quo and improve on the chronicity, however, it is likely that several things will need to happen:
- Better understand mechanisms of resistance that induce relapse
- Develop predictive biomarkers of response
- Identify novel therapeutic targets
Here. we focus on the latest preclinical findings that were recently presented at the American Society of Hematology (ASH) in San Diego and explore where the future might be headed in this disease.
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The first day of the 2016 EORTC-NCI-EORTC Molecular Targets meeting brought us chilly weather and a frozen lake outside the conference centre in Munich. Brrrr!
It also heralded a great lineup of cancer researchers largely characterised by unconventional thinking. This, of course, is a good thing because it is only by dismissing dogma that a field can move forward unconstrained.
There were several talks that I will come back to in a separate post, but here I wanted to focus on one particularly good talk on breast cancer, something we haven’t covered in a while.
A decade or two ago, breast cancer made a lot of progress – we saw the emergence of gene expression profiling, the identification of different histology types, treatments for hormonal sensitivity or HER2-positivity and then… nothing. Meanwhile, the issue of drug resistance plagued researchers – why don’t all women respond and why do they become resistant?
In the meantime, we’ve seen a wealth of progress in melanoma, lung, kidney and bladder cancers, enormous strides in hematologic malignancies and many other areas. Breast cancer, the early star, seems to have faded and we haven’t had much to be cheerful about aside from a few isolated cases.
The good news is that things are a-changin’ though and research is looking more promising as we learn from lessons in basic and translational research and how they can be applied to new therapeutics and drug resistance.
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Post 2016 US Election, we move on and get back to business with an in-depth review of some new science and clinical data.
Yes, 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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