At the recent Triple (EORTC-NCI-AACR) and ASH meetings, Blueprint Medicines (Cambridge, MA) presented data on some of their targeted compounds in early clinical development including data in KIT, PDGFα and FGFR4 driven cancers.
While many observers attention is currently distracted on cancer immunotherapies, let’s not take our eye off the ball and forget that when we do find driver oncogenes in rare tumours, the activity of TKIs can still be superior in these situations and offer exquisite sensitivity, leading to exceptional responses.
Here, we take stock with a look what Blueprint are doing, where they’re going and also offer some perspectives from senior company executives, whom we interviewed last month.
Which reminds me, someone recently asked why we do so many interviews, “You do a prodigious amount of interviews on BSB, why is that?”
The answer is very simple – to learn faster and share that knowledge with other like minded souls. Charlie Ambler, author of Daily Zen, sums it up well in an essay about Talk Less:
“In Zen tradition, I’d like the kill the Buddha that is Lao Tzu and revise his ancient saying. It’s not that those who talk don’t know and those who know don’t talk— it’s that talking often inhibits us from knowing.”
Thus, the corollary here is that if you undertake interviews with scientists and researchers regularly then you have the pleasure of talking less – the person in the hotseat naturally talks more – and you learn faster.
What’s not to like? We all learn different things depending on our perspective and knowledge base. Sometimes, I even find re-reading old interviews a year or so later while preparing for a new one a related topic teaches me something new I didn’t see or realise before, simply because my own understanding has improved. Hopefully that is also true for subscribers!
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ASH16 in San Diego
Today we resume our coverage from the recent American Society of Hematology (ASH16) annual meeting with a look at some fascinating and highly compelling science that was presented in an obscure and hard to find tiny hall in San Diego.
This story is also about how a small biotech company that many casual observers may not even be aware of, is taking advantage of advances recent research to grab a clinical lead in a very specialised field in oncology that may yield a novel approach worthy of taking notice of..
Genomics is increasingly becoming a core element of cancer research. Think of it as the alphabet soup of molecular biology concerned with the structure, function, evolution, and mapping of genomes.
Once we understand and identify the genomic landscape in health and diseases such as cancer, it allows numerous platforms to evolve whereby those unique differences can be identified (as driver vs. passenger mutations, for example), explored in depth, and later key ones targeted with therapeutics. Inevitably, there are many ways to do this.
Much of the focus in genomics has been on DNA, but what about RNA?
RNA is important because a mistake – even a single nucleotide – can be devastating to the cell, and a reliable, repeatable method of RNA processing is necessary to ensure cell survival. Mis-splicing can thus lead to the development of new point mutations and genomic instability deep in the cell nucleus, potentially causing the evolution of certain cancers.
Paradoxically, these aberrations also offer novel therapeutic targets – but are they druggable?
What we are exploring here is a completely different approach, both in terms of how a fledgling company is funded and also the type of research that is conducted.
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HI Koko Crater Flowers
Over the last week or so, we’ve received a lot of questions on the following topics relating to women’s cancers in breast and ovarian carcinomas:
- APHINITY impact – pertuzumab and neratinib
- PARPs in ovarian cancer – niraparib, rucaparib and olaparib
- Seattle Genetics and Immunomedics
So this is probably a good time for a February BSB Reader Q&A post on the hot topics of the moment in cancer research.
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The huge pile of interesting scientific papers yet to be read seems to breed overnight and one constantly feels like they’re 2,000 articles behind, even with spending Friday mornings attacking them with gusto.
This was as true in my PhD days as it is now. For a scientist, these represent a lifeline and an important necessity, rather than a luxury.
In the last journal club posting we covered some hot topics in cancer immunotherapy, so this one covers a very different topic, namely targeted therapies.
It’s a good time for a new journal club post, where we tackle some of the recent published literature in oncology and highlight some important new findings that could have an impact on cancer research and development.
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Today we continue the second of a two part interview with a global thought leader who is also a scientist-clinician and well versed in cancer research as well as clinical trials.
Old Town Hall, Munchen
We explore how we can do clinical trials better in order to learn via a more rigorous process what works, what doesn’t, and why. After all, we we don’t know why certain approaches didn’t work or what the mechanisms of resistance are, how can we possibly improve?
Randomness is not necessarily a good thing in clinical research, especially if you don’t know what target you’re actually trying to hit!
If you missed the first part of this latest KOL interview and want to catch up then you can find it here (Link).
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Two of the most intriguing developments in cancer research over the last 5 years have been checkpoint blockade and CAR T cell therapies. There’s no doubt that they work – in some patients – or that toxicities can be challenging to manage at times, but what has been very interesting to me has been physician reactions to the rise of immunotherapies.
There has been much noise about biomarkers, including whether they work or not in this niche, as well as how do we go about selecting patients for therapies and combinations?
Ultimately, immunotherapies will be no different from targeted therapies in that we need to better understand the underlying biology in order to move forward beyond the low hanging fruit and figure out how we can best select appropriate therapy for each individual based on their particular characteristics.
The worry that many researchers have is that we could end up making the same mistakes with immunotherapies as targeted therapies, i.e. treat them in a broad fashion akin to throwing mud at the wall. Indeed, some companies are already doing this, much to the consternation of the research community.
So how do we go about doing things better and thinking more strategically about what needs to be done?
Up next is the first in a two-part interview series with a global thought leader who is a scientist-clinician with expertise in both immunology and oncogenic pathways. What does he have to say about where we are now and importantly, what does the future hold?
This is the penultimate article in our coverage from the Triple meeting in Munich, held in November 2016.
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San Francisco: In the final post of the week, it’s time to focus on some of the interesting concepts and early ideas being explored in GI tumours such as pancreatic and colorectal carcinomas.
Gems from the Poster Hall or what Dog Drug Heaven really looks like?
Despite the image implied by the used poster bins (right), there were actually several encouraging signs from emerging IO approaches as well as some surprising results that lead to some compounds – or at least some indications – going off to dog drug heaven.
There were also some salutory lessons to be learned in terms of understanding biomarkers and useful these can be.
After years of incremental improvements with targeted therapies, it’s time to look at whether some immunotherapy combinations can make an impact in what is known as cold tumours.
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San Francisco: ASCO Gastrointestinal symposium 2017 – Update on metastatic colorectal cancer
It might surprise quite a few people that colorectal cancer (CRC) is the third most commonly diagnosed cancer globally, especially in the western hemisphere where hereditary, dietary and lifestyle factors can be important.
The bedrock of therapeutic approaches in this disease have largely centred around chemotherapy (FOLFOX or FOLFIRI) along with targeted therapies against EGFR (cetuximab, panitumumab) or VEGF (bevacizumab, ziv-aflibercept, regorafenib etc).
In our second report from #GI17, we take a look at some of the emerging monotherapy and combination approaches that are showing early signs of moving the needle in advanced CRC, an area that has been relatively dormant of late. This is partly because it’s a cold tumour and with the focus on cancer immunotherapies, it’s not the first tumour type that companies will necessarily rush to evaluate.
Things are changing though, even in colorectal cancer so it’s time to look at some key studies that may teach us more about this disease.
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San Francisco: A look at what’s new in gastric cancer (GC) from the 2017 ASCO GI meeting.
Day 1 of #GI17 is filling up…
There were several phase 3 trials presented in GC and gastro-esophageal junction (GEJ) carcinoma in both targeted therapies and immunotherapies this past weekend.
- When we look carefully at the latest data, what do we find?
- Where are the opportunities and challenges in this niche?
Another critical question that many observers will be interested in is…
Will BMS’s checkpoint inhibitor, nivolumab (Opdivo), overcome recent setbacks in lung cancer and make a mark in stomach cancer to challenge approved targeted therapies such as ramucirumab (Cyramza)?
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The most common cause of breast cancer death is when metastatic ER+ tumors develop resistance to treatment. There remains an huge unmet medical need for new effective treatment options.
One translational clinical researcher leading the way in understanding the molecular and genomic landscape in ER+ metastatic breast cancer is Nikhil Wagle, MD (@Nikhilwagle), Deputy Director, Center for Cancer Precision Medicine at the Dana-Farber Cancer Institute (DFCI).
He’s shown the power of partnering with patients via social media to speed research, with over 3,000 men and women joining the Metastatic Breast Cancer Project that launched in October 2015.
At the 2016 San Antonio Breast Cancer Symposium, Dr Wagle kindly spoke to BSB about the genomics research that he and colleagues are undertaking, and what this may mean for how metastatic breast cancer is treated:
- What if you could help accelerate clinical trial enrollment via a network of educated and empowered patients?
- What if you could access real world data to help learn about exceptional responders?
For those of you in Pharmaland, here’s one approach that should captivate the imagination of what’s possible.
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