Biotech Strategy Blog

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

Posts from the ‘Acute Leukemia’ category

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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Yesterday sudden and unexpected news from Seattle Genetics caused quite a stir…

“Seattle Genetics Announces Clinical Hold on Several Phase 1 Trials of Vadastuximab Talirine (SGN-CD33A).”

Part of the Seattle Genetics exhibit booth at #ASH16, taken with permission

In short, over 300 patients have been treated with the ADC and six experienced hepatotoxicity, including several cases of veno-occlusive disease, with four fatalities.

We’ve written about AML several times recently and also received a number of reader questions on this latest development, so it’s time to explore the issue in more depth and look at the implications. We also include some expert commentary from a leukemia specialist for their take on the issue.

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The 2016 annual meeting of the American Society of Hematology with over 27, 000 attendees, a record high, was the venue for the announcement of a major new initiative by the Leukemia Lymphoma Society (LLS), called Beat AML.

It is lead by three well respected researchers in the Hematology/Oncology field:

  • Dr John Byrd (Ohio State)
  • Dr Brian Druker (OHSU)
  • Dr Ross Levine (MSK)

Beat AML is a special project at LLS, who have developed a broad collaboration with academic researchers, pharmaceutical companies, a genomic provider, and a clinical research organization:

 

Source: LLS

Initially, there will be five trial sites, which will each offer all arms of the trial. The centers are:

  • Memorial Sloan Kettering Cancer Center in New York
  • The Ohio State University Comprehensive Cancer Center in Ohio
  • OHSU Knight Cancer Institute in Oregon
  • Dana-Farber Cancer Institute and
  • Massachusetts General Hospital Cancer Center, both in Massachusetts.

Further sites and (hopefully) also other drugs from pharma companies will be added in due course, so if you’re interested in joining this project, do contact them after checking out more details here!

For our industry readers, this would be a great opportunity to get involved in an exciting and landmark study for AML, whether you are a researcher or a company with a promising drug in early development. These types of trials can help speed up drug development if a therapy graduates in a particular subset.

Here, we offer an in-depth analysis of the scientific and clinical rationale behind this important landmark study and the targets/drugs selected to date.

BSB also spoke with Dr Brian Druker, Director of the Oregon Health and Science University (OHSU) Knight Cancer Institute in Portland, Oregon, who offers additional insights on the special project.

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Oncology R&D is tough and there are many more failures than successes, despite the FDA approving more than they’ve rejected over the last two years. That’s quite unusual in my experience.

Dr Mario SznolAs Dr Mario Sznol (Yale) told us at SITC recently, sometimes these things are sometimes more whimsical. He was referring to different types of modalities that can be used in conjunction with cancer immunotherapies, but the sentiment is also highly relevant to the FLT3 AML space.

The critical questions we need to think here about are:

  1. What’s different about the various approaches?
  2. What can we learn from the FLT3 experiences to date that give us clues about the changing landscape in AML?

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We have followed the roller coaster development of the Bcl2 inhibitor, venetoclax (ABT–199/GDC–0199), for several years now.  There have been some lowlights along the way, but lately, things have been much rosier for AbbVie and Genentech as a more sensible dosing and patient management approach has been paying off.

Recently at ASCO and ASH, we have seen encouraging new data emerge in leukemia (AML and CLL), lymhomas (NHL), and even multiple myeloma.

New data has now emerged that looks quite interesting in another blood disorder. Today, we took a look at the data and also the potential implications for venetoclax’s development program.

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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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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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