Biotech Strategy Blog

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

Posts tagged ‘Johns Hopkins’

Franciscan Crab Restaurant San FranciscoWe saw at ASCO last year that response to checkpoint immunotherapy is feasible in some patients with colorectal cancer, but what about other gastrointestinal tumours such as pancreatic, duodenal and biliary cancers?

Can their activity extend beyond the obvious hypermutated tumours such as melanoma, lung, renal and bladder cancers?

Many of you will know that most pancreatic cancers, for example, are detected late and prognosis in metastatic disease is generally poor. You also typically don’t see much coverage of the other GI non-CRC cancers from cancer conferences in the medical media outside of pancreatic cancer occasionally.

At the ASCO Gastrointestinal symposium (#GI16) this past weekend, there was some new data of note in these tumour types that is well worth highlighting and discussing because it may have a major impact on the GI landscape.

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After the intensity of gastrointestinal cancer, we now turn our attention to genitourinary (GU) cancers with the upcoming ASCO GU meeting later this week in Orlando.

Two of the big topics here will be prostate and renal cell (RCC) cancers.

Unfortunately, the long awaited data in adjuvant RCC demonstrated that early treatment with sorafenib or sunitinib did not improve outcomes in locally advanced kidney cancer after resection. According to the ASCO press release, the trial conducted by Dr Haas and colleagues at U Penn discovered that:

“The average period to disease recurrence was similar between those who received sorafenib or sunitinib after surgery (5.6 years) and those treated with placebo (5.7 years).”

We will therefore turn our attention to castration resistant prostate cancer (CRPC).

One of the recent and ongoing controversies is splice variants, especially AR-V7, which is thought by some research groups to confer resistance to the hormonal therapies, enzalutamide and abiraterone. The big question though, is does it, and how useful is an assay in helping to determine appropriate therapy? Are there other factors at play?

We looked at the latest data and put the findings in context with what we know from other published research.

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One of the exciting things about the biotechnology industry is its ability to innovate and translate developments in basic science into potential new drugs.

I previously wrote about denufosol in cystic fibrosis (CF), a disease that affects about 30,000 people in the United States and 70,000 worldwide.  The disease is characterized by the accumulation of mucus that leads to bacterial overgrowth and chronic lung infections. Mucus cannot be removed from the lung in CF due to abnormal mucociliary transport resulting from impaired epithelial chloride secretion and sodium hyperabsorption.  This is now known to be due to defective cystic fibrosis transmembrane regulator (CFTR) protein. A good overview of this can be found in the 2006 New England Journal of Medicine Editorial by Felix Ratjen, “Restoring Airway Surface Liquid in Cystic Fibrosis.”

A good overview of the pipeline of new drugs in development for CF can be found on the Cystic Fibrosis Foundation web site.  Vertex in particular has two drugs  (VX-809, VX-770) in late stage development that are cystic fibrosis transmembrane conductance regulators, aimed at increasing CFTR function.  Phase 3 registration data for VX-770 is expected in the first half of 2011. I look forward to writing about the results.

Recently, a team from Johns Hopkins led by Neeraj Vij published a paper in the January 2011 issue of Journal of Immunology on the “Critical Modifier Role of Membrane-Cystic Fibrosis Transmembrane Conductance Regulator-Dependent Ceramide Signaling in Lung Injury & Emphysema.”

The researchers found that lung damage in mice was associated with changes in the amount of CFTR in the cell surface membrane.  Decreases in the amount of CFTR were associated with increased ceramide, a trigger of inflammation of cell-death. Or as the the paper describes it:

“CFTR expression inversely correlates with severity of emphysema and ceramide accumulation in chronic obstructive pulmonary disease subjects compared with control subjects.”

The emergence of inflammation as a key role in chronic disease was the subject of a previous blog post about diabetes, so is interesting to see another area where it is involved.

This basic research shows that developing drugs that target CFTR and mediate ceramide may have an important role to play in the treatment of emphysema, a chronic obstructive pulmonary disease (COPD) that affects 2 million Americans.  Translational medicine that can take basic science and apply it to clinical practice is key to the long term success of the biotechnology industry.

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