Category Archives: Mutations

Targeted therapies for cancer – overview

The National Cancer Institute has a very informative, yet easy to understand overview  presented as a Q&A – Targeted Cancer Therapies. Continue reading

Cancer diagnostics in clinical use

Diagnostics that not only diagnose cancer, but also help select the appropriate therapy and monitor response are a mainstay in modern oncology, and their use is growing.  Precision medicine, identifying genetic markers of disease and designing treatments to exploit these, is the essence of  targeted cancer therapy.   Continue reading

Ras – targeting the “undruggable”

The Ras onogene has been studied for 30 years, with over 40,000 scientific articles published on it.  However, an inhibitor of the mutated form of Ras has not been able to be developed.  So, researchers have developed downstream blockers of the Ras cascade – MEK inhibitors and Raf inhibitors, for example.  But why can’t a molecule that blocks mutated Ras be identified? Continue reading

EGFRm+ non-small cell lung cancer – new treatments in development

For patients with activating mutations to the EGFR receptor kinase, small molecule specific inhibitors Iressa (gefintinib) or Tarceva (erlotinib) are administered as front-line treatments.  But, non-small cell lung cancers develop resistance after about one year of treatment via a single recurrent missense mutation (T790M) to the tyrosine kinase.  This is similar to resistance observed in patients with CML (Chronic Myeloid Leukemia) following treatment Gleevec (imatinib), which is mediated by a mutation to the Bcr-Abl transgene. Continue reading

Epigenetic signature in blood for breast cancer risk

As reported in Genomic Medicine on June 27, an epigenetic signature in patients with BRCA1 and BRCA2 mutations was found by examining DNA methylation (DNAme) patterns on whole blood samples.  Importantly, this signature predicted for a high risk of developing breast cancer even in those patients who did NOT have BRCA1 and BRCA2 mutations. Continue reading

PARP inhibitor rejected by FDA Advisory Committee, then Approved for refractory patients

In June 2014, the FDA Oncology Drug Advisory Committee voted 11-2 to delay approval of AstraZeneca’s olaparib, a PARP (poly-ADP ribose polymerase) inhibitor for maintenance therapy in patients with ovarian cancer.  The product received Accelerate Approval designation from the FDA, which provides for conditional approval pending follow-up studies, based on surrogate endpoints from Phase 2 trials, in this case, progression-free survival (PFS).  Continue reading

Genetic Analyses of Cancer – Precision Medicine a New Standard

Memorial Sloan Kettering has started testing the cancers of its patients for 341 cancer-related genes.  It is collaborating with Quest Diagnostics to perform the analyses in an effort to identify biomarkers that can best guide appropriate therapy.  This is precision medicine, and it is happening at other major cancer centers, as well… Continue reading

Precision Medicine: SWOG Lung-MAP study – unique public/private collaboration

Using genomics to take-on advanced non-small cell lung cancer, the Southwest Oncology Group announced the start of a master protocol that will screen patients for optimal eligibility into one of five clinical trials with experimental drugs… Continue reading

FDA Clinical Hold lifted on telomerase inhibitor

In clinical trials, “clinical holds” are imposed by the FDA when toxicities emerge that are unexpected, either new toxicities that have not been seen before, (or were not anticipated) or increased severity of known or anticipated side effects.  The hold allows the FDA to assess the data, demand more data from the sponsoring companies, and make appropriate changes in the study protocol… Continue reading