In 1938, it was discovered that the urine of a woman could be injected into frogs as a way to test for pregnancy (1). If the frogs laid eggs after the injection, then the woman was pregnant. The assay had a relatively quick turn-around time, was simple, and generally accurate…the principle being that the hCG hormone excreted in urine of pregnant women caused the frogs to lay eggs. However, as always, there was a better way.

When working on targeted therapeutic drug development in oncology, tumor mutation heterogeneity makes it difficult to treat all patients. However, there is one biomarker that has been found to affect 30% of all tumor cases- the RAS mutation. The abundance of this genomic abnormality was discovered via the ongoing investigations into targeted RAS cancer therapies. Researchers thus have been working to find a method to block the RAS pathway in all tumors, with the desired outcome to treat all patients regardless of differing cancer subtype.

I just came back from an educational and inspirational two days attending the Powering Precision Health Summit held in Boston, MA. Breakout sessions throughout the meeting focused on targeted therapies and biomarker detection for neurodegenerative, oncology, autoimmunity, cardiovascular and metabolism diseases.

Glioblastomas — the most common and aggressive brain tumors in adults — can be difficult to treat because therapies only affect a proportion of tumor cells, which in turn leads to a poor survival rate in the patient population. It is hypothesized that a subgroup of cells within these tumors, identified as Glioblastoma Stem Cells (GSCs), reproduce making identical drug-resistant copies of themselves.

With an incident rate of 55,000 diagnosed cases per year, thyroid cancer is becoming the fastest-growing cancer type in the United States. Like other tumor classifications, genetic abnormalities and mutations play a key role in the proliferation of cancer cells; and although previously identified point mutations are observed in 90% of thyroid cancers, driver mutation intricacies have an opportunity for exploration. For example, the insulin-like growth factor 2 mRNA-binding protein 3’s (IGF2BP3) activation is still not fully understood.

Telomeres are regions of highly repetitive DNA at the end of a chromosome that assists in cellular duplication and rejuvenation. This physiological function has been historically associated with biological aging as the length of the telomere shortens after division and subsequently alters the effectiveness of DNA replication. However, a team of researchers at the Jackson Laboratory at the University of Texas MD Anderson Cancer Center (UTMD) have found that the length is also impactful in genetic alterations in 31 different types of metastatic tumors.

Huge strides are being made in the realm of Immuno-Oncology by a team of scientists at Sichuan University in West China. Nature reports that for the first time — as part of a clinical trial — an individual with aggressive lung cancer has been injected with their own cells engineered to express edited genes leveraging the CRISPR-Cas9 technique and targeting the PD-1 encoding gene. PD-1, which upon T-cell activation blocks the recognition of cancer cells as a foreign entity, impedes the immune responses innate ability to disrupt tumor cell proliferation. The CRISPR-Cas9 strategy combines a DNA-cutting enzyme along with molecular guide that can identify targets (such as PD-1) and replace the removed sequence with stretches of non-coding DNA.

A professor at the University of Limerick has identified a gap in the classification of a portion of the digestive system. The mesentery, which connects the intestine to the abdomen, has for hundreds of years been mistakenly considered a fragmented structure made of separate parts. However, the Professor or Surgery at UL’s Medical School, J Calvin Coffey, challenged this ideology and found that the mesentery is one continuous structure. By acknowledging this differentiation, it allows researchers to target abnormalities and diseases as well as could lead to improved health outcomes.

BioIVT understands the importance of the R&D process, as there are multiple methods that can be leveraged from concept initiation to drug delivery. In an effort to assist in the advancement in personalized medicine we would like to break down the different validation methods that can be leveraged in offering patients faster diagnoses, fewer side effects, and better outcome prior to initiating pre-clinical trials.

It is widely accepted that the presentation and course of Crohn’s disease (CD) is highly variable. A new study conducted by the University of North Carolina at Chapel Hill and Cedars-Sinai Medical Center sought to conceptualize the cellular mechanisms behind CD and characterize the processes associated with disease phenotypes (http://gut.bmj.com/content/early/2016/10/13/gutjnl-2016-312518.abstract).