Accelerating Rare Disease and Orphan Drug Development: Opportunities for Biomarkers, Diagnostics & Patient Engagement

Rare diseases affect more than 350 million people worldwide but patients often face limited options for approved therapies. As a result, many patients have joined advocacy groups first and foremost to connect with others struggling with their rare disease, but also to promote research, unite multiple stakeholders and stimulate new possibilities in the therapeutic pipeline. Research and orphan drug development efforts are starting to follow suit by increasingly incorporating patients’ needs and examining potential outcomes.

Capitalizing on Global Drug Development Opportunities from China

The Chinese pharmaceutical market continues to grow steadily, but drug developers in China face similar challenges as their global counterparts: Development times are increasing and success rates are declining. Xiaoning Guo, PhD, PMP, clinical development program director of our Asia Pacific services, recently discussed potential strategies to improve investment returns and accelerate global development.

What Are the Emerging Noninvasive Biomarkers in NASH?

Novel biomarkers represent a promising means to improve diagnosis of nonalcoholic steatohepatitis (NASH). Currently, a definitive diagnosis requires a liver biopsy, a surgical procedure with many limitations. There are a variety of biomarkers that can assess liver status, but they do not always distinguish between patients with NASH and those with other disorders. Advanced imaging techniques, while useful for evaluating some liver features, can be impractical and costly.

Leveraging the Clinical Power of MRI in Preclinical Liver Tumor Imaging

The liver is an ideal organ for magnetic resonance imaging (MRI) due to its size, plus the inherent contrast it enables compared to other tissues and lesions; this results from the high iron content which acts as a natural MRI contrast agent. For such reasons, liver tumor MRI is a major area of clinical and preclinical oncology imaging. We have been performing liver tumor MRI for over a decade to evaluate mouse syngeneic intrasplenic liver metastasis models, orthotopic human tumor xenografts (PDXs), and GEM models. Liver tumor MRI facilitates use of models with more clinically relevant context, enabling:

Endotoxin inhibition and enhancement: Critical considerations for medical device testing

A known structural component of gram-negative bacteria, endotoxins can elicit strong inflammatory responses and fever if they enter the human bloodstream. Because endotoxins can leach into the body from contaminated medical devices, rigorous endotoxin testing is crucial for patient safety and an essential prerequisite for marketing new medical devices.

Use of liquid biopsy analysis to identify biomarkers related to solid tumors: Insights from a recent Japan symposium (Part 2)

Liquid biopsy analysis offers a promising, minimally invasive approach to detecting and monitoring cancer. It involves the analysis of tumor components like DNA, cells and exosomes found in body fluids. Unlike traditional tissue biopsies, liquid biopsies allow for repeated sampling to track tumor evolution and response to therapy in real time. In his presentation at the recent Japan Symposium 2023, Dr. Sriram Ramanan, associate director of oncology biomarkers at Labcorp’s Biomarker Solution Center, discusses the advantages and disadvantages of using liquid biopsy analysis to identify biomarkers related to solid tumors.

Multiple myeloma: Empowering the frontline – confronting a deceptive cancer affecting vulnerable communities

Multiple Myeloma: Empowering the Frontline – Confronting a Deceptive Cancer Affecting Vulnerable Communities

Longitudinal Evaluation of Orthotopic Lung Tumors With Imaging

Lung cancer is the most common cause of cancer related deaths for men and second for women (after breast cancer). Worldwide, 1.8 million diagnoses and 1.6 million deaths occur annually.1 While incidences have been decreasing over recent decades and advances in diagnosis and treatments are being made in cancer as a whole, only 17 percent of patients diagnosed with lung cancer will survive five years leaving an opportunity for new therapies to make a large impact on this population of patients. The combination of surgery, traditional chemotherapies, targeted therapies, radiation, and immunotherapy have been shown to be beneficial treatment options; and development of novel therapies and/or combinations continue.