Pre-Clinical and Clinical Trials

Cellular Communications

Like spectroscopy, as discussed in BPI Lab last month (1), cell signaling is not a laboratory technique but rather an area of scientific study. The environment of living cells — whether prokaryotic or eukaryotic, in vitro or in vivo — comprises not only water, nutrients, waste products, and metabolites, but also molecules released by other cells in response to intracellular events such as microbial infection and disease state or environmental factors such as temperature, osmolality, and pH. Receptor proteins on…

In Vitro Functional Testing Methods for Monoclonal Antibody Biosimilars

The pressure to contain rising healthcare costs — combined with the number of innovator biologic drugs coming off patent (30 licensed biological drugs by 2015) — offers huge opportunities for developers of biosimilar products. In 2011, the global market size of the biosimilars industry was around US$2.5 billion. Global demand for such products — and monoclonal antibody (MAb) biosimilars, specifically — is estimated to grow at 8–17% from 2012 to 2016 (1). The advent of biosimilars should bring more affordable…

T-Cell Suspension Culture in a 24-Well Microbioreactor

Cell therapy promises revolutionary new therapeutic treatments for cancer and other serious diseases and injuries. For example, T-cell therapy response rates of >50% and durable complete response rates of 20% have been reported in patients with metastatic melanoma who had failed other therapies (1). In another example, sustained remissions of up to a year were achieved among a small group of advanced chronic lymphocytic leukemia patients upon treatment with autologous T-cells expressing an anti-CD19 chimeric antigen receptor (2). Numerous other…

The Role of Medical Affairs in Moving from R&D to Commercialization

Bringing a new pharmaceutical product to market is a unique process based on a number of requirements for supporting a product launch. For a research and development (R&D) company, launching a product into market may seem to be an issue for someone else to handle in the far-distant future and at a much later time. But even at laboratory or early development stages, biotechnology companies should understand the language of pharmaceutical companies and know how that industry operates. Doing so…

Preformulation Development of a Recombinant Targeted Secretion Inhibitor

Our company carried out a preformulation study on a recombinant targeted secretion inhibitor (TSI) with contract research organization (CRO) Avacta Analytical. In this protein, the binding domain of botulinum toxin is replaced to broaden the toxin’s therapeutic potential and allow drug development to be targeted towards a specific disease. In our study, we took advantage of the high-throughput, microvolume protein analysis of Avacta’s Optim 1000 fluorescence and light-scattering instrument (which is distributed in the United States by Pall Corporation). It…

Understanding the Patient Journey

    The biopharmaceutical industry is abuzz with talk regarding a 2011 US Food and Drug Administration (FDA) guidance on human factors and the mitigation of user-based risk in the development of medical devices (1). As expected, his talk is often accompanied by a sense of anxiety. Device developers and the growing number of biomanufacturers developing combination drug–device products now need answers to usability questions they are hardly familiar with. Wrong answers may have direct (and troubling) implications from a…

The Global Cold Chain

In biopharmaceutical development, ancillary business operations such as logistics and supply chain management are frequently perceived as secondary objectives to the ultimate goal of discovering treatments and cures for devastating diseases. However, in the hypercompetitive world of drug development, forward-thinking companies have found that optimizing their clinical-trial supply chain can provide a strategic advantage to accelerating drug development. The globalization of clinical research has made the safe, punctual, and compliant transport of study drugs and other temperature-sensitive materials increasingly complex.…

A New Path in the Fight Against Melanoma

    Increased activation of cell survival signaling cascades helps tumor cells grow and makes cancer cells difficult to kill. Inhibiting proteins involved in those survival pathways is a useful strategy for selectively destroying such cells. The mitogen-activated protein kinase (MAPK) pathway is one such signaling cascade. Preclinical research finds that it is activated in most melanomas. One key kinase involved has become the focus of some targeted melanoma therapies. MAPK/ERK kinase (MEK), also known as MAPKK, has several known…

Listening to Patients’ Voices

    The role of patients in changing the pharmaceutical industry’s research agenda is evolving. Patients are greatly affected by drug R&D, with its potential to provide cures or treatments for a wide range of different medical conditions. But new product development does not always meet all patients’ needs. For example, drugs for Parkinson’s disease most often aim to treat movement disorders, whereas patients are also concerned about pain, sleep problems, lack of bowel and bladder control, and sexual dysfunction…

Advancements in Processing That Optimize Samples for Future Research

    Many factors contribute to the quality of biospecimen collections, and most are not mutually exclusive. How we assess the value of a biosample at the time of collection may be very different from at the time of analysis, which can be (and often is) an event in the distant future. To help ensure quality and create a sample resource that is not easily depleted, both novice and experienced “biobankers” can follow some general sample life-cycle management principles to…