Pre-Clinical and Clinical Trials

Emerging Treatments for Spinal Cord Damage

Injuries to the spinal cord can cause permanent paralysis and even lead to death, with little or no hope for patients to regain lost function after such trauma has occurred. News of my spinal cord research first came to prominence in the 1980s with a front-page story that chronicles a presentation at the annual Society for Neuroscience meeting (1). That reported the first time that crushed peripheral nerves had been regenerated back into a patient’s spinal cord. Regeneration of spinal…

Is the QbD Toolbox Ready for Cell and Gene Therapies? Integrating Patient Outcomes into Manufacturing Cell and Gene Therapy Bioproducts

In their lifecycle development and manufacturing models, biotechnology products and the biopharmaceutical industry have been founded on principles originating from the pharmaceutical small-molecule industry. Such principles define clinical programs that establish risk benefits of a dosage and its delivery system on healthy individuals and patients. A company then develops a process to manufacture that product consistently over several years. Product quality attributes set through manufacturing controls are expected to ensure patient outcomes in terms of safety and efficacy and deliver…

Partnerships in Immunotherapy for the Future of Cancer Treatment

Immunotherapy seeks to harness the power of our human immune system to fight disease. In this rapidly evolving field, collaboration among different stakeholders is essential to bringing new treatments to market. Patient advocacy groups, researchers, hospitals, manufacturers, and government entities all are working together to translate promising new research into life-saving products. Types of immunotherapy include monoclonal antibodies (MAbs) and antibody derivatives, checkpoint inhibitors (immune-modulating proteins), cancer vaccines, T-cell therapies, and cytokines — so the approach involves a range of…

Elucidation: Better Biomarkers Could Power Precision Medicine for Autoimmune Disease

Precision medicine has long been a tantalizing goal for the pharmaceutical and healthcare industries. Fields such as oncology and rare diseases have benefited greatly from implementation of genome-guided care. Drug developers likewise have made strides with biomarker-aided discovery programs and stratification for clinical trials. Current indications suggest that autoimmune diseases will be the next major focus of personalized medicine. The goal of precision medicine often is described as “getting the right drug to the right patient at the right time.”…

Your Brand Is the Patient’s Experience

The future success of biopharmaceutical businesses will depend at least partly on their ability to create meaningful brand experiences from the start of a drug program. By “brand,” I don’t mean logos and taglines. I’m talking about meaningfully unique experiences that directly affect clinical and patient needs — specifically, to address the growing demand for self-administered injectable therapeutics. Whether you are a biosimilar developer trying to carve out differentiated value or a market leader looking at your patent protection in…

Reverse Translational Medicine: The Promising Future of Failure

The drug development landscape is awash with candidates that have shown enormous promise and efficacy in preclinical models but failed when administered to clinical trial subjects. Although such failures occur for different reasons, one of the most pervasive causes is the inability of preclinical models to recapitulate human physiology accurately. Despite advances with both in vitro and in vivo models, improving those toward a more accurate avatar of the human physiological process remains a challenge. Central to that effort will…

Polysorbates, Biotherapeutics, and Anaphylaxis: A Review

Rapidly increasing use of monoclonal antibodies (MAbs) in the treatment of neoplastic, autoimmune, and inflammatory diseases has led to a dramatic increase in hypersensitivity reactions worldwide, complicating the use of MAbs as first-line therapies and limiting patient survival and quality of life (1). The origins of anaphylaxis are not well understood, though its mechanism is fairly straightforward (Figure 1). It is usually attributed to some undefined intrinsic property or properties of a biotherapeutic — despite the fact that biotherapeutic formulations…

The Clinical Side of Biosimilar Development

Biosimilars have become common on pharmacy shelves in Europe. The first biosimilar product — Sandoz’s Omnitrope version of Lilly’s Humatrope (somatropin) — was approved by the European Medicines Agency (EMA) in 2006. In the decade that followed, more than 20 biosimilars have gained regulatory approval in Europe. The first biosimilar monoclonal antibodies (MAbs) — comparators to Janssen’s Remicade (infliximab) — were approved in 2013. The pace of approvals in the United States has been much slower. The US Food and…

Driving Therapeutic Innovations: Academic Institutions Can Help Lessen Development Risks

During the Biotech Week in Boston this past October, I had a chance to talk with David DiGiusto (Stanford University) about his work toward advancing bioprocessing and cell therapy development. I asked him to comment on points from his keynote presentation about how academic research groups can sustainably cycle assets into the biopharmaceutical pipeline. University research departments have long made innovative technologies available for commercial licensing. But in the excerpt below, he details ways in which such groups are further…

Progress Toward Commercial Scale and Efficiency in Cell Therapy Bioprocessing

Regenerative medicine includes both cell and gene therapies. Currently 672 regenerative medicine companies operate around the world, and 20 products have been approved by the US Food and Drug Administration (FDA). Of 631 ongoing clinical trials by the end of 2015 (1), over 40% are in oncology, followed in prominence by cardiovascular and infectious diseases. Here I focus on gene and cell therapy bioprocessing in which the final products delivered to patients are cells. Cell therapies are either autologous (derived…