Entries in drug development (2)


The Emergence of Patient-centered outcomes in guiding clinical care

Last week, I had the opportunity to attend an educational forum offered by MassBio entitled “Communicating Clinical Benefit: The Role of Patient-Centered Measures” that opened my eyes to the importance of patient-reported outcomes (PRO) in improving clinical care. For those who are unfamiliar, PRO research aims to develop assessment tools that can be used to capture a patient’s self-reporting of health and quality of life. The assessment tools are often in the form of a questionnaire or interview asking patients directly about their symptoms, functioning, and overall mental state as they pertain to disease and/or treatment. These data are different from physiological measurements and investigator-reported measurements, which objectively measure and observe the patient’s biology. By creating standardized, reproducible methods to record reports from patients, these tools permit a more objective way to characterize the patients’ experience. Patient-centered tests obviously require thorough validation before FDA approval for use, as potential primary or secondary endpoints in clinical trials.

The compiled outcome data from these studies provides relevant guidance for patients in evaluating their care options and understanding the treatment-associated risks. In addition, these data offer insight for payers as to which clinical benefits are most meaningful to the patient, which in turn can help assign a monetary value to a particular therapy. The information generated in such studies may also be used to support labeling on the drug package insert and marketing indications for a product. Additionally, drug companies can use these data to confirm their product is fulfilling their patients’ needs early on in clinical development.

Growing interest in measuring patient-centered outcomes reflects a shift away from medicine’s former disease-centric approach. As the field of genomics uncovers an array of genetic and epigenetic influencers, diseases have become increasingly complex, easing the path for personalized medicine to emerge as a more direct way to help individuals solve health problems. While the need for research to identify biological mechanisms and markers of disease and therapeutic efficacy remains critical, it is exciting to learn about the research that helps to connect the biology to clinical outcomes important to the patient.



Nanoparticle antioxidants offer potential Alzheimer’s therapy

Mitochondria have been known as a cell’s power plants. The abnormal generation of reactive oxygen species (ROS) from dysfunctioning mitochondria can cause neuronal cell death. This pathologic process is a key factor to a number of neurodegenerative diseases, including Alzheimer’s disease (AD). Amyloid-β peptides, which are believed to cause AD, can interact with resident proteins inside mitochondria, inducing abnormal production of ROS. Thus, ROS scavengers, such as antioxidant molecules, targeting mitochondria would be useful for prevention and early state treatment of AD.

A research team lead by Taeghwan Hyeon designed and synthesized triphenylphosphonium-conjugated ceria (CeO2) nanoparticles (TPP-ceria NPs), which can selectively localize in mitochondria and behave as strong ROS scavengers. These nanoparticles function in a recyclable manner by shuttling between Ce(III) and Ce(IV) oxidation states. The study of an AD mouse model indicates that the nanoparticles effectively suppress neuronal cell death by protecting them from ROS. This research has paved the road for the development of novel strategy to prevent and treat AD and other neurodegenerative diseases. 



ACS Nano 2016, 10, 2860−2870.