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Despite the appeal of personalized medicine (that is treatment selection based on the presence of a particular marker), uncertainty remains regarding the broad utility of this selection strategy in oncology. A recent meta-analysis by Jardim et al. in the Journal of the National Cancer Institute attempted to provide some clarity by comparing efficacy outcomes between personalized… Continue reading Shedding (Dim) Light on Clinical Benefit in Biomarker-Based Drug Development

Launch of clinical investigation represents a substantial escalation in commitment to a particular clinical translation trajectory; it also exposes human subjects to poorly understood interventions. Despite these high stakes, there is little to guide decision-makers on the scientific and ethical evaluation of early phase trials. In our recent article published in Medicine, Health Care and… Continue reading How do researchers decide early clinical trials?

Days after receiving the experimental medication BIA 10-2474 in a first in human trial, one man was brain dead and another five hospitalized. According to recent reports, three are likely to have neurological deficits. To my knowledge, this is the first time since 2001 that a healthy volunteer has died in a medical experiment. And… Continue reading Clinical Trial Disaster in France

Attrition in drug development – that is, the failure of drugs that show promise in animal studies to show efficacy when tested in patients- is often viewed as a source of inefficiency in drug development. Surely- some attrition is just that. However, in our recent Feature article in eLife, my long time collaborator Alex London… Continue reading Why clinical translation cannot succeed without failure

Clinicians, sponsors, ethics review committees, and others are charged with ensuring that risk is in a favourable balance with benefit when patients enrol in trials. Yet how do they make this judgment, when the only evidence available is from preclinical animal studies? In our recent article published in the Journal of Medical Ethics1, we offer… Continue reading Balancing the Evidence: Animal efficacy studies should have more weight in the risk/benefit calculus ahead of clinical trials

A novel anti-cancer drug is found to shrink every tumour type tested in experimental animal models. Let’s rejoice and start clinical trials without delay! Well, not so fast. Preclinical experiments in animal models are aimed at showing that a new drug will be useful in human beings. However, individual animal experiments are often too small… Continue reading Too much of a good thing

The new buzzword in personalized cancer medicine is “actionable mutation”. This label is given to the genetic aberrations that are present in some patients’ tumors, and that are intended targets of new drugs. Increasingly, treatment decisions in routine clinical care, and enrollment in trials are being guided by the concept of “actionable mutations”. However, determining… Continue reading Acting on “Actionable Mutations”

Most trials are funded by public sponsors, charities, or private drug developers. Austere research funding environments, and growing engagement of patient communities, has encouraged many to seek alternative funding.  One such alternative is patient funding. In the August 6 issue of Cell Stem Cell, my co-authors Alex London and Dani Wenner ask whether “patient funded… Continue reading Is it ok for patients to pay for their own clinical trials?

The task set before clinical investigators is not easy. They are supposed to answer pressing scientific questions, using very few resources, and exposing patient-subjects to as little risk as possible. In other words, we expect them to be rigorous scientists, stewards of the medical research enterprise, and guardians of their patients’ interests all at the… Continue reading Predicting Risk, Benefit, and Success in Research