Phase 1 – STREAM research

Tea Leaves: Predicting Risk and Benefit in Translation

Every early phase trial begins with a series of predictions: that a new drug will show clinical utility down to road, that risks to study volunteers will be manageable, and perhaps, that patients in trials will benefit. Make a bad prediction here, and people potentially get hurt and resources wasted. So how good a job… Continue reading Tea Leaves: Predicting Risk and Benefit in Translation

ASGCT, cont’: Results on Fetal Tissue for Battens Presented

Robert Steiner, co-principal investigator in a fetal cell transplantation study involving the rare, fatal hereditary disease Neuronal Ceroid Lipofuscinosis (also known as Batten disease), presented results of a now completed phase 1 study. According to Steiner, the study involved the highest ever dose of stem cells delivered to the human brain. The trial involved six… Continue reading ASGCT, cont’: Results on Fetal Tissue for Battens Presented

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Who says the British Press isn’t all yellow? “Doctors have begun trials using gene therapy to treat patients for cystic fibrosis.” So proclaims an April 19 story in the Guardian (“Cystic fibrosis to be treated by gene therapy technology”). “Cystic fibrosis gene cure closer,” reads a Februrary 2009 BBC headline. Details available “‘We are not… Continue reading Untitled

Basic Science and Pharmaceutical Productivity

One of the great paradoxes of contemporary medicine has been a seeming inverse relationship between investment in basic science and registration of novel new drugs by regulatory agencies. The delay between basic science discoveries and clinical applications can be very long; many promising drug candidates are called on the basis of laboratory discovery, but few… Continue reading Basic Science and Pharmaceutical Productivity

Departing Milano Stazione? ADA-SCID and Gene Transfer

Greetings after a hiatus for teaching, grants, committees, book deadlines, wiping runny noses, and more. Much has happened since my last posting, and in the next two or three weeks, I hope to catch up. First item on the agenda is a Jan 29 report in New England Journal of Medicine (NEJM) describing successful reconstitution of… Continue reading Departing Milano Stazione? ADA-SCID and Gene Transfer

Northern Lights? Canada and the New Tricouncil Draft

Since it’s issuance in 1998, Canada’s Tricouncil Policy Statement (Canada’s policy on the ethics of human research) has had an influence on the practice of research ethics that has outsized Canada’s population. The three research councils– CIHR, NSERC, and SSHRC– are presently revising the Tricouncil, and a few days ago, a revised draft was presented… Continue reading Northern Lights? Canada and the New Tricouncil Draft

Ethics, Phase 1 Trials, and the Developing World

Under what conditions is it ethical to perform phase 1, translational clinical trials in low and middle-income countries? Together with lead author Alex John London (Carnegie Mellon, Philosophy), I attempt to answer that question in the July 5 issue of Lancet (“Justice in Translation: From Bench to Bedside in the Developing World”). The article makes… Continue reading Ethics, Phase 1 Trials, and the Developing World

Are Phase 1 Volunteers Vulnerable?

In the January 2008 issue of Archives of Internal Medicine, an NIH team led by Christine Grady reports the results of a study (Participants in Phase 1 Oncology Research Trials: Are They Vulnerable?) surveying the demographics of patients who participate in phase 1 cancer studies. They report that these volunteers are overwhelmingly white, have pretty… Continue reading Are Phase 1 Volunteers Vulnerable?