As Jonathan is fond of saying: Drugs are poisons. It is only through an arduous process of testing and refinement that a drug is eventually transformed into a therapy. Much of this transformative work falls to the early phases of clinical testing. In early phase studies, researchers are looking to identify the optimal values for the various… Continue reading The Landscape of Early Phase Research
Tag: first in human trials
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
Conditions of Collaboration: Protecting the Integrity of the Scientific Enterprise
So what does it take to keep medical research a well-oiled enterprise that efficiently and effectively delivers cures? Lots of cooperation–or so I argue, along with co-authors Alex John London and Marina Emborg in a piece appearing in Science [a publicly accessible version of the essay is available at Science Progress]. Unfortunately, we argue, the… Continue reading Conditions of Collaboration: Protecting the Integrity of the Scientific Enterprise
California Dreamin: CIRM Announces New Stem Cell Awards
California’s Institute for Regenerative Medicine just announced a series of large funding awards to fund translational research initiatives involving (mostly) stem cells. The projects funded are telling with respect to what was funded, and what they will attempt to achieve. First, notwithstanding a press release containing the words “bringing stem cell therapies to the clinic,”… Continue reading California Dreamin: CIRM Announces New Stem Cell Awards
Mice- Three Different Ones: Towards More Robust Preclinical Experiments
One of the most exciting and intellectually compelling talks thus far at the American Society of Gene Therapy meeting was Pedro Lowenstein’s. A preclinical researcher who works on gene transfer approaches to brain malignancies (among other things), Lowenstein asked the question: why do so many gene transfer interventions that look promising in the laboratory fail… Continue reading Mice- Three Different Ones: Towards More Robust Preclinical Experiments
Prime Time for Embryonic Stem Cells?
According to a recent report in the Washington Post, researchers at Geron have received approval from FDA to initiate the first ever human trial involving stem cells derived from human embryos. A story in the most recent issue of Nature provides more background. Briefly, the study will involve transplanting tissues derived from human embryonic stem… Continue reading Prime Time for Embryonic Stem Cells?
Stems and Blossoms (part 1): Justice
Shortly before I left for holiday, the International Society for Stem Cell Research (ISSCR) issued a policy paper, “Guidelines for the Clinical Translation of Stem Cells,” outlining ethical and scientific considerations for researchers designing translational trials involving stem cells (whether stem cell derived, adult, or embryonic). In my opinion, the document wins the award for… Continue reading Stems and Blossoms (part 1): Justice
In Brugge: The Cure
One of the most striking themes at the European Society of Gene and Cell Therapy was the extent to which continental European researchers conceptualize first-in-human gene transfer experiments as therapeutic interventions rather than research protocols. Perhaps the most extreme and explicit expression of this was view was presented by Bonn internest Thomas Heinemann (he also… Continue reading In Brugge: The Cure