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
Author: Jonathan Kimmelman
Yellow Light on Gene Transfer Studies
Among the greatest heartbreaks in the field of gene transfer have been problems encountered in trials involving a rare, hereditary immune disorder, X-SCID (known popularly as “Bubble Boy” syndrome). As is well known, a team of researchers based in Paris– and then in London– successfully reversed severe immunodeficiencies in 20 or so children using retroviral… Continue reading Yellow Light on Gene Transfer Studies
Toxic Waste?
Before testing new drugs in human beings, drug developers must first perform a series of safety tests in animals. Unfortunately, these preclinical toxicology studies are typically protected as trade secrets. In fact, many countries have laws that specifically bar drug regulators from releasing preclinical toxicology data submitted by drug developers. Unless you take the extreme… Continue reading Toxic Waste?
The Biotechnology of Neglect
What can the biotechnology industry do for neglected diseases (infectious diseases that, because they primarily afflict low-income countries, are not heavily researched in the public or private sector)? Biotechnology enthusiasts might answer “everything,” ignoring the dearth of research and development incentives for small biotechnology firms, much less the major challenges presented by the manufacture, distribution,… Continue reading The Biotechnology of Neglect
Guinea Pig Nation?
Here is an irresistible news headline: “Public Policy That Makes Test Subjects of Us All.”(New York Times, April 6, 2009). Then you open to the story only to discover, to your disappointment, that the piece is written by John Tierney, probably the most uninformed and underqualified members of the NYTimes staff. His argument is ridiculous:… Continue reading Guinea Pig Nation?
Red Handed: An Independent Review of IRBs
A decade or so ago, the Government Accounting Office published a series of reports faulting human protections at VA hospitals, raising concerns about HHS oversight of human research, and urging continued vigilance in human research. After a seeming pendulum swing toward protections bashing (witness the outpouring of condemnation after OHRP sanctioned a team of researchers… Continue reading Red Handed: An Independent Review of IRBs
Centralized Revue
In the most recent issue of Molecular Therapy, U Penn researcher Hildegrund Ertl provides a strong and eloquent defense of the Recombinant DNA Advisory Committee (RAC). RAC was initially formed to evaluate the safety of studies involving recombinant DNA. In the last decade, however, its most visible function has been to provide advise to researchers… Continue reading Centralized Revue
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
Found Figures: Picking up the Pieces after an HIV Vaccine Trial Fails
In the November 29, 2008 issue of Lancet, two reports (plus a commentary) report the famously disappointing outcome of a recent placebo-controlled study testing adenoviral vector-based vaccines against HIV. News reports over a year ago reported that the study was halted after an interim analysis failed to show any prospect of proving effective. More troubling,… Continue reading Found Figures: Picking up the Pieces after an HIV Vaccine Trial Fails
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?