Last week, I blogged on the issue of generic biologics: should companies that make vaccines, monoclonal antibodies, cell therapies, etc. get 12 years of data exclusivity before competing companies begin offering generics? Or should they be held to the same standard as makers of drugs, who get five years of exclusivity? Looks like the U.S.… Continue reading Orange Light on Generics
Author: Jonathan Kimmelman
Phased by Phase 0?
Today, Lancet ran an editorial asking whether Phase 0 trials will become a “platform for drug development.” The editorial responds to the first published ‘phase 0’ study, this June, in Journal of Clinical Oncology (Kummar et al). These studies involve delivering very small quantities of a new drug to test its properties before giving biologically… Continue reading Phased by Phase 0?
Generic Biologics
Gene transfer, cell transplantation, monoclonal antibodies, enzyme replacements, tissue engineering all have great potential to improve health care. But will we be able to afford them? Various economists have shown that a large proportion of health care cost inflation is attributable to new technology, and the significant costs of development and production for drugs involving… Continue reading Generic Biologics
Safe Harbor? Leukemia, Gene Transfer, and Lentiviral Vectors
A few further observations from the American Society of Gene Therapy Meeting… A recurrent theme in this blog is the frequency with which novel research fields encounter safety problems that confound laboratory predictions. One presentation at the 2009 ASGT meeting brought this point home. Recall my entry on May 12 discussing various refinements to retroviral… Continue reading Safe Harbor? Leukemia, Gene Transfer, and Lentiviral Vectors
The Vision Thing: Update on LCA
Last year’s “big ticket” item at ASGT was results from the first three patients in two gene transfer trials testing nearly identical products against a rare form of congenital blindness, Leber’s Congenital Amaurosis (LCA). I previously blogged (here and here and here and elsewhere) on the controversial decision to move the intervention into children, given the novelty… Continue reading The Vision Thing: Update on LCA
Clonal Trouble
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
ASGT in San Diego
This year’s annual meeting of the American Society of Gene Therapy is in San Diego. I’ve been to several interesting talks thus far, and plan to post entries on a few. For now, here’s an overview of some major (or some not so major) clinical developments in gene transfer that are being reported at this… Continue reading ASGT in San Diego
A Cure? "Compassionate Use" and Drug Regulation
Are government bureaucrats keeping dying patients from getting access to possibly life saving drugs? That’s one way to read Margaret Talbot’s story in the Sunday New York Times (“Fighting for a Last Chance at Life.” May 17, 2009). Talbot describes how mother Kathy Thompson sought access to an unlicensed therapy Iplex for her Amyotrophic Lateral Sclerosis… Continue reading A Cure? "Compassionate Use" and Drug Regulation
Untitled
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