Author: Jonathan Kimmelman

Targeted Cancer Drugs: The "Price of Progress"?

So here is the party line on the newest generation of cancer drugs.  Unlike older generation drugs, which are generalized poisons, newer cancer drugs hone in on very specific molecular targets.  Because of this specificity, they have fewer “off-target” effects, and hence fewer side effects. In the current issue of Journal of Clinical Oncology, Niraula… Continue reading Targeted Cancer Drugs: The "Price of Progress"?

Registration of Trials: A Census

Apologies to the millions of avid followers of Lost in Translation for the long haitus.  In response to an international petition campaign, with several Nobelist signatories, I am cautiously restarting this blog with the aim of (monthly??) blogposts on troubles and turmoil in clinical translation. We lead off with an article in this week’s JAMA,… Continue reading Registration of Trials: A Census

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

Dirty Windows of Drug Development

Think of clinical trial data as a window on the efficacy and safety of a drug. Think of data protection and trade secrecy as soot. The above picture? This is the public view on drug safety and efficacy. According to a recent report in Nature Biotechnology (Feb 2011), medicine may be getting some soapy water… Continue reading Dirty Windows of Drug Development

More Gray Matter: Parkinson’s Disease and Gene Transfer

Several groups are pursuing gene transfer strategies against Parkinson’s disease. No small task, because for these approaches to work, investigators have to deliver vectors deep inside the brain using surgery. I have previously written that early phase studies using surgical delivery press the boundaries of acceptable risk, because patients can generally manage their disease adequately-… Continue reading More Gray Matter: Parkinson’s Disease and Gene Transfer

Icarus, again: Adversity in another Gene Transfer Trial

Two weeks ago brought good news and bad news for gene transfer. First the good news. New England Journal of Medicine beatified a new gene transfer strategy for Wiskott-Aldrich Syndrome (WAS). WAS is a primary immunodeficiency that primarily affects boys. It is thus in the same family of disorders that have been, in varying degrees,… Continue reading Icarus, again: Adversity in another Gene Transfer Trial

Are Trials Necessary?

Today’s New York Times ran a heartbreaking story by Amy Harmon about two cousins who developed melanoma. One was entered into a cancer clinical trial and received the investigational drug PLX4032. The other was ineligible for the trial, and therefore unable to access the experimental drug. Guess which cousin died? The article is one in… Continue reading Are Trials Necessary?

Embryonic Stem Cell Trials Start Development

So, FDA has lifted a hold on the first ever clinical trial testing cells derived from human embyonic stem cells. The study- based in California and sponsored by the biotechnology company Geron (view press release here)- will administer cells derived from human embryos (“neural support cells”) to ten patients with recent spinal cord injury with… Continue reading Embryonic Stem Cell Trials Start Development

Information: Stem Cell Tourism Redux (part 1)

The current issue of Kennedy Institute of Ethics Journal contains the first installment in a two part series on the ethics of stem cell tourism, by long time stem cell watcher Cynthia Cohen and Peter Cohen. The Cohens pull together a large body of news reports and internet posts on Russian and Indian private clinics… Continue reading Information: Stem Cell Tourism Redux (part 1)