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
Author: Jonathan Kimmelman
ASGCT, continued: Eyes on Stage
Predictably, the big presidential symposium at ASGCT reserved a slot for Jean Bennett, who led one of the three teams that have tested a gene transfer strategy for a rare genetic form of blindness, Leber’s Congenital Amaurosis (LCA). Unpredictably, however, Bennett trotted out one of her “treated” patients, Cory Haas, along with his two parents,… Continue reading ASGCT, continued: Eyes on Stage
ASGCT in Washington DC
Another year, another annual meeting of the American Society of Gene Therapy- now rechristened American Society of Gene AND CELL Therapy. The meeting ends today, and I am way behind in posts. There have been, to my knowledge, no startling new revelations about high impact trials or disastrous adverse events. The studies of Leber’s Congenital… Continue reading ASGCT in Washington DC
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
Filing Cabinet Syndrome: The Effect of Nonpublication of Preclinical Research
Much has already been said about Filing Cabinet syndrome in medical research: the tendency of researchers to publish exciting results from clinical trials, and to stash null or negative findings safely away from public view in a filing cabinet. Nonpublication distorts the medical literature, because it prevents medical practitioners from accessing negative information about drugs.… Continue reading Filing Cabinet Syndrome: The Effect of Nonpublication of Preclinical Research
CAR Accidents: Unexpected and Serious Toxicity in Gene Transfer Immunotherapy
This month’s issue of Molecular Therapy– the premium journal covering developments in gene transfer- reports two deaths in recent cancer gene transfer studies. Both studies involved a similar anti-cancer strategy, in which a patient’s T cells are genetically modified to mount a strong and sudden immune attack against the patient’s cancer (the particular genetic modification… Continue reading CAR Accidents: Unexpected and Serious Toxicity in Gene Transfer Immunotherapy
Testing Testing…: Personal Medicine, Breast Cancer, and Policy
Personalized medicine is supposed to usher an era in which treatments are tailored to individuals. And HER2 testing has long been seen as heralding the promise of personalized medicine: tumors that test positive for an amplified HER2 gene are more likely to be responsive to drugs, like trastuzumab, that block the HER2 receptor. Some may… Continue reading Testing Testing…: Personal Medicine, Breast Cancer, and Policy
Teaching Kills Blogging: Somewhat Recent Developments…
Dear Faithful Readers: Teaching has cut my blogging to a trickle, though the teaching has now begun to taper off. My silence is not for want of major developments in the last two months. Among a few highlights: • Obama picks members for his Bioethics advisory panel: White house recently announced membership of its “Presidential… Continue reading Teaching Kills Blogging: Somewhat Recent Developments…
Ark, Troubled Waters, and Rainbows for Gene Transfer
This morning I awoke to a news report by National Public Radio’s Joe Palca on promising developments in gene transfer. In it, Palca provided a good account of the field’s travails, as well as some encouraging developments in the last few years. The story ended with the prediction that the coming “months and years” would… Continue reading Ark, Troubled Waters, and Rainbows for Gene Transfer
Canada Human Research Ethics Policies: Take 2
In the Vancouver Olympics, Canada is tied for the most gold medals as of this writing. Will Canada also “own the podium” when it comes to providing a clear and effective voice for ethical human research? Recall that, in a previous post, I mentioned that Canada was presently undertaking a major overhaul of its main… Continue reading Canada Human Research Ethics Policies: Take 2