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
Author: Jonathan Kimmelman
Just the FACS: Reprise on Insertional Mutagenesis
I‘ve just returned from the annual European Society of Gene and Cell Therapy meeting in Belgium. Lots of great material for upcoming posts. For now, I want to follow on the last posting on the leukemias in the X-SCID study. A warning: those lacking a stomach for science geek-talk might want to skip this posting.… Continue reading Just the FACS: Reprise on Insertional Mutagenesis
Burst Bubbles
Among the greatest traumas for gene transfer was the development of leukemias in several children participating in trials using retroviral vectors against X-linked Severe Combined Immune Deficiency (X-SCID– also known as “bubble boy syndrome”). About 20 or so children have had their immune systems fully restored by this gene transfer strategy. Tragically, however, five children… Continue reading Burst Bubbles
Keeping Alive with Hope
Hope has been a consistent theme in Barack Obama’s campaign, which thankfully came to a glorious end (many of us can now “hope” to actually get some work done after weeks of checking fivethirtyeight.com every ten minutes). His book was titled The Audacity of Hope. In the close of his victory speech, he stated “Let… Continue reading Keeping Alive with Hope
Cash Crash
Think you’re the only one stashing your financial statements in a filing cabinet without first opening them? How do you think biotechnology companies feel? Today’s New York Times ran a story by Andrew Pollack (“Broader Financial Turmoil Threatens Biotech’s Innovation and Cash”) describing the impact of the economic downturn on the biotechnology sector. Among the… Continue reading Cash Crash
The Future of Pharmaceutical Regulation
The October 2008 issue of Nature Reviews–Drug Discovery contains a very informative perspective piece on how drug regulators negotiate uncertainty, risk, and benefit when making approval decisions (“Balancing early market access to new drugs with the need for benefit/risk data: a mounting dilemma”). I have long argued that novel biologics like gene transfer will require… Continue reading The Future of Pharmaceutical Regulation
Cancer, Low and Middle-Income Countries, and Translational Research
In the October 20, 2008 issue of Journal of Clinical Oncology, oncologist Zeba Aziz describes morning rounds in a cancer ward in Lahore, Pakistan. The first patient earns $20 a month and requires a combination therapy costing $10K. In the second case, a father can only pay 15% of the treatment needed by his daughter.… Continue reading Cancer, Low and Middle-Income Countries, and Translational Research
From Bench to Ringside: The Presidential Debate
Last night, Obama and McCain confronted each other in the final Presidential debate. A flagging economy and two wars have left little room in the two campaigns for discussion of science, policy, and human research. Yet last night’s debate touched on two themes: embryonic stem cell (hES) research, and biomedical research funding. Obama accused McCain… Continue reading From Bench to Ringside: The Presidential Debate
Northward Migration?
“Since 2005, we’ve started seeing the big 20 pharma corporations making investments [in gene therapy],” says a deputy head in European Medicine Agency (EMEA) in the October 2008 issue of Nature Biotechnology (“Ark floats gene therapy’s boat, for now,” by Randy Osborne). “When you want to know what season is there and when the weather… Continue reading Northward Migration?
The Problem with Models
Chicago in plastic and balsa. If only animal models were as convincing as the one pictured above from the Museum of Science and Industry. The August 7 issue of Nature ran a fascinating feature on how many scientists are reassessing the value of animal models used in neurodegenerative preclinical research (“Standard Model,” by Jim Schnabel).… Continue reading The Problem with Models