Clinical trials are the statistical testing of new drug molecules in human patients to determine safety and effectiveness. You frequently hear about drugs having “promising results after Phase II clinical trials” and you may not know exactly what that means.
First, to be clear, a great deal of development effort goes into a candidate molecule before it ever enters a person. There will have been extensive lab tests, tests in animal models (an animal, such as a rat, which has a condition that mimics the human disease of interest) as well as toxicity tests in animals. In general, perhaps 1 in 1000 molecules makes it all the way to phase 1 trials. In biotech, the ratio is probably higher, but that should give you an idea. And once you enter into clinical trials, the majority (perhaps the great majority) of candidate drugs fail at each phase.
A quick summary is:
- Phase I - is it safe?
- Phase II - does it work?
- Phase III - fully, statistically define the drug
Continue reading ‘What are clinical trials?’
A pretty cool development here for fans of gene therapy. The essence of gene therapy is that if a person is born with a defective copy of some gene you can insert a good copy into a critical set of cells in that person and restore the broken functionality. In this case the researchers injected therapeutic viruses into 12 patients brains and saw therapeutic benefits for parkinsons sufferers.
The reason you use viruses for gene therapy is that a virus has evolved to attach to a cell and inject it’s own genes to force the cell to make copies of the virus. It’s possible to gut the virus and insert your own genes of interest, so when the virus infects a cell, all it does is inject your genes. There’s no replication, no spreading viruses, etc. Gene therapy has had a pretty rocky history though, and not a lot of medical success.
Continue reading ‘Gene therapy for parkinsons has successful phase 1 trial’
I came across a short piece in the New York Times recently that’s worth pointing out. It’s about a particular startup in the Bay Area that’s focused on using metabolic engineering to produce anti-malarial drugs and the next item on their agenda is fuel.
For the layperson, metabolic engineering is a step beyond genetic engineering. Metabolic engineering involves creating a new network of complementary reaction pathways within a cell, essentially creating whole new ways of making biological products. In a big picture sense, metabolic engineering treats the cell as a factory, and adds or optimizes structures within the cell for some design purpose.
Continue reading ‘Metabolic engineering as path to medicine and energy’
Last night we hit a place in downtown San Francisco called Cafe Claude and had the best meal we’ve had since moving to the bay area 5 months ago. It’s a small restaurant tucked away down an alley that you probably wouldn’t notice if you didn’t know it was there.
We were attracted to the place because they were had live jazz, but the music ended up being pretty forgettable background fare. The food was universally excellent though. Service was a little snooty and french, but it wasn’t obnoxious.
I had a flat iron steak which was incredible, and it’s hard to impress me with a steak. It was perfectly medium-rare with a delicious sweet pepper sauce. My wife had a pork filet that was in an almost dessert-sweet sauce with hints of caramel. Everyone at the table commented on how delicious the sauces were on their various dishes. The chocolate mousse for dessert was great, and didn’t just feel like pudding the way a lot of mousse does. Entrees were about $30, and although portions weren’t huge the quality was great. We’ve definitely spent a lot more for much worse food so this place is definitely recommended.