Ophthalmology Talk and Clinical Research Class

The weather early this morning was kind of cold and snowy. It wouldn't have been a problem--the weather was beautiful a few hours later--except that I went to CCF at 6:30 AM to see the Ophthalmology Grand Rounds. I wanted to see this particular seminar because it was about setting up clinical trials for children. The speakers were from CHOP (Children's Hospital of Philadelphia at U Penn). They are working on a treatment for a particular form of genetic blindness. So far, they've been very successful with treating blind dogs, which is their animal model. Basically, there is a defective gene in these animals that can be fixed by injecting viral vectors containing good copies of the gene into the dogs' retinas. (The researchers believe that they are having success doing this because, unlike most proteins in the body, eye proteins tend to stick around more or less for the animal's entire life.) Now they want to do clinical trials on children in particular, because the animal evidence they've amassed suggests that treating adults won't ultimately work.

You may remember the 1999 fiasco at U Penn involving Jesse Gelsinger, who was one of the first humans to receive experimental genetic therapy. Since there were apparently quite a few ethical mishaps that took place and are alleged to have contributed to Gelsinger's death, U Penn now has very stringent rules in place for genetic therapy clinical trials. The fact that the researchers plan to target kids made this talk especially interesting. There are tons of extra ethical concerns to take into account when it comes to kids, since they are not legally able to consent. It was an excellent talk, well worth getting up at the crack of dawn to attend it. Plus, I have now completed my first semester of seminar attendance requirement for my MS degree.

The clinical research class was pretty helpful today. We had all been asked to make powerpoint presentations of our methods sections. I went first, and the other students, the instructor, and the statistician who helps our class sometimes made some suggestions on how to improve it. I didn't know the answers to some of the questions, so I have made an appointment for tomorrow to discuss it with the physician who is helping me deal with the clinical part. In particular, I don't really understand what's going on with the stats yet.