A retrovirus called VRX496 has demonstrated that gene therapy has the potential to treat HIV and other serious human diseases. VRX496 is a genetically altered AIDS retrovirus that impairs HIV viral replication. It’s sort of like a Trojan Horse, but instead of carrying soldiers it carries genetic material that inhibits HIV replication.
Five patients with advanced AIDS experienced a decrease in viral load and an increase in white blood cells (the cells that fight off the bad things like bacterial and fungal infections) after being treated with VRX496. All of these patients had been unresponsive to at least two antiretroviral treatments. Normally white blood cells never go up in AIDS patients, but steadily decline year after year.
This phase I trial is very encouraging, but there’s still a lot of work to do. The phase II trial has started using AIDS patients whose virus is under control with the use of antiviral treatments. We’re looking at maybe another 20-25 years before we actually see VRX496 being used world wide to treat AIDS. Meanwhile 2.5 million people worldwide have died from this disease.
Let’s hurry things up people! Time’s a wastin’.
So what’s the hold-up? Do researchers just need more time for study and trial? Or is it a political/economic issue?
It’s a little of both. The FDA requires drug developers to file an Investigational New Drug application 30 days before begining clinical trials. The FDA can also put a “clinical hold” on something if they think there’s a problem. This could keep a drug in clinical trials for years and that’s just the begining. There’s a huge manual for clinical trials http://www.fda.gov/cder/about/smallbiz/CFR.htm and researchers have to be sure to dot every I and cross every T.
Also, the researcher has a huge responsiblity to the public. Lets say that 10 years down the road VRX496 mutates and no longer impairs HIV replication, but expediates it or even worse, turns HIV into an airborn pathogen. These are all things that they have to consider before they let everyone get VRX496.
Your field of study is so interesting. THe worst that’s going to happen if Limon and I screw up something is that 10 years down the road people are going to continue being idiots.
Very true, T. And, in fact, my students are all high-level professionals: the worst that will happen is they’ll get a promotion and, *gasp*, not deserve it. We need to go be Cindy’s assistants.