UT Southwestern creates customized treatments for rare diseases

One in 10 individuals in America – most of them children – is afflicted with a rare disease. UT Southwestern researchers are on a quest to address that sobering reality by creating safe viruses to deliver customized treatments to halt and sometimes reverse rare conditions.

Transcript

Steven Gray, Ph.D./Associate Professor, Pediatrics

This is a clean room facility and what that means is that this is basically a sealed facility. The air in here is very controlled.

Everything starts basically here where when we make the virus, we manufacture it in cells that are grown in culture in kind of a nutritive cool aid.

These cells are kept basically in this liquid nitrogen tank…You would pull out racks and get a vial of these cells out that’s going to be a small container about the size of your finger and then put that on a cold block to keep them frozen and then those will need to be carried into the facility.

If you’re just transferring material, then you can have one person on one side. One person on the other.

So we’re moving from basically that prep room into this is one of two rooms that are essentially the main production rooms.

Open up the tubes underneath in here and then move those cells into a small reactor about the size of a quart.

And really the next two weeks of production are just going from this small about a teaspoon of cells up to about 125 gallons.  And so, it’s just growing those cells. It’s keeping them happy and then they divide and replicate. Divide and replicate.

Then we’re giving those cells basically pieces of DNA that give them instructions to become factories for the virus, and each cell can make about 100,000 viruses.

And then that gets pumped through the wall and then there’s a whole other series of equipment in the other room that’s going to concentrate that 125 gallons down and purify the virus away from all the cell debris and everything else we want to get rid of until in the end, we’ve gone from 125 gallons down to about 1 cup of purified virus.

The doses that we’re doing, one cup would be enough to treat about 10 patients. Each patient would get about 2 teaspoons of virus solution and in that 2 teaspoons is about 1 quadrillion engineered virus so that’s a thousand times a trillion so it’s really a concentrated solution of virus to be able to deliver the genes to as many cells as we possibly can.  

And if everything goes right, then we’ll be making virus in here in the spring to try and help some kids.