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Decades of dedication lead to drug trial for rare, fatal illness

December 11, 2019


So RVCL is a disease that begins in middle-age. The disease then is a relentless and progressive dropout of small blood vessels throughout the body. And then these patients get
progressively more debilitated And lose mental function, lose physical function and become blind. So it’s a progressive relentless disease that leads into death in about five to 10 years. How’s it going? Good to see you. How are you? Kim was one of the original three
patients in the trial. And we follow her now for a number of years. We know how many lesions she has in her eye. We know how many lesions she has in her brain, where they are. And so what we can do then is start her treatment. And we infuse over a five-day period the drug. It’s a chemo therapeutic agent. A cousin of it is used to treat acute leukemia. But here we don’t want to kill the cells. What we want to do is make the cells work better. We’re giving them just enough we hope that will correct or partially correct the defect. We’ve completed the first six months on three patients. The good news is you’re overall stable and in addition the second good news is the toxicity has been minimal if any. We are hoping to stop the disease from progressing. The long-term goal would be using modern genetics to go in and actually correct
the defect in these patients. Go in where we get rid of the mutated gene. That will happen I think in the future, but we’re not there yet. So right now we’ve got to hope that we can prevent it with in-vitro fertilization. And the patients who have it, we’re hoping this drug will slow things down.

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