Monday, December 30, 2019

Sickle Cell Disease- Therapy Success, Anti-GE Failure

While the internet's 'experts' and celebrity doctors proclaim genetic engineering to be a dangerous and unnecessary foray into 'playing God", a young woman's life has changed forever because of a revolutionary therapy.  The story appeared on the CBS News magazine 20/20 on December 29, 2019. 

At this point, Jeannelle Stephenson appears to be cured of Sickle Cell Disease, a debilitating genetic disorder that caused her immense pain and suffering. 

Ms. Stephenson suffered from "bone crushing pain" and a sedentary lifestyle because of the disease. She considered herself "middle age" in her 20's because the disease kills its victims early. She was not alone, as Sickle Cell Disease affects about 100,000 Americans.  I covered the story and modern therapies on the Talking Biotech Podcast

First her bone marrow was destroyed using chemotherapy.  At that point she could produce no more blood cells.  Then scientists introduced stem cells genetically altered with the corrected genetic information. After a transfusion of these stem cells, they started to produce the correct cell types, eliminating sickle cell disease. 

Today she leads a normal and active life. 

I could not watch it without crying.  It is so powerful to see technology end suffering and change lives. I'm so grateful to the scientists, and the technology that allows it to happen.

But I also was choked up because this should have happened years ago, and while Jennelle is a success story many are suffering today with the disease.  The vocal opponents of genetic engineering and the politically-imposed restrictions on stem cell research delayed these kinds treatments. They should have been reaching patients years ago and in wide deployment, and we should be well past celebrating a single success story.   

In this episode I saw hope for the future and contempt for those that sought, and continue to seek, restrictions on technology than can help others, simply to satisfy an empty belief or political motivation.