There is hope. In addition to stem-cell transplants (which have been done for 2-3 decades), gene therapy for Krabbe is on the horizon!
You’ll have to Google gene therapy if you want a more scientific explanation, but essentially it corrects a typo. Example: Krabbe is caused by a lack of the GALC enzyme, so imagine the gene was spelled GACL – close but not correct; gene therapy takes the patient’s own cells, corrects the typo to be GALC, and puts the cells back into the body. The body then begins to produce the necessary enzyme with no risk of rejection.
It’s an incredible treatment already being used for other leukodystrophies!
THIS fact is why we continue to fight for Newborn Screening for Krabbe.
Every baby deserves a chance at life. We have the technology to detect the disease AND effectively treat it if caught at birth, and yet only SEVEN states are screening (that slide is coming).
Owen, Tygh, Ezra, Michael, Lennon…these five babies were transplanted EARLY and are thriving! We won’t stop fighting until every baby is given the same opportunity for life. After all, we would have done anything possible to save Tori, and we want every parent to have that chance.
Tori began to show symptoms at five months. Krabbe hit fast and hard, and it robbed her of her smile, and other abilities, immediately. She went to Heaven at 20 months.
But, it didn’t have to be this way. Later this month you will learn about the importance of Newborn Screening and the treatment that is possible if Krabbe is caught at birth.