St. Jude Symposium and Gene Therapy

June 13th and 14th, 2016 I was lucky enough to be invited to attend a SCID Symposium at St. Jude’s Research Hospital in Memphis, TN. Heather Smith and Mickey Morgan were also there. Looking back it was quite fortunate that I was able to attend. My mindset for the trip was set on learning about the newest therapies available in order to share the information with SCID families. I didn’t realize how important the information was going to become for my family.

We’ve watched the progress of gene therapy in XSCID for years. In fact, we had already collected stem cells from Ray back in 2001, during preliminary discussions on possibly utilizing gene therapy for Ray. However, it was during 2001 that problems arose in the early gene therapy trials. I knew the family from the French trials in 2000 whose son ultimately passed away after receiving gene therapy. His mom had shared his incredible treatment breakthrough with those of us in the SCID group and we were all devastated when we learned that he’d developed a form of leukemia and later learned that he did not survive.

A lot of time and research has gone into the study of gene therapy since then. Different vectors are used now and it’s been shown that the prospects are more successful if room is made in the marrow by using an ablative therapy such as busulfan. Most of those who’ve already gone through gene therapy have seen noticeable improvement in immune status and nutritional status, as well as clearing on board chronic infections. We’re hoping to see similar positive results.