We don’t yet know when NIH will be ready to start the next step for Gene Therapy for Ray. We hope to hear a plan soon. The waiting is hard. We have trouble deciding what we can plan to do and what we cannot. I know Ray is really anxious to get things moving.

Several people have asked us for more information on Gene Therapy. A few of you (especially the SCID families following this blog) may appreciate reading about the more technical details. Attached to this post is a PDF of an article published in April of 2016 at sciencemag.org, titled; Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency. The article gives the data on the 1st 5 patients in the trial, with the 1st patient 36 months post Gene Therapy. The results reported in this article are quite impressive. Below is a short section from the introduction.

Follow-up data from two older patients demonstrate that lentiviral vector gc transduced autologous HSC gene therapy after nonmyeloablative busulfan conditioning achieves selective expansion of gene-marked T, NK, and B cells, which is associated with sustained restoration of humoral responses to immunization and clinical improvement at 2 to 3 years after treatment. Similar gene marking levels have been achieved in three younger patients, albeit with only 6 to 9 months of follow-up. Lentiviral gene therapy with reduced-intensity conditioning appears safe and can restore humoral immune function to posthaploidentical transplant older patients with SCID-X1.

Click to download: XSCID Gene Therapy Article