Article published yesterday at The Journal of Clinical Investigation (where Dr. Orange published the NEMO article about Andy) from Dr. Alain Fischer regarding gene therapy.
I called Dr. Fischer once about 5 years ago and he told me he was not ready to use gene therapy to treat children with NEMO.
In this article he mentions NEMO:
Within the past 10 years, spontaneous, partial corrections of the phenotype of severe T cell immunodeficiencies (e.g., ADA deficiency, SCID-X1, Wiskott-Aldrich syndrome, RAG1 deficiency, CD3 deficiency, and NF-κb essential modulator (NEMO) deficiency syndrome) have been reported
And then he says,
These results constitute a strong rationale for the development of a gene therapy approach that recapitulates these rare spontaneous events.
His trials:
Between 1999 and 2002, 10 children with SCID (Severe Combined Immune Deficiency) under the age of 1 were enrolled into a gene therapy clinical trial with Dr. Fischer.
9 children developed corrected T and NK lymphocytes
7 of the 9 developed T cells and their counts were normal within 3 months and are still normal now.
2 of the 9 experienced partial T cell reconstitution.
Now, 7.5 years later, these 7 patients retain a functional immune system, enabling them to live normally.
There is a new trial and 20 children enrolled.
17 out of the 20 have been successfully treated in London and Paris.
WOW
Find the article here.
