Stem Cell Therapy for Fabry Disease
Using our STEM technology (Stem cells engineered into micropharmacies), 5 patients were treated with cell therapy for Fabry and followed for 5 years. These genetically-modified cells were able to produce significant quantities of therapeutic enzyme which lead to the reduction in toxic metabolites in patients by more than 50%.
When patients are removed from ERT (enzyme replacement therapy - the current standard of care), they see a quick jump in toxic metabolites by 44%. When they are put on cell therapy (CT), they see a drop in toxic metabolites that exceeds the levels they had when they were on ERT (more than 50%). This drop occur either with a patient administered only CT (down 66%) or in the remaining CT+ERT treated patients (average 80%). These are results indicate the Fabry CT treatment is at least as effective as ERT for controlling toxic metabolite levels.
From a publication on the biomarkers for the CT trial, we extracted the above data for lyso-Gb3 metabolite. (https://www.oaepublish.com/articles/rdodj.2024.14). Each patient has the same trend - at one month from start of study, while the patients remained on ERT, their toxic lyso-Gb3 metabolite held steady. When the patients were taken off the ERT and measured one month later (No ERT), their lyso-Gb3 levels had increased. After the patients received the gene-engineered cell therapy (CT Month 3-12), they saw a significant drop in the toxic metabolite. When the effectiveness was measured years later, all but one patient were showing at least a 5 year durability.
On average, patients see an increase in toxic metabolites with removal of ERT administration (up 44%). When cell therapy is provided with or without ERT, their toxic metabolites drop (down 77%). The effect on suppressing toxic metabolites is durable out to 5 years for 4 out of 5 patients. For more information, see the 5 year followup publication (PMID 39794302).
