New Fabry disease therapy shows 130x increase in effectiveness.
Study by a Canadian researchers uncovers a new therapy with 130x increase in length of effectiveness. Durability of cell-based gene therapy last at least 5 years in most patients tested in clinical study.
Problem
Patients with Fabry disease suffer from build up of toxic lipid metabolites, because they are missing a key enzyme, alpha-galactosidase A. Without this enzyme, patients are fated to have kidney failure followed by heart failure. Current standard of care is enzyme replacement therapy (ERT). ERT is sufficiently effective to control the levels of toxic metabolites, however it complicates the lives of patients because they must construct their lifestyle around tedious clinical visits, every other week, for the rest of their lives.
Solution
A recently published study by a Canadian group offers a promising new therapy that will change the treatment regime to a less frequent dosing (130x reduced - article link: https://pmc.ncbi.nlm.nih.gov/articles/PMC11726700/). The group used a cell-based gene therapy to deliver continuous secretion of enzyme in leukocyte populations. Because of the systemic reach of leukocytes, the missing enzyme becomes availability to all sensitive tissues.
Result Detail
In their study, the team observed the levels of the lyso-Gb3 toxic lipid metabolites was initially held steady for all patients because they had been on ERT. When they were taken off ERT, their toxic metabolites increased by 41%. Then when the patients were given the cell-based gene therapy (GT-GLA-S03), the level of toxic metabolites dropped 48% to a level that was lower than the starting place on ERT. When they measured the patients 5 years later, 4 of 5 patients were still producing sufficient plasma levels of enzyme. The levels of toxic metabolites was still in control, 5 years after that first initial dose.
Company Genesis
Previous attempts to commercialize the therapy had not been successful, primarily due to funding restraints. With the recent publication of the 5 year follow up, and its exhibition of 130x longer durability than standard-of-care ERT, the team behind these finding decide to form a new company, Glafabra Therapeutics, where the new company is currently raising funds to restart clinical trials in the US. Glafabra is actively seeking partnerships to help get this therapy into the hand of the Fabry disease community, so that patients can start to utilize a new therapeutic approach that will give them a less encumbered lifestyle
