Human trials to start very soon for Stem Cell-Based Gene Therapy for Huntington’s

Human trials to start very soon for Stem Cell-Based Gene Therapy for Huntington’s

Neural Regeneration Research has published a report in which the scientists at Universty of California Davis Health System summarize the shortcomings and advances of stem cell-based Gene Therapy for Huntington’s disease.

Gene therapy using mesenchymal stem cells for Huntington’s disease is showing promise in mouse studies, and preparations are underway to possibly move it into clinical testing. Before the technique might be ready for human trials, however, scientists need to master a few more steps, using larger animal models to investigate the therapy’s safety and likely long-term effects.

Because of their unique biological properties, mesenchymal stem cells have shown tremendous promise in stem cell-based gene therapy approaches in recent years, and applications targeting other neurological diseases, such as ALS and stroke, are now in clinical trials.

In preparation for a future Phase 1 human clinical trial of mesenchymal-BDNF stem cells for Huntington’s, a current study, PRE-CELL, is recording numerous measures of disease symptoms and markers in patients to serve as a base for the coming HD-CELL trial.

Read in detail here.