The study met its primary objective, which was to evaluate the safety of a one-time bilateral delivery of AB-1005 directly to the putamen.
Finding an effective ASO delivery system is a major challenge. Cancer cells have gatekeeper molecules that stop unwanted substances from entering.
This combined approach, developed by Florida International University cancer researcher Diana Azzam, was used successfully for the first time to guide treatment of relapsed pediatric cancer patients in collaboration with First Ascent Biomedical and Dr. Maggie Fader at the Helen & Jacob Shaham Cancer & Blood Disorders Institute at Nicklaus Children's Hospital in Miami.