Cell-Based Treatment for Stargardt Disease: Advanced Cell Technology (ACT)


Description: ACT has launched a Phase I/II clinical trial of transplanted retinal pigment epithelial (RPE) cells for the treatment of Stargardt disease. The RPE cells are derived from human stem cells. RPE are the first cells affected in people with Stargardt disease. They play a critical role in maintaining the health and function of photoreceptors. The study, taking place at Oregon Health and Science University and the Jules Stein Eye Institute (UCLA), will enroll up to 12 participants. The primary goal of the study is to demonstrate safety and tolerability. The Foundation funded many of the preclinical studies that made this human trial possible.

Trial status: Recruiting participants.

Partial List of Participation Criteria:

  1. 18 years of age or older
  2. Diagnosis of advanced Stargardt disease
  3. No better than hand-motion vision in eye to be treated

Clinical Trial Contacts:

James Bainbridge, M.A., M.B., B.Chir., Ph.D., FRCOpth
Phone: 44 (0) 20 7608 6823

Heather Kneale
Phone: 44(0) 207 608 4023
Moorfields Eye Hospital
London, United Kingdom

Grampian University Hospitals NHS Trust, Foresterhill, Aberdeen Royal Infirmary Eye Unit
Aberdeen, United Kingdom
Contact: Vikki McBain
01224 552543

Jules Stein Eye Institute, UCLA School of Medicine
Los Angeles, California
Contact: Logan Hitchcock

Wills Eye Institute-Mid Atlantic Retina
Philadelphia, Pennsylvania
Contact: Shellie Markun

Clinical Trial Underway for Stem Cell Treatment

Additional Information:

Listing of ACT’s Stargardt disease stem cell treatment on ClinicalTrials.gov