Second Patient With Stargardt’s Macular Dystrophy Treated With Stem Cells in Advanced Cell Technology Trial

July 2, 2012

Advanced Cell Technology, Inc. (Nasdaq: ACTC) today announced the treatment of the second patient in its Phase 1/2 clinical trial for Stargardt’s macular dystrophy (SMD) using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs). The surgery was performed on Friday, June 29 at Moorfields Eye Hospital in London, the same site as the first patient treatment in January, by a team of surgeons led by Professor James Bainbridge, consultant surgeon at Moorfields and Chair of Retinal Studies at University College London. The procedure was successfully performed without any complications.

The Phase 1/2 trial is designed to determine the safety and tolerability of hESC-derived RPE cells following sub-retinal transplantation in patients with Stargardt’s at 12 months, the study’s primary endpoint. It will involve a total of 12 patients, with cohorts of three patients each in an ascending dosage format. It is similar in design to the U.S. trial for Stargardt’s that was initiated in July 2011.

Gary Rabin, Chairman and CEO of ACTC, notes that the company has seen both subjective and objective evidence of improved visual acuity and quality of vision (ability to see colors better, etc) in seven other dry age-related macular degeneration (dry AMD) and Stargardt’s patients implanted with the cells. Importantly, the effects appear to persist, with the first two patients closing in on the one year mark this month and maintaining the increases in visual acuity measured shortly after their treatment.

Read about further developments in the trial on Gary Rabin’s blog.




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3 Responses to “Second Patient With Stargardt’s Macular Dystrophy Treated With Stem Cells in Advanced Cell Technology Trial”

  • Caio Ribas

    Hello good day!
    My father has Stargardt’s disease I was wondering how this research for treatment.

    srry english
    Caio Ribas

  • Dr. Ari Weitzner

    Good catch!

  • Dwayne

    ummm… SMD = Stargardt’s macular dystrophy. It’s macular, not muscular.