Stem Cell Trial for Eye Disease Commences

Researchers at the RIKEN Center for Developmental Biology will treat the first patient in its clinical trial testing an induced pluripotent stem cell-based treatment for age-related macular degeneration.

By | September 12, 2014

FLICKR, RUTH HARTNUPThe first ever trial to treat humans with a treatment derived from induced pluripotent stem cells (iPSCs) is set to begin. Soon—“within days,” Nature reported one RIKEN source as saying—researchers at the RIKEN Center for Developmental Biology (CDB) in Kobe, Japan, will transplant sheets of retinal pigment epithelial cells derived from an age-related macular degeneration (AMD) patient’s own skin cells to replace damaged cells of the retina.

CDB ophthalmologist Masayo Takahashi and her collaborators have tested the autologous treatment in monkeys, showing that the animals mounted no immune response that resulted in the cells’ rejection. Studies in mice and monkeys have also demonstrated that the therapy is unlikely to cause tumors, a concern with injecting iPSCs and embryonic stem cells into the body. To further ensure the safety of patients in this first trial, the researchers have performed genetic stability tests on the cells they plan to implant.

The first transplant surgery will be performed at the Institute of Biomedical Research and Innovation in Kobe. Trial researchers will eventually transplant autologous cells into six AMD patients, who will be monitored for one year following the procedure.

“While I worry that researchers in Japan may be moving a bit too fast to human studies, at the same time I have to admire their dedication and commitment to translating iPS cells into clinically relevant medicines,” Paul Knoepfler, a stem cell researcher at the University of California, Davis, wrote on his blog. “It’s tricky when it comes to getting iPS cell-based therapies to patients. The sweet spot—not too slow or too fast—for the speed of iPS cell clinical translation is not entirely clear today.”

(Stay tuned for next month’s biobusiness article on stem-cell therapies for blindness diseases, appearing in The Scientist’s October issue dedicated to vision.)

Add a Comment

Avatar of: You



Sign In with your LabX Media Group Passport to leave a comment

Not a member? Register Now!

LabX Media Group Passport Logo

Popular Now

  1. Thousands of Mutations Accumulate in the Human Brain Over a Lifetime
  2. 2017 Top 10 Innovations
    Features 2017 Top 10 Innovations

    From single-cell analysis to whole-genome sequencing, this year’s best new products shine on many levels.

  3. Search for Life on the Red Planet
  4. Two Dozen House Republicans Do an About-Face on Tuition Tax