Regenerative medicine opens new frontiers in treatment of AMD

VIENNA — Success with the first autologous transplantation of iPS-RPE sheets opens a new frontier in the treatment of age-related macular degeneration.

Masayo Takahashi, MD , PhD , keynote lecturer at the meeting of the European Society of Ophthalmology, approached the concept of stem cells at the Salk Institute in 1995 and pioneered research on stem cell transplantation into the retina.

Masayo Takahashi

Her research moved from somatic stem cells to embryo-derived cells and finally to induced pluripotent stem cells (iPS). Following preclinical trials and a long process of quality control for safety and genomic analysis, the first iPS-derived RPE cell sheet was transplanted in a patient, a Japanese woman with AMD that was nonresponsive to anti-VEGF therapy.

“No adverse event or immune rejection was observed. Retinal thickness decreased, and no recurrence of CNV occurred. While the patient was progressively losing vision in the course of anti-VEGF treatment, stabilization was achieved after RPE transplantation surgery, and she could stop the injections,” Takahashi said.

The patient reported vision to be “brighter than before” and white to be “clearer than before.”

This initial successful case proved that autologous RPE transplantation may be safe and effective. However, the cost, “comparable to an F1 Super Car,” is too high to be feasible, Takahashi said.

“That’s why we are moving to allogenic transplantation, evaluating immune responses, genetic compatibility and HLA matching,” Takahashi said.

Anti-VEGF, she said, is not a radical cure and relies on repeated treatment.

“If the RPE is damaged, RPE replacement is needed, and if photoreceptor cells are damaged, we need photoreceptor transplantation. Regenerative medicine is gradually improving, and these new treatments will become available in 10 to 20 years,” she said. – by Michela Cimberle

Disclosure: Takahashi reports no relevant financial disclosures.