CU Anschutz researchers launch “moonshot” to cure blindness through eye transplants

As part of a national “moonshot” to cure blindness, researchers at the CU Anschutz Medical Campus will receive as much as $46 million in federal funding over the next five years to pursue a first-of-its-kind full eye transplantation.

“This is no easy undertaking, but I believe we can achieve this together,” said Dr. Kia Washington, the lead researcher for the University of Colorado-led team, during a press conference Monday. “And in fact I’ve never been more hopeful that a cure for blindness is within reach.”

The CU team was one of four in the United States that received funding awards from the federal Advanced Research Projects Agency for Health, or ARPA-H. The CU-based group will focus on achieving the first-ever vision-restoring eye transplant by using “novel stem cell and bioelectronic technologies,” according to a news release announcing the funding.

The work will be interdisciplinary, Washington and others said, and will link together researchers at institutions across the country. The four teams that received the funding will work alongside each other on distinct approaches, though officials said the teams would likely collaborate and eventually may merge depending on which research avenues show the most promise toward achieving the ultimate goal of transplanting an eye and curing blindness.

Dr. Calvin Roberts, who will oversee the broader project for ARPA-H, said the agency wanted to take multiple “shots on goal” to ensure progress.

“In the broader picture, achieving this would be probably the most monumental task in medicine within the last several decades,” said Dr. Daniel Pelaez of the University of Miami’s Bascom Palmer Eye Institute, which also received ARPA-H funding. Pelaez is the lead investigator for that team, which has pursued new procedures to successfully remove and preserve eyes from donors, amid other research.

He told The Denver Post that only four organ systems have not been successfully transplanted: the inner ear, the brain, the spinal cord and the eye. All four are part of the central nervous system, which does not repair itself when damaged.

If researchers can successfully transplant the human eye and restore vision to the patient, it might help unlock deeper discoveries about repairing damage to the brain and spine, Pelaez said, as well as addressing hearing loss.

To succeed, researchers must successfully remove and preserve eyes from donors and then successfully connect and repair the optical nerve, which takes information from the eye and tells the brain what the eye sees.

A team at New York University performed a full eye transplant on a human patient in November 2023, though the procedure — while a “remarkable achievement,” Pelaez said — did not restore the patient’s vision. It was also part of a partial face transplant; other approaches pursued via the ARPA-H funding will involve eye-specific transplants.

Washington, the lead CU researcher, said she and her colleagues have already completed the eye transplant procedure — albeit without vision restoration — in rats.

The CU team will next work on large animals to advance “optic nerve regenerative strategies,” the school said, as well as to study immunosuppression, which is critical to ensuring that patients’ immune systems don’t reject a donated organ. The goal is to eventually advance to human trials.

Pelaez and his colleagues have completed their eye-removal procedure in cadavers, he said, and they’ve also studied regeneration in several animals that are capable of regenerating parts of their eyes, like salamanders or zebra fish. His team’s funding will focus in part on a life-support machine for the eye to keep it healthy and viable during the removal process.

InGel Therapeutics, a Massachusetts-based Harvard spinoff and the lead of a third team, will pursue research on 3-D printed technology and “micro-tunneled scaffolds” that carry certain types of stem cells as part of a focus on optical nerve regeneration and repair, ARPA-H said.

ARPH-A, created two years ago, will oversee the teams’ work. Researchers at 52 institutions nationwide will also contribute to the teams. The CU-led group will include researchers from the University of Southern California, the University of Wisconsin, Indiana University and Johns Hopkins University, as well as from the National Eye Institute.

The teams will simultaneously compete and collaborate: Pelaez said his team has communicated with researchers at CU and at Stanford, another award recipient, about their eye-removal research.