The National Eye Institute (NEI), a division of the National Institutes of Health, has awarded researchers at Icahn School of Medicine at Mount Sinai a five-year grant to support an effort to recreate a patient’s ocular stem cells and restore vision in those blinded by corneal disease.
About six million people worldwide have been blinded by burns, trauma, infection, genetic diseases, and chronic inflammation that result in corneal stem-cell death and corneal scarring. There are currently no effective long-term treatments for the vision loss that occurs. Corneal stem-cell transplantation is an option in the short term, but availability of donor corneas is limited.
Specifically, the grant will allow Mount Sinai researchers to recreate a patient’s own stem cells by taking mature cells, such as eyelid or oral skin cells, and coaxing them backward along the development pathways to become eye-specific stem cells again, and serving ultimately as needed replacements for damaged cells in the cornea, explains Albert Y. Wu, MD, PhD, Assistant Professor, Ophthalmology, and the study’s principal investigator.
“Because the stem cells are their own, patients will not require immunosuppressive drugs that are currently used after donor corneal transplants,” adds Dr. Wu, who is also Director of the Ophthalmic Plastic and Reconstructive Surgery, Stem Cell and Regenerative Medicine Laboratory and a member of The Black Family Stem Cell Institute at Icahn School of Medicine. “This would greatly improve their quality of life.”
The funding will also allow investigators to research the most viable stem cell sources, explore the molecular pathways involved in ocular and orbital development, and develop cutting-edge biomaterials to engraft a patient’s own stem cells and restore vision.
Ihor Lemischka, PhD, Lillian and Henry M. Stratton Professorial Chair of Gene and Cell Medicine, and Director, The Black Family Stem Cell Institute, and J. Mario Wolosin, PhD, Professor of Ophthalmology, are co-investigators with Dr. Wu.
The Institute is Mount Sinai’s hub for both basic and disease-oriented research on embryonic and adult stem cells. Investigators believe that understanding how stem cells signal one another and other cells may potentially yield diagnostic and therapeutic breakthroughs not only for corneal damage, but possibly type 1 diabetes, Parkinson’s disease, various cardiovascular diseases, liver disease, and cancer.
“It is an exciting time for stem-cell biology as we move forward and set the foundation for clinical breakthroughs,” says Dr. Lemischka. “Stem cells are likely going to play a part in virtually all of the major medical breakthroughs of the twenty-first century.”