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In 2019, Dr. Rachida Bouhenni, PhD, received a Cure Glaucoma Foundation grant to study protein therapy for a childhood vision defect. The study, Development of an Enzyme Replacement Therapy for Primary Congenital Glaucoma, paused during the 2020 COVID-19 pandemic. As of August 2020, the research resumed and is now fully operational. Dr. Bouhenni hypothesizes that injecting a lab-made enzyme into the eyes of patients with certain gene mutations reduces cellular stress. This approach aims to limit vision loss and improve the quality of life for affected children.
Successful Enzyme Uptake in the Eye Confirms Research Hypothesis
Her first step to confirm the hypothesis was proving that the eye could successfully receive the enzyme. Over the course of four weeks, researchers treated different groups of enzyme-deficient mice with lab-made enzyme at varying doses and times. Consequently, they could observe how dosage and timing affected enzyme uptake and effectiveness. The tests succeeded, and the figure below shows the enzyme uptake in the eye cells. Because of the success of this segment of the research, Dr. Bouhenni can move forward to the next phase, which is extensive analysis of the data.
Recently, Dr. Bouhenni added a graduate student to assist with the research. A private company has shown interest and may mass-produce the enzyme for future testing and treatment. When additional foundations or companies support research, projects can progress faster and expand more effectively. Historically, the biggest barriers to research are the lack of funding and personnel.
Dr. Bouhenni expressed her sincere gratitude to the donors and board of directors of the Cure Glaucoma Foundation for their generosity. Because of the initial grant, this promising research was able to begin and will continue to move forward.