The oldest eye and ear, nose, and throat hospital in the country is the newest member of Partners HealthCare. On Sunday, April 1, Massachusetts Eye and Ear (Mass. Eye and Ear) officially joined our network, and will now enhance our patients’ access to the highest quality, research-driven care for eye and ENT conditions.
Mass. Eye and Ear brings a long legacy of pioneering treatments that have brought new hope to patients with eye and ear conditions. That bold embrace of innovation was at work last month, when Mass. Eye and Ear became the first institution to treat a patient’s inherited retinal disorder with gene therapy.
The patient, a 13-year-old boy named Jack, was born with a defective version of the RPE65 gene, a mutation affecting 1,000-2,000 Americans that prevents the proper production of a protein that lets the eyes process light. For Jack, this meant struggles in the classroom, and poor night vision that ruled out sports with outdoor games that veered into evening hours. A drug called Luxturna, developed by Philadelphia-based Spark Therapeutics and approved by the FDA in December, could change all of that.
Unlike other gene therapies already in use, Luxturna works by delivering a healthy, lab-grown copy of the defective gene into patients, rather than genetically modifying patients’ own cells—making it the first “true” gene therapy approved by the FDA. For Mass. Eye and Ear and surgeon Jason Comander, MD, the opportunity to move Luxturna from therapeutic first to surgical first by injecting it into Jack’s eyes last month was in keeping with their medical mission. “It’s every eye doctor’s dream to take someone who has bad vision and give them the chance of getting better,” says Dr. Comander.
Though it will take some time to realize the full therapeutic benefit for Jack, there is reason to believe his sight will improve; in clinical trials, Luxturna improved most patients’ vision either moderately or significantly. Post-approval success as it is administered across the country will fuel hope for the role of gene therapies in treating patients with a wide range of conditions involving genetic variations.
Image credit: WBUR.