Saudi Arabia is the first site in the Middle East to utilize Luxturna as the first USA FDA approved gene therapy to treat blindness in children who are born blind and adults with a hereditary disease: night blindness
AL-BILAD — Professor Selwa Al-Hazzaa is a Saudi ophthalmologist and head of the ophthalmology department at King Faisal Specialist Hospital. She is one of the first Saudi women to achieve international success academically and professionally and had once served as the personal ophthalmologist for late King Fahd bin Abdulaziz Al Saud.
As Chief of Ophthalmology, Selwa Al-Hazzaa makes Saudi Arabia the Center of Excellence to utilize LUXTURNA, the first USA FDA/EU approved “Retinal Gene Therapy” to treat blind patients.
The journey and negotiations for Professor Al-Hazzaa started in October 2018 in Chicago USA, during the American Academy of Ophthalmology meeting to recognize Saudi Arabia as part of the world global GENOME map.
Negotiations continued with the global company in Europe who then declared Saudi Arabia as the first site in the Middle East and the 5th globally to utilize Luxturna as the first USA FDA approved gene therapy for any genetic disorder for “Retinal Gene Therapy” to treat blindness in children who are born blind and adults with a hereditary disease referred to as “Retinitis Pigmentosa” (night blindness) with a defective biallelic RPE65 Gene mutation.
The long journey continued with discussions with the Saudi diagnostic lab (SDL) under the supervision and efforts of Dr. Malak Thagafi in Riyadh, KSA and the identification of multiple patients from various Saudi families with biallelic RPE65 mutation in KSA who were all recalled and re-examined by Professor Al-Hazzaa with repeat photography; OCT’s and ERG’s to reconfirm the accurate diagnosis of this specific mutation in Retinitis Pigmentosa both in children and adults.
The retinal surgeons Professor Al-Hazzaa and Dr. Mohammed Al-Khuthaila obtained surgical accreditation in Europe for this complex and delicate sub-retinal microscopic surgery. In October and with the continuous efforts of Professor Al-Hazzaa, the Saudi FDA approved the genetic treatment “LUXTURNA” as a one-time administration of RPE65 gene therapy for all patients with Retinitis Pigmentosa making KSA a leader in the Genome Project which further represents a tremendous advance in medicine and in changing people’s lives in KSA.
Their first patient was a 9-year-old Saudi female born blind and followed by Professor Al-Hazzaa since the age of 1 year with her affected older sibling and cousins. In November 2019 the surgery was completed of subretinal injection of both eyes with the genetic treatment of LUXTURNA by Professor Al-Hazzaa and Dr. Mohamed Khuthaila with great success.
The proved safety and efficacy of LUXTURNA has shown significant improvement over the past 20 years of the conducted trials in both the USA & GB in improving markedly the navigational ability in dimly light conditions (hence night blindness) in children and adults of this intraocular administration of AAV2 of RPE65 Gene which is designed to deliver a normal copy of the mutated RPE65 gene to the diseased retinal cells that are lacking a normally functioning RPE65 gene to restore vision with only ONE-time treatment to each eye in 2 stages.
Professor Al-Hazzaa, an international expert in Genetics, Gene Therapy and hereditary diseases in the Middle East indicates new opportunities of this precision medicine will be extended to other forms of “Inherited Retinal Diseases” such as Stargardt disease, Chorideremia, Usher syndrome, LHOA, Retinoschisis, other forms of RP, and more genetic diseases that are extremely common in Saudi Arabia.
This is a new era in which patients with confirmed “Inherited Retinal Disorders” can now be treated with gene therapy or enrolled in relevant gene therapy clinical trials being conducted worldwide.
Professor Al-Hazzaa’s initiative has put Saudi Arabia at the forefront of worldwide translational gene therapy interventions and “Transforming Innovation Into Impact”.