17 Jul One Time Injection With Spark’s Gene Therapy LUXTURNA Demonstrated Lasting Visual Improvement
MedicalResearch.com Interview with:
Dr. Russell
Stephen R. Russell, MD
Dina J Schrage Professor of Macular Degeneration Research
Service Director, Vitreoretinal Diseases and Surgery
Professor of Ophthalmology and Visual Sciences
The University of Iowa
MedicalResearch.com: What is the background for this study? What are the main findings?
Response: This study examines the efficacy (and safety) of treating children and adults with a form of retinitis pigmentosa known as RPE65-associated Lebers congenital amaurosis, with an adeno-associated viral vector(AAV) delivered RPE65 construct. Building on successful phase 1/2b trials from multiple centers, the AAV-hRPE65v2 agent now designated as voretigene neparvovec, contains a highly optimized enhancing sequence and promoter.
The main findings were an improvement on a multiple light level mobility test (MLMT) and multiple additional supportive secondary endpoints which included improvements in full-field light sensitivity, Goldmann visual field, and others.
MedicalResearch.com: What should clinicians and patients take away from your report?
Response: The outcomes of this trial demonstrate a strong, statistically and clinically significant improvement with gene therapy in this condition.
- First, the study represent a milestone of phase 3 completion by a gene therapy study that met its pre-specified primary and several supportive secondary endpoints.
- Second, the results demonstrate successful improvement of a genetic disease with intervention, rather than a reduction of loss.
- Third, the safety profile suggests that the adeno-associated vector platform demonstrated little to no inflammation when injected into the subretinal space.
MedicalResearch.com: What recommendations do you have for future research as a result of this study?
Response: Combined with another recent publication that suggests that up to 75% of known retinal degeneration-causing genes are small enough to fit into an AAV payload, these results support a targeted approach of additional trials for other inherited retinal diseases (IRDs).
Disclosures: My institution has received grants from the sponsor, Spark Therapeutics. Additionally, I have consulted for Spark.
MedicalResearch.com: Thank you for your contribution to the MedicalResearch.com community.
Citation:
Russell, Stephen et al. The Lancet , Volume 0 , Issue 0 ,
DOI: http://dx.doi.org/10.1016/S0140-6736(17)31868-8
Note: Content is Not intended as medical advice. Please consult your health care provider regarding your specific medical condition and questions.
Last Updated on July 17, 2017 by Marie Benz MD FAAD