Professor, who works at the University of Pennsylvania Perelman School of Medicine and is the Director of the Rose H. Weiss Gene Therapy Center, one of the largest research centers in the field in the USA. Dr. Wilson is considered one of the leading names in gene therapy research against rare diseases in the world.
The gene therapy called “Zolgensma”, which was approved by the FDA for the use of patients in 2019 for spinal muscular atrophy (SMA), and the gene treatments for familial “lipoprotein lipase deficiency” and “retinal dystrophy” diseases caused by mutation in the RPE65 gene, were developed in Wilson’s laboratory and spread around the world. It is among the main treatments.
Prof., who conducted two clinical studies on gene therapy with Gazi University Faculty of Medicine Hospital Phase-1 Clinical Research Center in Turkey. Dr. In this context, Wilson visited the hospital yesterday.
“A CENTER WHERE GENE THERAPY CAN BE APPLIED FOR MANY COUNTRIES”
Stating that he is happy to be in Turkey, Prof. Dr. Wilson said that Gazi University Faculty of Medicine Hospital is at a very good point in gene therapy in terms of expertise, infrastructure and application.
Prof. Dr. Wilson said, “I think Gazi University Faculty of Medicine is an excellent center for the start of gene therapy applications in Turkey. In fact, it will be a center where gene therapy can be applied to many countries in the world. In addition to clinical research, gene therapies will also be produced and reproduced here and delivered to patients immediately.” “During my visit, I got the impression that gene therapy production studies could also begin because they have sufficient equipment and scientists.” he said.
“MORE EFFECTIVE AND CHEAPER” GENE THERAPY STUDIES FOR SMA PATIENTS HAVE BEEN STARTED
Wilson also stated that they are working on a new treatment in the USA that is closely related to SMA patients, and said:
“The virus of the gene therapy called ‘Zolgensma’, which is currently used in many countries around the world for SMA patients, was produced in my laboratory. Of course, this is a new treatment, there are still some question marks about its safety and effectiveness. Most importantly, it is a very expensive treatment. Many diseases are treated in our laboratory. “We are constantly working on the development of new gene therapies for our patients. In this context, we have created the first stages of the new generation gene therapy to be used in SMA disease. We expect this gene therapy to be more effective, have fewer side effects and be cheaper. Of course, we need a few more years to offer it to patients.”
“GENE TREATMENT IS EFFECTIVE WHEN APPLIED IN THE EARLY PERIOD”
Prof. Dr. Regarding the effects of gene therapy currently used in SMA, Wilson said, “SMA gene therapy, especially when applied early in newborns, prevents many loss of functions that will occur in the future. However, this has been observed in early treatment applications. The number of functions that can be regained if the disease has progressed to an advanced stage.” “It decreases considerably, and even if the disease is very advanced, there is no recovery of function.” shared his knowledge.
Stating that including SMA in heel blood screenings of newborn babies in Turkey is very important and valuable in terms of early treatment, Wilson pointed out that treatment research on rare diseases in the world has reached a very good point compared to the past.