Retinitis Pigmentosa Clinical Trial
Official title:
Management of Retinitis Pigmentosa by Wharton's Jelly Derived Mesenchymal Stem Cells: Preliminary Clinical Results
The aim of this study is to determine if umbilical cord Wharton's jelly derived mesenchymal stem cells implanted in sub-tenon space have beneficial effects on visual functions in retinitis pigmentosa patients by reactivating the degenerated photoreceptors in dormant phase.
The retinal pigment epithelium (RPE) forms the outer blood-retinal barrier between
photoreceptor cells and choroidal blood vessels. Photoreceptor cells are vitally and
functionally dependent on the RPE. The conversion of blood glucose to ATP, synthesis of
proteins in the visual cycle and removal of metabolic waste takes place in the RPE. For these
important processes, various peptide growth factors and their receptors are synthesized in
the RPE. More than 260 genes in the RPE are responsible for the production of these peptide
fragments. Mutations in any of these genes as well as ischemic, physical or chemical RPE
damage causes retinal degeneration. Retinal degeneration may be inherited, such as in
retinitis pigmentosa (RP), Stargardt's disease, choroideremia, Best vitelliform dystrophy and
Bietti's crystalline dystrophy. Retinal degeneration may also be acquired through genetic
mechanisms, such as age-releated macular degeneration. In retinal degeneration, there is a
developing loss of RPE and photoreceptors, regardless of the underlying cause.
Umbilical cord Wharton's jelly derived mesenchymal stem cells (WJ-MSCs) have significant
paracrine and immunomodulatory properties. WJ-MSCs secrete trophic factors that stimulate RPE
or secrete trophic factors that are similar to those produced by RPE. In studies using animal
models, WJ-MSCs have been found to be effective in stopping the progression of retinal
degeneration and for rescuing photoreceptors in the dormant phase. WJ-MSCs are
hypoimmunogenic and have significant immunomodulatory properties. WJ-MSCs have been shown to
suppress chronic inflammation and prevent apoptosis in animal models of neurodegenerative and
ischemic retinal disorders. WJ-MSCs also stimulate progenitor cells in the retina and elicit
self-repair mechanisms.
The aim of this preliminary clinical study is to investigate the efficacy of deep sub-tenon
injected WJ-MSCs as a stem cell treatment modality for the management of retinitis
pigmentosa, which creates outer retinal degeneration. These functional and structural effects
were investigated using microperimetry, electrophysiology and spectral domain optical
coherence tomography (SD-OCT). To the best of our knowledge, this is the first prospective
clinical study that utilizes a large number of RP cases, and cases that are in phase-3.
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