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Treatment of Retinal Disease by Activation of the Function of Bone Marrow-derived Stem Cell or Progenitor Cell Thereof
Efficacious therapeutic agent for retinal diseases including retinitis pigmentosa

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Case Number62
UploadedNov, 11, 2009
Potential ApplicationThe prophylactic and therapeutic agent of the present invention enables us to prevent and treat for various retinal neurodegenerative diseases including retinitis pigmentosa. For instance, we can apply the invented therapeutic agent for diseases of preventable in deterioration of vision such as glaucoma, age-related macular degeneration, retinopathy of diabetes and retinopathy of prematurity by preventing the degeneration in photoceptor cell or retinal vessel.
Technology
Area
Biotechnology
KeywordsRetinal disease
Neuroprotection
Bone marrow derived stem cell
ARMD
Retinitis pigmentosa
ResearchersAtsushi OTANI
Nagahisa YOSHIMURA
Manabu SASAHARA
Summary

Currently one out of every 4000 people are suffered from retinitis pigmentosa, which is a blinding disease. Efficacious treatment preventing the development has not been invented so far, and a development of breakthrough therapy has been expected.

We have invented a fact that the cell function in the bone marrow stem cell became significantly improved when administering two types of hematopoietic factor of G-CSF(Granulocyte-colony stimulating factor) and Erythropoietin to a modeling mouse at the same time. Thus, it was observed that the mouse administered G-CSF and Erythropoietin at the same time obviously had a slow advancement in retinal degeneration, and that the effect influenced not only the protection for degeneration of photoreceptor cell, but also the protection for degeneration of the retinal vessel. Remarkably, a significant inhibition was observed in the degeneration of the cone photoreceptor cell, which is greatly relevant to the visual acuity of patients suffering from retinitis pigmentosa. This experiment result shows that our invented method leads to the prevention of blindness.

Therefore, our invention provides:
1) Prophylactic and therapeutic agent for retinal diseases (including retinitis pigmentosa) combining G-CSF and erythropoietin. In this case, the retinal diseases are attributable to the degeneration in the photoreceptor cell or the retinal vessel.
2) The photoreceptor cell degeneration inhibitor combining G-CSF and erythropoietin.
In this case, the photoreceptor cell is pyramidal cell or rod cell.
3) Retinal vessel degeneration inhibitor combining G-CSF and erythropoietin.
4) Prophylactic and therapeutic agent for the diseases attributing to choroidal neovascularization including age-related macular degeneration, and the therapeutic agent is produced combining G-CSF and erythropoietin.

G-CSF and erythropoietin are safe and secure, enable applying for a clinical setting in a very short period.
Moreover, we are able to provide a breakthrough therapeutic agent in terms of a large number of patients who suffer from retinal diseases.
Also, the pharmaceutical agent of the present invention requires no gene introduction. Though the patients suffer from retinitis pigmentosa have various gene variation patterns, the pharmaceutical agent of the present invention is generally applicable without considering the variation pattern, and is greatly beneficial.

fig1
[Fig.1] Preservation of photoreceptor cell by sole or combinatorial administration of G-CSF or Epo

Related InformationRelated papers/documents/links
Reference: Prof. Atsushi OTANI
“Treatment of Retinal Disease by Activation of the Function of Bone Marrow-derived Stem Cell or Progenitor Cell Thereof ”
PCT/JP2009/052415
WO 2009/102021

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