Symposien Donnerstag

Änderungen im Programmablauf vorbehalten.

Saal 3 09:45 - 11:15 01.10.2015
Symposium Do11
Focus Research: Cell based therapy of the retina
Vorsitzende/r: Boris Stanzel (Bonn), Michael Koss (Heidelberg), Susanne Binder (Wien)

The retina is increasingly becoming the premier testing site for various cell-based therapeutics. Here we attempt to bring an overview of existing clinical and upcoming therapeutic modalities from preclinical development including autologous RPE transplantation, trophic cell-based therapy and stem cell based replacement using tissue engineered constructs.
Referent/in: Bernd Kirchhof (Köln)
The largest demand for an RPE substitute is geographic atrophy in age related macular degeneration. Recent investigations in the human eye showed that human RPE stem cells can survive and multiply under the retina. However, cell transplants could not support central vision. On the one hand, because the macula cannot be detached traumatically in end stage AMD, secondly because attachment of suspended single cells may be compromised by aged Bruch´s membrane, thirdly macular atrophy is irreversible. Since a combined RPE and retinal cell transplant is not yet in sight, isolated RPE transplants must aim at a stage of AMD prior to geographic atrophy. In early AMD the compliance to accept a surgical risk will be limited.
Referent/in: Udo Bartsch (Hamburg)
Neuroprotective approaches for the treatment of degenerative retinal diseases are currently being explored in patients with retinitis pigmentosa or geographic atrophy using intravitreal implants of encapsulated cells genetically modified to secrete a neurotrophic factor. To further evaluate and eventually optimize the efficacy of cell-based neuroprotective strategies, we have generated lentivirally modified clonal neural stem cell lines with a forced expression of different neurotrophic factors. The neuroprotective potential of these stem cell lines was analyzed in mouse models of retinitis pigmentosa, neuronal ceroid lipofuscinosis and glaucoma.
Referent/in: Boris Stanzel (Bonn)
Referent/in: Kirsten Borchers (Stuttgart)
Referent/in: Michael Koss (Heidelberg)
We will introduce a cost-effective re-innovated SoIodate application, that mimics outer retinal degeneration, like in dry AMD.
Referent/in: Kapil Bharti (Bethesda)
The recent successful phase I trial using embryonic stem (ES) cell derived retinal pigment epithelium (RPE) has provided hope for a cure for degenerative eye diseases. Induced pluripotent stem (iPS) cells are an alternate and an autologous source of stem cells potentially with fewer immune-challenges as compared to ES cells. Using a developmentally guided differentiation protocol we have developed fully polarized RPE tissue from iPS cells. The RPE monolayer along with its secreted ECM and a biodegradable scaffold form a tissue that well mimics the native tissue in structural and functional properties. This tissue has been functionally authenticated in vitro for its ability to perform several key RPE functions like phagocytosis of photoreceptor outer segments, ability to transport water from apical towards basal sides, and ability to secrete cytokines in a polarized fashion. Currently, we are testing the safety and the efficacy of this tissue in animal models. We have begun Phase I Investigational New Drug (IND) enabling studies with the goal to transplant autologous iPS cell derived RPE in patients in advanced Geographic Atrophy stage of age-related macular degeneration (AMD), one of the leading blinding diseases in the US. Our work will provide a potential personalized cell therapy for AMD patients.