Periodic Report Summary 1 - BAPREFINEMENT (Bioartificial Pancreas refinement)
The project “BAPrefinement” (bio-artificial pancreas refinement) aims at making a substantial contribution to the development of a functional cure for type 1 diabetes. The latter is an autoimmune disease resulting in the destruction of insulin producing beta cells in the pancreas. Patients depend on life-long insulin therapy, fine-tuned via frequent blood glucose measurements and administered as multiple daily injections. The only possible cure is beta cell transplantation. This has so far been conducted only on a few selected patients, due to the risks and side-effects associated with immunosuppressive medication to avoid transplant rejection and an acute shortage of donor tissue. The project has set out to overcome both limitations and pave the way for the bio-artificial pancreas into the clinic. It introduces a novel, very robust type of surrogate beta cells, namely genetically modified liver cells, and combines it with a highly bio-compatible material for cell encapsulation allowing for immune protection and long-term survival of the transplanted cells.
During the first reporting period, cell encapsulation technology was transferred by the researcher to the host organization. Capsules were characterised in terms of size and mechanical stability. Insulin producing cells were encapsulated, and the growth and viability of cells within the capsules was assessed. Several parameters were used to optimise the generation and culturing of encapsulated insulin producing cells. Cryopreservation and subsequent revival of encapsulated insulin producing cells was successful, both, in terms of capsule integrity and cell survival. This is an important prerequisite for development of a viable cell therapy product, fit for commercialisation, accessible to a large number of patients and affordable. A small pilot biocompatibility study has been carried out with the encapsulated insulin producing cells. Furthermore, the tumourigenicity of the cells at hand has been assessed. This is a requirement for the use of cells in patients, defined by the WHO. The results of the tumourigenicity study give hope that the cells will be acceptable to the regulatory authorities.
The study design has been finalised and ethics approval obtained for a first in-vivo dose-finding study to assess the number of capsules needed to normalise blood sugar levels in a rodent model for type 1 diabetes. This study will be subject of the second reporting period. If successful, results will serve as pre-clinical data required for taking the project to the next level of clinical testing (as follow-on to this project). Once this is completed, odds are high that the resulting clinical product will drastically improve the lives of type 1 diabetics as well as type 2 diabetics who require insulin therapy.
For further information, please contact Dr. Eva Maria (Lilli) Brandtner (lilli.brandtner@vivit.at).
During the first reporting period, cell encapsulation technology was transferred by the researcher to the host organization. Capsules were characterised in terms of size and mechanical stability. Insulin producing cells were encapsulated, and the growth and viability of cells within the capsules was assessed. Several parameters were used to optimise the generation and culturing of encapsulated insulin producing cells. Cryopreservation and subsequent revival of encapsulated insulin producing cells was successful, both, in terms of capsule integrity and cell survival. This is an important prerequisite for development of a viable cell therapy product, fit for commercialisation, accessible to a large number of patients and affordable. A small pilot biocompatibility study has been carried out with the encapsulated insulin producing cells. Furthermore, the tumourigenicity of the cells at hand has been assessed. This is a requirement for the use of cells in patients, defined by the WHO. The results of the tumourigenicity study give hope that the cells will be acceptable to the regulatory authorities.
The study design has been finalised and ethics approval obtained for a first in-vivo dose-finding study to assess the number of capsules needed to normalise blood sugar levels in a rodent model for type 1 diabetes. This study will be subject of the second reporting period. If successful, results will serve as pre-clinical data required for taking the project to the next level of clinical testing (as follow-on to this project). Once this is completed, odds are high that the resulting clinical product will drastically improve the lives of type 1 diabetics as well as type 2 diabetics who require insulin therapy.
For further information, please contact Dr. Eva Maria (Lilli) Brandtner (lilli.brandtner@vivit.at).