« Generation of hematopoietic stem cells from non-hematopoietic iPS in patients with acute irradiation syndrome an innovative therapeutic strategy of hematopoietic syndrome. » – GIPSIS

This proposed project aims at using cellular therapy to improve medical care of acute radiation syndrome (ARS) in active soldiers affected by nuclear radiation and in civilians after malevolent acts or nuclear accident. The recent breakthrough was the ability to generate adult induced pluripotent stem cell, so called iPScs. Up to recently, the major technological bottle-neck was their in vivo use in human. Strikingly, the first clinical trial has been accepted by the Japanese administration and is expected to start within 2013, paving the way to clinical development. Clinical iPS banks of selected universal donors should allow their use for large scale allogeneic grafts. Aplasia is one side effect of the acute radiation syndrome, but one of the most dangerous because the prognosis is at stake. The recovery implying a quick hematopoietic graft. The use of hiPS derived hematopoietic stem cells (HSC) is a promising medical option in the future.
We will demonstrate that 1) hiPS from healthy donor are capable to reconstitute a human hematopoiesis in a radioinduced aplasia preclinical model, 2) hiPS from aplastic anaemia patients or nuclear radiation affected patients retain this ability, 3) we can clinically define a product which would cure the aplasia as severe consequence of acute radiation syndrome.
The consortium already knows how to differentiate hematopoietic stem cell from hiPS, to deal with the in vivo acute radiation syndrome model and its medical care. Our level of attainment assures the fulfillment of the program.
In the midterm, any breakthrough of our innovative and ambitious project will be used to cure aplastic anaemia patients.