Novo Nordisk wants to develop new stem cell-based therapies to help patients with serious chronic diseases that will allow us to continue to offer life-changing treatments in the future. Novo Nordisk has for a long time been a partner in national and international cooperative projects involving stem cells. Our initial research activities on pluripotent stem cells from mice have been expanded to include cells of human origin. Human pluripotent stem cells include embryonic and induced pluripotent stem cells where the human embryonic stem cells (hESC) used in our research are derived from surplus embryos donated with freely given informed consent. Novo Nordisk also follows scientific breakthroughs within stem cell research, including the Nobel Prize-winning breakthrough where human induced pluripotent stem cells (hiPSC) were created from somatic cells.
Novo Nordisk finds that it is crucial to proceed with research in pluripotent stem cells (hESC as well as hiPSC), to gain basic knowledge about how cells grow and develop to allow us to confirm the possibility of using stem cells in cell replacement therapies. Novo Nordisk has embarked on hiPSC-based research, which provides unique access to novel models in disease development as well as in drug efficacy and safety testing. Research in hESC has evoked an important ethical debate and Novo Nordisk would like to be part of an open discussion about understanding the ethical and legal issues in the use of human embryonic stem cells. Novo Nordisk urges all countries to establish legislation that will ensure that this field is adequately regulated and controlled.
Adult and pluripotent stem cells differ in several ways; pluripotent stem cells allow unlimited expansion and have a greater developmental potential compared to most adult stem cells. Therefore, Novo Nordisk focuses on pluripotent stem cells as a basis for cell therapy.
Currently, the best defined and most extensively used stem cell treatments are based on adult stem cells, including blood stem cell transplantation to treat diseases and conditions of the blood and immune system, or to restore the blood system after treatments for specific cancers. Some bone, skin and corneal diseases or injuries are also treated with grafting of tissue that is dependent on the use of stem cells from these organs.
Pluripotent stem cells themselves cannot directly be used for therapies as in their undifferentiated state they will likely cause benign tumours (teratomas). They will first need to be coaxed into specialised cell types before transplantation. Therefore, it is critical that these cells are proven safe and efficacious in preclinical and controlled clinical trials.
Many potential pluripotent stem cell-based treatments are currently being tested in animal models and a few have been brought to clinical trials, with the first phase 1/2 clinical trial approved by the US Food and Drug Administration (FDA) in 2010.
A second type of pluripotent stem cells is induced pluripotent stem cells (iPSCs that were discovered/generated for the first time by Yamanaka et al in 2006, for which he received the Nobel Prize in 2012). Such cells can be obtained by treating any given cell culture with four factors that induce the reprogramming to eventually yield induced pluripotent stem cells. The four factors can be given very efficiently via viral infection, which is the widely used method.
More recently, more gentle ways have been developed to reprogramme mature cells into iPSC. International guidelines need to be developed on sources of iPSC starting-cell material and to allow an evaluation of the applicability of iPSC in cell-based therapy in general. Recently, a Japanese clinical trial was approved to use patient-specific iPSC-based cell therapy of macular degeneration (a chronic eye disease).