Recent Patents on Regenerative Medicine - Volume 5, Issue 2, 2015

Use of animal products to culture human stem cells poses risk of pathogens and other contaminations which may render stem cells therapeutically unsuitable. Hence, it is necessary to meet the standards of good manufacturing practices, clinical-grade like and xeno-free conditions. The protocol for culturing clinical grade stem cells needs to be modified so that maximum utilization can be done for stem cell therapies. Modifications can be in the media compositions, additional growth factors, small molecule compounds, and gene manipulations of the required transcription factors or the platform in which stem cells can grow. After the clinical grade standards are met it needs the approval of FDA for use as transplantation therapies. This review provides various methods and inventions for culturing clinical grade stem cells which have been patented.

Regenerative medicine seeks to restore normal architecture and function of tissues which have been damaged by disease, trauma and/or ageing. Stem cells are known to be an integral part of any regeneration process. Their isolation or generation, in vitro expansion, recruitment and transplantation to the damaged sites are being heavily pursued. Several applications of pluripotent cell derivatives have arrived at the clinical stage and are offering promising results. Stem cells themselves or their products can exert desired functions in regeneration. The source of stem cells for developing therapies is vast and developments have been made at a very fast pace during the last ten years. Pluripotent stem cells from discarded embryos or induced from postnatal tissues are at the base of these investigations as they can theoretically be permanently cultured in vitro and can be triggered to differentiate any cell in the human body. Induced or directed differentiation of these cells is still being studied and generally the process involves long manipulations and thorough selection, which prevent pluripotent cells from being useful in autologous transplants. So-called adult stem cells or stem cells isolated from postnatal individuals offer the possibility to develop autologous sources for cell therapies. Bone marrow, cord and peripheral blood, fat and oral mucosa are sources of such stem cells. In this case the drawbacks are the availability in sufficient numbers to develop the regenerative protocols and the limited applications of these cells due to their limited differentiation ability. Both pluripotent and adult stem cells are being investigated for the development of clinical trials at the moment. Pluripotent stem cells will be able to be applied widely in cell therapy when directed differentiation can be controlled to the point of producing safe and functional derived cells and tissues. The present review will focus on recent patents on directed differentiation of pluripotent stem cells to derivatives with potential therapeutic value.

Multipotent and pluripotent Stem cells are highly clonogenic populations of cells considered the utmost promise in the field of regenerative medicine. By definition stem cells are progenitor cells capable of self-renewal and differentiation hypothetically “ad infinitum” into more specialized cells and mature tissue. Stem cells are commonly classified based on the developmental stage from which they are isolated, although this has been a source of debate amongst stem cell scientists. A commonly accepted approach classifies stem cells into three different groupings: Embryonic Stem Cells (ESCs), Umbilical Cord Stem Cells (UCBSCs) and Adult Stem Cells (ASCs), which includes stem cells from bone marrow (BM), fat tissue (FT), engineered induced pluripotent (IP) and peripheral blood (PB). The increasing emergence of evidence in support of the clinical effectiveness of stem cells has had dramatic implications in our understanding of degenerative disease progression offering the opportunity to develop novel therapeutics that specifically target and eradicate the inherent cause. This review aims to provide an overview of the recent developments in the field of multipotent and pluripotent stem cell new models and therapeutics, embodied in the form of patent documents. Therefore, we refer to a wide range of inventions, with their existing development number of patented clinical trials, which suggest stem cells specific benefits as future degenerative disease guideline uses.

Gene therapy strategies have become involved in methods for the treatment of inherited or acquired diseases with the aim to correct the faulty genetic code or to modify gene expression. In order to develop safe and effective therapeutic methods for applying in humans, different nucleic acids, termed as vectors, have been engineered, as therapeutic molecules in addition to classical drugs. In this review we included an overview of recent research and patents on therapeutic nucleic acids, designed for use on safe and effective gene therapy protocols for the treatment of diseases, whose results have been obtained in animal models, and which suggests that the use of nucleic acids as therapeutics molecules holds great promise for the future of disease control or cure.

Genomic engineering has enormous potential along basic research, drug discovery and cell therapeutics. Many existing methods for targeted gene knockout mutagenesis or integration rely on homologous recombination. The low rate of spontaneous recombination in nearly all mammalian cell types, as well as the scale of screening, effort and time required to isolate the targeted events during genome modification, have hindered progress in this field. The present review has the objective to present latest improvements of technology to develop genetic modification to a clinical grade level so it can be used in human therapy.

Stem cells have the ability to self-replicate in vitro indefinitely under adequate conditions and provided that pluripotent signals are triggered exogenously. Moreover, they are able to differentiate into virtually all human cell types. This vast potential makes stem cells a powerful tool to be used in regenerative medicine and different therapeutic strategies as well as in clinical and basic research, contributing to the generation of novel therapeutic avenues to treat innumerable pathological disorders that today have no known cure. Despite increased disclosures at a high speed pace in terms of benefits, applicability and relevance of stem cell based therapies; more studies in humans are needed to advance in the establishment of safe and effective cell-based treatments. In this review we compile an overview of the latest reports and patents on stem cell accomplishments for the treatment of diseases and injuries from Patent databases as The European Patent Office, WIPO, Fresh patents and Freepatents online, as well as Pubmed references, regarding stem cell therapy. Nevertheless, the vast majority of these promising results have been established in animal models, suggesting that stem cells research must go on prior to transferring therapeutic protocols to the human being although it has much to offer to improve life quality and eliminate disability.

Stem cells possess a great potential of differentiating into various types of cells. They are capable of sustaining their stemness or getting converted into cells having a more specialized function in the body. The obstacles that pose as a barrier to the therapeutic and clinical applications of stem cells are the difficulty in deriving and controlling the fate of these stem cells. With the advancement of science and technology and with better knowledge, scientists have come up with the method of reprogramming for the generation of iPSCs. Reprogramming refers to converting a differentiated cell into its embryonic state, capable of re-differentiating into other cell types. These iPSCs phenotypically as well as morphologically resemble ESCs. Induced pluripotent stem cells are of great advantage for studying the pathogenesis of disease and drug discovery. Understanding the regulation of stem cells is of great importance so as to generate a controlled stem cell fate and function. A safe, more efficient and a cost-effective approach to this is the use of small molecules for reprogramming. Small molecules are easier to handle and provide a good tool for use in in vitro and in vivo studies for the development of therapeutics. Recently, fine-tuning of various combinations and concentrations of the small molecules helps provide control over the regulation of iPSCs and the reprogramming outcomes. In this article, we review patents and discuss the recent development with respect to the use of small molecules, their control over the cell fate, the effect on regulation of the reprogramming factors and the efficiency of reprogramming.