What we do
PLACEMA´s main objective is to offer cell reprogramming and differentiation services for research groups, for pharmaceutical companies and for clinical application, generated under national and international quality standards.
What is cell reprogramming?
Stem cells have the capacity to self-renew and differentiate into specific cell types. Until recently, only two types of stem cells existed: adult and embryonic. Embryonic stem cells have the ability to differentiate into all types of adult tissue (pluripotency), reason for which they have sparked great interest as a source of cells for use in regenerative therapies. However, they derive from fertilized oocytes, making their availability limited, and no legislation has been approved for their use in Argentina.
Thanks to work conducted by Dr. Shinya Yamanaka in 2007, stem cells presenting characteristics of human embryonic cells can be generated from adult human cells such as skin cells (Takahashi et al, 2006, Cell; 126(4):663-76). The process through which an adult cell is converted into a stem cell is called reprogramming. In other words, cell reprogramming generates specialized cells such as neurons or muscle cells from other adult cells such as fibroblasts or blood cells. Cell reprogramming is achieved by introducing 4 genes (Oct3/4, Klf-4, c-Myc y Sox-2) into adult cells (fibroblasts or blood cells).
The impact of this technology and the huge possibilities that will provide to the Health Sector became evident when the 2012 Nobel Prize for Medicine was awarded to Dr. Shinya Yamanaka for this research. Cell reprogramming will allow:
- To generate in vitro models of disease to:
- Study disease pathophysiology.
- Screen for drug efficacy and toxicity on cells from actual patients suffering from particular conditions.
- To generate future regenerative therapies:
- From individual patient cells.
- From cells of compatible donors.
In vitro disease models to study the pathophysiology of diseases
One of the first repercussions of this new technology has been the observation that specific cell types (neurons for example) obtained by reprogramming and differentiation of adult cells from a given patient, present functional characteristics similar to those of the patients disease. As a result, over 20 new disease models have been described for diseases that lacked study models in the past.
In vitro disease models to investigate drug efficacy and toxicity in cells from patients with specific diseases
This method allows use of specific cell types such as cardiomyocytes or neural cells as substrates for drug screening. These cell substrates may derive from patient suffering from specific conditions or from healthy donors.
Future regenerative therapies from patients own cells or from compatible donors
La reprogramación celular permite obtener células con funciones específicas a partir de células de la piel o de la sangre. Estas células específicas serán el sustrato de futuras terapias celulares basadas en trasplantes que remplacen o regeneren células enfermas. Para que este trasplante celular no sea rechazado, las células trasplantadas deben ser compatibles con el paciente trasplantado o éste debe ser inmunosuprimido.
Cell reprogramming generates new cells with specific functions derived from skin or blood cells. These specific cells will be the substrate for future cell therapies using transplantation to replace or regenerate sick cells. In order for these cell transplants not to undergo rejection, cells used must either be compatible with host cells, or patients will need immunosuppressive therapy.
In cases in which patient-specific cells are generated by reprogramming and applied to the same patient, rejection is no longer an issue. In the case of setting up a cell-bank using healthy donors, donor histocompatibility profiles will need to match recipient ones. In order to develop new therapies using cell reprogramming, different phases of clinical research need to be completed in controlled clinical trials. During the latter, interventions are tested in patients to make sure they are safe and efficacious. It is important to point out that experimental practices should be conducted under state regulatory framework policies. Specifically, all clinical research using stem cells should first be approved by INCUCAI and be free of charge to patients. Only after an experimental therapy applied to patients has demonstrated therapeutic efficacy and lack of undesired side effects can it be established as a treatment. Currently, only one clinical trial has been approved worldwide allowing reprogrammed cells to be used as substitute cells in 2013.
Consensus prevails that this new technology will become an indispensable tool for disease research in general, as well as for preclinical drug testing and development of novel therapies. Its recent discovery combined with its high future impact and the availability in Argentina of installed capacity, makes cell reprogramming a strategic growth niche for the country.