Stem cells are characterized as unspecialized cells of the human body, capable of differentiating into specific cells and having the ability to self-renew. They can either originate from embryos and adult cells or develop from other cells through the use of reprogramming techniques.
Early efforts to harness the potential of stem cells for disease treatment were predominantly focused on regeneration and the potential to repair tissues via cell therapies. However, as technology continues to advance, the focus is now transitioning to the use of stem cells in drug discovery applications, including toxicity testing, disease modeling, target identification, and various others. The pivotal role and applications of stem cells as drug discovery and development tools provide the global stem cell market with lucrative growth opportunities, expected to progress with a CAGR of 8.88% during the forecast period of 2022 to 2030.
Since stem cells offer the remarkable capacity to generate an unlimited source of disease-relevant cell types, they are rapidly becoming an invaluable part of the drug discovery process.
In this blog, we discuss how stem cells are being utilized in innovative ways to improve drug discovery – spanning biotech start-ups, large pharmaceutical companies, as well as academia.
The Unique Role of Stem Cells in Drug Discovery & Development
The distinct features of stem cells, including multipotency and self-renewal, have enabled both industries and academia to evaluate the use of stem cells in various disorders and diseases, in addition to medical problems. These include osteoarthritis, hematopoietic stem cell transplants, hearing loss, diabetes, multiple sclerosis, organ transplantation, spinal cord injury, and others. Moreover, according to Inkwood Research, the drug discovery and development segment under the application category is expected to grow with the highest CAGR of 9.83% during the forecast period.
An essential part of determining a drug’s safety is analyzing its cardiac toxicity. This factor refers to the possible side effects that a drug can have on heart function, including arrhythmias and sudden death. Accordingly, to test for toxicity, embryonic or tissue-specific stem cells from healthy individuals and patients with diseases can be collected and developed into hepatocytes, neurons, or cardiomyocytes.
Although researchers attempt to test for possible cardiotoxic effects, they are not always 100% accurate. Moreover, drugs can successfully pass numerous tests for safety; however, they still have chronic effects that cannot be discovered without the use of a human cell system. As a result, stem cells help researchers better predict long-term consequences associated with drug use, with lesser risk to real patients. In this regard, leading companies such as AllCells LLC (United States) offer products that are utilized in the avenue of drug discovery and toxicity testing.
Improved Disease Models:
A significant advantage of stem cells in drug discovery is that they produce better models of drug reactions and human disease, compared to animal models. While rates and mice are generally used in medical research, human beings have notably different and intricate bodily processes than rodents. A difference in heart rate, for instance, can lead to exponentially different drug interactions in a human versus a rodent. This is also a reason why reports of medical breakthroughs after rodent studies do not often impact real medical practice, even when the same principles are applied to human beings.
On the other hand, by using stem cells from a particular patient population, scientists can assess a drug in a relatively more specific application setting. This ensures that drugs are tested in conditions closer to real treatment, thereby making the entire process faster, more efficient, and affordable.
Target identification is defined as the process of recognizing a molecular target that has the potential to be modulated by a therapeutic agent. Stem cell-based models of disease provide a quicker and often more accurate way of investigating new disease mechanisms, leading to a greater understanding of the condition’s molecular basis.
Aligning with this, numerous large-scale academic collaborations have been established to gather biomedical data from induced pluripotent stem cells (iPSCs). A core example constitutes the Human Induced Pluripotent Stem Cell Initiative, where genomic, proteomic, transcriptomic, as well as phenotypic data were amassed from thousands of healthy and disease-associated iPSC lines. This open-source platform aims to provide researchers with an international resource that can be utilized to identify molecular targets that are disease-specific.
Future of Stem Cells
As the research on stem cells progresses, key players are set to recognize the value of stem cell models. Several pharmaceutical leaders are already competing in order to bring stem cell models up to speed. For example, while Roche contracted CDI for supplying iPSCs to accelerate the identification of new drug candidates, Bristol-Myers echoed the move through the acquisition of iPierian, a biotechnology company that develops novel therapies for neurodegenerative disease, for €675M.
A key advantage of iPS cells is that they can be utilized to make models specific or particular to individual patients. Moreover, the use of stem cell banks from a wide variety of genetic backgrounds would also enable the personalization of treatments to every patient’s individual cellular and genetic makeup. iPS cells also have an intrinsic capability for indefinite self-renewal, thus providing them with the potential to adopt different cellular fates through differentiation.
In this regard, autologic stem cell therapy, used for the treatment of certain types of leukemia, lymphomas, and multiple myeloma, is expected to be the fastest-growing treatment type during the forecast period. The segment’s notable growth is accredited to the rising demand for personalized and customized medicine, in addition to a rapid rise in the prevalence of chronic disorders.
Furthermore, as per the research team at the Johns Hopkins Kimmel Cancer Center, an effective treatment for one cancer could prove to be effective in another with an equivalent genetic signature, owing to the genetic variability between cancers. Accordingly, by sequencing tomors’ RNA and comparing the sequence to larger databases of cell lines, the researchers were able to match patients with specific pathologies with identified effective treatments. Such developments are set to boost the growth of the global stem cell market over the upcoming years.
What are the key restraints faced by the global stem cell market?
The absence of a well-defined regulatory framework for stem cell therapy, the high costs associated with stem cell procedures, ethical issues and social complexities, as well as less responsiveness in developed economies, are some of the key restraints faced by the global stem cell market.
What is the potential of stem cells in the avenue of treating neurological disorders?
Stem cells offer a potential treatment for neuro-generative diseases by achieving stem cell-based nerve cell replacement, in addition to the repair of the central nervous system. Moreover, stem cells also help modulate inflammation in the host environment or disease process.