Clinical Translation
Clinical translation is the multi-step process of turning scientific discoveries made in the laboratory into real-world medical treatments. This process can take years to decades and involves testing a potential new treatment in a series of experiments to assess its safety and effectiveness. When tested on people in the context of a rigorous clinical trial, many possible new treatments fail to be proven safe and effective.
Basic Research
This process starts in a laboratory with basic research. Basic research involves figuring out how living organisms, from the cellular level up to the whole animal or person, work and also what can go wrong in disease or injury. Scientists develop hypotheses (ideas that can be tested by experimentation), perform tests to determine whether the hypotheses are correct or incorrect, analyze the results, and make conclusions. These experiments in the lab are where scientists come to understand and test the scientific principles that underlie important medical discoveries and are pivotal for achieving real-world medical treatments.
A vital part of the research process is replication and external (or peer) review. Scientists share their methods, results and conclusions to the scrutiny of outside experts, typically through publication in peer-reviewed journals. Other scientists replicate the same experiments to reach the same outcomes. In this manner, scientists regularly collaborate with and build upon the discoveries of their peers.
Preclinical Research
Preclinical research builds upon the findings of basic research and applies this knowledge into the development of potential treatments. Scientists study how these new treatments work using laboratory models of disease, often involving cells, lab-grown tissues and organoids, and animals. Just because treatment shows promise in an animal, however, does not mean it will be effective in a human, which is why clinical trials are so important. Preclinical research should be reviewed by experts, protocols and results published in peer-reviewed journals and be reproduced, ideally by an independent laboratory, before a new experimental treatment is tested on patients in clinical trials.
Clinical Research
If the results of preclinical research demonstrate that the new treatment is likely to work and be safe, permission is sought from regulatory authorities and ethical review boards to conduct clinical research, or testing in humans, which is completed through clinical trials. In some cases, new experimental treatments might be tested on a very small number of people before a clinical trial is started. Regulatory structures vary across jurisdictions and impact how investigational treatments are tested. A clinical trial is a regulated research study designed to establish where a potential new treatment reliably produced the intended medical benefit and is safe.
Clinical trials start with a small number of people and are focused on testing safety. As the procedures are perfected and the risks evaluated, the number of participants is gradually increased and the effectiveness of the treatment is more closely examined. Learn more.
During the clinical trial process, there are a number of checks to protect the rights of patients.
Fundamental to the process are:
Monitoring of experimental treatments for patient safety and ethical practice. Before beginning, trials should be carefully reviewed by a group of people who together have broad expertise and experience in research, medicine and ethics. These groups, often called Institutional Review Boards (IRBs) or medical ethics review committees, evaluate a number of factors, including the potential risks weighted against the potential benefits.
Oversight by regulatory agencies. National oversight agencies, such as the European Medicines Agency (EMA), the U.S. Food and Drug Administration (FDA) or Japan’s Pharmaceuticals and Medical Devices Agency (PMDA), authorize and monitor the development of new treatments. The nature of regulatory agencies and their responsibilities vary from country to country, but most enforce a code of conduct or guidelines for researchers and clinicians to follow to promote safe and effective medical practice.
If you are thinking about a clinical trial for yourself or a loved one, learn more about things you should consider here.
Approval for Use
Once the clinical trial data are collected, a national or regional regulatory agency, for example the EMA, FDA, PMDA, or TGA, will review the data to determine if the effectiveness and safety of the treatment was formally demonstrated, and should or should not be approved for clinical use for particular diseases or conditions. The manner in which medical treatments are marketed is also regulated to ensure companies do not make health claims related to their products that have not been proven through the trial process.
However, the field of stem cell science is new and rapidly changing, and regulation is still catching up. There may come a time when stem cell treatments are regulated consistently by governments across the world. Until such time, people investigating stem cell treatments need to be aware of what is and isn’t regulated in the countries in which they seek treatment. A lack of regulation does not constitute approval or suggest safety or effectiveness. For example, clinics may offer autologous (from your own body) stem cell treatments, which may not be subject to oversight if the cells are minimally manipulated. However, cells taken from an individual are not necessarily safe for use in or as therapy for that same individual.
How do stem cell treatments differ from traditional drugs on the market?
Treatments that use stem cells or stem cell-derived cells are largely new and there is a lot that we still need to learn. There are particular challenges in preparing stem cells for therapeutic use. Unlike traditional prescription drugs which are synthesized from standardized chemical compounds, stem cell treatments are developed from living cells. Ensuring the uniformity, efficacy, and safety of the stem cell treatment requires new and specialized laboratory tests that in many cases are specific to the cell type being used.
For most diseases, it is still being determined which cells will work best to repair a particular damaged or diseased tissue, how to get those cells to the right place in the body, how many cells are needed, and how to ensure that they will survive in the body and perform the desired function.
Furthermore, side effects need to be documented and reported to regulators, and long-term safety must be determined, since transplanted cells may remain for many years, potentially a lifetime, in patients’ bodies. Potential patients should note that many clinics marketing unproven stem cell “treatments” do not have rigorous standards in place for monitoring and reporting side effects, nor do they have long-term plans to monitor the health of their patients or determine whether they are experiencing unanticipated side effects as a result of the administered unproven stem cell “treatment”. Careful monitoring and extended follow-up of patients who receive stem cell treatments is extremely important. These studies are necessary to reduce physical, psychological, and/or financial harm for current and future patients.