How Science Becomes Medicine

The process by which science becomes medicine is designed to minimize harm and maximize effectiveness.

There is a well-established path by which scientific discoveries are developed into new medical treatments

Clinical translation is the multi-step process of turning scientific knowledge into real-world medical treatments. The steps that scientists go through are essential to assuring a therapy is both safe and effective. They may discover that an approach that seemed promising in a culture dish, does not work in animals, or that an approach that worked in animals, does not work in humans. They may discover that a treatment effectively addresses symptoms of a disease or injury in humans, but that it carries unacceptable risks.

In this section:


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. Experiments in the lab are where scientists come to understand and test the scientific principles that underlie important medical discoveries.

Scientists in every field are taught to follow the scientific method, a process designed to acquire new knowledge in an objective manner. The basic steps of the scientific method are:

  1. Ask a question
  2. Background research
  3. Come up with a hypothesis, a proposed explanation for the question
  4. Test the hypothesis in a manner in which you can either prove or disprove the hypothesis
  5. Analyze the results of the testing
  6. Make a conclusion
Scientific Method

Though the scientific method is often presented as a linear sequence of steps, new information or thinking might cause a scientist to back up and repeat steps in the process. Not all steps take place in every scientific inquiry, and they are not always approached in the same order, thus, the scientific method is best considered as general principles.

A vital part of the research process is replication and external (or peer) review. Scientists open up 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 the understanding gained of disease. It involves the translation of this scientific knowledge into the development of potential treatments. Scientists study how these new treatments work in animals and may also test new treatments on lab-grown animal tissues or human tissues.  Just because a 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.


Clinical research and patient protections

If the results of preclinical research are promising, it may progress to clinical research, or testing in humans.

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 about Clinical Trials here.

Sometimes, in attempting new surgical techniques or where the disease or condition is rare, treatments might be tried on just one or two people outside the confines of a clinical trial.

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

In many countries, a national agency reviews clinical research for evidence of safety and effectiveness, and then approves treatments for use by patients. The manner in which drugs 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.


Buyers beware

Perhaps you’ve heard of “snake oil,” which comes from the 19th century practice of advertising a single elixir as a remedy for all sorts of ailments without clear evidence of quality or health benefit. Unfortunately, false advertising and exaggerated claims are still a problem, and unproven stem cell treatments are among the miracle cures being sold today. Be aware of these things to know if you are considering a stem cell treatment.

Snake Oil