How Scientific Discoveries Become Everyday Medicine

19/09/2016

To become real-world medical treatments, scientific discoveries made in the laboratory follow a well-established path to assess their safety and effectiveness.

Today, you can find stories about new breakthroughs in stem cell therapy or developments in regenerative medicine in nearly every important newspaper and media blog around the world.

Positioned alongside real-world events that are happening right now, such as political elections, religious conflicts, and the Olympics, they can sometimes appear to be a lot closer to reality than they actually are.

Down in the basement of some cutting-edge research institution, stem cell investigators are conducting lab studies on in vitro or animal models. Depending on the outcome, they progress to preclinical research and multi-stage clinic trials in human subjects. During which, over the span of a few years or even decades, they publish their results in scientific journals.

At various stages along this pathway, the progress of the scientist’s research makes its way to our screens via journalists who translate the results reported in publications into a more accessible language.

And so we hear about incredible findings that could potentially cure a life-threatening disease or reverse a debilitating illness. But what was the sample size of the study? Has it even been tested in people? Has it made its way through rigorous clinical trials and proven to be safe and effective? Do we know what adverse effects it has over the long-term?

These questions are somewhat overlooked by media organisations whose focus is to generate hype and buzz with their stories, leaving readers and patients in the dark about when they’ll have access to new personalised medical treatments. But with a little understanding of the clinical pathway, from basic research and preclinical research to clinical trials and approval for use, the public can decipher for themselves how far away discoveries really are from becoming a reality.

Step 1: Basic Research

All scientific discoveries were once a principle or hypothesis which scientists needed to test.

Basic research involves experiments in the lab, from the cellular level up to a whole animal or person, in which scientists can test their principles and determine how they relate to disease or injury.

The basis of this part of the process is the scientific method: a set of techniques for investigating ideas and theories and acquiring new knowledge in an objective manner.

There are six basic steps of the scientific method:

1. Ask a question
2. Do background research
3. Create a hypothesis: a proposed explanation for the question
4. Conduct an experiment and test the hypothesis in a manner in which you can either prove or disprove the hypothesis
5. Analyse the results
6. Make a conclusion

Scientists may introduce new thinking or find new information which causes them to back up and repeat certain steps, and so they act more as a general set of principles than a strict linear sequence.

After basic research, scientists share their methods, results, and conclusions for external or peer review. This is a vital part of the process and the first stage in which the wider public may become aware of the research, typically through publications in peer-reviewed journals like Cell Stem Cell, Nature, and Regenerative Medicine.

Step 2: Preclinical Research

Preclinical research is where scientists apply knowledge attained from basic research toward the development of potential treatments.

It’s basically the gateway into clinical trials. Scientists study how the accrued knowledge translates to lab-grown animal or human tissues and understand more about its effectiveness against disease. The studies are generally small-scale and therefore if a discovery has the potential to cause harmful or adverse effects it can be safely managed and adjusted. If the treatment shows promise it does not mean it will be effective in humans — this is the importance of the clinical research stage.

Step 3: Clinical Research and Trials

Given the results from the previous stages show promise, scientists progress to small clinical trials with a limited number of participants and begin safety testing.

The focus here is to evaluate the level of risk and perfect the procedures before progressing to trials with larger groups of participants. In some cases, such as if the testing involves a rare disease or new surgical techniques, scientists may perform treatments on smaller cohorts of just a handful of people outside the confines of a clinical trial.

As this is where people become involved, there are a number of procedures in place to ensure their protection. Any trial set to commence is reviewed by a group of experts in research, medicine, and ethics, often known as Institutional Review Boards (IRBs), to evaluate all the factors and gauge the potential benefits against any risks.

Clinical trials are also overseen by regulatory agencies such as the as the European Medicines Agency (EMA) or the U.S. Food and Drug Administration (FDA). They are the organisations which monitor and authorise the development of all new treatments, presenting a set of guidelines and code of conduct for researchers and clinicians to follow to ensure safe and effective medical practice.

Step 4: Approval For Use And Marketing Authorisation

Clinical research is reviewed by a national agency for safety and effectiveness before medical treatments are finally approved for public use. There is also a strict regulation process which companies go through to make sure the health claims relating to their products are proven and backed up by clinical data.

However, this isn’t to say treatments are not able to be marketed without approval from regulation authorities. In the stem cell therapy market which is rapidly expanding, many products and treatments are already available to purchase in several different countries.

Unfortunately, as the demand and promise is so high from individuals suffering from a wide range of ailments and injuries, stem cell therapy is seen by many companies as an opportunity to make some easy money. And as regulation is not yet widespread, exaggerated claims and false advertising is something that consumers and patients need to be aware of before seeking treatment.

In the not too distant future, it’s likely we’ll see stem cell therapies regulated consistently by governments around the world. But until that time, extra care needs to be taken. This even goes for autologous treatments which use stem cells from your own body. If a treatment is unregulated, it may be effective, or it may not be — without it being independently tested and assessed there is no way of knowing.

By understanding the clinical pathway, you’ll become a savvy patient able to analyse the figures and data for yourself. That means coming to well-informed decisions about the proven effectiveness of a treatment, and, whenever you see a breakthrough in stem cell therapy on the news, understanding how close or far away it really is.

Are you prepared for a future of personalised stem cell therapies? Contact us today to find out how we can help safeguard the health of your children by storing stem cells from milk teeth.