Published by the Students of Johns Hopkins since 1896
June 15, 2024

The understated value of clinical research

By DUY PHAN | April 6, 2017

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justinling/Cc-By-2.0 Clinical trials help determine the efficacy of drugs developed in labs.

uroscience bench lab research is currently very trendy. We have so many diseases affecting the nervous system that lack satisfactory treatments, and such treatment inadequacies stem from a poor understanding of disease mechanisms. Therefore we pour money and funding into bench lab research in hopes of discovering the biology of disease that can be used as the basis for developing new medical breakthroughs.

With all that effort and investment into neuroscience research, we’d think that most brain diseases would be eradicated by now. We all know that is far from the truth. Every day we read newspaper headlines about new treatment strategies for diseases such as Alzheimer’s and Parkinson’s, but very few of these lab findings actually even make it to the clinical trial stage. Once in clinical trials, even fewer survive the test of proving efficacy in human patients.

Neuroscientists might comment that the nervous system is complex and therefore that even more funding is still needed to better understand neurobiology.

Although I would certainly welcome more funding into neuroscience research, it is easy to understand why non-scientists would be upset: What are scientists doing with the millions and millions of dollars without producing tangible results? For results-oriented administrators and government officials, the lack of findings can seem like a reason to cut off research funding.

Is there a way that we can increase research productivity and get more bang for the buck? While funding for basic research is very important, I contend that clinical research is a major source of untapped potential that should be getting more attention.

What is the difference between basic research and clinical research? Basic research involves the lab activities you normally think of, concerned primarily with answering scientific questions about natural phenomena and making fundamental discoveries. Clinical research primarily concerns human patients, and it includes clinical trials of newly developed medications and retrospective studies analyzing existing patient records to answer clinically relevant questions.

Because of the focus on human patients rather than purely scientific questions, clinical research can produce data that immediately impact patient care. For example there are more than 20 antiepileptic drugs available to treat seizures. Even though these drugs are still not entirely satisfactory, clinical research can determine which population of patients each type of antiepileptic drug benefits most.

Establishing such patient stratification paradigms will certainly allow epileptologists to give patients the best possible medication treatment for the time being while waiting for better treatments currently being developed by basic neuroscience lab research.

More funding for clinical research can actually also tremendously help basic research. For example, in Fragile X syndrome (the most common genetic cause of autism), research progress in preclinical understanding of the disease has led to numerous clinical trials of newly developed therapeutics over the past several years. However these clinical trials have seemed disappointing, since these therapeutics failed to show improvements in primary endpoints.

Do such “failures” mean that the preclinical hypothesis about the biology of the disease has been proven to be wrong? That is certainly one way to look at it, but another interpretation is that shortcomings in the design and implementation of clinical trials masked areas of positive response. Indeed it is exceedingly difficult to objectively quantify complex responses, such as social behavior and aggression.

These difficulties make it challenging to assess whether the rationally designed therapeutics are truly beneficial and, therefore, whether the scientific basis of treatment from basic research is translatable to humans. More funding for clinical research into developing better ways to assess treatment responses will help tremendously.

Yes, we do need more funding for basic neuroscience research. However I believe that neuroscience research findings will be much better complemented with increased attention and funding for clinical research.


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