How New Cancer Treatments are Created: Translational Research

Ever wondered how do scientists find new treatments for cancer? The answer lies in understanding the process of translational research.

Translational cancer research is defined as the process of applying discoveries generated during research in the laboratory, and in preclinical studies, to the development of trials and studies in humans. This is based on the understanding that cancers involve specific gene mutations (alterations in the genetic material, which is DNA) that manifests as altered RNA and proteins in the cells (DNA--> RNA--> Protein).

The process of translational research consists of three main steps.

Step 1: Identification of molecular signatures

Like we have unique signatures, so do cancers. The first step of translational cancer research involves finding those signatures in one type of cancer, for example, breast cancer. The patients are then examined for the presence of certain molecular signatures also called biomarkers (specific sequences of DNA and RNA, and proteins), both in the solid tumor tissues obtained by surgery (either biopsy or resected tumor), and circulating tumor particles in the blood samples (liquid biopsy).

Image credit: Ochoa S et al. doi: 10.3389/fonc.2020.00845

The identification of molecular signatures from tissues requires highly specialized methods for their detection, such as polymerase chain reaction and electron microscopy for DNA/RNA sequences and immunohistochemistry for proteins. For instance, there are many specific molecular signatures, such as estrogen receptor (ER), progesterone receptor (PR) for breast cancer.

Step 2: Finding drug targets

The same set of patients is also studied over a period of time for disease progression and death, and response to existing treatments (chemotherapy, immunotherapy, hormone therapy, etc.). Not every molecular signature that is identified through the above process can become a drug target. Only after comparing the signatures with the disease progressions, patterns are identified that link the specific molecular signatures with disease progression and risk of death. One signature alone, or more than one signature in combination may indicate a good or a bad outlook (prognosis) of the disease and thus help to identify the most suitable drug targets.

Step 3: Drug development

Considering the above-identified patterns and drug targets, experimental drugs are developed either alone or in combination so as to reduce the risk of disease progression and death. The development of experimental drugs usually involves various stages including evaluation of the drug in preserved cancer cell lines in a laboratory, administration of the drug to animal models of cancers, and finally testing in human patients with cancers, which are known as clinical trials.

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Experimental drugs that successfully clear clinical trials are then licensed/approved for use in that particular variety of cancer harboring that particular molecular signature (personalized medicine). This approval for use is provided by the regulators, such as FDA (US), EMA (Europe), CDSCO (India), etc., first in that particular country where the human experiments were conducted, and later on in other locations depending on the local laws.

References and suggested reading:

1. From bench to bedside: the growing use of translational research in cancer medicine

2. Translational research—the need of a new bioethics approach

3. Defining Translational Research: Implications for Training

About the author: Dr. Naval Asija is a licensed MBBS Physician from India. MBBS is the equivalent of the MD degree offered by international medical schools. He is based in Delhi, India, and works as a medical writer, editor, and consultant. He supports medical researches as an author's editor, medical communication companies involved in medico-marketing activities, and medical technology companies in improving their products. He can be contacted via his LinkedIn Profile: https://www.linkedin.com/in/navalasija/