Short Communication - (2024) Volume 9, Issue 6
Received: 02-Dec-2024
Editor assigned: 04-Dec-2024
Reviewed: 16-Dec-2024
Revised: 23-Dec-2024
Published:
30-Dec-2024
, DOI: 10.37421/2576-3857.2024.09.281
Citation: Thurzo, Zsoldos. “Targeted Therapy: A New Era of Customized Treatment.” J Oncol Med & Pract 9 (2024): 281.
Copyright: © 2024 Thurzo Z. This is an open-access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution and reproduction in any medium, provided the original author and source are credited.
Targeted therapy is a revolutionary approach in modern medicine that is reshaping the landscape of treatment for various diseases, particularly cancer. Unlike traditional treatments such as chemotherapy or radiation, which are often broad and affect both healthy and diseased cells, targeted therapies are designed to precisely target specific molecules that play a crucial role in the growth, progression, and spread of disease. This targeted approach offers several advantages, including reduced side effects, higher effectiveness, and the potential for personalized treatment regimens. The idea of personalized medicine, where treatment is tailored to an individual’s unique genetic makeup, is a driving force behind the evolution of targeted therapies.
The foundation of targeted therapy lies in understanding the genetic and molecular basis of diseases. Over the years, advancements in molecular biology and genomics have revealed that many diseases, particularly cancers, are driven by specific genetic mutations, alterations, or abnormalities in cell signalling pathways. For instance, in cancer, the uncontrolled growth of cells is often the result of mutations in genes that regulate cell division or repair damaged DNA. These mutations can activate or deactivate proteins that play pivotal roles in maintaining cellular function. Targeted therapies aim to identify these molecular targets, whether they are proteins, enzymes, or genes, and intervene specifically at these sites to halt or slow down disease progression.
One of the most well-known examples of targeted therapy is the use of tyrosine kinase inhibitors in the treatment of certain types of cancer. Tyrosine kinases are enzymes that help regulate the cell cycle and are often implicated in cancer development. Mutations in the genes encoding these enzymes can lead to abnormal signalling that drives tumor growth. Imatinib, the first tyrosine kinase inhibitor, was developed to target a specific mutation in Chronic Myelogenous Leukemia (CML) that leads to the production of an abnormal enzyme. Imatinib has transformed the treatment of CML, offering patients a more effective and less toxic alternative to traditional chemotherapy. This success story was a significant milestone, demonstrating the potential of targeted therapies to revolutionize cancer treatment.
Targeted therapies can be divided into several classes based on the molecules they target. Monoclonal antibodies, for example, are laboratorymade molecules that can mimic the immune system’s ability to fight off harmful pathogens such as viruses and bacteria. These antibodies are designed to target specific antigens found on the surface of cancer cells. One prominent example of monoclonal antibody therapy is trastuzumab, which targets the HER2 receptor, a protein that is overexpressed in some breast cancers. Trastuzumab has shown significant efficacy in treating HER2-positive breast cancer, improving patient outcomes and survival rates. Small molecule inhibitors represent another class of targeted therapies. These are typically oral medications that target specific enzymes or proteins inside cells, interfering with their function. For instance, BRAF inhibitors are used in the treatment of melanoma with BRAF mutations [1].
These drugs work by inhibiting the mutated BRAF protein, which is involved in cell signalling that drives the uncontrolled growth of melanoma cells. The use of targeted therapies such as BRAF inhibitors has greatly improved survival rates for patients with advanced melanoma, a disease that was once considered difficult to treat. In addition to cancers, targeted therapies have also shown promise in treating other diseases, including autoimmune disorders and genetic diseases. For example, targeted therapies are being explored as treatments for diseases like rheumatoid arthritis, where the immune system attacks the body’s own tissues. In these cases, biologics that target specific immune system molecules, such as Tumor Necrosis Factor (TNF), have been developed to reduce inflammation and prevent tissue damage. Similarly, gene therapies, which aim to correct or replace defective genes, are another form of targeted treatment that holds great potential for treating inherited genetic disorders [2,3].
A key aspect of the success of targeted therapy is the advancement of diagnostic technologies that can identify molecular targets in patients. Diagnostic tests, such as Next-Generation Sequencing (NGS), have enabled clinicians to detect genetic mutations and alterations with high precision, making it possible to select the most appropriate targeted therapy for individual patients. For example, in Non-Small Cell Lung Cancer (NSCLC), genetic testing can identify mutations in the EGFR gene, allowing clinicians to choose EGFR inhibitors as a treatment option. The ability to match the right therapy to the right patient has improved treatment outcomes and minimized unnecessary side effects. However, the road to widespread implementation of targeted therapies has not been without challenges. One of the primary hurdles is the development of drug resistance. While targeted therapies can be highly effective at the outset, many patients eventually develop resistance to these treatments. This can occur through various mechanisms, such as the acquisition of new mutations that allow cancer cells to bypass the effects of the drug.
For instance, in the case of EGFR inhibitors used in NSCLC, some patients develop mutations that alter the structure of the EGFR protein, rendering the drug ineffective. Overcoming resistance is an active area of research, with scientists exploring combination therapies, which use multiple drugs to target different pathways simultaneously, to prevent or delay the development of resistance. Moreover, the cost of targeted therapies is another significant challenge. Developing and manufacturing these therapies is often complex and expensive, making them less accessible to many patients. The high cost of targeted treatments has led to concerns about the affordability and accessibility of these therapies, especially in low-resource settings. As targeted therapies become more widely used, efforts are being made to reduce costs and improve access, but this remains an ongoing issue [4,5].
In conclusion, targeted therapy represents a new era of medicine, offering the promise of more effective and personalized treatments for a wide range of diseases. By focusing on the molecular and genetic causes of disease, targeted therapies allow for treatments that are tailored to the individual patient, minimizing side effects and improving outcomes. Although challenges such as drug resistance and cost remain, the potential benefits of targeted therapy make it one of the most exciting developments in modern medicine. As research continues to advance and new technologies emerge, the future of targeted therapy looks increasingly bright, offering hope for patients around the world.
None.
No potential conflict of interest was reported by the authors.
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