Drug Repurposing in Oncology: A Promising Avenue

Introduction

Cancer is one of the leading causes of disease burden and mortality in the world, and its presence continues to challenge health care systems worldwide. Conventional drug discovery and development are said to be costly, time-consuming, and associated with high failure rates. However, in the past years, the concept of drug repositioning has emerged as a highly potential strategy to reduce these difficulties, especially in the area of oncology. Drug repositioning, on the other hand, is the process of identifying new therapeutic uses for existing drugs that have already been approved for other conditions. This is thought to be an approach of great recent interest because it allows the reduction of time, cost, and risk in developing new cancer therapies. With safety and pharmacokinetic data available, drug repurposing will enable translational investigations from the bench and a quick move toward effective treatments for patients. In this review, I discuss drug repurposing in oncology, including key examples and mechanisms through which these drugs show their anticancer effects.

The Rationale for Drug Repurposing in Oncology

Classical drug discovery is long, with an expensive proposition running into multiple decades and billions of dollars for the development of a new drug. Cancer adds complexity and heterogeneity, with drug repurposing offering a real solution through the already large repository of drugs available for utilization, many of which have well-characterized safety profiles. The approach not only saves time and resources, but the laboratory findings can be translated into clinical settings very quickly.

In the field of oncology, a particular concern is the demand for effective therapies against the backdrop of rapid disease progression. In this area, drug repurposing is a potent method to radically shorten the period of remedial search. Repurposed molecules often treat diseases through new MOAs, which have not been targeted by standard cancer therapy till now, thus providing an assurance of an effective treatment for a cancer patient who has refractory or resistant cancer.

The mechanistic action of repurposed oncology drugs involves multiple targetings of various pathways involved in cancer progression. Few repurposed drugs exploit mechanisms concerned with their original indications, giving an anti-cancer effect. No steroidal anti-inflammatory drugs, a very common pain-relieving and anti-inflammatory agent, have been found to have anti-cancer properties by COX inhibition. However, recently, it has been shown in studies that it can target other pathways involving NSAIDs in apoptosis and immune modulation, thereby making them a potent candidate for cancer therapy.

In addition, other antiprotozoal drugs, like atovaquone, developed mainly for the treatment of parasitic infections, have promising effects in inhibiting the growth of different cancers, which may include breast cancer. Atovaquone inhibits the pathway of signaling HER2/β-catenin, which is important for the viability and multiplication of cancer cells. Through this type of dual targeting, the treatment becomes effective, and the development of resistance is unlikely.

A striking example is the use of antimalarial agents in the treatment of cancer. Pyrimethamine, an inhibitor of the dihydrofolate reductase in the parasite responsible for causing malaria, has also been identified as an anti-cancer agent for similar reasons, that is, against the same targets in the folate pathway of tumor cells. Interestingly, its recent identification as an inhibitor of EMT, which is key to cancer metastasis, has added to its credentials as a drug repositioned for cancer treatment.

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Challenges and Considerations in Drug Repurposing

While drug repurposing holds a number of advantages, it is not without its challenges. Probably the most prominent ones are related to the establishment of firm evidence of efficacy in the new indication. Although much about the safety profile of drugs for repurposing is reasonably well documented, the evidence for effectiveness against cancer has to be subjected to rigorous testing through appropriate preclinical and clinical evaluations, which constitute a major research and development investment with close collaboration among academic, industrial enterprises, and regulatory authorities.

Another challenge is the potential for drug interactions, particularly repurposed drugs in combination with conventional therapy for cancer treatment. It is therefore important for careful consideration to be taken with the scientific optimization of the pharmacokinetic and pharmacodynamic properties of the drugs, respective of the disease states they were associated with, so that they do not act as drugs of cross purposes, hence providing treatment-related adverse effects while stopping the optimization of the therapeutic outcomes.

Moreover, the process of repurposing may be full of problems regarding intellectual property since most of these used drugs have already expired their patent terms. This serves as a great opportunity for pharmaceutical companies to develop them for new indications. However, creative business models such as public-private partnerships or government incentives could help overcome these barriers as well.

Landmark Repurposed Drugs in Oncology

A few drugs have already entered the field of oncology as repurposed drugs and have proven the worth of this approach in fully realizing the great potential of repurposed drugs in the area of oncology. Mebendazole, an anthelmintic agent against parasitic worms, is now being repurposed for treatment against thyroid cancer. Mebendazole was able to demonstrate its ability to inhibit the growth of papillary and anaplastic thyroid cancer in preclinical studies via cell cycle arrest and induction of apoptosis. These findings suggest that mebendazole may be a useful synthetic lethality agent for various nematodes, especially those intolerant of the Ro5-3335 compound.

Another example of repurposing is thalidomide. Initially marketed as a sedative, thalidomide showed clinical effectiveness in treating multiple myeloma. Its mechanism of anticancer activity involves changes in the immune response and the inhibition of angiogenesis, as in other, newer blood vessels growing in tumors. Pretty importantly, its repurposing resulted in the discovery of other drugs with a similar mechanism of activity inhibition of angiogenesis in cancer, lenalidomide and pomalidomide.

Besides, aspirin, one of the most commonly used NSAIDs among them, has the prospect of lowering the risk of colorectal cancer. In epidemiological research studies, long-term use of aspirin has been associated with a significant decline in the rate and number of deaths from colorectal cancer. This is considered the effect of COX enzyme inhibition and modulation of inflammatory pathways in tumor genesis.

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Future Directions in Drug Repurposing for Oncology

Advanced technologies, such as artificial intelligence and big data analysis, could drive drug repurposing in oncology. Tools for artificial intelligence and analytics can assist  in the identification of drug candidates from huge biomedical data—genetic, proteomic, and pharmacological information. AI-driven drug repurposing platforms screen existing drugs for their potential to hit certain cancer pathways and suggest various drug combinations, all at an accelerated rate compared to manual discovery.

Further advancing the principles of personalized medicine, which are treatments designed for the genetic and molecular profile of an individual’s tumor, represents new opportunities for drug repurposing. Specific identification of biomarkers associated with the response to the repurposed drug would further enable clinical doctors to select the most appropriate therapies in service to the patient, thus increasing the chances for better outcomes with fewer side effects.

Further advancement in drug repurposing research can be achieved through collaborative efforts between researchers, clinicians, and pharmaceutical companies. With shared data, resources, and expertise, these stakeholders can collaborate in surmounting challenges towards translation into clinical settings and shortening the drug development timeline.

Conclusion

Drug repurposing is a promising avenue in the development of new therapies for cancer, rapidly and more cost-effectively than through previous traditional techniques. Such has been the case in a body of work by researchers that very rapidly explored new therapeutic applications that may be found in existing drugs with well-characterized safety profiles, bringing life-saving treatments to cancer patients sooner. The success achieved so far in the face of challenges underscores the potential of this approach to meaningfully change the cancer treatment landscape. Along with these technological advancements, drug repurposing is set to play a very important role in the fight against cancer.

References

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