Translational Research and Drug Repurposing for Cancer


Many types of research investigate different cancer areas such as prevention and treatment, but one type that may not be so familiar is translational research.

With a focus on ‘translating’ basic science discoveries from the laboratory into the clinic, translational research aims to improve health outcomes and conditions for patients.

‘Translational research is the vital step between scientific discoveries and clinical practice,’ says A/Prof Nikola Bowden, a Cure Cancer alumna. ‘We need to use the huge amount of knowledge and discoveries we already have to more rapidly and cost-effectively treat cancer and to respond to situations like pandemics. It is logical to use what we already have to its full potential.’

Funded by Cure Cancer in 2010 and again in 2013, Nikola has made a name for herself in multiple areas of research: melanoma , ovarian cancer , and even COVID-19 . Back in 2010, she used her funding to help her prove that DNA repair is deficient in melanoma, and went on to confirm these findings in a large patient cohort. ‘Because of these findings we can alter the treatment to get DNA repair to work again,’ she explains.

Nikola and her team at the Hunter Medical Research Institute are using their discoveries for translational research. A breakthrough in one cancer can sometimes lead to a breakthrough in another. In fact, a variation of Nikola’s original melanoma research has been applied to work for ovarian cancer. She explains, ‘My melanoma research has progressed to a phase 2 clinical trial which is currently open and recruiting patients in Newcastle and Cairns. We’re preparing the first interim analysis to be published soon!’

As a result of Nikola’s original melanoma project using old chemotherapy drugs in a new way, she’s been able to move into the field of drug repurposing and established the Centre for Drug Repurposing and Medicines Research at the University of Newcastle.

In essence, drug repurposing is a method for identifying new uses for approved or investigational drugs, saving both time and money. An example of drug repurposing is the use of Thalidomide, a sedative originally used in 1957 to relieve morning sickness in pregnant women.

Nikola retells, ‘Widespread use of thalidomide in 46 countries resulted in skeletal defects in more than 15,000 children before marketing of the drug discontinued in 1961. Despite the severe and adverse events caused by thalidomide, in 1964 its use as a sedative for a severe form of inflammatory leprosy, erythema nodosum leprosum (ENL) led to the accidental discovery that it also completely heals ENL sores and relieves pain. World Health Organisation led clinical trials which confirmed thalidomide was an effective treatment for ENL in up to 90% of patients. In 1994, new anti-blood vessel actions of thalidomide were discovered which made it an attractive drug to re-purpose for cancer treatment.

Within 4 years, thalidomide was approved under the trade name Thalomid for the treatment of multiple myeloma. Researchers Ashburn and Thor eloquently describe the potential of drug repurposing with the quote “The lesson from the thalidomide story is that no drug is ever understood completely, and repositioning, no matter how unlikely, often remains a possibility”.’

Now an expert in the field of drug repurposing, Nikola explains, ‘A new drug requires approximately 12-16 years processing time and investment of US$1- 2 billion to achieve market approval. In contrast, repurposing a drug on average takes only 6.5 years to obtain approval and investment of US$300 million.’

‘The development of repurposed drugs is attractive both in terms of the substantial cost efficiencies it offers in comparison to drug discovery, and because therapeutic advances and new drug options for some cancer patients have been far slower than expected.’

‘Drug repurposing is an ideal way to rapidly find effective treatments and we recently published an editorial to explain that this is the ideal way to find treatments for the effects of COVID-19 while we wait for a vaccine to be developed. Similarly, we can use drug repurposing for really hard to treat cancers, while we wait for the next big drug discovery,’ Nikola says. ‘We aim to get more treatments approved for both melanoma and ovarian cancer.’

Her team recently received $2.69m from the Medical Research Future Fund to conduct a 5-year research program on Drug Repurposing for treatment resistant ovarian cancer. Their next project aims to screen 9,000 drugs to find potential new treatments for patients with ovarian cancer that currently have no treatment options.

Translational research like Nikola’s is the crucial connection between ground-breaking discoveries and clinical practice.

‘Supporting early-career researchers is a critical step to ensuring new ideas are constantly being researched for cancer,’ she says. ‘Big ground-breaking discoveries and changes to how we approach cancer come from the curiosity of these minds. Most of the Nobel Prizes awarded for medicine are for discoveries made by scientists in their 20s and 30s. The impact of the discovery often takes decades to be realised.’

Since 1967, Cure Cancer has funded 528 ground-breaking research projects like Nikola’s, giving novel ideas the opportunity to grow and develop into various hands-on practices used to effectively treat cancer patients.


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