Oncology R&D trends and breakthrough innovations
This report identifies the latest trends and emerging technologies in oncology R&D, providing scientific decision-makers with a roadmap of high-impact opportunities in an evolving landscape.
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Key R&D challenges for oncology
More resources are being channeled into the research and development of new cancer treatments than ever before. This growth runs parallel to the increasing incidence of cancer, which is predicted to rise globally by up to 75% by 2050. In 2023, more than 2,000 clinical trials started for new cancer therapeutics, and 25 oncological novel active substances were launched on the market, bringing the total to 192 since 2014.
While R&D transitions away from traditional chemotherapy, approaches such as immunotherapy, targeted therapies, and precision medicine are increasing in prevalence, alongside the use of AI. Despite promising advances, many clinical trials for cancer still fail, with an average approval rate of only 3.3% as of 2019. However, oncology R&D is making meaningful and significant progress, improving cancer diagnosis, treatment, and patient outcomes worldwide.
Challenge #1
Intense competition
The oncology R&D market is highly competitive, dominated by major players who produced 31 of 35 blockbuster treatments in 2020. Smaller firms face challenges such as securing funding, differentiating their offerings, and recruiting trial participants due to high competition. The preference for alliances over acquisitions in such a large market complicates independent development. Advanced technologies and digital strategies are crucial to remain agile while balancing innovation speed with regulatory demands.
Challenge #2
Complexities of solid tumors
Solid tumors present significant challenges in oncology R&D due to their complex biology, resistance mechanisms, and barriers such as dense tissue and hostile microenvironments, which hinder drug penetration. Innovative delivery systems, precise targeting, and personalized medicine are essential to effectively treat solid tumors but these methods are still developing. The heterogeneity within tumors and across patients further complicates treatment, making universally effective therapies difficult to achieve.
Challenge #3
Cancer heterogeneity
Cancer heterogeneity, the variation within tumors and between patients, complicates oncology R&D by hindering the development of universal therapies This variation occurs at multiple levels, evolves over time, and may lead to drug resistance. It challenges personalized treatments, requiring constant adaptation and biomarker identification. Technologies such as circulating tumor DNA analysis offer promise for early diagnosis, improving precision therapies and patient outcomes.
The role of science clusters in advancing oncology research
Oncology research is at a critical juncture, driven by the increasing demand for personalized treatments and rapid technological advancements. Science clusters play a critical role in this landscape, helping to facilitate early-stage research discoveries and fostering collaborations between academia, clinicians, patients, biotechnology firms, and pharmaceutical companies.
Top oncology innovations
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1. TIGIT-targeting antibodies for new possibilities in immunotherapy
It is possible for cancer cells to evade the immune system by exploiting the TIGIT receptor on T-cells, inhibiting their ability to defend against tumors. Many cancers overexpress CD155, a protein that binds to TIGIT and reduces immune response. Inhibiting this CD155/TIGIT interaction provides a potential avenue for improving cancer treatment outcomes by restoring the immune system's effectiveness.
At Bio-Rad , researchers have utilized Bio-Rad's Pioneer™ Antibody Discovery Platform to create ten diverse monoclonal antibodies, which demonstrate high sequence diversity and potent inhibition of TIGIT/CD155 interaction. Preclinical trials have shown positive results and the team is seeking out-licensing opportunities to help further develop cancer therapies.
2. Non-viral delivery of RNA therapeutics for hard-to-treat cancers
RNAi has potential in cancer treatment through silencing disease-causing genes. It targets specific mRNA to block protein production and subsequently address mutations and pathways driving tumor growth. Despite its potential, challenges like effective delivery and safety concerns have resulted in limited clinical success, demonstrating the need for new solutions.
At University College Cork , researchers have developed the CycloVector system, which delivers siRNA safely and precisely to tumors. Their non-viral platform demonstrates efficacy in cancers like prostate cancer and acute myeloid leukemia, with minimal toxicity. By silencing oncogenes and modifying tumor microenvironments, RNAi therapies can enhance existing treatments and provide targeted options for hard-to-treat cancers, with applications across precision oncology. The team are seeking a partner for co-development and licensing. have identified a novel, non-toxic therapeutic approach targeting a long non-coding RNA that inhibits angiogenesis in diabetic retinopathy and related ocular diseases. This non-surgical solution is more effective and stable than current anti-vascular endothelial growth factor (VEGF) therapies, offering long-term relief by reversing pathological progression. The team is seeking licensing, collaborations, and partnerships.
3. Harnessing the acidic tumor microenvironment to improve drug specificity
Traditional antibody-based cancer treatments risk off-target effects by attacking antigens on healthy cells. However, as the tumor microenvironment is acidic, this presents an opportunity for the development of antibodies with heightened tumor specificity through pH-dependent binding interactions.
At the University of Texas at Austin, scientists have developed an antibody that binds to CD16 in tumors in an acidic environment typical of tumors (pH 6.5), but has weaker affinity at normal pH (7.4). Their approach improves tumor specificity and reduces adverse effects by avoiding healthy cells. Their method could significantly enhance precision oncology and the team is seeking a partner for co-development and commercialization.
4. Restoring protein production in genetic diseases
Restoring protein production is crucial in genetic diseases and cancer, where mutations disrupt essential cellular functions. Over 2,000 disorders, including cystic fibrosis, Duchenne muscular dystrophy, and hereditary cancers arise from PTCs in a wide range of genes. Treatments such as gene editing or small-molecule drugs may have limitations such as toxicity and off-target effects, providing a need for a new approach that can restore proteins without harming cells.
At UMass Chan Medical School , a team of scientists have developed a therapy that targets the suppression of PTC mutations. Their mutation-specific antisense oligo approach minimizes toxicity while enhancing protein restoration, holding the potential to revolutionize treatments for over 2,000 genetic disorders and cancers. The researchers are seeking partners for development and licensing.
5. An RNA-based platform for developing cancer treatments
At the heart of molecular biology is the transcription of DNA and translation of mRNA, generating functional proteins for any living cells. RNA-based disease mechanisms and therapeutics have become an important emerging field based on the landmark development of mRNA vaccines against viruses,such as COVID-19.
A team at the University of Rochester have developed a platform to design novel translation-manipulating ASO compounds to target novelmRNAs for testing their therapeutical potential. Their platform offers the opportunity for drug developers to curate novel RNA-based therapeutics for cancer, undruggable targets in cardiac hypertrophy and organ fibrosis, among other human diseases like Huntington’s. The Rochester scientists are seeking exclusive licensing of their technology.
6. Highly selective kinase inhibitors for a range of cancers
Kinase inhibitors used in cancer treatment often lack selectivity, leading to off-target effects and resistance, particularly in therapies targeting RET, BTK, and EGFR. Existing cancer drugs such as Ibrutinib and Osimertinib are limited by side effects or reduced efficacy against resistant mutations. There remains a clear need for next-generation inhibitors that offer both potency and precision.
Researchers working in the Gustafson Lab at San Diego State University have developed a novel class of kinase inhibitors that useatropisomerism to enhance selectivity while maintaining strong anti-cancer activity. Their lead compounds have shown promising results in preclinical models of NSCLC, thyroid cancer, breast cancer, CLL, and other cancers. Notably, the team’s EGFR-targeting asset selectively inhibits the triple mutant without affecting healthy cells. The team areseeking partners to advance development and bring their highly targeted therapies to patients.
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