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Clinical Trials Today: Recent Milestones in Rare Cancer Research

By Renee Butler
On March 16, 2021

Cancer research has evolved exponentially over the last two centuries. The first time radiation was used to cure cancer happened in 1903. Less than 100 years later, cancer preventatives were receiving approval. However, rare disease is more involved. In our last article, we looked at rare cancer research in detail. In this article, we will discuss rare oncology research today and moving forward.

 

Rare Cancer Research in Practice

The year 2020 saw many changes and breaking news stories, but one that may have missed your radar is the Pan-Cancer Analysis of Whole Genomes study. It was a consortium that analyzed over 2,600 genomes covering nearly 40 types of cancer. These researchers matched those cells with normal tissues to determine the changes that happened at a molecular level. The end analyses help identify how cancers grow and spread. They also extended the genomic analysis to encompass the complete genetic composition of tumor cells.

The Pan-Cancer Analysis of Whole Genomes is not the only effort underway. There is also the International Rare Cancer Initiative, which began in 2011. It was formed through the collective efforts of several agencies, including the National Cancer Institute (US), Cancer Research UK, and the European Organisation for Research and Treatment of Cancer. The IRCI has a mission to test rare cancers with testable hypotheses that also have international interest and no existing forum. To date, they have completed several trials, including ones in high-grade uterine sarcoma and small bowel adenocarcinoma.

 

The Future of Rare Oncology

Collaborations are most certainly the future of rare oncology research. With so few people affected by specific rare cancers, the years it generally takes to receive a diagnosis (5.6 years in the UK and 7.6 years in the US), and the survival rate of those with such cancers, working together is important.

Technology

Tumor genome sequencing has evolved to the point that it can be conducted in a research or clinical setting. That fact allows oncology therapeutics to become uniquely targeted, but it also helps add to the small-molecule libraries that allow researchers to develop cancer treatments in a systematic way. Furthermore, having a full characterization of tumors on genomic and cellular levels with both germline and somatic variation can help determine causative variants as well as opportunities for therapeutic treatments. This dataset is essential for determining rare cancer predispositions. As such, there are several efforts underway to make that information widely available, including The Global Alliance for Genomics and Health, the Matchmaker Exchange (www.matchmakerexchange.org), and the Genomic Data Commons (GDC) (https://gdc.cancer.gov).

Large-Scale Studies

Large-scale studies of some common cancers are also proving beneficial to rare oncology research. They provide datasets that make it easier to interpret profiles of rare cancers. The benefit here is that these studies can help identify opportunities for targeted cancer treatment that would have been overlooked otherwise.

Online Communities

Another factor impacting the future of rare oncology research is the development of online communities. These platforms provide a place for rare oncology patients to organize and discuss their cancers. They can discuss treatments, and therapeutics, sharing their experiences. Groups may determine best practices in this way as well as share opportunities to participate in upcoming research studies.

Going the Distance to Create a Healthier World

As a contract research organization, Pharm-Olam is committed to conducting rare cancer studies that leverage these opportunities whenever possible. We have strong connections with advocacy groups as well as the technology and study capabilities to develop meaningful conclusions from a variety of study structures.

Do you have a forthcoming rare oncology study? Contact our team for a proposal today. We can help you understand the potential pitfalls in your current study protocol and develop strategies for mitigating those issues. Together, we can deliver a successful rare oncology study for your organization.

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Tags: Rare & Orphan Disease, Oncology-Hematology Disease

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