New Study into Benefits of RNA Tumor Profiling Provides Hope for More Effective Cancer Treatment

OmiCure is excited and hopeful about the future of precision cancer treatment, thanks to a recent study conducted by the National Cancer Institute. While there is still a considerable amount of information needed to enhance this approach, this new publication supports the idea that RNA tumor profiling is headed in the right direction to improve the outcomes for cancer patients.


The National Cancer Institute Brings Precision Cancer Medicine to More Patients

Conducted by Kenneth Aldape, M.D., chief of NCI’s Laboratory of Pathology and Eytan Ruppin, M.D., Ph.d., head of NCI’s Cancer Data Science Laboratory, this novel approach of analyzing tumors is finding a way to bring precision cancer treatment to more and more patients. Through the WINTHER trial, we started to understand how examining the gene expression in patients’ tumors can drastically change the outcomes by providing more effective, targeted treatment options. This new study reiterates the idea that RNA tumor profiling is a significant advance in cancer treatment and brings a deeper level of understanding to the analysis of gene expression.

The role and DNA and mRNA

When a gene is expressed, the DNA is copied into mRNA during a process called transcription. That mRNA is then used to make protein through a process of translation. Researchers and doctors have studied precision cancer treatment to understand the relationship between cancer cell genes better to find the right treatment options for each patient individually. Until the WINTHER trial, most clinical trials focused on precision medicine were based solely on the analysis of alterations in DNA. While this did bring significant advances into the field of precision medicine, there was little success in positively impacting the outcomes for patients.


The introduction of RNA tumor profiling seemed to close the gap that existed in finding successful outcomes for patients. Indeed, the issue with RNA-based testing initially was finding ways to translate the data found into information that would be helpful for doctors treating patients. Thanks to this new study by Aldape and Ruppin, it seems they’ve found another way to translate that data and to comprehensively test it to improve doctors’ ability to treat patients more effectively.


NCI researchers take the next step in putting the translation of that data into action by accurately predicting whether patients respond to previous treatment with targeted therapy or immunotherapy. With these significant advances, researchers can reanalyze data to match more and more patients to the most beneficial treatment option for their tumor profile. Although this breakthrough advance in precision medicine is relatively new, NCI researchers are gradually incorporating this new approach into patient care at the National Institute of Health’s Clinical Center. This only complements the DNA-based methods that are already in place, bringing the entire cancer community one step closer to more effective treatment plans and better outcomes for patients.


Understanding Relationships Between Genes Helps Oncologists Predict Responses to Targeted Therapy

As more and more voices and publications surface, predicting that RNA tumor profiling is the right direction for precision oncology, understanding what these studies mean helps doctors, patients, and caregivers find new hope for effective cancer treatment plans. The human genome carries between 20-25 thousand genes. When looking at cancer cells, analyzing the relationship between those genes can help influence drug therapy outcomes. While some connections between these genes help cells survive, others are harmful. Studies find that the relationships between our genes affect the effect a drug has on a patient’s cancer. Understanding those relationships is the key doctors need to help choose the best treatment option for each patient individually.


Dr. Eytan Ruppin, M.D., Ph.D.

Dr. Ruppin has spent the past several years developing computational tools that take information on gene expression and use it to infer relationships between pairs of genes. This is a major advance in RNA tumor profiling because it serves as the missing link that translates the information collected into actionable data.

By focusing on genes hit by targeted therapies, Ruppin has been able to analyze publicly available data sets containing molecular data from hundreds of patients and research the interactions between genes across an entire genome. Of course, there’s much more work to be done. With 500 million possible gene-pair combos in the human genome, further advances will take more testing, research, and time. But this new approach certainly has us headed in the right direction.


"Understanding interactions like those could help doctors choose the best treatments for individual patients",
explained Eytan Ruppin, M.D., Ph.D., chief of NCI’s Cancer Data Science Laboratory

With 21 different clinical trials of various targeted therapies, this new approach accurately predicted patients’ responses to treatment in 17 of 21 data sets covering several types of cancer and more than 12 different kinds of targeted therapies. That’s a significant advancement in the field of precision oncology! And, although Ruppin notes that data isn’t accurate all the time, this is a clear signal that precision medicine is a useful resource for cancer patients. This new hope gives researchers and oncologists the drive to push for further testing to learn more about targeted therapy and improve the approach altogether.


Comparing the NCI’s New Approach to the WINTHER Trial

One of the best ways to test a new approach is to pit it against an established trial to see if it further enhances the treatment options available to patients. That’s precisely what the NCI did with the WINTHER trial. In the WINTHER trial, clinicians matched patients to treatment based on DNA alterations or RNA levels in the patients’ tumors. But RNA-based therapy was only used for patients with no tumor DNA alterations that could be matched to an effective treatment option. As the first clinical trial to use RNA data to influence treatment decisions, the WINTHER trial provided a significant advance in precision oncology.


But further research and testing are always needed to continue towards a conclusion treatment option for patients. The WINTHER trial used the RNA approach based on the expression of the gene or genes in a patient’s tumor targeted by each drug used. Of 86 patients, 23 responded to treatment in the WINTHER trial. At 27% success, that’s still a considerable development in precision oncology. But there’s more to be excited about with this new approach.

If this new approach guided the WINTHER trial, Ruppin predicts that 74 patients could be estimated to respond to RNA tumor profiling and targeted therapy. That’s a whopping 86% of patients. Even considering false-positive tests, Ruppin predicts that oncologists could determine which of 32 FDA-approved targeted cancer therapies would work best for 60% of patients in the trial.


What’s Next?

NCI researchers are still testing this new approach. It’s slowly making its way into use in clinical cancer trials at the NIH Clinical Center. But as NCI leaders continue to consider how to incorporate the approach into patient care effectively, the future is very bright. NCI researchers are currently collaborating to plan randomized clinical trials of this new approach. This will address questions about its reliability and solidify the potential benefits RNA tumor profiling has for cancer patients. The National Cancer Institute’s overall goal is to gradually and carefully learn more about the potential benefits of RNA tumor profiling for more effective treatment options through precision oncology.


At OmiCure, we’re excited and hopeful for the future of precision oncology. As we continue to assist the entire cancer community in the quest to find the best possible outcomes for patients, these new publications shine a light on the fact that precision medicine brings us one step closer to better treatment.