In a remarkable advancement in pediatric oncology, a team from the International University of Florida has introduced a novel method that marries genetic testing with an innovative drug testing technique directly on tumor samples. This method, recently detailed in the journal Nature Medicine, shows significant improvement in 83% of challenging cancer cases in children, offering new hope where traditional treatments falter.

Revolutionary Approach to Cancer Treatment

The conventional treatment landscape for pediatric cancers, while seeing strides in survival rates for certain types, remains bleak for high-risk and recurrent cases. The new approach by Dr. Diana Azzam and her team addresses these gaps by introducing a method that provides rapid, precise testing results, enhancing treatment options significantly.

Dr. Azzam's method involves taking a sample of blood or tumor tissue and cultivating the cancer cells in lab conditions that mimic their natural environment in the human body. The cells are then exposed to over 120 approved therapeutic drugs, including both cancer-specific and non-cancer drugs, tested in various combinations to identify the most effective treatment for each patient. This process, remarkably, takes about a week—much faster than current genomic profiling methods.

"The team's combined approach eliminates guesswork and provides a list of the most effective drugs, helping oncologists tailor treatments that are most likely to succeed for each patient," said Thomas R. Gilarte, dean of Stempel College and a co-author of the study. This method not only speeds up the determination of effective cancer treatments but also personalizes care to meet individual patient needs more accurately.

Clinical Trial Success

The effectiveness of this innovative approach was proven in a clinical trial involving 25 children with relapsed or refractory hematological malignancies. The traditional care options for these patients had been exhausted, making the success of this new method particularly significant.

Of the patients who received personalized treatment recommendations through this approach, 19, diagnosed with various cancers, showed promising results. Six of these children received follow-up treatments, with five displaying improved survival outcomes. This contrasted starkly with other participants who did not receive personalized care recommendations—most of them showed no improvement.

Dr. Azzam hopes these findings will encourage more widespread adoption of this functional medicine approach, not just as a last resort but as a primary treatment option. "This could be the way to turn cancer into a manageable disease," she noted.

Shifting Paradigms in Cancer Treatment

The traditional cancer treatment model generally applies a one-size-fits-all approach, which doesn't work for everyone due to the complex nature of cancer. Azzam's method not only includes a genomic component, which is a staple in precision medicine, but it also assesses how living cancer cells react to multiple drugs in real-time, which significantly broadens treatment options.

The latest data shows that only a small fraction of adults benefit from genomic-driven therapies, and the numbers are even less encouraging in children. This integrated approach, therefore, represents a significant shift towards truly personalized medicine—finding the right treatment for the right person at the right time.

This innovative method developed by Dr. Azzam and her team could redefine the treatment of pediatric cancers, moving from a standard to a more targeted and effective regime, reducing the trial-and-error method in choosing the right drugs for treating young patients.