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Precision Medicine: Targeted Therapy in Paediatric Oncology Patients

For several decades, cancer has been one most of the devastating diseases affecting paediatric patients. A huge number of clinical trials and research papers are aimed at solving the puzzle of cancer; however, most scientists and healthcare providers are often shocked by the unpredictable attitude and response of this disease.

Nevertheless, huge progress has been made in this field as about 50 years ago, the 10-year survival rate didn’t exceed 20% (patients < 20 years old); but nowadays, it has reached 83%. This has been made possible by the efforts of cooperative group protocols and multidisciplinary treatment.1 However, the toxicities and adverse effects exhibited by chemotherapeutic agents remain a barrier while treating paediatric oncology patients. In such circumstances, precision medicine can help us deal with the above issues.2

In 2015, Mr Barack Obama, the president of the United States, launched the “Precision Medicine Initiative”.3 Furthermore, he described it as being equivalent to the first moon landing as it aimed at providing individualised care to cancer patients.1

What is Targeted Therapy? And What are its Advantages?

Targeted therapy depends on targeting unique receptors or proteins in the malignant cells, thus leading to fewer chemotherapy-induced adverse effects.

In this regard, a study published in the Oncology Times aimed at evaluating the efficacy of targeted therapy in paediatric oncology patients with poor prognoses.4 The trial included 149 children with relapsed, refractory, and progressive high-risk malignancies (survival rate: less than 20%; median survival: 9.5 months). By using a particular algorithm, the paediatric oncologists could identify twenty patients with very high-priority targets who could benefit from targeted therapy. After receiving the appropriate targeted therapies, these children showed longer progression-free survival than other children (204.5 days versus 114 days).

Thus, to achieve the highest benefit from the targeted therapy, we would have to focus on developing the diagnostic tools to facilitate identifying the highest priority targets in each patient.

But although targeted therapy can achieve better results, there are only a few approved targeted therapies available for paediatric oncology patients. Thus, the American Society of Clinical Oncology (ASCO) urges the scientific community to undertake precision medicine research and treatment approaches as a critical research priority. But even as it does so, we must be aware of the various challenges faced while developing and using targeted therapy in the paediatric population.5

Challenges of Including Paediatric Population in Clinical Trials

To include paediatric patients in clinical trials is not an easy task. According to the European Society for Medical Oncology (ESMO), the legal age in Europe for participation in clinical trials is above 18 years. However, it’s lowered to 12 years in the USA.6