Precision Medicine Advances Target Rare and Pediatric Cancers with Personalized Therapies
Researchers are developing personalized treatment approaches for rare and pediatric cancers using genetic profiling, AI-driven dosing, and 3D-printed medications to improve outcomes while reducing long-term toxicity.
Investigators are advancing personalized treatment strategies for rare and pediatric cancers through genetic profiling, artificial intelligence, and novel drug delivery technologies. Rare cancers affect fewer than 40,000 people per year but collectively represent 27% of all cancers and account for 25% of cancer deaths.
A clinical trial known as the Investigation of Profile-Related Evidence Determining Individualized Cancer Therapy, or I-PREDICT, uses the unique genetic blueprint of each patient's tumor to craft personalized drug treatment, combining multiple drugs to target several of the tumor's key genetic drivers at once. Most tumors have multiple cancer-causing mutations, and addressing secondary molecular alterations simultaneously can improve treatment effectiveness.
The I-PREDICT approach has been successfully applied to gastrointestinal stromal tumors as well as a wider range of both rare and more common cancers. One breast cancer patient who entered the trial in 2016 is now cancer free, having been on the cusp of hospice care nine years ago.
For rare abdominal cancers, particularly those of the pancreas and gastrointestinal tract including tumors of the appendix, researchers have combined tumor-removing surgery with heated chemotherapy (HIPEC) delivered directly to the abdomen. The first use of appendix cancer tissue models to test new drugs in the laboratory led to the discovery that Palbociclib, a drug traditionally used to treat breast cancer, showed remarkable potential in treating this rare disease. As an existing FDA-approved drug with a known side effect profile, it could be moved quickly into trials.
Results published in the Journal of Clinical Oncology demonstrated Palbociclib as the first truly effective targeted therapy for appendix cancer, and possibly other cancers which share similar genetic mutations. Patients across the U.S. are starting to benefit from this discovery, and insurance companies have extended coverage for the treatment based on the report.
In pediatric oncology, more than 400,000 children are diagnosed with cancer annually, making it one of the leading causes of childhood illness and death worldwide. While survival rates for acute lymphoblastic leukemia are above 90% in high-income countries, they remain below 40% in many low and middle-income countries. Long-term survivors face risks of non-communicable diseases such as heart disease from anthracyclines, hearing loss from cisplatin, and learning difficulties after cranial irradiation.
AI-driven molecular profiling can analyze genetic, clinical, and treatment data to predict how a child will respond to therapy, adjust doses, and reduce the risk of side effects. 3D nano printing enables the production of personalized medicines on demand. A structured literature search of PubMed, Scopus, Web of Science, and Google Scholar (2005-2024) identified studies showing that across leukemia, neuroblastoma, brain tumors, bone sarcoma, and lymphoma, AI-supported platforms improved individualized chemotherapy exposure, anticipated toxicity based on clinical or pharmacogenomic markers, and assisted clinicians in modifying early treatment.
3D nano printing enabled child-friendly medicines, multi-drug polypills, and controlled-release formulations that reduced dosing errors and improved treatment adherence. Early hospital-based experience with Bayesian therapeutic drug monitoring and on-demand pediatric drug printing suggested high feasibility for real clinical settings. Genetic variability within tumors causes parts of the same tumor to respond differently, increasing the risk of treatment failure.
Most medicines are designed for adults and then adapted for children, which can lead to dosing errors, poor adherence, and harmful side effects. AI-guided dosing and nano-printed formulations enhanced precision, lowering acute and late toxicities that support healthier long-term outcomes in children with cancer, particularly when linked to disease-specific needs.