We use cookies to ensure our website works properly and to personalise your experience. Cookies policy
1Research Scholar, Yashodeep College Of Pharmacy Saralgaon – Nagaon Road, Saralgoan, Tal – Murbad, Dist – Thane 421401.
2Assistant Professor, Yashodeep College Of Pharmacy Saralgaon – Nagaon Road, Saralgoan , Tal – Murbad , Dist – Thane 421401.
3Principle, Yashodeep College Of Pharmacy Saralgaon – Nagaon Road, Saralgoan , Tal – Murbad , Dist – Thane 421401.
Precision oncology is an approach in which cancer treatment is selected according to the molecular, genomic and clinical characteristics of an individual patient and tumour. Artificial intelligence (AI), including machine learning, deep learning and natural language processing, can analyses large and complex datasets generated by genomics, pathology, radiology and electronic health records. AI may help identify actionable biomarkers, predict treatment response and resistance, support drug discovery, match patients to clinical trials and anticipate adverse drug reactions (ADRs). This review describes the principles of AI-driven precision oncology, major biomarkers, pharmacogenomics, mechanisms of selected targeted and immune therapies, drug-specific adverse effects, AI-assisted toxicity prediction, clinical workflow, advantages, limitations, ethical considerations and future directions. Selected therapies including osimertinib, trastuzumab, dabrafenib/trametinib, olaparib and pembrolizumab demonstrate how molecular findings can be translated into individualized treatment. The future of oncology is likely to involve multimodal AI combined with longitudinal molecular monitoring and multidisciplinary clinical decision-making.
Cancer is a heterogeneous group of diseases in which genetic and epigenetic alterations drive uncontrolled cell growth. Patients with the same anatomical cancer type may have substantially different molecular profiles and may therefore respond differently to the same treatment. Conventional treatment based mainly on tumor site and stage can consequently result in variable outcomes. Precision oncology attempts to address this problem by incorporating molecular information into therapeutic decision-making. [1,2]
AI provides computational methods for analyzing high-dimensional data that may be difficult to integrate using conventional statistical approaches. In oncology, AI can learn patterns from genomic profiles, digital pathology, radiological images, laboratory results, treatment records and clinical outcomes. The objective is not simply to predict disease but to generate clinically useful information for selecting, monitoring and adapting therapy. [3,4]
The development of targeted therapies and immunotherapies has increased the importance of biomarker testing. EGFR, HER2, BRAF, BRCA1/2, ALK, ROS1, RET, NTRK, KRAS, PD-L1 and mismatch-
repair/microsatellite-instability status are examples of molecular characteristics that may influence treatment selection in appropriate clinical settings. [5,6]
AI therefore has the potential to connect three major components: molecular characterization, therapeutic selection and longitudinal monitoring. Its use must, however, be supported by rigorous validation, appropriate clinical oversight and protection of patient data. [3,7]
PRECISION ONCOLOGY CONCEPT
Precision oncology integrates tumor biology with patient-specific clinical information. Molecular testing may identify mutations, gene fusions, copy-number changes, protein expression or DNA-repair abnormalities. The result is interpreted together with cancer type, stage, previous treatment, organ function, performance status and patient preferences. [1,5]
Table 01: Simplified Precision-Oncology Pathway.
| PATIENT | TUMOUR SAMPLEM | OLECULAR TESTIN | GAI / DATA ANALYSIS | BIOMARK ER PER | SONALIS ED THERA | PYMONITO RING |
|---|---|---|---|---|---|---|
| Clinical data | Biopsy / blood | NGS / IHC / ctDNA |
ML + clinical data | Actionable target | Targeted / immune | Response + ADR |
ROLE OF ARTIFICIAL INTELLIGENCE
Machine Learning
Supervised learning can predict outcomes using labelled clinical datasets, while unsupervised learning can identify molecular clusters or patient subgroups. Classification and regression models may support response or toxicity prediction. [3,4]
Deep Learning
Deep neural networks can analyse complex imaging and pathology data. Convolutional neural networks have been investigated for tumor detection, segmentation and histopathological classification. [8,9]
Natural Language Processing
NLP can extract diagnoses, medications, laboratory findings, adverse events and biomarker information from clinical notes and medical records. [3,4]
Multi-Omics Integration
AI can integrate genomic, transcriptomic, proteomic and clinical data to identify biological patterns and potential therapeutic targets. [3,4]
Drug Discovery
AI can assist target identification, molecular screening, prediction of drug–target interactions and prioritization of candidate compounds, potentially reducing time spent in early drug discovery. [10]
BIOMARKERS AND MOLECULAR PROFILING
Biomarkers are measurable characteristics that provide information about a biological process, disease state or treatment response. Companion or complementary diagnostic testing can help determine whether a patient is likely to benefit from a particular therapy. [5,6]
Table 02: Biomarker Interpretation.
| Biomarker | Common clinical relevance | Therapeutic example |
|---|---|---|
| EGFR mutation | Selected NSCLC | Osimertinib |
| HER2 amplification/over expression | Breast and other cancers | Trastuzumab |
| BRAF V600 | Melanoma and other cancers | Dabrafenib + trametinib |
| BRCA1/2 / HRD | Selected breast, ovarian, prostate, pancreatic cancers | Olaparib |
| ALK rearrangement | NSCLC | ALK inhibitors |
| ROS1 fusion | NSCLC | ROS1 inhibitors |
| RET alteration | NSCLC / thyroid cancers | RET inhibitors |
| NTRK fusion | Tumor-agnostic settings | TRK inhibitors |
| MSI-H / dimmer | Multiple tumor types | Immune-checkpoint inhibitors |
| PD-L1 expression | Several cancers | Selected PD-1/PD-L1 inhibitors |
Biomarker interpretation is context-dependent; not every biomarker is appropriate for every tumor type, and treatment eligibility depends on current evidence, regulatory status and clinical guidelines. [5,6]
AI-DRIVEN CLINICAL WORKFLOW
Table 03: AI-supported Precision Oncology Workflow.
| Patient assessment | History, stage, performance status, laboratory data | |
|---|---|---|
| Sample acquisition | Tumor biopsy / blood / liquid biopsy | |
| Molecular & imaging analysis | NGS, IHC, pathology, radiology, ctDNA | |
| AI-assisted integration | Machine learning + clinical and molecular datasets | |
| Biomarker identification | Actionable mutation / pathway / immune biomarker | |
| Therapy selection | Targeted therapy / immunotherapy / trial | |
| Monitoring | Response, resistance and adverse reactions | |
| Reassessment | Repeat molecular/clinical evaluation when indicated |
MECHANISM OF ACTION OF SELECTED PRECISION THERAPIES
EGFR pathway – Osimertinib
Activating EGFR mutations can promote receptor tyrosine-kinase signaling and downstream pathways such as RAS–RAF–MEK–ERK and PI3K–AKT. Osimertinib is a third-generation EGFR tyrosine-kinase inhibitor designed to inhibit mutant EGFR signaling, including the clinically important T790M resistance mutation. Inhibition reduces proliferative and survival signaling in susceptible tumor cells. [11,12]
HER2 – Trastuzumab
Trastuzumab binds the HER2 receptor and interferes with HER2-mediated signaling. It can also promote antibody-dependent cellular cytotoxicity. HER2-targeted treatment is therefore an example of receptor-directed precision therapy based on tumor biomarker status. [13]
BRAF/MEK – Dabrafenib + Trametinib
BRAF V600 alterations can constitutively activate the MAPK pathway. Dabrafenib inhibits BRAF signaling, while trametinib inhibits MEK, a downstream kinase. Dual pathway inhibition provides complementary suppression of MAPK signaling. [14]
BRCA/HRD – Olaparib
PARP participates in DNA damage response. In tumors with defective homologous-recombination repair, including selected BRCA1/2-mutated cancers, PARP inhibition can create synthetic lethality and preferentially impair tumor-cell survival. [15]
PD-1 – Pembrolizumab
PD-1 is an inhibitory receptor on immune cells. Pembrolizumab blocks PD-1 signaling and can restore T-cell activity against tumor cells. Response is influenced by tumor and immune biomarkers and the clinical context. [16]
Detailed drug table
Table 04: Mechanism of Action of Selected Precision.
| Drug | Target / biomarker | MOA | Major ADRs | Monitoring | ||||
|---|---|---|---|---|---|---|---|---|
| Osimertinib | EGFR mutation |
Irreversible mutant EGFR-TKI | Rash, diarrhea, paronychia; ILD/pneumonitis, QT prolongation |
ECG when indicated; pulmonary symptoms; labs |
||||
| Trastuzumab | HER2 | HER2 blockade + ADCC | Cardiac dysfunction, reactions |
infusion n | LVEF/ cardiac assessment; infusion monitoring |
|||
| Dabrafenib trametinib | + | BRAF V600 | BRAF + MEK inhibition |
Fever, rash, ocular/cardiac effects | fatigue e; | Temperature e, skin, ocular and cardiac monitoring | ||
| Olaparib | BRCA/HRD | PAR P lethal it |
inhibit on; | synthetic ice | Anemia, nausea, fatigue; rare MDS/AML | CBC; renal/ hepatic assessment as appropriate |
||
| Pembrolizumab b | PD-1 | Immune-checkpoint blockade | Immune-related dermatitis, colitis, hepatitis, pneumonitis, endocrinopathies |
Symptoms, LFT, thyroid and other tests as indicated |
||||
| Alectinib | ALK fusion | ALK tyrosine-kinase inhibition | Myalgia, fatigue, liver changes, bradycardia | enzyme e | LFTs, heart rate, symptoms | |||
| Larotrectinib | NTRK fusion | TRK inhibition | Dizziness, fatigue, nausea, liver enzyme changes | Liver tests and neurological symptoms | ||||
ADVERSE DRUG REACTIONS AND AI-BASED PREDICTION
Adverse drug reactions remain a major challenge even when treatment is molecularly targeted. Precision medicine can reduce exposure to ineffective therapies but does not guarantee absence of toxicity. AI may help identify early signals from laboratory results, medication records, vital signs, imaging and free-text clinical documentation. [3,17]
Table 05: Adverse Drug Reaction and AI- Based Prediction.
| Therapy | Important ADRs | Potential AI/clinical signal |
|---|---|---|
| EGFR inhibitors | Skin toxicity, diarrhea; rare pneumonitis | Rash documentation, stool symptoms, oxygenation/imaging patterns |
| HER2 therapy | Cardiac dysfunction | LVEF trends, symptoms, cardiac investigations |
| PARP inhibitors | Anemia, thrombocytopenia, fatigue | CBC trends, treatment interruptions |
| Checkpoint inhibitors | Immune-related organ toxicity | LFT/thyroid changes, inflammatory markers, symptom notes |
| BRAF/MEK inhibitors |
Pyrexia, skin, ocular/cardiac toxicity | Temperature, dermatology/ophthalmology findings, ECG/echo |
AI-based ADR prediction should be viewed as an early-warning or decision-support approach. False positives and false negatives can occur, and clinical confirmation is necessary before changing therapy. [3,17]
PHARMACOGENOMICS
Pharmacogenomics evaluates how inherited genetic variation influences drug exposure, efficacy and toxicity. Oncology provides important examples in which genetic information can contribute to safer treatment. DPYD-related variation can increase the risk of severe fluoropyrimidine toxicity. Tumor genomic biomarkers, although distinct from germline pharmacogenomics, also determine whether selected targeted treatments are biologically appropriate. Integrating germline, tumor and clinical information can move precision medicine toward both efficacy and safety. [18,19]
AI can potentially combine pharmacogenomic results with medication history, laboratory values and previous toxicity to generate individualized risk estimates. Such systems require robust validation and careful handling of genetic privacy. [3,18]
APPLICATIONS OF AI IN ONCOLOGY
1. Cancer detection and image-based diagnosis
2. Digital pathology and tumor grading
3. Molecular biomarker discovery
4. Treatment-response prediction
5. Resistance prediction and longitudinal monitoring
6. Clinical-trial matching
7. Drug discovery and repurposing
8. Adverse drug reaction and toxicity prediction Prognostic modelling and survival prediction
ADVANTAGES
AI-driven precision oncology can improve patient stratification by identifying clinically relevant patterns across multiple data types. It may help reduce ineffective treatment exposure, prioritize actionable molecular alterations, support trial matching and detect subtle changes that precede clinically obvious progression or toxicity. From a pharmaceutical perspective, AI can also assist target identification, drug screening and prediction of drug–target interactions. [3,4,10]
However, improved algorithmic accuracy does not automatically translate into improved patient outcomes. Clinical utility must be established through appropriate validation and prospective evaluation. [3,7]
LIMITATIONS AND ETHICAL ISSUES
Data quality and bias
Models can inherit errors and biases present in training data. Under-representation of certain populations may reduce generalizability. [7,20]
Interpretability
Complex models may produce predictions without an easily understandable biological explanation. Explainable AI methods can improve transparency but may not eliminate uncertainty. [7,20]
Privacy
Genomic and clinical datasets are highly sensitive. Secure storage, controlled access and appropriate governance are essential. [20,21]
Clinical validation
Models developed retrospectively may perform less well in new hospitals or populations. External and prospective validation is therefore important. [3,7]
Cost and accessibility
Advanced molecular testing and computational infrastructure may be expensive and may not be equally available to all patients. [20]
Responsibility
AI should support, not replace, clinical judgement. Healthcare professionals must interpret AI outputs in the context of the individual patient. [7,20]
FUTURE PERSPECTIVES
Future systems may integrate single-cell sequencing, spatial transcriptomics, liquid biopsy, digital pathology, radiomics and longitudinal electronic health records. Multimodal AI could create dynamic patient profiles that change as tumor biology evolves. Federated learning and other privacy-preserving methods may support collaboration between institutions while reducing unnecessary transfer of sensitive data. Prospective clinical trials and real-world evidence will be important to determine whether AI-guided precision oncology improves survival, quality of life, safety and cost-effectiveness. [3,4,7]
The pharmacist may have an increasingly important role in interpreting medication-related risks, monitoring ADRs, reviewing pharmacogenomic information, supporting medication optimization and
educating patients about targeted and immune therapies. [18,19]
CONCLUSION
AI-driven precision oncology is transforming the concept of personalized cancer therapy from a largely descriptive approach into a data-integrated clinical strategy. By combining molecular biomarkers with imaging, pathology, clinical information and treatment outcomes, AI can support patient selection, therapeutic matching, resistance assessment and toxicity prediction. Targeted drugs such as EGFR, HER2, BRAF/MEK and PARP inhibitors, together with immune-checkpoint inhibitors, illustrate the importance of matching mechanism with tumor biology. Nevertheless, responsible implementation requires high-quality data, rigorous validation, privacy protection, ethical governance and multidisciplinary oversight. The most appropriate model is therefore human-guided precision oncology in which AI augments the expertise of oncologists, pharmacists, pathologists and other healthcare professionals. [1,3,4]
REFERENCES
Nikita Gholap, Prachi Desale, Shriram Bairagi, Ai-Driven Precision Oncology: Revolutionizing Personalized Cancer Therapy, Int. J. of Pharm. Sci., 2026, Vol 4, Issue 10, 4706-4712. https://doi.org/10.5281/zenodo.23762774
10.5281/zenodo.23762774