What is the difference between targeted therapy and chemotherapy?

Chemotherapy kills all rapidly dividing cells (including healthy ones), causing widespread side effects. Targeted therapy specifically blocks molecules that drive cancer growth, based on the genetic makeup of your tumour. Side effects are usually different and often less severe, though targeted therapies still have significant side effects.

Do I need genetic testing for a targeted therapy trial?

Most targeted therapy trials require proof that your tumour has the specific genetic alteration the drug targets. This is usually confirmed through genomic testing of your biopsy or blood sample (liquid biopsy). Ask your oncologist about NHS genomic profiling.

Can targeted therapy cure cancer?

While targeted therapies have dramatically improved outcomes for many cancers, they rarely cure advanced cancer on their own. However, they can control disease for years and are increasingly being used in earlier stages (adjuvant setting) where they may contribute to cure. Combining targeted therapies with other treatments is a major focus of current trials.

What is a tumour-agnostic treatment?

A tumour-agnostic treatment targets a specific genetic mutation regardless of where the cancer started. For example, larotrectinib targets NTRK gene fusions in any cancer type. Pembrolizumab is approved for any MSI-H/dMMR tumour. These represent a paradigm shift from treating cancer by organ to treating by molecular profile.

Treatment Guide

Targeted Therapy Clinical Trials in the UK 2026 — Find Active Studies

26 May 2026 12 min read

400+

UK targeted therapy trials

70+

NHS sites

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Cancer types

Targeted therapy is the cornerstone of precision medicine in oncology. Unlike chemotherapy which attacks all rapidly dividing cells, targeted therapies are designed to interfere with specific molecules involved in cancer growth and spread. The UK is a global leader in precision oncology trials, with the NHS Genomic Medicine Service enabling biomarker-driven trial matching for patients across the country.

Types of Targeted Therapy

Kinase Inhibitors

Small molecules that block cancer-driving enzymes. Examples include EGFR inhibitors (osimertinib for lung cancer), BTK inhibitors (ibrutinib for CLL), ALK inhibitors, BRAF inhibitors, and MEK inhibitors. Usually taken as daily oral tablets.

Monoclonal Antibodies

Laboratory-made proteins that target specific cancer cell surface proteins. Trastuzumab (HER2), cetuximab (EGFR), bevacizumab (VEGF), and rituximab (CD20). Given by IV infusion, typically every 2-3 weeks.

Hormone Therapies

Target hormone-driven cancers. Aromatase inhibitors, SERDs, and androgen receptor inhibitors are precision therapies for breast and prostate cancer respectively. New oral SERDs and PROTAC degraders are in UK trials.

Epigenetic Modifiers

Target the regulatory mechanisms that control gene expression in cancer. EZH2 inhibitors, HDAC inhibitors, and hypomethylating agents. An emerging field with trials in haematological cancers and solid tumours.

Biomarker-Driven Trials

The future of targeted therapy is biomarker-driven — matching drugs to the specific genetic alterations driving each patient's cancer:

EGFR-mutant lung cancer — Osimertinib resistance trials, EGFR exon 20 insertion trials, and combination approaches
HER2+ breast cancer — New HER2-targeted ADCs, bispecific antibodies, and kinase inhibitors
BRAF V600E colorectal cancer — Encorafenib + cetuximab combinations; next-generation BRAF inhibitors
RET-mutant thyroid cancer — Selpercatinib and pralsetinib; trials in earlier disease stages
FLT3-mutant AML — Gilteritinib and quizartinib; combinations with chemotherapy
BRAF-mutant melanoma — Resistance mechanisms and next-generation combinations
Tumour-agnostic trials — Targeting specific mutations regardless of cancer type (e.g. NTRK fusions, KRAS G12C, MSI-H)

Genomic Testing for Targeted Therapy

Access to targeted therapy trials increasingly requires comprehensive genomic profiling:

NHS Genomic Medicine Service — Provides genomic testing for cancer patients across England through 7 Genomic Laboratory Hubs
Whole Genome Sequencing (WGS) — Available on the NHS for certain cancers; identifies all clinically relevant mutations in one test
Targeted panels — Focused tests for specific cancer types (e.g. 300-gene solid tumour panel, haematology panel)
Liquid biopsy — Blood tests (ctDNA) that can detect mutations without a tissue biopsy. Increasingly used in trial screening

Tip: If you haven't had genomic testing, ask your oncologist about it. The NHS is expanding access to comprehensive genomic profiling, and having your results opens up targeted therapy trial options. Our Smart Matcher can help identify trials based on your specific mutations.

What to Expect on a Targeted Therapy Trial

Most targeted therapies are oral tablets taken at home — much more convenient than IV chemotherapy
Some are IV infusions given every 1-3 weeks at a day unit (no overnight stay needed)
Side effects are typically different from and often milder than chemotherapy, but can still be significant
Regular monitoring includes blood tests, scans, and sometimes specific biomarker tracking
Treatment continues as long as it's working — targeted therapies can control cancer for years
Resistance eventually develops in most cases; trials are addressing this with combination approaches

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