The Future of Oncology: Developing Safer and More Targeted Cancer Therapies

• Targeted therapy blocks specific molecules that drive cancer cell growth, while chemotherapy damages all rapidly dividing cells.
• Biomarker testing comes first, because only tumors that carry the relevant target can respond to a given targeted drug.
• In the FLAURA trial, a third-generation EGFR inhibitor extended median progression-free survival in EGFR-mutated lung cancer from 10.2 to 18.9 months.
• Targeted drugs have their own side effects, such as skin reactions with EGFR inhibitors and lung inflammation with some antibody-drug conjugates.
• Blood tests for circulating tumor DNA can guide decisions after surgery, and in one trial they halved the use of chemotherapy in stage II colon cancer without worse outcomes.
• Aegis Capital, a HealthTech & Longevity VC, invests in early-stage startups from Central and Eastern Europe working on diagnostics, digital therapy and AI in health.

What Is Targeted Cancer Therapy?

Targeted cancer therapy is a form of cancer treatment that acts on specific proteins or genetic changes that help cancer cells grow, divide and spread. Chemotherapy is cytotoxic and affects all rapidly dividing cells without differentiation between healthy and malignant tissue, which explains side effects such as hair loss, nausea and damage to the bone marrow. Targeted drugs interfere with a particular pathway, so they are effective only when the tumor depends on that pathway.

The need for more effective and tolerable treatment keeps growing. According to global cancer statistics published in CA: A Cancer Journal for Clinicians in July 2026, about 21 million people were newly diagnosed with cancer in 2024 and nearly 10 million died from the disease. The same report projects that new cases will rise to 34 million by 2050 due to population growth and aging alone.

How Targeted Therapy Differs From Chemotherapy

The two approaches differ in three main ways:

• Target: chemotherapy damages cells that divide quickly, while targeted therapy blocks a specific molecule, such as a mutated receptor or an overactive enzyme.
• Selection: chemotherapy is chosen mainly by cancer type and stage, while targeted therapy also depends on the result of biomarker or genetic testing.
• Side effects: chemotherapy often causes fatigue, nausea and low blood counts, while targeted drugs cause side effects linked to their specific target.

Many treatment plans combine both. Targeted therapy can be given together with chemotherapy or radiation, and the oncologist decides the combination, dose and cycle based on the type of cancer, its stage and the patient's condition.

Why Biomarker Testing Comes First

Only some cancers carry the molecular targets that specific drugs are designed to hit. Before targeted therapy is prescribed, genetic or biomarker testing on tumor tissue or blood helps determine whether the relevant change is present. A patient without the target is unlikely to respond, so testing is essential and protects patients from ineffective treatment and its side effects.

Testing also changes how cancers are classified. In 2017, the FDA approved the first treatment based on a tumor's genetic feature instead of its location in the body, for cancers with high microsatellite instability or mismatch repair deficiency. Oncology is gradually moving from organ-based categories toward categories defined by specific mutations.

Tip: Before starting treatment for advanced cancer, ask your oncologist for advice on whether biomarker or genetic testing has been done and which results could affect the choice of therapy.

Main Types of Modern Cancer Treatment

Modern oncology combines several classes of therapy, each with a different mechanism and a different side effect profile. The table below offers a comparison of the main options in terms of mechanism and side effects.

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Targeted Therapy in Practice: Three Landmark Examples

Three cancer types show how targeted therapy moved from concept to standard care. Each example also shows its limits.

Imatinib and Chronic Myeloid Leukemia

Imatinib targets the abnormal enzyme produced by the gene fusion that drives chronic myeloid leukemia. Final results of the IRIS trial, published in the New England Journal of Medicine in 2017, reported an estimated 10-year overall survival of 83.3% among patients assigned to imatinib. The authors' conclusion was that long-term treatment was not associated with unacceptable cumulative or late toxic effects.

The success of imatinib in the IRIS trial turned a disease with a poor prognosis into one that many patients manage for years with a daily tablet. They also set the model for later targeted drugs: identify the molecular driver, block it, and monitor response with specific tests.

EGFR Inhibitors in Lung Cancer

The epidermal growth factor receptor (EGFR) is a protein on the cell surface that signals cells to grow. An EGFR mutation can switch that signal on permanently, driving cancer cell growth in non-small cell lung cancer. According to the European Society for Medical Oncology, EGFR mutations occur in about 15% of non-small cell lung cancers in Western populations and about 35% in Asian populations.

EGFR inhibitors outperform chemotherapy as first treatment for these patients, but resistance develops over time, most often through a second mutation called T790M. In the FLAURA trial, osimertinib, which targets both the original EGFR mutations and T790M, extended median progression-free survival from 10.2 to 18.9 months compared with earlier EGFR inhibitors. Median overall survival reached 38.6 months versus 31.8 months, showing how drug design can respond to known resistance mechanisms.

HER2 and Antibody-Drug Conjugates

Antibody-drug conjugates combine a targeted antibody with a cytotoxic payload, delivering chemotherapy directly to cells that carry a specific protein. In the DESTINY-Breast06 trial, trastuzumab deruxtecan achieved a median progression-free survival of 13.2 months and a response rate of 62.6%, compared with 34.4% for chemotherapy, in HR-positive breast cancer with low or ultralow HER2 expression. Around 85 to 90% of HR-positive, HER2-negative breast cancers show some HER2 expression, so the approach expanded the group of patients who can benefit.

In December 2025, the FDA approved trastuzumab deruxtecan with pertuzumab as first treatment for HER2-positive metastatic breast cancer, based on a trial of 1,157 adults. HER2 has become one of the clearest examples of how a single target can support several generations of drugs.

Are Targeted Therapies Safer?

Targeted therapies spare many healthy cells, but they are not free of risk. Skin reactions are common with EGFR-targeted drugs, diarrhea is a frequent side effect of several targeted therapies, and for some drugs used to treat cancer, regular monitoring of liver and kidney function and a review of other medications are required. Therapy may need a dose reduction or an interruption when side effects become severe.

Antibody-drug conjugates illustrate both the progress and the remaining risks. An early pooled analysis of 1,150 patients treated with trastuzumab deruxtecan found drug-related interstitial lung disease in 15.4%, with 2.2% fatal cases, while a 2026 meta-analysis of 35 studies and 6,840 patients reported 8.8% for all grades and 0.26% for fatal cases. Heavily pretreated patients carried the highest risk of severe events.

Did you know: Dose made a measurable difference in lung toxicity: in a meta-analysis of trastuzumab deruxtecan in metastatic breast cancer, interstitial lung disease occurred in 22.7% of patients receiving 6.4 mg/kg and 9.3% of those receiving 5.4 mg/kg.

Resistance is the second major limitation. Cancer cells can acquire new mutations that let them bypass the blocked pathway, so many regimens combine drugs, such as a BRAF inhibitor with a MEK inhibitor in BRAF-mutated melanoma, to delay resistance.

Immunotherapy: Helping the Immune System Find Cancer

Immunotherapy helps the patient's own immune system recognize and attack cancer, and a growing understanding of tumor immunology has made it possible to enhance that response. Many tumors produce proteins that switch off immune cells, and blocking these signals allows T cells to respond.

Immune Checkpoint Inhibitors

Checkpoint inhibitors are antibodies that block proteins such as PD-1 and CTLA-4, which normally keep immune responses in check. In the CheckMate 067 trial in advanced melanoma, median overall survival reached 71.9 months with nivolumab plus ipilimumab, 36.9 months with nivolumab alone and 19.9 months with ipilimumab alone, according to 10-year results published in the New England Journal of Medicine in 2024. More patients now survive for years: among patients who were alive and free of progression at 3 years, 10-year melanoma-specific survival was 96% with the combination.

Checkpoint inhibitors can also trigger immune-related side effects, because an activated immune system may attack healthy organs. Most of these effects are manageable when recognized early, which requires close monitoring by the treating team.

Cell Therapies

Cell therapies go a step further by modifying the patient's immune cells outside the body. In CAR-T cell therapy, T cells are engineered to recognize a specific protein on cancer cells and then returned to the patient, and the FDA approved the first CAR-T therapy in 2017 for a form of leukemia. Dendritic cells, which present tumor antigens to T cells, form the basis of cancer vaccine approaches, including a cellular immunotherapy approved in the United States in 2010 for advanced prostate cancer.

These therapies are complex to manufacture and can cause serious reactions such as cytokine release syndrome. Research now focuses on extending their benefit to solid tumors and on making them safer and more widely available.

Liquid Biopsy and Smarter Follow-Up

Liquid biopsy analyzes fragments of circulating tumor DNA (ctDNA) in a blood sample. After surgery, the presence of ctDNA can indicate microscopic residual disease that imaging cannot see, which helps identify patients at high risk of relapse. The same principles apply beyond colon cancer and may help doctors decide who needs additional treatment and who can safely avoid it.

The DYNAMIC trial, published in the New England Journal of Medicine in 2022, tested this approach in stage II colon cancer. Patients whose treatment was guided by ctDNA results were less likely to receive adjuvant chemotherapy, 15% compared with 28% under standard management, while 2-year recurrence-free survival was similar at 93.5% and 92.4%. Sparing patients unnecessary chemotherapy is one of the most direct ways to make cancer care safer.

Example: A patient with stage II colon cancer has a ctDNA blood test 4 and 7 weeks after surgery. Both results are negative, so under a ctDNA-guided approach the oncologist may recommend follow-up without adjuvant chemotherapy. A positive result at either time point would lead to a recommendation for chemotherapy.

Supporting Patients Beyond the Drug

Cancer affects mental health as well as the body, which makes psychological care an important part of treatment, and anxiety and depression are common during and after treatment. Digital therapeutics and other digital interventions deliver structured psychological support, such as cognitive behavioral therapy, through a smartphone or online platform. They can reach patients between clinic visits and in places where specialists are scarce.

A systematic review and meta-analysis of 22 randomized controlled trials, published in JMIR Cancer in 2025, found that digital interventions tailored to specific content and duration can reduce depression and anxiety in patients with cancer. The authors called for larger, more rigorous studies, so digital support works best as part of care planned with the treating team.

Why Oncology Innovation Needs Early-Stage Capital

New cancer therapies and the tools around them take years of development and clinical trials before patients can access them. Diagnostics that select the right patients, software that monitors response and digital programs that support patients all need clinical evidence and regulatory approval. Early-stage investors with healthcare experience help founders plan that path and connect them with clinicians and research centers.

Aegis Capital is a HealthTech & Longevity VC that invests in early-stage startups from Central and Eastern Europe, with diagnostics, digital therapy and mental health, and AI and digital health among its investment areas. The fund has a capitalization of PLN 80 million, backed by 22 private investors, and offers an initial ticket of up to PLN 3 million, with total funding of up to PLN 8 million per company across follow-on rounds.

FAQ

What is the difference between targeted therapy and chemotherapy?

Chemotherapy damages all rapidly dividing cells, including healthy ones. Targeted therapy blocks specific molecules that cancer cells depend on, so it works only when the tumor carries the relevant target.

Is targeted therapy suitable for every patient?

No. Targeted therapy is effective only when biomarker or genetic testing confirms that the tumor has the specific change the drug is designed to block.

What is an EGFR mutation?

An EGFR mutation is a change in the gene for the epidermal growth factor receptor that keeps the receptor switched on and drives cancer cell growth. It is most often tested for in non-small cell lung cancer, where it guides the use of EGFR inhibitors.

Can targeted therapy be combined with chemotherapy?

Yes. Targeted drugs can be combined with chemotherapy, radiation or other targeted drugs, and combinations are sometimes used to delay resistance.

Why do targeted therapies stop working?

Cancer cells can acquire new mutations that bypass the blocked pathway. Newer drugs, such as EGFR inhibitors designed for the T790M mutation, are developed to overcome known resistance mechanisms.

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