Pharma & Cancer Treatment 2025: Breakthroughs, Market Leaders, and Industry Trends

The landscape of cancer treatment and the pharmaceutical industry is undergoing rapid transformation in 2025, driven by scientific breakthroughs, shifting drug sales rankings, and evolving strategies among top pharma companies. This article synthesizes the latest advances in cancer therapy, highlights the world’s top-selling drugs, and examines the leading pharmaceutical players shaping the future of oncology and global health.

Pharma & Cancer Treatment: Breakthroughs, Market Leaders, and Industry Trends (2025)

Latest Breakthroughs in Cancer Treatment (2025)

The fight against cancer is being reshaped by a wave of innovations, many of which are already impacting clinical practice or are in advanced stages of development:

1. Next-Generation Immunotherapy

Perhaps the most transformative breakthrough in modern oncology has been the rise of immunotherapy—a set of treatments that awaken the immune system to recognize and attack cancer cells. It is an idea as beautiful as it is powerful: train the body to see the enemy hiding in plain sight.

Checkpoint inhibitors were the first major triumph in this domain. Cancers like melanoma, lung cancer, and Hodgkin’s lymphoma once carried grim prognoses. Now, drugs such as nivolumab (Opdivo), pembrolizumab (Keytruda), and atezolizumab have offered durable responses—sometimes even complete remission—by blocking the “brakes” cancer places on immune cells.

Advances in immunotherapy continue to improve survival rates. Novel checkpoint inhibitors, CAR-T cell therapies, and bispecific antibodies are expanding treatment options for hard-to-treat cancers like pancreatic and brain tumors.Using immunotherapy as a Neo-adjuvant Strategy (before surgery or other treatments) is also gaining traction, with evidence that checkpoint inhibitors alone may be sufficient in some cases, potentially changing standard care protocols (AACR 2025).

Intra-tumoral immunotherapy represents a promising strategy to convert tumors into in situ vaccines, offering targeted immune activation with reduced toxicity. While clinical success has been observed in accessible tumors like melanoma, challenges persist in achieving consistent systemic responses and treating visceral metastases. Future advancements in delivery techniques and combination therapies hold significant potential to broaden its applicability.


Related: Combining Repurposed Drugs with Immunotherapy: A Novel Approach to Cancer Treatment

2. Precision Oncology and Genomics

AI-driven analysis of tumor genetics and microenvironment is enabling highly tailored therapies that improve outcomes and reduce side effects, making precision medicine a cornerstone of modern oncology  (4).

3. Tumor-Infiltrating Lymphocyte (TIL) and TCR-Engineered Therapies

Following approvals in 2024, TIL and TCR therapies represent a major advance in immunotherapy for solid tumors, enabling the immune system to better recognize and attack cancer cells4.

4. Antibody-Drug Conjugates (ADCs)

ADCs deliver chemotherapy directly to cancer cells, minimizing side effects. In 2025, new ADCs are being developed and approved for a wider range of cancers, improving treatment precision and efficacy48.

5. Drugging the “Undruggable” Targets (KRAS, GPCRs)

Second-generation inhibitors targeting challenging oncogenes like KRAS G12D and G12V are showing promising clinical results, expanding treatment options for cancers previously considered difficult to target (AACR 2025).

Here are ten cancers where chemotherapy is used less often, replaced by more effective or better-tolerated targeted treatments:

1. Melanoma: Immunotherapy and targeted treatments are now the primary tools. Chemotherapy is rarely used.

2. Chronic Myeloid Leukemia (CML) Oral tyrosine kinase inhibitors like imatinib allow most patients to live normal lifespans without chemotherapy.

3. Chronic Lymphocytic Leukemia (CLL) Targeted drugs like venetoclax and BTK inhibitors are commonly used first-line. Chemotherapy is now the exception.

4. MSI-High Colorectal and Endometrial Cancers Immunotherapy can provide long-lasting responses for patients with mismatch repair deficiency.

5. ER+ Breast Cancer (Low Oncotype DX Score) Hormonal therapy alone is often appropriate when genomic testing shows a low recurrence risk.

6. PD-L1 High Non-Small Cell Lung Cancer Single-agent immunotherapy may be more effective and better tolerated than chemotherapy in selected patients.

7. Advanced Prostate Cancer Hormone-targeting agents like enzalutamide and abiraterone are now preferred over chemotherapy in many cases.

8. Kidney Cancer Most patients now receive immunotherapy and VEGF inhibitors, not chemotherapy.

9. Liver Cancer (HCC) The combination of atezolizumab and bevacizumab has become a standard first-line treatment.

10. Multiple Myeloma: Treatment now often starts with monoclonal antibodies and other targeted agents, reducing the need for traditional chemotherapy.

6. Molecular Glues and Protein Degraders

These novel small molecules induce cancer-causing proteins to self-destruct, offering a new mechanism to tackle tumors. Investment and clinical trials in this area are accelerating in 20254.

7. Next-Generation CAR-T Cell Therapies

CAR-T therapies continue to expand beyond blood cancers into solid tumors, with new FDA approvals and innovations like in vivo CAR-T generation making these treatments more accessible and effective for a broader range of patients (48).

But checkpoint inhibition is just the beginning. CAR-T cell therapy has taken the world by storm, especially in blood cancers. This process involves harvesting a patient’s T-cells, genetically engineering them to recognize a cancer-specific antigen, and infusing them back into the bloodstream. These modified cells become hunters. In diseases like B-cell leukemia, CAR-T therapy has shown cure rates of over 80% in patients who had exhausted every other option.

A treatment that makes immune cells hunt down and kill cancer cells was declared a success for leukaemia patients in 2022. In the journal Nature, scientists at the University of Pennsylvania announced that two of the first people treated with CAR-T-cell therapy were still in remission 12 years on.

More recently, the same journal announced that a woman treated with CAR-T therapy as a four-year-old is in remission 19 years later.

However, the US Food and Drug Administration is currently investigating whether the process can in fact cause cancer, after more than 30 cases of secondary cancer were observed in patients receiving CAR-T therapies. The jury is still out as to whether the therapy is to blame but, as a precaution, the drug packaging now carries a warning.

New research is pushing CAR-T therapy into solid tumors—a far more complex challenge due to the tumor microenvironment and antigen heterogeneity. Trials in glioblastoma, pancreatic cancer, and ovarian cancer are underway, with engineered cells being further augmented with “logic gates” and “safety switches” to navigate the maze of normal tissues.

Related: CAR T-Cell Therapy for Glioblastoma: 3 Case Reports

8. AI-Based Risk Profiling, Drug Discovery and Radiotherapy

  • AI and machine learning are being used for early cancer detection and risk assessment, particularly in regions with limited access to imaging specialists.
  • Artificial intelligence is also accelerating the identification of new cancer drugs, cutting development time and uncovering novel treatment avenues.
  • AI is making radiotherapy more precise, reducing collateral damage to healthy tissue.

9. CRISPR Gene Editing

Clinical trials are leveraging CRISPR to edit genes within cancer or immune cells, aiming to correct mutations and enhance immune targeting of tumors.

10. Tumor Microenvironment Targeting

Drugs that alter the tumor’s surrounding environment are being developed to enhance the effectiveness of existing therapies.

11. Repurposed Drugs: Unlocking New Potential in Alternative Cancer Treatment

A significant breakthrough in 2025 is the growing use of repurposed drugs as alternative cancer treatments—medications originally developed for non-cancer conditions but now showing promise in oncology. Drugs like ivermectin, mebendazole, and fenbendazole, traditionally used as antiparasitic agents, are being studied for their anticancer properties. A peer-reviewed protocol published in September 2024, led by researchers such as Dr. Ilyes Baghli and Dr. Paul Marik, demonstrates that these drugs can disrupt cancer cell growth by targeting microtubules, essential for cell division [Baghli et al 2024].

A significant breakthrough in 2025 is the growing use of repurposed drugs—medications originally developed for non-cancer conditions but now showing promise in oncology. Drugs like ivermectin, mebendazole, and fenbendazole, traditionally used as antiparasitic agents, are being studied for their anticancer properties. A peer-reviewed protocol published in September 2024, led by researchers such as Dr. Ilyes Baghli and Dr. Paul Marik, demonstrates that these drugs can disrupt cancer cell growth by targeting microtubules, essential structures for cell division. When used in combination, they exhibit a synergistic effect, offering a potent, affordable treatment option [Baghli et al 2024].

Additionally, drugs like metformin, originally developed for diabetes, and anastrozole, a breast cancer treatment now repurposed for prevention, are gaining traction. Metformin disrupts cancer cell metabolism, while anastrozole reduces estrogen levels to lower breast cancer risk. These repurposed drugs are particularly valuable in low- and middle-income countries, where access to expensive therapies is limited, and their established safety profiles accelerate their integration into clinical practice.

References:
While anecdotal reports, such as case studies on fenbendazole (more than 200 case reports), suggest benefits, controlled clinical trials are needed to confirm efficacy. Patients considering these treatments should consult integrative oncologists to tailor protocols to their needs.

Holistic and Integrative Approaches

Complementary therapies, including dietary interventions and lifestyle changes, are also increasingly incorporated into cancer care.

12. Liquid Biopsies

Non-invasive blood tests for early cancer detection and monitoring are becoming more refined and accessible.

13. Gut Microbiome Research

The gut microbiome’s influence on immunotherapy efficacy is under investigation, with personalized probiotics being explored as adjuncts to cancer treatment.

14. Pancreatic Cancer Advances

Early detection tests and new insights into the disease’s molecular mechanisms are offering hope for one of the deadliest cancer types.

15. Personalized Cancer Vaccines

mRNA-based vaccines are now tailored to individual tumors, training the immune system to recognize and attack cancer cells. Trials in the UK and Europe are underway, with hopes of fewer side effects and reduced recurrence compared to traditional chemotherapy.

Top 10 Drugs by Worldwide Sales in 2024 (Updated for 2025)

The global pharmaceutical market in 2025 continues to be dominated by a mix of oncology immunotherapies, metabolic drugs, and treatments for autoimmune diseases. The latest forecasts and sales data reveal significant growth in cancer immunotherapy led by Merck’s Keytruda, alongside the rising prominence of GLP-1 receptor agonists for diabetes and weight management.

Key Highlights:

  • Keytruda (Pembrolizumab) remains the world’s bestselling drug, with sales projected to exceed $31 billion in 2025. Its growth is driven by continuous expansion of approved indications, now totaling around 40 worldwide, including recent approvals for cervical cancer and other malignancies. Merck reported an 18% year-on-year sales increase in the first three quarters of 2024, underscoring its dominant position in oncology.
  • GLP-1 receptor agonists dominate metabolic therapy sales, with Novo Nordisk’s Ozempic (semaglutide) and Eli Lilly’s Mounjaro (tirzepatide) ranking second and third, generating $21 billion and $19 billion respectively. These drugs target type 2 diabetes and obesity, reflecting growing global demand for effective metabolic disease treatments.
  • Dupixent (dupilumab) and Skyrizi (risankizumab) continue to lead in autoimmune disease treatment, maintaining strong sales above $13 billion each, driven by expanding indications in dermatology and inflammatory diseases.
  • Other oncology drugs such as Darzalex (daratumumab) and Opdivo (nivolumab) hold significant positions, with Darzalex generating around $13 billion in revenue and Opdivo also maintaining multi-billion dollar sales.
  • Biktarvy, an antiretroviral for HIV, and Eliquis, an anticoagulant, continue to be among the top sellers, each with approximately $13 billion in sales.
  • Wegovy, another semaglutide-based drug for chronic weight management, has surged to fifth place with $14.3 billion in sales, highlighting the expanding market for obesity treatments.

Market Trends:

  • Four of the top ten drugs belong to the GLP-1 class (semaglutide and tirzepatide variants), underscoring the blockbuster status of metabolic therapies.
  • Immuno-oncology remains a cornerstone of pharma growth, with Keytruda’s sustained leadership and Opdivo’s continued presence.
  • Autoimmune therapies maintain strong demand with Dupixent and Skyrizi.
  • The pharmaceutical market is increasingly shaped by combination therapies, expanded indications, and innovative delivery systems.

Pharma Industry Leaders: R&D and Oncology Focus

The pharmaceutical industry’s biggest players are investing heavily in research and development, particularly in oncology:

  • Merck & Co. leads with Keytruda, the world’s top-selling cancer drug, and is actively pursuing new indications and combination regimens.
  • Novo Nordisk has transformed the obesity and diabetes market with Ozempic and Wegovy, signaling a shift toward metabolic disease as a major growth area.
  • AbbVie, despite biosimilar competition for Humira, remains a top player through diversification and pipeline development.
  • Bristol Myers Squibb (BMS) and Pfizer continue to dominate in cardiovascular, oncology, and immunology segments.

Trends Shaping the Future of Cancer and Pharma

  • Personalization and Precision: From vaccines to gene editing, therapies are increasingly tailored to individual patients’ genetic and molecular profiles.
  • AI and Digital Health: Artificial intelligence is transforming everything from drug discovery to diagnostics and treatment planning.
  • Repurposing and Combination Therapies: Old drugs are finding new life in oncology, often in combination with cutting-edge treatments.
  • Global Access and Equity: Efforts are underway to make advanced therapies more accessible worldwide, with clinical trials expanding beyond traditional markets.
  • Regulatory and Safety Challenges: The rapid pace of innovation brings new safety and regulatory considerations, as seen with CAR-T therapy’s potential risks.

Conclusion

2025 marks a pivotal year in cancer treatment and the pharmaceutical industry. Breakthroughs in personalized medicine, gene editing, and AI are converging with market shifts driven by blockbuster drugs and strategic R&D investments. As the world’s leading pharma companies race to bring new therapies to market, patients stand to benefit from more effective, targeted, and accessible treatments than ever before.


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