Latest Breakthroughs in Cancer Treatment 2025: What You Need to Know

Despite trillions spent on cancer research, cancer still kills around 10 million people a year and is a leading cause of death globally, according to the World Health Organization. Cancer research continues to advance at an unprecedented pace, with new treatments offering hope to millions of patients worldwide.

 

Credit: Statista

Breast, lung and colon cancer are among the most common, while the number of new cancer cases annually is expected to grow by more than 75% between 2022 and 2050.

As we enter 2025, several groundbreaking therapies and innovations—including personalized vaccines, gene-editing technologies, repurposed drugs, and alternative cancer treatments—are reshaping cancer care.

Here are some of the most promising breakthroughs to watch for this year:

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 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
• Immunotherapy 101: What You Need to Know 2025 (Part 1)
• Approaches to Overcome the Current Treatment Plateau in Immunotherapy (European Journal of Cancer 2025)

CAR-T-cell therapy

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

Immunotherapy vs Chemotherapy

Chemotherapy still saves lives and remains essential in many cancers. But for a growing number of patients, we now have better options. These are treatments that are more targeted, more personalized, and often less toxic.

According to Dr Daniel Flora, an oncologist:

I specialize in melanoma and early-phase clinical research, and I haven’t prescribed chemotherapy for melanoma in years. Instead, I offer immunotherapy, targeted therapy, and clinical trials with promising approaches like bispecific T-cell engagers (BiTEs) and tumor-infiltrating lymphocytes (TILs). These therapies are designed to work with the immune system or go after specific cancer targets. We’ve made real progress, though there is still more work to do.

This shift is happening across oncology. Here are ten cancers where chemotherapy is used less often, replaced by more effective or better-tolerated 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.

We still rely on chemotherapy when the evidence supports it, particularly when it improves cure rates. But in many cases, we are moving toward more refined treatment strategies.

People are often surprised when I tell them I rarely prescribe traditional chemotherapy anymore.. As an oncologist, it’s meaningful to be able to offer patients better options. And when a clinical trial is available that could replace chemotherapy with something more precise or less toxic, I do my best to make that available. - Dr Daniel Flora

2. AI-Driven Drug Discovery and Clinical Research

Artificial intelligence is revolutionizing drug discovery by identifying new cancer-fighting compounds at an accelerated rate. AI algorithms analyze vast datasets to pinpoint potential treatments, significantly reducing the time required for drug development.

Randomized controlled trials (RCTs) are costly and time-consuming endeavors. Incorporating computational simulations and AI algorithms into RCT design could enhance trial efficiency, optimize protocols, and ultimately inspire more real-world research. This innovative approach holds promise to reduce costs, speed up the development process, and improve the relevance and accuracy of clinical findings.

Related: AI Predicts High-Dose Ivermectin and Mebendazole Combined with Supplements and Tailored Diet/Lifestyle Improved Overall Survival in Stage 4 Colorectal Cancer

3. CRISPR Gene Editing in Cancer Therapy

CRISPR technology has progressed to clinical trials, allowing scientists to edit genes within cancer cells or immune cells to enhance treatment. This approach aims to correct genetic mutations responsible for tumor growth and improve immune system targeting.

4. Personalized Cancer Vaccines

Cancer vaccines have evolved beyond prevention, with new personalized vaccines targeting individual tumors. These mRNA-based therapies train the immune system to recognize and attack cancer cells, offering a promising avenue for highly tailored treatments. 

Thousands of NHS cancer patients in England could soon access trials of a new vaccine treatment. It's designed to prime the immune system to target cancer cells and reduce recurrence risk. These vaccines are also hoped to produce fewer side effects than conventional chemotherapy. Thirty hospitals have joined the Cancer Vaccine Launch Pad, which matches patients with upcoming trials using the same mRNA technology found in current COVID-19 jabs. Over 200 patients from the UK, Germany, Belgium, Spain and Sweden will receive up to 15 doses of the personalized vaccine, with the study expected to complete by 2027.

Repurposed drugs like metformin are being explored as part of alternative cancer treatment strategies to enhance immune responses by altering cancer cell metabolism, potentially boosting vaccine efficacy in combination therapies [PubMed 2021].

5. AI-based Risk Profiling

In India, World Economic Forum partners are using emerging technologies like artificial intelligence (AI) and machine learning to transform cancer care. For example, AI-based risk profiling can help screen for common cancers like breast cancer, leading to early diagnosis. AI technology can also be used to analyze X-rays to identify cancers in places where imaging experts might not be available. These are two of 18 cancer interventions that the Centre for the Fourth Industrial Revolution India, a collaboration with the Forum, hopes to accelerate.

Credit: Science/Cambridge University Hospitals

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

Old meets new. A significant breakthrough in 2025 is the growing and trending use of repurposed drugs as alternative cancer treatments—medications originally developed for non-cancer conditions but now showing promise in oncology. 

Repurposing drugs as a market development strategy is a well-established and highly effective approach within the pharmaceutical industry. Rather than developing entirely new compounds from scratch—a process that can be costly, time-consuming, and fraught with uncertainty—companies identify new therapeutic uses for existing drugs. This strategy not only accelerates the development timeline but also leverages existing safety and efficacy data, reducing regulatory risks.

Many blockbuster drugs owe their commercial and clinical success to this approach. For example, Viagra was initially developed to treat hypertension and angina but found enormous success as a treatment for erectile dysfunction. Keytruda, originally designed for melanoma, has since been approved for a wide range of cancers, including lung, bladder, and head and neck cancers, significantly expanding its market potential. Similarly, Ozempic was first developed for type 2 diabetes management but has gained attention for its effectiveness in weight management, tapping into a new patient population.

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.

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

7. Tumor Microenvironment Targeting

Researchers are focusing on modifying the tumor microenvironment to make cancer cells more vulnerable to treatment. New drugs are being designed to alter the surrounding cells, cutting off the tumor’s support system.

8. Liquid Biopsies for Early Detection

Liquid biopsies—blood tests that detect cancer-related genetic mutations—are becoming more refined and accessible. These non-invasive tests allow for early cancer detection, real-time monitoring, and personalized treatment adjustments.

9. Gut Microbiome and Cancer Therapy

The gut microbiome’s role in cancer treatment is gaining attention, with research showing that certain gut bacteria can enhance the effectiveness of immunotherapy. Personalized probiotics and microbiome-targeted therapies are under investigation.

10. AI-Assisted Radiotherapy

Radiotherapy is becoming more precise with AI-powered systems that tailor radiation doses to individual patients. This reduces damage to healthy tissues and improves overall treatment outcomes.

11. Fighting pancreatic cancer

Pancreatic cancer is one of the deadliest cancers. It is rarely diagnosed before it starts to spread and has a survival rate of less than 5% over five years. At the University of California San Diego School of Medicine, scientists developed a test that identified 95% of early pancreatic cancers in a study. The research, published in Nature Communications Medicine, explains how biomarkers in extracellular vesicles – particles that regulate communication between cells – were used to detect pancreatic, ovarian and bladder cancer at stages I and II. And now a new test, coined PAC-MANN, can pick up signs of the disease from just one drop of blood, say researchers.

Scientists are also getting closer to a cure. A US/UK study has discovered that pancreatic cancer shuts down particular molecules in a key gene. The hope now is that the new knowledge "could lead to the development of more effective treatment options in the future”, Dr Chris Macdonald, head of research at Pancreatic Cancer UK, told The Guardian.

Related: Potential Role of Fenbendazole and Ivermectin in the Treatment of Stage 4 Pancreatic Cancer: A Compilation of Case Reports and Mechanistic Insights.

12. Precision Medicine and Genomics

Precision medicine, powered by advances in genomics, is revolutionizing how cancer is diagnosed, treated, and even prevented. Unlike the traditional “one-size-fits-all” approach, precision medicine tailors prevention and treatment strategies to each person’s unique genetic makeup, environment, and lifestyle. 

Precision oncology is the “best new weapon to defeat cancer”, the chief executive of Genetron Health, Sizhen Wang, says in a blog for the World Economic Forum. This involves studying the genetic makeup and molecular characteristics of cancer tumours in individual patients. The precision oncology approach identifies changes in cells that might be causing the cancer to grow and spread. Personalized treatments can then be developed.

The 100,000 Genomes Project, a National Health Service initiative, studied more than 13,000 tumour samples from UK cancer patients, successfully integrating genomic data to more accurately pin-point effective treatment. Because precision oncology treatments are targeted – as opposed to general treatments like chemotherapy – it can mean less harm to healthy cells and fewer side effects as a result.

This shift is leading to more effective therapies, fewer side effects, and earlier interventions (1, 2, 3). Read More: How Precision Medicine & Genomics Are Transforming Cancer Care and Prevention.

Emerging therapies presented at ASCO 2025 (MD Anderson)

• Combination therapy (dabrafenib + trametinib + pembrolizumab)
• Cancer: BRAF V600E-mutated anaplastic thyroid cancer (Stage IV)
• Finding: Given before surgery, about two-thirds of patients had no remaining cancer; two-year survival ~69%.
• Why it matters: Brings hope for a cancer type once considered nearly untreatable.

• First-line regimen for BRAF V600E-mutated metastatic colorectal cancer
• Cancer: Colorectal cancer with BRAF V600E mutation
• Finding: Encorafenib + cetuximab + chemotherapy achieved ~61% response rate, vs ~40% with the old standard.
• Why it matters: Offers a much stronger first-line option for an aggressive colorectal cancer subtype.

• mRNA-encoded bispecific antibody (BNT142 → RiboMab02.1)
• Cancer: Tumors expressing CLDN6 (testicular, ovarian, lung, others)
• Finding: Uses lipid nanoparticle-delivered mRNA to generate a bispecific antibody inside the patient, showing early anti-tumor activity.
• Why it matters: A first-of-its-kind approach combining mRNA technology with immune redirection.

• Oral KIF18A inhibitor (VLS-1488)
• Cancer: Tumors with chromosomal instability, in heavily pretreated patients
• Finding: Early trials show safety and initial anti-tumor effects by targeting a protein cancer cells depend on for survival.
• Why it matters: Could open a new drug class aimed at “chromosomally unstable” cancers.

• Antibody-drug conjugate PVEK (pivekimab sunirine)
• Cancer: Blastic plasmacytoid dendritic cell neoplasm (BPDCN), a rare aggressive leukemia
• Finding: Achieved durable remission rates by targeting CD123 with an antibody-drug conjugate; FDA approval is being pursued.
• Why it matters: May soon provide the first targeted therapy for a rare, deadly leukemia.

Why These Matter & Potential Impact

• They represent first-in-class or pioneering combinations, not just tweaks of existing treatments. E.g. mRNA-encoded bispecific antibodies, targeting of KIF18A, etc. MD Anderson Cancer Center

• Several of them address cancers with poor prognoses: anaplastic thyroid cancer, BRAF V600E colorectal cancer subtype, rare leukemia (BPDCN). MD Anderson Cancer Center

• Some show promise in earlier settings (neoadjuvant treatment to enable surgery) or as first-line therapies, which may more substantially improve survival and quality of life. MD Anderson Cancer Center

Final Thoughts

The landscape of cancer treatment in 2025 is marked by rapid innovation, from high-tech solutions like CRISPR and AI to practical, cost-effective alternative cancer treatments like repurposed drugs and holistic integrative approaches.

These advancements bring new hope for patients and medical professionals, particularly in making advanced care more accessible globally. While many of these breakthroughs are still in early stages, their potential to revolutionize cancer care is undeniable. Staying informed about the latest advancements, including the promise of repurposed drugs, can help patients and caregivers make more informed treatment decisions.

Related: 

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