Targeted and Immunotherapy Drugs for Liver Cancer: Treatment Options and Latest Advances
Liver cancer, especially advanced hepatocellular carcinoma (HCC), is increasingly treated with targeted therapy and immunotherapy as important systemic treatment approaches.
So, what are the targeted and immunotherapy drugs for liver cancer? What are the differences between them? What new combination treatment strategies have emerged in recent years?
This article introduces common liver cancer targeted and immunotherapy drugs, their mechanisms, and key treatment trends.
What Is Liver Cancer and When Are Targeted Therapy and Immunotherapy Used?
Liver cancer is a malignant tumor that develops in the liver, with hepatocellular carcinoma (HCC) being the most common type of primary liver cancer.
In general, treatment approaches include:
- Early-stage HCC: Surgery, liver transplantation, and local ablation are commonly considered.
- Intermediate-stage HCC: Locoregional treatments such as transarterial chemoembolization (TACE) are commonly used.
- Advanced or unresectable HCC: Targeted therapy, immunotherapy, and combination regimens are important systemic treatment options.
Therefore, targeted and immunotherapy drugs for liver cancer are mainly considered when local treatments cannot adequately control HCC.
👩⚕️ The specific treatment plan should be determined by a physician based on disease stage, liver function, previous treatments, overall health, and other individual factors.
What Is Targeted Therapy for Liver Cancer?
Liver cancer targeted therapy uses drugs that target specific molecules and signaling pathways involved in tumor growth and angiogenesis.
In HCC, VEGF/VEGFR inhibition is an important strategy, while other multi-target drugs act on pathways such as RAF, FGFR, and MET to inhibit tumor growth and progression.
Overall, targeted therapies for liver cancer work through different mechanisms, and treatment selection depends on the patient’s disease characteristics and treatment history.
What Are the Common Targeted Therapy Drugs for Liver Cancer?
Common targeted therapy drugs for HCC include multi-target tyrosine kinase inhibitors (TKIs) and monoclonal antibodies targeting tumor angiogenesis.
Sorafenib
Sorafenib is one of the most important multi-target tyrosine kinase inhibitors in the development of systemic treatment for HCC.
It acts on multiple signaling pathways, including VEGFR, PDGFR, and RAF, thereby affecting both tumor cell proliferation and angiogenesis.
Sorafenib was an important systemic treatment option for advanced HCC for many years and helped advance liver cancer treatment from traditional approaches toward molecular targeted therapy.
Lenvatinib
Lenvatinib is also a multi-target tyrosine kinase inhibitor.
Its major targets include VEGFR, as well as pathways involving FGFR.
Lenvatinib has become an important targeted treatment option for advanced HCC.
Regorafenib
Regorafenib is another multi-target kinase inhibitor that acts on several targets, including VEGFR, RAF, and KIT.
It has a role as a subsequent-line treatment option for certain patients with advanced HCC who have previously received sorafenib.
Cabozantinib
Cabozantinib targets multiple pathways, including VEGFR, MET, and AXL.
Its activity across several signaling pathways associated with tumor progression has supported its use as a later-line treatment option for advanced HCC.
Ramucirumab
Ramucirumab is a monoclonal antibody targeting VEGFR2. It inhibits VEGF-related angiogenic signaling and thereby interferes with tumor blood vessel formation.
Its use in certain patients with advanced HCC is associated with factors such as alpha-fetoprotein (AFP) levels.
Donafenib
As a representative Chinese innovative targeted drug for HCC, Donafenib has been approved for first-line treatment of certain patients with unresectable HCC.
Apatinib
In HCC treatment, Apatinib has been used as a subsequent-line treatment option for certain patients and has also been investigated and used in combination with camrelizumab in selected patients with advanced HCC.
What Is Immunotherapy for Liver Cancer?
Unlike targeted therapy, which directly interferes with tumor-related molecular pathways, immunotherapy primarily works by regulating the patient’s immune system to enhance the body’s antitumor response.
Cancer cells can exploit immune checkpoint mechanisms to evade immune system attack. The PD-1/PD-L1 pathway is one of the important immune checkpoint pathways involved in this process.
Under normal conditions, these pathways help prevent excessive immune activation. However, tumor cells may exploit them to reduce the antitumor activity of T cells.
Immune checkpoint inhibitors can block these immune “brakes,” helping restore part of the T-cell response against cancer cells.
What Are the Common Immunotherapy Drugs for Liver Cancer?
Current immunotherapy drugs for liver cancer mainly include immune checkpoint inhibitors, particularly PD-1 and PD-L1 inhibitors.
PD-1 Inhibitors
Representative drugs include:
- Nivolumab
- Pembrolizumab
- Camrelizumab
- Sintilimab
- Tislelizumab
These drugs block the PD-1 pathway and help restore T-cell antitumor activity.
However, the approved indications, treatment lines, and specific combination regimens for different drugs may vary between countries and regions.
PD-L1 Inhibitors
Representative drugs include:
- Atezolizumab
- Durvalumab
PD-L1 inhibitors primarily act on the PD-L1-related immune checkpoint pathway to regulate the antitumor immune response.
What Is the Difference Between Targeted and Immunotherapy Drugs for Liver Cancer?
This is an important question for many patients.
Simply put, targeted therapy mainly acts on tumor-related molecules and signaling pathways, while immunotherapy primarily works by modulating the immune system to attack cancer cells.
| Comparison | Targeted Therapy | Immunotherapy |
|---|---|---|
| Main action | Blocks pathways involved in tumor growth | Restores or enhances antitumor immune responses |
| Common targets | VEGFR, RAF, FGFR, MET, etc. | PD-1, PD-L1, etc. |
| Common drug types | Small-molecule kinase inhibitors, monoclonal antibodies | Immune checkpoint inhibitors |
| Examples | Sorafenib, lenvatinib, etc. | Nivolumab, pembrolizumab, etc. |
| Treatment approach | Can be used alone or in combination | Can be used alone or in combination |
| Main feature | Directly interferes with tumor-related pathways | Activates or restores the patient’s antitumor immune response |
Specific treatment plans should be comprehensively evaluated and determined by a physician based on tumor stage, liver function, overall health, previous treatment, locally approved medicines, and other individual factors.
Treatment Trends in Targeted and Immunotherapy Drugs for Liver Cancer
In recent years, systemic treatment for HCC has gradually evolved from single-drug approaches toward targeted therapy, immunotherapy, and multi-mechanism combination treatments.
From Single-Agent Therapy to Combination Treatment
Immunotherapy combined with anti-angiogenic therapy is an important treatment approach for advanced HCC.
For example, atezolizumab combined with bevacizumab targets the PD-L1 and VEGF pathways, respectively. Durvalumab combined with tremelimumab works through PD-L1 and CTLA-4.
These approaches reflect the growing interest in combining different mechanisms to achieve broader control of tumor progression.
From Advanced Disease to Earlier Treatment Stages
Research is increasingly extending beyond advanced HCC to intermediate-stage disease, the perioperative setting, and disease management following locoregional treatment.
Studies are exploring combinations of immunotherapy with surgery, ablation, TACE, and other treatment approaches.
New Drugs Are Becoming Major Research Priorities
Future development of targeted and immunotherapy drugs for liver cancer is expected to focus increasingly on bispecific antibodies, new immune checkpoints, and emerging molecular targets.
Examples include PD-1/VEGF bispecific antibodies and emerging immune targets such as LAG-3 and TIGIT.
Overall, HCC treatment is moving toward multi-mechanism combinations, precision patient stratification, and personalized treatment, providing more potential options for patients.
Conclusion
The treatment landscape for HCC is moving from single-treatment approaches toward targeted therapy, immunotherapy, and multi-mechanism combination strategies.
As new immunotherapies, bispecific antibodies, and precision targeted drugs continue to advance, patients with HCC may have access to increasingly diverse and individualized treatment options.
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FAQ about Targeted and Immunotherapy Drugs for Liver Cancer
What Are the Most Common Targeted and Immunotherapy Drugs for Liver Cancer?
Common targeted drugs include sorafenib, lenvatinib, donafenib, regorafenib, cabozantinib, and ramucirumab, while immunotherapy options include nivolumab, pembrolizumab, camrelizumab, sintilimab, tislelizumab, atezolizumab, and durvalumab.
How Does Targeted Therapy Work for Liver Cancer?
Targeted therapy for liver cancer blocks specific pathways involved in tumor growth and angiogenesis, including VEGFR, RAF, FGFR, and MET.
How Does Immunotherapy Work for Liver Cancer?
Immunotherapy helps restore the body’s antitumor immune response by blocking immune checkpoints such as PD-1, PD-L1, or CTLA-4.
Can Targeted Therapy and Immunotherapy Be Used Together for Liver Cancer?
Yes, targeted therapy and immunotherapy can be combined in certain patients with advanced HCC, although the appropriate regimen depends on clinical factors and local treatment guidelines.
What Are the Latest Treatment Trends for Liver Cancer?
Current treatment trends include combination therapies, treatment in earlier disease stages, bispecific antibodies, new immune targets, and more personalized treatment strategies.
