Targeted Immunotherapies: Overview

What "targeted immunotherapy" means

Targeted immunotherapies are prescription medicines that act on a specific part of the immune system. Some push immune cells to attack cancer. Others block a specific immune signal to calm harmful inflammation in autoimmune and inflammatory diseases. They are not "immune boosters", and they are very different from supplements, diets or devices sold for immune wellness. This page is an overview for information only, not medical advice. It does not describe doses or tell anyone to start, stop or change a medicine.

Three things people mix up

Approval belongs to a particular drug and a particular use. This page does not list individual drugs, and we have not checked each one's regulatory status. Check the regulator's own listing for any specific drug.

In cancer

NCI lists several types of immunotherapy used in cancer: immune checkpoint inhibitors, T-cell transfer therapy, monoclonal antibodies, treatment vaccines and immune system modulators. [1] Checkpoint inhibitors block checkpoints so immune cells can respond more strongly to cancer. [1] Monoclonal antibodies are lab-made immune proteins designed to bind specific targets on cancer cells. [1] NCI also says immunotherapy is not yet as widely used as surgery, chemotherapy or radiation therapy. [1]

Risks. Many side effects happen when the revved-up immune system also acts against healthy cells and tissues. [1] For checkpoint inhibitors, NCI lists common effects of rash, diarrhea and fatigue, and rarer widespread inflammation that can affect the lungs, colon, liver, pituitary gland, heart muscle, kidneys, thyroid, pancreas or nervous system. [2] A review of these immune-related adverse events says their mechanisms are unclear. [3] Monoclonal antibodies can cause needle-site reactions and flu-like symptoms. [4]

In autoimmune and inflammatory disease

For rheumatoid arthritis, the National Institute of Arthritis and Musculoskeletal and Skin Diseases says biologic response modifiers, a type of disease-modifying drug, are used if the disease does not respond to initial therapies, and that they target specific immune messages and interrupt the signal, helping to decrease or stop inflammation. [5] That description applies to rheumatoid arthritis; other conditions have their own evidence and approvals. For immune-system background see how the immune system works, and for broader drug-class context see small molecules vs peptides vs biologics.

The trade-off is infection risk. NIAMS notes that many rheumatoid arthritis medicines may increase the risk of infection, so doctors may monitor for infections. [5] In a meta-analysis of 71 randomized trials (22,760 participants) of anti-TNF drugs, a class of biologics, any infection was 20% more frequent, serious infections 40% more frequent and tuberculosis 250% more frequent than with placebo or no treatment, with moderate-quality evidence. [6] A review adds that screening for latent tuberculosis and management of hepatitis B are part of risk minimization, and that observational data can be distorted by confounding by indication. [7] See also immunodeficiency and autoimmune conditions.

Evidence labels for this area

• Approved medicine: specific drugs for specific cancers and rheumatoid arthritis, as described by NCI and NIAMS above.
• Established: that targeting immune checkpoints or immune signals changes immune activity and can cause immune-related side effects. [1][5]
• Investigational: newer targets and combinations; a registered trial proves nothing about effectiveness.
• Marketing claim: any "immunotherapy" offered outside a regulated drug or trial.