Mesothelioma Gene Therapy

Gene therapy is considered a third-line treatment for patients diagnosed with advanced mesothelioma. Doctors use new therapies, like gene therapy, to contain mesothelioma and prevent it from spreading to distant sites. Gene therapy works by enhancing existing cells in order to make mesothelioma cells easier to kill. It is still undergoing clinical trials, but gene therapy remains promising for the future of mesothelioma treatments.

Written and Fact-Checked by: Laura Wright

What Is Gene Therapy?

Research technician with multipipette in genetic laboratory

Cancer appears in the body when a human carcinogen, such as asbestos, causes an otherwise healthy cell to divide and disperse abnormally. This abnormal multiplication of cells is a genetic mutation that scientists hope to resolve with the help of gene therapy.

Gene therapy replaces the cells with synthetic cells in the hope that they will provide the immune system with enough energy to do its job of defending the body against attack by cancer cells.

Gene therapy is an experimental treatment that has been in its testing phase for several decades but has not yet been approved by the FDA for exclusive first- or second-tier treatment.

The preferred treatment options for mesothelioma include:

However, gene therapy can be introduced as an additional, supporting treatment to counteract the defective genes in a cell. Gene therapy has so far proven to aid some patients fighting mesothelioma.

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How Does Gene Therapy Work?

Gene therapy involves the replacement or addition of mutated genes into a cancerous cell.

Genes injected through gene therapy may affect cancerous cells in multiple ways, including:

  • Causing healthy cells to multiply
  • Helping other genes that mend damaged genes or cells
  • Suppressing cancerous cell advancement

One type of protein and tumor suppressor gene is BAP1, which acts as a moderator for environmental toxins like asbestos—the only known cause of mesothelioma. Cells with lower levels of BAP1 appear to have a reduced ability for DNA repair once compromised by environmental carcinogens. Doctors feel this is a possible genetic reason why healthy cells in mesothelioma patients become triggered by asbestos and turn into cancer.

By targeting the BAP1 gene through gene therapy, doctors can help patients better defend against mesothelioma cell growth. 

While the process is difficult and still being studied, the best practice thus far is to inject new genes into the cell using a vector (an organism that spreads infection). Viruses typically act as gene therapy vectors. They get injected into cells so that they can deliver genetic material and target cancer cells, leaving healthy cells intact so that they can do their job.

2023 Update on BAP1 Gene Mutations

Surprisingly, even though patients with BAP1 mutations are more likely to develop mesothelioma, studies show that their cancer is less aggressive and easier to treat, with data indicating an average life expectancy of 7 years.

According to a study published in January 2023, a group of researchers that included Dr. Michele Carbone found that when only one copy of the BAP1 gene is present, the HIF1 protein is absent, which makes it more difficult for mesothelioma tumor cells to thrive in a low-oxygen environment.

This new finding shows that targeting BAP1 activity may make mesothelioma and other cancers more responsive to therapy and help improve survival rates.

“Two clinical trials have been opened at the National Cancer Institute Clinical Center in Bethesda, Maryland, to treat and study affected individuals in these [BAP1] families.

We hope findings from this study may enable clinicians to offer their patients more targeted treatment options faster.”
—Dr. Michele Carbone, University of Hawai’i Cancer Center

Types of Gene Therapy

A scientist using a microscope to assist with her work on gene therapy

There are different types of gene therapy that have been tested and used for patients with mesothelioma. These various trials target specific genes so that they can effectively attack cancerous cells. One common approach is the use of gene transfer, which works by injecting foreign substances to trigger effects such as suicide genes (see below).

Angiogenic inhibitors are used to stop the growth of blood cells, which ultimately starves the tumor. Oncolytic viruses are the most common use of gene therapy. They’re used to genetically alter viruses to attack cancerous cells once injected.

Each of these forms of gene therapy act in different ways to aid the fight against mesothelioma.

Some further types of gene therapy and how they work include the following:

  • Suicide Gene Therapy: This type of gene therapy is used as a gene transfer, which works to kill mesothelioma cells. This is done by modifying cells to produce enzymes, which triggers what is known as “suicide cells”, and causes the cell to die. One type of suicide gene therapy is called gene-directed enzyme-producing therapy or GDEPT.
  • Cytokine Gene Therapy: This type of gene therapy is signaled by cytokines, which act as molecules that signal communication from cell to cell within the immune system. This communication tells cells where to move to attack cancerous areas. Cytokine for gene therapy speeds up this communication to rush those cells to attack the cancer.
  • Tumor-Based p53 Gene Therapy: p53 is a tumor-suppressor gene that is one of the genes mutated when cancer is present. Experts believe that targeting this gene specifically to repair it will be a step forward in finding a longer-lasting treatment for mesothelioma.

Benefits of Gene Therapy for Mesothelioma

The benefits for mesothelioma using gene therapy reflect those of other similar treatments such as virotherapy. Gene therapy is limited in its ability to treat mesothelioma fully without the help of other treatments. However, one of the benefits of gene therapy is its ability to target cancerous cells specifically, leaving the patient room to fight off the disease using the remaining healthy cells.

Did You Know?

Various clinical trials have indicated that the use of gene therapy has shown impressive results. In one trial, 12 patients tolerated the treatment well, experiencing a stable response after just one month of treatment. The median survival rate for these patients was 449 days, or 15 months, with the assistance of gene therapy. It was observed that this treatment boosted pre-existing anti-tumor antibody responses and transitory inflammatory responses.

The best way to determine whether you’re a good candidate for gene therapy is to speak with a mesothelioma doctor. Use our Free Doctor Match to find top specialists in your area.

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Current and Ongoing Research into Gene Therapy

The FDA has not yet approved the mainstream use of gene therapy for patients with mesothelioma. However, in August 2017, the FDA approved it as a treatment for certain types of cancer. Specifically, the use of Kymriah gene therapy was approved for pediatric and young adult patients who have been diagnosed with a form of acute lymphoblastic leukemia.

“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer. New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses.”

- Scott Gottlieb, M.D., FDA Commissioner

Gene Therapy Side Effects

Gene therapy has been known to cause varying levels of side effects. Typically, these side effects are slight and disappear within 24 to 48 hours.

The most common side effects include flu-like symptoms such as:

  • Fever
  • Severe chills (or rigor)
  • Changes in blood pressure
  • Nausea
  • Vomiting

Experts have been concerned about the potential side effects of injecting genetic mutations within the body, such as exposing healthy cells to harmful viruses. However, patients have not exhibited these symptoms in clinical trials, and gene therapy remains an asset in treating patients with mesothelioma.

Undergoing Gene Therapy and Other Mesothelioma Treatments

Gene therapy is a promising treatment with the potential to restore hope to patients living with mesothelioma. With its varying abilities and methods at targeting and destroying cancerous cells, it is one of the most promising treatments in expanding research for mesothelioma.

Clinical trials are currently accepting patients diagnosed with mesothelioma to test out gene therapy treatments. Contact our Patient Advocates today at (866) 608-8933 to learn more about this emerging mesothelioma treatment.

Mesothelioma Gene Therapy FAQs

Can gene therapy be used to treat mesothelioma?

Yes. In clinical trials, mesothelioma specialists are using gene therapy as a third line of treatment against mesothelioma. Gene therapy, when used in combination with primary mesothelioma treatment methods such as surgery, chemotherapy, or radiation, can help halt the spread and growth of mesothelioma tumors.

To learn more about gene therapy or find a clinical trial near you, contact our Patient Advocates at (866) 608-8933. They can help connect you with a doctor and answer any ongoing questions you might have.

Are there side effects of gene therapy?

Unfortunately, yes. Like most other mesothelioma treatment options, there are side effects of gene therapy. Common side effects resemble cold or flu symptoms, and typically ease after 24-48 hours after treatment.

Written by:

Lead Editor

Laura Wright is a journalist and content strategist with more than 15 years of professional experience. She attended college at the University of Florida, graduating magna cum laude with a bachelor’s degree in journalism in 2008. Her writing has been featured in The Gainesville Sun and other regional publications throughout Florida.

  1. American Cancer Society. “What’s New in Malignant Mesothelioma Research?” Retrieved from: Accessed on December 20, 2022.

  2. Bononi A, Wang Q, Zolondick AA, Bai F, Steele-Tanji M, Suarez JS, Pastorino S, Sipes A, Signorato V, Ferro A, Novelli F, Kim JH, Minaai M, Takinishi Y, Pellegrini L, Napolitano A, Xu R, Farrar C, Goparaju C, Bassi C, Negrini M, Pagano I, Sakamoto G, Gaudino G, Pass HI, Onuchic JN, Yang H, Carbone M. BAP1 is a novel regulator of HIF-1α. Proc Natl Acad Sci U S A. 2023 Jan 24;120(4):e2217840120. doi: 10.1073/pnas.2217840120. Epub 2023 Jan 19. PMID: 36656861

  3. Cancer Gene Therapy. (2013). “Gene Therapy for malignant mesothelioma: current prospects and challenges.” Retrieved from: Accessed on December 20, 2022.

  4. Mayo Clinic. “Gene Therapy.” Retrieved from: Accessed on December 20, 2022.

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