Key Takeaways
- Gene therapy seeks to reverse or compensate for the genetic mutations that drive mesothelioma — restoring healthy cell function without modifying the actual genes themselves, since repairing mutations at their origin is currently not feasible.
- Gene therapy works through several approaches: introducing the proteins that mutated genes can no longer produce, genetically modifying the immune system to attack mesothelioma cells, or introducing “suicide genes” that trigger intentional cancer cell death.
- Key genetic targets include BAP1 (mutated in 60–70% of mesothelioma cases) and p53 — both are tumor-suppressing genes whose loss removes the body’s natural safeguards against uncontrolled cell growth.
- Gene therapy is targeted and patient-specific — it attacks cancerous cells while leaving healthy cells intact, with relatively minor side effects compared to systemic treatments like chemotherapy.
- Gene therapy is currently only available through clinical trials and is not yet approved for widespread clinical use. However, as more trials demonstrate success, FDA approval for mesothelioma-specific gene therapies may follow.
- Used in conjunction with other treatments in a multimodal approach, gene therapy has the potential to be a significant advancement for mesothelioma patients.
What is Gene Therapy?
Gene therapy seeks to reverse genetic mutations—a characteristic of all cancers—in hopes of
restoring healthy functioning and growth to cancerous cells. In mesothelioma, asbestos fibers
hijack local immunity, reasonable growth patterns, and cells’ gene signatures, or their unique
expression of DNA into proteins and other biological molecules. Gene signatures can be
interpreted as indicative of a patient’s prognosis, receptivity to certain treatments, likelihood of
metastasis, or the odds of cancer recurrence. Obviously, genes and gene signatures are critical for
a.) understanding how mesothelioma develops; b.) formulating new targets for treatment; and c.)
reversing the damage inflicted by damaged genes. For instance, mutations in the BAP1 gene are
known to be associated with mesothelioma (with the mutation presenting in ~60-70% of all
cases), as well as the p53 gene. These genes, while multifaceted in their effects, have largely anti
tumor signatures. This means that the proteins they encode for are responsible for regulating the
cell cycle and to what extent cells reproduce. It’s nearly impossible to fix these genes at their
origins, so doctors can either supplement, re-program, or alter other cells to account for these
mutations.
Gene therapy can include introducing the proteins/molecules that impaired genes usually encode
for. Gene therapy can also include genetically modifying the immune system to attack
mesothelioma cells. Gene therapy can also include introducing “suicide genes” that incite
intentional cell death. Obviously, gene therapy encompasses many avenues of treatment targets,
which makes the field exciting and promising.
Is Gene Therapy to Treat Mesothelioma a Viable Option?
Gene therapy is currently only available in clinical trials, given that it’s still in the earlier stages
of its development. Plus, because it isn’t feasible to actually revert the genes themselves, it has
been challenging to devise ways to effect biological changes like genes would. These challenges
have made gene therapy largely trial-based; but with more trials, though, these obstacles can be
mitigated.
The Future of Using Gene Therapy to Treat Mesothelioma
As research further elucidates the genetic mechanisms of mesothelioma—how mutations affect
prognosis/treatment, how mutations spark carcinogenesis, etc.—gene therapy will ideally
become a more common form of treatment. Because it can involve several different mediums to
impart change, this widens the scope of patient eligibility. As another plus, gene therapy is
largely context-dependent and targeted, meaning that it’s both unique to the patient and doesn’t
affect healthy, non-cancerous cells.
As more clinical trials prove successful, the Food & Drug Administration (FDA) can give these
treatments the go-ahead for clinical use. If used in conjunction with other therapies in a
multimodal approach, gene therapy can be a huge “win” for mesothelioma patients: healthy cells
are left intact, side effects are relatively minor, and functionality is returned to defunct genes.
If you or a loved one has been diagnosed with mesothelioma or another
asbestos-related disease, please call (800)-505-6000 or fill out our contact form. We are here
to help you navigate the legal process of filing a claim to receive compensation for your
mesothelioma diagnosis. We help mesothelioma victims and their families in
Pennsylvania.
Frequently Asked Questions
What is gene therapy for mesothelioma?
Gene therapy is a treatment approach that aims to reverse or compensate for the genetic mutations that allow mesothelioma to develop and spread. In mesothelioma, asbestos fibers damage genes responsible for immune function, tumor suppression, and normal cell growth. Because it is currently not possible to repair these genes at their origin, gene therapy instead works around the damage — by introducing the proteins that mutated genes can no longer produce, reprogramming immune cells, or introducing new genetic material that targets cancer cells.
How does gene therapy work?
Gene therapy encompasses several different treatment approaches:
- Protein supplementation — introducing the proteins or molecules that damaged genes (like BAP1 or p53) would normally produce, restoring some of their tumor-suppressing function
- Immune system modification — genetically modifying the patient’s immune cells to recognize and attack mesothelioma cells more effectively
- Suicide genes — introducing genetic material into cancer cells that triggers intentional, programmed cell death (apoptosis)
The common thread is that these approaches are targeted and patient-specific — they affect cancerous cells while leaving healthy cells intact.
Which genes are targeted in mesothelioma gene therapy?
The two most significant genetic targets in mesothelioma are:
- BAP1 — a tumor-suppressing gene mutated in approximately 60–70% of all mesothelioma cases. BAP1 normally controls cell growth, programmed cell death, and DNA repair.
- p53 — often called the “guardian of the genome,” p53 prevents damaged cells from dividing. A 2024 study found that supplementing p53 during chemotherapy enhanced treatment effectiveness and reduced side effects.
Both genes are anti-tumor in nature — when they are functioning, they prevent cancerous growth. When they are mutated, the body loses critical safeguards against cancer.
What are gene signatures and why do they matter?
A gene signature is a cell’s unique pattern of expressing DNA into proteins and other biological molecules. In mesothelioma, analyzing gene signatures can help doctors:
- Predict prognosis — how the cancer is likely to progress
- Assess treatment receptivity — which therapies are most likely to be effective
- Evaluate metastasis risk — how likely the cancer is to spread
- Predict recurrence — the odds of cancer returning after treatment
A mesothelioma-specific gene signature (COL5A2, ITGAV, SPARC, ACTA2) was identified in a 2022 study and is being investigated as a potential therapeutic target.
How is gene therapy different from chemotherapy or immunotherapy?
The three approaches work through fundamentally different mechanisms:
- Chemotherapy — a systemic treatment that disrupts cell division throughout the entire body, affecting both cancerous and healthy cells. Significant side effects.
- Immunotherapy — restores or strengthens the patient’s existing immune system to fight cancer. Fewer side effects than chemo but relies on the immune system being functional enough to respond.
- Gene therapy — directly addresses the genetic mutations driving the cancer by replacing lost gene functions, reprogramming cells, or triggering targeted cell death. Highly specific, patient-tailored, and generally spares healthy cells.
In a multimodal approach, all three could potentially be used together — each attacking the cancer through a different pathway.
Is gene therapy available to mesothelioma patients now?
Gene therapy for mesothelioma is currently only available through clinical trials — it has not yet received FDA approval for widespread clinical use. The field is still in earlier stages of development, and the challenge of compensating for genetic damage without directly repairing the genes themselves remains a significant hurdle. However, as more trials demonstrate safety and effectiveness, FDA approval for mesothelioma-specific gene therapies may follow.
What should I do if I’ve been diagnosed with mesothelioma and want to explore gene therapy?
If you or a loved one has been diagnosed with mesothelioma, ask your oncologist about active clinical trials involving gene therapy. Eligibility depends on your specific diagnosis, stage, cell type, and overall health. For information about your legal options, call (800) 505-6000 or fill out our contact form for a free consultation.
