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“Historic” Action by FDA Could Make Gene Therapy a Reality for Mesothelioma Patients

The U.S. Food and Drug Administration (FDA) has cleared a gene-altering therapy known as Kymriah (tisagenlecleucel) for pediatric leukemia. Approved for distribution by Novartis Pharmaceuticals Corp., the treatment is now the first gene therapy to be cleared in the U.S., which the FDA says is an “historic” action with major consequences for patients with cancer and other diseases—including mesothelioma.

In a recent Washington Post article, FDA Commissioner Scott Gottlieb said, “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 cure intractable illnesses. He said companies are pursuing hundreds of experimental treatments involving gene therapy products.

A clinical trial for one promising experimental treatment, involving the same gene-altering therapy (chimeric antigen receptor (CAR) T-cell immunotherapy (‘Living Drug’)) and mesothelioma, is currently underway at Memorial Sloan Kettering Cancer Center (MSK). Led by Dr. Prasad S. Adusumilli, the Thoracic Cancer Research team at MSK has identified mesothelin as an ideal target for cellular immunotherapy. Mesothelin is a cancer-cell surface antigen expressed in a majority of patients with mesothelioma, lung adenocarcinoma, and triple-negative breast cancer.

Explains Adusumilli, “published studies from our laboratory have shown that mesothelin expression is associated with cancer cell aggressiveness. Using clinically relevant mouse models, we have demonstrated the antitumor efficacy of mesothelin-targeted CAR T cells when injected directly into the pleural cavity.”

“Chimeric antigen receptor (CAR) T cell immunotherapy, a targeted therapy that genetically redirects the patient’s own T cells to lyse cancer cells,” he says, “has achieved dramatic success in hematological malignancies. CARs are genetically engineered antigen-specific receptors that, when transduced into T cells, can specifically recognize and kill cancer cells. Groundbreaking MSK studies have shown complete remissions in patients with therapy-refractory lymphoma and leukemia,” so far.

Researchers for the clinical trial known as Malignant Pleural Disease Treated With Autologous T Cells Genetically Engineered to Target the Cancer-Cell Surface Antigen Mesothelin are still recruiting participants.

If you have been diagnosed with mesothelioma, ask your doctor about groundbreaking studies such as the MSK gene therapy clinical trial. Participating in a clinical trial such as this could be effective in helping treat your specific type of mesothelioma, so talk to your doctor today.



Adusumilli, Prasad S. "Exciting New CAR T Cell Therapy Trial Opens at MSK." Memorial Sloan Kettering. Memorial Sloan Kettering Cancer Center (MSK), Thoracic Surgery Service, 23 Sept. 2016. Web. 08 Nov. 2017.

"FDA Approval Brings First Gene Therapy to the United States." U.S. Food and Drug Administration. Office of the Commissioner, 30 Aug. 2017. Web. 08 Nov. 2017.

McGinley, Laurie, and Carolyn Y. Johnson. "FDA Clears First Gene-altering Therapy - ‘a Living Drug’ - for Childhood Leukemia." The Washington Post. WP Company, 30 Aug. 2017. Web. 08 Nov. 2017.

"What's New in Malignant Mesothelioma Research and Treatment?" American Cancer Society. American Cancer Society, Inc., 2017. Web. 8 Nov. 2017.