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Medical Milestone: Gene Therapy Drug Approved To Fix Genetic Code Typos

FDA-Approved Gene Therapy

Mark your calendar: A new era in medicine has begun.

The European Union has licensed for sale the first gene-therapy drug in the Western world. Gene therapy is a type of medicine that treats disease by replacing defective genes with functioning genes. It holds great potential for treating diseases caused by defective genes, though it has remained largely experimental and confined to research laboratories to date. One day, patients with mesothelioma may benefit.

The European Commission on Friday granted approval to Glybera, a gene therapy medicine that treats a rare inherited disorder called lipoprotein lipase deficiency (LPLD), according to the BBC News.


People with the extremely rare disease are unable to produce enough of an enzyme to digest fat properly. Fat levels in the blood may increase dramatically. A person with the enzyme deficiency may suffer life-threatening pancreatitis attacks as well as early onset of diabetes.

The drug developed by a Dutch company, UniQuire, in a single injection that contains a gene that helps the body produce the necessary enzyme.

According to The Wall Street Journal, the European action is a milestone for the field of gene therapy. In an article in the WSJ, Len Seymour, a professor of gene therapy at Oxford University who is unaffiliated with Glybera, said the approval begins to exemplify what genetically coded medicines can do.

UniQure plans to file an application with the U.S. Food and Drug Administration next year, seeking regulatory approval for the drug, the company said in a statement. No gene therapy medicines have been approved in the United States so far.

Researchers have been studying the effectiveness of gene therapy in treating various diseases in clinical trials since a landmark discovery in 1989 that an abnormality in one gene caused the disease cystic fibrosis. That led to the premise that doctors could treat a patient’s disease by identifying an abnormal gene mutation and replacing the defective gene with a corrected copy. But scientists have run into many set backs in the development of gene therapy.

While the initial applications of gene therapy are likely to involve rare diseases that may be cured by replacing a single defective gene, researchers also are exploring the potential of gene therapy for treatment of cancer including mesothelioma, a cancer caused by asbestos exposure.

The University of Pennsylvania has an ongoing gene therapy clinical trial for patients who are newly diagnosed with mesothelioma and patients whose cancer has not responded to other treatments. Patients receive a combination of chemotherapy and a new type of gene therapy called immuno-gene therapy that uses a modified common cold virus to trigger the patient’s immune system to destroy cancer cells. The doctors have been encouraged by the response of mesothelioma patients receiving the experimental treatment, Dr. Daniel Sterman an associate professor at the University of Pennsylvania said in a news release.


Approximately 2,500 to 3,000 people are diagnosed with mesothelioma each year in the United States. Most are older workers, retired workers and veterans who were exposed to asbestos fibers decades ago in a workplace or in military service.

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