StemCell Therapy

The world's first mass-market gene therapy has been legalized in Europe. Glybera, which treats a painful disorder that leads to pancreatitis, will be available in hospitals beginning next year.

Chances are you haven't heard much about lipoprotein lipase deficiency (LPLD), a disease that leads to pancreatitis. This rare disease, however, is at the center of world-changing medical advances.

Last week the European Union approved a gene therapy treatment for LPLD--and this marks the first time any medical treatment that rewrites a patients' DNA has been approved for commercial use.

The treatment, called Glybera, will be released by Dutch firm uniQure in the second half of 2013. Glybera will be administered to patients by specially trained doctors at a limited number of European hospitals. Patients receiving treatment have their DNA altered by a series of injections into their leg muscles, which helps normalize the metabolism of fat particles carried in the blood. LPLD prevents sufferers from properly metabolizing these particles, leading to a host of side effects including pancreatitis.

Gene therapy is an emerging form of medicine, focusing on the use of DNA to rewrite or supplement existing genes. This experimental practice is expected to lead to a series of breakthroughs over the next 50 or so years for various types of cancers, Parkinson's disease, sickle cell anemia, and a host of inherited conditions. Studies involving different forms of gene therapy are currently under way in Europe and the United States--one famous case in 2007 involved a man who was cured of HIV through stem cell transplantation. Stem cell transplants are one type of gene therapy used to give patients therapeutic DNA; other methods use different sources.

LPLD affects approximately one in 1,000,000 people worldwide. Apart from (frequently fatal) acute pancreatitis, patients regularly suffer from yellow spots on their skin, swollen abdomens, enlarged livers, severe abdominal pain, and diarrhea. The condition is currently treated through diet--patients are encouraged to go on a strict low-fat, alcohol-and-red-meat-free diet to mitigate symptoms.

By helping to normalize the metabolism of fat, Glybera prevents inflammation of the pancreas thereby averting the associated pain and suffering and, if administered early enough, the associated co-morbidities, said Professor John Kastelein of the University of Amsterdam in a statement.

Gene therapy treatments in the United States are primarily focused on cancer, including leukemia. No other gene therapy products are expected to be approved by major medical regulator agencies in 2012. The European Commission, which handles medical approvals for Europe, is widely considered to have a bureaucratic pipeline that is far more open to gene therapy than the United States.

[Image: DNA damage via Tom Ellenberger/Washington University School of Medicine]

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