Gene therapy nears regulatory approval in Europe
The New York Times reports that the European Medicines Agency has recommended approval of gene therapy to treat a rare disease. Should the European Commission follow the advice, it would become the first approval of a gene therapy drug in the Western world.
According to the NYT:
Gene therapy involves providing the body with genes it needs, such as correct copies of defective genes that cause genetic disorders. Its use in the West so far has been confined to clinical trials.
The therapy recommended for approval in Europe, called Glybera, was developed by uniQure, a Dutch company. It treats lipoprotein lipase deficiency, a disease that affects only several hundred people in the European Union and a similar number in North America.
People with the disease have a genetic mutation that prevents them from producing an enzyme needed to break down certain fat-carrying particles that circulate in the bloodstream after meals. Without the enzyme, so much fat can accumulate that the blood looks white rather than red.
Glybera provides correct copies of the lipoprotein lipase gene, which allows patients to make some of the needed enzyme. A single treatment, consisting of injections into multiple spots on the leg muscles on the same day, is expected to last for several years, if not longer, said Jörn Aldag, chief executive of uniQure.
ALS patient feels great after stem cell transplant
Took part in Phase I of Dr. Eva Feldman's human clinical trial
Ted Harada tells Crain's Detroit Business that nearly nine months after receiving stem cell injections to his spinal cord, improvement persists.
The birth of two human clinical trials
The Taubman Institute’s overriding purpose is to discover potential new treatments that can be tested in clinical trials. Watch as two Taubman Scholars explain how they made it happen.
Institute training video helps physicians overseas
U-M exam method for diabetic nerve damage translated to Mandarin Chinese. The Taubman Institute has produced video of an exam protocol that will help doctors in Asia and elsewhere as they grapple with growing diabetes epidemics and the resulting complications.
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Taubman Scholars direct 31 human clinical trials
Science funded by the Taubman Institute has led to 31 current human clinical trials, studying potential therapies for diseases including breast cancer, muscular dystrophy, diabetes and ALS. See the complete list of trials.
People who care
Generous donors fund institute's summer students
Leadership advisory board members fund Tauber Family Student Internship Program
Three future medical scientists will work with Taubman Institute researchers starting in June.
State leaders laud Taubman Institute accomplishments
Leaders of state and local government visited the Taubman Institute on March 18 to tour Taubman Scholar labs and discuss the potential medical research offers for both improving the health of residents and establishing new jobs and businesses in Michigan.