25 February 2014

Advances in Treating Huntington’s Disease

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Huntington’s Disease is a rare neurological disorder with no effective treatment, yet. But there is hope, at least for the future, coming from a number of research endeavours in Italy and Great Britain.

Juliet Ross is suffering from Huntington’s Disease, a rare neurological disorder that affects one in 10.000 people. Due to a genetic defect, her body produces a toxic protein that damages neurons in her brain. There is no cure for this condition and big pharmaceutical companies usually show little interest in investing in new drugs for rare diseases, but recently the European Commission supported a number of research projects on advances of Huntington’s Disease.

One project is called PADDINGTON, where researchers of Siena Biotech in Italy have developed a new compound that binds to the toxic protein of Huntington’s Disease and disables it. This could slow down the progression of the disease, and preliminary clinical tests are underway.

The second research endeavour is called NEUROSTEMCELLS, where a team in Milan led by Professor Elena Cattaneo studies how the neurons in Huntington’s patients are lost. The researchers want to use stem cells to grow exactly those neurons which are being lost by the disease. Their vision: to use these lab-grown neurons one day for experimental transplants. 

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