Huntington’s disease treated successfully for first time in gene therapy trial | Huntington’s disease
Huntington’s disease, which is a destructive degenerative disease working in families, was successfully treated for the first time in the breakthrough gene therapy study.
The disease caused by a single gene defect constantly kills the brain cells that cause dementia, paralysis and ultimately death. The chances of developing a parent with Huntington have the chance to develop the disease that has never been cured so far.
Three years later, gene therapy slowed the progression of the disease by 75%.
“Now we are being treated for one of the world’s more terrible diseases in the world, Prof said Prof Sarah Tabrizi, Director of Huntington Disease Center. “This is absolutely big. I’m really glad.”
The drug, which inactivates the mutant protein that causes Huntington, is given to the brain in a single shot during a 12-20-hour surgical procedure, ie it will be expensive. The breakthrough sends hoping fluctuations through the Huntington community, many of which witnesses the ruthless effect of the disease on family members.
Typically, the first symptoms in your 30s or 40s include mood changes, anger and depression. Later, patients develop uncontrolled shaking movements, dementia and ultimately paralysis, some people die in ten years.
Tabrizi said that with treatment, people can work significantly independently and the dramatic effect of treatment increases the likelihood of preventing symptoms if the treatment is given at a previous stage.
In the UK, it means that there are at least 20,000 people who are 6,000 to 10,000 people with Huntington’s disease in the UK and at least 20,000 people. However, in families affected by Huntington, only one -fifth has now chosen to test the gene, because existing treatments only help symptoms instead of slowing down the brutal progress of the disease.
“Now I think many people will come to the fore for genetic test because there is a treatment,” Tabrizi said.
Mutant Huntington gene contains instructions of cells to make a toxic version of a brain protein called Huntingtin. The therapy is given by a modified harmless virus to give a specific DNA sequence to neurons.
To prevent negative reactions, the virus is very slow to two separate brain regions from a micro-coater, which is a complex procedure that lasts 12 to 20 hours.
After DNA is given to neurons, the cells tell the cells to prevent the production of the toxic version of Huntingtin.
Results obtained from experiment containing 29 patients treated in the UK and the United States Released by UNIQure companyHowever, although all the details have not yet been published. Three years after treatment, the high -dose of the drug had an average slowing of a 75% slowdown in the progression of the disease based on motor function, cognition and daily life experience.
There were also clear indicators that neurons were saved in the brain, and neurophylament levels (symptoms of cell death) were significantly lower in the treatment group.
Uniqure Chief Medical Officer Walid Abi-Saab added that the company hoped to approve the drug for approval in the US at the beginning of next year and said, uz We are incredibly excited about what they can represent for individuals and families affected by Huntington’s disease, ”he said.
