Major breakthrough in Huntington’s disease drug trial
A research team at University College London’s Huntington’s Disease Centre investigating the first potential treatment for Huntington’s disease has announced “results of ground-breaking importance for Huntington’s disease patients and families”.
A new drug, called IONIS-HTTRx, has been proven to successfully lower the level of the harmful protein called Huntingtin, which causes Huntington’s disease.
Huntington's is caused by an error in a section of DNA called the huntingtin gene. Normally this contains the instructions for making the huntingtin protein, but a fault in the gene damages the protein. The new treatment is designed to ‘silence’ the gene.
The research team at University College London, led by Professor Sarah Tabrizi, say there is now hope the degenerative disease can be stopped.
It took over a decade of pre-clinical development before the first human trial of the huntingtin-lowering drug could begin in 2015, and experts say it could be the biggest breakthrough in neurodegenerative diseases for 50 years.
But the scientists caution that the research is at a very early stage and that while it isn’t a cure, it is a step closer to being able to delay the onset of the disease’s symptoms.
The experimental drug, which is injected into spinal fluid, safely lowered levels of the toxic proteins in the brain during it’s first human trail, which involved 46 participants with early stage Huntington’s disease at 9 study centres in the UK, Germany and Canada.
During the trial, sponsored by Ionis Pharmaceuticals, each patient received four doses of either the drug IONIS-HTTRx or a placebo. As the trial progressed, the dose of the drug was increased. The completion of the trial showed the drug was safe, well tolerated by patients and importantly reduced the levels of huntingtin in the brain.
“The results have been hailed as ‘enormously significant’ because it is the first time any drug has been shown to suppress the effects of the Huntington’s mutation that causes irreversible damage to the brain. Current treatments only help with symptoms, rather than slowing the disease’s progression.”
The Guardian
Professor Tabrizi, Director of the UCL Huntington’s Disease Centre and IONIS-HTTRx Global Chief Investigator, said:
“For the first time a drug has lowered the level of the toxic disease-causing protein in the nervous system, and the drug was safe and well-tolerated. The key now is to move quickly to a larger trial to test whether the drug slows disease progression.”
As a result of these successful outcomes, Ionis’ partner, Roche, will take on the development of the drug.
Cath Stanley, Chief Executive of the Huntington’s Disease Association, said:
“This is a great day for the Huntington’s community. Today's announcement of the results of the trial are of ground breaking importance to families affected by Huntington's disease. Although there is still some way to go before the overall results are known, this is a big step forward.”
If you would like more information about getting involved in Huntington’s disease research visit www.enroll-hd.org
To read about the science behind this major breakthrough and what it means for the Huntington’s community, take a look at en.hdbuzz.net/249
The research is supported by The National Institute for Health Research (NIHR) University College London Hospitals Biomedical Research Centre. The centre is a partnership between UCL and University College London Hospitals NHS Foundation Trust funded by the NIHR to translate scientific breakthroughs into better patient treatments.
A research team at University College London’s Huntington’s Disease Centre investigating the first potential treatment for Huntington’s disease has announced “results of ground-breaking importance for Huntington’s disease patients and families”.
A new drug, called IONIS-HTTRx, has been proven to successfully lower the level of the harmful protein called Huntingtin, which causes Huntington’s disease.
Huntington's is caused by an error in a section of DNA called the huntingtin gene. Normally this contains the instructions for making the huntingtin protein, but a fault in the gene damages the protein. The new treatment is designed to ‘silence’ the gene.
The research team at University College London, led by Professor Sarah Tabrizi, say there is now hope the degenerative disease can be stopped.
It took over a decade of pre-clinical development before the first human trial of the huntingtin-lowering drug could begin in 2015, and experts say it could be the biggest breakthrough in neurodegenerative diseases for 50 years.
But the scientists caution that the research is at a very early stage and that while it isn’t a cure, it is a step closer to being able to delay the onset of the disease’s symptoms.
The experimental drug, which is injected into spinal fluid, safely lowered levels of the toxic proteins in the brain during it’s first human trail, which involved 46 participants with early stage Huntington’s disease at 9 study centres in the UK, Germany and Canada.
During the trial, sponsored by Ionis Pharmaceuticals, each patient received four doses of either the drug IONIS-HTTRx or a placebo. As the trial progressed, the dose of the drug was increased. The completion of the trial showed the drug was safe, well tolerated by patients and importantly reduced the levels of huntingtin in the brain.
“The results have been hailed as ‘enormously significant’ because it is the first time any drug has been shown to suppress the effects of the Huntington’s mutation that causes irreversible damage to the brain. Current treatments only help with symptoms, rather than slowing the disease’s progression.”
The Guardian
Professor Tabrizi, Director of the UCL Huntington’s Disease Centre and IONIS-HTTRx Global Chief Investigator, said:
“For the first time a drug has lowered the level of the toxic disease-causing protein in the nervous system, and the drug was safe and well-tolerated. The key now is to move quickly to a larger trial to test whether the drug slows disease progression.”
As a result of these successful outcomes, Ionis’ partner, Roche, will take on the development of the drug.
Cath Stanley, Chief Executive of the Huntington’s Disease Association, said:
“This is a great day for the Huntington’s community. Today's announcement of the results of the trial are of ground breaking importance to families affected by Huntington's disease. Although there is still some way to go before the overall results are known, this is a big step forward.”
If you would like more information about getting involved in Huntington’s disease research visit www.enroll-hd.org
To read about the science behind this major breakthrough and what it means for the Huntington’s community, take a look at en.hdbuzz.net/249
The research is supported by The National Institute for Health Research (NIHR) University College London Hospitals Biomedical Research Centre. The centre is a partnership between UCL and University College London Hospitals NHS Foundation Trust funded by the NIHR to translate scientific breakthroughs into better patient treatments.