Now in its eighth year, World Alzheimer’s Month is an international campaign to raise awareness and challenge the stigma that surrounds dementia. For the UK DRI, these 12 months have been marked by significant milestones for both the institute, and crucially the field of neurodegenerative disease and search for viable therapeutics.
New hope for trials
One of the standout breakthroughs came late last year with news that a Phase1/2a trial targeting Huntington’s disease, led by UK DRI Group Leader Prof Sarah Tabrizi, had successfully lowered levels of the harmful huntingtin protein in the nervous system and that the drug was safe and well-tolerated. Following full publication of results in May 2019, Sarah said she is excited to be moving forward with global Phase 3 trials.
Just this week, Dr Ahmad Al Khleifat broke news that thefirst dose of gene therapy targeting C9orf72 in ALS was successfully delivered at King’s College Hospital. This study, led by UK DRI Associate Director Prof Chris Shaw, is a key milestone for the motor neuron disease community and if successful, will have wider implications for the neurodegenerative field. On this theme, UK DRI announced its first ‘Directors’ Strategic Initiative’ in July, establishing an Adeno-associated virus (AAV) core facility at UK DRI at King’s. This will aid the whole institute in the design, pre-clinical validation and clinical trials of AAV vectors.