CUBRIC enters its first multi-centre phase 1 clinical trial
25 May 2017
Huntington's disease patients have received their first doses on an experimental RNA targeting drug as CUBRIC celebrates its first clinical trial involvement since opening last June.
Huntington’s Disease (HD) patients underwent their first MRI scans in one of CUBRIC’s Siemens Prisma 3T scanners in March this year, an occasion that marks the centre’s first clinical trial involvement. The trial is set to recruit patients with very early symptoms of HD, and CUBRIC is just one of several MRI sites across the UK and Europe who are involved.
Ionis Pharmaceuticals has partnered with Roche to develop the experimental drug, IONIS-HTTRx, to treat Huntington’s disease. The trial aims to test the safety of the drug which is administered intrathecally, by injection into spinal fluid, to improve its delivery to the brain.
This first-in-human drug works by targeting the known cause of the disease: a toxic protein called mutant huntingtin which slowly damages and kills neurons, leading to the progressive and ultimately fatal decline in mental and physical abilities that is the devastating hallmark of Huntington’s disease. This approach has the potential to prevent or slow the progression of this devastating disease.
Using MRI, scientists hope to identify subtle changes in brain microstructure and function, called ‘biomarkers’ that may help to inform the design if it rolls out to phase 2 or 3. To do this, researchers at Cardiff are scanning HD patients at multiple time points over several months, during which they receive multiple doses of the drug.
If this first-in-human trial proves that the drug is safe, then Ionis and its partner Roche hope to advance the drug to the next clinical study with the ultimate goal of commercialising the drug.
Dr Hannah Furby, the lead imaging researcher for the trial at CUBRIC, cheered the news that Cardiff University would be entering the trial.
I am overjoyed that the trial will be opening up its remit to patients within Wales. The fact that the drug trial has already come so far is really encouraging, and brings us closer to finding a cure for this devastating disease
She also commented on what this means for the new brain imaging centre. “The IONIS-HTTRx trial will hopefully mark the first in a series of clinical trials to take place in CUBRIC in the future”.
The trial is still ongoing in Cardiff, but we are not currently recruiting any more patients.