Science & Technology, UK (Commonwealth Union) – Parkinson is a neurodegenerative condition that can disrupt essential amino acids. The condition is usually identified by confusion, tremors, small handwriting, loss of smell and trouble with sleeping or walking. Many of these symptoms can manifest itself in other conditions hence a final conclusion is made with the use of other tests.
A large-scale Phase 3 clinical trial to determine the role of the drug ambroxol to slow the progression of Parkinson’s disease (PD), is set to commence early this year.
Ambroxol is presently used for the treatment of respiratory conditions, by enhancing the clearance of mucus, reduces coughing and has anti-inflammatory properties.
Pre-clinical studies, led By Professor Schapira at the University College London (UCL) Queen Square Institute of Neurology marked ambroxol as a candidate drug to lower progression of PD.
Findings of the Phase 2 clinical trial by Professor Schapira that was conducted at UCL tested ambroxol in persons with PD. The findings revealed that ambroxol could effectively move into the brain and elevate levels of the protein GCase (glucocerebrosidase). Gcase permits cells to take out waste proteins, such as alpha-synuclein (a protein that gathers up in Parkinson’s and is believed to be a key factor of the condition), with increased efficiency.
The Phase 2 trial further indicated that ambroxol was safe for people with PD with good tolerance.
The world 1sr Phase 3 trial, known as ASPro-PD, is led by Professor Anthony Schapira in collaboration with UK charity, Cure Parkinson’s and Van Andel Institute, after 8 years of work with the PD community.
The trial will see 330 individuals having PD across 10-12 clinical centers in the UK. It will be placebo (medication which gives the perception of effectiveness but no real effect) controlled and participants will use ambroxol for 2 years.
The effectiveness of ambroxol will be gaged on how capable it is to slow the progression of PD by applying a scale including quality of life and movement. Preparations for recruitment of persons for the trial have already commenced.
“I am delighted to be leading this exciting project. This will be the first time a drug specifically applied to a genetic cause of Parkinson’s disease has reached this level of trial and represents ten years of extensive and detailed work in the laboratory and in a proof of principle clinical trial,” explained Professor Schapira.
“The study design is the result of valuable input from people with Parkinson’s, leaders in the field of Parkinson’s, trial design and statistics from the UCL Comprehensive Clinical Trials Unit (CCTU), the MHRA and a consortium of funders led by Cure Parkinson’s, all operating as an effective team to ensure we have reached this stage.”
“We look forward to working with all these groups to ensure successful completion of the study.”
The Phase 2 data from Professor Schapira’s group at UCL discoverd that ambroxol can enhance alpha-synuclein removal, the international Linked Clinical Trials (iLCT) programme gave significance to studies on the drug.
Formed and operated by Cure Parkinson’s and Van Andel Institute, the iLCT programme’s mission is to lower, stop and reverse the progression of PD. The target is to significantly bring down the time to make these treatments available for persons with PD by testing promising drugs presently having extensive safety data and, certain instances, approved by regulators for other medical conditions.
The waiting time for many conditions to patients requiring them urgently often ends up in lengthy trials that can last decades often not reaching patients requiring it urgently as many safety protocols and ethical concerns need to be addressed, however in recent times with the advancement of technology and the use of existing drugs in trials may help speed up its use.
