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Neurodegenerative disease trials

A phase I/II study for bilateral putamen delivery of recombinant glucocerebrosidase in patients with Parkinson's disease using MR-guided focused ultrasound induced opening of the blood-brain barrier

The treatment of Parkinson’s disease currently uses medications that help manage symptoms, however, new medicines aiming to slow the progress of the disease are still being developed. A potential therapy for PD being evaluated here, is known as enzyme replacement therapy (ERT), and is approved for a treating other diseases. ERT is able to replace an important enzyme deficient in patients with PD. One goal of this clinical trial is to evaluate this ERT which is, a large molecule, can gain access to brain areas most affected by PD.

An emerging therapeutic device, called focused ultrasound (FUS) delivers ultrasound energy across skull bone to targeted areas of the brain, temporarily opening small regions of the blood brain barrier or BBB. Using focused ultrasound in conjunction with GCase delivery, allows large therapeutics to access areas of the brain where they otherwise could not reach.

In this study patients will undergo up to 3 blood brain barrier opening treatments, combined with ERT therapy every 2 weeks. Following treatment, patients will be followed up for up to 12 months and undergo a series of imaging scans, questionnaires and clinical safety evaluations.

Status: Expected 2022

Investigation of the safety of low-intensity focused ultrasound in opening the blood-brain barrier and delivery of therapeutic in Parkinson’s disease

The goal of this pilot trial was to investigate the safety of opening the blood-brain barrier (BBB) in key motor areas known to be implicated in Parkinson’s disease (PD) as well as delivering a drug therapeutic directly to these areas of the brain using the low-intensity MRI-guided focused ultrasound. The putamen, which plays a role in movement was the area of brain that was targeted with ultrasound.. The study also explored the safety of delivering enzyme replacement in the brain as a possible approach to reduce or stop neurodegeneration in Parkinson’s disease.

In this initial trial PD patients aged 35-73, receive three BBB opening sessions along with doses of an enzyme replacement therapy every two weeks. Imaging and follow up visits where completed up to six months after the last focused ultrasound treatment session.

Status: Completed

Safety data from this study will help build on this technique and allow us to expand therapeutics options for patients diagnosed with PD.

For more information or to inquire about eligibility for the trial, please email Please note FUS Parkinson’s in the email subject line

A Phase 2a, Open-label, Single-Arm Clinical Trial of Oral Drug Therapy Coupled with Primary Motor Cortex Blood-Brain Barrier Opening using Transcranial MR-Guided Focused Ultrasound in Amyotrophic Lateral Sclerosis (ALS)

In the initial safety trial, investigators at Sunnybrook showed that it was safe to open the BBB in the motor cortex of patients with ALS. Building upon these results, the current study will investigate the safety of opening up the blood brain barrier in the motor cortex while delivering a drug therapeutic. In this new study patients will undergo serial BBB treatments spaced a few weeks apart. Follow-ups will include neurological examination MRI scans, MRI and clinical visits and will occur up to 6 weeks after the last BBB treatment session.

Successful outcomes from this trial may allow us to use this technique to deliver stem cells, gene therapy or large molecules to key areas of the brain of patients with ALS.

Status: Expected 2022

For more information or to inquire about eligibility for the trial, please email Please note FUS ALS in the email subject line

ExAblate Blood-Brain Barrier Opening for Treatment of Alzheimer's Disease

This ongoing study is investigating the safety and feasibility of repeated BBB opening in multiple regions of the brain in patients with mild to moderate Alzheimer’s disease (AD).

The brain areas targeted with ultrasound will include regions displaying amyloid and tau as demonstrated on PET imaging, as well as structures implicated in memory and early involvement in AD. Exploration of cognitive test scores and clinical biomarkers in blood and cerebrospinal fluid (CSF) will be evaluated over the 6 months following the final ultrasound procedure. These findings will inform further trial development of focused ultrasound as a tool for enhancing new drug therapies in AD and other cognitive disorders.

Status: Expected 2022

For more information or to inquire about eligibility for the trial, please email Please note FUS Alzheimer’s disease in the email subject line.