The Focused Ultrasound Foundation and The ALS Association established a partnership funding program earlier this year with the goal of advancing the development of new focused ultrasound applications for the treatment and diagnosis of amyotrophic lateral sclerosis (ALS). The organizations will extend up to $1 million in funding to support research projects that address critical unanswered questions to treat ALS patients.
While all projects submitted related to the use of focused ultrasound technology for ALS will be considered, the selection committee will prioritize projects that address the following two research areas.
Research Area #1: Focused Ultrasound and Blood-Brain Barrier Opening (BBBO)
The quantification of neurofilament light chain (NfL) in the blood and cerebrospinal fluid has emerged as a critical endpoint biomarker for pharmaceutical clinical trials in patients with ALS. In addition, studies have reported that the presence of NfL precedes clinical diagnosis by up to 12 months.
The potential to detect NfL earlier in the disease could allow for the delivery of neuroprotective agents and even gene-targeted therapies that could significantly delay or halt the typical course of ALS. Focused ultrasound has been reported to safely and reversibly open the blood-brain barrier (BBB) within the motor cortex of patients with ALS, which could allow for the targeted delivery of therapeutics. This technique could also permit two-way trafficking of NfL and other analytes back into the circulation for earlier and more sensitive detection, characterization of disease subtypes, and disease monitoring.
Some potential areas of interest include:
- Investigating the safety of focused ultrasound BBBO in the motor strip by analyzing the kinetics of NfL release into the circulation and effects on motor function.
- Enhancing our knowledge of biomarker release with focused ultrasound BBBO, including but not limited to proteomics, transcriptomics, and metabolomics.
- Researching the ability of focused ultrasound BBBO to enhance the delivery of various classes of therapeutics for ALS, including but not limited to, existing approved medications, new antisense oligonucleotides (ASOs), gold nanoparticles, and gene-targeted therapies.
Research Area #2: Focused Ultrasound and Neuromodulation (NM)
The ALS Foundation and Focused Ultrasound Foundation encourage projects that will improve the current quality and longevity of life for patients until a cure or new treatments are available. Recent technological advances with indwelling neuromodulators improve clinical function in ALS patients. Clinical trials using focused ultrasound are currently underway to assess noninvasive NM to treat an assortment of neurological diseases. We believe there is the potential to also noninvasively modulate the motor cortex with the goal of improving function in patients with ALS.
Some potential areas of interest include:
- Evaluating the safety of focused ultrasound NM in the motor strip by analyzing the kinetics of NfL release into the circulation and effects on motor function.
- Demonstrating the feasibility and efficacy of focused ultrasound NM to the motor cortex.
Requirements
For consideration, proposed projects must meet the following:
- The project must be collaborative and require a team of at least one focused ultrasound researcher and one ALS expert. Both The ALS Association and the Focused Ultrasound Foundation are committed to building new research teams for ongoing collaborations moving forward. If you need assistance identifying an expert for your team, please contact us.
The following metrics must be captured:
- Biological: analyses of blood samples pre- and post-focused ultrasound as well as histopathological evaluation when necessary
- Acoustic: intensity/pressure (derated and non-derated), voltage/power/energy or whichever parameter used on the focused ultrasound device, pulsing parameters, sonication duration, frequency, and transducer characteristics, including diameter, focal depth, etc.
- Imaging: ultrasound, MR protocols (MR thermometry, acoustic radiation force impulse, shearwave imaging, elastography, functional MRI), or nuclear imaging used for treatment monitoring
Funding
A variety of research funding is available based on the type of project.
- Preclinical research projects will be funded for one to two years with a budget range of $150,000 to $200,000.
- Clinical research projects will be funded for one to three years (with an option to extend based on patient recruitment) with a budget range of $300,000 to $600,000 dependent upon protocol and number of patients.
Project budgets outside of these ranges will be considered on a case-by-case basis with sufficient justification. All monetary figures are in USD.
Eligibility
Applicants must hold a faculty appointment as a tenure-track assistant professor or higher rank at award activation.
Submission Instructions
The deadline to apply for this funding opportunity was Monday, October 2, 2023.
Selected applicants will be invited to submit a full research proposal, with a submission deadline of December 1, 2023, at 5pm Eastern. After review and assessment, the Focused Ultrasound Foundation and The ALS Association will share the costs of funding, and the Focused Ultrasound Foundation will administer the grants.
Questions related to the application process should be directed to Emily White, MD, the Foundation’s managing director of operations.