Newswise — CHARLOTTESVILLE, VA (January 10, 2025) — Results from a clinical trial using focused ultrasound to treat Alzheimer’s disease were published today in the Journal of Neurosurgery, representing a critically important new direction in the treatment of Alzheimer’s. The study, led by Jin Woo Chang, MD, PhD, in Seoul, Korea, demonstrated that repetitive focused ultrasound–mediated blood-brain barrier (BBB) opening on both frontal lobes is safe and can reduce amyloid plaques – a hallmark of Alzheimer’s – even without the co-current administration of Alzheimer’s drugs. The study also found improvement in common neuropsychiatric symptoms associated with the disease.
A clinical trial, sponsored by the Focused Ultrasound Foundation, assessed the safety and effectiveness of more frequent and larger openings of the blood-brain barrier (BBB) compared to earlier research. The study involved six female participants aged between 50 and 85, each receiving BBB openings in both frontal lobes three times, spaced two months apart. The procedures utilized DEFINITY® (perflutren lipid microsphere) microbubbles administered via intravenous injection, along with the ExAblate Neuro 220 kHz low-intensity focused ultrasound system.
The study’s average focused ultrasound–mediated BBB opening volume of 43.1 cubic centimeters is twice as large as Dr. Chang’s previous trials and larger than any other previous clinical trial. This achievement exposed more amyloid plaques in the brain to focused ultrasound than ever before, representing an important milestone in Alzheimer’s treatment.
Dr. Chang, a neurosurgery professor at Korea University Anam Hospital who worked alongside neurologists from Yonsei University on this research, stated, “We created this protocol to maximize benefits and to explore the application of focused ultrasound in broader brain regions impacted by Alzheimer’s disease. This study marks the beginning of a deeper insight into the effects of blood-brain barrier (BBB) opening on its own, and it will lay the groundwork for upcoming trials that will assess the potential advantages of incorporating drug delivery into the affected brain areas.”
Levels of amyloid plaques and clinical evaluations were assessed prior to and following the treatment. Without the administration of any medications, researchers were able to specifically analyze the impact of the focused ultrasound-induced opening of the blood-brain barrier.
Enhancements in the CGA-NPI score—a clinical assessment measuring neuropsychiatric symptoms linked to Alzheimer's disease, including delusions, agitation, irritability, and anxiety—were noted in five out of six participants (83%). Additionally, there were no reported adverse events related to the treatment, highlighting the safety of this method.
"The findings from this preliminary study are promising, stimulating, and thought-provoking, yet they require validation through more extensive research," stated Dr. Neal Kassell, the founder and chairman of the Focused Ultrasound Foundation. "Research into Alzheimer’s has seen little progress over the last several decades, but focused ultrasound presents a beacon of hope in a domain that has persistently been in search of groundbreaking solutions and could fundamentally change the trajectory of this debilitating condition. Understanding the effects of blood-brain barrier opening without the influence of medication in Alzheimer’s patients significantly enriches the body of knowledge in this area."
Following the encouraging findings of the study finalized in 2023, the Focused Ultrasound Foundation is sponsoring a new research initiative aimed at examining the cognitive effects associated with larger volumes of blood-brain barrier (BBB) openings. This trial will additionally assess the possible advantages of integrating focused ultrasound with drug delivery methods to enhance treatment efficacy for individuals with Alzheimer’s disease.
For more information, please visit www.fusfoundation.org.
For interviews with Dr. Neal Kassell, Dr. Chang, or another representative of the Focused Ultrasound Foundation, please contact Allison Preston-Smith at (434) 326-9820 or [email protected].
About Focused Ultrasound Focused ultrasound uses ultrasound energy guided by real-time imaging to treat tissue deep in the body without incisions or radiation. It is FDA-approved in the United States to treat essential tremor, Parkinson’s disease, liver tumors, uterine fibroids, pain from bone metastases, osteoid osteoma, and the prostate. Dozens of additional indications are approved outside of the US. The technology is in various stages of research and development for more than 180 diseases.
The Focused Ultrasound Foundation is an organization dedicated to advancing the field of focused ultrasound technology for medical applications. By supporting research, clinical trials, and education, the foundation aims to improve patient outcomes and promote the use of this non-invasive treatment method. Through collaborations with scientists, healthcare professionals, and industry partners, the foundation works to raise awareness and accelerate the development of focused ultrasound therapies for various conditions, including cancer, neurological disorders, and more.
Located in Charlottesville, VA, the Focused Ultrasound Foundation was established with the mission of enhancing the lives of millions globally by hastening the advancement of focused ultrasound, an innovative and noninvasive technology. The Foundation aims to facilitate worldwide acceptance as quickly as possible by coordinating and financing research initiatives, promoting collaboration, and raising awareness among both patients and healthcare professionals. Since its inception in 2006, it has grown to be the foremost nongovernmental funding organization for focused ultrasound research.
