The Ultrasound and Elasticity Imaging Laboratory at Columbia University, led by Elisa Konofagou, Robert and Margaret Hariri Professor of Biomedical Engineering, is making strides in the realm of ultrasound-based medical technology.
Their recent work has shown promising advancements in the treatment of Alzheimer’s disease.
Advancing Ultrasound Technology
The laboratory’s work spans several areas, from developing algorithms to detect microscopic changes in tissue caused by physiological processes to using focused ultrasound for drug delivery to the brain.
One of their most significant achievements has been in using focused ultrasound to non-invasively open the blood-brain barrier (BBB), which could enable the delivery of gene-editing vectors to the brain.
Crossing the Blood-Brain Barrier
The BBB has traditionally been a significant obstacle in treating neurological diseases like Alzheimer’s and Parkinson’s. The barrier protects the brain but also prevents most drugs from reaching their target areas.
Collaborating with physicians at Columbia University Irving Medical Center, the team has explored methods to circumvent this challenge.
Two Papers, Two Breakthroughs
The team has recently published two significant papers. One paper, published in PNAS, highlights the use of focused ultrasound and systemically administered microbubbles to open the BBB.
This technique not only allows genome editing but also boosts the gene copies by 20 times. This is a significant advance, allowing targeted gene therapy for diseases like Alzheimer’s.
Another paper, published in Theranostics, indicates that opening the BBB can also awaken the brain’s immune system, reducing the beta amyloid and tau loads in Alzheimer’s patients and improving working memory.
A clinical trial in Alzheimer’s patients also showed modest beta amyloid reduction in the region where the BBB was opened.
Therapeutic Implications
These papers demonstrate that focused ultrasound could be a critical tool in Alzheimer’s treatment, either by enhancing gene-editing or through immunomodulation.
Konofagou notes that the technology’s potential for simultaneous gene editing and immunomodulation could be particularly important for treating Alzheimer’s, especially in its early stages.
With these promising results, the Konofagou group is now testing their focused ultrasound technique on Alzheimer’s patients.
They aim to reduce tau and beta amyloid loads and improve memory deficits. The team is also collaborating with the Leong group to move their gene-editing technique closer to clinical application.
Conclusion
The research coming out of Elisa Konofagou’s laboratory represents a considerable leap forward in our understanding and potential treatment of Alzheimer’s disease.
By exploiting focused ultrasound technology, researchers are getting closer to effective therapies that can cross the BBB, opening new avenues for treating this devastating disease.
If you care about Alzheimer’s disease, please read studies that bad lifestyle habits can cause Alzheimer’s disease, and this new drug may help treat Alzheimer’s disease.
For more information about brain health, please see recent studies about a new early sign of Alzheimer’s disease, and results showing this brain problem can increase risk of stroke for up to five years.
The research findings can be found in PNAS.
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