From the April 2016 issue of HealthCare Business News magazine
For example, two separate laboratories using two different Alzheimer’s mouse models have demonstrated that focused ultrasound and micro-bubbles alone, through temporary opening of the BBB, appear to facilitate the brain’s clearance of beta amyloid, a key pathologic protein related to Alzheimer’s. The approach also demonstrated improved memory and cognitive function with no unwanted damage to brain tissue. While the results are preliminary, having two sites validate this approach is very promising. Based on these two pre-clinical studies, a pilot clinical trial using focused ultrasound to treat Alzheimer’s is being organized.
Safe, reliable and temporary opening of the BBB remains an unmet critical medical need for the treatment of a range of diseases. The current methods of getting through this barrier, either via direct brain injections or using mannitol, are far from ideal, and focused ultrasound may offer a better option. Furthermore, the ability of focused ultrasound to non-invasively induce various other effects in the brain, including precise ablation, immunomodulation and neuromodulation, offers potential future benefits to disorders beyond Alzheimer’s, -Parkinson’s, epilepsy, glioblastoma and possibly others — and has the potential to impact millions of patients worldwide.
Grisham book raises hope, increases awareness
For widespread clinical adoption of focused ultrasound to become a reality, there is a continuous need to raise awareness of this promising and multi-functional platform technology among important stakeholder groups including physicians, payers, hospital administrators and patients. In an effort to raise awareness on a wide scale, John Grisham has written a short book about focused ultrasound called The Tumor. It is a fictional account portraying the potential of focused ultrasound to impact the lives of many suffering from deadly or debilitating diseases such as brain tumors.
Grisham has been a Board member and steadfast supporter of the Focused Ultrasound Foundation for years. He decided to write this book to ignite widespread interest in the field and potentially drive donations to fund future research. The book is available through the Foundation (www. thetumor.org) and a free e-book on Amazon. More than 200,000 books have been ordered to date.
What’s ahead?
Before the end of 2016, the FDA should rule on focused ultrasound to treat essential tremor using a dedicated brain system. Promising results of a pilot trial of 15 patients, published in the New England Journal of Medicine, led to a larger multi-site pivotal trial. The FDA is currently reviewing the PMA submission of this pivotal trial. Approval will open the door to treatment of other movement disorders, as well as psychiatric disorders, using precise ablation of known targets in the brain. Clinical trials are ongoing in the U.S. for Parkinson’s tremor and dyskinesia, and are on the horizon for epilepsy, OCD, depression and dystonia.
