Focused Ultrasound changed the life of one uterine fibroid patient
Focal Drug Delivery
Introduction
The American Cancer Society estimated that in 2009 nearly 1.5 million new cases of cancer would be diagnosed and just over half a million individuals would die from cancer. Of those 1.5 million patients diagnosed with cancer in 2009, most will be treated with what is still the standard combination of surgery, radiation therapy and chemotherapy, all of which have well documented deleterious side effects and low success rates. Imagine a treatment option by which all three could be performed with minimal invasiveness and side effects—we at the FUS Foundation believe MR-guided focused ultrasound-mediated drug delivery has the potential to be that solution.
The FUS Foundation is dedicated to accelerating research and development of patient treatments using one of today’s most revolutionary and promising medical technologies: non-invasive Magnetic Resonance-Guided Focused Ultrasound (FUS). The foundation’s work is motivated by the belief that FUS treatments could become the ultimate in minimally invasive surgery, serve as a viable alternative to radiation therapy and offer a new platform for precise drug delivery— applications with the potential to alleviate suffering, save lives and quicken recovery times for millions of patients worldwide.
Rationale
FUS-mediated drug delivery has the potential to impact the largest number of patients numerically in an epidemiological sense, yet has considerable challenges to surmount as it sits at the intersection of multiple industries (both device and medical therapies) and stakeholders. The foundation has an established track-record of engaging various stakeholder groups through its Brain Program and has a unique capacity to aid in the direction and collaboration within this important and game-changing field.
Strategy
The FUS Foundation will assume facilitative and leadership roles in the development of an internally-driven research program in focal drug delivery (Mission Statement below). This is consistent with the foundation's vision and mission and role in bringing together industry, academia, and funding to accelerate the development and adoption of focused ultrasound. Modeled after the foundation’s Brain Program, the goal of the foundation’s Focal Drug Delivery Program is to work with the key stakeholders, including academic researchers, clinicians, pharmaceutical companies, focused ultrasound manufacturers, governmental agencies and disease-specific foundations, to accelerate clinical applications of FUS-mediated drug delivery. The approach will combine advanced imaging techniques, activation of the immunological response, tumor ablation and the targeted delivery of drugs, thus reducing the system-wide effects of current drug therapies. Success will hinge on collaboration among the best and the brightest in the field.
Focal Drug Delivery Program Mission Statement
The aims of this program are to define and engage the research community in FUS-mediated drug delivery in order to:
- Clarify the clinical applications that will benefit most from MR guidance
- Develop a research roadmap for clinical applications for MR-guided FUS-mediated drug delivery
- Accelerate research by fostering collaboration among the academic researchers, pharmaceutical companies, focused ultrasound companies and clinical community
Organization
Core Stakeholder group
- Academicians already working with ultrasound and drug delivery systems
- Will serve as the foundation for the program’s Steering Committee
Broader Stakeholder group
- Researchers and Clinicians with experience in drug delivery systems and/or focus ultrasound in clinical applications relevant to FUS-mediated drug delivery
Priority Clinical Indications
Based on the mission of the foundation and input from the Broader Stakeholder group, the Core Stakeholder group defined five clinical indications as top priority:
- Pancreatic cancer
- Blood-brain barrier opening in brain for the treatment of glioblastoma multiforma (GBM)
- Liver cancer
- Head and Neck cancer
- Prostate cancer
Program Activities
July 2010: Core Stakeholder group meeting
A key objective of the Core Stakeholder group's work has been to provide an answer to the question:
Is there a compelling role for the foundation in FUS-mediated drug delivery whereby it can add unique value while contributing to its mission of accelerating the adoption of MR-guided focused ultrasound clinical applications?
To help define the role of the foundation in drug delivery, a survey was completed by the Core Stakeholder group followed by a face-to-face meeting in July 2010. Meeting outcomes included:
Identification of five clinical indications as top priority (above).
The initial treatment approach should emphasize focused ultrasound mild hyperthermia using existing chemotherapeutics in conjunction with high intensity focused ultrasound (HIFU or FUS).
While the initial emphasis is on focused ultrasound mild hyperthermia in combination with chemotherapy, experimental models should be designed with multiple arms to include microbubbles, as literature suggests significant drug delivery enhancement with the addition of microbubbles.
Indication-specific research questions were identified as well as a set of pre-clinical, mechanistic and/or technical questions common to all five indications.
Recommended role to the foundation on its role in FUS-mediated drug delivery
- Serve as a nexus for collaboration, through faciliation and leadership of working groups, workshops, etc.
- Supply seed funding to initiate research projects
