Scientists at the University of Virginia Comprehensive Cancer Center have developed an experimental treatment for glioblastoma that uses focused ultrasound and nanoparticles to deliver cancer-fighting genetic molecules across the blood-brain barrier, slowing tumour growth and extending survival in animal models.
The approach, published in the Journal of Clinical Investigation, targets multiple cancer-driving genes at once using small pieces of genetic material called microRNAs. The researchers combined these microRNAs with nanoparticles and used MRI-guided focused ultrasound together with microscopic bubbles to temporarily open the brain's protective barrier, allowing the treatment to reach the tumour.
Glioblastoma is the most common malignant brain tumour in adults and one of the most aggressive forms of cancer, claiming more than 13,000 lives in the United States each year. It is exceptionally difficult to treat because cancer cells spread into healthy brain tissue, making complete surgical removal nearly impossible, and because the blood-brain barrier blocks many drugs.
Current treatment generally begins with surgery to remove as much of the tumour as possible, followed by radiation and chemotherapy. The UVA strategy takes a different path, using microRNAs that can suppress the activity of several problematic genes simultaneously, rather than relying on single-target drugs. This is significant because many of the molecules that drive cancer growth cannot be targeted by existing medications, and combining multiple conventional drugs can produce harmful side effects.
Roger Abounader, MD, PhD, a professor at UVA's School of Medicine and the study's lead researcher, said the approach could help address molecules for which no drugs exist. "This new approach could help target numerous molecules that promote cancer growth, including those for which no drugs exist, at the same time to achieve better therapies," he said. "We are hoping to translate our findings into future clinical trials for patients with glioblastoma and other brain tumors."
Laboratory tests and animal models showed that the microRNA treatment slowed tumour growth and helped the animals survive longer. The strategy remains experimental and will need further research before it can be tested in humans.
Abounader said the technology could have broader applications. "This approach could have broad applications for several brain and other human diseases," he said. "We and many others are intensively working to lighten the burden of cancer, and hopefully also completely cure it, but this requires a continued investment in cancer and medical research."
UVA Health has a long history in focused ultrasound research. It partnered with the Focused Ultrasound Foundation to establish the Focused Ultrasound Immuno-Oncology (FUSION) Center, the first centre in the world devoted to investigating how focused ultrasound can be combined with cancer immunotherapy. The work also supports the Manning Institute of Biotechnology, which aims to speed the discovery of new medicines.
The study was funded by the National Cancer Institute, the National Institute of Neurological Disorders and Stroke, a UVA Comprehensive Cancer Center pilot grant, a Schiff Foundation grant, the Ben and Catherine Ivy Foundation, and the Focused Ultrasound Foundation.
Analysis
Why This Matters
- Glioblastoma kills more than 13,000 Americans each year and has limited treatment options; a new way to cross the blood-brain barrier could open avenues for many brain conditions.
- The approach targets multiple cancer-driving genes simultaneously, which may overcome drug resistance and avoid the toxicity of combining multiple conventional drugs.
- If the technique advances to human trials, it would represent a significant shift from current single-target therapies to a multi-target genetic strategy.
Background
Glioblastoma is the most common and aggressive malignant brain tumour in adults, with cancer cells that infiltrate healthy tissue and resist standard treatments. The blood-brain barrier, a protective layer of cells that blocks toxins and pathogens, also prevents most drugs from reaching brain tumours. The UVA team has been investigating focused ultrasound for years; the university partnered with the Focused Ultrasound Foundation to create the FUSION Centre, the world's first dedicated to combining focused ultrasound with cancer immunotherapy.
What to Watch
- Whether the UVA team secures regulatory approval and funding to begin Phase I clinical trials in humans.
- The development of similar nanoparticle and ultrasound delivery systems for other brain diseases such as Parkinson's or Alzheimer's.
- Ongoing results from the FUSION Centre's immunotherapy combinations that may complement this microRNA approach.
Checked
12 claims in this story checked against 1 source: 12 supported.
Show each claim
- SupportedSound waves and nanoparticles are being used to deliver an experimental attack on brain cancer.Latest Science News -- ScienceDaily: “To deliver these molecules into the brain, the researchers combined specialized nanoparticles, focused ultrasound and microscopic bubbles.”
- SupportedUVA researchers developed an experimental glioblastoma strategy using focused ultrasound, microbubbles and microRNAs carried by nanoparticles to reach the brain.Latest Science News -- ScienceDaily: “Together, these technologies offer a way to temporarily cross the brain's protective barrier”
- SupportedThe researchers used MRI-guided focused ultrasound and microbubbles to temporarily open the blood-brain barrier.Latest Science News -- ScienceDaily: “The ultrasound works with tiny "microbubbles" to temporarily open the blood-brain barrier.”
- SupportedIn laboratory experiments and animal models, the microRNA treatment slowed tumour growth and helped the animals survive longer.Latest Science News -- ScienceDaily: “Their findings showed that the microRNA treatment slowed tumor growth and helped the animals survive longer.”
- SupportedGlioblastoma claims more than 13,000 lives in the United States each year.Latest Science News -- ScienceDaily: “It claims more than 13,000 lives in the United States each year.”
- SupportedThe study was published in the Journal of Clinical Investigation.Latest Science News -- ScienceDaily: “Abounader and his colleagues published their experimental approach and initial laboratory findings in JCI, the Journal of Clinical Investigation.”
- SupportedAbounader said: “This new approach could help target numerous molecules that promote cancer growth, including those for which no drugs exist, at the same time to achieve better therapies.”Latest Science News -- ScienceDaily: ““This new approach could help target numerous molecules that promote cancer growth, including those for which no drugs exist, at the same time to achieve better therapies,” said Abounader”
- SupportedAbounader said: “We are hoping to translate our findings into future clinical trials for patients with glioblastoma and other brain tumors.”Latest Science News -- ScienceDaily: ““We are hoping to translate our findings into future clinical trials for patients with glioblastoma and other brain tumors.””
- SupportedAbounader said: “This approach could have broad applications for several brain and other human diseases.”Latest Science News -- ScienceDaily: ““This approach could have broad applications for several brain and other human diseases,” Abounader said.”
- SupportedAbounader said: “We and many others are intensively working to lighten the burden of cancer, and hopefully also completely cure it, but this requires a continued investment in cancer and medical research.”Latest Science News -- ScienceDaily: ““We and many others are intensively working to lighten the burden of cancer, and hopefully also completely cure it, but this requires a continued investment in cancer and medical research.””
- SupportedThe FUSION Center is the first centre in the world devoted to investigating how focused ultrasound can be combined with cancer immunotherapy.Latest Science News -- ScienceDaily: “It is the first center in the world devoted specifically to investigating how focused ultrasound can be combined with cancer immunotherapy.”
- SupportedThe research received funding from the National Cancer Institute, the National Institute of Neurological Disorders and Stroke, and the listed foundations and UVA grant.Latest Science News -- ScienceDaily: “Additional support was provided by a UVA Comprehensive Cancer Center pilot grant, a Schiff Foundation grant, the Ben and Catherine Ivy Foundation, and the Focused Ultrasound Foundation.”