A roundup of today’s big neuro rehab stories from the NR Times team
Study to explore whether gene therapy can work against brain tumours
Grant of up to $11 million will fund a clinical trial at UCSF that uses a smarter new CAR-T guided by precision technology.
A type of gene therapy called CAR-T that has extended survival for thousands of patients with leukaemia and other blood cancers is being adapted at UC San Francisco to treat people with glioblastoma, the most common and deadly adult brain tumour.
This new more powerful version of CAR-T employs a novel technology developed at UCSF called synthetic notch (synNotch) that both protects healthy tissue from damage and enables the treatment to work more effectively.
UCSF opened enrolment this week for a clinical trial that is using the technology for the first time in people. A second trial, also at UCSF, is slated for 2025.
Approximately 12,000 Americans are diagnosed each year with glioblastoma. Patients survive on average for just 15 months after their diagnosis, and new treatments are urgently needed.
Study reveals potential diagnostic marker for multiple sclerosis years before symptom onset
A new study published today in Nature Medicine unveils a significant breakthrough in the understanding and early detection of multiple sclerosis (MS).
Researchers have identified a unique autoantibody signature present in approximately 10 per cent of patients with MS years before the onset of clinical symptoms.
Danillo Augusto, Ph.D is an assistant professor in biology at the University of North Carolina at Charlotte and a co-author of the study.
The researcher said: “This study sheds light on the preclinical phase of MS and provides a promising avenue for early detection and intervention.
“Identifying patients at high risk of developing MS before symptom onset could revolutionize patient care and treatment strategies.”
Research uncovers new therapeutic target for traumatic brain injury
Researchers at Gladstone Institute in the US used state-of-the-art imaging technology to study mouse brains, as well as brains from people who experienced a traumatic brain injury.
They also produced three-dimensional imaging of a whole intact mouse brain, showing blood-brain barrier leaks and abundant fibrin in traumatic brain injury.
In both mouse and human brains, fibrin was present together with activated immune cells.
The team leveraged genetic tools with a specific mutation in fibrin that can block it from activating immune cells without affecting the protein’s beneficial blood-clotting abilities.
“This study identifies a potential new strategy to diminish the devastating impacts of brain injuries,” says Lennart Mucke, MD, director of the Gladstone Institute of Neurological Disease.
“Brain injuries can have profound effects on a person’s cognitive abilities, emotional health, and motor skills, touching every part of their life.
“It will be interesting to explore whether blocking the disease-promoting effects of fibrin can improve the outcome of brain surgeries and reduce disability when implemented after traumatic brain injuries have occurred.”
Signs of multiple sclerosis show up in blood years before symptoms
UC San Francisco scientists have discovered a harbinger in the blood of some people who later went on to develop the disease.
In about 1 in 10 cases of MS, the body begins producing a distinctive set of antibodies against its own proteins years before symptoms emerge.
These autoantibodies appear to bind to both human cells and common pathogens, possibly explaining the immune attacks on the brain and spinal cord that are the hallmark of MS.
The findings are published in Nature Medicine.
MS can lead to a devastating loss of motor control, although new treatments can slow the progress of the disease and, for example, preserve a patient’s ability to walk.
The scientists hope the autoantibodies they have discovered will one day be detected with a simple blood test, giving patients a head start on receiving treatment.
“Over the last few decades, there’s been a move in the field to treat MS earlier and more aggressively with newer, more potent therapies,” said UCSF neurologist Michael Wilson, MD, a senior author of the paper.
“A diagnostic result like this makes such early intervention more likely, giving patients hope for a better life.”
