Drug Developed for Inherited Bleeding Disorder Shows Promising Trial Results
A study led by Mass General Brigham researchers found that engasertib lessened bleeding events for patients with HHT, the second most common inherited bleeding disorder.
Press Release5 Minute ReadDec | 3 | 2019
Susanne van Veluw, PhDIf we direct therapeutic strategies towards promoting healthy vasculature and therefore improve clearance of amyloid-beta from the brain, we may be able to prevent or delay the onset of Alzheimer’s disease in the future.”
BOSTON – In patients with Alzheimer’s disease, amyloid-beta protein fragments accumulate in the tissue and blood vessels of the brain, likely due to a faulty clearance mechanism. In experiments conducted in mice, investigators at Massachusetts General Hospital (MGH) have found that very slow spontaneous vessel pulsations – also known as ‘vasomotion’ – drive the clearance of substances from the brain, indicating that targeting and improving this process may help to prevent or treat amyloid-beta accumulation.
In their study published in Neuron, the researchers injected a fluorescently labeled carbohydrate called dextran into the brains of awake mice, and they conducted imaging tests to follow its clearance. Their experiments revealed that vasomotion was critical for clearing dextran from the brain and stimulating an increase of the amplitude of these vessel pulsations could increase clearance. Also, in mice with cerebral amyloid angiopathy, a condition that causes amyloid-beta to build up in the walls of the brain’s blood vessels, vessel pulsations were hindered and clearance rates were reduced.
“We were able to show for the first time that large dilations and contractions of vessels that happen spontaneously at an ultra-low frequency are a major driving force to clear waste products from the brain,” said lead author Susanne van Veluw, PhD, an investigator in the department of Neurology at MGH. “Our findings highlight the importance of the vasculature in the pathophysiology of Alzheimer’s disease. If we direct therapeutic strategies towards promoting healthy vasculature and therefore improve clearance of amyloid-beta from the brain, we may be able to prevent or delay the onset of Alzheimer’s disease in the future.”
About the Massachusetts General Hospital
Massachusetts General Hospital, founded in 1811, is the original and largest teaching hospital of Harvard Medical School. The MGH Research Institute conducts the largest hospital-based research program in the nation, with an annual research budget of more than $1 billion and comprises more than 8,500 researchers working across more than 30 institutes, centers and departments. In August 2019 the MGH was once again named #2 in the nation by U.S. News & World Report in its list of "America’s Best Hospitals."
A study led by Mass General Brigham researchers found that engasertib lessened bleeding events for patients with HHT, the second most common inherited bleeding disorder.
The Sean M. Healey & AMG Center for ALS at Mass General Brigham has selected Tiziana Life Sciences as one of the companies to enter the Healey ALS MyMatch Program. Learn more.
Mass General Brigham study of more than 11,000 adolescents and young adults found those who took buprenorphine for 12 months had almost half the risk of overdose compared to those who discontinued early.
In a study of nearly 30,000 female nurses under age 50, Mass General Brigham researchers found that a high intake of ultra-processed foods was associated with increased risk of adenomas, colon polyps which can be precursors of colorectal cancer.
The Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital has contracted with Inflammasome Therapeutics to design a new Healey ALS MyMatch trial. Learn more.
Rakesh Jain, PhD, of the Mass General Brigham Cancer Institute, is the senior author of a paper published in The Proceedings of the National Academy of Sciences (PNAS), “Physiologically-based pharmacokinetic model for CAR-T cells delivery and efficacy in solid tumors.”