CurePSP has just announced their latest Venture Grant recipients. These grants total $320,000 and will all support the research of neurodegenerative disease treatments.
CurePSP gives these grants twice every year. All applications are reviewed by an independent scientific advisory board.
If you are interested in applying or the next grant cycle, applications are due on December 17th, 2021.
CurePSP is a nonprofit organization based out of New York and founded in 1990. The organization focuses on a few specific rare neurodegenerative diseases including progressive supranuclear palsy (PSP), multiple system atrophy (MSA), and corticobasal degeneration (CBD).
All of these conditions are known as “prime of life” neurodegeneration. This is because they tend to begin during middle age. Symptoms of these conditions include speech difficulties, swallowing difficulties, cognitive impairment, impaired motor control, as well as behavioral disinhibition. Each of the conditions is caused by a toxic accumulation of tau, a protein that is naturally present. When this protein is overproduced in the brain, it destroys neurons.
Unfortunately, there has yet to be an approved treatment, let alone a cure, for any of these conditions. CurePSP is helping to change that by funding research specifically for conditions like these. To date, they have funded around 200 studies.
Additionally, CurePSP provides support, education, and information to patients, their families, researchers, doctors, healthcare workers, caregivers and more.
Rachel Bailey from UT Southwestern Medical Center located in Dallas, Texas, was awarded one of the grants for her work in gene therapy. She is investigating a gene replacement therapy that would be able to fight the accumulation of tau within the brain.
She plans to utilize a viral vector to deliver two forms of RNA. The first will prevent the abnormal tau protein from being manufactured. The second will create an aggregation-resistant tau protein.
Daniel Geschwind from UCLA will be working on investigating four new oral drugs in mice models. The mice models will all produce the abnormal tau with a bit of help from genetic engineering.
The therapies enhance an enzyme which can cut up the abnormal tau. The enzyme can also enhance the autophagy system in the brain cells. This system disposes abnormal proteins that are present, including the tau protein.
The enzyme is called puromycin-sensitive aminopeptidase.
Maria Lima da Silva from Harvard will be investigating molecules which activate autophagy through an enzyme ULK1. When activated, the toxic accumulation of the tau protein may be cleared. While similar to Dr. Geschwind’s study, this investigation will not use mice. Instead, Maria is using stem cells from human biopsies.
Marta Olah from Columbia is using the grant to study microglia. Microglia are the immune cells within the brain which are well known to be involved in neurodegeneration.
Marta will be using a novel method of RNA sequencing to investigate individual immune cells. She will then be able to map which cells are encoding proteins that are close to the degenerating neurons.
The hope is that this map will help researchers to develop new drug targets and more knowledge surrounding the disease.
You can read more about these new grant winners here.