A New Approach for Treating CADASIL

Background:

CADASIL is a genetic disease that can lead to stroke and dementia.  Caused by a gene mutation in nearly four out of every thousand people, this condition mainly affects young and middle-aged adults. It damages small blood vessels in the brain,  leading to a reduction in neuron structures, synapses and neurogenesis that can result in migraines, epilepsy, multiple strokes, changes in mood and personality, dementia, and early death. Though scientists have identified the gene that causes CADASIL, little is known about how it functions, and no treatment is currently available for the condition.

Technology Overview:

Inventors at SUNY Upstate Medical University developed a new therapeutic approach that can stop the progress and even reverse the effects of CADASIL. A combination treatment comprising the biological molecules stem cell factor (SCF) and granulocyte colony-stimulating factor (G-CSF) improves cognitive function in CADASIL by increasing brain blood vessel density and promoting neural network rewiring.

Advantages:

Novel therapeutic approach for delaying CADASIL progression.

Improves spatial learning and memory.

Increases formation of new blood vessels.

Promotes growth of new neurons.

G-CSF is an FDA approved biological product (Filgrastim).

Applications:

CADASIL and other cerebral small vascular diseases.