CADASIL Eligibility in the NEST Neurologic Stem Cell Treatment Study

Westport, CT, October 30, 2019 --(PR.com)-- The most common inherited stroke syndrome, CADASIL, was only discovered in the early 1990’s and can start with mild mood disorders and migraines before developing more serious neurologic issues. With a long and descriptive name, “Cerebral Autosomal Dominant Arteriopathy with Subcortical Infarcts and Leukoencephalopathy” is due to a a single gene mutation typically inherited from an affected parent and symptoms often start in early middle age. The gene is called NOTCH3 on Chromosome 19 and its alteration causes deposits in the small blood vessels in the body. In the brain this can lead to mini-strokes and to mini-strokes and to serious neurologic symptoms including cognitive or memory impairments.

The Neurologic Stem Cell Treatment Study or NEST is a National Institutes of Health (NIH) registered clinical study (NCT 02795052) that uses the patient’s own bone marrow stem cells (BMSC) in different neurologic diseases. These include Stroke (CVA), Traumatic Brain Injury, Multiple Sclerosis, Parkinsons, ALS, PSP, MSA, CADASIL, Peripheral Neuropathy and others. The study is under an Institutional Review Board and compliant with FDA regulations regarding use of stem cells. Improvements have been seen across a number of neurologic diseases. Of special interest to CADASIL patients is how the BMSC may specifically help in their condition.

In key research using an animal model of CADASIL, scientists have shown that the NOTCH3 mutation can reduce an important chemical for blood vessels called Vascular Endothelial Growth Factor (VEGF). This can cause loss of brain vessels, neurons and synapses, leading to dementia and other neurologic problems. However they could counter this problem by using stem cell factors which increased the amount of VEGF /VEGF A which in turn enhanced repair, restored vasculature and improved brain function. Other researchers have shown that BMSC will release VEGF in response to various factors and help heart cells that have lack of oxygen. Finally, it has been shown that Mesenchymal Stem Cells (MSC), which are found in BMSC, stick to early endothelial cells (EPC) and release VEGF which helps them grow.

In summary, it appears that BMSC can release a factor called VEGF which helps the cells lining blood vessels to grow and which is reduced in CADISIL. Therefore, BMSC as provided in the NEST study may potentially help regrow lost brain vessels and neuron structures, helping to improve brain function.

Patients interested in the actual articles may contact Dr. Levy and request links or further information on applying to the NEST study. As with any clinical study, it is not possible to predict whether an individual patient will improve from treatment or continue to have progression of their disease. The purpose of the NEST study is to potentially benefit patients as well as to gather data to determine whether statistical significance is present for any improvements.

Patients may receive information about the study by emailing the Study Director, Steven Levy MD, at stevenlevy@mdstemcells.com; using the "contact us" page on the website www.mdstemcells.com ; calling 954-417-5533 and leave a voice message with their telephone number; alternative number 203-423-9494 (no messaging). MD Stem Cells has no grant support for NEST and is not a pharmaceutical company; this is a patient sponsored study and the patients pay for both treatment and travel.