Relapsing-Remitting Multiple Sclerosis Clinical Trial
Official title:
Mitochondrial Dysfunction and Disease Progression
While the last several years have seen great strides in the treatment of relapsing forms of
MS, progressive MS, responsible for the majority of MS-related disability, lags far behind.
Despite much research, the lack of understanding related to what causes patients' relentless
decline in function results in an inability to develop targeted treatment strategies suitable
for clinical trials. This grant has two main goals.
The first goal is to extend the investigators preliminary study on rat neurons treated with
the CSF of MS patients to a larger number of Progressive patients in order to validate the
initial findings and extend the study to include analysis of human neurons. The initiating PI
(Dr. Casaccia) and the Partnering PI and Clinical Neurologist (Dr. Katz Sand) have recently
identified components that are present in the CSF of progressive patients that impair the
ability of rat neurons to produce energy. The partnering PI, Dr. Quinzii (Columbia
University) together with collaborator Dr. Fossati (NY Stem Cells Foundation), have
characterized human neurons generated from stem cells derived from skin biopsies of
progressive patients and detected the presence of energetic deficits. The experimental plan
will build on these results and test hypotheses of disease progression. The overall goal is
to improve understanding on how to stop neurons from degenerating and stop clinical
progression.
The second goal is to ask whether it is possible to define a progressive disease course on
the basis of combined biochemical, functional and imaging measurements. The initiating PI
will be responsible for the biochemical assessment of CSF and serum samples and, together
with partnering PI Quinzii, will also provide functional bioassays measurements of
mitochondrial bioenergetics impairment in patients. These data will be combined with clinical
assessment and MRI evaluations conducted by the partnering PI Katz Sand and collaborator
Inglese. A two year clinical and imaging follow up from the initial recruitment will allow to
define whether the combined measurements can be used by clinical neurologists to define the
disease course and better identify therapeutic options for patients.
The expectation is that the completion of the stated aims of research will allow an
advancement of the current knowledge of the progressive form of MS and lead to potential new
therapeutic targets.
n/a
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