Edison
Pharmaceuticals, Inc. recently launched clinical trials in both Parkinson’s Disease and ALS using
the same compound, known as EPI-589. The trials are designed to measure any
impact the compound has on the levels of an antioxidant, called glutathione
(GSH), in blood samples collected from enrolled participants. According to the stated aims, this is primarily a
biomarker trial. Measures of disease progression are a secondary outcome
measures.
This is an “open label” clinical trial, meaning all 20 participants accepted will be given the active
compound (which is delivered in pill form) during
the 30 day treatment phase of the trial, followed by a 90-day period in which
participants will be monitored and samples presumably collected to determine duration of
response to the treatment in people diagnosed with ALS. According to published information about the clinical trial, 20 people diagnosed with ALS are expected to be
enrolled at a single site, the California Pacific Medical Center in San
Francisco.
What
is EPI-589
To
date, there have not been published data regarding the mechanism of action of
EPI-589, sometimes also referred to as (R)-troloxamide quinone. Similarly, no
published preclinical studies of EPI-589 in models of ALS were found at the
time of publication of this statement
on this clinical trial. However,
according to the company’s website, public statements and other information
available, Edison
Pharmaceuticals believes that GSH levels will be modified in those treated with
EPI-589. According to a company spokesperson, this
compound acts “catalytically,” meaning small doses may be all that is required
to impact the presence of GSH in cells. GSH is found in nearly all
cells and plays an important role, together with other antioxidants, in maintaining
the effective handling of reactive oxygen species such as free radicals.
In ALS, the pathway proposed to be targeted by EPI-589 may sometimes be
referred to as oxidative stress. Many different response elements are required
for the health of cells. Many of these change over time in ALS. Glutathione is
one among hundreds of response elements. This study aims to measure GSH
to determine whether or not its levels are changed by treatment with EPI-589.
Bottom
line
This clinical trial is not
open for enrollment today (6/9/2015), however, Edison states it hopes to open the
trial at the single
site as soon as the end of July 2015. It is
not known whether EPI-589 is an
effective treatment for ALS, and will not be known until this and subsequent
larger trials are completed and data analyzed. Similarly, it is important to
note that GSH should not be misinterpreted at this time as a biomarker of
disease progression, rather a proposed biomarker of drug response. This trial
aims to test that hypothesis. The ALS Therapy Development Institute will
continue to monitor this clinical trial and add any new information learned to our global ALS clinical trial database.