[tabi] possible treatment for RP

  • From: "Allison and Chip Orange" <acorange@xxxxxxxxxxx>
  • To: <tabi@xxxxxxxxxxxxx>
  • Date: Sat, 24 Jul 2010 18:05:10 -0400

"Valproic acid shown to halt vision loss in patients with retinitis
Contact: Jim Fessenden
University of Massachusetts Medical School
Valproic acid shown to halt vision loss in patients with retinitis
UMass Medical School coordinating a $2.1 million three-year clinical trial
WORCESTER, MASS. - Researchers at the University of Massachusetts Medical
(UMMS) believe they may have found a new treatment for retinitis pigmentosa
(RP), a severe neurodegenerative disease of the retina that ultimately
results in
blindness. One of the more common retinal degenerative diseases, RP is
caused by the death of photoreceptor cells and affects 1 in 4,000 people in
the United
States. RP typically manifests in young adulthood as night blindness
or a loss of peripheral vision and in many cases progresses to legal
blindness by
age 40.
In the July 20 online edition of the British Journal of Ophthalmology,
Shalesh Kaushal,
MD, PhD, chair of ophthalmology and associate professor of ophthalmology
and cell biology at UMMS, and his team, describe a potential new therapeutic
between valproic acid and RP, which could have tremendous benefits for
patients suffering from the disease. In a retrospective study, valproic
by the FDA to reduce seizures, treat migraines and manage bipolar
disorder-appeared to have an effect in halting vision loss in patients with
RP and
in many cases resulted in an improved field of vision. Results from
this study, in conjunction with prior in vitro data, suggest valproic acid
may be
an effective treatment for photoreceptor loss associated with RP.
UMass Medical School will be the coordinating site for a $2.1 million,
clinical trial funded by the Foundation Fighting Blindness/National
Research Institute quantifying the potential of valproic acid as a treatment
RP. The clinical trials will build upon Kaushal's work in the retrospective
study in which patients were treated off-label with doses of valproic acid
from 500mg to 750mg per day over the course of two to six months. Treated
at a time when patients normally experience rapid vision loss as a result of
five of the seven patients in the study experienced improvement in their
field of vision.
"Inflammation and cell death are key components of RP," said Kaushal. "It
the valproic acid protects photoreceptor cells from this. If our
can be further substantiated by randomized clinical trials then low dose
acid could have tremendous potential to help the thousands of people
suffering from RP."
To date, discovery of a treatment for RP has been complicated by the fact
that more
than 40 different genes have been linked to the disease, making many
interventions impractical or impossible; as a result, the disease remains
untreated for an estimated 100,000 patients in the U.S. Most RP therapies
currently being investigated focus on nutritional supplementation, vitamin A
light reduction or gene therapy.
Dr. Kaushal and colleagues, having previously demonstrated the use of the
small molecule,
retinoid, as a pharmacological agent capable of increasing the
yield of properly folded RP rhodopsins, began screening other small
molecules for
similar attributes. Because of its already known qualities as a potent
inhibitor of the inflammatory response pathway and cell death, valproic acid
believed to have a unique profile making it a potential candidate as a
retinal disease treatment.
"Traditionally, moving a new scientific discovery from the bench to the
patient requires
a significant investment of time and resources," said Kaushal.
"Repurposing drugs already approved by the FDA and which have been shown to
be safe,
such as valproic acid, is an economical and time-efficient way to
quickly bring new treatments to patients."
"The Foundation Fighting Blindness is delighted to be moving Dr. Kaushal's
work with valproic acid into our clinical trial network, because
the drug has the potential to preserve vision for thousands of people
affected by
retinal diseases," said Steve Bramer, Ph.D., chief drug development officer,
National Neurovision Research Institute, a clinical support arm of the
Fighting Blindness. "It's an exciting research collaboration for us,
because of the drug's potential, and the knowledge and expertise Dr. Kaushal
the University of Massachusetts Medical School bring to the clinical study."
About the University of Massachusetts Medical School
The University of Massachusetts Medical School, one of the fastest growing
health centers in the country, has built a reputation as a world-class
research institution, consistently producing noteworthy advances in clinical
basic research. The Medical School attracts more than $240 million in
research funding annually, 80 percent of which comes from federal funding
The mission of the Medical School is to advance the health and well-being
of the people of the commonwealth and the world through pioneering
education, research,
public service and health care delivery with its clinical partner,
UMass Memorial Health Care. For more information, visit
 IN Partners In Policymaking:
Here is a second abstract that was received about this.
Br J Ophthalmol doi:10.1136/bjo.2009.175356
Clinical science
Therapeutic potential of valproic acid for retinitis pigmentosa
Correspondence to Dr Shalesh Kaushal, Department of Ophthalmology,
University of
Massachusetts Medical School, 381 Plantation St., Biotech 5, Suite 250,
Worcester, MA 01605;
Contributors C M Clemson and R Tzekov contributed equally to this work.
Accepted 6 March 2010
Published Online First 20 July 2010
Background/aim To examine the efficacy and safety of valproic acid (VPA) in
with retinitis pigmentosa (RP).
Methods Thirteen eyes were examined before and after brief treatment
(average 4 months)
with VPA. Visual fields (VF) for each eye were defined using digitised
Goldmann Kinetic Perimetry tracings. VF areas were log-transformed and VF
relative to baseline was calculated. Visual acuity was measured using
a Snellen chart at a distance of 20 feet (6.1 m). Values were converted to
the logarithm
of the minimum angle of resolution (logMAR) score.
Results Nine eyes had improved VF with treatment, two eyes had decreased VF
and two
eyes experienced no change, with an overall average increase of 11%.
Assuming typical loss in VF area without treatment, this increase in VF was
significant (p<0.02). An average decrease (0.172) in the logMAR
scores was seen in these 13 eyes, which translates to a positive change in
score of approximately 20/47 to 20/32, which was significant (p<0.02)
assuming no loss in acuity without treatment. Side effects were mild and
well tolerated.
Conclusion Treatment with VPA is suggestive of a therapeutic benefit to
with RP. A placebo-controlled clinical trial will be necessary to assess
the efficacy and safety of VPA for RP rigorously.

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