Coalition Duchenne Funds $25,000 Under a Sponsored Research Agreement With Phrixus Pharmaceuticals For Development Work On Carmeseal™
CheckOrphan Press Release
Newport Beach - April 17, 2012
Coalition Duchenne, a Newport Beach charity that raises funding and awareness for Duchenne muscular dystrophy (DMD), today announced the funding of $25,000 as part of a sponsored research agreement (SRA) with Phrixus Pharmaceuticals, a Michigan based specialty pharmaceutical company. The goal of the SRA is to advance Carmeseal, a treatment for heart failure in DMD
patients and the general population and a potential skeletal muscle enhancer.
The initial funding is part of a larger agreement coordinated through Duchenne Alliance, a group of over 30 non-profit organizations dedicated to Duchenne muscular dystrophy research. Coalition Duchenne is spearheading Carmeseal funding for parent
organizations and acting as the liason between Duchenne Alliance and Phrixus.
Coalition Duchenne has to date coordinated $33,000 of funding under the SRA.
Coalition Duchenne brought Carmeseal to the attention of the Duchenne Alliance and
secured participation by eight other non-profits. Coalition Duchenne hopes to
complete the total grant funding through the Duchenne Alliance before mid-year.
Duchenne muscular dystrophy is the most common lethal childhood genetic disease
affecting 1 in 3,500 males.
Phrixus was founded in 2006 by Thomas Collet and Dr. Bruce Markham to validate the
potential of Carmeseal. They were building on the work of Professor Joseph Metzger
Ph.D., then at the University of Michigan and now Chair of Integrative Biology and
Physiology at the University of Minnesota. Dr. Metzger conducted experiments that
showed that Carmeseal could boost the blood pumping capacity of damaged hearts by binding to the damaged regions of cell membranes.
The funding by Coalition Duchenne and the Duchenne Alliance will demonstrate that
Carmeseal prevents the development of dilated cardiomyopathy in mdx mice, a mouse model of Duchenne, after subcutaneous administration, a novel route of administration for Carmeseal. Phrixus plans to obtain echocardiography data on 185 mdx and control mice currently being studied for respiratory function. Phrixus will also attempt to determine if chronic treatment with Carmeseal improves overall skeletal muscle function in mdx mice.
Carmeseal, generically known as poloxamer 188 (P-188), has been shown to boost the blood pumping capacity of damaged hearts. When Carmeseal, which appears to act as a molecular band-aid, is infused into the bloodstream, it encounters and binds to microscopic tears in the heart muscle. This may prevent the pathological leak of
calcium into the heart cells, which could cause calcium overload and keep the heart
from delivering sufficient oxygenation to the vital organs. Carmeseal, which has been
shown to be effective in four animal models of DMD and heart failure, is expected to
have its effect in patients with DMD irrespective of the genetic defect that causes the disease.
“20,000 boys are born each year with Duchenne, more than 50 each day. Most do not live into their 20s.” said Catherine Jayasuriya. “We need to focus on changing the
course of the disease, we lose many young men to cardiac issues. Developing
Carmeseal is one way we will eventually change that outcome.”
The Duchenne Alliance uses a proprietary collaborative online software called the
Duchenne Dashboard. The Duchenne Dashboard provides scientists and foundations
with a single entry point for proposals to advance Duchenne research. "The Duchenne Dashboard is going to radically improve and expedite the scientific process in the field of muscular dystrophy. The non-profit foundations came together and set the new standard for effective biomedical funding," said Duchenne Dashboard creator Carlo Rago of OpenOnward. International participants in the Carmeseal funding include Action Duchenne, Jett Foundation, JB’s Keys, Suneel’s Light, Hope for Gus, Zack Heger Foundation, Ryan’s Quest, and Michael’s Cause.
Catherine adds, “Duchenne is a difficult road, and presents significant challenges along
the way. Duchenne steals many things, the ability to walk, to hug, to move, to talk, to breathe, but there is that something inside of you, that it can never get to, that it can never take… Hope.”
Catherine founded Coalition Duchenne in 2011 to raise global awareness for Duchenne muscular dystrophy, to fund research and to find a cure for Duchenne. Coalition Duchenne is a 501c3 non-profit corporation. Coalition Duchenne is a call to action; it is about bringing not just Duchenne charities together, but everyone. It calls for humanity to come together as nations, as people, as individuals, to help. As Martin Luther King Jr said, “We are confronted with the fierce urgency of now.”
Duchenne muscular dystrophy is a progressive muscle wasting disease. It is the most
common fatal disease that affects children. Duchenne occurs in 1 in 3,500 male births, across all races, cultures and countries. Duchenne is caused by a defect in the gene that codes for the protein dystrophin. This is a vital protein that helps connect the muscle fiber to the cell membranes. Without dystrophin, the muscle cells become
unstable, are weakened, and lose their functionality. Life expectancy ranges from the
mid teenage years to the mid 20's. Their minds are unaffected.
For more information about Coalition Duchenne, please visit
www.coalitionduchenne.org. Donations can be made on the website at or at
http://www.active.com/donate/duchenneexpedition2.
For more information about Phrixus Pharmaceuticals, Inc., please visit
www.phrixuspharmaceuticals.com. |
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