CureDuchenne Announces Webinar Featuring New Approach To Bone Regeneration in DMD

A webinar hosted by CureDuchenne featuring an informative program by Mesentech is scheduled for 4:30pm EST on Thursday, December 10th. The community is invited to join in and learn directly from Mesentech about its novel therapeutic approach to bone regeneration.

The webinar is entitled Mesentech and The Importance of Bone Health giving attendees a view of Mesentech’s novel therapeutic approach towards improving bone density and strength.

While standards of care call for monitoring bone density in Duchenne and also intervening with osteoporosis drugs at the first sign of fractures, there is still much unmet medical need to better combat bone fragility in this disease. That is why CureDuchenne is so pleased to help support the efforts of Mesentech, Inc in developing a novel drug with the goal of bone regeneration.

About Duchenne Muscular Dystrophy
Duchenne is a severe X-linked form of muscular dystrophy that affects approximately 1 in 5000 males. DMD is caused by the absence of the dystrophin protein.  Dystrophin is a large protein that provides multiple cellular functions and helps to protect skeletal and cardiac muscle against injury, inflammation and fibrosis. Individuals with Duchenne show progressive muscular degeneration and lose the ability to walk by early to mid-teens and progress to full loss of upper body function.  Cardiopulmonary complications are the primary cause of death.

How is bone compromised in Duchenne muscular dystrophy?

Individuals with Duchenne are at risk for osteoporosis, or thinning of the bones, for three main reasons:

  1. Reduced weight-bearing activity (which normally drives the development of strong and dense bones)
  2. Side effects of glucocorticoid therapies
  3. The effects of cytokines released due to the chronic inflammatory response in dystrophin-deficient muscles

How does low bone density impact individuals with Duchenne?

When bones have reduced density, they become brittle and prone to fracture.  Studies have indicated that in Duchenne, at least half of individuals will sustain bone fractures due to low bone density.

Dr. Michael Kelly, Chief Scientific Officer at CureDuchenne and a member of Mesentech’s scientific advisory board offered the following information:

MES-1007 is a bi-specific drug-conjugate consisting of two parts:

  1. A targeting moiety that uses a novel proprietary molecule that delivers the conjugate to bone, and
  2. A drug moiety that delivers a PGE2 prostaglandin analogue to activate the EP4 receptor specifically on bone when released from the conjugate.

MES-1007 is a prodrug. Typical prodrugs are designed to improve specific properties of drugs such as bioavailability, pharmacokinetics or half-life. The bi-specific drug conjugate combines this property with improved targeting to the tissue of interest.

This novel conjugate [MES-1007] has no biological activity on its own but is designed to only release its active drug component at the target site (bone). This specifically reduces the multiple side-effects associated with the activity of the drug in other (non-target) tissues, and greatly improves its efficacy while reducing toxicity and mechanism-related side-effects.

A Newly-Formed Partnership

CureDuchenne recently partnered with Mesentech to find out if Mesentech’s targeted therapy for reversion bone wastage will be useful in Duchenne muscular dystrophy.

CureDuchenne’s investment supports Mesentech’s lead program, MES-1007, into clinical development and its evaluation in Duchenne muscular dystrophy.  Pediatric osteoporosis is a significant problem for DMD patients, leading to frequent fractures and premature loss of ambulation.  There are currently no approved therapies for bone wastage for individuals affected by Duchenne. There is tremendous potential for MES-1007 to be the first targeted therapy to specifically address bone wastage in these individuals.

About CureDuchenne Ventures

CureDuchenne Ventures, the research funding arm of CureDuchenne, a leading global nonprofit dedicated to finding and funding a cure for Duchenne muscular dystrophy (DMD), announced an investment in Mesentech Inc., a regenerative medicine company with a prodrug platform that selectively delivers therapeutics to bone.  The investment is part of a new joint funding collaboration with the Charles H. Hood Foundation (CHF) that looks to advance early stage research for pediatric conditions.

About CureDuchenne
CureDuchenne is recognized as a global leader in research, patient care and innovation for improving and extending the lives of those with Duchenne muscular dystrophy. As the leading genetic killer of young boys, Duchenne affects more than 300,000 individuals living today. CureDuchenne is dedicated to finding and funding a cure for Duchenne by breaking the traditional charitable mold through an innovative venture philanthropy model that funds groundbreaking research, early diagnosis, and community education. For more information on how to help raise awareness and funds needed for research, please visit cureduchenne.org.

About the Charles H. Hood Foundation
Since 1942, the Charles H. Hood Foundation has carried on the legacy of founder Charles H. Hood by funding groundbreaking and innovative pediatric research. After decades of successful grant programs, the Foundation broke new ground in 2015 by establishing its program-related investment fund to support entrepreneurial efforts to significantly improve health outcomes of children. Through both its grants and investments, the Foundation has made meaningful change possible by filling the gaps in the medical research and innovation funding marketplace. To learn more about the Hood Foundation please visit charleshoodfoundation.org.

Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia four years ago. He was treated with a methylating agent While he was being treated with a hypomethylating agent, Rose researched investigational drugs being developed to treat relapsed/refractory AML.

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