RESTEM, a pioneering, next-generation, clinical-stage cell therapy company focused on harnessing the unique power of cell therapy and JAR of Hope, a 501(3)(C) non-profit foundation dedicated to ongoing research efforts to find a cure for Duchenne Muscular Dystrophy (DMD), today announced a strategic collaboration and launch of a landmark clinical trial to evaluate RESTEM’s novel, umbilical cord lining stem cell (ULSC) therapy for the treatment of Duchenne Muscular Dystrophy.

The Phase I trial will include 12 patients who will receive intravenous infusions of ULSCs to treat DMD, which is a severe type of muscular dystrophy that primarily affects boys. Muscle weakness usually begins around the age of four, with rapid symptom progression. Most young boys are wheelchair-bound by the age of twelve.

The trial, which has received authorization to proceed from the U.S. Food and Drug Administration (FDA), seeks to provide early clinical evidence of the potential of this novel cell therapy to improve the health of DMD patients and is the first clinical trial focused on evaluating ULSCs in DMD. If the Phase I trial is successful, a Phase II trial is expected to follow, which will assess effectiveness of ULSC therapy in a larger number of DMD patients.

“As a pioneering next-generation, clinical-stage cell therapy company committed to advancing game-changing cell-based therapeutics, we are honored to have the support of JAR of Hope to provide the critically important financial support to bring this novel therapeutic approach to children with Duchenne Muscular Dystrophy”, said David Pyrce, CEO of RESTEM.

“DMD is an extremely serious disease that over time can affect all voluntary muscles, including the heart and breathing muscles in later stages of the disease resulting in significantly shortened life expectancy. With few effective therapies available, there is a significant need for better treatment options and this trial, using RESTEM’s novel, umbilical cord lining stem cell therapy, offers the potential to improve or slow disease progression with hopes of giving these children a more carefree childhood and an improved quality of life,” said James Raffone, Founder and CEO of JAR of Hope.

Mr. Raffone continued, “JAR of Hope was born because no child should have to suffer or die from DMD and no family should have to suffer from the devastating consequences of this disease. Our mission is to better educate, raise awareness and provide funding for innovative new treatment options that potentially will lead to a cure for DMD, giving children to get the chance to become healthy adults. Our goal is to one day see the words Duchenne muscular dystrophy only in history book, just like polio. We are pleased to have the opportunity to provide the financial support for this clinical trial with transformative potential using a novel cell therapy approach as the first step down the path to a cure for DMD. We encourage others to join with us in our relentless pursuit to find a cure for DMD.”

“We are thrilled to have the opportunity to bring this potentially transformative therapy, based on over a decade of research, to Duchenne Muscular Dystrophy patients”, said Rafael Gonzalez, Ph.D., Senior Vice President of Research and Development at RESTEM. “We are honored to have the generous financial support from JAR of Hope to bring this potentially game-changing therapy to DMD patients as quickly as possible.”

Dr.Gonzalez continued, “Our mission is to fast-track the development of transformative new therapies for serious, life-threatening diseases, with a laser-focus on fast-tracking the launch of this study evaluating the potential effectiveness of our novel and proprietary umbilical cord lining stem cell therapy in patients with DMD.”

Clinical Trial Overview RESTEM has received FDA authorization for its Investigational New Drug Application (IND) entitled “A Phase I, randomized, double-blind, placebo-controlled cross over study to assess the safety, tolerability, and preliminary efficacy of Umbilical Cord Lining stem cells (ULSC) in pediatric patients with Duchenne Muscular Dystrophy (DMD)”. The trial is planned to enroll 12 patients. In previous clinical trials in other indications, RESTEM’s ULSCs have demonstrated an excellent safety record with promising clinical results achieved across a range of indications. If encouraging clinical results are achieved in this Phase I study, a Phase II trial is planned, with the goal of bringing this potentially transformative new therapeutic option to patients with DMD as quickly as possible.

[Source(s): RESTEM, PRWeb]