RESTEM and JAR of Hope Foundation Announce Strategic Partnership and Clinical Trial Launch of Next-Generation Cell Therapy for Patients with Duchenne Muscular Dystrophy

RESTEM Developing First-in-Class, Umbilical Cord Lining Stem Cell Therapies. JAR of Hope Providing Critical Philanthropic Financial Support for Clinical Trial.


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.

About Duchenne Muscular Dystrophy 
DMD is a rapidly progressive form of muscular dystrophy that occurs primarily in boys. It is caused by an alteration (mutation) in a gene, called the DMD gene that can be inherited in families in an X-linked recessive fashion, but it often occurs in people from families without a known history of the condition. Individuals who have DMD have progressive loss of muscle function and weakness, which begins in the lower limbs. Children with Duchenne Muscular Dystrophy do not produce the muscle protein dystrophin, which is encoded by the DMD gene and is crucial for muscle structure and function. Duchenne muscular dystrophy affects approximately 1 in 3500 male births worldwide. Because this is an inherited disorder, risks include a family history of Duchenne Muscular Dystrophy.

RESTEM is a pioneering, innovation-driven, clinical-stage, biotechnology firm based in Corona, California, dedicated to the discovery, development and commercialization of novel, next-generation, cell-based therapeutics and tools that aid in the treatment of a broad range of degenerative disorders. The company’s mission is focused on improving the quality of life for those who suffer from disabling diseases of the immune system, age-associated disorders, and other degenerative diseases. The company is based on over 14 years of research and development focused on the company’s novel and proprietary umbilical cord lining stem cell therapy, or ULSCs, which have demonstrated promising regenerative properties in preclinical and human clinical trials. With an established strong leadership position, we continue to seek to be a major contributor to next-generation, cell-based therapies with ongoing clinical trials that address significant unmet medical need across a range of diseases and disorders. For additional information, please visit,

About JAR of Hope 
JAR of Hope was born on September 13th, 2013, when the founder, Jim Raffone’s precious son, James (“Jamesy”) Anthony Raffone, was diagnosed with Duchenne Muscular Dystrophy (DMD). JAR of Hope’s mission is to find a cure for DMD. 
Jamesy was 4 years old when he was diagnosed. Today he is 13. Each day without a cure his muscles continue to waste away as will all children who suffer from this horrific disease. Statistically, by the age of 12 they will be in a wheelchair, by 15 they will be a quadriplegic, by 18 they will not be able to breathe on their own and eventually will live on a ventilator until their bodies are no longer strong enough to fight this disease. Many don’t survive past their mid-20s. The only way this disease will continue to cheat these children of a future is if we stand by and do nothing to find a cure. 
DMD is a genetic disorder characterized by progressive muscle degeneration. It primarily affects boys, although there are a few rare cases of girls who have DMD. Duchenne affects 300,000 boys worldwide; 20,000 children in the United States alone. These children are robbed of their childhood as they can’t run and play. 
In over 200 years, a cure for Duchenne has not yet been found. Not one child has survived this disease, and as you can probably imagine, it takes millions of dollars, massive amounts of time and people committed to finding a cure. JAR of Hope has and continues to dedicate each and every day to finding a cure and living in a world where Duchenne no longer exists. With the help of donors and sponsors, JAR of Hope is funding a team of research scientists and doctors determined to eliminate Duchenne. We also provide financial and emotional support for children and their families who are and have been affected by DMD. We have purchased wheelchair accessible vans, installed chair lifts on stairs in homes for these children to be able to move from one floor to another, funded studies, assisted with medical bills and, unfortunately, have paid for funeral expenses for children that have lost their fight to Duchenne MD. 
JAR of Hope’s founder, Jim Raffone, has been relentless in his pursuit to defeat Duchenne. Some of his efforts in educating others, raising awareness and funds include breathtaking world-record events such as climbing Mt. Everest, creating race teams to participate in marathons and ultra marathons around the world, gathering thousands of people to simultaneously do pushups together, and creating the longest Lego chain at a school in New Jersey, to name a few. You can see the many events that have taken place on JAR of Hope’s website. Jim has received Keys to Cities from various Governors and Mayors as he has traversed around the globe determined to make Duchenne a household name and push science forward. Jamesy is the inspiration behind JAR of Hope and Jim is committed to all of the children with DMD around the world. Jamesy’s courage, strength, positive attitude and willingness to do whatever it takes to get healthier and stronger every day motivates Jim to carry on until the first child is cured. Jim and the JAR of Hope team believes that this disease has gone on for too long. Jim’s motto is: “I can, I will & I must” find a cure; however, he knows that he cannot do it alone. Together, we can make history and save lives. To learn more, please see

For more information, please contact: 
Rafael Gonzalez, PhD 
Founder and SVP R&D 

James Raffone 
Founder & CEO 
JAR of Hope