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Duchenne Muscular Dystrophy Therapy Receives Rare Pediatric Disease Designation

Duchenne Muscular Dystrophy Therapy Receives Rare Pediatric Disease Designation

Duchenne Muscular Dystrophy is characterized by progressive muscle degeneration paired with weakness.  This is caused by the lack of dystrophin.  This protein is the key to muscles staying strong and uninjured.  This disorder is typically found when a person is very young, as early as three years old.  It mainly affects boys but it can be seen in girls as well. 

This disorder is one that will eventually kill the patient.  The treatments that are available are not enough to help the patient overcome the disorder.  Instead, they are only able to help them prolong their life.  Some treatments are able to help them have a better quality of life and help to retain what healthy muscles they have.

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With the lack of the dystrophin protein, the child grows up with muscles that are unable to stay fully functional.  Their muscles continually break down until they have chronic muscle damage.  Many patients die as early as their twenties.  They typically see heart disease is a key factor in their death.  It is important for researchers to find a way to help these muscles develop and avoid being damaged.

Capricor Therapeutics

Capricor Therapeutics is a biotechnology company that is in clinical stages.  They are trying to create therapies for disorders like DMD.  They are working on the CAP-1003 which is a cell-free treatment.  They are also looking at the CAP-1002 as a treatment. CAP-1002 uses cardiosphere-dervided cells.  These cells are a form of a base cell that helps boost activity with in the immune system.  The CDCs have been part of many publications from scientists.  There have been over one hundred and forty human subjects that have helped in clinical trials pertaining to CDCs.    

When tested in human subjects scientists found that patients have profound advancements statistically in how well their upper limbs functioned.  After being used for six months the cell treatment was found to be tolerated by the patients well. The cells with in CAP-1002 may be able to exert vesicles that influence the immune system.  They may help boost anti-inflammatory responses in the body.  It could also help to reduce the scarring found on connective tissues on the muscles as they are injured.  The vesicles could also help to reduce the amount of cell death.

Improvements in Patients

With all of these byproducts of the CAP-1002 patients have found improvements in their muscle structures.  These muscles were able to function better compared to ones not treated in the same manner.  This treatment could be beneficial as a simple treatment or it could be paired with others for better muscle retention. Capricor Therapeutics was striving to get a Rare Pediatric Disease Designation for CAP-1002.  This designation is set by the FDA and must follow certain guidelines.  The FDA considers a rare pediatric disease one that is serious or life-threatening to the person that is anywhere from birth to age eighteen.  It must affect less than 200,000 people in the US.

With a designation, there can be an FDA Rare Pediatric Disease Priority Review Voucher available.  This voucher will help anyone that has a voucher obtain priority review over other sponsors.  This priority is for their new drug or their biologics license application to be reviewed in a more timely manner.  Typically within six months from application versus the typical ten months.

Rare Pediatric Disease Designation

As of July 18, 2017, the Food and Drug Administration gave CAP-1002 the Rare Pediatric Disease Designation.  With this designation, Capricor is able to continue to research CAP-1002 for a general treatment of Duchenne Muscular Dystrophy.  They will also be in line to get a Priority Review Voucher. This is not the only designation that the CAP-1002 has received.  It has also had the Orphan Drug Designation.  With both of these designations, Capricor will be able to receive the benefits and extra motivation to continue the research with this cell based therapy.  As they move forward they are hoping to conduct another human based trial for the treatment. 

Currently there is an ongoing HOPE-Duchenne study.  This study is called Halt cardiomyOPathy progrEssion in Duchenne.  Up to thirty subject may be selected for this study where they will be randomized into two groups.  There will be subjects who will receive the active treatment.

The active treatment will be a dose of seventy-five million CAP-1002 cells that are combined with twenty-five million cells into three ventricle cardiac locations.  There will also be subjects that will randomly be selected to have the usual care.  They will continue the typical care and will not receive the CAP-1002 cells. 

Goal of Study

The main goal of this study is to learn about how CAP-1002 cells are tolerated in a human subject.  They would also like to examine the cells in a safety perspective to make sure that they are able to interact with the body without doing damage or altering other structures in a negative manner.  With this information they will be able to develop the CAP-1002 treatment concept even further to better the treatment.

As the observe the changes that the cells bring they will be looking at changes in the body like major cardiac events, coronary blood flow, laboratory assessments, physical examinations, vital signs, ECG, and any major adverse events as well. The point of this study is not to push this treatment forward into production but to ensure the quality of the treatment for patients.  If there are aspects that do not work well with the human subjects the treatment will need to be reevaluated and retested.

There are four separate secondary goals for the study.  The first goal concentrates on studying the cardiac structures and how they have changed due to the treatment.  It will be studied through a cardiac MRI twelve months after the infusion.

The second goal is about the functionality of the treatment.  They are looking to see how much change in mobility patients have.  They are using a few different tests to determine this change.  The Performance of Upper Limb scale, spirometry, and a six minute walk test.  These tests will be performed twelve months after infusion.

The third goal is for quality of life.  This is assessed using a Pediatric Quality of Life Inventory.  It will be used to determine if the treatment is helping or hindering the human subjects. The last goal is to assess biomarkers.  These assessments will be conducted twelve months after the infusion.


With the advancements that Capricor has had with the FDA, it is clear that there is a need for treatments for Duchenne Muscular Dystrophy.  While this disorder is rare its affects are devastating to the patients.  They typically start having symptoms of their disorder early in childhood and will spend the rest of their lives living with the effects.

The current treatments are not able to help these patients live their lives to the fullest.  Many patients are dying in their twenties due to complications from the disorder.  Many of these complications stem from their heart becoming to weak to support them. 

A treatment that can help strengthen and repair their muscles is needed.  This can help them live longer lives.  It will also help them have a better quality of life.  If they are not hindered by their disorder deteriorating their muscles they will be able to participate in more activities and events.  As treatments are created for DMD the patient's lives with continue to improve.