We're all Joining Jacks fight against Duchenne Musculare Dystrophy, are you?

Creating new muscle by using stem cells

Joining Jack is delighted to announce that we have invested £140,000 as part of Duchenne UK's DMD INSPIRE Major Grant of $945,654 (approx. £780,000) to support the University of Minnesota’s pioneering work on regenerating muscle in DMD patients with stem cells

Stem cells are a potentially exciting approach to generate new healthy muscle in patients with Duchenne Muscular Dystrophy (DMD).

The stem cells we are looking at are called human induced pluripotent stem cells (iPSCs). These cells replace the diseased muscle tissue with healthy muscle and with stem cells that can create healthy myofibers (mature muscle cells) which are able to regenerate.

The aim of this project is to produce and test these stem cells to get the preclinical data required to progress to a clinical trial. The team will use our grant to manufacture the stem cells and test a number of things including what dosing should be used and the safety and toxicity.

If the project is successful, the group will commence planning for a Phase I Clinical Trial to start testing this approach on patients.

The team at the University of Minnesota are world leaders in cell therapy, so we are very excited to be funding their work. This group of expert scientists and clinicians have been working hard for the last 3 years to take this potential treatment from early stage research to the exciting reality of manufacturing these cells.

Dr. Rita Perlingeiro, Professor at the Department of Medicine at the University of Minnesota, said:

“I am delighted to receive funding from Duchenne UK as we enter the important preclinical phase of our project. This partnership is critical to moving our cell therapeutic approach forward to enable clinical trials” 

Alex Johnson, co-founder of Joining Jack said:

“Joining Jack is absolutely committed to accelerating potentially exciting therapies into clinical trial so that they can be tested on patients. Stem cells offer the potential hope of creating new healthy muscle in DMD patients, and we are pleased to be supporting this innovative and ground-breaking project.”

 

Duchenne UK would like to thank partner charities and family funds for supporting this project: Joining Jack, Alex's Wish, Jacobi's Wish, William's Fund, Project GO, Cure4George, Jack's Mission, Action for Arvin, The Smedley Family

 

Q&A with Dr. Rita Perlingeiro, Professor at the Department of Medicine at the University of Minnesota,

What are stem cells?

Stem cells are cells that can develop in to many different cell types in the body. In the case of pluripotent stem cells, they are able to divide and replenish all cell types of the body including muscle fibres. When exposed to specific culture conditions, it is possible to generate very large numbers of tissue specific skeletal muscle stem/progenitor cells.

What do we hope stem cells will do?

As we have observed in extensive proof-of-concept studies using mouse models of DMD, we hope transplantation of pluripotent stem cell-derived skeletal muscle stem/progenitor cells into DMD patients will reduce the muscle degeneration caused by lack of dystrophin. We expect that injected stem cells will i) produce functional muscle and ii) populate this new muscle with muscle stem cells, and therefore contribute to long-term regeneration.

Why is cell-based therapy important for treating DMD?

Skeletal muscle is a highly regenerative tissue. In DMD, muscle stem cells work hard to replace damaged tissue with new muscle fibers, but these also lack dystrophin, leading to successive cycles of degeneration/regeneration. A successful cell-based therapy will be able to break this cycle and counteract DMD pathology, as transplanted cells will give rise to dystrophin-expressing functional muscle fibers.

What are the potential risks or side effects of this approach?

Potential side effects: No clinically significant adverse effects are expected. Mild effects (e.g. inflammation, hematoma) at the site of injection. This approach will require the use of immunosuppression. Therefore, there is potential for side effects associated with this type of medication.

Potential risks: Undifferentiated cells are cells that are not specialized to perform a specified function in the body.  Cell therapies require differentiation of undifferentiated cells into specialized cell types, such as our target cell population, skeletal muscle stem/progenitor cells.  If differentiation fails or some other error leads to accidental injection of large numbers of undifferentiated pluripotent stem cells, a teratoma may result. This risk applies to any cell therapy based on pluripotent stem cells and is extremely low in the study described here as quality control procedures rigorously evaluate cells prior to transplantation.  

If this project is successful, what will the next steps be? 

Filling of an Investigational New Drug (IND) with the FDA, and initiating of a First-in-Human Phase 1 Safety/Dose Escalation Trial of pluripotent stem cell-derived skeletal myogenic progenitors for DMD.