Each month, we gather partners from the MAGIC project to discuss aspects in relation to gene therapies. Readers will learn who the people behind the MAGIC Project are, why we are committed to advancing gene therapies, and how our roles in MAGIC are crucial for achieving better health outcomes for people living with muscular dystrophies. This time, we are speaking with Gustavo Dziewczapolski from Cure CMD.

In brief words, please let us know who you are individually and on behalf of which project partner organization. 

My name is Gustavo Dziewczapolski, and I am a biologist with a Ph.D. in Neuropharmacology from the University of Buenos Aires, Argentina. To further my scientific career, I relocated to the United States, where I have worked at UC San Diego and the Salk Institute. In 2016, I joined Cure CMD, an Advocacy Patient Organization, as the Scientific Director. Alongside Rachel Alvarez, the Executive Director, I represent Cure CMD in the MAGIC consortium.

Cure CMD was established in 2008 with the mission to advance research for congenital muscular dystrophy (CMD) treatments and to provide educational and supportive resources for the stakeholder community. CMD is an ultra-rare group of diseases with significant unmet medical need. One of Cure CMD’s primary objectives is to fund promising research that directly impacts the development of therapies and optimization of care for CMD.

Why are you participating in the MAGIC project? How can your perspectives complement the MAGIC project goal to accelerate the development of genetic therapies for muscular dystrophies? 

Part of the “MAGIC” of this consortium lies in its interdisciplinary and inter-stakeholder nature. With a robust foundation in academic labs to generate research hypothesis and results, and the involvement of small and medium-sized enterprises to synergize translational research, this consortium is also integrated with international patient advocacy groups to ensure that the research has a patient-centric approach. Our role includes disseminating the goals, progress and potential of the experimental results obtained. Additionally, we bring the “voice” of the affected community to the Consortium and provide biological samples when needed. 

What have been the current challenges regarding the development of genetic therapies for muscular dystrophies for you? 

One of the major obstacles for the development of genetic therapies for muscle disorders is the lack of humanized models of the targeted diseases, which hinders the development of precise strategies and tools for gene-directed therapies with a higher likelihood of translating into real human treatments. Additionally, challenges such as developing appropriate vectors to carry and deliver gene correction tools to the affected muscles will be addressed by MAGIC. By creating accurate humanized muscle organoids modeling CMDs, Duchenne Muscular Dystrophy, and X-linked Myotubular Myopathy, we aim to design and evaluate safer, more efficient, and more precise gene therapy vectors for these muscle disorders.

What are the main outcomes (direct results) you expect from the project? 

We anticipate that this ambitious project will deliver a set of pre-clinical proof-of-concept results for gene-directed therapies, significantly increasing the chances of these therapies translating into effective genetic treatments for individuals affected by each of the muscle disorders studied here.

What are the expected impacts on your organisation from participating in the project? 

Having direct access and a voice in the development and execution of the experimental plan, along with the opportunity to disseminate this information to our affected community, provides, on one side, significant lessons about the complexities of the research endeavor. On the other hand, it highlights the expertise and dedication of this group in overcoming the challenges of bringing safer and more precise gene therapy to the clinic.

Cure CMD has been managing a patient registry, a tissue repository, and a cell bank. Some of the cell samples used to create muscle organoids are from patient cells in our bank. Publicizing this direct contribution from the community to cutting-edge research will encourage more patients and their families to become involved in research.