Donate now

Friends of Garrett Cumming Research Chair

What Is the Friends of Garrett Cumming Research Chair?

Muscular Dystrophy Canada, in partnership with the University of Alberta, established the Chair in order to enhance national and international research capacity with the ultimate goal of finding a cure for all neuromuscular disorders.

How is the Chair funded?

The Chair was named for Garrett Cumming, a young man from Alberta living with Duchenne muscular dystrophy. Garrett’s determined family, friends and community worked tireless to raise more than $1.6 million from supporters. Muscular Dystrophy Canada leveraged each donation, dollar for dollar, by obtaining $1.5 million dollars from the Government of Alberta’s Access to the Future Fund. By working together, we established the first endowed Chair for neuromuscular research in Canada.

Who holds the position?

Dr. Toshifumi Yokota

Toshifumi Yokota, PhD, is the first and current Friends of Garrett Cumming Research Chair for Muscular Dystrophy Canada at the University of Alberta.

Dr. Yokota received his PhD in 2003 from The University of Tokyo School of Science. Upon completing his post-doctoral training, he worked as a Research Associate for Dr. Eric Hoffman at the Children’s National Medical Center, Washington DC, where he studied the molecular mechanisms of neuromuscular disorders such as and worked on developing safe and effective therapeutic options.

What is Dr. Yokota’s research focus?

Since being appointed Friends of Garrett Cumming Research Chair in 2011, Dr. Yokota has continued researching new and innovative therapies for treating muscular dystrophy. At present, his work focuses on three projects:

  • Antisense and Exon-Skipping Therapy Duchenne muscular dystrophy is caused by mutations in the gene encoding dystrophin. The Exon-Skipping approach employs synthetic DNA-like molecules as a DNA “patch” to skip over the mutated parts of the dystrophin gene, which is responsible for causing Duchenne muscular dystrophy. Dr. Yokota and his team have already demonstrated the first successful systemic treatment of dystrophic dogs using multiple exon-skipping.
  • Analysis of Dystrophin Revertant Fibres These fibres, which consist of functional dystrophin, can found in patients with Duchenne Muscular Dystrophy, who otherwise lack functional dystrophin proteins. By analysing these fibres, researchers may be able to develop new, more effective targets for treatments.
  • Muscle Membrane Imaging Some forms of muscular dystrophy including 2B and Miyoshi myopathy have a primary defect in skeletal muscle membrane repair. Dr. Yokota is using state-of-the-art technology to analyze the molecular mechanisms involved in muscle membrane repair. A better understanding of this process could lead to better treatments for patients.

Dr. Yokota’s research has received much attention, both in academic circles and the mainstream media. His goal is to one day find a cure for all forms of muscular dystrophy. Learn more about Dr. Yokota’s work and publications

Interested in finding out how you can support neuromuscular research? Contact us

An Interview with Dr. Toshifumi Yokota

In 2011, Dr. Yokota was named to the newly-created Friends of Garrett Cumming Research Chair, Muscular Dystrophy Canada at the University of Alberta. The first Research Chair of its kind in Canada, Dr. Yokota’s position offers an exciting new opportunity for research into neuromuscular disorders.

The following is an interview with Dr. Yokota.

Muscular Dystrophy Canada: Can you briefly describe your current research?

Dr. Yokota: We are working towards a cure for muscular dystrophy using a kind of “DNA band-aid” called morpholino. We use the small DNA-like molecules (called “morpholinos”) to cove” a mutated part of the gene like a band-aid, [allowing] the gene to jump over the mutated part of the gene and produce near-normal protein. We have recently achieved the first successful exon skipping therapy in a dog model of muscular dystrophy.

MDC: What are some the implications of your research, and what kind of therapies might it open up?

Dr. Yokota: The exon-skipping therapy was thought to be applicable to a very limited number of DMD patients, but we demonstrated that by using multiple morpholinos as a cocktail, we can potentially treat more than 90% of DMD patients.

We are also trying to apply this DNA band-aid to many different types of mutations of DMD and other types of muscular dystrophies, such as Limb-Girdle Muscular Dystrophy (LGMD) type IIB, Miyoshi myopathy, and Myotonic dystrophy.

MDC: What does your appointment to The Friends of Garrett Cumming Research Chair, Muscular Dystrophy Canada mean for your research?

Dr. Yokota: This is really a great opportunity for me, and I appreciate everyone who supported and made this possible. It will be my first experience starting an independent lab as a principal investigator, and I am very excited and honoured to do that at one of the most famous and prestigious universities in Canada. I am also looking forward to working with researchers and students there. I want to contribute to the continuing success of Canadian medical research on muscular dystrophy.

MDC: What do you think the creation of The Friends of Garrett Cumming Research Chair, Muscular Dystrophy Canada means for muscular dystrophy research in Canada?

Dr. Yokota: There are already many famous muscular dystrophy researchers in Canada; Canada is one of the leading countries in the field of muscular dystrophy research, especially in muscle stem cell research.

But there are not many researchers working on antisense therapeutics or exon-skipping therapy for muscular dystrophy as far as I know, so I think my specialty will fit very well.

I also want to collaborate with researchers in Canada. Since I did my PhD at the University of Tokyo and then worked at Imperial College [in the UK] and Children’s National Medical Center in Washington DC [as a post-doctoral researcher], I know researchers everywhere in the world. I would like to use these ties to promote more international collaborations in this field.

MDC: What direction do you see your research taking in the next 5-10 years?

Dr. Yokota: In the short term, I want to continue work on exon-skipping and antisense therapeutics for DMD and other forms of muscular dystrophies.

My longer term goal is to find another potential target of DMD treatment, especially to determine the factors which promote exon-skipping. DMD patients have mutations in the DMD gene and lack a protein called dystrophin, but most patients have a small proportion of dystrophin-positive fibres for unknown reasons (“revertant fibres”). Th” “revertant fibres” have spontaneou” exon-skipping, which means they can somehow jump over the mutated part of the gene spontaneously. This is a very similar mechanism to exon-skipping therapy with antisense morpholinos, and I am interested in performing molecular analyses of these positive fibres, which might lead to finding of a new target of muscular dystrophy treatment.

Muscular Dystrophy Canada: In addition to your work, what part of the move to Edmonton is most exciting for you?

Dr. Yokota: Edmonton reminds me of my hometown, Morioka, a city about 500 km north of Tokyo. It gets very cold there in the winter and I like winter sports, so I am looking forward to winter sports the most.

X
WP-Backgrounds by InoPlugs Web Design and Juwelier Schönmann