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Treating Duchenne muscular dystrophy focus of new research partnership

For Immediate Release – April 4, 2019

Toronto, Ontario – Muscular Dystrophy Canada (MDC) and The Foundation for Gene & Cell Therapy (Jesse’s Journey) are joining forces to accelerate ground-breaking research focused on new treatments for Duchenne muscular dystrophy to the sum of $600,000.

Through this partnership, MDC will provide $300,000 with Jesse’s Journey matching the commitment. Two research projects will be funded:

Dr. Anthony Gramolini: Non-viral, immune-modulatory nanoparticles for delivery of CRISPR/Cas9 as a treatment intervention for Duchenne Muscular Dystrophy.
Dr. Michael A. Rudnicki: Exosomal Delivery of Wnt7a for Treating Duchenne Muscular Dystrophy.

“For anyone impacted by a neuromuscular disorder, research offers hope that better treatments will be uncovered, and ultimately, a cure. Both of these research projects show very promising initial results and we are thrilled that this investment will help move the research forward,” said Barbara Stead-Coyle, CEO, Muscular Dystrophy Canada. “We are thrilled to be working with Jesse’s Journey to fund this promising research and can’t wait to see what positive impacts it has on our clients and families.”

Both projects focus on improving a critical component, the delivery vehicle, used in gene therapies like gene transfer and gene editing.

“Jesse’s Journey is proud to partner with Muscular Dystrophy Canada to fund these two exceptional research studies,” said Perry Esler, Executive Director Jesse’s Journey. “For nearly 25 years, Jesse’s Journey has funded the most promising Duchenne research worldwide and we’ve seen first-hand the difference that is made in the life of a person facing Duchenne when they are able to benefit from scientific discovery. Jesse Davidson – the inspiration behind Jesse’s Journey – once said, “When researchers work together, miracles can happen.” We believe the same can be true when funding organizations work together, and it’s in the spirit of collaboration we welcome the opportunity to join forces with MDC.”

There are already several clinical trials exploring gene transfer (a type of gene therapy) underway for Limb-Girdle and Duchenne Muscular Dystrophies. These make use of AAV (adeno-associated virus) as the transport vehicle but limitations exist in using AAVs including small packaging capacity, difficulty in producing large amounts, and potential to cause an immune response especially if they need to be administered more than once.

To circumvent some of the limitations of AAV, both research projects will be testing alternative non-viral vehicles.

Dr. Gramolini and his team will test BIPHIP, a nanoparticle which will be used to transport CRISPR gene editing tools into cells. with the goal of editing a genetic change that causing this disorder. Dr. Rudnicki and his team will test exosomes as vehicles to deliver the Wnt7a gene, which has been shown to help muscle cells regrow.

If successful, the results could have broader applicability to other types of muscular dystrophies.

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ABOUT MUSCULAR DYSTROPHY CANADA

Muscular Dystrophy Canada’s mission is to enhance the lives of those impacted with neuromuscular disorders by continually working to provide ongoing support and resources while relentlessly searching for a cure through well-funded research. To learn more about Muscular Dystrophy Canada, please visit www.muscle.ca or call our toll-free number at 1-800-567-2873.

ABOUT JESSE’S JOURNEY

Jesse’s Journey is a partnership of science and families dedicated to the discovery of treatment and cure of Duchenne muscular dystrophy – because parents should not out live their children. To learn more about Jesse’s Journey visit www.jessesjourney.com or call 519-645-8855.

MEDIA CONTACT INFORMATION:

Heather Rice
Muscular Dystrophy Canada
Heather.Rice@muscle.ca
902-440-3714

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Federal government commits to national strategy to improve access to rare disease drugs

Toronto, Ontario – Muscular Dystrophy Canada commends the Government of Canada for committing $35 million, in its 2019 budget, to develop a Canadian Drug Agency to oversee the development of a new national formulary of prescribed drugs and a strategy to provide support for Canadians with rare diseases.

A national strategy to alleviate the high-cost of drugs for rare diseases will ensure more Canadians are able to access effective, life-changing treatments.

“This is an important first-step towards ensuring individuals diagnosed with a rare disease, such as a neuromuscular disorder, are able to access treatment without cost as a barrier,” said Barbara Stead-Coyle, CEO, Muscular Dystrophy Canada. “We call on all Federal parties to focus on this very real issue facing Canadians.”

According to the Health Charities Coalition of Canada (HCCC), recent studies show that while a majority of Canadians have access to prescription medicines under the current mix of private and public insurance, approximately 22% of prescription drug costs are still paid for out of pocket by patients.

Stead-Coyle added, “MDC has always been committed to ensuring patients can access treatments at an affordable price, and we will continue to advocate on behalf of patients until that’s a reality.”

Muscular Dystrophy Canada, along with its partners in HCCC, looks forward to integrating the perspectives of patients and the health charity sector as we work with the Federal Government to develop and implement these important initiatives.

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ABOUT MUSCULAR DYSTROPHY CANADA

Muscular Dystrophy Canada’s mission is to enhance the lives of those impacted with neuromuscular disorders by continually working to provide ongoing support and resources while relentlessly searching for a cure through well-funded research. To learn more about Muscular Dystrophy Canada, please visit www.muscle.ca or call our toll-free number at 1-800-567-2873.

MEDIA CONTACT:

Heather Rice
Muscular Dystrophy Canada
Heather.Rice@muscle.ca

902-440-3714

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CADTH review and Spinraza

Spinraza decision signals the need for a Canadian Rare Disease (or Orphan Drug) Framework

MDClogo-stacked

The drug review and approval process in Canada is complex. Many agencies from Health Canada, to Canadian Agency for Drugs and Technologies in Health (CADTH) and Institut national d’excellence en santé et en services sociaux (INESSS), to the Pan-Canadian Pharmaceutical Alliance and each individual provincial and territorial governments all share in the decision-making.

While ensuring real world evidence and improved health outcomes should be at the top of the list of considerations, cost becomes a significant determining factor. We understand that governments are faced with significant healthcare challenges and budgets are strained. However, timely and equitable access to care is the foundation of the Canadian health care system. It is the value we share, one that defines us and our health care system.

That is why the recent decision released by CADTH on March 1, 2019 related to the use of Spinraza to treat Spinal Muscular Atrophy (SMA) types 1, 2, 3 and 4, a rare neuromuscular disorder, cannot and should not be ignored. read more

With this one decision, the neuromuscular community and the entire rare disease community is wondering whether we will ever have timely and equitable access to care. This decision has highlighted the fact that Canada desperately needs to redefine an approach to the treatment and management of rare diseases.

Health Canada defines rare diseases as life-threatening, debilitating or serious and chronic conditions that affect a small number of individuals. It is estimated that about 1 in 12 Canadians are affected by a rare disorder, the majority of who are children. The 160 plus neuromuscular disorders that fall under the umbrella of Muscular Dystrophy Canada are part of the rare disease landscape.

SMA affects patients with widely ranging degrees of severity depending on age of onset. SMA Type I presents by the age of six months and is the most common genetic cause of infant mortality. In SMA Type II, age of onset is six to 18 months, and patients have delayed motor milestones, respiratory issues and possibly shortened life expectancy. SMA Type III patients are those with onset from 18 months to 18 years of age, and they experience muscle weakness. SMA Type IV is adult onset with varying degrees of muscle weakness. Common to all types of SMA is a progressive decline in muscle function.

SMA causes major problems with walking, muscle strength, fine motor skills, (deep) breathing, and swallowing or feeding. The inability to walk means relying on wheelchairs and other mobility aids and dealing with associated barriers. In more severe cases, patients cannot execute basic movements such as sitting up or feeding themselves.

Difficulty in breathing may lead to reliance on mechanical ventilation, and difficulty in swallowing may necessitate use of a feeding tube. For young adults, all of these difficulties present barriers to moving away from home, finding work, and making friends. There is also awareness of the burden placed on family members.

Nusinersen, marketed as Spinraza, is a medication used in treating SMA. An “antisense” drug, Spinraza is designed to target genetic instructions at the RNA stage (an intermediate step between DNA and the protein manufacturing stage inside cells), resulting in increased production of the needed survival motor neuron (SMN) protein.

In December 2016, Spinraza became the first approved drug in Canada to treat this disorder. It also has orphan drug designation in the United States and the European Union. In fact, clinical trials that involved testing Spinraza in infants and children were stopped prematurely based on early results that clearly showed participants receiving treatment with the drug were achieving significantly more motor milestones than those who were receiving a placebo. All participants in those trials were then invited to transition into an open-label study in which they all would receive the drug.

One parent described the treatment as a “miracle”. Demonstrated improvements include stronger breathing, speech, and coughing; increased physical strength, energy, and movement; ability to perform new actions such as holding one’s head up, rolling over, bearing weight on legs, raising arms and legs to and above the head, sitting or standing up with and without assistance, and using mobility aids. Parents also described faster and more complete recovery from illness and positive effects on mood and confidence.

CADTH and INESSS each undertook separate and distinct review processes that led to markedly different recommendations for access, with CADTH recommending significant restrictions on access, while INESSS recommended no such restrictions. This has led to “luck by postal code” access for SMA patients in Canada. In Quebec, and 13 countries (Germany, Austria, Italy, Luxembourg, Israel, Japan, Spain, Belgium, Romania, Hong Kong, Portugal, Poland, Scotland) broad reimbursement is provided – presymptomatic, for types 1-3.

Given that both organizations reviewed the same scientific evidence, how could the decisions be so markedly different? CADTH’s decision is not in keeping with what other countries around the world are recommending. It would seem that the process needs to be reconsidered.

CADTH has not developed a review process that is fitting for rare diseases. We should not ignore the patient experience related to improvements in quality of life. Small, incremental improvements can be life changing for our community. Going from being fed to being able to feed yourself or from not being able to stand to being able to walk has value and needs to be weighed more strongly as part of the overall review process.

On their website it says – CADTH believes that credible, objective evidence should inform every important health care decision. When you want to know what the evidence says, ask CADTH. We fully support the need for scientific evidence to drive the review process; however, this needs to be balanced with real-world evidence, and the patient experience is a critically important part of that real-world evidence.

In fact, in a recent report of the Standing Committee on Health entitled “Canadians Affected By Rare Diseases and Disorders: Improving Access To Treatment,” one of the 19 recommendations recognized the importance of this type of evidence. It states:

“That the Government of Canada provide funding through the Canadian Institute of Health Research for research into the diagnosis of patients with rare diseases and the collection of real-world evidence regarding the effectiveness of treatments for these conditions.”

The Report recommendations include various approaches to strengthen and streamline the regulatory process with the aim of improving access to drugs for rare diseases and are fully supported by MDC.

The CADTH decision sends a signal that incremental breakthroughs are not important – yet this is how science generally works, one finding builds upon another.

With this decision, the challenge now moves to the provinces to determine how they will approach Spinraza as part of a provincial formulary. MDC will work with the community and with regulators to continue to advocate for access for this life-changing treatment. In reviewing the CADTH recommendation, we ask governments to recognize the inequity this decision creates, and seriously consider how to expand the availability of this treatment for all patients across Canada.

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MDC welcomes Daria Wojtal as Director of Research!

We are pleased to announce that Daria Wojtal has joined Muscular Dystrophy Canada as the new Director of Research. Daria comes to us from the Hospital for Sick Children and the University of Toronto, where she worked under the leadership of Dr. Ronald Cohn on her PhD in developing CRISPR gene editing technology for the treatment of neuromuscular disorders.

 

Daria Wojtal

 

Daria is also a long-time volunteer for Muscular Dystrophy Canada as a Planning Committee Member where she acted as a liaison between MDC and a team of scientists from SickKids who run scientific activities during the Walk for Muscular Dystrophy in Toronto.

Muscular Dystrophy Canada is excited about the work that Daria will be focusing on and the level of neuromuscular research knowledge she brings to the organization. She will be building out new programming to increase Muscular Dystrophy Canada’s investment and impact in research in Canada and internally through strategic partnerships.

Please join us in welcoming Daria Wojtal to the Muscular Dystrophy Canada family!

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Muscular Dystrophy Canada applauds Quebec for approval of SPINRAZA

For Immediate release – December 19, 2018
Toronto, Ontario

Muscular Dystrophy Canada applauds Quebec for approval of SPINRAZA for patients impacted by Spinal Muscular Atrophy Types 1, 2 and 3

Recommendations provided by the Institut national d’excellence en santé et en services sociaux (INESSS), the regulating body in Quebec, have resulted in the approval of SPINRAZA (nusinersen) for Spinal Muscular Atrophy (SMA) Types 1, 2 and 3 patients in that province.

This decision is the result of a refiling of the drug approval by Biogen early in 2018, after being reviewed and approved for use in Type 1 patients only.

The effectiveness of SPINRAZA was established in the pivotal ENDEAR and CHERISH studies and supported by multiple clinical studies. For individuals with infantile-onset (Type 1) and presymptomatic SMA results showed marked improvements in muscle function. How spinraza works

“Muscular Dystrophy Canada (MDC) was pleased to support both the initial and subsequent reviews by providing a comprehensive patient submission. Ensuring our community has a strong and credible voice in all matters related to trials and treatments is at the forefront of our work in Canada,” commented Muscular Dystrophy Canada CEO, Barbara Stead-Coyle. “This is a life changing treatment for our SMA community. We applaud the Minister of Health and Social Services Danielle McCann, and the Quebec government for reconsidering their initial decision and expanding access for more clients in Quebec.”

Stead-Coyle added “MDC’s commitment to ensuring SMA clients can access this treatment at an affordable price remains steadfast. There is still much work to do and we will continue to work tirelessly to ensure that all patients who may benefit from Spinraza will receive access as quickly as possible in Canada.”

A review from the Canadian Agency for Drugs and Technologies in Health (CADTH), the regulating body for the rest of Canada, is expected in the new year.

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ABOUT MUSCULAR DYSTROPHY CANADA

Muscular Dystrophy Canada’s mission is to enhance the lives of those affected with neuromuscular disorders by continually working to provide ongoing support and resources while relentlessly searching for a cure through well-funded research. To learn more about Muscular Dystrophy Canada, please visit www.muscle.ca or call our toll-free number at 1-800-567-2873.

MEDIA CONTACT INFORMATION:

Heather Rice
Muscular Dystrophy Canada
Heather.Rice@muscle.ca
902-440-3714

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