STAT3 a new research to treat DMD

Canadian Research series

This video series of interviews features Canadian scientists who work on solutions for Duchenne muscular dystrophy (DMD). We wish to connect you – the DMD community – with these scientists. You’ll be delighted to learn about their motivation and dedication to this cause. In turn, they appreciate knowing more about our community, because we’re the people who are impacted by their discoveries. We hope that the interviews will help to foster an understanding of scientific concepts that aren’t always easy to grasp.

 

Meet the Gunning Group

We met and interviewed Patrick Gunning, Ph.D., who leads the Gunning Group, and one of his Ph.D. students, Yasir Raouf. Professor Gunning holds a Canada Research Chair in Medicinal Chemistry and is Director of the Centre for Medicinal Chemistry, Department of Chemical and Physical Sciences, at the University of Toronto’s Mississauga campus.

The Gunning Group is a team of scientists who pursue projects that focus on drug discovery. They work at the molecular level to design new drugs. Their goal is to develop better treatments and diagnostic options for devastating and under-explored human diseases. One of those diseases is DMD.

Dr. Gunning: “Yasir and myself are very motivated to try and see whether a STAT drug would work in DMD,”

 

What to know about STAT3

Their research targets STAT3, a protein that plays an essential role in cell regeneration. STAT3 is a transcription factor that controls or regulates, to some extent, how cells proliferate or grow and divide.

That’s very relevant to DMD, says Yasir Raouf. In DMD, muscle cell regeneration is poor. “Usually in humans, when your muscle cells die, they regenerate, and you get new muscles. If you have DMD, that regeneration is slow or nonexistent.”

If you can control STAT3, you can control the regenerative capacity of muscle cells, he believes.

 

What’s the timeline for development of this treatment?

Over the last decade, the Gunning Group developed a STAT3 drug that inhibits this protein. Over the next two years, they plan to test its effectiveness in mouse models. They will study how it affects muscle cell proliferation in mice over an extended period. For this work, they will collaborate with Dr. Michael Rudnicki, Professor and holder of a Canada Research Chair in Molecular Biology at the University of Ottawa.

 

Words of hope for the DMD community

Dr. Gunning has a message for families in the DMD community: “It’s very important to raise awareness for this disease.”

He urges families to highlight to the industry that people need help and new drugs to treat this orphan disease. “If money and efforts in research are put towards this disease, I’m very confident that academics like myself or industry can find something that will spare the lives of these children.”

Yasir Raouf hopes that we will soon live in a world that has cured DMD.“That’s what I hope for. That’s what I work for.”

 

More about the Gunning Group: http://www.gunninggroup.ca

 

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Girls with Duchenne: The Great Forgotten

Only one woman in 50 million is affected by Duchenne muscular dystrophy (DMD). Considered as a male disease, DMD usually affects boys (1 in 3500). Some people don’t know that they exist. Those women affected by DMD are there, and they are suffering from this lack of education. Know that DMD is a rare disease and difficult to live for a boy. So, just imagine what it could mean for girls. They face many additional obstacles; they aren’t unicorns they exist, and DMD girls deserve to be heard. Here’s what Feriel had to say and without censorship.

 

Feriel’s seeks equality

Feriel Cheba: “As a woman with this myopathy, I am fighting to say that us, girls with Duchenne’s myopathy, we exist. We are here, we too, we do want to live and have the right to dream about recovery and a better life. »

 

A world traveller despite DMD

Feriel is a young woman of 26 years old, native of France. Tiredness related to DMD didn’t stop her to become a world traveller. She visits many countries going through Europe, Tunisia, Mauritania, Cuba and Senegal to only name them. She is also a cinephile and loves everything that involves culture. Simply put, she loves life. She fights to live life to the fullest every single moment.

 

A sense of abandonment

It is in 1998 that she received the diagnosis of Duchenne’s myopathy (myopathy is the term used in France). She was only six years old, and it was already challenging to hear this news. However, repeating continually that she has a male disease amplifies her pain.

Feriel Cheba: ” It’s very hard as a parent to learn that your child has this disease especially when doctors tell you constantly that it’s a male disease. Incomprehension and unfairness are high. Why do so many doctors think this disease doesn’t affect women? It is time now to tell talk about it with young medical students, it has to be told during their genetic courses that Duchenne’s myopathy affects mostly boys and sometimes girls too”.

 

Hurtful words

She also gets many hurtful comments. There is a lack of resources and answers. In fact, it is even harder for girls to access clinical trials. Two neurologists told her that some clinical trials were open for boys exclusively, what she thinks is an injustice.

Feriel Cheba: “People do not believe me. They tell me that it’s impossible that I am affected by this disease. People don’t like what is rare. An internist even told me that it was an anomaly. Am I unnormal? No, I don’t think so. I am only different as everyone else”.

 

The hope for recovery and everybody

Despite all the difficulties encountered, she remains hopeful. She takes advantage of every moment spent with her family and friends. She also knows the existence of a treatment that can help with her specific mutation. Although, she can’t access it since its exclusive for walking patients. In her heart, she keeps hoping and dreaming of the day there will be a treatment for everyone suffering from DMD.

 

More about Duchenne: Duchenne muscular dystrophy – La Force DMD

Facebook Group: Girls can get Duchenne Too

MDA > Girls don’t get Duchenne or do they

Myopathie de Duchenne

 

 

Portrait of Duchenne – For the love of his brother!

What’s it like to live with DMD? As a parent, to face the reality that your child has a fatal disease? As a person with DMD, to face an inevitable, premature death in your twenties? As a sibling, relative or friend, to face mourning your loss?

The journey is beyond words.

Our Web short documentary series, “Portrait of Duchenne Canada”, will present the experiences of families and children with DMD. You will meet five Canadian families who are taking on the challenges of raising money and leading the way in advocating for access to new treatments. Today, more than ever, it’s time to share their stories. New treatments are on the horizon and families need to be heard to advocate for ways to access them quickly.

SAMUEL & GUILLAUME

Dream of a robotic arm becomes real, too late…

Samuel Fleurant-Beauchemin’s brother Guillaume had DMD. Guillaume was gradually losing the use of his arms. One day, Samuel came to visit his brother with a sparkle in his eyes. He had just seen a JACO robotic arm. He believed that the wheelchair-mounted device would restore Guillaume’s independence. Samuel decided to get his brother the robotic arm at all costs! He would start a non-profit organization to raise funds. He would quickly succeed in his task. And he did. But on the day that the arm was installed, destiny struck. Guillaume was admitted to the hospital, where he passed away shortly afterwards. Despite this ordeal, Samuel decided to continue to give back. Today, he runs an organization called A bridge to autonomy. To date, they have given 20 robotic arms to children with DMD in Quebec.

 

 

 

Quotes from the video

 

Mourning with his brother

Samuel Fleurent-Beauchemin:“To me, what has been the hardest was to live with the bereavements of my brother, who should no longer be able to do such and such.”

 

Samuel Fleurent-Beauchemin: “I was a bit angry with life, to make him suffer and to make us go through all of this.”

 

Samuel Fleurent-Beauchemin:“I think for him, as a human being who has already had his autonomy, I think the biggest challenge is to understand that you once have done it, but no more today.”

 

When the arm was installed, he went to emergency, never to return

Samuel Fleurent-Beauchemin:“On January 24th, Guillaume’s arm was installed. He had just been officially installed and, that day, he went to the emergency.”

 

Samuel Fleurent-Beauchemin: “We have created a foundation to help others. We help people by providing them with technical assistance and tools that are complementary to what the government offers. Currently, it involves the gift of robotic arms.”

 

Paying forward in memory of his brother

Samuel Fleurent-Beauchemin: “What my brother has brought me in life continues to grow on me. It is the project that he inspired in me; it is a project that represents him. It’s always inside me and it’s always growing. When you are in mourning, you really must focus on the person you have become, because of these past ones. Without this person, we would not have become who we are. I am glad because he’s still making me grow.”

 

Join the fight!

Duchenne muscular dystrophy (DMD) is a rare disease with no cure. Orphan drugs that target its causes are being developed now. But we need your help. Research is the only hope. And research requires funding.

For the past 20 years, Canadian families in the DMD community have been raising funds for and awareness of DMD. Their actions and successes have funded current research. Now that new treatments are emerging, families need to unite and be heard to access new treatments quickly.

 

Bruce Babington, our own La Force athlete, will cycle across Canada in July 2018 to raise funds and awareness for DMD. Our part is to tell their story.

 

Sharing these stories is one step forward towards the cure

  • We invite you to share this story with your community.
  • Encourage Bruce’s challenge: to cycle from Quebec to British Columbia in 24 days! Donate / here
  • Make a donation to our organization, so we can continue to help get closer to treatment. Donate / here

 

For more information:

About Samuel’s organization: Un pont vers l’autonomie / here

About Kinova’s JACO robotic arm: Kinova / here

Portrait of Duchenne – Victor’s unforgettable experience!

 

Giving an incredible experience to your child

Victor Morand, 11 years old: “I like it because they give you a lot of encouragement and it’s one of the only activities I can do with my dad.”

It took about two years of going back and forward to doctor appointments before the Morand family got a DMD diagnosis. Like every other parent, Jean-Philippe Morand turned to the Internet looking for answers. He understood…  the best hope for Victor was research. Exactly one year after receiving the diagnosis, they started a fundraiser event called Dystrospin.

They’ve raised thousands of dollars for the past four years, but Jean-Philippe wanted to do more so he started running and cycling with Victor which made them on top for raising funds awareness for DMD. In 2017, the father and son travelled over 600 km; they ran two marathons (Québec and Ottawa) and accomplished a track cycling of 650 km called La randonnée Jimmy Pelletier. This is the inspiring story of Jean-Philippe and Victor.

 

 

Quotes from the video

The hope lies in the research

Jean-Phillipe Morand: “Hope lies in research. A few weeks after I learned about Victor, I thought: well, after going through all the information on the internet about muscular dystrophy, I realized that my only hope lies in research. So, from there, we started exactly a year after the diagnosis the Dystrospin for collecting money to give it to research so we can find a cure to help Victor.”

 

The importance of their actions for funding research

Jérôme Frenette, Professor: “People alike Jean-Philippe who put so much energy in developing recruitment projects, it really matters. Finally, it puts a face on those affected by the disease, we now know why we wake up to work. We have children…I have pictures of many children at my office, I put them in the laboratory. People like the foundation give us a real picture of the tangible reality about everyday work that you tend to lose sight of when you work in a laboratory. Foundations are helpful, they give us credibility when we discuss with granting agencies. I think that for them, we also put a face on the children concerned, on people and on the importance of research in those fields.”

 

Join the fight!

Duchenne muscular dystrophy (DMD) is a rare disease with no cure. Orphan drugs that target its causes are being developed now. But we need your help. Research is the only hope. And research needs funding.

For the past 20 years, Canadian families in the DMD community have been raising funds for and awareness of DMD. Their actions and successes have funded current research. Now that new treatments are emerging, families need to unite and be heard to access new treatments quickly.

 

Bruce Babington, our own La Force athlete, will cycle across Canada in July 2018 to raise funds and awareness for DMD. Our part is to tell their story.

 

Sharing these stories is one step forward towards the cure

  • We invite you to share this story with your community.
  • Encourage Bruce’s challenge: to cycle from Quebec to British Columbia in 24 days! Donate / here
  • Make a donation to our organization, so we can continue to help get closer to treatment. Donate / here

 

For more information:

Jean-Philippe Morand event Distrospin/ here

La Randonnée Jimmy Pelletier/ here

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Capricor initiates HOPE-2 clinical trial

One of the objectives of our team is to inform you about new treatments. Here’s the latest news from a recent press released by Capricor which announces the clinical trial HOPE-2. Enjoy reading and thank you for sharing these articles within the DMD community.

CAP-1002 aim to maintain or improve cardiac and skeletal muscle function

Capricor’s lead candidate, CAP-1002, is a cell therapy that is currently in clinical development for the treatment of Duchenne muscular dystrophy (DMD). CAP-1002 is an allogeneic product, meaning that it is manufactured from donor heart tissue and then stored until needed for use. CAP-1002 consists of allogeneic cardiosphere-derived cells, or CDCs, a unique population of cells that contains cardiac progenitor cells. CAP-1002 has been shown to exert potent immunomodulatory activity and alters the immune system’s activity to encourage cellular regeneration. CDCs have been the subject of over 100 peer-reviewed scientific publications and have been administered to approximately 140 human subjects across several clinical trials.

Status by the FDA

CAP-1002 has been granted orphan drug designation by the FDA for the treatment of DMD.

About the HOPE study

Capricor’s previous clinical trial, the HOPE-Duchenne trial, evaluated the safety and efficacy of a single dose of CAP-1002 in boys and young men with heart disease related to Duchenne muscular dystrophy. It found CAP-1002 was safe, well tolerated and demonstrated significant and sustained signals of improvement in cardiac and skeletal muscle function.

HOPE 2

Participants in the HOPE-2 trial will be randomized to receive either placebo or CAP-1002, delivered intravenously every three months for a total of 4 administrations. Participants will be followed for a yearlong period following randomization, and an open-label extension (OLE) study is planned. The trial evidence should suggest an appropriate risk/benefit profile of CAP-1002 in the medical indication. Capricor – HOPE-2

Where is the trial?

The medical center in Sacramento is the first site in the nation to begin enrolling and treating participants. Approximately 12 to 15 investigative sites are expected to participate in the trial.

About Capricor

Capricor Therapeutics is a clinical-stage biotechnology company focused on the discovery, development and commercialization of first-in-class biological therapeutics for the treatment of rare disorders. Capricor’s lead candidate, CAP-1002, is an allogeneic cell therapy that is currently in clinical development for the treatment of Duchenne muscular dystrophy.

What is DMD?

Duchenne muscular dystrophy (DMD) is a disease that almost exclusively affects boys and whose incidence is 1 in 3,5000. It is extremely rare that Duchenne muscular dystrophy (DMD) will affect girls. Those affected are usually diagnosed around the age of five, but symptoms may be visible from early childhood. It is a degenerative disease of the muscles caused by a genetic mutation. The Duchenne muscular dystrophy (DMD) – for which no treatment is currently available – directly affects skeletal muscles. Without treatment, the consequences of the disease are dire for those afflicted and their families.

For more information:

Rare Disease Report

GlobeNews Wire – Press Release

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Edasalonexent: Positive results and no side effects

One of the objectives of our team is to inform you about new treatments. Here’s the latest news from a recent Catabasis press release about edasalonexent. Enjoy reading and thank you for sharing these articles within the DMD community.

The MoveDMD trial through 48 weeks of edasalonexent treatment

 

“ We believe that these effects will ultimately translate to boys with Duchenne maintaining functional abilities longer.”

Said Jill C. Milne, Ph.D., Chief Executive Officer of Catabasis, in a press release this week.

The fact that no evidence of side effects or safety has been observed after more than 37 patient-years of exposure to treatment is also encouraging.

What is Edasalonexent?

Edasalonexent (CAT-1004)  is being developed as a potential foundational disease-modifying therapy for all patients affected by DMD, regardless of their underlying mutation. It is an investigational oral small molecule. Edasalonexent inhibits NF-kB, a protein that is activated by DMD and drives inflammation and fibrosis, muscle degeneration and suppresses muscle regeneration.

You can watch our video here to have a more in-depth explanation about the basis of this treatment.

 

Positive results:

Statistically significant improvement was observed compared to the off-treatment control period. These improvements show a slowing of disease progression and are in addition to the improvements found in all assessments of muscle function through more than a year of edasalonexent treatment.

What’s next?

Catabasis is preparing for a Phase 3 trial that will enroll approximately 125 boys with DMD between the ages of 4-7 years old regardless of mutation type and who have not been on steroids for at least six months. It is planned to be a single, global, placebo-controlled Phase 3 trial with two boys receiving edasalonexent for every one boy receiving placebo. After 12 months in the trial, all boys are expected to receive edasalonexent in an open-label extension.

Approbation in the US: FDA Status

The FDA has granted orphan drug, fast track and rare pediatric disease designations and the European Commission has granted orphan medicinal product designation to edasalonexent for the treatment of DMD. For a summary of clinical results reported to-date, please visit www.catabasis.com.

About DMD

Duchenne muscular dystrophy (DMD) is a disease that almost exclusively affects boys and whose incidence is 1 in 3,500. It is extremely rare that Duchenne muscular dystrophy (DMD) will affect girls. Those affected are usually diagnosed around the age of five, but symptoms may be visible from early childhood. It is a degenerative disease of the muscles caused by a genetic mutation. The Duchenne muscular dystrophy (DMD) – for which no treatment is currently available – directly affects skeletal muscles. Without treatment, the consequences of the disease are dire for those afflicted and their families.

For more information:

More information about the trial:  DMDtrials@catabasis.com and Catabasis -clinical trials

More: Portrait of Duchenne – edasalonexent cat-1004 – La Force DMD

Press release: www.catabasis.com

Business Wire: www.businesswire.com

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A clinical trial on utrophin modulation is completed

One of the objectives of our team is to inform you about new treatments. You can catch up on the last news in the latest press release by Summit Therapeutics company about ezutromid, a utrophin modulation. We wish you a happy reading and thank you for sharing those articles within the DMD community.

 

“PhaseOutDMD” clinical trial for ezutromid is completed

“We believe the early improvements seen in muscle health in the interim data from PhaseOut DMD indicate ezutromid is reducing DMD disease severity. In the full trial results, we aim to see continued utrophin modulation and sustained changes in magnetic resonance parameters. These results, if positive, could form the basis of a regulatory filing of ezutromid, bringing this universal treatment to patients more rapidly.”

Said Dr. David Roblin, Chief Medical Officer and President of R&D of Summit.

What is “PhaseOutDMD”?

PhaseOut DMD is a Phase 2 open-label, multi-centre trial of the Company’s utrophin modulator, ezutromid, in patients with DMD. Previously announced 24-week interim data from PhaseOut DMD showed evidence of activity across three different measures. Specifically, ezutromid:

• Maintained the production of utrophin, a naturally occurring protein that can potentially substitute for dystrophin, as measured by muscle biopsy;

• Significantly and meaningfully reduced muscle damage, as measured by muscle biopsy; and

• Significantly reduced muscle inflammation, as measured by magnetic resonance.

What is utrophin?

The human body naturally produces utrophin, a protein, when a muscle is first forming or when a muscle is repairing. As a muscle matures, dystrophin replaces utrophin. However, in people with Duchenne muscular dystrophy (DMD), dystrophin does not function properly. Utrophin is functionally and structurally similar to dystrophin. Preclinical trials that have simulated sustained utrophin production have shown that it could potentially replace dystrophin in people with Duchenne muscular dystrophy (DMD). The replacement of dysfunctional dystrophin with functional utrophin might have a highly positive impact on muscle performance.

Summit Therapeutics believes that utrophin may slow or even stop the progression of DMD.

 

This video explains what is utrophin.

About Summit Therapeutics

Summit is a biopharmaceutical company focused on the discovery, development and commercialization of novel medicines for indications for which there are no existing or only inadequate therapies. Summit is conducting clinical programmes focused on the genetic disease Duchenne muscular dystrophy and the infectious disease C. difficile infection. Further information is available at www.summitplc.com and Summit can be followed on Twitter (@summitplc).

What is DMD?

Duchenne muscular dystrophy (DMD) is a disease that almost exclusively affects boys and whose incidence is 1 in 3,5000. It is extremely rare that Duchenne muscular dystrophy (DMD) will affect girls. Those affected are usually diagnosed around the age of five, but symptoms may be visible from early childhood. It is a degenerative disease of the muscles caused by a genetic mutation. The Duchenne muscular dystrophy (DMD) – for which no treatment is currently available – directly affects skeletal muscles. Without treatment, the consequences of the disease are dire for those afflicted and their families.

Useful links

Here are a few additional links including the initial news release of the Summit Therapeutics company.

Link to the news release: www.summitplc.com

Link to additional information on utrophin modulation:

Positive data about utrophin modulator – La Force DMD

Portrait of Duchenne – Utrophin modulator – La Force DMD

 

  • 2018 > Summit Therapeutics ends development of ezutromid therapy for DMD after trial failure. Read more here.
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Portrait of Duchenne – Every action counts!

A chance encounter launched groundbreaking DMD research

When Max Sedmihradsky was two-years-old, he received the DMD diagnosis. The news was devastating beyond words for his parents Kerry and Andrew. After a moment of profound sadness, Andrew came up with the idea for Max’s Big Ride. Every year he cycles between Ottawa and Hamilton, Ontario, with Max in front of the cargo bike. The ride aims to raise funds and awareness for Duchenne muscular dystrophy.

Andrew works at the University of Toronto, where the researcher Patrick Gunning also works. Andrew took the chance to reach up to him to see if he would do some research for DMD. At that time, Patrick Gunning reached out to his graduate students to start a research on DMD. They found out that the cell they are working on, “Stat 3”, plays a decisive role in DMD. A year later, after this chance encounter, the Gunning Group received a $300,000 grant and are performing ground-breaking research for DMD. This is the story of Max’s Big Ride and the Gunning Group.

 

 

Quotes from video

Magic happened

Andrew Sedmihradsky: “You know, it’s the very random kind of thing that happened and the fact that I work in the building where there is a man who potentially could have the cure for Duchenne is tremendous”

 

Kerry Sedmihradsky: “Patrick Gunning is a researcher at the University of Toronto where Andrew works. And Andrew attended a talk which mention the research that Patrick was doing and for what he has told me, he is kind of the rock star of researchers at the University of Toronto”

 

Patrick Gunning, Associate Professor: “Would you be interested in looking at developing drugs for Duchenne?’’ And I said: “Well, we are typically oncology based and I don’t think that the targets that we make drugs for are involved in Duchenne.” I asked my graduated student Yasir to investigate whether STAT proteins in particular were involved in Duchenne.”

 

A ride of hope: Max’s Big Ride

Andrew Sedmihradsky: “You know, a few days into it, we just went on a family trip to the museum and I remember in the gift shop seeing a card that said: “Falling down is part of life but getting up is living.  It really responded to me, it resonated with me. I think that’s kind of the attitude that we’ve adopted you know, from that point forward. And you know, you have to fight, and we’re determined as a family to do so. To try and beat this.”

 

Kerry Sedmihradsky: “So, he worked out the route between Hamilton and Ottawa which is 600 km with Max riding in the front of the Cargo bike and it’s been a really, really positive experience for our family.”

 

Groundbreaking research with the Gunning Group

Yassir, Graduated student: “It’s very rare that we get to see what we do have an impact. So yeah, my motivation is, just all the young children that have Duchenne and if I can do anything, you know one percent that I can do to try to push this a little bit more, maybe five years later someone pushes it more and hopefully, we live in a world soon where Duchenne is something that is normal and we can cure it.”

 

Join the fight!

Duchenne muscular dystrophy (DMD) is a rare disease with no cure. Orphan drugs that target its causes are being developed now. We need your help. Research is the only hope. And research needs funding.

For the past 20 years, Canadian families in the DMD community have been raising funds for and awareness of DMD. Their actions and successes have funded current research. Now that new treatments are emerging, families need to unite and be heard to access new treatments quickly.

 

Bruce Babington, our own La Force athlete, will cycle across Canada in July 2018 to raise funds and awareness for DMD. Our part is to tell their story.

 

Sharing these stories is one step forward towards the cure.

  • We invite you to share this story with your community.
  • Encourage Bruce’s challenge: to cycle from Quebec to British Columbia in 24 days! Make a donation /Here
  • Donate to our organization, so we can continue to help get closer to treatment.  /Here

 

For more information:

About Max’s Big Ride:  Max’s Big Ride / Here

About the Gunning group: The Gunning Group  / Here