sIn early August 2016, Biogen and Ionis Pharmaceuticals announced that their investigational drug for the treatment of spinal muscular atrophy (SMA), nusinersen has met the pre-specified primary endpoint for the interim analysis of ENDEAR (A study testing the clinical efficacy and safety of IONIS_SMN Rx in patients with infantile-onset SMA). The observed effect of nusinersen on infants with infantile-onset (consistent with type 1) SMA was a statistically significant improvement in the achievement of motor milestones in comparison to infants who had not received the treatment. Nusinersen’s safety profile was also deemed acceptable during the study. The results obtained in the pre-specified interim analysis have led to the closure of the ENDEAR study, thus transitioning participants into the SHINE open-label study in which all patients will be receive nusinersen.
In light of this news, Biogen will develop and commercialize nusinersen globally and will process regulatory filings in the coming months. The executive vice president and chief medical officer at Biogen, Alfred Sandrock, M.D., Ph.D., highlighted “we share the community’s sense of urgency as we strive to bring the first treatment of SMA, the leading genetic cause of infant mortality, to families facing this devastating disease. We remain committed to understanding the potential of nusinersen in the broader SMA population and will continue to focus on the rapid completion of our ongoing studies.”
In September 2016, Biogen will open the global expanded access program (EAP) for eligible patients with infantile-onset SMA (consistent with Type 1). In Canada, the EAP program is being initiated at existing nusinersen clinical trial sites where there is a path supporting long-term availability of nusinersen, namely at BC Children’s Hospital, the Hospital for Sick Children, Montreal Children’s Hospital, and Children’s Hospital – London Health Sciences Centre. Upon initiation of the EAP along with the appropriate local approvals, enrolment may start at individual sites after transitioning ENDEAR study participants to the open-label extension study.
If you are interested in obtaining more information on the EAP program, please contact the following lead investigators at either of the sites:
BC Children’s Hospital, Vancouver BC – Dr. Kathryn Selby
Hospital for Sick Children, Toronto ON – Dr. Jiri Vajsar
Montreal Children’s Hospital, Montreal QC – Dr. Maryam Oskoui
Children’s Hospital – London Health Sciences Centre, London ON – Dr. Craig Campbell
For more information on the specific study please go to
Welcoming the New Year & new opportunities for collaboration
The Canadian Neuromuscular Diseases Network (CAN-NMD) would like to extend its best wishes for 2016 to all its stakeholders and partners, including the Neuromuscular Now blog readers. This year, we hope to further collaborate, share much more knowledge with our stakeholders and establish stronger partnerships. Please feel free to send us any ideas you may have for blog posts throughout this year. The Network also welcomes guest blog posts from its members. If you would like to write a guest blog, please feel free to contact the National Director of Operations, Megan Johnston at firstname.lastname@example.org or the National Knowledge Broker, Gracia Mabaya at Gracia.Mabaya@lhsc.on.ca with your innovative ideas for knowledge sharing via this platform.
From its inception, the Network has adopted knowledge brokering as a role based knowledge translation strategy with the ultimate goal of enhancing neuromuscular clinical practice, research and education through knowledge sharing and exchange and increasing collaborations among neuromuscular health care providers, researchers, patients, families and other stakeholders.
To date, the Network has accomplished the following knowledge brokering preparation activities:
Upcoming activities for 2016 include:
Application of the Knowledge Translation Framework: A Call to all Task Forces
During its second annual meeting in Ottawa in September 2015, the CAN-NMD released its knowledge translation framework. This framework has been developed using existing knowledge management and translation frameworks to act as a roadmap for all knowledge translation activities of the Network. The National Knowledge Broker is available to assist task forces and all network stakeholders in their use and evaluation of the framework for all knowledge related activities. The Network aims to undertake a couple of demonstration projects this year on the implementation and evaluation of its knowledge translation framework. If you have already begun or are in the process of initiating a knowledge sharing and exchange activity in the context of the CAN-NMD, such as the development of patient education material or clinical care guidelines, we would ask you to please get in touch with Gracia Mabaya at Gracia.Mabaya@lhsc.on.ca to use these as demonstration projects for the implementation.
Knowledge Translation Web-Based Platform
One of the exciting activities the Network hopes to achieve this year is the development of a knowledge translation web-based platform in the form of a Wiki. Through this platform, the Network hopes to help professionals to connect and exchange best practice experiences in relation to neuromuscular disease clinical care, research and education. Some of the features expected to appear on this platform include, but are not limited to, a personal user page for each user, a scientific resources database, the ability to upload and download documents, a forum/message board, a case of the week discussion section, as well as lunch & learns. If you have not been able to participate in the needs assessment preparing for the development of this web-platform, please feel free to do so here.
In addition, the Network’s Knowledge Broker will be conducting workshops with health care providers at various adult and pediatric neuromuscular clinics on the CAN-NMD’s objectives in general as well as specific knowledge brokering goals and objectives. Feedback will also be sought from healthcare providers on knowledge sharing and exchange activities that would be useful to them. The first round took place at Holland Bloorview Kids Rehabilitation Hospital and The Hospital for Sick Children (SickKids) in Toronto, Ontario on February 2nd, 2016. Sessions are being planned for Kingston, Ottawa, Montreal and Quebec City. If you would like to have a session held at your clinic or via videoconference in order to give your staff a broader understanding of the CAN-NMD and to promote their involvement, please contact Gracia Mabaya at Gracia.Mabaya@lhsc.on.ca.
We look forward to hearing your comments on knowledge brokering in general, the knowledge translation web-based platform, and on any other ideas you may have or may be looking forward to seeing the Network disseminate in the year 2016. Please post your comments and suggestions below.
 The Canadian Institutes of Health Research (CIHR) defines knowledge translation as a process consisting of the synthesis, dissemination, exchange and application of knowledge aiming to improve health outcomes through the delivery of effective health services and products and an overall strengthened healthcare system.
 The Canadian Institutes of Health Research (CIHR)’s Knowledge to Action (KTA) Framework (Straus, Tetroe, & Graham, 2013), the Global Knowledge Management Framework (Pawlowski & Bick, 2012), and Imagine Canada’s Effective Knowledge Transfer & Exchange For Nonprofit Organizations Framework (Zarinpoush, Von Sychowski, & Sperling, 2007).
RECOMMENDATIONS ON THE USE OF THE FLUMIST & RESPIRATORY SYNCYTIAL VIRUS VACCINES FOR NEUROMUSCULAR DISEASE PATIENTS
General considerations for neuromuscular disease patients
Neuromuscular disease (NMD) patients are considered to have compromised immune systems and this comes with both a greater risk for influenza infection alongside a greater risk for side effects and complications from influenza vaccination. Adults over the age of 65 and aboriginals are also considered to be at increased risk for influenza infection even if they are otherwise healthy. The most important thing a family can do to prevent influenza from entering their home and infecting a person with a neuromuscular disease is to get immunized against influenza virus. The entire family and all caregivers are strongly recommended to get influenza vaccine as soon as it is available.
What is the FluMist® vaccine?
Perhaps you have already come across this conversation in your respective practice this flu season. In any case, one of the issues we will be discussing in this edition of NeuromuscularNow is whether or not it is safe for your neuromuscular (NM) patients to take the live attenuated influenza vaccine (LAIV), also referred to as FluMist. FluMist is to be administered through the nose (intranasal administration). The 2015-2016 LAIV contains protective agents against all four flu viruses: an influenza A (H1N1) virus, an influenza A (H3N2) virus and two influenza B viruses. The LAIV vaccines are made from actual disease pathogens, which are weakened in the laboratory setting.
Recommendations for your neuromuscular disease patients
The statement on seasonal influenza vaccine 2015-2016 released by the Public Health Agency of Canada (PHAC) provides some recommendations on the use of the FluMist vaccine(1). Although LAIV is the recommended preferential vaccine for healthy children and adolescents 2 to 17 years of age, PHAC does not recommend its use for children, adolescents and adults (2-59 years of age) with immune compromising conditions.
Specific contraindications for the LAIV vaccine include:
PHAC also recommends that LAIV recipients avoid close contact with persons with severe immune compromising conditions for at least two weeks following vaccination, because of the risk for transmitting a vaccine virus and causing infection. Individuals caring for people with compromised immune systems and family members residing in the home should be vaccinated with inactivated vaccine.
The information above is taken from the Canadian Immunization Guide Chapter on Influenza and Statement on Seasonal Influenza Vaccine for 2015-2016 published by the Public Health Agency of Canada and is available at: http://www.phac-aspc.gc.ca/naci-ccni/flu-2015-grippe-eng.php
Administration of RSV vaccine for children with muscle disease: Not to be given to ANYONE outside of the first year of life
Another vaccine being discussed in this edition of NeuromusclarNow for your NM patients with a disease of the muscle is palivizumab (RSVAb). RSVAb is a humanized murine monoclonal immunoglobulin anti-respiratory syncytial virus antibody used to prevent respiratory syncytial virus (RSV) (2). This vaccine is produced by DNA technology and is made up of 95% human and 5% murine amino acid sequences (2,5).
RSV is the most common virus that can affect lungs and breathing tubes, and cause lower respiratory tract infection (LRTI) in infants and children (2,3,4). Severe RSV has an increased prevalence among infants with a neuromuscular condition due to risk factors such as an impaired ability to clear airway secretions, respiratory muscle weakness, a high prevalence of gastro-esophageal reflux and swallowing dysfunction leading to aspiration(2).
The Canadian RSV season usually begins in November or December, lasting for about four to five months (3). In addition to many other indications, the Canadian Immunization Guide published by PHAC recommends that children at risk for severe RSV receive RSVAb (5). It is under this category that you can request RSVAb prophylaxis for your NM patients. Children residing in Ontario may be eligible for reimbursement of vaccination costs for RSV prophylaxis (6).
The Canadian Paediatric Society (CPS) has recently conducted an analysis on the efficacy and cost effectiveness of palivizumab for preventing RSV(2). Randomized controlled trials have demonstrated a 40% efficacy of the vaccine in children with chronic lung disease (CLD) and 45% in those with congenital heart defect (CHD). In terms of cost effectiveness analysis, it was concluded that it is unlikely that palivizumab is cost-effective in reducing cost of hospitalizations for children with CLD or CHD and palivizumab’s cost effectiveness can only be evident in settings where RSV hospitalizations are exceedingly common and very expensive(5).
Health care providers must note that because the RSV infection does not confer protective immunity, for children who get infected with RSV while taking the RSVAb prophylaxis, the scheduled monthly dosage should be given throughout the RSV season.
For specific guidelines, please visit the references enclosed. We look forward to receiving your thoughts and comments in the discussion section below.
(1)Public Health Agency of Canada (2015). An Advisory Committee Statement (ACS) – National Advisory Committee on Immunization (NACI). Retrieved from http://www.phac-aspc.gc.ca/naci-ccni/flu-2015-grippe-eng.php
(2)Robinson,J. L., Le Saux, N. (2015). Canadian Paediatic Soceity, Infectious Diseases and Immunization Committee. Preventing hospitalizations for respiratory syncytial virus infection. Paediatric Child Health, 20(6), 321-326.
(3)Resch, B., Manzoni, P., Lanari, M. (2009). Sever respiratory syncytial virus (RSV) infection in infants with neuromuscular diseases and immune deficiency syndromes. Paediatric Respirartory Reviews, 10(3), 148-153. doi:10.1016/j.prrv.2009.06.003.
(4)Welliver, T. P., Garofalo, R. P., Hosakote, Y., Hintz, K. H., Avendano, L., Sanchez, K., Velozo, L., et al. (2007). Severe human lower respiratory tract illness caused by respiratory syncytial virus and influenza virus is characterized by the absence of pulmonary cytotoxic lymphocyte responses. Journal of Infectious Diseases, 2007(195), 1126-1136. doi: 10.1086/512615
(5)Public Health Agency of Canada. (2013). Canadian Immunization Guide. Part 5: Passive Immunizing Agents. Retrieved from http://www.phac-aspc.gc.ca/publicat/cig-gci/p05-01-eng.php#pass
(6)Government of Ontario: Ministry of Health and Long-Term Care. (2015). Re: 2015-2016 Season for Respiratory Syncytial Virus Prophylaxis for High Risk Infants. Retrieved from http://www.health.gov.on.ca/en/pro/programs/drugs/funded_drug/pdf/rsv_info.pdf
CAN-NMD ANNUAL MEETING UPDATE
On September 23 & 24, 2015 the 2nd annual CAN-NMD meeting was held in Canada’s Capital City of Ottawa.
Dr. Lawrence Korngut, Chair of the Network, kicked off Day 1 with opening remarks followed by a series of mini-talk presentations from many of our members, both old and new, providing research and clinical care updates from across the country in neuromuscular diseases.
The talks provided great insight into what our attending members have been working on in their field. Duchenne muscular dystrophy, Charcot-Marie Tooth disease, amyotrophic Lateral sclerosis (ALS or Lou Gehrig’s disease), familial amyloid polyneuropathy, and spinal muscular atrophy were all represented in the research updates including new diagnostic tools and potential new treatments. Clinical care was also well represented with talks on knowledge sharing, enhancing care, supporting youth in clinics, new training programs and multidisciplinary approaches to rare disease diagnosis and treatment.
A sneak peek of the upcoming Neuromuscular Nation virtual social community was presented and excited attendees were invited to test the site. Neuromuscular Nation aims to be a secure place where patients and families who may never otherwise have a chance to interact with each other can connect, share, and gather information.
Day 1 ended with the inaugural edition of National Neuromuscular Disease Rounds as a multidisciplinary team from Moncton, New Brunswick presented exceptional cases encountered recently. They highlighted a multidisciplinary approach of research, diagnostic, and clinical care teams working closely together which allowed them to diagnose and treat these cases with greater success. The Moncton team aims to improve diagnosis and treatment though the integration of Next Generation Sequencing (NGS) and development of biomarkers using mRNA and NGS. They are also conducting systematic studies on familial ALS and MS (Multiple Sclerosis) in communities where a “rare” disease occurs more “frequently” in order to better understand, diagnose and educate those communities. Video of the NMD Rounds will be available soon.
The morning of Day 2 focused on knowledge translation as the CAN-NMD’s national knowledge broker, Gracia Mabaya (based at the London Health Sciences Centre in London, Ontario) presented the Network’s knowledge translation framework. This framework will be used to advance knowledge sharing and exchange in neuromuscular research, clinical care and education across stakeholder groups represented within the Network, as well as between Canadian clinical care facilities to improve overall care, and facilitate more rapid uptake of new findings from research across all centers. Examples of how the framework could be applied were provided and there was a general consensus among members on the comprehensiveness, applicability and usefulness of the framework for future Network-specific knowledge sharing and exchange initiatives.
Representatives from 5 companies in the pharmaceutical industry were also in attendance on Day 2 and participated in a round table discussion sharing ideas around forging mutually beneficial outcomes in neuromuscular disease research and clinical care.
Meeting attendees spent the afternoon within their respective task force to review progress on activities undertaken in 2015 and looked ahead to plan the work to be accomplished in 2016. Each task force presented its yearly plan to the rest of the network before a representative from Muscular Dystrophy Canada joined Dr. Korngut for closing remarks on a successful meeting. The dates for next year’s annual meeting will be finalized in the next couple of months. Watch your inbox for an opportunity to provide input on the best timing.
About the Blog
Neuromuscular Now is the Canadian Neuromuscular Diseases Network (CAN-NMD) blog, intended to share the work of the Network broadly throughout Canada and beyond. We aim to provide new content twice per month including updates from the Network, as well as featured posts on noteworthy topics spanning the neuromuscular disease field from guest bloggers and Canada’s researchers and neuromuscular care providers. If you have a great idea for a topic you’d like to see please let us know!
Join the Network
Since its inaugural meeting in the picturesque town of Banff, Alberta, in October 2014, the Network has begun setting out its mandate and vision. During the first three years the Network aims to become a sustainable national entity consisting of all interested stakeholders across neuromuscular disease and contributing to the delivery of world class clinical care; the enhancement of capacity for world-class clinical and basic science research; and the creation of world-class educational opportunities in neuromuscular diseases.
The CAN-NMD welcomes academic, as well as community memberships. To become a member, please see the types of memberships offered by clicking here and click on the “BECOME A MEMBER” button below the page to register.
A glance at Network Activities!
Over the past year, the Network has been working to obtain an understanding of stakeholder needs through needs assessment and environmental scan surveys. You can provide your input here. The results collected from these surveys will enable the Network national staff to identify existing gaps and assets with respect to the national neuromuscular clinical care landscape, knowledge translation and research talent engagement.
The Network is preparing to launch a virtual social community “Neuromuscular Nation”, which will be a great collaborative platform uniting patients, families, and caregivers, from across Canada and facilitating new relationships and sharing of experiences. Specific features of the portal will include a personal profile, live chat, secure mailbox, knowledge repository and research notifications from the Canadian Neuromuscular Disease Registry (CNDR)… and many more! Stay tuned for more information on the launch of the Neuromuscular Nation.
To date, the CAN-NMD has been presented at various conferences, namely the “Colloque interdisciplinaire sur les maladies neuromusculaires et la sclérose laterale amyotrophique” in Laval, QC on April 16th, 2015; at the rehabilitation clinic network (IRDPQ) in Quebec City on May 15th, and at the International Myotonic Dystrophy Consortium (IDMC) in Paris, France on June 9, 2015. On September 20, 2015, CAN-NMD will be showcased at the Muscle Study Group Society Scientific Meeting in Snowbird, Utah.
The Network has also begun work on tools for scientists and clinicians working in translational research; a knowledge translation framework and an education framework.
Upcoming Annual Meeting
The Network will hold its annual meeting in the heart of the nation’s capital, at the Westin Ottawa Convention Centre from September 23-24, 2015. Look for updates from that meeting on our next blog post.
Whether you are a neuromuscular disease patient, a parent or caregiver, a health care provider, a researcher, a student or simply one who is passionate about neuromuscular diseases, your feedback is highly important to us! We look forward to receiving your comments in the area below, or here.