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25th April 2014
 
Unique opportunity to find out about TACT at ECRD 2014

To date the TREAT-NMD Advisory Committee for Therapeutics (TACT) has held 9 review meetings across Europe and the US, and has reviewed a total of 24 program applications for neuromuscular diseases. The goal of each TACT review is to help the sponsor to position the candidate compound along a realistic and well-informed plan to clinical trials, and eventual registration. The reviews and subsequent recommendations are focussed on generating meaningful and rigorous data that can enable clear go/no-go decisions and facilitate longer term funding or partnering opportunities. The TACT review process thereby acts to comment on viability, de-risking the process of proceeding on a development programme.

At this year’s ECRD 2014: The European Conference on Rare Diseases & Orphan Products, TACT Chair, Dominic Wells, and Core Group Committee member, Cristina Csimma, will co-lead a tutorial, Supporting the pathway to trials for rare diseases: clinical trial design and other considerations. During this tutorial Dominic and Cristina will share their experiences in advising on the drug development pathway for neuromuscular diseases and shall explain how this could be exported as a model for other rare diseases. Attendees will gain a greater appreciation of how TACT reviews act as a constructively critical and educational resource for researchers and drug development sponsors evaluating research programs with a translational goal toward entry into clinical trials in neuromuscular diseases. During this tutorial, Professor Ralf-Dieter Hilgers, IDeAL coordinator, will also discuss clinical trial design for small sample population groups.

Pre-conference tutorials will take place on Thursday 8 May, 2014 from 14:30-17:00.

Registration details can be found here.

 
 
 
European Medicines Agency launches
adaptive licensing pilot project
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The adaptive licensing pilot launched by the European Medicines Agency (EMA) aims to improve timely access for patients to new medicines. This approach, which is also known as staggered approval or progressive licensing, is part of the Agency’s efforts to improve timely access for patients to new medicines.

The proposed process, starts with the early authorisation of a medicine in a restricted patient population and is followed by iterative phases of evidence gathering and adaptations of the marketing authorisation to expand access to the medicine to broader patient populations.

As a holistic approach, adaptive licensing requires the involvement of all stakeholders who have a role in determining patient access, including the EMA, industry, health technology assessment (HTA) bodies, organisations issuing clinical treatment guidelines and patient organisations. All discussions will take place in a ‘safe harbour’ environment to allow free exploration of the strengths and weaknesses of all options for development, assessment, licensing, reimbursement, monitoring, and utilisation pathways in a confidential manner and without commitment from either side.

Further information about this pilot project can be found on the EMA website.

 
 
 
 
Recruitment complete for dysferlinopathy study
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We are delighted to announce that 193 patients have been recruited into the Clinical Outcome Study for Dysferlinopathy (LGMD2B/Miyoshi), exceeding the original goal of 150 patients.

The study will evaluate a large group of dysferlinopathy patients to determine which tests are best at measuring changes in the disease. The goals are to:
-  Define the best tests for measuring disease progression for use in clinical trials.
-  Collect biological samples for the identification of disease markers that are needed to non-invasively monitor the disease during clinical trials.
-  Collect accurate details about the full clinical spectrum of the different forms of dysferlinopathy by measuring disease progression. This information will help enhance patient care.

The outcome measures identified in this study are needed for the development and success of future clinical trials. In addition, the information gained in this study will provide a better understanding of the disease, ensure optimal care and treatment, and will lead to new and better therapies.

Co-leading this study are Kate Bushby from Newcastle University and Laura Rufibach from the Jain Foundation.

Find out more about the study which is funded by the Jain Foundation.

 
 
 
 
New stretching videos for Duchenne muscular dystrophy
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A comprehensive series of stretching videos for those affected by Duchenne muscular dystrophy have been developed by therapist Tina Duong and are now available to view on the Cooperative International Neuromuscular Research Group (CINRG) website.

The Stretch Out videos are in two parts, the first provides detailed instructions for stretching the muscles commonly affected by DMD whilst the second gives individuals a chance to actively perform the stretches along with the boys in the video.

Tina, along with an amazing group of boys, features in this fun filled educational video.

Please ensure that you understand how to correctly perform these stretches before beginning the second section of the video.

Always remember to consult with your medical team and physiotherapist before starting any new exercise routine.

Please visit the CINRG website to view the videos.

 
 
 
 
NeurOmics patient newsletter now available
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The latest annual patient newsletter for the NeurOmics project is now available for download.

This EU-funded research project addresses ten rare neurodegenerative and neuromuscular disorders. Next generation whole-exome sequencing and detailed clinical information is now being combined and used to improve the diagnoses and understanding of these diseases with an aim to developing future therapies - contributing to the IRDiRC goals of delivering 200 new therapies for rare diseases by 2020.

This current newsletter focuses on the project's achievements in its first year of operation and explains some of the techniques used to analyse the samples donated by patients. It also touches upon a number of new genes that have been discovered as a result of the project, gives an update on biomarkers and concludes with a look to what the coming months will hold for the project.

Download the newsletter from the NeurOmics website.

 
 
 
 
European Commission publishes report on Advanced
Therapy Medicinal Products Regulation
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Advanced therapies (gene therapy, somatic cell and tissue engineered products) have the potential to bring major benefits to patients. That said, there are still many unknowns so it is important to have appropriate controls in place to prevent any possible detrimental effects of those therapies.

The Advanced Therapy Medicinal Products (ATPM) Regulation was developed in 2007 to protect patients. It requires experts across the EU carry out an independent review of the product in question and high standards of quality, efficacy and safety must be evident before the product is made available to patients.

Care must be taken to ensure that the process does not have detrimental consequences for public health but it should not hinder the emergence of potential treatments. Regulation in this area, whilst continually ensuring public health, should not only create conditions that facilitate the appearance of new medicinal products but also rapidly adapt to scientific advances.

Based on experiences gathered since the Regulation was implemented in 2008 the European Commission has adopted a report (based on a public consultation). This report looks not only at the impact this has had on advanced therapies but also considers the future and the areas where the process can be improved.

Further information about advanced therapies can also be found on the European Commission website.

Download the full report here.

 
 
 
 
OPTIMISTIC myotonic dystrophy study: recruitment begins
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Recruitment is now open in Newcastle and Munich for this observational trial in myotonic dystrophy type 1. Sites in Nijmegen and Paris are undergoing final preparations and will begin recruitment soon.

The study aims to increase activity and improve quality of life through a tailored cognitive behavioural intervention. It is a randomised controlled trial with the control arm receiving standard care. Nearly 300 patients will be involved in total across the four sites.

This is the first international trial with intervention for this population and as well as assessing the effectiveness of the behavioural therapy it will also collect samples for genetic and biomarker analysis; increasing our understanding about all aspects of this condition.

For more information including who to contact if you are interested in taking part please visit the project website www.optimistic-dm.eu

 
 
 
 
Research grant opportunity for nemaline myopathy announced
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A Foundation Building Strength for Nemaline Myopathy (AFBS) has announced its "Building Muscle Initiative" research grant opportunity.

AFBS will fund up to $1 Million (US) towards this grant to find treatments for Nemaline Myopathy (NM). The goal of the grant is to advance understanding of the pathogenesis of NM in order to design and optimize treatment and management strategies.

The application deadline is 1 June 2014 at 5:00pm (Pacific Standard Time).

 
 
 
 
Candidates for 9th TACT review meeting in Berlin announced
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TACT is pleased to announce that the following applications will be reviewed at the TACT meeting scheduled for 10 May in Berlin, Germany:

-  Richard Franklin, Tarix Orphan LLC, MA, Cambridge, USA: TXA127, pharmaceutical formulation of angiotensin (1-7)

-  Mimoun Azzouz, Sheffield Institute for Translational Neuroscience (SITraN), University of Sheffield: Viral vector containing DNA coding for the human SMN protein; for treatment of 5q spinal muscular atrophy

Following the face-to-face review meeting, TACT will generate a detailed report with program development recommendations to the applicant within 6 weeks.  A non‐confidential report summary, developed in collaboration with the applicant, will be available via the TREAT‐NMD website within 8 weeks following the meeting.

The next TACT review meeting will be held in Chicago, USA 25-26 October 2014.  Individuals interested in submitting an application should send an expression of interest to the TACT secretariat Louise Johnston.

 
 
 
 
Medical Research Council PhD studentship -
myotonic dystrophy opportunity
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Applications for a PhD studentship in Glasgow working on defining the cis- and trans-genetic modifiers of disease severity and treatment response as an approach to patient stratification in myotonic dystrophy type 1 are now being taken.

The successful applicant will be expected to start on 1 October 2014 and will be supervised by Professor Darren Monckton and Dr Graham Hamilton.

Closing date for applications is Friday 2 May 2014.

For those interested, further details of the studentship can be found on the TREAT-NMD website. Alternatively please contact Darren Monckton directly.

 
 
 
 
World Muscle Society Congress 2014 -
early bird registration closing soon
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Early bird registration for this year's World Muscle Society Congress will close on 30 April.

The 19th congress will take place in Berlin, Germany from 7-11 October and is the premier annual international Congress on neuromuscular disorders.

This year´s focus will be on Protein Aggregation, Autophagy and Proteomics, Limb-Girdle Muscular Dystrophies (LGMDs) and, as usual, on the Advances in Treatment of Neuromuscular Disorders.

The dedicated congress website has details of the preliminary programme, information about registering along with further information to help you make the most of your time in Berlin.

 
 
 
 
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25th April 2014
TREAT-NMD newsletter - 25th April 2014
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