Archive news 2017
IMP sends its season's greetings to all of you, wherever you are! We wish you a happy time with your loved ones.
We are looking back on a very pleasant and successfull cooperation with many of you and are looking forward to do so again in the next year. Thank you for your support and involvement in our fight against mitochondrial disease.
In the meantime have a good time with your family and friends and enjoy! We wish you a happy and loving 2018!
Please find below the official pressrelease by Khondrion
Khondrion presents phase II KHENERGY trial data
Data supporting Phase III development of KH176 in mitochondrial disease
Nijmegen, The Netherlands - Khondrion, a leading clinical-stage pharmaceutical company focussing on small molecule therapeutic for mitochondrial diseases, today (22.11.2017) announced results from its KHENERGY study, a Phase II exploratory trial with oral KH176 in the m.3243A>G multisystem mitochondrial MELAS and MIDD syndromes and mixed phenotypes. The results of the trial were presented by Prof. Jan Smeitink, Khondrion's CEO, at the Dutch Life Sciences Conference.
This year, as in the last few years, the Global Mitochondrial Disease Awareness Week was held, in the third week of September. The GMDAW is focussed on creating awareness, education, fundraising and advocacy efforts with respect to mitochondrial disease all around the world. In many countries national events were organised with this aim. This varied from walking- and biking tours to raise funds, to patient information days, and general information campaigns.
For 2017 IMP wanted to set the GMDAW globally on the map by beating the world record of lighting out the most monuments/landmarks ever. Of course in green light, the colour which represents the disease best. The Guinness World Record was held with 38 monuments. The goal for IMP was to light out 55 monuments for 24 hours worldwide in the night of the 16th of September. The number 55 was chosen because it was 55 years ago that the first mitochondrial diagnosis was set.
The search for a cure for mitochondrial disorders is of the essence. Nobody will deny this.
However, we all know that scientific developments take time and the process of approval of drugs is a lengthy one. And even when a drug/cure is on the market, it is likely that not all mitochondrial patients will benefit from it. Mitochondrial disease concerns a group of complex, hetergenous disorders.
In the meantime mitochondrial patients have to live their daily lives. Therefore, the quality of life and the concurring carepaths require our full attention. It is for this reason that IMP initiated a full study into the quality of life of mitochondrial patients. If we know and understand the daily situation of patients and have analysed the most important complaints which affect the quality of life, we can stimulate the research into developing good carepaths with proper therapies. In the end we wish that patients can be in control of their daily lives and be empowered in such a way that they can manage their own situation.
In the world of mitochondrial medicine many topics are being discussed and many issues are to be resolved. If these are purely medical, it is obvious that they must be dealt with by the medical (and other) specialists. Yet, there are also issues which should be dealt with in a broader circle, with patient representatives, other scientists, regulatory authorities (EMA, FDA) or with representatives from pharmaceutical industry.
Therefore, IMP has taken the initiative to organise and (partly) fund international workshops to deal with different issues. Main goal is to have all relevant stakeholders together so that different subjects can be discussed, consensus can be achieved, and the path to a therapy/cure will be smoother.
Last year in November in Rome the first workshop took place. This was done with the great support of a select group of academic researchers. Key opinion leaders from the academic world, all involved in mitochondrial disease, and representatives from different patient advocacy groups met. The aim was to achieve consensus on a list of outcome measures for trials on primary mitochondrial myopathy. Consensus on this is essential for the process of having trials and drug development approved by FDA and EMA. This would speed up the whole process. And after a couple of days of hard work and additional work by four KOL'S a consensus list was achieved. The list will serve as guidance for researchers organising trials for primary mitochondrial myopathy.
The academic researchers are now planning another comparable workshop, focussing on another type of mitochondrial disease.
A second workshop took place in March this year in Amsterdam. This time also representatives from (7) pharmaceutical companies participated. The aim was to share knowledge and experiences with organising research studies and trials and with the FDA and EMA. All participants were open in sharing and eager to learn from each other. It is unique that within a disease community all stakeholders meet and discussions take place in such an open atmosphere. The added value of such workshops became clear and the need for more was expressed.
As it is in the interest of mitochondrial patients that such meetings take place with the aims of learning from each other, having in-depth discussions, achieving consensus, and as such speeding up the path to a cure. Therefore, IMP will continue with its efforts to organise these.
Last June the annual member meeting of IMP took place in Cologne. Coinciding with the Euromit2017 conference this was a fantastic opportunity to combine both.
For years Euromit has been a place to meet for researchers, clinicians, care professionals and patient representatives, all involved in mitochondrial disease. The medical programme was attended by dozens of young researchers who wish to specialize in mitochondrial disease. This makes us very hopeful for the future. As such the entire conference breathed optimism and positive energy.
During the IMP annual member meeting a new board had to be elected. All five sitting board members were re-elected for another period of 3 years and the board was reinforced with a sixth member: Philip E. Yeske, Ph.D, Science & Alliance Officer of the United Mitochondrial Disease Foundation (USA).
Additionally, the Scientific Committee of IMP which reviews applications from researchers for endorsement/support by IMP, was reinforced with a fourth member: Alison Maguire, BSc FCA, Head of Research of the Lily Foundation (UK).
Next year's annual member meeting will take place in Cambridge, UK, directly after the Mitochondrial Medicine Conference in May.
Each year, at the third week of September, the Global Mitochondrial Disease Awareness Week is held. The Global Mitochondrial Disease Awareness Week (GMDAW) is marked by awareness, education, fundraising and advocacy efforts to raise awareness about mitochondrial diseases (mito) all around the world. Many events are organised on a national level, but this 'light up' campaign is an international event.
This year the international mito community wants to set the Global Mitochondrial Disease Awareness Week globally on the map by beating the world record of lighting out the most monuments/landmarks ever. Of course in green light, the colour which represents the disease best. The current Guinness World Record for most monuments lighted out (24 hours) is held by Estee Lauder for Breast Cancer Awareness with 38 monuments. The goal for us is to light out 55 monuments for 24 hours worldwide on Saturday/Sunday September 16th-17th 2017. 55 monuments, because in 2017 it is 55 years ago that the first mitochondrial diagnosis was set.
The Guinness World Record organisation has officially recognised the attempt to break the record. And there is a big chance that we are going to make it! There still are many things to organise and communicate but we are getting there.
Watch the world turn green on the 16th of September!
Questions?
Maureen Latocki (Canada) This email address is being protected from spambots. You need JavaScript enabled to view it.
Rebecca Davis (Australia) This email address is being protected from spambots. You need JavaScript enabled to view it.
Alfons Heetjans (Netherlands) This email address is being protected from spambots. You need JavaScript enabled to view it.
Are you interested in the latest scientific developments and research studies regarding mitochondrial disorders? If so, the patient meeting at the international medical congress, Euromit2017, is the place to be. The programme, especially developed for patients and their families, is taking place on Wednesday the 14th and Thursday the 15th of June (two half days), in Cologne, Germany. Presentations are given by leading researchers specialised in mitochondrial disease from all over the world. The programme also offers a great opportunity for patients to ask questions to these specialists. The main language is English but the presentations will also be translated simultaneously in German (headphones). And last but not least the patient meeting is an excellent event to meet other mitochondrial patients.
Each year, at the third week of September, the Global Mitochondrial Disease Awareness Week is held. The Global Mitochondrial Disease Awareness Week (GMDAW) is marked by awareness, education, fundraising and advocacy efforts to raise awareness about mitochondrial diseases (mito) all around the world.
This year we want to set the Global Mitochondrial Disease Awareness Week globally on the map by beating the world record of lighting out the most monuments/landmarks ever. Of course in green light, the colour which represents the disease best. The current Guinness World Record for most monuments lighted out (24 hours) is held by Estee Lauder for Breast Cancer Awareness with 38 monuments. The goal for us is to light out 55 monuments for 24 hours worldwide on Saturday/Sunday September 16th-17th 2017. 55 monuments, because in 2017 it’s 55 years ago that the first Mitochondrial diagnosis was set.
Available online, the official Rare Disease Day poster, is out now! Based on the theme of RESEARCH, the poster embraces the possibilities that research can bring, a bright and hopeful future. The visual also emphasizes the important role that everyone has to play in furthering rare disease research. Let's raise more awareness of rare diseases than ever before, by sharing the poster on all the social media channels and websites that we can!
For the whole information package on Rare Disease Day you can go to http://www.rarediseaseday.org/downloads
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