At the initiative of IMP a scientific workshop on potentially harmful drugs for mitochondrial patients was organised. It took place in Amsterdam on the 1st and 2nd of November. Mitochondrial specialists/clinicians and pharmacologists from all over the world came together to reach consensus about drugs which may or may not be safe to use by mitochondrial patients, children and adults.

 

At this moment IMP publishes a list of potentially harmful drugs on the website. This list needs to be updated on the basis of new and existing scientific evidence and clinical experience. The entire workshop was sponsored by patient organisations: AEPMI, DGM, Eurordis, IMP, the Lily Foundation, MitoCanada, Mitocon and Muscular Dystrophy UK. They strongly believe that thorough and trustworthy information on drugs is essential for mitochondrial patients.

The group of experts carried out extensive research into existing scientific literature beforehand. At the workshop they discussed safety of all relevant drugs with the purpose of reaching consensus. The two-day workshop was hard work but the complete list of drugs was reviewed and their safety was discussed. The experts reached consensus on all of them. In the coming weeks they will work on a solid scientific document, to be published open access. The following step will be to develop a new table for patients and their families on the basis of the scientific document. There is still much work to do but all parties are dedicated to complete this important task.

We will keep you informed!

Stealth Biotherapeutics is a pharmaceutical company focussing on the development of drugs against mitochondrial diseases and many common age-related diseases. The company is currently conducting a phase III clinical trial called MMPOWER-3. It concerns a randomized trial to evaluate the efficacy and safety of elamipretide in patients with Primary Mitochondrial Myopathy. This is followed by an open-label treatment extension with elamipretide.

Once a drug has proved satisfactory after phase III trials, the regulatory authorities (FDA and EMA) will review everything and, it is hoped, give approval to market the drug so that it becomes available for patients. In Europe, after EMA’s approval, each individual country carries out its own review and then decides on approval for the national market.

The drug elamipretide will be administered through daily subcutaneous (under the skin) injections. Elamipretide is an investigational drug that associates with cardiolipin, a key structural component of the inner mitochondrial membrane, and has shown to improve mitochondrial function in preclinical and early clinical studies. 

The trial will assess approximately 200 patients, aged 16-80, across North America and Europe.

Visit  https://www.clinicaltrials.gov/ for a list of trial sites.

Patients that have a genetic confirmation of mitochondrial disease are potentially eligible for participation in MMPOWER-3.

The clinical trial investigator will identify potential patients.

At this time Stealth is providing access to elamipretide only through clinical trials.

For more information:

Website: https://www.stealthbt.com/ or https://mitotrials.com/

Protocol (pdf): MMPOWER-3 protocol for patient advocacy groups

For contact:

Twitter: @stealthbt

Email: This email address is being protected from spambots. You need JavaScript enabled to view it.

 

 

 

 

 

 

  

Canada: Mark your calendars!

In the Mitochondrial Disease Awareness Week MitoCanada has planned a number of exciting fundraising and awareness activities. There truly is something for everyone; MitoCanada invites you to join along!

The funds raised with these activities will support transformational research and MitoCanada's advocacy efforts.

Joining is easy. Simply browse through the evens and select one or two that best suits you. You can also create your own mito awareness event and MitoCanada will help you get started. 

Inspired? Go the website here here

Global Mitochondrial Disease Awareness Week

Around the world Global Mitochondrial Disease Awareness Week is marked with educational, fundraising and advocacy efforts designed to raise awareness about mitochondrial disease (Mito). Every year IMP members around the world are working together to raise awareness for mitochondrial disorders, by setting up special events that week to raise awareness. For additional information and to get involved you can take a look at our special website http://gmdaw.org/ or on the special Facebook page https://www.facebook.com/GlobalMitochondrialDiseaseAwarenessWeek The GMDAW website will also inform you about any national events taking place in your country.

Light Up for Mito 

 #lightupformito

Light Up for Mito is a global campaign hosted by International Mito Patients (IMP) involving illuminating landmarks in green to raise awareness of mitochondrial disease (Mito). This year on 22 September 2018, IMP members are collaborating to light up landmarks around the world. 

How you can get involved:

Help us break the world record! You can help us make Light Up for Mito even bigger this year by organising a monument to be lit up green in your area. Please contact This email address is being protected from spambots. You need JavaScript enabled to view it. to get involved

Install a green light bulb in your porch or outside your home or office and light up for mito. Share a photo on social media using #lightupformito or email This email address is being protected from spambots. You need JavaScript enabled to view it. to have your photo included on the website

Change your profile picture on your social media accounts to the green mito ribbon to help raise awareness

Senate Committee recommends laws change so Australian babies don’t suffer mitochondrial disease

Australian women at risk of having babies suffering severe mitochondrial disease may soon be able to have healthy children, following last night’s Senate Community Affairs References Committee recommendation to move towards legalising mitochondrial donation, a ground-breaking IVF technique that replaces energy-generating DNA in the mother’s egg with donor material.

The Committee’s report has been welcomed by the Australian Mitochondrial Disease Foundation (AMDF), the peak body supporting Australians with mitochondrial disease, a serious and potentially fatal genetic disorder that starves the body’s major organs of energy.

AMDF Chairman Dr Doug Lingard thanked the Senators for their hard work inquiring into mitochondrial donation, and called on the Australian Government to act on the Committee’s recommendations as soon as possible.

“At least 60 Australian babies each year could be prevented from suffering severely disabling and potentially fatal forms of mitochondrial disease if mitochondrial donation was available here. It offers the first practical hope for future generations to live free of maternally inherited mitochondrial disease,” Dr Lingard said.

“In the AMDF’s experience engaging with the Australian public, politicians and other stakeholders, including a Citizens’ Jury in 2017, mitochondrial donation receives overwhelming support when people understand the procedure and its ramifications.”

“By acting promptly to change our laws, Australia could become the second country in the world to establish a regulated system to provide mitochondrial donation to families affected by this devastating disease,” Dr Lingard said.

“After ten years of scientific research, ethical review and consultation, in 2015 the UK made pioneering legislative changes to allow mitochondrial donation; these were endorsed in 2016 by the Human Fertilisation and Embryology Authority, and the first clinic and patient licences were issued in 2017.

“The Committee clearly recognises that the UK’s strict regulatory system for mitochondrial donation provides a sound basis for Australia, with only minor changes likely to be required to reflect our local context.

“The AMDF supports the pathway towards legislation recommended by the Committee.

“We stand ready to help in the public consultation process and urge the Australian Government to seek the advice of the National Health and Medical Research Council as soon as possible,” Dr Lingard said.

 

Background

Mitochondrial donation involves replacing the faulty mitochondrial DNA in the mother’s egg (0.1% of its genetic material) with healthy donor mitochondrial DNA so the resulting baby will not suffer mitochondrial disease.

The mother’s and father’s nuclear DNA contributes more than 20,000 genes or 99.9 per cent of the baby’s genetic make-up and determines its appearance, intelligence, behaviour and other personal characteristics. The 0.1 per cent contribution (37 genes) from the donor egg means the resulting baby’s cells can effectively convert food and oxygen into the energy needed to power its organs.

Depending on which parts of their bodies are most affected and to what extent, people with mitochondrial disease can lose their sight or hearing, be unable to walk, eat or talk normally, have strokes or seizures, develop liver disease or diabetes, suffer cardiac, respiratory or digestive problems, or experience developmental delays or intellectual disability.

 

For interviews with the AMDF, experts and affected families, please contact:

Carol Moore, Moore Public Relations

02 9560 2826, 0402 382 363, This email address is being protected from spambots. You need JavaScript enabled to view it.  

 

For further media information:

·         AMDF Media Resources Dropbox: https://tinyurl.com/AMDFmedia

·         Senate Standing Committee on Community Affairs Science of mitochondrial donation and related matters https://www.aph.gov.au/Parliamentary_Business/Committees/Senate/Community_Affairs/MitochondrialDonation

·         Scimex Expert Background Briefing: https://www.scimex.org/newsfeed/background-briefing-senate-to-report-on-allowing-three-person-ivf-to-treat-mitochondrial-disease  

           A recording of the 15 June briefing is in the Attachments at the bottom of the page, featuring:

-       Professor Carolyn Sue: Professor and Director of Mitochondrial Research, Royal North Shore Hospital, Kolling Institute, University of Sydney; Founding Director, Australian Mitochondrial Disease Foundation; Co-Chair, AMDF Scientific & Medical Advisory Panel.

-       Professor David Thorburn: Head, Mitochondrial Research Group, Murdoch Childrens Research Institute; Co-Chair, AMDF Scientific & Medical Advisory Panel

-       Dr Ainsley Newson: Associate Professor of Bioethics and Deputy Director, Sydney Health Ethics, The University of Sydney

 

© 2018 Int. Mito Patients