Successful first annual meeting in Bologna!

Representatives of our 11 partners gathered in Bologna for the first annual meeting, 29-30th October.

What happens you combine 24 scientists, clinicians, clinical trial officers, charity workers and professional organisers with the finest tagliatelle al Bolognese ever tasted? Our first annual meeting in Bologna of course!

On Monday 29th and Tuesday 30th October, representatives from MCDS-Therapy’s 11 partners descended on the city’s Instituto Ortopedico Rizzoli (the base of partner number six) to give updates each other’s progress and discuss the best approaches to particular challenges.

The meeting was kicked off by our Coordinator, Prof Mike Briggs, who welcomed everyone and detailed our exciting agenda. We discussed every aspect of the trial, including sponsor requirements, patient identification and recruitment, safety reporting, clinical trial monitoring, health economics, biostatistics and data management, biomarker discovery, communication and dissemination, and coordination and management. Phew – it’s a good job Italy is also known for great coffee!

We would like to say a huge thank you to Finovatis’ Maud, David and Elodie for organising such a productive meeting, to Luca at the Rizzoli Institute for hosting us, and of course to all the representatives who came along. We’re looking forward to collaborating further as the trial progresses.

If you would like to find out more about each of the 11 partners and their roles in the trial, head over to our Partners page.

Why are we doing biomarker discovery?

What is a biomarker and why is the MCDS-Therapy team trying to find one? Sciomics, the partner focusing on this part of our work, have written this handy blog to keep you in-the-know.

Having a rare disease is challenging for patients and their loved ones as the diagnosis is often delayed or inaccurate, specialists are scarce and even upon being successfully diagnosed a treatment may not be readily available.

The whole Sciomics team is very grateful to be part of the MCDS Therapy project having the aim of repurposing a safe and proven drug to treat Metaphyseal Chondrodysplasia, Type Schmid (MCDS). Important questions are still to be answered, does the drug work in every child, do the children respond quickly or are there adverse events to be expected?

How can we try to answer these questions? We need biomarker. A biomarker is something we can measure in order to be able to say the drug works or does not. An unrelated example for a biomarker is measuring your temperature if you have a cold. Is it elevated you are likely to be sick or if it is normal you are probably not. Here at Sciomics we measure more than 1000 proteins from a blood sample to identify different proteins as biomarkers potentially being able to judge whether the new therapy is working or has only little to no positive effect.

This is especially important for MCDS as the only biomarker so far is growth rate which can be very slow for natural reasons during a certain period of time or caused by the disease. Children need to be monitored for years until it can be concluded whether the drug has an effect or not. Having a simple biomarker test which can indicate a response to the drug quickly, maybe even after a few weeks, using an easily obtainable blood sample would be of great value to the children and their parents.

We at Sciomics can investigate proteins in tiny blood samples in order to find such biomarkers. For current diagnostic measurements 10ml of blood are taken to look at a handful of parameters some of them already being proteins. Our technology can measure more than 1000 proteins using a thousand times less blood while giving us a hundred times more information.

Looking at very small blood samples has many advantages, the sample is almost pain-free to obtain, every patient can give a tiny amount of blood even sick children and it minimises the children’s risk to suffer from any negative events due to the test. In order to be able to look at 1000 and more proteins, usually your Doctor only looks at 5-10 proteins, we had to invent and develop a whole new method. This was done over more than 15 years of research and out of the 12 people working at Sciomics many are scientists.

We hope that our research within the MCDS Therapy consortium will have a positive impact on many children’s and their family’s lives.

MCDS presented for the first time

Professor Mike Briggs presented the work behind MCDS-Therapy at Findacure’s Drug Repurposing for Rare Diseases conference.

As an academic-led, industry-free drug repurposing project for an ultra-rare disease, the MCDS-Therapy team is keen to spread the word of our work as far and wide as possible. Professor Mike Briggs, Project Coordinator, was invited to do just that at Findacure’s Drug Repurposing for Rare Diseases conference on 27th February 2018.

The conference was organised to showcase the excellent drug repurposing work being done across Europe in rare diseases. It was a perfect way to present the MCDS-Therapy project for the first time, and to announce receipt of funding from the European Union’s Horizon 2020 research grant. Over 100 people attended despite the snowy weather, including Dr Melita Irving who will be leading the MCDS-Project at the Guy’s and St Thomas’ Hospital trial site.

The conference was held in celebration of International Rare Disease Day, and gave Mike the chance to share our plans for MCDS-Therapy, and the journey to receive funding, with other academics, clinicians, patient groups, and life scientists.

Findacure, the organisation that arranged the conference, is another of the 11 MCDS partners. They are responsible for patient engagement, communication, and dissemination. You can find out more about the event by reading their conference blog. Alternatively you can listen to Mike’s talk in the video below.

Kick-off meeting

The 11 partners of MCDS-Therapy met in Brussels to officially launch the project.

The MCDS-Therapy project is off to a great start. Having been awarded a grant of €5,700,000 from the European Union’s Horizon 2020 fund in December 2017, 10 of the 11 partners involved met in Brussels for a kick-off meeting in February 2018.

The meeting was a chance for all the partners to meet face-to-face for the first time, to learn about what each other does, to go over the overall plan for the MCDS-Therapy, and to discuss the next steps that need to be taken. Each partner gave a presentation to the rest of the consortium to explain their own aims, plans and expected outcomes for the project.

We were pleased to welcome Iiro Eerola from the European Commission, who gave an overview of the Horizon 2020 grant and the EC’s work and focus on rare diseases. This was followed by number of productive discussions about the overall trial design, and data analysis, which will help to ensure that the trial is in a great place when launched later in the year.

A number of smaller break out meetings were planned and all of the partners left with a clear sense of the road ahead, and the work they need to focus on over the next few months.

H2020 grant success!

The MCDS-Therapy project has been awarded €5,700,000 from the European Union’s Horizon 2020 fund.

The MCDS-Therapy project has received the fantastic news that our application to the European Union’s Horizon 2020 fund has been successful. An award of €5,700,000 was confirmed in December 2017, and was met with delight by all our global partners. The grant was awarded from H2020’s ‘SC1-PM-08-2017 New Therapies for Rare Diseases’ call for projects, which is part of their Research and Innovation action.

The grant will be used to fund the observational study and clinical trial in all trial sites, biomarker discovery research, health economic research, patient engagement, and public and patient engagement for the MCDS-Therapy project. We are delighted that over 20 years of hard work in pre-clinical research, as well as the potential of the project to deliver an effective treatment to patients, was recognised by the European Union grant. Of the 127 projects submitted to this particular H2020 call, only 10, including MCDS-Therapy, were funded. Without this funding, the multi-site and in-depth research we had planned would not have been able to take place.

We would like to say a huge thank you to the Horizon 2020 team for their financial support, and to everyone involved in our application. The whole consortium is excited to get started.