MCDS-Therapy and Covid-19

Due to the current COVID-19 situation, we have had to make the difficult decision to halt the MCDS-Therapy study. This means that we are stopping study visits for the foreseeable future and all activity relating to the trial.

Safety is always our top priority and we feel that halting the trial, before participants receive any of the study treatment, is the best decision.

We do not know how long the study will be halted as the COVID-19 situation is changing daily.  We cannot gauge how this will affect when we can restart the study. We will be posting updates here, on the MCDS-Therapy website, and on our social media accounts.

We recommend that any participants in the trial with concerns or seeking further information should get in touch directly with the local study doctor. Anyone else should feel free to contact us if you have any questions. The team will do everything they can to respond to you; however do be aware that many NHS workers who are part of the trial have been redeployed to assist with the COVID-19 response.

Thank you for your interest and support for MCDS-Therapy. While the halt of the trial is disappointing, we will be back and moving MCDS research forward again as soon as it is safe to do so.

Productive second annual meeting in Oslo

On Tuesday 10th September, the MCDS-Therapy consortium descended on Oslo, Norway, for our second annual meeting. Although Norway is not a partner in our project, the location was ideal because the International Skeletal Dysplasia Society annual meeting (ISDS), which most of our partners were attending, was held there directly afterwards.

The purpose of our meeting was to bring all the project’s partners under the same roof to provide updates on progress and discuss the next steps. After a delicious working meal the night before, the consortium gathered bright and early at the city’s Hotel Bristol. The meeting started with a welcome from Deputy Coordinator Michael Wright and was shortly followed by training on trial assessments, protocol, and pharmacy. The Newcastle Clinical Trials Unit and Sponsor representatives then met independently with each international trial site to discuss their own processes and needs.

Following an incredible buffet lunch, the consortium set about the afternoon’s activities. This included updates from non-clinical partners, including Findacure, who are working on communications, Sciomics, who are leading the exploratory biomarker discovery research, and Finovatis, who are doing a fantastic job of managing the project as a whole.

The meeting was a great opportunity to put names to faces and proved to be very productive. A big shout out must go to the Murdoch Children’s Research Institute representatives who travelled all the way over from Melbourne, Australia, for the meeting!

The MCDS-Therapy clinical trial is now open!

We are delighted to announce that MCDS-Therapy is now recruiting patients in the UK. We know that many people will have questions about the MCDS-Therapy clinical trial, and we have tried to answer the big ones in this blog.

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.

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.