Making the RARE voice heard
LifeArc joins the NRG Collective’s #RareYouthProject as a 2018/19 sponsor
LifeArc’s vision of collaborative approaches to improving outcomes for rare disease medicines has led to the medical research charity joining the #RareYouthProject. The project takes the form of a year-long programme that works with children and young people affected by rare disease. The project will create a magazine with articles that matter to Young People, written by Young People in the rare community.
Launched in 2017 by independent not-for-profit publishers, NRG Collective Ltd, creators of Rare Revolution magazine, this annual programme works with the young Rare community to ensure their voices are heard through the creation of their own dedicated Youth Magazine.
The young editorial team are given complete control of topics, assignments and design for the magazine. The Rare Revolution Team, in collaboration with the RareTogether project, support all aspects of their journey from the initial planning meeting through to the successful launch of their final publication.
The #RareYouthProject first project took place in 2017 finishing in March 2018 at a dedicated Youth Event in London where around 50 young people and their families took part in the Rare Youth Issue launch.
The finished 2017/18 magazine is outstanding and a testament to the hard work and passion that each member of the youth editorial team put into the project
Nicola Miller, Creative Director and Youth Co-ordinator
Commenting on the collaboration Dr Catriona Crombie, LifeArc’s Philanthropic Fund Manager said;
While LifeArc tends to be involved early in the process of developing new drugs for patients, a collaboration like this reminds us why we do what we do and allows us to better understand the needs of those affected by a rare disease
Rebecca Stewart, CEO, NRG Collective Ltd, noted;
“Following on from the success of our pilot programme we want to secure this project for future years to ensure our talented young rare community can continue to be heard and have access to the long-term benefits that come from the work experience, mentoring and friendships made during this programme. We are thrilled to have the support of LifeArc as Silver Sponsor of our 2018/19 Project and hope other companies will join them in supporting our annual programme.”
The 2017/18 Rare Youth Issue can be read here https://bit.ly/2IBjAhL If you would like to find out more about supporting this year’s #RareYouthProject please contact Rebecca on firstname.lastname@example.org
NRG Collective Press Contact
Rebecca Stewart, Title, NRG Collective
Tel: +44 (0) 13398 83838
Mobile: +44 (0) 7795663589
LifeArc Press Contact
Glenn Mursell, Communications, LifeArc
Tel: +44 (0) 207 391 2754
Mobile: +44 (0) 7799 261 060
About NRG Collective Ltd.
An independent not for profit company NRG Collective Ltd are dedicated to making rare voices heard. Through their free publications Rare Revolution and the Rare Youth Edition they are raising awareness and providing valuable information for the whole rare disease community.
LifeArc is a medical research charity with a 25-year legacy of helping scientists and organisations turn their research into treatments and diagnostics for patients. LifeArc is pioneering new ways to turn great science into greater patient impact. The charity brings together a network of partners to tackle specific diseases and directly funds academic and early stage research. So far, LifeArc’s work has helped to develop four drugs (Keytruda®, Actemra®, Tysabri® and Entyvio®) and a test for resistance to carbapenems. www.lifearc.org Twitter @lifearc1
In Summer 2017 we published 'Let's Work Together' an article by Dr. Neil Rajan highlighting the need for research in CYLD cutaneous syndrome (CCS) and his success in being granted a clinical trial. In this follow up we hear from Dr Rajan what the results mean for the CSS community.
History of CSS Research
Around 1985, people affected by a rare skin disease in Newcastle, joined forces with Prof Sir John Burn to push for research on inherited cylindromas, now called CYLD cutaneous syndrome (CCS).
Half of people with CCS have at least one painful skin tumour and up to a quarter undergo complete scalp removal.
These patients wanted research to improve this outcome, and wanted alternative treatments to surgery. The first steps to this was establishing what was different in the DNA of these patients and their tumours and in 1995 in a partnership led by Professor Burn with collaborators at the Sanger Institute, a difference was found. This gene locus was called CYLD. In 2000 CYLD was cloned, and in 2003 Prof Alan Ashworth published one of three papers in the journal Nature highlighting CYLD’s function.
It has been a great privilege to work alongside Professor Ashworth and Professor Chris Lord, and in 2011 we highlighted a dependency on an enzyme called TRK in cylindroma cells. This enzyme, when blocked in cylindroma cells in the lab, would kill them off. But would it work in patients? The next seven years involved delivering a clinical trial to Newcastle patients that used a drug that would block this very enzyme.
This month we reported on this, the first ever targeted, randomized, placebo-controlled clinical trial for CCS called “TRAC”. In this study, we tested an ointment that blocked TRK in cylindroma skin tumours. Partnering with a company who had already made this ointment and found it was safe to use in patients with other skin conditions like psoriasis, we used the highest strength with human safety data available in the TRAC study. Although we showed this was safe for the study period, this ointment doesn’t appear to be strong enough. We now we need to see if stronger strengths of the ointment can have an effect, in dose escalation studies.
For more information on this report read here
We are not there yet, but have learnt a lot about CCS. The TRAC trial has allowed us to study the natural history of this poorly understood rare disease, teaching us about the rate of growth cylindroma tumours. We have learnt about the impact of this disease on patient quality of life, and have developed clinical trial tools to measure symptoms such as pain, which interestingly, improved in some tumours in the TRAC study.
Importantly we have developed a roadmap for future trials, and this is relevant at a time when newer drugs that block TRK are being developed.
Lessons learned: Perseverance, patience, and patient partnership are crucial to take steps forward for rare disease and to keep going.
Figure 1. By studying multiple tumours in each patient with CCS, we were able to design a placebo-controlled statistically powered clinical trial – an uncommon feat in rare disease. Also, at the end of the study, we biopsied selected skin tumours, allowing us to study the effect of the drug and its penetration into tumour tissue. (Image by Marina Danilenko)
Rare Revolution Editor