Professor Veronica Kinsler and her researchers at the Francis Crick Institute and Great Ormond Street Institute of Child Health have published an article in the Investigate Dermatology Medical Journal detailing a huge breakthrough in their CMN research work.
Professor Veronica Kinsler and her CMN research team have designed a new genetic therapy that has silenced the NRAS gene in cells in a dish and in mice. NRAS is one of several genetic mutations causing Congenital Melanocytic Naevus (CMN). The research team used a genetic therapy called silencing RNA, which silences the mutated NRAS in CMN skin cells. The therapy was delivered through special particles directly to CMN cells. The team gave injections containing the therapy to mice with CMN; silencing the NRAS gene after just 48 hours. They also tested it in cells and whole skin sections from children with CMN. Importantly, silencing the gene triggered the CMN cells to self-destruct.
It is hoped this approach will form the basis for clinical trials within the next 24 months, subject to raising enough funds. This means that in the future the treatment could potentially be used to reverse CMN, and therefore reduce the risk of affected children and adults from developing melanoma. It could also potentially reverse other commoner types of at-risk moles as an alternative to surgery.
Prof Kinser said “These results are very exciting, as not only does the genetic therapy trigger self-destruction of the CMN cells in the lab, but we have managed to deliver it into the skin in mice. These results suggest that the treatment in future could potentially reverse CMN in people, however more testing will be needed before we can give it to patients.
“We are very grateful to our patients at Great Ormond Street Hospital, who have been actively participating over many years to help us produce this new potential therapy. After more studies, we hope the therapy can soon enter clinical trials in people.”
You can read a plain language summary of the research findings here.
It is important to remember there are many different expressions of CMN, including neurological complexities and the risk of melanoma. Our vision is to live in a world where no one suffers because of CMN and so for some of our members this newly developed treatment could prove to be life transforming.
Looking ahead, we have lots of work to do over the next 4 years and beyond, such as, publicising the research breakthrough on a global scale; supporting patients who take part in clinical trials alongside continuing to deliver our Support Pathway to all families affected by CMN; educating medical professionals about CMN and the research breakthrough so families can make informed medical choices, continuing to raise the profile of CMN through our fantastic ‘how do you C Me Now?’ campaign. To do all this we must raise £500,000 over the next 4 years. We are so close to finding a treatment for CMN; you can join us on our journey by donating here.
We do hope you are able to take time to reflect on the truly incredible dedication of Professor Kinsler and her team to get the CMN research to this point of discovering a potential treatment. Over the past 26 years, as a charity, we have been able to provide Professor Kinsler with over one million pounds of research funding raised by our members and supporters.
We have all been a part of this journey and so I would like to personally thank all our members and supporters for partnering with us to achieve our 3 main aims; supporting those affected by CMN, raising awareness of CMN and funding pioneering research to develop effective treatments for all expressions of CMN; providing choice for our members.
Jodi Whitehouse
Founder & CEO
