Researchers from the Francis Crick Institute, UCL Great Ormond Street Institute for Child Health and Great Ormond Street Hospital for Children (GOSH) have created a new genetic therapy to help with large moles in a rare skin condition. The therapy has the potential to reverse the giant moles and lower the likelihood of affected individuals developing cancer. It may also have applications in treating other conditions.less common forms of vulnerable moles rather than opting for surgery.
Most people have small skin moles, but children with congenital melanocytic nevus syndrome (CMN) are born with large, painful, or itchy moles covering up to 80% of their bodies due to mutations acquired before birth. These moles have the potential to develop into a dangerous type of cancer known as melanoma.
A recent study published in the Journal of Investigative Dermatology reveals that researchers were able to suppress the activity of a gene called NRAS, which is mutated in the cells of these moles, in cells in a dish as well as in mice. NRAS is part of a group of genes known as RAS genes.The team utilized a genetic therapy known as silencing RNA to silence the mutated NRAS in mole skin cells, which, when mutated, can cause moles and predispose individuals to cancer. The therapy was administered in specialized particles directly to mole cells.
Injections containing the therapy were given to mice with CMN, resulting in the silenced NRAS gene after just 48 hours. The team also conducted tests on cells and entire skin sections from children with CMN. Silencing the gene prompted the mole cells to self-destruct.
Veronica Kinsler, Principal Group Leader of the Mosaicism and Precision Medicine Laboratory at the CriDr. Veronique Bataille, a professor at GOSH/UCL specializing in paediatric dermatology and dermatogenetics, as well as a NIHR Research Professor, expressed the challenges faced by individuals and families living with CMN. The study’s findings are promising, as the genetic therapy not only caused the mole cells to self-destruct in the laboratory, but it was also successfully delivered into the skin of mice. This suggests that the treatment could potentially reverse moles in humans, although further testing is necessary before it can be used in patients. Dr. Bataille also expressed gratitude towards the patients involved in the study.The researchers at Great Ormond Street Hospital have been actively involved in helping to develop this new potential therapy over the course of many years. After further research, they are hopeful that the therapy will be ready for clinical trials in humans soon. This project received funding from the National Institute for Health and Care Research (NIHR), Caring Matters Now Charity and Patient Support Group, LifeArc, and the NIHR Great Ormond Street Hospital Biomedical Research Centre. Jodi Whitehouse, CEO of Caring Matters Now, expressed optimism about the breakthrough in finding a treatment for CMN, believing that it could significantly improve the lives of the families they support who are living with the condition.CMN. The news is incredibly inspiring and exciting for someone like me, who was born with CMN covering 70% of my body and underwent over 30 operations in childhood in an attempt to remove the CMN due to the risk of melanoma, but without success. This brings real hope to those living with CMN.”
Catriona Crombie, Head of Rare Disease at LifeArc, stated: “This work is part of our dedication to improving the lives of people with rare diseases, by investing in promising research and assisting scientists in overcoming translational research obstacles. If successful, we anticipate human clinical trials for this therapy within the next few years.
.”
The scientists have been collaborating closely with the Crick’s Translation team to advance the technology for the benefit of patients. This has involved securing funds for translation from LifeArc, in order to conduct further research in mice to gain a better understanding of how the treatment functions over an extended period.
