THIS year’s Nikolas Symposium has been hailed a landmark as clear targets have been set at which to aim new drug treatments for LCH.
H R Trust Trustee, Professor Peter Beverley, has advised that following the discovery of BRAF V600E, a mutation found now in both some samples of LCH and certain cancers, a huge understanding in LCH has resulted.
The conference, the 21stto be held since its formation, was entitled Dendritic Cell Genomics and Metabolomics: Clues for LCH Causes and Cures and was attended by scientists and doctors from all over the world, including H R Trust Trustees Drs Vasanta Nanduri and Johann Visser and Professor Beverley.
It was Professor Barrett Rollins of Harvard University, who had attended a previous Nikolas Symposium, who identified in 2010 a change or mutation, in the DNA in half of LCH samples. Tellingly the mutation is in a kinase and these molecules are the key to controlling cell growth.
Mutations in kinases are often found in cancers and the particular mutation found in LCH samples, called BRAF V600E, has already been found in other forms of cancer. Because the discovery of V600E in LCH is a breakthrough in understanding the cause of LCH, much of Nikolas 21 focused on the implications.
No scientific advance is considered secure until confirmed, so it was important that two independent labs presented data confirming Professor Rollins’s finding. As the mutation is found in only half of LCH samples, it is likely that other mutations are present in the other half.
Delegates discussed methods for searching for further mutations and another important question that was considered was whether only the mature LCH cells found in LCH tissue samples, contain the mutation. Alternatively the mutation might be present in blood or bone marrow stem cells as well, which would have implications for treatment.
Though these and many other questions remain, the presence of V600E indicates that LCH is a form of cancer as well as providing a clear target for drug development. Encouragingly, many pharmaceutical companies are developing drugs against kinases for cancer therapy and the meeting heard a preliminary report on a clinical trial of one of these as a treatment for LCH.