As we put the essential pieces together to launch a major precision medicine clinical trial for children with acute leukemia – LLS PedAL - one of the most important aspects of this trial will be its global reach. This LLS-led trial, which will test targeted therapies in children with acute myeloid leukemia, and eventually other high-risk leukemias, will benefit not only children in the United States, but in several European countries, as well as the UK, Australia, New Zealand and Canada. We anticipate opening the trial in approximately 200 sites around the U.S., Canada and across the globe.
In November 2019, representatives from Europe, including Christian Michel Zwaan, MD, PhD, professor of pediatric oncology, senior PI developmental therapeutics and head of trial and data center, at Princes Maxima Hospital in Utrecht, Netherlands and Dirk Reinhart, MD, director of pediatric oncology at the University Hospital in Essen, Germany, visited our headquarters in Rye Brook, New York, to discuss how this collaboration would work.
And in early February 2020, the chairs of LLS PedAL clinical trial were invited speakers at the annual ACCELERATE conference in Brussels, Belgium, an international gathering of pediatric oncologists, researchers, patient advocacy organizations, biopharmaceutical and regulatory representatives committed to advancing new treatments for children with cancer. The LLS PedAL team gave a presentation on the trial’s concept, which can stand as a model for international collaboration to execute clinical trials with intent to file for approval of novel therapies with the U.S. Food and Drug Administration, as well as regulatory agencies in other countries.
Our European colleagues recognize the importance of this trial and its potential impact for children who urgently need better treatments for leukemia and they are eager to join us in our effort. Drs. Zwaan and Reinhart are working with us to help form a European AML consortium to join LLS PedAL to ensure as many children in Europe as possible will have access to the trial when it launches later this year.
LLS PedAL is a key component of the larger LLS Children’s Initiative – a $100 million multi-year endeavor to take on children’s cancer from every direction. In addition to the unprecedented clinical trial, we are developing a platform to share pediatric cancer data so more researchers and physicians have access to this vital information. Our initiative also includes new research grants to advance novel treatments such as immunotherapy, gain greater understanding of which patients are most likely to respond to therapy, and address the long-term effects of treatment such as cardiotoxicity, infertility and cognitive impairments. We are also enhancing our free education and support services for children and their families and pressing for policies to improve access to care and treatment.
Dr. Reinhartt, MD, presenting at the ACCELERATE meeting in Brussels