5th Paediatric Strategy Forum

Paediatric Strategy Forum

for Medicinal Product Development of Epigenetic Modifiers in Children and Adolescents

23-24 January 2020 

Wyndham Philadelphia Historic District Hotel, 400 Arch Street, Philadelphia, USA

PRESS RELEASE: Groundbreaking Childhood Cancer Collaborative Comes to the U.S. for the First Time

| Background and objectives | 

The fifth multi-stakeholder Paediatric Strategy Forum, organised by ACCELERATE in collaboration with the European Medicines Agency (EMA) and with participation of the Food and Drugs Administration (FDA) will focus on Epigenetic Modifiers, including BET inhibitors, in children and adolescents. Paediatric Strategy Forums have been created to evaluate science, facilitate dialogue and provide an opportunity for constructive interactions between relevant stakeholders (patient advocates, clinicians, academics, biotechnology/pharmaceutical companies and regulators) on specific topics requiring open discussion on development of medicines in the best interests of children and adolescents with cancer. The goal of this meeting is to share information, in a pre-competitive setting, to facilitate the developments of innovative medicines and ultimately their introduction into the standard-of-care of children with malignancies.

The first four Paediatric Strategy Forums were on anaplastic lymphoma kinase inhibition held in January 2017, on mature B-cell malignancies in November 2017 and immune checkpoint inhibitor combinations in September 2018 and acute myeloid leukaemia in April 2019  

DNA methylation and covalent histone modifications are precisely and dynamically controlled by various epigenetic modifiers and are critical aspects of transcriptional regulation. Dysregulation of these enzymes results in cancers. Recent research highlights that expression and epigenetic changes are particularly important in paediatric cancers. The goal of epigenetic therapies is to reverse epigenetic dysregulation, restore the epigenetic balance, and revert malignant cells to a more normal condition. In recent years, epigenetic modifiers have become the focus of intensive preclinical and clinical development efforts.

Currently, several drugs have been approved for clinical use in cancer treatments and many more are on the horizon. The introduction of RACE Act in the USA requires that drugs and biological products should be developed for paediatric cancers, if the drug or biological product is directed at a molecular target determined to be substantially relevant to the growth or progression of a paediatric cancer and there are a number of epigenetic targets on the FDA Paediatric Molecular Target List. This change in regulatory requirements, complementary to the EU Paediatric Regulation stresses even more the necessity for paediatric development of these medicinal products to be considered early.

The objective of the Forum is to determine the paediatric therapeutic needs and to define the landscape for the development of epigenetic modifiers in children and adolescents, whilst supporting global regulatory requirements.

The output of the Forum will be a published summary and manuscript from all participants addressing the challenges and documenting the conclusions of the meeting. Potentially further specific meetings relating to areas which require more in-depth discussion will be arranged.

| Summary – Paediatric Strategy Forum Medicinal Product Development for Epigenetic Modifiers in Children |

5th Paediatric Strategy Forum is presented by Alex’s Lemonade Stand Foundation


For more information, please contact contact@accelerate-platform.org
You can tweet using #ACCELERATEplatform