Childhood Cancer R&D Program: Developing and Accelerating Novel Therapies for Children With Cancer

Over the past 60+years, survival rates for children with cancer have greatly improved, most notably for most leukemias and lymphomas, along with a number of solid tumors such as Wilms tumor, a tumor of the kidney. Despite this progress, a number of childhood cancers, subtypes of certain cancers, or cancers that relapse, often lack curative treatments [1]. Furthermore, the majority of children who survive cancer, experience one or more significant long-term side effects later in life as a result of treatments that may include intensive chemotherapy, radiotherapy or surgery [2] [3].

Childhood cancers, which comprise only 1% of all cancers, exhibit distinct biologic traits that usually present rapidly and follow an aggressive growth pattern. Under the microscope, many childhood cancers contain features seen in fetal tissues and are the result of dysregulated development [4]. Compared to adult cancers, childhood cancers harbor fewer genetic mutations but generally show more gene fusions and epigenetic changes [5].

Developing new treatments for childhood cancers has been challenging as each cancer is rare or ultra-rare, lacking clear economic models that can support research & development in the private sector. Despite legislation in the US and Europe that incentivize or require the conduct of pediatric studies [6], clinical trials for children still encounter a 6.5-year delay compared to adults [7].

The goal of this program is to promote the development of novel and safer drugs for children with cancer, to accelerate the start of clinical trials for children whenever scientifically appropriate, and to become a role model for other pharma companies by showing that the pharmaceutical industry can help lead development of new treatments under the umbrella of corporate social responsibility.

We implemented a proactive approach to identify drugs from the Sanofi R&D pipeline that could be of potential benefit for children with cancer. By systematically evaluating the pipeline early in the R&D value chain, we identify compounds targeting pathways that are relevant for childhood cancer. We conduct preclinical studies and initiate protocols for clinical trials with children once the scientific relevance is confirmed. In 2023, we thus started a clinical trial in children with leukemia in less than 2 years after adults, an impressive improvement compared to the median of 6.5 years delay between trial initiation in children versus adults.

We want to accelerate drug development for children with cancer by joining forces and sharing knowledge with expert institutions and consortia in the field. To this end, we have built strategic partnerships and collaborations with a number of premier organizations, including but not limited to the Innovative Therapies for Children with Cancer (ITCC) and the Foundation for the National Institutes of Health (FNIH) COACH consortia. In 2023, together with ITCC, we have launched a multistakeholder Childhood Cancer Working Group at the Paris-Saclay Cancer Cluster (PSCC). One of the aims of this PSCC Childhood Cancer Working Group is to encourage biotech- and start-up firms to work on childhood cancer. In addition, we foster innovation for childhood cancer research by leveraging Sanofi’s existing i-Awards / iDEA-iTECH Awards and the Sanofi Golden ticket initiative.

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Raising awareness about childhood cancers and the urgent need for new therapies is a top priority for us. We therefore organize events at Sanofi throughout the year, including specialized webinars on different research topics and informative seminars focused on childhood cancer. Additionally, every September during childhood cancer awareness month, we host an annual symposium where we gather key opinion leaders in the field. We also actively participate in international conferences and use these platforms to inform on our dedication to childhood cancer and to exchange with the community in the field. These events are invaluable opportunities for collaboration and knowledge exchange.