Why we must prioritise childhood cancer
Young cancer patients around the world are suffering from a lack of new, targeted therapies and access to effective care, but paediatric oncologists are determined to change this
Cancer remains a leading cause of death from disease in children1. Each year around 400,000 children and young adults up to the age of 19 will receive an often devastating cancer diagnosis. In Europe, 35,000 children are told they have cancer annually and, while 80 per cent will survive for five years or more,2 6,000 will die. In lower- and middle-income countries (LMICs) the picture is even bleaker: as a result of delay or misdiagnosis, obstacles to care and a lack of access to medication, fewer than 30 per cent of children are cured3.
The French vaccines and pharmaceuticals giant Sanofi has created a two-pronged approach to tackle gaps in cancer care. It has dedicated a small part of its R&D to develop medicines to eliminate childhood cancer deaths and build partnerships with leading scientific institutions. At the same time it reaffirmed its commitment to improving survival rates of children with cancer in LMICs through its existing My Child Matters programme. In May, the company launched Foundation S – the Sanofi Collective to manage all of its philanthropic efforts. It has three key aims: to improve the health of communities most affected by climate change and pollution, to broaden access to life-saving medicines and vaccines and to continue tackling childhood cancer in LMICs, a programme the company has been pursuing for 17 years.
In 2005, the Sanofi Espoir Foundation launched My Child Matters, a multi-partner collaboration with NGOs and governments in countries where the needs of children and families were most evident. The programme has helped more than 120,000 children, trained more than 50,000 healthcare professionals, and is credited with increasing the survival rate of children by a median of 5.1 per cent over 10 years, thus saving the lives of more than 1,300 children.
As Vanina Laurent-Ledru, the new Director General of Foundation S, explains, the company will continue to fund awareness of and research into childhood cancer, with the aim of supporting the World Health Organization objective of achieving at least a 60 per cent survival rate for all children with cancer by 2030, which would save an additional million lives over the next decade. “For us it was essential to continue the fight against childhood cancer because in a number of countries, notably in francophone Africa, there is no one else,” she says. “We need to continue training those healthcare professionals and advocating with the entire community on behalf of those children.
“Our commitment is driven by the fact that we can leverage our expertise in capacity building, pain control, access to care and oncology, especially for those children in developing countries who mostly need support in terms of access to care more than access to innovation.”
For survivors everywhere, cancer leaves an enduring physical and psychological legacy4 that also needs to be addressed, says Peter Adamson, Global Head of Oncology Development and Paediatric Innovation at Sanofi, who has worked in paediatric oncology for 35 years. “The drugs we use today, with few exceptions, were approved in the 1950s, 1960s and a small number in the 1970s,” he explains. “It really wasn’t through new drugs that we achieved the current rates of long-term survival, but through an understanding that certain diseases needed more intensive administration of the drugs we have, and that has been leveraged to its fullest.”
Adamson explains that more than half of all children who survive cancer will be left with a long-term consequence, with the intensification of treatment exacting a painful price. “These are cytotoxic chemotherapy drugs, and they impact virtually every organ system. Children may lose hearing, never attain full adult height, have early onset heart disease, lung disease, kidney disease or bone disease,” he says.
The need to develop more targeted and innovative therapies, not only to improve childhood cancer survival rates but also to improve the quality of life for those who beat this terrible disease, is clearly overwhelming from a humanitarian perspective, and there are practical, clinical and financial obstacles to be overcome as well. While there are incentives and requirements enshrined in legislation in Europe and the US for drug companies to develop child-specific drugs, this has had a relatively small impact on the development of new paediatric oncology therapies.
Childhood cancers are commonly the effect of gene mutations5 and, as such, many of the 140 types of early childhood cancer can be considered ultra-rare; for example, only 4,300 children are diagnosed with the most common childhood cancer, acute lymphoblastic leukaemia (ALL), each year across the US and Europe.
With numbers of patients in the hundreds for some cancers, early drug development studies are hard to run, despite the fact that more than 60 per cent of children with cancer participate in clinical research in patient networks across the US and Europe.
For Dietmar Berger, Chief Medical Officer and Head of Global Development at Sanofi, research into innovative therapies for childhood cancers has been hampered by what he describes as an “abundance of caution” and a preconceived notion that children are more vulnerable than adults. Legislation demands that therapies are tested on adults before children, for example.
“The regulatory requirements are generally focused on individual drugs and on developing them in indications in children that are similar to the adult indications,” says Berger. “We decided to go beyond that, realising that the biology in some of the diseases in children is different.”
A study comparing the time taken for an investigational new drug to reach a first in-adult study, compared with a first in-child study, found the child-focused trials lag behind their adult counterparts by six years on average, a disparity that has convinced Peter Adamson that a new, accelerated R&D effort into childhood cancers is essential. Adamson’s team is making efforts to compress the time delay from adult to child-centred research into new cancer treatments from six years to 18 months by identifying research into adult cancer therapies that shows potential to treat childhood cancers with the support of additional laboratory studies.
“We are prioritising a small number of drugs in our pipeline,” Adamson says. “And we are not going to wait for the requirements and incentives to do so.” In parallel, Sanofi’s paediatric oncology team is working in partnership with patient networks in the US and Europe “because no single centre sees enough patients,” says Adamson. They are also working in tandem with specialist biotech companies and laboratories with the appropriate expertise to further research and drug development.
“We’re not going to have half a dozen drugs every year, but if we can get one leading candidate every one to two years, where we can very quickly show dramatic impact, that will be a success,” says Adamson. “The vision I am trying to put forward is to demonstrate that you can commit a fraction of your R&D budget, and make a significant impact on childhood cancer. I think other companies will follow Sanofi’s example.
“All companies are responding to legislated incentives and requirements, but no big pharma companies are saying, ‘We’re not going to wait for the requirements, we are going to take leading candidates – not all the drugs – and do this.’” For Adamson, and others working with children and their families to treat childhood cancers, the potential reward is enormous. “The goal is different,” he says. “It’s not to extend life by a few months. The goal is to give childhood cancer survivors normal life expectancy. We are willing to incur more risk, because that risk allows for a long-term cure.”
