More ambitious collaborative models are needed - Open Letter

We often hear from childhood cancer charities calls for deeper and wider collaborations among key stakeholders. I fully agree that collaborations are beneficial but I do feel that we must further elaborate on this concept and focus more on outcomes other than procedures. We also need to challenge existing models and explore new ones. 

One of the most puzzling observation I made when I set up aPODD and joined the childhood cancer community is how fragmented this community is and how is lacking a strategic vision when it comes to drug development. We have thousands of childhood cancer charities often working separately on small, local projects that have very little chances of delivering tangible benefit to patients on a global scale. This happens because we are trying to solve problems that are massive in scale, requiring an equally massive approach. Instead we are literally fighting a fire with a little cup of water at a time.

We see encouraging examples of charities here and there that attempt some new approaches when it comes to research. But I would challenge the childhood cancer community (myself included) to do more and aim higher. Collaboration is unlikely to deliver better results if it just means doing more of the same together on a bigger scale. Scaling up an inefficient process is more likely to generate a larger inefficiency.

Every year approximately $100M are invested in childhood cancer research in the US alone. It is not unreasonable to assume that at least $1B has been invested over the past 20 years. And yet we have seen practically no results when it comes to new drugs approved for childhood cancer treatment. This is because we have been working on a single model that is supporting and engaging with academia only. It’s great to have hospitals and research institutes receiving funding and being able to research into the molecular machinery of childhood cancer but this will not translate into new medicines approved for use in children if we don’t make it attractive for companies to take advantage of this research and get new treatments all the way to regulatory approval and commercialisation. While I would not advocate that we abandon academic research funding, we must also consider new partnerships. Childhood cancer charities must understand the operational and financial challenges of new drug development and take more responsibility for that. 

Yes, we do need to collaborate, but we urgently need to do so with biotech and pharma companies because, whether we like it or not, they are the ones developing new drugs. The Cystic Fibrosis Foundation did not revolutionize the medical outlook of their patients by doubling their investment in basic research. They did so by creating a drug development unit and starting to work with biotech companies like Vertex Pharmaceuticals.

Why can’t we do the same in paediatric oncology?

Why don’t we want to create a drug development entity for childhood cancer?

I believe a starting point is the Kids Cancer ACT NOW drug development consortium launched by aPODD and the Medical Research Council Technology (MRCT) last October during the SIOP conference in Dublin.  

Together with MRCT we want to identify promising treatments in the industry pipeline that have the potential to deliver benefits to childhood cancer patients. We want to accelerate their clinical development by raising funds from the consortium members and other funding sources (e.g. venture philanthropy, social/impact investors) and create co-development partnerships with industry. This approach, driven by science and patients’ unmet needs, has the potential to accelerate the development of much needed therapies.

Take DIPG, for instance, surely the most lethal form of brain cancer in children. Why not leveraging the efforts of DIPG charities by hav8ing them to join Kids Cancer ACT NOW and creating an even bigger drug development fund that could be used to support a specific DIPG drug development project? The opportunities might be out three. We just need to grab them.

This change will not happen because suddenly industry will become child-focused and philanthropic and because academic research will have more funds available for their research. Real change will come when childhood cancer charities will jump into the driving seat and start leading the way.

In keeping with an old, famous quote by Mahatma Gandhi, we should “be the change we want to see in the world”.

Let's work together and make this happen.

Cesare Spadoni, on behalf of the aPODD team