Wednesday 22 June 2016

Guest Post - Crowdfunding for Pediatric Cancers

Cesare Spadoni, founder of aPODD (accelerate Paediatric Oncology Drug Development), talks to Pan Pantziarka about the problems in drug development in children's cancers, and about the crowdfunding campaign to find a new treatment for Medulloblastoma.

Pan: What is the aPODD foundation aiming to do?
Cesare: aPODD (accelerate Paediatric Oncology Drug Development) was set up with the mission to speed up the development of better and safer treatments for children with cancer. This is a cause that is very close to my heart. I lost my first daughter to cancer a few years ago. That is when I began thinking about doing something for children with cancer.

Obviously, you can have a positive impact on sick children and their families in many different ways. In my case, because of my professional background in drug development, I felt compelled to do something to address the major problem preventing any further clinical improvement for children and adolescents with cancer: the lack of therapeutic options and the delayed access to the most innovative treatments.

Specific anti-cancer drugs are not developed for younger patients because it is not profitable for industry to do so. This is an area where patients’ organisations may play a vital role. Drug repurposing is certainly an area we are very much interested in. By looking at existing drugs we may be in a position to identify possible new treatments much faster and at a fraction of the cost and risk of new drug development

Pan: How is this campaign different to others?
Cesare: We are looking to identify a potential new treatment for Medulloblastoma, a rather aggressive form of brain cancer that is more frequent in children and adolescents. The current therapeutic options for this cancer are limited and very harsh, including high dose chemotherapy and radiotherapy. The impact on patients may be devastating as the lucky survivors may face severe health problems later in life.

For this project we are partnering with Healx, a company based in Cambridge (UK), which offers very advanced technologies and strong expertise in drug re-purposing. Healx is applying advanced computational biology tools, data analytics and machine learning to make sense of complex biological data sets and match those with the profiles of known drugs.

We are now in the process of finalising a list of drugs that we would like to test experimentally in Medulloblastoma cell lines in view of progressing further with the most promising ones.
We are very excited by these early results and we are really hope that this crowdfunding is successful so that we can proceed as fast as we can.

Pan: What do you see as the key impediments to advancing drug development for paediatric oncology?
Cesare: Paediatric oncology is neglected in terms of new drug development. The pharmaceutical industry tends to focus on adult drug development and largely fails to develop specific treatments for younger patients with cancer. Children are currently treated with old cytotoxic drugs that were approved as far as 40-50 years ago. Children do not immediately benefit from the latest advancement in targeted therapies as these usually get to be evaluated in children only several years after approval in adults.  In practical terms, children are treated as second-class citizens when it comes drug development efforts

Pan: Why is paediatric oncology different to adult oncology?
Cesare: Childhood cancers are generally different from adult ones. There are some diseases that are almost exclusively seen in younger patients.

This is where part of the problem lies. Industry develops drugs for the more common adult cancers (e.g. Lung, Breast, Prostate). As these diseases do not generally occur in children and adolescents, there is not obligation imposed by the regulators onto companies to evaluate these investigational drugs in children and adolescents too.

However, because of the similar mechanisms of action in some cases, we have the potential to see a therapeutic effect in some childhood cancers too. For example a class of anti-cancer compounds defined as ALK inhibitors was developed to treat lung cancer in adults. These compounds, however, hold promise to treat a subset of paediatric neuroblastoma patients.  Paediatric development was not initially pursued by companies only to find out years later that a clinical effect was indeed shown in children.

All these are truly missed opportunities for children and adolescents with cancer.

Pan: Do you see any signs of hope in the near future?
Cesare: I believe that real change in paediatric oncology must be driven by parents and patients. Recently we have seen more and more childhood cancer charities willing to get more directly involved in drug development efforts. We need to realise as a community that we must understand the challenges of drug development and actively work to shorten this highly expensive, risky and challenging process.

Drug repurposing is one approach but we should also considering partnering with industry for selected co-development projects in order to “de-risk” childhood cancer drug development. To this end we are working on the creation of a global drug development fund for childhood cancer, together with other leading charities in EU and US.

Pan: Aside from Medulloblastoma are there other childhood cancers you want to focus on in the near future?
Cesare: If this drug repurposing project for Medulloblastoma proves to be successful we are planning to initiate other projects for hard to treat childhood cancers that have currently a poor prognosis and are responsible for the majority of deaths. We need to shorten the time it takes to identify possible treatments for children and adolescents with cancer. Sadly, time is not on their side but if we can save time in the process we will certainly save more lives.

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