Children with cancer often don’t have access to innovative drugs in the way adult patients do. Why? Today we talk to Cesare Spadoni, founder of the aPODD foundation (Accelerating Paediatric Oncology Drug Development), about the current situation with childhood cancer and how it can be changed.
1. What is your background and how did you get involved with the project?
I am a scientist and a drug development professional that has been working in business development roles for the past 12 years. But I am also a parent that one day heard the most terrifying words any parent may hear. My daughter was diagnosed with cancer at the age of three. After a year-long struggle with the disease she passed away in my arms. This was the time I resolved to do something for children with cancer.
2. What is the aim of your project and how did it start?
The major problem facing children with aggressive forms of cancer is the lack of access to innovative treatment options. Children with cancer are generally treated with radiotherapy and old cytotoxic drugs that were approved 40-50 years ago in some cases. Sick children do not have access to the most innovative therapies because these are largely developed and registered for use in adults, only.
Childhood cancers are generally different diseases. Specific drugs for these cancers are not developed by industry because it is not profitable to do so. The result is that children lack treatment options when conventional therapies fail.
We cannot save more children’s lives if we don’t make new treatments available more quickly. Moreover, we should not forget that cure with the existing harsh treatments comes at a price as survivors are likely to suffer from severe health problems later in life, including secondary cancers.
That is precisely why I set up aPODD (Accelerating Paediatric Oncology Drug Development). aPODD is a charity registered in London. Our mission is to accelerate the development of new drugs that are better and safer to treat cancer in children. We want to work alongside both academia and industry and effectively “de-risk” drug development for childhood cancers.
3. Could you please tell us more about your project with HealX? Provided that your crowdfunding campaign is successful (which we hope it will be!), what steps will need to be taken in order to accomplish this project (i.e. bring new drugs to the market)?
We believe that drug repurposing is an ideal approach to overcome the lack of new therapeutic options for children with cancer. Drug repurposing is about looking at medicines already approved for use in one disease and see whether they may be efficacious in another disease. Whereas new drug development is massively expensive and risky and way beyond the means of a medical charity such as aPODD, interesting drug repurposing opportunity may be identified more quickly and any further clinical development work can be completed at a fraction of the cost of new drug development. Indeed, there are several examples of drug re-purposing in the history of medicines. Aspirin is the best known example. It was originally developed as a treatment for pain and fever. It is now widely used as anti-coagulant too.
We decided to partner with Healx because they have an interesting technology to predict new uses for old drugs. They also have strong academic connections and two of the biggest drug repurposing experts worldwide in their leadership team: David Cavalla and David Brown.
With the current project we are looking at new possible treatments for Medulloblastoma, which is an aggressive form of brain cancer in children, for which we have limited therapeutics options.
Healx’ approach is based on advanced computational biology, machine learning and data analytics. Complex data sets derived from Medulloblastoma patients (mainly gene expression data) are analysed by Healx in order to match the profile of Medulloblastoma with the profile of a specific drug. The most promising hits are then manually curated by the drug repurposing experts within the company, who will study the possible mechanism of action and dig into any existing pieces of prior clinical evidence.
The team at Healx is now completing the first stage of the project. They are finalising a list of drugs that are predicted to have an effect against Medulloblastoma. We will soon be in the position to test these drugs in the laboratory. We are launching a crowdfunding campaign on May 25 to raise funds to support extensive evaluation in patient-derived Medulloblastoma cell lines. These experiments will be crucial to see whether any of these drug repurposing candidates may be taken further. Ideally, the best compounds will be evaluated in animal models in view of selecting the best compound as clinical candidate.
We are very excited about this project as any new compound could reach patients relatively quickly if the pre-clinical data support its use for Medulloblastoma.
4. How do you see the future of your organisation?
Clearly, we would like to be in the position to select a clinical candidate for Medulloblastoma at some point. We are aware that this is still a research and development project and there is no guarantee that we will be able to identify a clinical relevant compound. We are however confident that drug re-purposing is a sound approach for identifying new therapeutic options for disease areas that are neglected by the pharmaceutical industry. We believe that patients’ organisations like aPODD can play a pivotal role by partnering with innovative technologies providers such as Healx. More and more information is generated and it is imperative to make sense of these data in the smartest possible way. If we are successful with this Medulloblastoma project we may expand this approach to other childhood cancers and thus deliver tangible benefits to patients.
We simply need to speed up the identification and development of new drugs for children with cancer. Because childhood cancer won’t wait!
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