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Blog | 9 May 2019

Childhood cancer treatment: the challenges and solutions

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All childhood cancers are considered rare. Each year, around 300,000 new cases are diagnosed among children (0-19 years) worldwide. Advancements in treatment mean that the survival rate for children’s cancer has doubled since the 1960s, yet, despite these successes, cancer is still a leading cause of death amongst children.

Here we explore what makes childhood cancers so difficult to treat, along with how the latest technologies and drug discovery methods can be used to find new and effective treatments for rare cancers.

The challenge of treating childhood cancers

The complexity of such cancers, their fast and malignant progression and the fact that many of the current approved cancer treatments that increase survival rates also cause health problems, are just three of the many reasons why childhood cancers are so difficult to treat.

These are some of the challenges that we’re working to overcome through our work here at Healx. By collaborating with patient groups and using the latest technology and drug discovery methods, we’ve been able to progress new treatments for childhood cancers such as diffuse intrinsic pontine glioma (DIPG), medulloblastoma and neuroblastoma. In doing so, we’ve had to overcome a number of treatment challenges.

Three key treatment areas for tackling childhood cancers:

1. Discovery of less toxic cancer drug treatments

The high toxicity of drugs used in conventional cancer therapies, such as chemotherapy and radiation, can be difficult for children to tolerate. These treatment types can cause a number of late effects in children whose bodies are still growing, and whose normal cells are dividing more quickly than in adults.

This is particularly problematic for the treatment of brain cancers such as medulloblastoma (the most common type of paediatric brain cancer) where the standard treatment relies heavily on radiotherapy. In such cases, radiation therapy can cause damage to a child’s developing central nervous system, potentially resulting in changes to their level of intelligence and ability to learn.

One way of uncovering new treatment options that are both effective and less toxic is to identify drugs and nutraceuticals already in clinical use that can be repurposed as safe treatments for childhood cancers. Identifying drugs that have already been tested for safety and efficacy in previous studies makes it more likely that the new drug regimens discovered are more tolerable for childhood cancer patients. We adopted this drug repurposing strategy in our work with childhood cancer charity, aPODD (Accelerating Paediatric Oncology Drug Development), to find new treatments for neuroblastoma and medulloblastoma

Using Healnet, our treatment prediction platform which harnesses artificial intelligence (AI) technology to identify possible new drug treatments, we were able to shortlist 13 existing drugs with the potential to treat neuroblastoma. The most promising of these drug candidates are now being validated in patient-derived xenograft.

2. Reducing the drug discovery timeline

The speed at which childhood cancers often progress is in stark contrast to the slow and complex process of drug discovery and approval. This is the case in DIPG, the second most common type of primary, high-grade brain tumour in children, where tumours usually grow quickly and are more likely to spread to other parts of the brain or spinal cord.

As it stands, there is a real lack of approved treatment options, and whilst it is an exciting time for gene therapies, these will still take in excess of 10 years to reach the market. This is too long for children with DIPG, and other aggressive cancers, to have to wait.

The use of new technologies, such as AI, offers opportunities for developing new treatments whilst also dramatically speeding up the drug discovery process. Healnet, for instance, is able to screen thousands of drug profiles, matching them to the molecular biology of the disease – all in a relatively short amount of time compared with the traditional drug discovery process. The treatment predictions made by Healnet then have the potential to reach the clinic as safe, effective treatments within two years – and in time to make a difference to children with these devastating cancers.

Our work with aPODD is a good example of this approach in action. Thanks to Healnet, we were able to predict 13 new treatment options for neuroblastoma within the space of 13 months – dramatically speeding up a process which typically takes ten times as long using traditional research methods.

3. Exploration of cancer drug combinations 

Many childhood cancers are highly malignant, have large numbers of genetic mutations and show dysregulation of many signalling pathways. This makes the task of finding specific disease targets to focus therapeutic efforts on very difficult. In addition, these types of tumours are able to rapidly evolve by accumulating genetic mutations in response to therapy, making therapeutic resistance a substantial issue too.

One way of overcoming these challenges is to opt for a combination therapeutic approach (where two or more drugs are used together to treat a disease). Carefully designed combination strategies can simultaneously hit different pathways and targets that are critical for a tumour to survive and grow. This, in turn, helps improve therapeutic outcomes, avoid resistance, minimise side effects and reduce drug dosage for patients.

Discovering new combination therapies is also something that Healnet can help with. From the data stored in our vast knowledge base of rare diseases, Healnet is able to make new connections between related childhood cancers – in terms of their biology, their symptoms and the drugs that may be effective on them. Healnet’s suite of AI algorithms then mine this data further to predict combination therapies that have the best chance of treating the rare cancer in question.

These predictions have helped us to progress combination therapies for a number of childhood cancers which are now being validated in disease models.

Collaborate with us

Our commitment to accelerating treatments for rare diseases is at the heart of everything we do here at Healx. We achieve this through our innovative technology, dedicated team of experts and collaborations with patient groups. We are currently working on childhood cancer projects focused on DIPG, medulloblastoma and neuroblastoma and are about to initiate projects on related rare cancers.

If you are a patient group and would like to work with us to find treatments for rare cancers, please contact us here. We’d be happy to hear from you.