Lila’s Remarkable Journey

The goal of the Zero Childhood Cancer pilot study was to establish the components of the program ready for the National Clinical Trial. It achieved that…and so much more. For at least one of the youngsters enrolled in the study, Lila [1], the personalised medicine she received as part of the study has been credited with prolonging her life.

Lila was just five months of age when doctors removed her first cancerous brain tumour.

By early 2016, Lila was under the care of Prof Richard Cohn, Head, Clinical Oncology at the Kids Cancer Centre at Sydney Children’s Hospital Randwick. While Lila initially did well on her maintenance chemo, at 2½ years old, symptoms returned, and she underwent a second serious surgery. It was a success…but it was not to last.

In January 2017, scans showed there were multiple tumours, including one on her spine. Lila’s condition deteriorated rapidly. She lost the ability to walk, stand, talk and sit up unaided. Another surgery was out of the question due to the location and the number of tumours. So serious was her condition, that her family was introduced to the palliative care team.

But there was a glimmer of hope. During her earlier treatment, Lila’s parents had consented to provide a sample of her tumour to test as part of the Zero Childhood Cancer pilot study. Prof Cohn had explained that the program was still in its very early stages and there was no guarantee that a drug match would be found and even if a drug was found, there was no guarantee that it would be able to help Lila.

“As Lila’s treating clinician, we were delighted when the Zero Childhood Cancer program was able to find a drug match that offered another treatment option for Lila when all other treatments had failed, and at a time that there was very little hope for her survival,” Prof Cohn said.

Then came the real hope. Testing on Lila’s samples as part of the Zero Childhood Cancer program showed the possibility that a new drug might be able to help her. Zero Cancer identified a rare DNA change in the tumour cells with the potential to drive the ongoing growth of the cancer and for which there was fortunately a new drug with the potential to target that change and inhibit the cells from continuing to grow.

Improvement in Lila’s condition once she began the new treatment was almost immediate. Within weeks, there were positive improvements in her responsiveness, interactivity, speech and mobility. Eventually, she started standing on her own – by the third week, she started walking again. Within a few more weeks on the new treatment, Lila was even seen dancing in the wards.

Lila’s most recent scans have shown very promising early results, according to Prof Cohn.

“While it is still very early stages, her response to this drug has been very positive and we have seen significant early improvements in her condition that none of us could have expected.

[1] Patient’s name has been changed for anonymity

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