Scientists have developed a drug that destroys brain tumours. Cancerous cells are wiped out as the drug passes through the blood-brain barrier that protects neurons from foreign invaders.
Experiments found the small molecule killed them all, while leaving healthy tissue alone. Results were described as "very promising". The mice were cured - with no relapse after more than six months.
It works in combination with chemotherapy. Tumours quickly returned in peers given only the latter - and spread rapidly. Improved therapies are urgently needed for glioblastomas - which are often incurable.
The international team reckon it could be introduced in clinical practice within five years. The breakthrough has potential implications for other aggressive cancers.
Lead author Professor Leif Eriksson, of Gothenburg University, said: "These are the first clear results with brain tumours that can lead to a treatment which completely avoids surgery and radiation. We have also begun studying the use of our substance on other aggressive tumour forms like pancreatic cancer, triple-negative breast cancer and certain liver cancers."
Glioblastomas are notoriously lethal. Around 2,500 cases are diagnosed in the UK each year. Only seven per cent of patients survive.
They have claimed the lives of The Wanted singer Tom Parker and the US president's eldest son Beau Biden. Most patients succumb within two years and few make it past five - a statistic that hasn't improved in decades.
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The drug, named Z4P, blocks a mechanism that fuels the tumour's protein production - causing cells to die of stress. Cancer cells, especially those that form aggressive tumours, are out of control. To manage this pressure they hijack healthy cells.
Prof Eriksson explained: "We have now succeeded in stopping this by inserting a specially developed molecule in the cells that inhibits one of these hijacked adaptive mechanisms in the cancer cells. This causes the cancer to self-destruct."
Advanced simulations on super computers came up with a version that got it through the blood-brain barrier that prevents uptake of most pharmaceuticals. Prof Eriksson said: "Today cancer treatment consists of surgery, radiation and chemotherapy. Unfortunately all cancer cells are not killed and the tumour returns.
"Once the cancer relapses the tumour cells have often spread and developed resistance."
The technique described in iScience does not apply to other forms of brain cancer because they develop differently Current treatments for brain tumours often have severe side effects. None were identified in Z4P.
The treated animals maintained weight, had no apparent changes in behaviour and there was no sign of impact on the liver. Extensive lab tests on cells have shown the substance is non-toxic - even at very high doses.
Prof Eriksson and colleagues are now optimising the treatment procedure and planning additional animal studies. But they are confident it should be able to arrive relatively quickly into clinical treatment.
Prof Eriksson said: "It largely depends on whether funding comes in that allows taking the different steps as smoothly as possible. If I'm optimistic perhaps it might take five years. That's a short timeframe, but at the same time glioblastomas are nearly 100 per cent fatal, so any improvement in medical care is major progress."
The study is published in the journal iScience.
