Hope for infant leukaemia sufferers with new treatment

Little Tilly (centre) who was diagnosed with leukaemia. Photo / Supplied

When little Tilly Buttini was diagnosed with leukaemia at just nine months old she had a 50 per cent chance of survival.

But new research has led to the discovery of a promising new approach to treating some of the worst types of leukaemia, including the aggressive kind that mostly affects babies.

Infant leukaemia is a subtype of leukaemia that has a gene rearrangement, news.com.au reports.

"It's one of the worst types of leukaemia children can get," said Professor Richard Lock, of the Children's Cancer Institute.

"A child who gets acute lymphoblastic leukaemia will experience close to 90 per cent survival, whereas infants have a 50 per cent survival.

"It really is a horrible type of leukaemia, and we desperately need new drugs to treat this in kids.

"With the current drugs they're treated with, which are very toxic, they suffer the long-term effects of conventional treatment if they survive into adulthood."

Clinical trials on the new treatment have already been successful in mice and have moved on to human trials in the US.

Prof Lock said they hoping there would be a clinical trial in children as early as next year.

The treatment would help so many kids like Tilly, whose family got the worst news possible when she was a baby.

It was just a regular morning for mum Chantal, getting her daughters ready for school.

Her eldest, Lucy, said baby Tilly had chickenpox in her ear.

Chantal took her to the GP and was sent immediately for a blood test.

By 6.30pm that evening, Tilly had been diagnosed with leukaemia.

Tilly was diagnosed at nine months old. Photo / Supplied

"I woke up at 7.30am on a normal, sunny day. By 6.30pm, my daughter had cancer – and our world changed forever," Mrs Buttini said.

"I felt like throwing up. It was like being punched in the stomach … we were completely blindsided."

Thankfully, Tilly completed her treatment in 2011, and today she is a bubbly eight-year-old who loves swimming, ballet dancing and crafts.

The type of leukaemia she had occurs in about 80 per cent of acute leukaemia cases in infants and up to 10 per cent of all leukaemia cases.

Often resistant to chemotherapy and extremely difficult to treat, the leukaemia claims the lives of many of those affected.

The latest research, published this month in the international journal Cancer Cell, says the new therapy has "outstanding pharmaceutical properties".

When used to treat specially bred mice that were growing the leukaemia derived from human patients, the therapy produced a "dramatic response", curing many of the mice.

She's now a bubbly eight-year-old. Photo / Supplied

"The combination of drugs currently used for treatment is often not effective and causes significant side effects," Prof Lock said.

"This is undesirable in anyone but is particularly a problem in children, whose growing bodies are very susceptible to the damaging effects of toxic drugs."

The newly developed agent is one of a new breed of targeted therapies that are designed to specifically target molecules that are critical for the survival and growth of cancer cells.

"What is particularly exciting about this new agent is that it shows remarkable anti-leukaemia activity when used on its own," Prof Lock said.

"It is highly unusual to see a single agent cause such a dramatic response, so we are extremely optimistic that it will prove effective in humans."