A US study has shown high dose Vitamin C halts the progression of blood cancer in mice by encouraging "faulty" stem cells in the bone marrow to die.

The findings, published in journal Cell, has raised the possibility of new new combination therapies for leukaemia patients carrying a specific gene mutation known as TET2.

"We're excited by the prospect that high-dose vitamin C might become a safe treatment for blood diseases caused by TET2-deficient leukemia stem cells, most likely in combination with other targeted therapies," said Dr Benjamin Neel, director of the Perlmutter Cancer Center.

The TET2 gene carries a protein that produces and matures stem cells, a process beneficial to blood cancer patients.

It's estimated TET2 mutations are found in 10 per cent of patients with acute myeloid leukemia (AML), 30 per cent of those with a form of pre-leukemia called myelodysplastic syndrome, and in nearly 50 per cent of patients with chronic myelomonocytic leukemia.

Previous research had suggested that TET2 could be activated by high-doses of Vitamin C.

"So we had the idea that high-dose Vitamin C be used as a therapy for some forms of Myelodysplastic syndrome and acute myeloid leukemia, particularly those forms who have mutations in this gene called TET2," said Dr Neel.

In the lab, scientists at the Perlmutter Cancer Center in New York added high doses of the Vitamin C to human leukemia cells carrying the TET2 mutations.

"We saw that that stops the growth," said pathologist Dr Iannis Aifantis.

A similar result was produced when tested on genetically engineered mice, according to the study.

It was also found the Vitamin C treatment had an effect on leukemic stem cells that resembled damage to their DNA, says first study author Luisa Cimmino.

"For this reason, we decided to combine Vitamin C with a PARP inhibitor, a drug type known to cause cancer cell death by blocking the repair of DNA damage, and already approved for treating certain patients with ovarian cancer," she said.

The combination had an enhanced effect on leukemia stem cells, further shifting them from self-renewal back toward maturity and cell death.

Scientists are now trying to apply the findings in clinic, with plans underway for a human clinical trial later this year.

Read more:
Vitamin C stops blood cancer in mice - SBS

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