Trials Hint At Major Progress In Brain Cancer Treatment

The results of new trials for treating brain cancer in children have shown very promising results, suggesting this may soon be added to the options for helping fight the disease.

Swiss pharmaceuticals firm Novartis has revealed the trial, using a combination of two drugs called Tafinlar and Mekinist, saw tumour shrinkage in 47 per cent of the patients, compared with 11 per cent in a comparative group that was being treated with chemotherapy. Moreover, the time without disease progression nearly trebled to 20 months.

The tests were being carried out on youngsters aged between one and 17 with low grade gliomas (LGGs), which are the most common childhood brain cancer. The drugs were administered orally.

However, the treatment itself was limited to a certain category of patients; they all had a genetic condition called BRAF V600, which is a factor in between 15 and 20 per cent of LGG occurrences in children. 

The drug combination itself has already been approved to treat some other cancer conditions, including skin, lung and thyroid cancer, but again this is focused on those with the BRAF V600 mutation.

While these results have been presented to the American Society of Clinical Oncology (ASCO) at its annual meeting and will now be passed on to regulators in an application for approval, it is therefore clear that they are only applicable to a minority of cases for one particular condition, based on a genetic mutation that accounts for no more than a fifth of LGG cases.

While that is not to understate the benefits of the treatment for those who can benefit from it, it is clear that this is not a catch-all miracle drug. Nor does it provide a permanent cure. This is why using radiotherapy for brain tumour conditions remains for many the most viable and effective form of treatment.

Novartis is far from alone in seeking to develop new pharmaceutical treatments for brain cancer. Oncology drugs maker Kazia Therapeutics has just presented the latest findings of research into paxalisib, a drug designed to tackle brain metastases. 

Phase II trials have shown that patients enjoyed longer survival times, a median of 15.7 months compared with 12.7 months for those using another drug, temozolomide, which the patients in the trial had a genetic resistance to. Progression free survival was also longer, at 8.6 months compared with 5.3 months in the temozolomide cohort. Side effects were few and mostly mild.

This was significant because at present, temozolomide is the only drug approved for this type of condition in the US. 

Kazia CEO Dr James Garner said: “The directionality of these analyses gives us greater confidence in the efficacy signals observed and appear encouraging for future development.”

Like the Novartis results for its combination therapy, the results were presented at the annual ASCO event and will have provided plenty of reasons for optimism going forward. 

One again, however, they are limited results that may offer less of a solution for brain tumour patients than radiosurgery, which as well as being non-invasive will not require continual treatment. These drug developments may be good news, but the possibilities offer by a Gamma Knife may remain far greater.

Novartis says drug combination outperformed chemo in children with brain cancer (NYSE:NVS) | Seeking Alpha

Novartis drug combo shows promise in childhood brain cancer (

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