Those of you who read the Daily Express may have come across the headline “Prostate cancer CURED” (http://www.express.co.uk/life-style/health/738632/prostate-cancer-symptoms-treatment-testosterone-PSA-tumours) on December 1st. Sounds great, but one blogger called Roger was a tad cynical – “like so much cancer research which comes to nothing and is never heard of again”. So was Roger right to doubt it?

The treatment used for the “cure” was first described on January 7th 2015 in the prestigious scientific journal Science Translational Medicine (Vol 7: 269ra2).  The primary and most effective therapy for life-threatening prostate cancer is hormone therapy, which removes nearly all the testosterone from the body. But what these scientists from the world famous John Hopkins Institute in Baltimore did was completely counter-intuitive. They took patients who had failed hormone therapy and continued to give them hormone therapy. Then, once a month, they gave them a massive shot of testosterone to give very high levels of testosterone. So, the men were alternating between having very high levels and very low levels of testosterone. Consequently it was called bipolar androgen therapy (BAT) because of the large swings between high and low levels of testosterone.

Just to make it more complicated, for the first 3 cycles they also gave etoposide – a chemotherapeutic drug. Their reasoning was that high testosterone might kill the cancer cells by attacking the cancer cell DNA and etoposide can also attack their DNA. One of their patients died, probably due to the side-effects of etoposide, of pneumonia and shortage of blood cells.

The bipolar androgen therapy had some benefit, giving PSA responses in 7/14 patients, in 4 of whom the PSA fell to less than 50% of the baseline value and in 5 of whom the cancer appeared to shrink. The scientists felt that this was good evidence that BAT might be useful for men with life-threatening prostate cancer. But this was only an observational study and the next step will presumably be a formal clinical trial where patients are randomised between treatments. 

It then went quiet for 2 years until the scientists described a single patient treated with BAT in the journal European Urology (10.1016/j.eururo.2016.09.020). This patient had 22 months of BAT and has no evidence of disease and undetectable PSA. Men who have failed hormone therapy might expect, on average, another 12 months, so the fact that all the signs are looking good after 22 months is very unusual. Mind you, the scientists are not claiming a cure, just an extreme response.

But this patient is a one-off in more ways than one. He was born with two DNA repair defects in all his cells, one in the gene BRCA2 and one in the gene ATM. These mutations might help to explain his “extreme response”. The other thing that is strange is that etoposide is not mentioned, because that might also be good for patients with DNA repair defects.

So – what’s the conclusion? Certainly the Daily Express journalist went way over the top for the sake of a good story – it was only one patient and the scientists were not claiming a cure. So, I’ve got lots of sympathy for Roger’s view, but I still look forward to this new therapy being properly tested in a clinical trial and hope that maybe it will help a small proportion of men with life-threatening prostate cancer.

Professor John  Masters