Breakthrough that means most lethal cancer is a death sentence no more: How miracle drug that makes tumors disappear has stunned doctors

Until recently, the future for Sara Sjölund seemed, at best, uncertain. Having been diagnosed with brain cancer in 2018, at the age of just 38, the businesswoman from London had undergone a raft of treatments, to no avail.

Radiotherapy, chemotherapy, surgery – all failed to halt the cruel march of the disease that hits more than 12,000 Britons a year and vanishingly few survive. Despite her medical team’s best efforts, her cancer – a type known as an astrocytoma – kept growing.

While the outlook for many cancers, from breast to skin and even lung cancer – once considered a death sentence – have improved dramatically over the last few decades, the odds for those with a brain tumour remain stubbornly poor.

Overall just one in ten patients are alive ten years after diagnosis. This is partly to do with the speed brain cancer often spreads, but also due to a lack of treatments able to successfully combat it.

Sara Sjölund was offered the chance to trial an experimental new treatment being pioneered by Dr Paul Mulholland, a brain cancer specialist at University College London – and it has proved remarkably successful

By the middle of last year, Sara was out of options: it was hoped chemotherapy would slow the cancer’s progression. But that was as good as it would get. ‘It was disconcerting,’ admits Sara.

‘The chemotherapy I was on is considered the gold standard, but it’s older than me – I was furious that there was nothing else, and nothing more could be done.’

Today, however, there is cause for hope.

She was offered the chance to trial an experimental new treatment being pioneered by Dr Paul Mulholland, a brain cancer specialist at University College London – and it has proved remarkably successful.

The breakthrough treatment plan uses ipilimumab – an immunotherapy drug given intravenously that is already used on the NHS to treat skin cancer – to first shrink the tumour.

The drug ‘takes the breaks off the immune system’, according to experts, helping it seek out and destroy cancerous cells wherever they are.

Patients are then offered surgery to remove what’s left of the growth, or chemo and radiotherapy, or both.

The hope, say experts, is that the radical approach will leave patients cancer-free.

So far, just a tiny handful of patients have been offered it but Dr Mulholland, backed by the National Brain Appeal charity, plans to launch a full-scale trial at the beginning of next year.

After six months on ipilimumab, Sara’s tumour is all but gone and ‘inactive’, according to Dr Mulholland. Sara herself describes it as ‘like a dead tree, it is still there but at the moment it does not look like it will regrow’.

Previously, she said that she ‘lived in fear and uncertainty of the cancer growing’.

But the treatment has given her a new lease of life.

Following the initial rounds of chemotherapy she had to take a break from her job at a media company. Now she plans to write a book about her journey and has founded a new company.

Dr Mulholland says: ‘Sara’s scans show remnants of the tumour. As long as it stays as it is she will be fine.’ Although Sara still has cancer – it’s far too early to know the outcome of her treatment – she feels like she has dodged a bullet. ‘I got incredibly lucky,’ she says. ‘I feel well. I’ve been given my life back.’

There is huge excitement around immunotherapy for brain cancer.

Sara speaking at a fundraiser. The treatment has given her a new lease of life and she now plans to write a book about her journey

‘It is not out of the question that it could be a cure,’ says Dr Mathew Clement. from the Cancer Research Centre in Wales.

‘We know ipilimumab is effective for other cancers and we have shown that we can apply them to treat brain tumours. We could see this treatment offered on the NHS within five years.’

And Sara’s isn’t the only astonishing story of recovery to have emerged thanks to the new breakthrough.

Earlier this year Ben Trotman, 41, became the first person with glioblastoma – an aggressive form of brain cancer that kills most patients within nine months – to undergo treatment with Dr Mulholland. In May 2018, former Labour MP and Culture Secretary Dame Tessa Jowell died after a year-long battle with glioblastoma.

Earlier this year Ben Trotman, 41, became the first person with glioblastoma – an aggressive form of brain cancer that kills most patients within nine months – to undergo treatment with Dr Mulholland

After choosing the trial drug Ben and his partner Emily brought their wedding forward and married last January

She used her final speech in the House of Lords – during which she received a poignant standing ovation – to call for more funding to develop better treatments for brain cancer.

The same kind of cancer also killed pop star and father-of-two Tom Parker at the age of just 33, in March 2022. He had only been diagnosed in October 2020.

In November that year, Ben received his first dose of immunotherapy.

Recalling the events that led to his diagnosis a month before that, Ben, an investment banker from West Sussex, says: ‘I was visiting the optician in Brighton when I suffered a seizure.

‘I remember walking along the High Street and feeling light-headed, and then struggling with the stairs leading up to the shop.

‘The next thing I knew, I was coming round in an ambulance being rushed to A&E.’ At the hospital, scans revealed a damaged area, or lesion, inside Ben’s skull – which was belatedly confirmed to be a brain tumour.

Ben’s wife Emily, who was with him throughout, says: ‘It was chaos. We were referred to a neurologist and a surgeon. No one could give us a straight answer about what was wrong. In December, we rushed Ben back to A&E when he began suffering a terrible headache. The doctor came back after more scans and said he’d had a bleed around the site of his tumour, which was the first time anyone had actually mentioned cancer.’

Frustrated, Emily began researching online and sent an email to the National Hospital for Neurology and Neurosurgery, where Dr Mulholland also works, asking if anyone there could help.

She says: ‘I found a generic email address and wrote to that – and we got a reply back from Dr Mulholland, who arranged a video appointment the next day. We feel incredibly lucky.’

It was at that point the full terrifying reality of Ben’s diagnosis became apparent. ‘Dr Mulholland said it was likely it was a neuroblastoma and offered Ben the chance to take part in the immunotherapy trial,’ says Emily.

‘The next day, we saw the team in Brighton who said Ben could have surgery, then radiotherapy and chemo but the typical life expectancy was 18 months.’

Choosing the trial drug was an easy decision. Initially, there was surgery to remove the tumour. Then two rounds of ipilimumab, which is given via intravenous infusion over the course of day, were planned.

Ben only had one of these. ‘I reacted quite badly to the first treatment,’ he says.

‘I developed this terrible, stabbing headache that made me want to vomit.’

He was admitted and given powerful steroid medication to control the headache. ‘On the upside, Dr Mulholland told us the reaction showed the treatment was working.’ The couple, who were engaged at the time, decided to rush forward their wedding to January 2023. ‘I have no idea how we did it,’ says Emily. ‘But it was a magical day.’

And then came the miraculous news: scans showed Ben’s tumour had disappeared.

He has since undergone intensive radiotherapy and is still undergoing a week of chemotherapy once a month. However, life is returning to some semblance of normality.

‘I have a scan every three months,’ says Ben. ‘They say it’s clear or no visible tumour. But they also tell me that, although they can’t see anything, that doesn’t mean the disease is gone.’

Emily says: ‘Ben’s been told he still has incurable cancer – that hasn’t changed and he’s not been given the all-clear. But every time we are told the tumour hasn’t come back, it’s a huge win.

‘The idea that he could be cured is probably too much to think about at the moment.’

Ben adds: ‘Life is better than we ever could have imagined. I have been able to get married and I am living normally. I am living proof this treatment works.’

The same treatment programme was also used successfully this year for Professor Richard Scolyer in Australia, advised by Dr Paul Mulholland, following Ben’s recovery.

The same treatment programme was also used successfully this year for Professor Richard Scolyer in Australia

18 months after his diagnosis with a glioblastoma Professor Scolyer’s tumour has completely disappeared, already doubling the average life expectancy for the disease

Remarkably, 18 months after his diagnosis with a glioblastoma Professor Scolyer’s tumour has completely disappeared, already doubling the average life expectancy for the disease (see box below).

Part of the challenge is that most cancer drugs can’t cross the blood brain barrier – a wall of dense cells and blood vessels that protects the brain from any invaders in the blood that could cause damage. However immunotherapy, such as ipilimumab, doesn’t need to, says Dr Mulholland.

These drugs work by helping the body’s fighter T-cells to spot and attack tumours – bypassing the age-old problem.

The forthcoming clinical trial will focus on offering the drug as early as possible after patients are diagnosed, in the hope that is the key for finding a cure. Sara only received the drug after standard treatment had failed but with Ben and Professor Scolyer, they were able to use it straight after diagnosis and their tumours have been cleared.

Experts believe the greatest benefit could come from giving the drug before standard treatment begins.

‘What we want to do is get patients early on in the disease before the body is weakened by chemotherapy and radiotherapy. Essentially we’re saying, let’s protect the immune system so it has the best shot at fighting the cancer,’ says Dr Mulholland. However, some experts warn that it is too early to tout success for these drugs, due to the small number of patients who have been treated.

‘Currently there is no good data to show that these drugs work on a large scale. We aren’t at a stage where we can say this works,’ says Dr Matthew Williams, a clinical oncologist at Imperial College London. ‘We fall into the trap of a hype disappointment cycle, where we overpromise and underdeliver.

‘There are exciting developments but it is very much a work in progress at this stage.’

It is hoped that the clinical trial set to start next year will provide the data required to see the treatment rolled out on the NHS.

Experts say one of the challenges they face in carrying out the necessary research is a lack of funding for brain cancer.

Dr Mulholland’s research team had previously tried to trial a widely available cancer vaccine that he believes could unlock a new treatment for glioblastoma.

However, the drug company that makes it refused to supply the medicine at a reduced rate in order to make research possible.

‘The vaccine – in the same way the one for Covid worked – uses genetic material from the patient’s brain tumour to stimulate an immune system response that clears the cancer.

‘In a small American study published earlier this year, researchers at the University of Florida found it was successful. This approach is also being used successfully to treat skin cancers.

‘The problem is, glioblastoma affects relatively few people a year in the UK and is still classed as a rare disease.

‘The pharmaceutical industry tends to focus attention on the common cancers, which is understandable. However, we need to incentivise the industry to prioritise these forgotten-about cancers, such as glioblastoma, too,’ says Dr Mulholland.

Running the campaign for greater funding and access to drugs for clinical trials in the UK is Dame Siobhain McDonagh, whose sister Baroness Margaret McDonagh died last year from glioblastoma.

The Baroness was also a patient of Dr Mulholland and had her life extended by two years after taking a course of immunotherapy drugs, and next year’s clinical trial will be named in her honour.

In May 2018, former Labour MP and Culture Secretary Dame Tessa Jowell died after a year-long battle with glioblastoma

‘Having cared for my sister for 18 months whilst my sister suffered I know just how poor the current treatment options are,’ says Dame Siobhain, the Labour MP for Mitcham and Morden.

Following her sister’s diagnosis she has taken up the mantle of Dame Tessa Jowell.

‘The disease has been left at the ‘bottom of the pile’ for too long,’ says Dame Siohbain.

She has been lobbying the Government to introduce new laws to guarantee funding and regular clinical trials for glioblastoma.

‘To make real advancements we need to work hand in hand with the NHS, pharmaceutical companies and the Government.

‘In the UK we are great at science and developing drugs.

‘We could be at the forefront of developing drugs for a disease that is currently a death sentence.’