A mum with multiple sclerosis has become the first patient in the UK to receive an experimental treatment that could slow or even stop progression of the disease.
Emily Henders, 37, was given an infusion of her own cells that had been weaponised in the lab to hunt down rogue elements in her immune system that drive the disease’s disabling relapses.
Sky News was given special permission to be present as the cells arrived deep frozen in liquid nitrogen before being carefully thawed and infused into her bloodstream.
“Someone’s got to do it. Someone’s got to be the first,” she told me.
Emily has had MS for four years, but existing drug treatment hasn’t worked. Three times she’s woken to find she’s unable to move her legs.
Her dad also has the disease and is now in a wheelchair.
“I want to avoid getting to that point as much as possible,” she said.
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“The thought of not being able to chase after my children or even walk up and down the stairs is something that I want to avoid at all costs.
“I want to stop it (the disease) before it has a chance to stop me.”
Multiple sclerosis is caused by rogue cells in the immune system turning against the body and attacking the protective covering of nerves in the brain and spinal cord.
It can cause blurred vision or problems with movement, memory or thinking, depending on which nerves have been damaged. There is currently no cure.
But doctors at University College London Hospitals (UCLH) are leading a new global trial of so-called CAR T therapy.
They took blood from Emily and isolated T cells that normally defend the body against infections.
These were genetically modified in the lab to target the rogue B cells in the immune system instead, and then infused back into Emily’s bloodstream.
The hope is that the treatment will stop further damage to the nerves and halt progress of the disease.
Dr Claire Roddie, who has been instrumental in designing the treatment, said it was one of the most significant developments in her clinical lifetime.
“If we can intervene with something that essentially reaches deep into all the areas of the body where the disease-causing cells are, and eradicate them, it is almost like flicking a switch,” she said.
“You get this immune (system) reset where the bad B cells don’t recover, and you get just normal B cells.”
CAR T therapy has already been successfully used to treat – and sometimes cure – aggressive blood cancers by harnessing the immune system to hunt down abnormal cells.
There is now growing interest in also testing it against autoimmune diseases, where the immune system starts to attack the body.
Early-stage clinical trials on patients with the chronic disease lupus are promising.
The MS trial at UCLH will recruit 18 patients at hospitals around the world.
It is primarily designed to confirm the treatment is safe enough to progress to larger studies, but scientists will also monitor the impact on the rogue cells.
Dr Wallace Brownlee, who is the principal investigator of the trial, said: “None of the medications we have available are fully effective at preventing relapses.
“This can be given as a single one-off treatment. It could be a real game-changer in terms of how we manage MS in the future.”
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The form of CAR T cell therapy being tested in the trial was invented by scientists at University College London’s Cancer Institute, and licensed by the UCL spinout Autolus.
It is called Obe-cel and is less likely to have serious side effects on the immune system than the first generation of CAR T therapies.
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Emily is a secondary school biology teacher and understands the scientific rationale for the treatment – but also for the potential of side effects.
“What are my options here?” she said.
“I could just let this disease take over and we could wait and see and I might be lucky and nothing happens to me.
“Or I might have a massive relapse next week and never be able to walk again.
“Are we willing to take that chance? And I think we’ve come to the conclusion that no, we’re not. We have to trust science and trust medicine to do its job.”
There are more than 150,000 people with MS in the UK. Nearly 7,100 are newly diagnosed each year.
The trial, called AUTO1-MS1O, is open to UK patients who have been diagnosed with relapsing or progressive forms of MS and whose disability is worsening despite existing treatment.
Dr Frederick Vonberg, neurology research fellow at UCL and UCLH, said: “We encourage patients aged between 18 and 60 years to speak to their neurologist and explore whether they might be eligible for a referral to this trial.”
