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Beating blood cancer: how decades of research makes us excited about the future

2nd Feb 2020

Last week Professor Pamela Kearns, one of the UK’s leading blood cancer researchers, gave a talk at St James’s Palace about the progress we’ve made in blood cancer research, and why we believe blood cancer can be beaten in the next few decades.

"The progress [research has] made in some types of blood cancer is truly outstanding.

ln the last thirty years, research has driven progress in the treatment of blood cancers. Thousands more people are now surviving after being diagnosed with blood cancer.

[In terms of ten year survival rates] we've gone from three in ten children surviving leukaemia to eight in ten. Myeloma has gone from one person in twenty, to six in twenty. And Non-Hodgkin lymphoma has increased from two in ten to six in ten.

Bloodwise is a major investor in the research that has contributed to this change. I will give you three examples of research that is making a real difference to patients now. I'd also like to say a bit about what the future holds.

Bloodwise supports research to improve how we treat blood cancer starting from how we diagnose the disease through to bringing new innovative drugs into the clinic.

Research can develop new tests to help doctors decide how to treat each patient - and often the difference that good tests makes is profound.

And if you haven't thought of that before, then it may surprise you to know that the huge leap in the numbers of children surviving acute lymphoblastic leukaemia (ALL) has happened despite the fact that there have been no new drugs for 30 to 40 years.

The life-saving improvement has come from learning how to use the treatments we have more effectively. Over the years, what happened was that, step by step, doctors increased the number of children they cured by using more and more intensive chemotherapies and combinations.

Whilst this saved many lives, it did increase the side effects suffered by these young patients. Side effects that sometimes had profound effects on a child for the rest of his or her life.

The questions were raised: 'do all these children need intensive treatment or can some be cured with less?', 'how can we know which children can be cured with less treatment?'

'What if you create a test that means doctors can identify which children need the highest intensity of treatment to survive, and which children need less treatment?'"

Less intensive treatments thanks to the Minimal Residual Disease test

"Today, the test known as the Minimal Residual Disease (MRD) test is helping more patients to have lower dose chemotherapy with all the benefits of reduced side effects.

The MRD test has moved from a research test to being routinely available within the NHS. This success of MRD in childhood leukaemia led to this type of technology being developed for other types of blood cancer, including acute myeloid leukaemia and for some solid cancers too.

MRD is not the only test to identify which patients need different or more intensive treatments. Bloodwise-funded research with a genetics group in Newcastle showed just last year that they can identify in advance a group of children who are at higher risk of relapse with current treatments. This test, and tests like this, will not only allow us to identify which patients need the most intensive treatment, but opens up the potential to identify treatments that are specifically targeted to that leukaemia.

Acute myeloid leukaemia (AML) is a very hard type of leukaemia to treat. Cure rates have improved very little and we're only just starting to see some specific targeted treatments being approved now for some subsets of patients. Getting new drugs approved is very slow.

But bringing new tests into the mainstream sometimes happens quickly."

Transforming acute myeloid leukaemia treatment

"Two years ago, a Bloodwise-funded team at King's College London developed a test that could identify a mutated gene called NPM1, that is defective in some people who have AML.

The Kings team designed a test that could accurately identify the defective NPM1 gene. After checking that the test worked in a clinical trial, treatment for AML is now totally transformed for this group of patients.

The standard treatment approach for AML is several cycles of chemotherapy and for some patients high-dose chemotherapy followed by a stem cell (or bone marrow) transplant.

But the NPM1 test allows doctors to see whether the leukaemia is completely gone or if it's starting to come back.

They can now make a choice that is more specific to each individual patient. lf it's starting to come back, then they can say, ‘we'll need to put you on more aggressive treatment again as fast as we can.'

Or when the NPM1 gene doesn't show up on the test, doctors can say, ‘we don't need to give you a stem cell transplant - we don't need to put you through the risk and trauma of this intensive type of treatment.’

And here's another thing that may surprise you. The speed that all this happened.

The NPM1 test went from a paper in the New England Journal of Medicine, to being available in the pathology labs in London, and then around the country, within a few months. And every week now it's changing conversations doctors have with their patients.

What's happening now?

For blood cancer, drugs have always been the main hope of a cure.

Blood cancer easily moves around the body in the blood stream. So, treatments like surgery and radiotherapy alone cannot cure our patients.

Medicines, like chemotherapy and, more recently, targeted biological therapies, have been the mainstay of treatment usually given in complex combinations and often over long periods of time; for example, 2 to 3 years for ALL. For some blood cancers, when drug treatment alone is not enough, patients undergo a blood stem cell transplant is which is a highly intensive treatment with significant side effects."

"Bloodwise drove the establishment of a brave and exciting new initiative to accelerate the clinical testing of medicines for blood cancers. This became known as the Trials Acceleration Programme and is based within my Clinical Trials Unit at the University of Birmingham.

Through this programme Bloodwise has funded series of 'early phase' trials of new treatment combinations that are showing real promise.

Chronic lymphocytic leukaemia (CLL) is the most common type of leukaemia and is more common in older people. CLL usually develops very slowly which is why it's called a chronic leukaemia - but today there is no cure. However, there are also lots of drugs available now that could be effective in CLL. The challenge for doctors is how best to treat each individual patient? Which drugs should be used in which combinations?

Professor Peter Hillman and his team in Leeds worked closely with the Bloodwise TAP team to bring these drugs to the patients who need them through clinical trials. These trials can tell Professor Hillmen which drug combinations look promising and which don't - and he can rapidly choose the most promising treatments to take forward into much bigger trials to fully test if the drugs work well enough to convince regulators that these new treatments should be made widely available in the UK.

The TAP CLL trials are already producing really promising results and changing people's lives. One of Professor Hilmen's patients, Brian who is 62, and has been on a trial since December 2016 says, ‘I've been able to keep working as a car mechanic and I don't worry about the leukaemia. The drugs on the trial have given me the chance to live the rest of my life, taking just one pill a day.'

And what might the future hold?

"One of the big challenges in blood cancer is that of cancer prevention. You are probably all aware of known risk factors that are associated with an increased risk of developing some cancers, for example; smoking, alcohol, obesity. Unfortunately for most blood cancers, we do not know why a patient develops blood cancer so at the current time we do not know how to prevent it.

What we can do though is think about how we might detect a blood cancer earlier and intervene to stop it becoming so serious. Here is an example:

Some blood diseases are related to one another, and one good example is monoclonal gammopathy of unknown significance (MGUS). MGUS is related to myeloma, a type of blood cancer that grows in our bones. Not everyone who gets MGUS will get myeloma, but everyone who gets myeloma has had MGUS - so if we could find ways to intervene before MGUS turns into myeloma that could make a big difference to the numbers of people living with myeloma.

Professor Chris Bunce in Birmingham is particularly interested in this issue. He and Bloodwise looked at the number of people who were getting myeloma and then at the number of people who were getting MGUS and realised that MGUS was already causing a lot of deaths even on its own.

MGUS is surprisingly common. Above the age of about 50, three in every 100 people will have it. Testing to identify these people would be possible - but to justify that we would need to know more about how MGUS is affecting people's health today, and we would need to have treatment options to offer them.

These are early days, but whatever happens, the next 30 years will be significantly different from where we are now."

Cutting edge treatment: CAR-T therapy

"Looking to the future, there is a huge amount of innovative research into using the body's own immune system to tackle cancer and it is in the blood cancer arena where this innovation has translated into a treatment now available on the NHS: CAR-T cells.

You may have heard of this new treatment in the many news programmes that surrounded the NICE announcement of its approval, making it available on the NHS for some children.

CAR-T therapy is just starting to be made available in children for whom the best current therapies have failed. But the question many parents ask their children's doctors is, ‘when can my child just have CAR-T and not have to have any chemotherapy?' Because the current standard drug treatments work well for the majority of children with ALL, and because CAR-T treatment has not yet been tested to see if it is better than current treatments, it is not appropriate to give ‘front-line’ at diagnosis. lt has only been tested in those children whose disease has failed to respond to all currently available treatment.

The results of CAR-T cell clinical trials have been remarkable and led to the rapid licensing of the drug. But the crucial next step is to design new trials to answer how best to use this new type of treatment. We must answer the questions like, ‘can we use it earlier in treatment?’, ‘Can it be used to replace any of the current treatments?’ We need to design trials to answer these questions to make sure this new treatment is available to the right patients at the right time.

We are entering a whole new era for the treatment of cancer.

We've gone from blasting every cell, including normal cells, with chemicals, to using actual cells from a person's immune system; putting them back into their bodies to help their own cells make the patient better.

This exceptional progress means that there are patients who can now say, ‘I'm alive, when there was nothing left for me.'

And this is just the beginning.

The type of CAR-T that is being used today is just the first. There are dozens of more sophisticated approaches, coming along behind them. Different types of CAR-T, and combinations of CAR-T with other therapies and other novel drug treatments are all being tested.

Bloodwise has been part of this story so far and will continue to invest in innovative research where they can help speed up progress towards curing more patients with blood cancers."

Looking to the future: beating blood cancer

"In 20 or 30 years from now, it is my hope that the question will not be just whether a treatment will stop people dying from blood cancer but how can we do this with the least side effects, so patients can go on to lead normal lives.

This day is coming. It's a matter of ‘when’. Not ‘if’.

We need to work on how fast we can bring all the promising new developments to the 'bedside', to patients.

One answer is that we can't do anything without you and others like you, who want to bring an end to blood cancer sooner.

You are the source of accelerating the work we are doing now and driving it quickly towards new tests and personalised, individual treatments. The speed of progress is directly linked to the amount of money Bloodwise is able to invest in research. And the answer to the question of when we can beat blood cancer is something that the lives of thousands of people rely on.

Together, you, me and the whole Bloodwise family of researchers; scientists and students; Bloodwise ambassadors; local groups and individual supporters can contribute to accelerating progress.

Speed that will bring new tests, treatments and therapies to (hundreds or thousands) of men, women and children as quickly as humanly possible.

Speed that means surviving blood cancer becomes normal for just about everyone.

Speed that changes everything for generations. Just as chemotherapy did 50 years ago."

We fund research to help find cures, better tests and kinder treatments for everyone living with blood cancer. Where we've invested, survival rates and quality of life have improved.