Case study: Treating multiple myeloma
Multiple myeloma is a rare and incurable form of blood cancer which occurs in the bone marrow.
The disease affects the skeleton, resulting in bone pain or fractures, tiredness, shortness of breath and repeated infections. It can be difficult to diagnose as symptoms are generally vague in the early stages - often resulting in delays in diagnosis.
Although existing treatments can be effective at controlling the disease and prolonging life, less than 50 per cent of adults survive for more than five years after diagnosis - so there is a pressing need to develop new myeloma diagnostic techniques and treatments through research.
For people who are fit and well enough, myeloma is usually treated with chemotherapy followed by a stem cell transplant. Unfortunately almost all patients will relapse following this, at some point. After relapse there are a number of treatment options but eventually the disease will become resistant to further therapy.
NIHR research into myeloma includes clinical trials testing innovative new drugs which may have therapeutic benefit for patients.
One such trial, Cardamon, is looking at new treatment approaches for newly diagnosed patients who have not started treatment. The phase 2 clinical trial, which is being supported by NIHR, aims is to establish whether a potentially promising new chemotherapy drug - a cancer growth blocker - is effective in making the disease go into good remissions when used in combination with the two standard drugs. It is also looking at the timing of the stem cell transplant.
Making a difference
Patients treated with this new drug combination may be able to maintain their disease free remission period - the period where they are free from cancerous symptoms - without having a stem cell transplant (the current treatment). This allows stem cells to be stored for later use, when the patient relapses from their myeloma.
Avoiding the need for a stem cell transplant would bring considerable benefits to people with myeloma, allowing the person to return to their lives and regain wellbeing and normal social functioning earlier. It also means patients avoid the risk of severe, potentially life-threatening infection during the transplant process - as well as the long recovery period afterwards.
In October 2015, PhD student Thomas Nealon from Oxford was diagnosed with multiple myeloma. Soon after, he was offered the chance to take part in the Cardamon study.
Taking part in the trial has had a profound impact on the quality of Thomas’s life.
He said: “When I started treatment, I couldn’t get out of bed unaided. Like with all cancers, little problems like tiredness suddenly reach a crescendo with all these different side effects.”
“Thanks to treatment, I can now take public transport, I can wander around London and take the underground. I think that’s a good example of how much better I’m feeling.”
“I can now do all the normal kind of activities required for an office job. I can still enjoy life late into the evening, whereas previously I had to have afternoon naps just to get the energy to do something else.”
Everybody who knows somebody with a condition like multiple myeloma should realise treatments are progressing really fast in this area. It’s made a great improvement in all of our lives.
In this film, Thomas talks about taking part in the trail and how it benefited him:
Cardamon trial Chief Investigator: Professor Kwee Yong from University College London, said:
"We designed the Cardamon study to bring a new and effective treatment to myeloma patients in the UK, in a combination with traditional chemotherapy drugs. The Cardamon study seeks to find out whether patients who receive a highly effective treatment like Carfilzomib can safely delay their stem cell transplant until their myeloma relapses.
"This approach would allow many patients to return to normality and pick up the threads of their lives sooner, without undergoing the risks and suffering of a transplant.
“Of course, not all patients will benefit from such an approach, and in Cardamon, we are looking at the genetic changes in the myeloma cells of patients, to understand which patients will benefit from delaying their stem cell transplant.”
We need to continue to perform research from the laboratory bench to beside so we can quickly turn scientific understanding into tngible benefits for patients
- Dr Jaimal Kothari
- NIHR has supported over 90 myeloma studies - with nearly 50 of these delivered in partnership with the life sciences industry
- Last year alone (2017/18), NIHR had over 40 myeloma studies open on its portfolio
- In 2017/18, NIHR helped nearly 13,000 people access and take part in myeloma studies
- There are around 5,700 new myeloma cases in the UK every year, that's 15 every day (2013-2015)
- An estimated 17,500 people in the UK are living with myeloma at any one time
- Incidence rates for myeloma in the UK are highest in people aged 85 to 89 (2013-2015)