Last year in the UK over 60,000 cancer patients enrolled on clinical trials aimed at improving cancer treatments and making them available to all.
A trial of panobinostat with bortezomib, thalidomide and dexamethasone for myeloma that has come back or is no longer responding to treatment (MUK Six)
This trial looked at a drug called panobinostat alongside bortezomib, thalidomide and dexamethasone for myeloma.
It was for people whose myeloma had come back or treatment had stopped working.
The trial was open for people to join between 2013 and 2014. The results were published in 2016.
More about this trial
The usual treatments for myeloma are chemotherapy, steroids and targeted drugs. You might have a combination of treatment called VTD. This includes the drugs:
But sometimes treatment stops working or the myeloma gets worse.
In this trial, researchers wanted to see if adding a drug called panobinostat to VTD improved treatment.
Panobinostat is a drug that blocks certain proteins (
The aims of the trial were to:
- find the best dose of panobinostat to have with VTD
- see how well VTD and panobinostat works for people with myeloma
- learn more about the side effects
Summary of results
The trial team found that VTD and panobinostat worked well for people whose myeloma had got worse. And most of the side effects weren’t too bad.
About this trial
57 people took part in this phase 1/2 trial.
The trial was in 2 parts. In part 1, the first few people had a low dose of panobinostat with VTD. If they didn’t have bad side effects the next few had a higher dose. And so on, until they found the best dose of panobinostat.
In part 2, people had this dose of panobinostat alongside VTD.
The researchers found the recommended dose of panobinostat. This was 20mg.
46 people had this dose alongside VTD.
People had treatment in cycles. Each 3 week period was a cycle of treatment. They had an average of 10 cycles. They could have up to 16 cycles.
Of these 46 people:
- 24 stopped treatment early and went on to have a
stem cell transplant
- 20 completed all 16 cycles of treatment
Of the 20 who completed treatment, 15 had more panobinostat for up to a year. The aim was to keep the myeloma under control for longer. This is called maintenance treatment.
4 people were still taking panobinostat when the researchers analysed the results.
They looked at how well treatment worked in all 46 people who had 20mg of panobinostat. They found, in:
- 3 people the myeloma went away completely
- 18 people the myeloma went away quite a bit (a very good partial remission)
- 21 people the myeloma went away a little bit (a good partial remission)
- 2 people the myeloma went away a tiny bit (a minimal partial remission)
- 2 people the myeloma stayed the same (stable disease)
They looked at how long people lived before their myeloma got worse. This is called progression free survival. On average this was 15.6 months.
Most of the side effects were mild to moderate. They included:
- tiredness (fatigue)
- numbness or tingling in the hands or feet
- diarrhoea or constipation
- bone pain
- feeling sick
Some people had more serious side effects. These included:
- an increased risk of infection
- an increased risk of bleeding and bruising
- a low level of phosphate in the blood
- liver changes
- colds and a sore throat
The trial team found a safe dose of panobinostat to have alongside VTD. They concluded that having panobinostat with VTD worked well for people whose myeloma had got worse. Most people were able to tolerate treatment well.
The trial team don’t plan to look at panobinostat in more trials. This is because there are newer drugs available that look more promising.
Where do these results come from
We have based this summary on information from the research team. The information they sent us has been reviewed by independent specialists (
How to join a clinical trial
Professor Jamie Cavenagh
Experimental Cancer Medicine Centre (ECMC)
NIHR Clinical Research Network: Cancer
University of Leeds