Clinical trials and novel drugs

Clinical trials are planned research investigations in which patients take part. They test new drugs or new combinations of current treatments, or compare different ways of using current treatments. They aim to provide reliable evidence about how effective treatments are and whether they should be made available to patients.

There are different types and phases of clinical trials. Each trial is designed to ask and answer specific questions about the treatment involved – for example, is this treatment safe? Is this treatment effective? Is it better than current treatments?

Clinical trial results are used to help governing bodies decide whether the treatment should be available for use on the NHS. For more information about this process download the Health technology assessment Infosheet.

If you’re a healthcare professional who would like to find out more information on clinical trials, see clinical trials in our research section.

What to consider before you take part in a clinical trial

Your doctor may discuss taking part in a clinical trial with you. Before you decide whether to take part in a trial it’s important you understand what’s involved, so you can make an informed decision.

Here are some of the benefits of taking part:

• Getting access to an effective current treatment or a new treatment that might not otherwise be available
• Being closely monitored by specialist doctors and nurses throughout the trial
• Helping researchers improve the treatment given to patients in the future

Other things to consider before you take part include:

• The new treatment might not be better than the current treatment
• The new treatment might cause unexpected side effects
• The new treatment might not work for every patient (although the same could be said for any treatment)

How to participate in a clinical trial

If you’re interested in taking part in a clinical trial, you should speak to your doctor. Every trial has a list of entry criteria and your doctor will need to check you’re eligible to take part.

Remember, taking part in a clinical trial is entirely voluntary. You should definitely speak to your doctor or nurse if you have any questions before making your decision.

What new treatments are being investigated?

Immunotherapies

The immune system is made up of specialised cells, tissues and organs which work together to protect the body from foreign organisms and abnormal cells in the body. Immunotherapies are treatments that help the immune system to kill myeloma cells.
The main types of immunotherapies being investigated for the treatment of myeloma include:

• Monoclonal antibody drugs
• Immune checkpoint inhibitors
• Adoptive T cell transfer
• Oncolytic viruses

Monoclonal antibody drugs

Monoclonal antibody drugs are designed to recognise and attach to specific proteins on the surface of cells. This helps the immune system recognise that the myeloma cell is abnormal and should be killed. Read more about Daratumumab (Darzalex®).

Adoptive T cell transfer

Adoptive T cell transfer involves the removal and genetic modification of T cells – a type of white blood cell – from a patient. The modification enables them to recognise myeloma cells. When they are reintroduced into the patient’s blood, these cells can seek out and kill myeloma cells. Read more in our Adoptive T cell transfer Infosheet.

Proteasome inhibitors

Proteasomes remove, break down and recycle damaged or unnecessary proteins in cells. Proteasome inhibitor drugs prevent them from being able to do this, which leads to a build-up of proteins in cells. Once this reaches toxic levels, the cell dies.

You can find out more about specific proteasome inhibitors being investigated for the treatment of myeloma by downloading the following Infosheets:
Carfilzomib Infosheet
Ixazomib Infosheet

Chemotherapies

Chemotherapy drugs are common in the treatment of myeloma. They work by damaging the DNA in myeloma cells, preventing them from multiplying and causing them to die.

Download the Bendamustine Infosheet.

Selective Inhibitor of Nuclear Export (SINE) compounds

SINE compounds block a particular protein found in cells. This protein is responsible for moving other proteins out of the nucleus of a cell. In myeloma cells, by blocking this protein, other proteins are not moved and the myeloma cell dies.

Download the Selinexor Infosheet.

Pro-survival inhibitors

Pro-survival inhibitors target specific proteins found in cells that prevent cell death. By interfering with these proteins, pro-survival inhibitors cause myeloma cells to die.

Download the Venetoclax Infosheet.