Liz Burtally
Posted by

Our Trials Acceleration Programme – fast-tracking clinical benefits to patients

Liz Burtally
Posted by
16 Dec 2016

If we think a drug could save lives, we want to be able to test as quickly as possible whether it’s safe and works better than current treatments. Find out how our Trials Acceleration Programme is helping us achieve this.

The problem

Before we set up our Trials Acceleration Programme (TAP), a researcher could have a great idea, but might not have everything they needed to get a trial off the ground. This meant that it was slow and complicated to test potentially life-saving new treatments.

Doctor and patient. Credit: Wellcome Images.

Clinical trials are expensive to run, and can take many years to complete because of the paperwork involved and the difficulty in patient recruitment.

If we think a drug could save lives, we want to be able to test as quickly as possible whether it’s safe and works better than current treatments. And for patients who often have no other hope of a cure or life-extending treatment, being active in this research can give them access to promising new treatments.

The solution

In 2011 we set about finding a solution.

TAP uses a hub and spoke model to navigate the logistical steps in setting up and delivering a clinical trial. The hub contains dedicated experts skilled at setting up and running clinical trials, meaning researchers and trial centres are free to do what they do best: coming up with new treatment ideas and supporting patients through the clinical trial. Linked to the hub is a network of hospitals that carry out research, meaning that more than 20 million people in the UK could have access to the very latest promising blood cancer treatments if they needed it.

Nurse preparing for a clinical trial. Credit: Wellcome Images.

So far nineteen trials have been adopted into the network – thirteen are currently open, one is in setup and five have now been completed. To date, over 876 patients have taken part in TAP clinical trials.

Impact so far

Finding the right combination therapies in CLL and AML

Chronic lymphocytic leukaemia (CLL) is characterised by a build-up of faulty versions of white blood cells, called B cells, in the bone marrow and the blood, crowding out the healthy B cells and other blood cells. Ibrutinib is a new targeted drug that is already showing great promise in patients with CLL, as demonstrated in our IciCLLe trial, which was led by researchers at our Leeds TAP centre. But some patients will experience side-effects, or start to become resistant to ibrutinib after prolonged treatment.

CLL cells. Credit: Wellcome Images.

In the IciCLLe extension study, researchers assessed a small group of patients with CLL who had received iburtinib with a biological therapy called obinutuzumab for one month. Early results suggest that adding obinutuzumab, another targeted drug, to ibrutinib can lead to big drops in CLL cells in the bone marrow and blood after just one month of treatment. Combination treatment appeared to be safe and patients could withstand the treatment. These early results look promising for patients with CLL who aren’t doing well on ibrutinib, and we will be watching the progress of the trial closely.

The RAvVA trial tested azacitidine and vorinostat in people who can’t have intensive chemotherapy for acute myeloid leukaemia (AML) or high risk myelodysplastic syndrome (MDS). Although a new drug called azacitidine is already used to treat people with AML who can’t have intensive treatment, patients inevitably relapse, or experience just a modest response. Vorinostat is a type of biological therapy that blocks cancer growth, and earlier studies have shown that it can improve the activity of azacitidine.

AML cells. Credit: Wellcome Images.

After six months of treatment, researchers saw that patients didn’t respond better to azacitidine when given vorinostat. Even though these trial results aren’t what were expected, trials with negative results really matter because they tell us what doesn’t work in a particular patient population — which can be as important as knowing what does. We hope that patients would be comforted by the idea that contributing to a trial, regardless of how it turns out, is really important.

Newly opened trials

We are excited about our three trials that opened this year: CLARITY, BUBBLE and TAMARIN.

Nurse with patient. Credit: Wellcome Images.

Finding new treatment options for people with CLL, myeloma and MPNs

CLARITY is looking at people with CLL that has come back or treatment has stopped working. Researchers want to know if combining ibrutinib with a new targeted drug called venetoclax, will be a useful treatment for CLL. The aims of the trial are to find out how well ibrutinib and venetoclax work in patients with CLL, and to find out more about the safety and side effects of this drug combination.

BUBBLE is looking at treatment for people whose myeloma has come back or treatment has stopped working. Researchers are looking for certain gene changes in their myeloma cells, which predicts whether the myeloma is more likely to come back or can lead to the chemotherapy to stop working. Bortezomib is one of the usual biological therapies for myeloma, and researchers want to know if combining this with a new drug called buparlisib will help people with these specific gene changes. BUBBLE aims to find the best and safest dose of buparlisib combined with bortezomib, and is the first trial in myeloma to aim to develop a specific treatment approach based on individual’s biological profile.

Myeloma cells. Credit: Nephron.

TAMARIN is looking at people with a type of blood disorder called myeloproliferative neoplasms (MPNs), where the bone marrow produces too many blood cells. Most treatments for MPNs aim to control the number of blood cells in the blood. Many people with MPNs have changes to certain genes called JAK2 and the CALR, which affects how blood cells are made. There are already some treatments that reduce the number of cells that have these gene changes, but some people can’t cope with the side effects. Tamoxifen is a drug currently used to treat breast cancer, and studies in the laboratory show that it is able to reduce the number of abnormal blood cells, and this happens most in the blood cells carrying the JAK2 and the CALR gene changes. Doctors want to see if adding tamoxifen alongside the usual treatment for MPN helps to reduce the number of cells carrying genetic changes.

We are also excited to announce a new trial called AVAIL-T, which is looking at people with peripheral T-cell lymphoma (PTCL), which is an aggressive and difficult to treat type of non-Hodgkin lymphoma. AVAIL-T will be the first trial to test a new immunotherapy drug called avelumab in patients with PTCL that has come back, or treatment has stopped working. Avelumab is an immunotherapy and works by harnessing the body’s immune system to mount an immune response against cancer, and researchers want to know how well it works in patients with PTCL, and if there are any side-effects associated with the treatment.

We hope you enjoyed the update on our TAP trials. We are expected more exciting results next year, so watch this space!

Nurse looking for supplies. Credit: Wellcome Images.

Why we need your support

We've invested £8 million in TAP so far, because we know it works.

But TAP has been a victim of its own success. We know that TAP works and now researchers know it too, so more and more of them are coming to us asking for support to get their trial up and running.

TAP has proven its worth, and we are grateful to our supporters for helping us transform the way we treat patients. We recently have committed three more years of funding to our TAP trials totalling nearly £2 million. But we need your support to ensure that we can continue to bring hope to patients, where before there was none.

Find out how you can help us continue with our life-changing research.

Find out more about our TAP trials


Related blogs