MPNs or MDS clinical trials

MPNs or MDS clinical trials

We are supporting numerous MPN and MDS trials.

In MPN, we are looking to see if a drug that is already used in breast cancer, will work for people who have MPN who have certain gene changes commonly found in this type of blood cancer. We also want to see if ruxolitinib will work for people with MPN who have relapsed after receiving chemotherapy. Another trial is testing if two drugs that are usually given alone - chemotherapy and ruxolitinib - can be safely given together.

For MDS, we want to improve the way people are treated by fine tuning the drug combinations. We are also supporting trials that want to boost the chances of a successful stem cell transplant, and looking at ways to treat people who have relapsed MDS after a stem cell transplant. And we are finding alternative treatments for older people who have high-risk MDS who are unable to tolerate high intensity chemotherapy.

Myelodysplastic syndromes (MDS)

Treatment options for MDS can vary widely. Some people may just need treatment to control their symptoms, but others may need more intensive treatment like chemotherapy, or even a stem cell transplant. 

A recent trial has shown that a chemotherapy called azacitidine improves survival in people with high risk MDS. But many people in this trial had to have their azacitidine reduced or stopped because of the drug's side effects of low platelet count and bleeding. We are supporting a trial called ELASTIC that wants to see if adding a drug called eltrombopag helps to keep the platelet count up during azacitidine treatment.

We are also supporting trials that want to boost the chances of a successful stem cell transplant, and looking at ways to treat people who have relapsed MDS after a stem cell transplant. And we are finding alternative treatments for older people who have high-risk MDS who are unable to tolerate high intensity chemotherapy.

FIGARO trial

Chief investigator - Professor Charles Craddock, University Hospital Birmingham
Acute myeloid leukaemia (AML) Myelodysplastic syndromes (MDS)
A randomised trial of the FLAMSA-BU conditioning regimen in patients with acute myeloid leukaemia and myelodysplasia undergoing allogeneic stem cell transplantation
This trial is looking at a reduced intensity conditioning regimen called FLAMSA-BU. Researchers think that this treatment could improve outcomes in older people because of reduced side effects and a lower risk of AML or MDS returning. FLAMSA-BU will be compared with standard intensity conditioning.

PRO-DLI

Chief investigator - Dr Victoria Potter, Kings College Hospital, London
Acute myeloid leukaemia (AML) Myelodysplastic syndromes (MDS) Childhood Hodgkin Lymphoma
A phase II prospective trial of prophylactic donor lymphocyte infusions for the prevention of relapse post HSCT in patients with high risk myeloid malignancy
Stem cell transplantation may cure acute myeloid leukaemia (AML) and myelodysplastic syndrome (MDS), but unfortunately people can suffer a relapse after transplant. Researchers want to see if giving white blood cells from the stem cell transplant donor can be used after transplantation to prevent relapse.

RAvVA trial

Chief investigator - Professor Charles Craddock, University Hospital Birmingham
Acute myeloid leukaemia (AML) Myelodysplastic syndromes (MDS)
A randomised phase II trial of 5-azacitidine versus 5-azacitidine in combination with vorinostat in patients with relapsed acute myeloid leukaemia ineligible for salvage chemotherapy
In this trial, researchers want to know if azacitidine given with vorinostat is better than azacitidine alone in people with AML or high risk MDS who are unable to tolerate intensive chemotherapy.

VIOLA trial

Chief investigator - Professor Charles Craddock, University Hospital Birmingham
Acute myeloid leukaemia (AML) Myelodysplastic syndromes (MDS)
A phase I trial of combined azacitidine and lenalidomide salvage therapy in patients with acute myeloid leukaemia who relapse after allogeneic stem cell transplantation
People with AML or MDS are usually treated with high doses of chemotherapy, followed by a stem cell transplant. But AML and MDS can come back after a transplant, and when it does it becomes much more difficult to treat. Researchers want to know if combining azacitidine and lenalidomide can help this group of people.

Myeloproliferative neoplasms (MPNs)

People with MPN can be given a variety of drugs to treat their condition, which includes aspirin, a mild chemotherapy called hydroxycarbamide, or interferon or anagrelide. Many people with MPN have changes in the JAK2 and CALR genes, and drugs that target these changes, such as ruxolitinib, have also been successful. 

But some people are unable to tolerate the side effects of these treatments, so researchers are searching for new ways to treat MPN.

We are supporting a trial that is testing if tamoxifen, which is already used in breast cancer, will work for people who have MPN with the JAK2 and the CALR gene change. We also want to see if ruxolitinib will work for people who have relapsed after receiving chemotherapy. Another trial is testing if two drugs that are usually given alone - chemotherapy and ruxolitinib - can be safely given together.