LI-1 Trial

Chief investigator - Professor Robert Hills, Cardiff University
LI-1 Trial: AML Pick A Winner Programme
Amount awarded: £758,553
Award start date: 10 Jan 2014
Recruitment start date: 01 Dec 2011
Award duration: 5 years (60 months)

People with AML and high risk MDS are usually given intensive chemotherapy, but many people over the age of 60 are unable to have intensive chemotherapy because they are too frail. These people are normally given another chemotherapy called cytarabine, which is given at a lower dose than in intensive chemotherapy. Cytarabine was established as the standard-of-care for this group of people by an earlier Bloodwise funded trial (LRF AML14).

Cytarabine (also known as cytosine arabinoside [Ara-C]) is an anti-metabolite, meaning it is very similar to molecules that are naturally found in the body. Because cytarabine’s structure is very similar to deoxycytidine - an essential component of DNA – the drug incorporates itself into the DNA of the cancer cell, preventing cell growth and division. Although cytarabine works well, researchers want to know if combining this treatment with other drugs can boost its effectiveness.

In this trial some people will have cytarabine and others will have cytarabine in combination with newer chemotherapies or biological drugs that are undergoing development. The “Pick A Winner” trial design was developed to rapidly test these new combinations, quickly discontinuing combinations that do not add benefit, and progressing those that do.

Treatment groups include:

  • Cytarabine (standard treatment)
  • Cytarabine and AC220 (a tyrosine kinase inhibitor that blocks signals that cancer cells use to divide and grow)
  • Cytarabine and tosedostat (a new drug that stops signals that cancer cells use to divide and grow)
  • Cytarabine and selinexor (the first drug in a new class of agents known as Selective Inhibitor of Nuclear Export (SINE) compounds. Selinexor works by inhibiting XPO1, a protein found in the nucleus of cancer cells, which causes cell death)
  • Cytarabine and lenalidomide (affects all sorts of cell processes, including how cells divide and grow)

The aim of the trial is to:

  • Compare treatment groups to find out which is best for treating people with AML and high risk MDS

You may be eligible to join this trial if:

  • You have AML or MDS
  • You are 60 years of age or older – some younger patients may be able to take part