Current Clinical Trials in Leukemia Research


Leukemia is a progressive disease of the body’s blood-forming tissues including bone marrow and the lymphatic system. In leukemia the bone marrow produces abnormal white blood cells which do not function properly. The proliferation of these abnormal leukocytes displaces normal blood cells, disrupting the body’s ability to fight infections, transport oxygen and control bleeding. 

Clinical trials for leukemia are research studies designed and implemented by specialists to improve therapies and treatment for those suffering from blood cancers. They exist to diagnose and treat cancer, relieve side effects, prolong remission and enhance quality of life. Trials that are more effective than current standard treatments and have fewer side effects are good candidates for approval by the U.S. Food and Drug Administration as novel therapies. Most standard treatments for cancer have grown out of clinical trials. Participating in a clinical trial might be the best option for someone with leukemia. 

Leukemia is classified as myeloid or lymphoid depending on the type of white blood cell that composes the cancerous cell. Further, they are categorized as acute or chronic contingent on how rapidly the disease progresses. Acute forms develop quickly, producing large numbers of abnormal cells in a short period, chronic leukemias progress more slowly over time.The four main types of leukemia include acute lymphocytic leukemia (ALL), chronic lymphocytic leukemia (CLL), acute myelocytic leukemia (AML), and chronic myelocytic leukemia (CML). Current clinical trials target all four of these categories. We highlight two current studies related to CLL below.

The Mayo Clinic is currently running an interventional trial to assess the efficacy of obinutuzumab and chlorambucil compared with ACP-196 in combination with obinutuzumab for the treatment of previously untreated chronic lymphocytic leukemia. Obinutuzumab is used in conjunction with chlorambucil as a first-line treatment for CLL. ACP-196 is an orally administered BTK inhibitor with a good side-effect profile. This drug is in clinical trials for CLL patients and many patients have reached remission taking ACP-196 alone. UCLA Hematology/Oncology is currently studying the effects of ACP-196 versus ibrutinib in previously treated subjects with high risk chronic lymphocytic leukemia. In studies on CLL cells, ibrutinib has been reported to promote apoptosis, inhibit proliferation, and prevent CLL cells from responding to survival stimuli.

Translating basic research into the clinical setting and advancing the understanding of clinical or population-based research is facilitated by the availability of a repository of tissue samples alongside accompanying pathogenic and clinical data procured from patients with leukemia as well as from normal controls. Tissue procurement resources for blood and bone marrow coupled with such data offer the opportunity for true translational research and medicine, facilitating the design of novel protocols and resulting in new treatments that more effectively target leukemia. Clinical trials offer the possibility for leukemia patients to participate in and benefit from the development of new treatments. Current advances in clinical research, when combined with tissue procurement and accompanying data, can be augmented with basic research to create translational medicine with astonishing power.