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Researchers at the Kennedy Institute, part of NDORMS have repurposed the approved drug miglustat to successfully slow the progression of multiple myeloma.

Researchers at the Kennedy Institute, part of NDORMS have repurposed the approved drug miglustat to successfully slow the progression of multiple myeloma.

Multiple myeloma is the second most common blood cancer and there is no known cure for it. Patients suffering from the condition have debilitating pain caused by bone damage, show tiredness, and are prone to infections.

The condition leads to the accumulation of plasma cells, a type of white blood cell, in the bone marrow, interfering with the production of normal blood cells. In addition, myeloma cells promote the activity of osteoclasts – bone cells that destroy and remove bone tissue.

Whilst incurable, multiple myeloma is currently managed using either drugs that target the myeloma cells thus reducing the burden on the bone marrow or drugs which reduce the activity of the osteoclasts. Occasionally, treatment will involve a mix of different therapies and/or a bone marrow transplant.

The research led by Professor Nikki Horwood used a commercial drug developed by Oxford GlycoSciences for the treatment of Gaucher disease – miglustat – and showed it to act on both aspects of multiple myeloma, slowing disease progression and alleviating its symptoms. Whilst still not a definitive cure, miglustat has clear benefits over current therapies for myeloma and would keep disease at bay for the 5,000 people diagnosed with multiple myeloma each year in the UK

Professor Horwood said: "This research is an important step towards targeting myeloma bone disease on more than one front, the cancer cells themselves and the bone destroying cells, using only one drug, which is a fantastic improvement for patients. Miglustat is a commercial drug already available on the market thus translation into clinical practice could happen quicker.  We are continuing to investigate Miglustat, and related drugs, in multiple myeloma and this research provides the proof of principle platform we needed for clinical translation."

You can read the full paper here.

 

This work was supported by the Multiple Myeloma Research Foundation (AE, NH, AK), Leukaemia and Lymphoma Research (KX, AE, AC, ES, MH, AR, IR, DSA, NH and AK), Arthritis Research UK (NH, LD), Kennedy Institute Trustees (YV) and the Glycobiology Institute, University of Oxford (GT, RAD and TDB).

Image: Left - Control group shows holes straight through the bone (blue background). Right - in the group treated with miglustat these holes are almost completely absent.