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Could stem cell therapy be an effective treatment for Friedreich’s ataxia?

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Posted April 20, 2018

Stethoscope. Image credit: Bru-nO via Pixabay, CC0 Public Domain

The small, pilot study aims to recruit seven people to find out if the bone marrow stem cell mobilising drug, granulocyte-colony stimulating factor (GCSF), could improve blood markers and potentially treat the condition.

GCSF, which is commonly used in diseases related to the blood, as well as in healthy people prior to bone marrow donation, activates stem cells within the bone marrow and causes them to circulate around the body.

People with FA will be invited to take part in the study and if they are suitable, GCSF will be injected just under the skin for five consecutive days. The injections will be at identical doses to those given to ‘healthy’ people prior to bone marrow donation.  Blood samples will then be taken over the subsequent two weeks to find out whether the drug has led to changes in blood markers, which would indicate a positive response to the drug.

The blood samples will be analysed to determine how GCSF changes blood markers that are relevant to FA.  The research team will also analyse the biochemical composition to see if there are changes and how long the change lasts for.

The patient taking part in the study will be assessed by a doctor before the initial drug administration, at the final drug administration and two weeks after the final drug dose.  A further blood test to monitor baseline blood tests will be taken two weeks after the final dose to ensure there have been no effects of GCSF on baseline blood tests.

Dr Alastair Wilkins, Reader in Neurology in the Bristol Medical School (THS) and North Bristol NHS Trust, said: “There has been much excitement and hope over a number of years that stem cell therapies might provide an effective treatment for a variety of neurodegenerative diseases including FA.

“Of all the types of stem cell therapies which have been put forward, we believe that bone marrow stem cell mobilisation currently holds the most promise, not least as these treatments are safe and have been used extensively for many years for other conditions.”

Dr Julie Greenfield, Head of Research at Ataxia UK, added: “We are delighted to be supporting this exciting research study. It is the first trial of this kind in people with Friedreich’s ataxia, a currently incurable condition for which there is an urgent need to develop treatments.”

Paul Howe, a supporter of the charity Ataxia UK and whose niece has the condition, commented: “I’ve watched my niece, Victoria, suffer for years with progressively worsening muscle problems throughout her body.  By taking part in the trial, I hope a treatment can be found for this little-known condition that affects mobility, speech, movement and, which currently has no cure.”

The research team suggest a larger and longer clinical trial should be carried out if the study shows there is an improvement in blood markers and a positive response to the drug.  The pilot study will also help researchers to define the dosage and selection of patients for a future larger clinical trial.

Source: University of Bristol

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