Plant extract hope for infant motor neurone therapy

March 4, 2014, University of Plymouth

(Medical Xpress)—Researchers from Plymouth University Peninsula Schools of Medicine and Dentistry have been part of an international team led by the University of Edinburgh, who have identified that a chemical found in plants could reduce the symptoms of a rare muscle disease that leaves children with little or no control of their movements.

Their study is published today (3rd March 2014) in the Journal of Clinical Investigation.

The research team have found that a plant pigment called quercetin – present in some fruits, vegetables, herbs and grains – could help to prevent damage to the nerves associated with the childhood form of , (SMA).

SMA, also known as 'floppy baby syndrome', is a leading genetic cause of death in children. It affects approximately one in 6,000 to 10,000 children and around half of children with the most severe form will die before the age of two. There is currently no cure for this kind of .

The condition is caused by a mutation in a gene that is vital for the survival of that connect the brain and spinal cord to the muscles, known as motor neurons. Until now, it was not known how the mutation damages these cells and causes disease.

The study reveals that the mutated gene affects a key housekeeping process that is required for removing unwanted molecules from cells in the body. When this process doesn't work properly, molecules can build-up and cause problems inside the cells.

Children with SMA experience progressive muscle wastage and loss of mobility and control of their movements. The disorder is often referred to as '' because of the weakness that it creates.

The team has found that the build-up of a specific molecule inside cells – called beta-catenin – is responsible for some of the symptoms associated with the condition.

In tests on zebra fish, mice and fruit flies (the latter in Plymouth), scientists found that treating the disease with quercetin – which targets beta-catenin – led to a significant improvement in the health of nerve and muscle .

Although quercetin did not prevent all of the symptoms associated with SMA, researchers hope that it could offer a useful treatment option in the early stages of disease.

Professor Tom Gillingwater from the University of Edinburgh, who led the study, said: "This is an important step that could one day improve quality of life for the babies affected by this condition and their families. There is currently no cure for this kind of neuromuscular disorder so new treatments that can tackle the progression of disease are urgently needed."

Dr. Iain Robinson, Associate Professor in Neuroscience at Plymouth University Peninsula Schools of Medicine and Dentistry, added: "By working to understand how SMA operates at a molecular level we have been able to apply a naturally occurring compound which targets the one of the key culprits, with the potential to introduce an effective drug therapy for children in the early stages of the disease."

Explore further: New information about the causes of 'floppy baby' syndrome discovered

Related Stories

New information about the causes of 'floppy baby' syndrome discovered

June 28, 2012
(Medical Xpress) -- New information on the potential cause of Spinal Muscular Atrophy (SMA), known as “floppy baby syndrome”, has been discovered by cell biology experts at the University of St Andrews.

New research sheds light on childhood neuromuscular disease

November 20, 2012
A study by scientists at the Motor Neuron Center at Columbia University Medical Center suggests that spinal muscular atrophy (SMA), a genetic neuromuscular disease in infants and children, results primarily from problems ...

Researchers identify genetic mutation causing rare form of spinal muscular atrophy

May 10, 2012
Scientists have confirmed that mutations of a gene are responsible for some cases of a rare, inherited disease that causes progressive muscle degeneration and weakness: spinal muscular atrophy with lower extremity predominance, ...

Study provides new insights into cause of human neurodegenerative disease

December 18, 2013
A recent study led by scientists from the National University of Singapore (NUS) opens a possible new route for treatment of Spinal Muscular Atrophy (SMA), a devastating disease that is the most common genetic cause of infant ...

Novel mechanisms underlying major childhood neuromuscular disease identified

October 11, 2012
A study by scientists from the Motor Neuron Center at Columbia University Medical Center (CUMC) suggests that spinal muscular atrophy (SMA), a genetic neuromuscular disease in infants and children, results primarily from ...

Low oxygen levels may decrease life-saving protein in spinal muscular atrophy

August 21, 2012
Investigators at Nationwide Children's Hospital may have discovered a biological explanation for why low levels of oxygen advance spinal muscular atrophy (SMA) symptoms and why breathing treatments help SMA patients live ...

Recommended for you

Researchers illustrate how muscle growth inhibitor is activated, could aid in treating ALS

January 19, 2018
Researchers at the University of Cincinnati (UC) College of Medicine are part of an international team that has identified how the inactive or latent form of GDF8, a signaling protein also known as myostatin responsible for ...

Bioengineered soft microfibers improve T-cell production

January 18, 2018
T cells play a key role in the body's immune response against pathogens. As a new class of therapeutic approaches, T cells are being harnessed to fight cancer, promising more precise, longer-lasting mitigation than traditional, ...

Weight flux alters molecular profile, study finds

January 17, 2018
The human body undergoes dramatic changes during even short periods of weight gain and loss, according to a study led by researchers at the Stanford University School of Medicine.

Secrets of longevity protein revealed in new study

January 17, 2018
Named after the Greek goddess who spun the thread of life, Klotho proteins play an important role in the regulation of longevity and metabolism. In a recent Yale-led study, researchers revealed the three-dimensional structure ...

The HLF gene protects blood stem cells by maintaining them in a resting state

January 17, 2018
The HLF gene is necessary for maintaining blood stem cells in a resting state, which is crucial for ensuring normal blood production. This has been shown by a new research study from Lund University in Sweden published in ...

Magnetically applied MicroRNAs could one day help relieve constipation

January 17, 2018
Constipation is an underestimated and debilitating medical issue related to the opioid epidemic. As a growing concern, researchers look to new tools to help patients with this side effect of opioid use and aging.

0 comments

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.