Zebra fish point the way towards new therapies for amyotrophic lateral sclerosis

August 27, 2012

Leuven scientists (VIB/KU Leuven) are using zebrafish as a model in their search for genes that play a role in the mechanism of amyotrophic lateral sclerosis (ALS). As a result, they have identified a molecule that could be the target for a future ALS treatment. ALS is a progressive degenerative motor neuron disease for which there currently is no treatment. Their study has been published in the prestigious journal Nature Medicine.

ALS is a progressive paralyzing disease caused by the destruction of the connecting to the muscles. As a result, ALS patients gradually lose control over their muscles and eventually become completely paralyzed while their mental capacity remains intact. In spite of the enormous medical and social impacts of this grave degenerative illness, the mechanisms behind its remain a mystery. There is no known treatment.

Using an unusual model for ALS that these scientists developed earlier, Wim Robberecht's research team searched for genes that either worsened or improved the phenotype of the disease. In the current study, Annelies Van Hoecke and Wim Robberecht and their colleagues identified the EphA4 receptor as a that modifies the clinical picture of ALS in zebrafish. Eliminating this receptor in zebrafish led to the disappearance of the illness, while blocking the receptor in mice resulted in a clear improvement of the animals' . The research also showed a link in ALS patients between the expression of the EphA4 receptor and the severity of the illness. ALS patients who express the receptor in limited amounts develop the disease later and have a better life expectancy than ALS patients who produce large amounts of the receptor. They also found that EphA4 prevented neurons from recovering from damage and that the cells that were actually the most susceptible to ALS expressed high levels of the receptor.

This study is very promising and proves that small model organisms such as zebrafish can be valuable tools in the search for a therapy for ALS. It also shows that molecules that are essential for the development of the nervous system can play a role in the mechanism of neurodegenerative disorders in adulthood. Finally, the results suggest that blocking EphA4 modifies the course of the disease, meaning that the receptor may be a good target for a future ALS drug. Of course, there is still a long road ahead before such a drug will be available to patients.

Explore further: Scientists identify new gene that influences survival in amyotrophic lateral sclerosis

Related Stories

Scientists identify new gene that influences survival in amyotrophic lateral sclerosis

August 26, 2012
A team of scientists, including faculty at the University of Massachusetts Medical School (UMMS), have discovered a gene that influences survival time in amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig's disease). ...

Disease progression halted in rat model of Lou Gehrig's disease

December 12, 2011
Amyotrophic lateral sclerosis (ALS; also known as Lou Gehrig's disease) is an incurable adult neurodegenerative disorder that progresses to paralysis and death. Genetic mutations are the cause of disease in 5% of patients ...

Potential new drug target in Lou Gehrig's disease

November 14, 2011
Two proteins conspire to promote a lethal neurological disease, according to a study published online this week in the Journal of Experimental Medicine.

A drug-screening platform for ALS

August 2, 2012
A research group at the Center for iPS Cell Research and Application (CiRA) at Japan's Kyoto University has successfully recapitulated amyotrophic lateral sclerosis (ALS)-associated abnormalities in motor neurons differentiated ...

Recommended for you

Lactic acid bacteria can protect against Influenza A virus, study finds

December 13, 2017
Lactic acid bacteria, commonly used as probiotics to improve digestive health, can offer protection against different subtypes of influenza A virus, resulting in reduced weight loss after virus infection and lower amounts ...

Lyme bacteria survive 28-day course of antibiotics months after infection

December 13, 2017
Bay Area Lyme Foundation, a leading sponsor of Lyme disease research in the US, today announced results of two papers published in the peer-reviewed journals PLOS ONE and American Journal of Pathology, that seem to support ...

Aging impairs innate immune response to flu

December 13, 2017
Aging impairs the immune system's response to the flu virus in multiple ways, weakening resistance in older adults, according to a Yale study. The research reveals why older people are at increased risk of illness and death ...

Drug blocks Zika, other mosquito-borne viruses in cell cultures

December 12, 2017
If there was a Mafia crime family of the virus world, it might be flaviviruses.

Study seeks to aid diagnosis, management of catatonia

December 11, 2017
Catatonia, a syndrome of motor, emotional and behavioral abnormalities frequently characterized by muscular rigidity and a trance-like mental stupor and at times manifesting with great excitement or agitation, can occur during ...

New compound stops progressive kidney disease in its tracks

December 7, 2017
Progressive kidney diseases, whether caused by obesity, hypertension, diabetes, or rare genetic mutations, often have the same outcome: The cells responsible for filtering the blood are destroyed. Reporting today in Science, ...

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.