Friday, 19 April 2013

Researchers discover new promising drug for amyotrophic lateral sclerosis

Researchers from the Harvard University reported today that by using a new, stem cell-based, drug-screening technology they have found a compound, called kenpaullone, that is cost-effective and more efficient than current drugs are in treating patients suffering from Amyotrophic Lateral Sclerosis (ALS).

During the study, the researchers first generated a large number of motor neurons, that were derived from stem cells harvested both from human patients and mouse models for ALS. Then, they used a new screening technique to search for molecules that could prolong the survival of these neurons.

A colony of Mouse embryonic stem cells
Mouse embryonic stem cells

The new screening technique was originally developed by Lee Rubin, a member of the Harvard Stem Cell Institute (HSCI) and professor at Harvard’s Department of Stem Cell and Regenerative Biology (SCRB).

"We carried out a small molecule screen using stem-cell-derived ALS motor neurons for the first time, we believe that we have demonstrated the potential value of an entirely new system by which potential ALS therapeutics can be discovered." said Rubin

Their research was very thorough and after trying out more than 5,000 molecules they found a compound, called kenpaullone, that greatly improved motor neuron survival. The study also notes that kenpaullone is more efficient than olesoxime and dexpramipexole are, two drugs that recently failed to pass Phase III clinical trials, due to being ineffective compared to placebo treatment.

"We have shown that there may be value in testing compounds before clinical trials on human ALS motor neurons as an addition, or as an alternative, to simply testing them in mouse disease models.Moreover, we believe that the stem-cell-based method by which we discovered a new ALS drug target might be used broadly in drug discovery for other diseases." said Rubin

At the moment, there is only one approved treatment for ALS (Riluzole) which lengthens survival ratio by only a few months. Hopefully, further research will reveal that kenpaullone is both safe and efficient in treating this deadly disease.

Amyotrophic lateral sclerosis (also known as motor neurone disease and as Lou Gehrig's disease) is a debilitating disease with varied etiology. ALS is characterised by rapid  progression of the following symptoms:
  • Overall weakness
  • Muscle atrophy and fasciculations
  • Muscle spasticity
  • Difficulty speaking
  • Difficulty swallowing
  • Difficulty breathing 

Most patients die from respiratory failure within 3 to 5 years after the onset of symptoms. About 20,000-30,000 people in the United States have ALS, and an estimated 5,000 people in the U.S are diagnosed with the disease each year.

Related Posts

Yang, Y., Gupta, S., Kim, K., Powers, B., Cerqueira, A., Wainger, B., Ngo, H., Rosowski, K., Schein, P., Ackeifi, C., Arvanites, A., Davidow, L., Woolf, C., & Rubin, L. (2013). A Small Molecule Screen in Stem-Cell-Derived Motor Neurons Identifies a Kinase Inhibitor as a Candidate Therapeutic for ALS Cell Stem Cell DOI: 10.1016/j.stem.2013.04.003

1 comment:

  1. A friend of mine got the bad news, newly diagnosed...Fingers crossed <3


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