Apr 25, 2017 10:27 AM EDT
Harvard scientists from the Stem Cell Institute recently found that amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA) can be treated by a common drug. In 2012, Harvard Medical School discovered the link between ALS and SMA, which lead them to a conclusion that treatment for both fatal conditions can be common. Now, five years later, they found that common drug might do just the trick.
A group of Harvard scientists identified a compound that protects the cells damaged by SMA, Harvard Gazette reported. Five years ago, scientists from the same institution concluded that the two fatal diseases are linked therefore; their treatment could be applied to the other. It's the discovery of the key compound just this recently that is offering hope to the patients and the family of those who have ALS or SMA.
This breakthrough is very significant in the quest of finding the treatment for both ALS and SMA. Most of the approaches have been to fix a dysfunctional gene, which is very common in SMA patients. SMA patients are unable to produce a very special protein called survival of motor neuron (SMN).
But the scientists from Harvard identified a compound that stabilizes the SMN both in the Petri dished human neurons and in mouse subjects. They discovered that motor neurons produce SMN and these neurons are more likely going to survive from toxic environments and stressors. This is also the case for ALS patients.
So what they learned is that SMN protein is necessary for the survival of motor neurons. According to Lee Rubin, this discovery opens up a line of new treatment. If the amount of SMN protein is increased, the motor neurons targeted by ALS or SMA can be protected.
A common drug that controls SMN protein stability can do the trick. This might also be relevant in the treatment of Schizophrenia as a new study suggests that the neurodegenerative disease is linked to ALS, ALS News Today reported. But it's quite improbable as ALS is a motor disease whereas Schizophrenia is a mental illness, hence more study is needed.
Meanwhile, Rubin's team has already filed a patent application for a common drug that promotes survival of all motor neurons. As to when this treatment can be made available or will it ever be made available remains to be a mystery.
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