Canadian researchers are making great strides toward finding a successful treatment for spinal muscular atrophy (SMA). The disease typically causes death within the first five years of life, but thanks to Prolactin, a pregnancy hormone, patients may be able to extend life up to 60-percent longer.
While this extension is nowhere near a cure, it is a measurable success for science and medicine.
SMA affects more than 1,000 children in Canada alone. Children with the disease lose motor function and muscle strength. The genetic disorder is triggered by a common gene carried by all patients, but there is a copy gene that does not activate. When this gene is activated it could potentially cure the disease.
The study on Prolactin and SMA is still in animal stages, but the results of the study are positive. When lab rats with the SMA gene were given Prolactin, it activated and regulated the copy gene. The copy gene then started producing SMN protein extending the life of the lab animals by 60-percent. In addition to extending lifespan, Prolactin also increased motor control.
The Prolactin/SMA study should move from animal trials to human trials shortly. Researchers are quick to note that the results, while not curative, are a step in the direction of a viable treatment. Ideally, patients with SMA would be treated before symptoms occur. Increases SMN protein could reduce the advancement of the disease, preserving muscle function and motor control.
There are four types of SMA. Type I progresses quickly leading to death before the age of 5 in most cases. Type IV is the adult form of the disease. There is no mention of how SMN protein or Prolactin therapy would affect adults with SMA or children with a milder form of the genetic disorder.
Source: Journal of Clinical Investigation. CM Lutz, S Kariya, S Patruni, MA Osborne, D Liu, CE Henderson, DK Li, L Pellizzoni, J Rojas, DM Valenzuela, AJ Murphy, ML Winberg, and UR Monani. 25 July 2011.