Each year, nearly 6,000 people in the U.S. are diagnosed with ALS. And at any given time, there are at least 20,000 Americans battling with the disease. A person diagnosed with ALS typically survives for 2 to 5 more years, with about 10% surviving for more than 10 years. Within that period, loss of muscle control and functioning ultimately results in not being able to do anything much except lie down and simply continue to breathe.
As hopeless as ALS and other spinal muscular diseases are, there might be a way to prevent the paralysis and muscle degeneration these diseases cause. Based on the results of a study published by the New York University, the solution might be cell reprogramming — an approach that will enable a person to grow new, healthy cells from his/her own stem cells.
Prior to this development, the researchers were able to show how mouse embryonic stem cells can be transformed into motor neurons — the cells responsible for muscle control and sensory reactions. The process required introduction of what are referred to as transcription factors — genes that control the expression of other genes — into the stem cells. After two days, the transformation into motor neurons took place.
To understand how the transformation works, the researchers observed the progression of what happens by checking out the changes and development in the cells once every six hours. What they discovered was a complex process that involved 3 transcription factors and 2 separate processes that eventually merge into 1 single process. At the onset, two of the transcription factors — Isl1 and Lhx3 — work together, while the third factor — Ngn2 — works independently. Later on, Isl1 and Lhx3 become dependent on Ngn2 to complete the process.
Based on these findings, the researchers postulate that direct programming will only work successfully if it is able to coordinate the activity of the two processes, bypassing the progressive changes by instantaneously replacing the gene transcription network with a completely new and functioning one.
According to Penn State Assistant Professor Shaun Mahony, one of the authors of the study, “By detailing the mechanisms underlying the direct programing of motor neurons from stem cells, our study not only informs the study of motor neuron development and its associated diseases, but also informs our understanding of the direct programming process and may help with the development of techniques to generate other cell types.”
Although the direct programming technique might be oversimplifying what is naturally a complex process, the hope is that it can pave the way for the development of other techniques that can eventually allow us to repair damaged cells or replace lost ones by transforming some of our other cells as replacement.