ALS patients: cell reprogramming opens the door to future treatments

Worldwide, there are between 5,760 and 6,400 new diagnoses of amyotrophic lateral sclerosis.

Better known by its acronym ALS, each year. It is a progressive disease of the nervous system that.

Affects neurons in the brain and spinal cord, causing loss of muscle control.

In the quest to provide better answers for ALS patients. A team of researchers at the University of Southern California in the United States identified two promising avenues.

The development of new treatments for various forms of the devastating disease.

ALS patients: cell reprogramming opens the door to future treatments

The findings were published in a pair of studies: the first in the journal Cell Stem Cell on 2 February. The second in the journal Cell on 7 February.

A minority of patients have various genetic causes of ALS that can be inherite in the family. The majority have what is known as sporadic disease because its causes are unknown. Explains Gabriel Linares, a postdoc in Ichida’s lab and co-author of both studies.

These differences make it “a difficult challenge to find a treatment that works for all ALS patients,” he said.

ALS patients: cell reprogramming opens the door to future treatments

To overcome this challenge, the researchers collecte skin or blood samples from patients. With both familial and sporadic ALS.

The scientists reprogrammed the skin and blood cells into motor neurons.

Which are the nerve cells responsible for movement that degenerate in the disease.

In the Cell Stem Cell study, co-authors Linares and Yichen Li found that several of the most effective drugs and similar molecules increase the activity of androgens.

The well-known group of sex hormones that includes testosterone. However, because drugs that increase androgen activity can have undesirable or dangerous side effects for ALS patients. The scientists set out to identify a genetic change that could produce similar results.

To do so, they used a publicly available bioinformatics database known as Connectivity Map. Develope by the Broad Institute of Harvard and MIT.

By analysing this database of how drugs affect the genetic landscape of disease. The scientists accurately predicted that deleting the SYF2 gene would increase the survival of motor neurons derived from patients with various forms of ALS.