By Jacob Ahlstrom | Posted - Oct 21st, 2019





Mitochondrial Treatment for ALS

Treatment of ALS drug SBT-272 on mouse models shows promising results. Stealth bioTherapeutics recently announced that they are making plans to move on to a Phase 1 safety trial by the end of this year. According to their research the drug that they are testing, SBT-272, improves health in the mitochondria and effectively decreases nerve damage.


What is the Mitochondria?

About 80% of your body’s energy comes from the part of the cell called the mitochondria. The mitochondria produces energy molecules and ships them out to be used elsewhere in the cell. Nerve cells that do not have functioning mitochondria will die. A specific protein assigned to protecting the mitochondria from toxins within the cell is missing in roughly 20% of hereditary ALS cases. This protein is critical in keeping the mitochondria alive. The cells that lack this protein are susceptible to decay. Unfortunately this is problematic because damaged neural cells (cells in charge of brain and motor function) do not regenerate or heal themselves like other cells in the body. Stealth BioTherapeutics is researching a drug that has proven to increase mitochondrial function and reverse the effects of cell decay in dying nerve cells. 

The purpose of SBT-272 

The drug, SBT-272, is designed specifically for ALS and other neurodegenerative diseases. Stealth BioTherapeutics presented their research for SBT-272 at the 18th Annual Northeast Amyotrophic Lateral Sclerosis Consortium. 

In the study, 60 ALS model mice were randomly divided into 3 groups and given one of three types of daily injections for up to 10 weeks. The first group received a placebo injection, to use as a control. The other two groups were given either a large dose of SBT-272(5.0 mg/kg) or a small dose (0.5 mg/kg). 

Study Results

The male mice treated with the higher dose of SBT-272 showed an increase in muscular function and an extended lifespan. The Biomarkers in these mice showed a reduction of nerve damage in the mice with the large dose compared to the placebo. A biomarker is a measurable substance in an organism whose presence is evidence of some disease or dysfunction in the body. 

No significant differences were seen in the female mice. When looking at the biomarker levels between those treated with a higher dose of SBT-272 and those given a placebo there was relatively no change. One reason for this may be that in general females tend to produce less of the biomarker that was examined in this experiment.

Stealth Research promises to work “diligently to progress SBT-272 into the clinic later this year to evaluate its safety ahead of anticipated efficacy studies”. We hope that such research will give us further insight into the benefits treating mitochondria for ALS and open doors to new drugs in the future.

Jacob Ahlstrom
About the Author

Jacob Ahlstrom - Jacob is a Neuroscience undergraduate at Brigham Young University. Jacob's interest in researching and writing about ALS is fueled by his hope to make the process easier for everyone else. Over the last year he has worked alongside Seth Christensen to find ways to educate and connect ALS patients.