Kirchhoff Family Fellow Proposes New Therapeutic Strategy in ALS

Joesph Klim, PhD, a Kirchhoff Family Fellow at the Lab of Kevin Eggan, PhD at Harvard University has discovered that restoring expression of the gene Stathmin2, or STMN2, rescues motor neuron degeneration, a hallmark of ALS. Findings were published on January 14th, 2019 in Nature Neuroscience.

Dr. Klim has spent the last three years studying TDP-43, a protein previously known the be involved in ALS. His recent discovery shows that TDP-43 sustains levels of STMN2, a mediator of motor neuron growth and repair. So when TDP-43 is perturbed, STMN2 is diminished and motor neurons suffer as a consequence, causing the body to lose the ability to move. Klim used patient stem cell models of ALS, as well as spinal cord samples donated by dozens of ALS patients, to show that boosting STMN2 could slow motor neuron degeneration in patients, regardless of their cause of ALS.

Said Klim, “Our work highlights STMN2 as a credible drug target for ALS and a potential new biomarker.”

Named in honor of former Project ALS board member and inspiration Tom Kirchhoff who passed away from ALS in 2015, the Tom Kirchhoff Family Post-Doctoral Fellowship at Project ALS is given to promising young scientists already making a difference in ALS research. “After meeting the Kirchhoff family”, said Klim, “I was inspired by their strength and courage to pursue ALS treatments as Tom battled this devastating disease and by their resilience and determination to continue this fight after his passing. As the Tom Kirchhoff Family Postdoctoral Fellow, I had the freedom to pursue this complicated and long-term project, and I was bolstered in my efforts by the strong Kirchhoff family spirit.”

Klim’s breakthrough demonstrates the power and interconnectedness of those who are affected by the disease, and those researching it – an important tenet of the Project ALS mission. Said Dr. Kevin Eggan, “These experiments…point towards a clear path for testing whether repairing STMN2 in our friends and family can slow or stop their disease.”

Read more about the study on the Harvard Department of Stem Cell and Regenerative Biology’s website.

(Photo: Dr. Kevin Eggan discusses Klim’s findings at the Project ALS gala onstage with the Kirchhoff family)