Scientist whose mother and sisters died of ALS complications hopes experimental treatment will save his life

Jeff Vierstra’s life has been touched by tragedy, with his mother and two sisters all succumbing to complications from ALS, a devastating neurodegenerative disease. But now, this scientist and avid skier is hoping that an experimental treatment can help him avoid the same fate.

Vierstra was just a toddler when his mother passed away from ALS. Tragically, his sisters Erin and Leigh also tested positive for a mutation of the FUS gene, putting them at high risk for developing the disease. This genetic predisposition has loomed over Vierstra, shaping his outlook on life.

Despite the grim prognosis, Vierstra has not let fear hold him back. He has embraced adventure, from skiing down icy slopes in Chile to skydiving in British Columbia. But deep down, he knows that his family history of ALS is a shadow that he cannot escape.

ALS, also known as Lou Gehrig’s disease, is a progressive condition that causes the degeneration of motor neurons, leading to loss of muscle control and eventually the ability to breathe. While there is currently no cure for ALS, researchers are exploring new treatments to combat this devastating disease.

At the Eleanor and Lou Gehrig ALS Center at Columbia University, Vierstra and his sisters participated in a clinical trial for an experimental treatment targeting the mutated FUS gene. Dr. Neil Shneider, a leading neurologist at Columbia University, identified abnormalities in Vierstra’s muscle testing that indicated early signs of ALS. Without hesitation, Vierstra opted to undergo the same treatment that had extended his sisters’ lives.

For the past three years, Vierstra has been receiving spinal infusions aimed at disabling the mutated gene. Despite the loss of his sisters to ALS, Vierstra credits the treatment with prolonging their lives. His own health has improved, with his muscle testing showing positive results after just one year.

Vierstra’s journey is a beacon of hope in the fight against ALS. Dr. Shneider believes that the research being conducted at Columbia University could revolutionize the treatment of ALS, turning it into a manageable disease rather than a death sentence. As Vierstra continues to live his life to the fullest, he is grateful for the opportunity to plan for a future that once seemed uncertain.

The ALS Center at Columbia University is at the forefront of research on familial forms of ALS, with plans to expand their studies to include other rare genetic mutations. Their initiative, Silence ALS, aims to develop personalized gene-based therapies for patients with various forms of ALS. For more information on participating in Silence ALS, individuals can contact silenceals@cumc.columbia.edu.

In the face of adversity, Jeff Vierstra is a shining example of resilience and determination. His story serves as a reminder that with perseverance and cutting-edge research, we can overcome even the most daunting challenges.