Terazosin for ALS

(TZ-ALS Trial)

This trial tests terazosin, a medication that might benefit people with amyotrophic lateral sclerosis (ALS), a condition affecting nerve cells in the brain and spinal cord. The study primarily assesses whether terazosin is safe and tolerable for people with ALS and if it can boost cellular energy levels. Participants will undergo various procedures, such as blood tests and scans, to aid researchers in gathering information. Suitable candidates have received an ALS diagnosis within the last three years and can swallow pills. As an Early Phase 1 trial, participants will be among the first to help researchers understand terazosin's effects in people with ALS.

The trial requires that you refrain from starting new medications like Riluzole or Edaravone during the study. You cannot be using terazosin or similar medications like doxazosin, alfuzosin, prazosin, or tamsulosin at the time of screening or within 3 months prior. Also, you cannot be on therapeutic anticoagulants or have noncompliant use of medications affecting the central nervous system or blood pressure.

Research has shown that terazosin is usually well-tolerated. One study found that terazosin helps protect motor neurons, which are nerve cells that control movement, by improving the body's energy use. This might address some energy problems seen in ALS.

Other studies have used terazosin at doses up to 10 mg daily for several months without major issues. Although terazosin is often used for high blood pressure and other conditions, its effects on ALS remain under investigation.

Terazosin is unique because, unlike most treatments for ALS that focus on managing symptoms, it targets cellular energy production. Researchers are excited about terazosin because it may enhance the body's ability to produce ATP, the energy currency of cells, which could slow the progression of ALS. While current standard care options like riluzole and edaravone provide limited benefits in extending survival or slowing disease progression, terazosin's approach of boosting cellular energy presents a novel avenue that could lead to more effective interventions for ALS.

Research has shown that terazosin might help treat ALS (amyotrophic lateral sclerosis). Studies have found that terazosin protects nerve cells in ALS models by increasing ATP, the energy cells need to function. Terazosin also enhances glycolysis, a process that helps cells produce energy, and can protect motor neurons from damage. These findings suggest that terazosin, the treatment under study in this trial, may help manage energy shortages and protect nerve cells in ALS.¹³⁶⁷⁸