Pridopidine for ALS: Expanded Access Protocol for S1R-targeted Therapy

Who could benefit from this research

What this research studies

If you have ALS, this program explores whether pridopidine can make a difference by acting on the Sigma-1 receptor in the brain and spinal cord. Researchers will monitor safety and clinical effects in participants through a structured protocol, including an efficacy-focused portion and an open-label follow-up. The study builds on previous findings that S1R activation may influence cellular processes linked to ALS, such as protein handling and cellular stress responses. It leverages an expanded-access framework to provide potential access to a promising therapy while gathering real-world data on outcomes and tolerability.

Why it matters

Where this research is happening

• Massachusetts General Hospital
  Boston, United States Active

Researchers

• Principal investigator: Paganoni, Sabrina — Massachusetts General Hospital
• Study coordinator: Paganoni, Sabrina

Related research

Other NIH-funded projects on the same topics.

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• Understanding how work exposures and genes influence ALS risk

  A study is building a long-term group of people at higher risk for ALS to see how their jobs, exposures at work, and genes together affect if and when the disease might develop.

  NIH research University of Michigan at Ann Arbor · Ann Arbor, United States · NIH-11631673

• NUZ-001: a treatment option for ALS to protect nerve cells

  A small molecule called NUZ-001 is being tested to help people with ALS by improving cell systems that keep proteins balanced and prevent harmful protein clumps.

  NIH research Massachusetts General Hospital · Boston, United States · NIH-11564156

• Alpha5 integrin's role in ALS: how a cell switch worsens disease

  Aims to understand how a specific protein called alpha5 integrin in immune cells affects ALS progression and whether blocking it can help extend life.

  NIH research Cedars-Sinai Medical Center · Los Angeles, United States · NIH-11540166

• C9orf72 ALS/FTD Prevent: prepping for future treatment

  This grant aims to get ready for a preventive treatment for people who carry the C9orf72 genetic change that can lead to ALS or frontotemporal dementia.

  NIH research University of Miami School of Medicine · Coral Gables, United States · NIH-11431628

• Air pollutants and ALS: mapping environmental links to motor neuron disease

  This NIH grant explores whether different air pollutants are linked to the development of ALS and uses a large U.S. data set to look for environmental risk factors.

  NIH research Washington University · Saint Louis, United States · NIH-11423391

About this research

1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
3. For full project details, budget, and progress reports, visit the official NIH RePORTER page.