Metformin for ALS
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests whether Metformin is safe and effective for treating a specific type of ALS linked to the C9orf72 gene mutation. Researchers aim to determine if Metformin can reduce certain proteins in the body that may be harmful in this type of ALS. Suitable candidates for the trial have been diagnosed with ALS caused by the C9orf72 gene change and can eat and take pills normally. Participants will take Metformin for about six months to assess its efficacy and safety. As a Phase 2 trial, this research focuses on evaluating the treatment's effectiveness in an initial, smaller group of people.
Will I have to stop taking my current medications?
The trial does not specify if you need to stop taking your current medications, but it does exclude those using potentially liver-damaging drugs and those who have taken experimental drugs recently. It's best to discuss your specific medications with the trial team.
Is there any evidence suggesting that Metformin is likely to be safe for humans with C9orf72 ALS?
Research has shown that metformin, a common diabetes drug, might be safe for people with ALS, a disease affecting the nervous system. Some studies suggest that metformin could lower the risk of developing ALS, indicating it might be well-tolerated. However, in certain animal studies, metformin produced harmful effects, but these results do not always apply to humans.
This trial is in an early stage, so researchers are carefully monitoring for any side effects. While some uncertainty remains about metformin's safety for ALS, its long-standing use for diabetes might offer some reassurance. It's important to remember that reactions can vary, and ensuring safety for ALS patients is the main goal of this trial.12345Why do researchers think this study treatment might be promising for ALS?
Researchers are excited about using Metformin for ALS because it offers a new approach to treating this challenging condition. Unlike traditional ALS treatments, which mainly focus on managing symptoms, Metformin targets cellular energy processes, potentially slowing disease progression. This is particularly promising for patients with C9orf72 positive ALS, as Metformin's mechanism of action may address underlying genetic factors. The possibility of repurposing a well-known diabetes medication like Metformin for ALS also brings hope for a faster transition from trials to clinical use, given its established safety profile.
What evidence suggests that Metformin might be an effective treatment for ALS?
Research suggests that Metformin might help people with ALS, especially those with the C9orf72 mutation. In this trial, participants with C9orf72 positive ALS will receive Metformin. Studies have shown that Metformin reduced harmful protein levels in cells and mice with C9-ALS and improved their symptoms. Another study found that Metformin use is linked to a lower risk of developing ALS. However, some research indicates Metformin might not benefit all ALS patients and could be harmful, particularly for women. While there is hope, the results are mixed, and more research is needed.12678
Who Is on the Research Team?
Laura Ranum, PhD
Principal Investigator
University of Florida
Are You a Good Fit for This Trial?
This trial is for individuals with C9orf72 positive ALS/FTD who can take oral food and medication, have no severe allergies to Metformin or barium sulfate, and no implanted electrical devices or metal in their body. Pregnant women, those trying to conceive, breastfeeding mothers, people with recent cancer (except skin), liver disease, renal impairment (eGFR below 30 mL/min/1.73 m2), or on hepatotoxic drugs are excluded.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive Metformin with dose escalation schedule twice daily for 24 weeks
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Metformin
Metformin is already approved in European Union, United States, Canada, Japan, China, Switzerland for the following indications:
- Type 2 diabetes
- Type 2 diabetes
- Type 2 diabetes
- Type 2 diabetes
- Type 2 diabetes
- Type 2 diabetes
Find a Clinic Near You
Who Is Running the Clinical Trial?
University of Florida
Lead Sponsor
ALS Association
Collaborator