Intravenous Immunoglobulin for ALS

(ALSFUS003 Trial)

This trial tests a new method to treat ALS, a condition affecting nerve cells in the brain and spinal cord, leading to loss of muscle control. Researchers aim to determine if intravenous immunoglobulin (IVIg, a therapy using antibodies) combined with a focused ultrasound technique can safely and effectively reach the brain area responsible for movement. The study seeks individuals diagnosed with ALS who can lie flat comfortably and are not currently experiencing breathing issues without support devices. Participants should be stable on any ALS medications before joining the trial. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this innovative therapy.

You may need to stop taking certain medications before joining the trial. Specifically, you should not take medications that increase the risk of bleeding, like aspirin, for at least 7 days before treatment, and you should not take Avastin for 30 days prior. Also, if you are using steroids, immunosuppressants, or NSAIDs, you should not have used them in the past 30 days.

Research has shown that intravenous immunoglobulin (IVIG) is generally safe for people with ALS, a disease affecting nerve cells in the brain and spinal cord. In previous studies, patients received doses similar to those planned for this trial, and their condition did not significantly worsen due to the treatment.

Some patients might experience mild side effects like headaches, fever, or nausea. Importantly, research has not commonly reported any serious side effects related to the treatment.

Intravenous Immunoglobulin (IVIG) for ALS is unique because it combines IVIG with focused ultrasound to enhance drug delivery to the brain. Most treatments for ALS, such as Riluzole and Edaravone, work by slowing disease progression, but they don't specifically target drug delivery to the brain. This new approach uses focused ultrasound to temporarily open the blood-brain barrier, potentially allowing more of the IVIG to reach critical areas like the primary motor cortex. Researchers are excited because this could lead to more effective treatment outcomes by directly enhancing drug delivery to the areas most affected by ALS.

Research has shown that Intravenous Immunoglobulin (IVIG) might help with ALS by reducing inflammation and slowing the disease's progression. In this trial, participants will receive IVIG combined with focused ultrasound (FUS) to temporarily open the blood-brain barrier, enhancing IVIG delivery to the brain. Studies suggest that using these two methods together could improve treatment delivery and possibly lead to better outcomes for ALS patients. Although limited data exist from human studies, this treatment is promising because it can directly target the brain, which is important for managing ALS symptoms.¹³⁶⁷⁸