Amyloid Clearing Drugs
In neurodegeneration and adjacent disorders the clearest established example is disease-modifying immune therapy in multiple sclerosis. These treatments include injectables, oral therapies, and infusion therapies that reduce or change inflammatory activity and alter the disease course. In the broader landscape, immune therapies are being investigated for many diseases but as of yet are not approved for those usages.
Biological Rationale
The rationale is straightforward in MS: the immune system is driving demyelination and contributing to later axonal injury. By reducing inflammatory attacks, limiting immune-cell trafficking, or depleting specific immune populations, these drugs can reduce relapses and lower new lesion formation.
In Alzheimer’s disease, immune modulation is more complicated, but it is an important area of research. Many older adults have amyloid and tau buildup in the brain without developing dementia, suggesting that protein aggregation alone may not fully explain who becomes sick. The brain’s immune response may be one reason some people age normally while others develop symptoms. Genes like TREM2, which help microglia respond to amyloid and cellular damage, support this idea. The goal would not be to broadly suppress immunity, but to help the brain’s immune cells respond in a way that contains damage instead of worsening it.
Evidence Strength
Strong in relapsing MS, emerging in other disease contexts
Early and ongoing treatment with approved disease-modifying therapies reduces relapses, limits new inflammatory activity, and can delay disability progression in MS. The evidence is weaker for complete control of chronic progressive neurodegeneration, but the category is still one of the biggest success stories in this space.
Novartis now has a drug targeting TREM2 in microglia for ALS and Alzheimer's, which while promising has a ways to go before being used in patients.
Limitations
These treatments are better at controlling inflammation than reversing established damage. They also come with tradeoffs, including infection risk, monitoring burden, cost, and variable effectiveness across progressive disease forms.
Sources
- National Multiple Sclerosis Society. Disease-Modifying Therapies for MS.
- Hauser, S. L., Bar-Or, A., Comi, G., et al. (2017). Ocrelizumab versus Interferon Beta-1a in Relapsing Multiple Sclerosis.
- Montalban, X., Hauser, S. L., Kappos, L., et al. (2017). Ocrelizumab versus Placebo in Primary Progressive Multiple Sclerosis.
- Rae-Grant, A., Day, G. S., Marrie, R. A., et al. (2018). Practice guideline recommendations summary: Disease-modifying therapies for adults with multiple sclerosis.
- Novartis Pharmaceuticals. (2026). A Clinical Trial to Learn About the Effects of VHB937 in People With Early Alzheimer's Disease