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SUPPLEMENT: A Novel Immunotherapy for the Treatment of Parkinson’s Disease

Study Rationale: There is currently no treatment that slows or stops the progression of Parkinson’s disease (PD). We are developing a novel therapeutic approach that targets neuroinflammation and protects the brain’s blood vessels, which are known to be compromised in PD. Uniquely, our therapy does not need to enter the brain to be effective. It works by blocking a harmful interaction between two proteins—tissue plasminogen activator (tPA) and NMDA receptors (NMDARs)—which contributes to inflammation, breakdown of the blood-brain barrier (BBB), and the loss of dopamine-producing neurons. In multiple models of PD, our treatment—using a mouse antibody called glunomab or its humanized version, LYS241—significantly reduced brain inflammation, protected neurons, and improved movement, even when administered at later stages of the disease. The therapy also lowered levels of neurofilament light (NF-L), a blood-based marker of nerve cell damage, reduced immune cell activation in the brain, and decreased toxic aggregation of alpha-synuclein protein.

Hypothesis: Blocking the interaction between tPA and NMDARs with LYS241 can protect the brain, reduce inflammation, and slow or stop progression of Parkinson’s disease.

Study Design: We will conduct studies to:

  1. Determine the most effective dose range of LYS241 for clinical translation.
  2. Identify human fluid biomarkers to support drug development.
  3. Manufacture a human-grade batch of LYS241 to confirm quality and consistency and support IND-enabling studies.
  4. Complete safety and toxicity studies in two animal species, as required before human testing.

Impact on Diagnosis/Treatment of Parkinson’s disease: This therapy represents a new class of treatment that goes beyond symptom management by targeting disease mechanisms directly. If successful, LYS241 could slow or prevent progression of PD. In addition, identifying blood-based biomarkers could improve how we monitor disease activity and treatment response in patients.

Next Steps for Development: Successful outcomes from pharmacokinetic, safety, and toxicity studies for LYS241, along with evidence of robust manufacturing quality control, will strengthen the drug’s investigational new drug (IND) application and support progression to Phase 1 and subsequent clinical trials in PD patients.


Researchers

  • Manuel Blanc, PhD, MBA

    Lyon & Caen France


  • Daniel A. Lawrence, PhD

    Ann Arbor, MI United States


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