A new antibody-based therapy for SPG4 hereditary spastic paraplegia
Official title: Testing a new therapy for SPG4 Hereditary Spastic Paraplegia
Funded at Drexel University · NIH-11388925 · Data updated 2026-11-12
A small NIH grant is funding a therapy aimed at reducing mutant M1 spastin protein to help people with SPG4-HSP, a gradual leg-weakening neurodegenerative condition.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Drexel University NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project start | June 15, 2026 |
| Project end | March 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $228,688 |
| NIH project number | 1R21NS149629-01 |
| Project ID | NIH-11388925 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are individuals with SPG4-HSP caused by SPAST mutations who can participate in biomarker and potentially neuronal studies.
✕Less likely to help
Patients without SPG4-HSP or those unable to participate in neuronal-targeted research may not benefit.
What this research studies
This project focuses on a genetic form of spastic paraplegia caused by SPAST mutations. Researchers have created monoclonal antibodies that are designed to work inside neurons to lower the harmful mutant M1 protein and protect axons. The approach uses intrabodies engineered to direct the mutant protein toward cellular degradation pathways, with the goal of slowing or reversing disease progression. The study will test whether this strategy can deplete the toxic protein effectively without harming normal protein function. If successful, this could lay the groundwork for a new treatment for SPG4-HSP.
Why it matters
Potential benefit: If successful, patients with SPG4-HSP could experience slowed disease progression and preserved mobility.
How similar studies have gone: Similar intrabody approaches have shown promise in other diseases, but this specific mutant M1 SPG4-HSP strategy is novel.
Where this research is happening
-
Drexel University
Philadelphia, United States Active
Researchers
- Principal investigator: Baas, Peter W. — Drexel University
- Study coordinator: Baas, Peter W.
Related research
Other NIH-funded projects on the same topics.
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Understanding axon degeneration in hereditary spastic paraplegia and ALS using patient-derived neurons
Aimed at understanding why certain nerve fibers die in hereditary spastic paraplegia and ALS by studying neurons made from patients’ own stem cells.
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Combinatorial Therapy for SPG4 hereditary spastic paraplegia
Aims to test gene-based treatments to help people with SPG4-related hereditary spastic paraplegia manage mobility and related symptoms.
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Understanding how full-length spastin works and its role in hereditary spastic paraplegia
This grant aims to uncover how the spastin protein uses ATP to cut and regrow microtubules, shedding light on the most common inherited cause of a neurodegenerative leg-weakness disorder.
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Finding hidden genetic causes of hereditary spastic paraplegia
Using whole genome sequencing to find genetic causes for people with hereditary spastic paraplegia whose earlier genetic tests came up inconclusive.
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Understanding nerve damage in hereditary spastic paraplegia
Researchers are using genetically modified rats and patient-derived stem cells to learn how gene mutations cause nerve fiber damage in people with hereditary spastic paraplegia.
-
Understanding and treating a genetic condition that affects movement in children
This study is looking at how certain genes affect movement problems in children with hereditary spastic paraplegia and is testing new treatments that might help improve their nerve function and overall health.
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.