ID: h-SDA-2026-04-26-gap-20260426-001521-7
Hypothesis

Plasma NfL Elevation Secondary to BBB-Associated Transport Dysfunction Enables Longitudinal Neurodegeneration Tracking

Neurofilament light chain (NfL) is released from damaged neurofilaments into the extracellular space, flowing into CSF and ultimately into peripheral blood via degraded BBB transport mechanisms.
🧬 NEFL🎯 Composite 49%💱 $0.61▲2.1%proposed
neurodegeneration
EvidencePending (0%)📖 0 cit🗣 1 debates 3 support 3 oppose
✓ All Quality Gates Passed
Mechanistic 0.78 (15%) Evidence 0.30 (15%) Novelty 0.00 (12%) Feasibility 0.00 (12%) Impact 0.00 (12%) Druggability 0.00 (10%) Safety 0.00 (8%) Competition 0.00 (6%) Data Avail. 0.00 (5%) Reproducible 0.00 (5%) KG Connect 0.50 (8%) 0.485 composite

🧪 Overview

Neurofilament light chain (NfL) is released from damaged neurofilaments into the extracellular space, flowing into CSF and ultimately into peripheral blood via degraded BBB transport mechanisms. Early BBB disruption increases permeability of neurofilament-derived peptides into circulation, causing disproportionate plasma NfL elevation relative to CSF levels. This makes plasma NfL a sensitive indicator of BBB permeability-augmented neurodegeneration, enabling peripheral blood-based disease progression monitoring. Multiple FDA-cleared platforms (Simoa, Elecsys, Lumipulse) provide validated detection.

🧬 Mechanism

🧬 Curated Mechanism Pathway

Curated pathway from expert analysis

flowchart TD
    A["Neurofilament Light<br/>Chain Release"]
    B["Damaged<br/>Neurofilaments"]
    C["Extracellular<br/>CSF Flow"]
    D["BBB Transport<br/>Dysfunction"]
    E["Peripheral Blood<br/>NfL Elevation"]
    F["Longitudinal Neurodegeneration<br/>Tracking"]
    B --> A
    A --> C
    C --> D
    D --> E
    E --> F
    style A fill:#b71c1c,stroke:#ef9a9a,color:#ef9a9a
    style E fill:#e65100,stroke:#ffab91,color:#ffab91
    style F fill:#1b5e20,stroke:#a5d6a7,color:#a5d6a7

⚖️ Evidence

⚖️ Evidence Matrix3 supports3 contradicts
Supports
LRP1 mediates Aβ efflux across the BBB, with expression declining in AD
Supports
sLRP1 levels in plasma inversely correlate with brain Aβ burden and cognitive function
Supports
ADAM10/17 responsible for LRP1 ectodomain shedding in response to inflammatory stimuli
Contradicts
Liver LRP1 significantly contributes to plasma sLRP1
Contradicts
LRP1 expression on peripheral monocytes correlates with AD risk
Contradicts
Majority of circulating sLRP1 originates from peripheral tissues
📖 Linked Papers

No linked papers recorded for this hypothesis yet.

🏥 Translation

🧬 3D Protein Structure — NEFL

No curated PDB or AlphaFold mapping for NEFL yet. Search RCSB →

🧠 GTEx v10 Brain ExpressionJSON

Median TPM across 13 brain regions for NEFL from GTEx v10.

Frontal Cortex BA9478 Cortex336 Anterior cingulate cortex BA24216 Hypothalamus144 Nucleus accumbens basal ganglia89.4 Substantia nigra83.0 Hippocampus76.4 Amygdala68.1 Caudate basal ganglia53.5 Cerebellum50.5 Putamen basal ganglia40.6 Cerebellar Hemisphere39.3 Spinal cord cervical c-121.1median TPM (GTEx v10)

💉 Clinical Trials

No clinical trials data linked to this hypothesis yet.

No curated ClinVar variants loaded for this hypothesis.

Run scripts/backfill_clinvar_variants.py to fetch P/LP/VUS variants.

🔍 Search ClinVar for NEFL →

No DepMap CRISPR Chronos data found for NEFL.

Run python3 scripts/backfill_hypothesis_depmap.py to populate.

🏆 Tournament

🏆 Arenas / Elo

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📊 Market Indicators

7d Trend
Stable
7d Momentum
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Volatility
High
0.1247
Events (7d)
1
Price History
▲2.1%

💾 Resource Usage

No resource usage or linked notebooks recorded for this hypothesis yet.

🔮 Predictions

🔎 Predictions vs Observations2 predictions · 0 with recorded observations
PredictionPredictedObservedStatusConf
IF plasma NfL is measured every 6 months over 24 months in patients with evidence of active neurodegeneration vs neurologically stable controls, THEN plasma NfL percent change will correlate with longSpearman correlation ρ≥0.50 between plasma NfL percent change and percent brain volume loss (FreeSurfer) over 24 months in active neurodegeneration group; ≥80% — no observation —pending0.71
IF participants are stratified by quantitative BBB permeability measured via dynamic contrast-enhanced MRI (DCE-MRI) to identify high-permeability (BBB-disrupted) vs low-permeability (BBB-intact) grouPlasma NfL/CSF NfL ratio ≥2-fold greater in high-BBB-permeability group (mean plasma NfL/CSF NfL ratio: ≥0.8) compared to low-permeability group (mean ratio: ≤0— no observation —pending0.68
🔮 Falsifiable Predictions (2)
pendingconf 71%
IF plasma NfL is measured every 6 months over 24 months in patients with evidence of active neurodegeneration vs neurologically stable controls, THEN plasma NfL percent change will correlate with longitudinal brain volume loss (ρ≥0.50, p<0.01) in the active neurodegeneration group, with ≥80% of fast
Predicted outcome: Spearman correlation ρ≥0.50 between plasma NfL percent change and percent brain volume loss (FreeSurfer) over 24 months in active neurodegeneration gr
Falsification: No significant correlation between plasma NfL change and brain volume loss (ρ<0.30, p>0.05) in active neurodegeneration group, OR plasma NfL remains stable (change <15%) despite substantial atrophy (>
pendingconf 68%
IF participants are stratified by quantitative BBB permeability measured via dynamic contrast-enhanced MRI (DCE-MRI) to identify high-permeability (BBB-disrupted) vs low-permeability (BBB-intact) groups, THEN the plasma NfL/CSF NfL ratio will be ≥2-fold higher in the high-permeability group than the
Predicted outcome: Plasma NfL/CSF NfL ratio ≥2-fold greater in high-BBB-permeability group (mean plasma NfL/CSF NfL ratio: ≥0.8) compared to low-permeability group (mean
Falsification: No significant difference in plasma NfL/CSF NfL ratio between BBB permeability strata (p>0.05, 95% CI includes 1.0-fold), OR CSF NfL exceeds plasma NfL proportionally in high-permeability group, indic
Metadatasource: v1_phase_c_backfill · origin_type: gap_debate
sourcev1_phase_c_backfill
origin_typegap_debate
_schema_version1
📊 Evidence Profile
Evidence Balance
+0%
Certainty
0%
Debates
0
Incoming
0
Outgoing
0
0 supporting 0 contradicting 0 neutral
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