Interferon Beta-1B

證據等級: L5

預測適應症: 2 個

Interferon Beta-1b: From Multiple Sclerosis (Established Use) to Hairy Cell Leukemia

One-Sentence Summary

Interferon beta-1b is a Type I interferon whose established clinical use — per the mechanistic evidence in this pack — is in multiple sclerosis; formal regulatory records for original indication and market authorization are not available in this dataset. The TxGNN model predicts a repurposing signal for Hairy Cell Leukemia, currently supported by 0 registered clinical trials and 4 older publications (1987–1990), with no Norway market presence on file.

Quick Overview

Item	Content
Original Indication	Not available in regulatory dataset (0 licenses on file; see Data Gap DG001)
Predicted New Indication	Hairy Cell Leukemia
TxGNN Prediction Score	99.16%
Evidence Level	L3
Norway Market Status	Not Marketed
Number of Authorizations	0
Recommended Decision	Hold

Why is This Prediction Reasonable?

Detailed mechanism-of-action data for this drug is currently not available (Data Gap DG002). Based on the mechanistic rationale supplied with this evidence pack, interferon beta-1b is a Type I interferon (IFN-β) with antiproliferative and immune-modulatory activity, signaling through the IFNAR receptor pathway. It belongs to the same Type I interferon family as interferon alpha, which is an already-established treatment for hairy cell leukemia.

The rationale for this prediction rests on drug-class analogy rather than a direct indication overlap: because IFN-α and IFN-β share the same receptor and downstream antiproliferative signaling in lymphoid/myeloid precursor cells, IFN-β-1b's activity against hairy cell leukemia has biological plausibility. This is corroborated by older clinical literature (1987–1990) directly testing IFN-β-ser in hairy cell leukemia patients, some of whom had failed or preceded standard alpha-interferon therapy.

However, this body of evidence predates modern standard-of-care agents for hairy cell leukemia (e.g., purine analogues such as cladribine/pentostatin), and no clinical trials have been registered on ClinicalTrials.gov to re-confirm this signal in a contemporary setting. The prediction should therefore be read as a plausible, mechanistically grounded hypothesis rather than a validated repurposing pathway.

Clinical Trial Evidence

Currently no related clinical trials registered.

Literature Evidence

PMID	Year	Type	Journal	Key Findings
2736487	1989	Prospective comparative study	Cancer	10 HCL patients treated with recombinant IFN-β-ser (90×10⁶ U SC, 3x/week); 63% normalized peripheral blood counts, 25% partial hematologic improvement, compared prospectively with IFN-α outcomes.
2198792	1990	Case series (post-IFN failure)	American Journal of Clinical Oncology	3 HCL patients who failed IFN-α or IFN-β achieved complete response with pentostatin (DCF), demonstrating a salvage option after IFN-β failure.
2082943	1990	Case series/small trial	American Journal of Hematology	12 heavily pretreated HCL patients (90–100% marrow hairy cells) treated with IV beta-ser IFN 90 MU 3x/week; describes dosing and tolerability.
3312839	1987	Retrospective cohort/experience	Leukemia	UCLA experience across 51 HCL patients on type I IFN trials; hematologic improvement in ~71% of patients starting recombinant beta-ser-IFN, comparable to alpha-IFN response rates.

Norway Market Information

This product currently has no authorization records on file in Norway (0 licenses; market status: Not Marketed).

Safety Considerations

Please refer to the package insert for safety information.

Additional Signal (Supplementary): Autoimmune Disease of the Central Nervous System

Separately from the primary repurposing candidate above, this evidence pack also contains a second, much stronger-evidence prediction for interferon beta-1b: autoimmune disease of central nervous system (TxGNN score 99.02%), supported by 24 clinical trials (including multiple completed Phase 3/Phase 4 studies such as BENEFIT and BENEFIT 11, n=278–2,878) and 18 publications, including a Cochrane network meta-analysis. Evidence level is L1, decision stage S3, with a pack-recommended decision of Proceed with Guardrails.

This signal is consistent with interferon beta-1b's mechanistic rationale of modulating Th1/Th17–Treg balance and inhibiting T-cell migration across the blood–brain barrier — the established mode of action in multiple sclerosis — and most likely reflects confirmation of an already well-established therapeutic use rather than a novel repurposing opportunity. It is noted here for completeness but is not the subject of this report's primary Go/Hold decision, since it does not represent a new indication.

Conclusion and Next Steps

Decision: Hold

Rationale: The hairy cell leukemia signal rests entirely on small, decades-old (1987–1990) case series and cohort literature with no registered clinical trials and no evidence generated against modern standard-of-care comparators. Combined with the L3 evidence level and "Research Question" stage flagged in the source data, the evidence is insufficient to justify proceeding at this time.

To proceed, the following is needed:

TFDA/Norway package insert data — warnings, contraindications, DDI (Data Gap DG001, currently blocking safety screening)
Confirmed mechanism-of-action documentation (Data Gap DG002)
Contemporary clinical trial data for interferon beta-1b in hairy cell leukemia, ideally benchmarked against purine analogue therapy
Route-of-administration compatibility assessment (currently "pending" in the evidence pack)
Confirmation of original approved indication(s) and market history, which are currently absent from the regulatory dataset
Disclaimer

This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.