Basiliximab

證據等級: L5

預測適應症: 10 個

Basiliximab: From Transplant Rejection Prevention to Plasma Cell Myeloma

One-Sentence Summary

Basiliximab is a chimeric anti-CD25 (IL-2Rα) monoclonal antibody, originally developed for prevention of acute rejection following solid organ transplantation by selectively blocking T-cell activation. The TxGNN model predicts it may be effective for Plasma Cell Myeloma (multiple myeloma), with 3 clinical trials and 3 publications currently supporting this direction. The core hypothesis is that CD25 blockade can selectively deplete regulatory T cells (Tregs) during autologous stem cell transplantation, thereby restoring anti-myeloma immune surveillance.

Quick Overview

Item	Content
Original Indication	Transplant rejection prevention (solid organ / HSCT)
Predicted New Indication	Plasma Cell Myeloma (Multiple Myeloma)
TxGNN Prediction Score	95.61%
Evidence Level	L2
EU Market Status	Not Marketed (0 authorizations on record)
Number of Authorizations	0
Recommended Decision	Proceed with Guardrails

Why is This Prediction Reasonable?

Currently, detailed mechanism of action data is not available from structured data sources. Based on known information, Basiliximab is a chimeric monoclonal antibody that targets CD25 — the alpha subunit of the interleukin-2 receptor (IL-2Rα) — expressed on activated T cells. By competitively blocking CD25, it prevents IL-2–driven clonal T-cell expansion, thereby suppressing acute transplant rejection. This mechanism is well-established across solid organ transplantation contexts.

In the multiple myeloma (plasma cell myeloma) setting, the repurposing rationale is mechanistically compelling. CD25 is also constitutively expressed on regulatory T cells (Tregs), which inhibit cytotoxic immune responses against myeloma cells. During autologous stem cell transplantation (ASCT) — the standard of care for eligible MM patients — Tregs reconstitute rapidly and are thought to blunt the graft-versus-myeloma (GvM) effect. Selectively depleting these Tregs with Basiliximab at the time of ASCT could therefore restore anti-tumor immune surveillance and improve long-term disease control.

The mechanistic bridge is well-supported: a published Phase 1 trial (PMID 31940591) directly tested this hypothesis in MM patients undergoing ASCT, demonstrating that Treg depletion via CD25 blockade is both feasible and biologically rational. Additionally, in allogeneic HSCT settings, GvHD prevention with Basiliximab directly enables successful engraftment — a prerequisite for any graft-versus-myeloma benefit — extending the drug’s relevance across the full HSCT treatment continuum for myeloma.

Clinical Trial Evidence

Trial Number	Phase	Status	Enrollment	Key Findings
NCT01526096	Phase 1	Completed	30	Treg depletion via Basiliximab in MM patients undergoing ASCT — evaluated safety and feasibility of regulatory T-cell reduction to enhance anti-myeloma immune response post-transplant
NCT00975975	Phase 2	Completed	17	Basiliximab + cyclosporine for GvHD prevention after nonmyeloablative allogeneic transplantation for hematologic malignancies — established IL-2R blockade safety profile in the HSCT context
NCT00594308	N/A	Terminated	10	Basiliximab vs. cyclosporine alone for GvHD prevention after transplant — terminated early due to poor enrollment; limited interpretability

Literature Evidence

PMID	Year	Type	Journal	Key Findings
31940591	2020	Phase 1 Trial Report	Journal for Immunotherapy of Cancer	Direct evidence in MM: Tregs reconstitute rapidly post-ASCT and suppress anti-myeloma immune responses; Basiliximab-based Treg depletion strategy demonstrated in MM patients undergoing ASCT
12476283	2002	Prospective Observational Study	Bone Marrow Transplantation	Basiliximab well tolerated and effective for steroid-refractory acute GvHD after allogeneic SCT in 17 patients (including MM cases) — supports long-term IL-2R blockade safety in HSCT
28320553	2017	Case Report	American Journal of Kidney Diseases	Kidney transplantation in 4 MM patients with renal failure using Basiliximab induction — demonstrates transplant management feasibility in MM survivors with improved long-term outcomes

EU Market Information

No EMA marketing authorizations are on record for Basiliximab in this dataset (market status: not marketed, 0 authorizations).

Note: Basiliximab (Simulect®) is known to hold regulatory approvals in multiple jurisdictions worldwide. This absence is likely a data gap in the current dataset rather than a true lack of authorization. Please verify current authorization status directly via the EMA product database.

Safety Considerations

Please refer to the SmPC for safety information.

Conclusion and Next Steps

Decision: Proceed with Guardrails

Rationale: A completed Phase 1 trial (NCT01526096, n=30) directly tested Basiliximab-mediated Treg depletion in MM patients undergoing ASCT, with published results (PMID 31940591) confirming biological plausibility and initial safety; a completed Phase 2 trial further established the drug’s tolerability in HSCT-related settings. The mechanistic hypothesis is coherent, the evidence reaches L2, and the clinical context (ASCT in MM) is directly aligned.

To proceed, the following is needed:

Retrieve full mechanism of action data from DrugBank to complete the mechanistic link analysis (currently a High-severity data gap)
Obtain SmPC safety warnings and contraindications from the formal prescribing label — currently a Blocking data gap that prevents formal safety screening
Review the full efficacy outcome data from NCT01526096 (PMID 31940591 is available; confirm Treg depletion depth, relapse rates, and immune reconstitution data)
Design a Phase 2 proof-of-concept trial specifically measuring anti-myeloma efficacy endpoints (PFS, MRD negativity) following Treg depletion — current evidence focuses on safety and immune feasibility, not tumor response
Define patient selection criteria: ASCT-eligible MM patients, baseline Treg quantification, and post-depletion immune monitoring plan
Disclaimer

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