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Related Experiment Video

Updated: May 28, 2026

Establishment of a Human Multiple Myeloma Xenograft Model in the Chicken to Study Tumor Growth, Invasion and Angiogenesis
10:04

Establishment of a Human Multiple Myeloma Xenograft Model in the Chicken to Study Tumor Growth, Invasion and Angiogenesis

Published on: May 1, 2015

Melflufen in Multiple Myeloma: Clinical Limitations, Biological Rationale, and Future Perspectives.

Matteo Garibotto1, Debora Soncini2, Roberto Massimo Lemoli1,2

  • 1Clinic of Hematology, IRCCS Azienda Ospedaliera Metropolitana, Ospedale Policlinico San Martino, 16132 Genoa, Italy.

Cancers
|May 27, 2026
PubMed
Summary

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Leukemia & lymphoma·2026

Melflufen, a novel peptide-drug conjugate for multiple myeloma (MM), showed initial promise but failed to improve survival in a key trial. Its development was halted, highlighting challenges in treating refractory MM.

Area of Science:

  • Oncology
  • Pharmacology
  • Drug Development

Background:

  • Multiple myeloma (MM) is an incurable blood cancer with frequent relapses and treatment resistance.
  • Patients with relapsed or refractory MM have limited therapeutic options and poor prognoses.
  • Melflufen is a peptide-drug conjugate designed for targeted delivery of alkylating agents in MM.

Purpose of the Study:

  • To review the biological basis, clinical trials, and regulatory history of melflufen in MM.
  • To critically evaluate melflufen's place in the evolving treatment landscape of MM.
  • To discuss lessons learned from melflufen's development for future strategies.

Main Methods:

  • Review of preclinical data and early-phase clinical studies of melflufen.
  • Analysis of the Phase III OCEAN trial results, focusing on overall survival.
Keywords:
aminopeptidasesmelflufenmultiple myelomapeptide–drug conjugateprecision medicine

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Repression of Multiple Myeloma Cell Growth In Vivo by Single-wall Carbon Nanotube (SWCNT)-delivered MALAT1 Antisense Oligos

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Last Updated: May 28, 2026

Establishment of a Human Multiple Myeloma Xenograft Model in the Chicken to Study Tumor Growth, Invasion and Angiogenesis
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Establishment of a Human Multiple Myeloma Xenograft Model in the Chicken to Study Tumor Growth, Invasion and Angiogenesis

Published on: May 1, 2015

Repression of Multiple Myeloma Cell Growth In Vivo by Single-wall Carbon Nanotube (SWCNT)-delivered MALAT1 Antisense Oligos
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Repression of Multiple Myeloma Cell Growth In Vivo by Single-wall Carbon Nanotube (SWCNT)-delivered MALAT1 Antisense Oligos

Published on: December 13, 2018

  • Examination of the regulatory decisions and market withdrawal of melflufen.

    • Main Results:

    • Melflufen demonstrated early encouraging activity in advanced MM, leading to accelerated FDA approval.
    • The Phase III OCEAN trial did not show an overall survival benefit for melflufen compared to standard care.
    • Concerns regarding overall survival led to the withdrawal of melflufen from the U.S. market.

    Conclusions:

    • Negative survival outcomes in the OCEAN trial significantly impacted melflufen's clinical trajectory.
    • The principles of enzyme-targeted drug delivery may still inform future MM therapies.
    • Biomarker-driven strategies and novel peptide-drug conjugates hold promise for refractory MM treatment.