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Related Concept Videos

Treatment Resistant Cancers02:56

Treatment Resistant Cancers

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Cancer is the second leading cause of death in the United States. A cancer cell is genetically unstable and hence can mutate faster. They can also modify their microenvironment and escape immune surveillance. The difficulties in treating cancer are further compounded by the emergence of rapid resistance to anticancer drugs. The most common ways to attain resistance in cancer cells include alteration in drug transport and metabolism, modification of drug target, elevated DNA damage response, or...
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Plain language summary of publication of how long people with relapsed multiple myeloma lived after treatment in the IKEMA study: Isatuximab plus carfilzomib and dexamethasone versus carfilzomib and dexamethasone.

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Melflufen for relapsed and refractory multiple myeloma.

Albert Oriol1, Alessandra Larocca2, Xavier Leleu3

  • 1Institut Josep Carreras and Institut Català d'Oncologia, Hospital Germans Trias I Pujol , Badalona, Spain.

Expert Opinion on Investigational Drugs
|September 14, 2020
PubMed
Summary

Melphalan flufenamide (melflufen) shows promise for treating relapsed or refractory multiple myeloma. This novel peptide-drug conjugate offers a new mechanism of action and a manageable safety profile for patients with limited options.

Keywords:
Melflufenmelphalan flufenamiderelapsed/refractory multiple myeloma

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Area of Science:

  • Oncology
  • Hematology
  • Pharmacology

Background:

  • Multiple myeloma survival has improved but remains incurable.
  • Novel agents are increasingly used, creating a need for new mechanisms of action in refractory disease.
  • Melphalan flufenamide (melflufen) shows activity in relapsed/refractory multiple myeloma.

Purpose of the Study:

  • To review the mechanism of action of melflufen.
  • To summarize clinical trial data for melflufen in combination therapies.
  • To describe the safety profile of melflufen.

Main Methods:

  • Review of melflufen's mechanism as a peptide-drug conjugate.
  • Analysis of Phase I and II clinical trial data.
  • Evaluation of safety data, focusing on adverse events.

Main Results:

  • Melflufen targets aminopeptidases, delivering alkylating agents to tumor cells.
  • Clinical trials investigated melflufen with dexamethasone, daratumumab, or bortezomib.
  • The safety profile is mainly characterized by manageable hematologic adverse events.

Conclusions:

  • Melflufen offers a new mechanism of action for multiple myeloma treatment.
  • It demonstrates clinically meaningful efficacy in patients refractory to multiple novel agents.
  • Melflufen presents a favorable safety profile, addressing an unmet medical need.