Targeted Therapy for Multiple Myeloma
Medically reviewed by: Craig Cole, MD
Last Updated: August 2026
Targeted therapy is a type of treatment that uses special drugs to attack multiple myeloma cells. The type of targeted therapy you receive depends on the results of biomarker testing. These drugs block certain genes or proteins that cancer cells need to grow, divide, and spread. Because they target cancer cells more precisely, they may cause less damage to healthy cells.
Targeted therapy may be given as a pill (by mouth), as an IV (through a vein), or as an injection (shot).
There are different types of targeted therapy to treat multiple myeloma:
- Immunomodulatory drugs (IMiDs): Help the immune system work better so it can attack myeloma cells.
- Cereblon E3 ligase modulatory drugs (CELMoDs): Work in a similar way as IMiDs. They are newer and stronger medicines that are designed to work better, including against some myeloma that has become resistant to IMiDs.
- Proteasome inhibitor therapy (PIs): Stop myeloma cells from getting rid of their “protein trash.” When this waste builds up, the cancer cells become overwhelmed and die.
- Selective inhibitors of nuclear export (SINE): Block a specific protein inside myeloma cells called exportin 1 (XPO1).
These are the targeted therapy drugs that are used most often to treat multiple myeloma. New treatments become available all the time, so this may not be a complete list.
Please note: The side effects listed here do not represent a comprehensive list. Each targeted therapy and drug has its own set of side effects and ways of working. It's important to discuss all potential side effects with your healthcare team. Be sure to tell them about any side effects you have.
All Available Treatments
Treatment Type
Immunomodulatory drugs (IMiDs)
Drug Names
Potential Side Effects
Nausea/vomiting
Diarrhea
Constipation
Fatigue/weakness
Infections
Drowsiness
Shortness of breath
Neuropathy
Low blood counts
Important Things to Know
- Neuropathy is a condition caused by damage to the peripheral nervous system. It can cause symptoms like pain, weakness, tingling, numbness, or sensitivity, often in the hands or feet. The neuropathy can be severe, and it might not go away after the drug is stopped.
- These drugs should not be used during pregnancy. They can cause severe, life-threatening birth defects or death to an unborn baby.
- Low blood counts may put you at risk for anemia, infection, bleeding or bruising.
Treatment Type
Cereblon E3 ligase modulatory drugs (CELMoDs)
Drug Name
Potential Severe Side Effects
Blood clots
Secondary cancer
Severe birth defects if taken during pregnancy
Potential Side Effects
Nausea/vomiting
Diarrhea
Constipation
Fatigue/weakness
Infections
Dizziness
Sleeping problems
Muscle pain
Neuropathy
Skin/nail changes
Low blood counts
Important Things to Know
- These drugs should not be used during pregnancy. They can cause severe, life-threatening birth defects or death to an unborn baby.
- Low blood counts may put you at risk for anemia, infection, bleeding, or bruising.
Treatment Type
Proteasome inhibitor therapy (PIs)
Drug Names
Potential Side Effects
Nausea/vomiting
Fatigue/weakness
Diarrhea
Constipation
Fever
Shortness of breath
Weight/appetite changes
Swelling
Body Pain
Low blood counts
Important Things to Know
- Low blood counts may put you at risk for anemia, infection, bleeding or bruising.
- Proteasome inhibitor therapy (PIs): PIs stop myeloma cells from getting rid of their “protein trash.” When this waste builds up, the cancer cells become overwhelmed and die.
Treatment Type
Selective inhibitors of nuclear export (SINE)
Drug Name
Potential Severe Side Effects
Infections
Potential Side Effects
Nausea/vomiting
Fatigue/weakness
Diarrhea
Constipation
Fever
Weight/appetite changes
Low blood counts
Important Things to Know
- Low blood counts may put you at risk for anemia, infection, bleeding or bruising.
- Selective inhibitors of nuclear export (SINE): Block a specific protein inside myeloma cells called exportin 1 (XPO1).