Targeted Therapy
Targeted therapy is a treatment approach that uses drugs designed to act on specific genetic or molecular changes found in cancer cells, rather than affecting all rapidly dividing cells broadly. In blood cancers, it is used when testing identifies a specific mutation or marker that a targeted drug can act against.
Understanding Targeted Therapy
Cancer cells often carry specific genetic mutations or protein changes that drive their growth. Targeted therapy uses drugs designed to interfere with these specific changes, aiming to affect cancer cells more precisely while sparing more of the surrounding healthy tissue compared to traditional chemotherapy. This precision depends entirely on identifying the right target through diagnostic testing.
In blood cancers, targeted therapy is used for specific conditions where a known genetic marker exists, such as certain forms of chronic myeloid leukemia, some subtypes of acute leukemia, and particular lymphoma or myeloma subtypes with identifiable molecular features. Because these drugs act on a specific target, they are only effective if that target is actually present in your cancer, which is why molecular and genetic testing is a required step before starting treatment.
Targeted therapy is often given as oral tablets, which can make it more convenient than intravenous chemotherapy for some patients, though this varies by specific drug. It may be used as a primary treatment, in combination with chemotherapy, or as ongoing maintenance therapy, depending on the blood cancer type and how the disease responds.
What the treatment involves
Molecular and genetic testing
Blood and bone marrow samples are tested for specific genetic mutations or molecular markers that determine whether a targeted therapy drug is likely to be effective for your particular cancer.
Treatment selection
Based on the identified marker and your specific diagnosis, an appropriate targeted therapy drug is selected, following recognized treatment protocols for that particular genetic subtype.
Baseline assessment
Organ function and blood counts are checked before starting, since some targeted therapies require monitoring for specific effects on the heart, liver or blood counts.
Administration and dosing
Most targeted therapies are taken as daily oral tablets at home, though some are given by infusion; your care team explains the specific dosing schedule for your drug.
Ongoing monitoring
Regular blood tests and, for some conditions, molecular testing track how well the cancer is responding and monitor for any drug-specific side effects that need dose adjustment.
Long-term follow-up
Depending on the disease and response, targeted therapy may continue for an extended period as maintenance treatment, with periodic reassessment to confirm it remains the right approach.
Who this treatment may be considered for
What to expect
Many targeted therapies are taken as daily tablets at home, which can reduce the number of hospital visits compared to intravenous treatments, though regular monitoring visits are still required.
Side effects vary by specific drug and can include fatigue, skin changes, gastrointestinal symptoms or effects on specific organs, which the care team monitors through scheduled blood tests.
Treatment duration varies considerably; some targeted therapies are taken for a defined period, while others, particularly for certain leukemias, may be continued long-term as maintenance treatment.
Because effectiveness depends on the presence of a specific molecular target, follow-up testing is used to confirm the drug continues to be appropriate over time.
Adherence to the prescribed dosing schedule is important for targeted therapy to work as intended, and your care team will discuss this with you.
Why choose AP Blood & Blood Cancer Centre
AP Blood & Blood Cancer Centre's single-specialty focus means molecular testing results and targeted therapy selection are interpreted within a dedicated hematology framework, supporting continuity between diagnosis and treatment choice.
Dr. Amarnadh Polisetty's clinical haematology training and experience managing hematologic malignancies, including targeted therapy protocols, allows treatment selection to be matched to each patient's specific genetic and molecular findings, in line with recognized national and international guidelines.
Common questions about Targeted Therapy
Medically reviewed by Dr. Amarnadh Polisetty, MD, DNB (Clinical Haematology)
Last reviewed on 1 July 2026
Curious whether targeted therapy applies to your diagnosis?
Speak with a clinical haematologist about molecular testing and whether targeted therapy is a suitable option for you.