for Cancer Treatment
Targeted therapies are designed to attack or kill cancer cells, while sparing normal cells as much as possible. These therapies are often designed to attach to abnormal proteins, receptors or genes that are found in high quantities in cancer cells or the surrounding tissue.
This section covers the following topics.
How are targeted therapies selected?
Some, but not all targeted therapies work best against cancer cells with certain markers or mutations. Two important types of tests can help guide selection of these targeted therapies.
- testing involves looking at tumor, blood or other other tissue samples for abnormal markers that might indicate the cancer is likely to respond to a particular . You can learn more about testing for selecting targeted therapies in our Biomarkers sections.
- Genetic testing for inherited mutations can also help guide treatment with targeted therapies. PARP inhibitors, for example, are a type of that are most effective for treating cancer in people with a or mutation.
Each drug has different indications. Like all cancer treatments, targeted therapies can have side effects. Visit our section on Side Effects for more information.
PARP inhibitors
PARP inhibitors work by blocking a protein used to repair damaged . They were initially developed to treat cancers in people with an inherited or mutation. Since then, research and additional approvals have expanded use of PARP inhibitors to more people and situations. There are five PARP inhibitors that have received approval for treating different types of cancers.
Indications vary by:
- type and of cancer: PARP inhibitors have been approved to treat breast, ovarian, pancreatic and cancers.
- presence of a mutation or biomarker: PARP inhibitors have been approved in different settings for:
- people with certain inherited mutations.
- people with certain acquired mutations.
- people with certain tumor biomarkers.
- women with certain types of ovarian cancer, regardless of the presence of an or .
- number of and response to prior treatments: have been approved in different settings for:
- platinum-sensitive or partially platinum-sensitive ovarian cancer.
- castration-resistent cancer (mCRPC).
- after chemotherapy.
- to treat progression or recurrence after a specific number of prior treatments.
Clinical trials studying PARP inhibitors in new settings or combinations are enrolling patients. As research continues, these approvals may expand to include treatment for additional cancers, earlier stages of cancer, people with other inherited mutations, and based on different tumor biomarkers.
Drug |
Cancer Type |
|
Use |
|
Lynparza () |
Breast cancer |
Early breast cancer at high risk for recurrence |
Given for one year as after completion of or chemotherapy and local treatment (surgery and, or radiation). |
or inherited mutation () |
Lynparza () |
Breast cancer |
For treatment of patients who have previously received chemotherapy, or hormone therapy for patients with hormone receptor disease. |
or inherited mutation and |
|
() |
Breast cancer |
For treatment of breast cancer. |
or mutation and |
|
Lynparza () |
Ovarian cancer |
3 or 4 |
maintenance therapy for people who had a complete or partial response to platinum chemotherapy. |
|
Lynparza () |
Ovarian cancer |
3 or 4 |
Combined with Avastin (bevacuzimab) for people who had a complete or partial response to platinum chemotherapy. |
|
Lynparza
|
Ovarian cancer |
3 or 4 |
Second-line or later for people who had a complete or partial response to platinum chemotherapy. |
No needed |
() |
Ovarian cancer |
3 or 4 |
For people who had a complete or partial response to platinum chemotherapy. |
No required |
Lynparza () |
Pancreatic cancer |
For people whose disease has not progressed on at least 16 weeks of platinum-based chemotherapy. |
in or |
|
Akeega ( and acetate) |
cancer |
castration-resistant cancer (mCRPC) |
In combination with prednisone for or later treatment of mCRPC. |
Inherited or tumor mutation in or based on FoundationOne tumor test |
Lynparza () |
cancer |
castration-resistant cancer (mCRPC) |
Combined with Zytiga and prednisone or prednisolone for or later treatment of mCRPC. |
|
Lynparza () |
cancer |
castration-resistant cancer (mCRPC) |
For treatment of mCRPC which has progressed following treatment with Xtandi () or Zytiga (). |
|
() |
cancer |
castration-resistant cancer (mCRPC) |
For treatment of mCRPC which has been treated with androgen receptor-directed therapy and a taxane-based chemotherapy. |
|
() |
cancer |
castration-resistant cancer (mCRPC) |
In combination with for mCRPC which has not yet been treated in the castration-resistant setting. |
Inherited or tumor mutation in one of the following genes: , , , ATR, CDK12, , FANCA, , MRE11A, , , or |
Other targeted therapies
There is a growing list of FDA-approved targeted therapies for cancer treatment. Most are classified by the abnormal target that they are designed to bind to and attack. Many of these new therapies are approved for use in all types of cancers () as long as the cancer contains the right marker or target. Drugs that are approved across cancer types are sometimes called pan-tumor or tumor-agnostic therapies.
Some drugs are classified as both immunotherapies and targeted therapies because they use antibodies to target abnormal proteins or receptors that are found in high quantities in cancer cells or the surrounding tissue. Antibody drug conjugates (ADCs) are drugs that combine two different types of molecules. A chemotherapy drug is linked to an antibody that delivers the chemotherapy directly to the cancer cells.
The table below lists some common targeted therapies used in cancer treatment. Clincal trials are studying new agents for treating cancer.
Drug |
Cancer Type |
|
Use |
|
Type of Agent |
Afinitor |
Breast cancer |
or advanced |
Combined with exemestane for postmenopausal women with advanced breast cancer which progressed with letrozole or anastrozole. |
, |
MTOR inhibitor |
Afinitor |
Pancreatic neuro-endocrine tumors (PNET) |
Progressive PNETs |
Used to treat PNETs that have progressed on other treatments. |
No needed |
MTOR inhibitor |
Avastin |
Ovarian, or primary peritoneal cancer |
2-4 |
Combined with Lynparza () for maintenance therapy for platinum-sensitive cancer. |
() |
VEGF inhibitor |
Avastin |
Ovarian, or primary peritoneal cancer |
3-4 |
Combined with chemotherapy, followed by Avastin as a single agent following initial surgical resection. |
No needed |
VEGF inhibitor |
Avastin |
Ovarian, or primary peritoneal cancer |
Recurrent |
Combined with chemotherapy for treating platinum-resistant, recurrent disease in people who received no more than 2 prior chemotherapy regimens. |
No needed |
VEGF inhibitor |
Avastin |
Ovarian, or primary peritoneal cancer |
Recurrent |
Combined with chemotherapy, followed by Avastin as a single agent, for platinum-sensitive recurrent disease. |
No needed |
VEGF inhibitor |
Avastin |
Colorectal cancer |
Combined with intravenous 5-fluorouracil-based chemotherapy for first-, or second-line treatment. |
No needed |
VEGF inhibitor |
|
Avastin |
Colorectal cancer |
Combined with chemotherapy for second-line treatment in patients who have progressed on a Avastin-containing regimen. |
No needed |
VEGF inhibitor |
|
Braftovi |
Colorectal cancer |
Combined with cetuximab, for the treatment of adult patients with colorectal cancer. |
BRAF V600E tumor mutation |
BRAF inhibitor |
|
Braftovi |
Melanoma |
Combined with Mektovi (binimetinib), for the treatment of patients with unresectable or melanoma. |
BRAF V600E or V600K tumor mutation |
BRAF inhibitor |
|
Cotellic |
Melanoma |
Combined with Zelboraf (vemurafenib) for the treatment of patients with unresectable or melanoma. |
BRAF V600E or V600K tumor mutation |
BRAF inhibitor |
|
Cyramza |
Colorectal cancer |
Combined with FOLFIRI chemotherapy, for treatment after disease progression on, or after prior therapy with bevacizumab, oxaliplatin, and a fluoropyrimidine. |
No required |
VEGF inhibitor |
|
Datroway (datopotamab deruxtecan-dlnk) |
Breast cancer |
Treatment for cancers that have progressed after hormone therapy and chemotherapy. |
Hormone receptor position (), |
Antibody-drug conjugate (chemotherapy attached to antibody targeting TROP-2) |
|
ELAHERE (mirvetuximab soravtansine-gynx) |
Ovarian cancer |
3-4 |
Used as second-line or later treatment of platinum-resistant or platinum-sensitive recurrent ovarian cancer. |
Positive for FRα (folate receptor alpha) |
Antibody-drug conjugate (chemotherapy attached to antibody targeting FR-α receptor) |
Enhertu (fam-trastuzumab-deruxtecan-nxki) |
Breast cancer |
Treatment for people who have received a prior anti-HER2-based regimen either:
|
overexpression () |
Antibody-drug conjugate |
|
Enhertu (fam-trastuzumab-deruxtecan-nxki) |
Breast cancer |
As treatment for people who have tumors that are HER2-low, received chemotherapy in the setting and whose cancer no longer responds to hormonal therapy. |
and HER2-low |
Antibody-drug conjugate |
|
Enhertu (fam-trastuzumab-deruxtecan-nxki) |
Breast cancer |
As treatment for people with , HER2-low or ultralow that came back or got worse after one or more hormone therapies in the setting. |
and HER2-low or HER2-ultra low |
Antibody-drug conjugate |
|
Enhertu (fam-trastuzumab-deruxtecan-nxki) |
(pan tumor) |
or unresectable |
For adult patients with unresectable or , solid tumors (including colorectal cancer) who have received prior systemic treatment and have no alternative treatment options. |
overexpression () |
Antibody-drug conjugate |
Erbitux |
Colorectal cancer |
Combined with FOLFIRI for treatment, or combined with irinotecan for cancers that no longer respond to irinotecan-based chemotherapy or as a single agent in patients who have progressed after oxaliplatin- and irinotecan-based chemotherapy. |
EGFR positive and KRAS mutation negative |
EGFR inhibitor |
|
Fruzaqla |
Colorectal cancer |
Used as a single agent when cancer has progressed after treatment with chemotherapy and . |
No required |
VEGF inhibitor |
|
Herceptin |
Breast cancer |
Early |
The treatment of breast cancer. |
overexpression () |
Antibody targeting receptors |
Herceptin (trastuzumab) and Tukysa (tucatinib) combination |
Colorectal cancer |
or unresectable |
For people who progressed after chemotherapy. |
overexpression () |
Antibody targeting receptors and a kinase inhibitor that targets receptors |
Ibrance |
Breast cancer |
Combined with an aromatase inhibitor as treatment of advanced cancer as initial hormone therapy in postmenopausal women or in men. |
and |
known as a kinase inhibitor that blocks the CDK4/6 pathway |
|
Ibrance |
Breast cancer |
Combined with Faslodex (fulvestrant) as treatment in postmenopausal women or in men whose disease progressed following endocrine therapy. |
and |
known as a kinase inhibitor that blocks the CDK4/6 pathway |
|
Kadcyla |
Breast cancer |
Early |
therapy for people with early breast cancer who still have disease after taxane and treatment with Herceptin |
overexpression () |
Antibody targeting receptors |
Kadcyla |
Breast cancer |
For treatment in people whose cancer got worse after receiving Herceptin and chemotherapy in the following settings:
|
overexpression () |
Antibody targeting receptors |
|
Kisqali |
Breast cancer |
Combined with an aromatase inhibitor for the treatment of pre/perimenopausal or postmenopausal women as initial hormone based therapy. |
and |
known as a kinase inhibitor that blocks the CDK4/6 pathway |
|
Kisqali |
Breast cancer |
Combined with Faslodex (fulvestrant) for the treatment of postmenopausal women, as initial hormone based therapy. |
and |
known as a kinase inhibitor that blocks the CDK4/6 pathway |
|
Krazati (adagrasib) |
Colorectal cancer |
In combination with cetuximab for locally advanced or colorectal cancer (CRC) that has progressed after treatment with chemotherapy. |
KRASG12C mutation |
against the KRASG12C protein |
|
Lenvima (lenvatinib) |
Endometrial cancer |
Advanced disease |
Combined with pembrolizumab, for the treatment of patients whose cancer has progressed after treatment and who are not candidates for surgery or radiation. |
Tumors that are not MSI-H or (or ) - they may be referred to as MSI-Low, MSS, pMMR or MMR-P) |
known as a tyrosine kinase inhibitor |
Mekinist (trametinib) |
Melanoma |
Unresectable or |
As a single agent and in combination with dabrafenib for the treatment of unresectable or melanoma. |
BRAF V600E or V600K tumor mutation |
known as a MEK inhibitor |
Mekinist (trametinib) |
Melanoma |
Lymph node positive |
Combined with Taflinar (dabrafenib) as treatment of people with melanoma and involvement of lymph node(s), following complete resection. |
BRAF V600E or V600K tumor mutation |
known as a MEK inhibitor |
Mektovi (binimetinib) |
Melanoma |
Unresectable or |
Combined with Braftovi (encorafenib), for the treatment of people with unresectable or melanoma. |
BRAF V600E or V600K tumor mutation |
known as a MEK inhibitor |
Orserdu |
Breast cancer |
Used alone to treat men or postmenopausal women with , breast cancer, which progressed after at least one line of hormone therapy therapy. |
, with an ESR1 mutation |
Type of targeted hormonal therapy known as SERD (selective receptor degrader or downregulator) |
|
Perjeta (pertuzumab) |
Breast cancer |
Locally advanced, inflammatory or early |
Combined with Herceptin (trastuzumab) and docetaxel as treatment before surgery (). |
overexpression () |
Antibody targeting receptors |
Phesgo (pertuzumab, trastuzumab combined injection) |
Breast cancer |
Early |
|
overexpression () |
Antibody targeting receptors |
Piqray |
Breast cancer |
Combined with Faslodex (fulvestrant) as treatment in men or post-menopausal women who progressed on or after treatment with hormone therapy. |
, and positive for a PIK3CA tumor mutation |
known as a kinase inhibitor that blocks the PIK3 pathway |
|
Pluvicto (lutetium Lu 177 vipivotide tetraxetan) |
cancer |
castration-resistant cancer (mCRPC) |
For treatment of mCRPC which has stopped responding or got worse after treatment with hormonal therapy using an androgen receptor inhibitor and taxane-based chemotherapy. |
Imaging with a that looks for cancers with the marker PSMA |
Targeted radiation therapy (radioligand therapy) |
Retevmo (selpercatinib) |
(pan tumor) |
or unresectable |
Second-line or later treatment for solid tumors for which there are no other treatment options. |
RET fusion |
Kinase inhibitor |
Stivarga (regorafenib) |
Colorectal cancer |
|
For treatment of colorectal cancer that has progressed after treatment and for which there are no other treatment options. |
No required |
known as a multi-kinase inhibitor |
Tafinlar |
Melanoma |
Unresectable or |
Combined with Mekinist (trametinib) for the treatment of people with unresectable or melanoma. |
BRAF V600E or V600K tumor mutation |
known as a BRAF inhibitor |
Tafinlar |
Melanoma |
Lymph node positive |
Combined with Mekinist (trametinib) as treatment of people with melanoma and involvement of lymph node(s), following complete resection. |
BRAF V600E or V600K tumor mutation |
known as a BRAF inhibitor |
Trodelvy (sacituzumab govitecan-hziy) |
Breast cancer |
For breast cancer that progressed, recurred or did not respond to at least two previous lines of treatment. |
Triple-negative (, ) |
Antibody-drug conjugate (chemotherapy attached to antibody found in ) |
|
Truqap |
Breast cancer |
Combined with fulvestrant as treatment for , advanced or breast cancer which recurred or got worse after standard hormone therapy. |
, PIK3 or AKT1 mutation in the tumor |
known as a kinase inhibitor that blocks the AKT pathway |
|
Tukysa (tucatinib) |
Breast cancer |
or unresectable |
In combination with Herceptin (trastuzumab) to treat cancer which has progressed after at least one prior treatment with an anti-HER2 treatment in the setting. |
overexpression () |
known as a kinase inhibitor that targets receptors |
Vectibix |
Colorectal cancer |
Combined with FOLFOX for treatment. |
Negative for KRAS and NRAS mutations |
that targets a receptor known as EGFR |
|
Vectibix |
Colorectal cancer |
As a single therapy following disease progression after prior treatment with fluoropyrimidine, oxaliplatin, and irinotecan-containing chemotherapy. |
Negative for KRAS and NRAS mutations |
that targets a receptor known as EGFR |
|
Verzenio (abemaciclib) |
Breast cancer |
Used alone to treat breast cancer that has progressed after treatment with hormone therapy and chemotherapy in the setting. |
and |
known as a kinase inhibitor that blocks the CDK4/6 pathway |
|
Verzenio (abemaciclib) |
Breast cancer |
Combined with Faslodex (fulvestrant) as treatment in women whose disease progressed following endocrine therapy. |
and |
known as a kinase inhibitor that blocks the CDK4/6 pathway |
|
Vitrakvi (larotrectinib) |
(pan tumor) |
or unresectable |
For treatment in solid tumors for which there are no other treatment options. |
NTRK fusion |
Kinase inhibitor |
Xofigo (Radium 223 dichloride) |
cancer |
castration-resistant cancer (mCRPC) |
For treatment of mCRPC that has spread to the bones but has not to other organs. |
No needed |
Targeted radiation therapy (radioligand therapy) |
Zelboraf (vemurafenib) |
Melanoma |
Unresectable or |
Combined with Tecentriq (atezolizumab) and Cotellic (cobimetinib) in people with melanoma that has the BRAF gene mutation, when the cancer can’t be removed by surgery or has spread to other parts of the body. |
BRAF V600E or V600K tumor mutation |
known as a BRAF inhibitor |
Clinicaltrials.gov identifier: NCT03803553
Study of a New InvestigationaI Inhibitor to Treat People with Advanced Solid Tumors
Clinicaltrials.gov identifier: NCT05932862
Study of the Drug Sovilnesib in People with Ovarian Cancer
Clinicaltrials.gov identifier: NCT06084416
NePtune: Using PARP Inhibitors Before Surgery in Localized Prostate Cancer
Clinicaltrials.gov identifier: NCT05498272