Are there any drug treatments for EMPD?

Extramammary Paget's disease (EMPD) presents a unique challenge due to its rarity, which has resulted in a lack of dedicated therapeutics specifically formulated and tested for its treatment. However, in many countries, medical practitioners may resort to using approved therapies "off-label" for conditions like EMPD, though this practice is not without its limitations.

Traditionally, chemotherapy and immunotherapy have been the primary treatment modalities, leveraging anti-cancer drugs and the body's immune system, respectively. Some patients have explored hormone therapies and traditional medicines as alternative options. Yet, the efficacy of these conventional treatments remains limited, prompting a shift towards targeted therapies as a promising avenue for EMPD manage

Leading medical institutions have adopted next-generation sequencing (NGS) to identify molecular targets within EMPD tumors, expanding the range of therapeutic possibilities. By analyzing DNA mutations, clinicians can gauge the potential effectiveness of existing treatments tailored to similar genetic profiles. This proactive approach holds the promise of personalized treatment strategies, optimizing outcomes for individual patients.

Memorial Sloan Kettering Cancer Center (MSKCC) is a leader in next generation targeted therapies. The MSK IMPACT program – which stands for integrated mutation profiling of actionable cancer targets – was approved by the US Food and Drug Administration in 2017. It is now being used routinely by EMPD patients at MSKCC. It is hoped that as more data on EMPD is obtained, and shared, a clearer course of EMPD treatment will be formulated. Others in the USA providing genomic profiling tests include: Foundation One, Caris Life Sciences and the University of Washington Department of Laboratory Medicine and Pathology.

Despite the absence of dedicated treatments for extramammary Paget's disease (EMPD), patients have explored various therapies with varying levels of effectiveness. The names and availability of these treatments can differ significantly from one country or region to another.

The following list, compiled from experiences shared by diverse extramammary Paget's disease (EMPD) patients, serves as illustrative examples. However, it's imperative to emphasize that this information should not be utilized to devise treatment plans. Instead, individuals should consult their healthcare provider for personalized care tailored to their specific situation.

Adriamycin

Generic Name: Doxorubicin

Other Name: Rubex, Doxil

Summary: Adriamycin is an anthracycline antibiotic chemotherapy drug used to treat lymphomas, leukemias, bone sarcoma, breast cancer, endometrial cancer, gastric cancer, liver cancer, kidney cancer, multiple myeloma, neuroblastoma, ovarian cancer, small cell lung cancer, and thyroid cancer. Adriamycin is made from the bacterium streptomyces. Adriamycin is sometimes referred to as "The Red Devil" because of its red color.

Adrucil

Other Names: 5-fluorouracil , 5-FU, Carac, Fluorouracil (5FU), Fluoroplex, Efudex, and Tolak

Summary: Fluorouracil (5FU) is a chemotherapy drug. It is often used to treat breast, bowel, skin, gastrointestinal, oesophageal, and pancreatic cancers. In topical use (cream or solution) it is sometimes used on basal cell cancer and actinic keratoses.

Agaricus

Other Names: Himematsutake, Agarikusutake, Kawarihiratake, Cogumelo do Sol, Sun mushroom

Summary: Agaricus blazei is an edible mushroom grown in Japan and Brazil. In traditional medicine, it has been used to treat various ailments including diabetes and cancer.

Aldara

Generic Name: Imiquimod

Other Names: Perrigo, Zyclara

Summary: Aldara is a an immune response modifier used to treat actinic keratoses, basal cell carcinoma (BCC), intraepidermal carcinomas, squamous cell carcinoma (SCC) and genital warts. Imiquimod stimulates the immune system to release cytokines. More information on Aldara (Imiquimod) can be found on the Topical Treatments for Extramammary Paget’s Disease (EMPD) page.

Arimidex

Generic Name: Anastrozole

Summary: Anastrozole is an aromatase inhibitor designed to treat postmenopausal women diagnosed with hormone-receptor-positive breast cancer. Arimidex is sometimes taken in combination with Tamoxifen.

Aromasin

Generic Name: Exemestane

Summary: Exemestane is an aromatase inhibitor designed to treat postmenopausal women diagnosed with hormone-receptor-positive breast cancer.

Casodex

Generic Name: Bicalutamide

Summary: Casodex is an oral non-steroidal anti-androgen hormone therapy for prostate cancer.

Camptosar

Generic Name: Irinotecan

Other Name: Onivyde

Summary: Medication used to treat colon and small cell lung cancers. It is sometimes used with fluorouraci or other cancer medicines in a combination chemotherapy.

Cytoxan

Generic Name: Cyclophosphamide

Other Name: Neosar

Summary: Cytoxan is an alkylating chemotherapy often used to treat lymphomas, leukemias, multiple myeloma, neuroblastoma, retinoblastoma, breast cancer, and ovarian cancer.

Docetaxel

Other Names: Taxotere, Docefrez

Summary: Docetaxel is used in treatment of breast cancer, non-small cell lung cancer, advanced stomach cancer, head and neck cancer and metastatic prostate cancer. It is also being investigated to treat small cell lung, ovarian, bladder, and pancreatic cancers, soft tissue sarcoma and melanoma.

EFUDEX

Generic Name: Fluorouracil Topical

Other Names: Carac, Fluoroplex, Tolak

Summary: Efudex is a topical that interferes with the growth of skin cells. Efudex works by causing the death of abnormal skin cells which are typically growing the fastest.

Evista

Generic Name: Raloxifene

Summary: Raloxifene is classified as a Selective Estrogen Receptor Modulator (SERM). It is a hormone therapy used to decrease the risk of breast cancer in postmenopausal women who have osteoporosis or a high risk of breast cancer.

Eligard

Generic Name: Leuprolide

Other Names: Lupron, Lupron Depot, Viadur

Summary: Eligard is a hormone therapy. Eligard is used to treat prostate, breast, ovarian and endometrial cancers.

Ellence

Generic Name: Epirubicin

Summary: Epirubicin is a chemotherapy drug often used in treating breast cancer.

Enhertu

Generic Name: Trastuzumab Deruxtecan

Summary: Enhertu is a HER2-directed antibody and topoisomerase inhibitor that is used to treat types of breast cancer, stomach cancer, and non-small cell lung cancer that have specific HER2 gene mutations.

FECOM

Summary: A combination regimen of 5-Fluorouracil, Epirubicin, Carboplatin, Vincristine, and Mitomycin is often known as FECOM.

Femara

Generic Name: Letrozole

Summary: Letrozole lowers estrogen levels in postmenopausal women, which may slow the growth of certain types of breast tumors that need estrogen to grow in the body.

FOLFOX

Summary: FOLFOX is a chemotherapy regimen for treatment of colorectal cancer including the drugs Folinic acid (leucovorin), Fluorouracil (5-FU) and Oxaliplatin (Eloxatin).

Fucoidan

Other names: Sulfated alpha-L-fucan, Fucoidin, Fucan, Mekabu fucoidan

Summary: Fucoidan is a complex polysaccharide found in many species of brown seaweed. Some studies suggest that it can prevent the growth of cancer cells and has antiviral, neuroprotective, and immune-modulating effects. However, it has not yet been studied in humans.

Gemzar

Generic Name: Gemcitabine

Summary: Gemzar is a chemotherapy drug often used in treating pancreas cancer, non-small cell lung cancer, bladder cancer, soft-tissue sarcoma, metastatic breast cancer and ovarian cancer.

Halaven

Generic Name: Eribulin

Summary: Eribulin is an anti-cancer medication used to treat locally advanced or metastatic breast cancer and liposarcoma.

Herceptin

Generic Name: Trastuzumab

Other Names: Herzuma (Biosimilar)

Summary: Herceptin is a monoclonal antibody. Herceptin is used to treat metastatic breast cancer. It is most effective against tumors that have the HER2/neu protein.

Imlygic

Generic Name: Talimogene Laherparepvec

Other Names: T-VEC

Summary: Imlygic is a biopharmaceutical drug used to treat melanoma lesions that cannot be operated on.

Kisqali

Generic Name: Ribociclib

Summary: Kisqali is an anti-cancer kinase inhibitor of cyclin D1/CDK4 and CDK6, and is used for the treatment of certain kinds of breast cancer.

Keytruda

Generic Name: Pembrolizumab

Summary: Keytruda is a immunotherapy that works with the immune system to help fight certain cancers. Keytruda has been used alone or in combination with other medicines to treat various cancers including: skin cancer, lung cancer, Hodgkin lymphoma, liver cancer, stomach cancer, squamous cell carcinoma of the esophagus, renal cell carcinoma and endometrial carcinoma.

Nerlynx

Generic Name: Neratinib

Summary: Nerlynx is an anti-cancer (antineoplastic) chemotherapy drug used on breast cancer.

Opdivo

Generic Name: Nivolumab

Summary: Nivolumab is a targeted therapy. It is a human programmed death receptor-1 (PD-1) blocking antibody. It is used to treat metastatic melanoma, non-small cell lung cancer (NSCLC), advanced renal cell cancer, Hodgkins lymphoma (cHL), etastatic squamous cell carcinoma of the head and neck (SCCHN), metastatic small cell lung cancer (SCLC) and hepatocellular cancer (HCC).

Paraplatin

Generic Name: Carboplatin

Summary: Carboplatin is an anticancer drug chemotherapy drug used to treat ovarian, lung, head and neck, endometrial, esophageal, bladder, breast, and cervical cancers.

Perjecta

Generic Name: Pertuzumab

Summary: Projecta is used to treat HER2-positive breast cancer.

PET

Summary: A combination regimen of Cisplatin, Epirubicin and Paclitaxel is often known as PET.

PIQRAY

Generic Name: Alpelisib

Summary: Piqray is often used in combination with the hormonal therapy Faslodex to treat metastatic hormone-receptor-positive, HER2-negative breast cancer with a PIK3CA mutation.

Platinol

Generic Name: Cisplatin

Summary: Cisplatin is a chemotherapy drug often used in treating bladder cancer, metastatic ovarian cancer, and metastatic testicular cancer.

Rapamycin

Other Name: Sirolimus

Summary:  Rapamycin is an immunosuppressant topical cream used to coat coronary stents. The drug’s name comes from Rapa Nui, the indigenous name of Easter Island, where the compound was originally discovered in soil samples.

Soltamox

Generic Name: Tamoxifen

Other Names: Nolvadex, Apo-Tamox, Tamofen and Tamone

Summary:  Tamoxifen is a selective estrogen receptor modulator (SERM). It is used to reduce breast cancer risk in women.

T-DM1

Generic Name: Ado-trastuzumab emtansine

Other Names:  Kadcyla, Trastuzumab emtansine

Summary: T-DM1 is a drug used to treat HER2-positive breast cancer. Known as an antibody–drug, T-DM1 chemically links the Trastuzumab antibody to the chemotherapy drug Emtansine (also known as DM1).

Taxol

Generic Name: Paclitaxel

Other Names: Onxal, Abraxane

Summary: Taxol is a chemotherapy drug. It is often used to treat breast, ovarian, lung, bladder, prostate, melanoma, esophageal, and other types of solid tumor cancers.

Verzenio

Generic Name: Abemaciclib

Summary: Abemaciclib is a kinase inhibitor. Abemaciclib works by blocking the action of an abnormal protein that signals cancer cells to multiply. Abemaciclib is sometimes used in combination with an aromatase inhibitor such as anastrozole (Arimidex), exemestane (Aromasin), or letrozole (Femara) to treat a certain type of hormone receptor-positive, early breast cancer.

Xeloda

Generic Name: Capecitabine

Summary: Often used to treat breast or colorectal cancers.

Yervoy

Generic Name: Ipilimumab

Summary: Ipilimumab is a type of immunotherapy known as a checkpoint inhibitor, which helps the immune system attack cancer cells. Ipilimumab is an anti-CTLA-4 inhibitor and has been approved to treat colorectal cancer, liver cancer, malignant pleural mesothelioma, melanoma, non-small cell lung cancer and renal cell carcinoma.

Zoladex

Generic Name: Goserelin

Summary: Hormone associated therapy for prostate and breast cancers.

This list is created from information shared by varied extramammary Paget's disease (EMPD) patients. The content presented on this website, encompassing both textual material and visual media, serves educational and informational purposes aimed at aiding individuals affected by extramammary Paget’s disease (EMPD) and their families in comprehending the complexities associated with EMPD. It is imperative to note that the website is not designed to serve as a replacement for professional medical advice, diagnosis, or treatment. Furthermore, the website does not advocate for or endorse any specific treatments, tests, physicians, procedures, or products. Should you suspect that you are experiencing symptoms of EMPD, we strongly advise you to seek guidance from your healthcare provider.