Exploring leptomeningeal disease treatments
The information presented here is intended to empower patients to start a conversation about treatment options with their doctor.
The goals of treatment for leptomeningeal disease (LMD) are to prolong survival & improve or stabilize neurological symptoms
Selecting which options are best for your particular case will depend upon the recommendations from the doctor guiding your treatment, as well as your own personal preferences.
Depending on your breast cancer subtype, chemotherapy, endocrine therapy, immunotherapy, and/or targeted therapy may be used to treat LMD. Radiation therapy may also be recommended, particularly to relieve pain or neurological symptoms. All interventions mentioned below can be used in combination, sometimes simultaneously. For example, a patient with LMD may receive IV chemotherapy to control the cancer outside of the central nervous system; intrathecal chemotherapy to control the LMD; and radiation therapy to address issues with pain.
Radiation therapy
Radiation therapy may be delivered to either the brain or spinal cord, depending on the location of the metastasis. Typically SRS is not used for leptomeningeal disease.
Learn more about radiation therapy in the brain metastasis treatments section
Chemotherapy
Patients with LMD can receive chemotherapy in three different ways – by swallowing a pill (oral), through a needle in your vein (IV), or intrathecally (IT) which means directly into the cerebral spinal fluid (CSF).
Intrathecal delivery of chemotherapy can occur via a lumbar puncture, or via a special port called an Ommaya. Similar to a port placed in the chest to access blood and deliver drugs directly to the bloodstream, the Ommaya port is surgically implanted in the skull and allows for access to the CSF to deliver drugs directly to the central nervous system. The patient receives a local injection (shot) of an anesthetic to numb the site where the Ommaya port will be placed, which makes the procedure very tolerable for most patients.
| Treatment Options Delivered Directly to the Brain or Spinal Cord for Leptomeningeal Disease | |||
| Drug Class | Drug Delivery | Drug Name | Breast Cancer Type |
| Therapeutic Antibody | IT infusion | trastuzumab (Herceptin®) | HER2+ |
| Chemotherapy | IT infusion | methotrexate, liopsomal cytarabine, topotecan | All |
Treatments in Clinical Trials
While research is still limited, there are several ongoing trials to investigate novel treatment options including immunotherapy, targeted therapy, chemotherapy, and radiation therapy for patients with LMD. You can find a summary of the drugs and biologics currently in trials below. You can also use the tool on this page to search for clinical trials after answering a few questions about your breast cancer.
To see all leptomeningeal disease trials, check out our trial search
| Novel Treatment Options for Leptomeningeal Disease in Clinical Trials | |||
| Drug Class | Drug Delivery | Drug Name | Breast Cancer Type |
| Chemotherapy | IV infusion | ANG1005 (GRN1005) | HER2- negative |
| Antibody-drug conjugate | IV infusion————— | ado-trastuzumab emtansine (T-DM1, Kadcyla®), trastuzumab deruxtecan (DS-8201, Enhertu®) | HER2+———————————— |
| ATR inhibitor | Oral pill | ceralasertib (AZD6738) | HER2+ |
| EGFR inhibitor | Oral pill | afatinib (Gilotrif®) | HER2+ |
| HER2 inhibitor | Oral pill | tucatinib (Tukysa®), neratinib (Nerlynx®), lapatinib (Tykerb®) |
HER2+ |
| Immunotherapy | IT infusion | HER2 CAR T-cell therapy | HER2+ |
| Therapeutic Antibody | IT infusion | pertuzumab (Perjeta®), trastuzumab (Herceptin®) | HER2+ |
| PI3K/mTOR inhibitor | Oral pill | paxalisib (GDC-0084) | All with PI3K mutation |
| Chemotherapy | IV infusion | high-dose methotrexate (HD-MTX), irinotecan hydrochloride, pemetrexed (Alimta®) | All |
| Chemotherapy | Oral pill | temozolomide (Temodar®) | All |
| Immunotherapy | IT infusion | anti-HER2 bispecific antibody armed activated T cells (HER2 BATs) | All |
| Immunotherapy | IV infusion | ipilimumab (Yervoy®), nivolumab (Opdivo®), pembrolizumab (Keytruda®), pritumumab | All |
| Radiation Therapy | Directly to brain or spine | proton radiation therapy | All |
| Therapeutic Antibody | IT infusion | iodine I 131 3F8, iodine I 131 MOAB 8H9, 177Lu-DTPA-omburtamab |
All |
| Therapeutic Antibody | IV infusion | avelumab (Bavencio®) | All |
| VEGF inhibitor | Oral pill | lenvatinib (MK-7902, Lenvima®) | All |
Explore our frequently asked questions
The information on this website is intended for general knowledge only and is not a substitute for medical advice or treatment. The content for this website has been reviewed by our medical advisory board who are experts in the field.
