Melphalan: What It Is, How It's Used, and What You Need to Know

When you hear Melphalan, a chemotherapy drug classified as an alkylating agent used to kill fast-growing cancer cells. Also known as L-PAM, it’s been a backbone treatment for decades in cancers like multiple myeloma and certain types of ovarian cancer. Unlike newer targeted therapies, Melphalan works by directly damaging the DNA inside cancer cells, stopping them from dividing and spreading. It’s not flashy, but it’s proven—and for many patients, it’s still the most effective option they have.

Melphalan doesn’t work alone. It’s often part of a combo plan, especially before a stem cell transplant. Doctors use high doses to wipe out bone marrow, then rebuild it with healthy stem cells. This approach, called high-dose Melphalan with autologous stem cell rescue, is standard for younger multiple myeloma patients who can handle the intensity. For others, lower doses are given orally or intravenously to slow disease progression without the transplant. The choice depends on age, overall health, and how far the cancer has spread.

It’s not just about the drug itself—it’s about what it connects to. Melphalan is tied to multiple myeloma, a blood cancer that affects plasma cells in the bone marrow, which is why most of the research and patient stories revolve around it. But it also plays a role in treating ovarian cancer, especially when other drugs stop working, and occasionally in some lymphomas and sarcomas. Side effects like low blood counts, nausea, and fatigue are common, but they’re managed with supportive care—something every patient needs to understand before starting.

What you won’t find in every guide is how Melphalan fits into the bigger picture of cancer care. It’s one of the older drugs, but it’s still in use because it works where newer options fail. It’s cheaper than many biologics, which matters for long-term treatment. And while newer drugs like lenalidomide or bortezomib get more attention, Melphalan remains a critical tool—especially in resource-limited settings or when patients need a reliable, time-tested option.

Below, you’ll find real patient guides, clinical comparisons, and practical advice on how Melphalan fits into treatment plans. You’ll see how it stacks up against other chemo drugs, what side effects to watch for, and how to prepare for the process. Whether you’re a patient, caregiver, or just trying to understand what’s happening, this collection gives you the facts without the fluff.