Novantrone (mitoxantrone for injection concentrate) is an intravenously delivered synthetic molecule for the treatment of multiple sclerosis (MS). It is indicated to reduce neurologic disability and frequency of relapses in patients with worsening relapsing-remitting MS (RRMS), patients with secondary progressive multiple sclerosis (SPMS), and patients with progressive-relapsing MS (but not primary-progressive MS).
In 2000, Novatrone became the first treatment approved by the U.S. Food and Drug Administration (FDA) for the treatment of SPMS.
It was originally developed by Immunex, which is now part of Amgen. The rights to distribute Novantrone were sold to EMD Serono, who has since chosen to discontinue distributing Novantrone. This was not due to safety concerns. The drug is still available under the generic name mitoxantrone hydrochloride.
History of Novantrone
Two major clinical trials provided the safety and efficacy data for Novantrone in multiple sclerosis patients, that led to its approval by the FDA. Patients in the first study were diagnosed with secondary progressive or progressive relapsing MS. They were randomized into three groups: placebo (control), 5 mg/m² Novantrone, or 12 mg/m² Novantrone. Treatments were administered intravenously every three months over two years. Patients were also evaluated for side effects at the three-month follow-up. Any relapses were treated with high-dose methylprednisolone. A total of 149 patients completed the full two years of treatment.
Patients in the second study were diagnosed with secondary progressive or worsening RRMS with residual neurological deficit between relapses. After an initial screening period to identify patients who developed at least one new Gd-enhancing MRI lesion in two months, 42 patients were treated with a combination of methylprednisolone and Novantrone. Half were treated with an intravenous dose of 1g of methylprednisolone alone, and half were treated with an intravenous combination of approximately 12 mg/m² of Novantrone and 1g of methylprednisolone. Treatment lasted six months, and patients were evaluated monthly.
At the conclusion of these two studies, researchers identified fewer new lesions, fewer relapses, less neurological decline, and less physical decline in Novantrone-treated patients.
How Novantrone Works
Novantrone interacts with DNA. The molecule inserts itself within DNA strands, causing the DNA to break apart. This affects the proliferation of cells, specifically B-cell, T-cell, and macrophage cell proliferation. These are the key auto-reactive cells in that cause inflammation in MS, so preventing their proliferation reduces disease activity.
Novantrone also reduces the production of interferon gamma, tumor necrosis factor-alpha (TNF), and interleukin-2 (IL2), which are inflammatory proteins over-produced in MS patients. Reducing the inflammatory environment of the nervous system helps protect neurons from further damage.
Common side effects from Novantrone treatment can include nausea, temporary hair thinning, temporary blue-green urine, urinary tract and airway infections, and changes to menstruation pattern. More serious side effects can include heart disease, liver damage and therapy-related acute leukemia (TRAL).
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