Novel Vaccine for Myelodysplastic Syndrome Granted Orphan Drug Designation


One-third of patients with myelodysplastic syndrome progress to acute myeloid leukemia.

Boston Biomedical recently announced that the FDA has granted the novel drug DSP-7888 orphan drug designation. DSP-7888 is an experimental cancer peptide vaccine for the treatment of myelodysplastic syndrome (MDS), a group of rare blood disorders characterized by the abnormal development of blood cells in the bone marrow, according to a press release.

The candidate vaccine contains peptides that induce Wlms’ tumor gene 1 (WT1)-specific cytotoxic lymphocytes and T cells, which attack cells that express WT1 found in numerous cancer types.

Previously, the National Cancer Institute ranked WT1 as a priority for cancer immunotherapy.

Boston Biomedical reports that up to 15,000 patients are diagnosed with MDS in the United States each year, with one-third of patients progressing to acute myeloid leukemia. The high prevalence of cancer cases resulting from MDS highlights the need for preventative treatments, according to the release.

Clinical data from a phase 1/2 clinical trial of patients who progressed on first-line treatment with azacitidine treatment were previously presented at the 2016 American Society of Hematology Annual Meeting and Exposition, according to the release.

The investigators reported that DSP-7888 was well-tolerated in patients with MDS. They also noted preliminary signs of clinical benefits, according to the release.

DSP-7888 is currently being evaluated in 3 studies: a phase 1/2 study in MDS, a phase 1/2 study in pediatric patients with relapsed or refractory high-grade gliomas, and a phase 1 study in advanced cancers.

The vaccine will also be examined in combination with bevacizumab in a phase 2 study in patients with recurrent or progressive glioblastoma, according to Boston Biomedical.

“Receiving an Orphan Drug Designation for DSP-7888 reinforces our dedication to helping address an unmet medical need for people with myelodysplastic syndrome, where other therapies are still needed,” said Patricia S. Andrews, chief executive officer of Boston Biomedical, Inc. “We are committed to uncovering the potential therapeutic benefit of cancer peptide vaccines, and we look forward to advancing the clinical development of this first-in-class compound.”

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