The FDA granted orphan drug status to Lentigen's brain tumor stem cell treatment, giving the Maryland company market exclusivity for 7 years once the therapy reaches regulatory approval.
Lentigen founder and Chief Scientific Officer Boro Dropulic told FierceDrugDelivery that the orphan drug status, coveted by many companies, is an indication that "the FDA looked very seriously at our product and is happy with the outcome."
Lentigen's LG631-CD34 is designed to protect healthy cells from a particularly toxic cancer drug, temozolomide, a standard of care for patients with the aggressive malignant brain tumor glioblastoma. Using stem cells to modify DNA in a patient's blood, the treatment makes healthy cells resistant to the drug's side effects, allowing the safe delivery of larger and more frequent doses.
On the delivery side, the company's stem cell approach makes novel use of what is called a lentivirus, which is the "most efficient vector system known to permanently genetically modify human cells," Dropulic told FierceDrugDelivery. The vector, which is a virus tailored to deliver genetic material, is particularly adept at integrating protective measures into DNA over a long period of time, Dropulic said.
The National Institutes of Health is currently funding a Phase I clinical trial of the therapy.
"Glioblastoma multiforme is a serious brain cancer with an average life expectancy of less than 12 months after diagnosis," Dropulic told FDD. "The goal for this therapy is to permit dosing with Temodar that is therapeutically beneficial for patients without toxicity, resulting in more favorable patient outcomes."
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