According to a story from BioSpace, the drug company Plus Therapeutics has recently announced that its investigational therapy Rhenium NanoLiposomes (RNL™) has earned Orphan Drug designation from the US Food and Drug Administration (FDA). The therapy is being developed as a treatment for recurrent glioblastoma, a deadly form of brain cancer. In addition, the therapy is proceeding into Cohort 6 of the phase 1 trial in which it is currently being investigated following approval from the trial’s Data Safety and Monitoring Board.

About Glioblastoma

Glioblastoma is a rare brain cancer. It is also the most aggressive cancer to originate in the brain. It is characterized by its rapid progression and poor response to most treatments. In most cases, the cause of glioblastoma is not known. A small number of cases evolve from another type of tumor called an astrocytoma. Risk factors for glioblastoma include genetic disorders such as Turcot syndrome and neurofibromatosis, exposure to pesticides, smoking, and a career in petroleum refining or rubber manufacture. Symptoms of glioblastoma include personality changes, headaches, memory loss, seizures, vomiting, and nausea; patients may lose consciousness in late stages. Treatment approaches include anticonvulsants, steroids, chemotherapy, radiation, and surgery. While a small number of patients can survive for several years, treatment is often ineffective, with the tumor relapsing quickly. Five year survival rate is only three percent. To learn more about glioblastoma, click here.

About Orphan Drug Designation

Orphan Drug designation is reserved primarily for treatments that are being developed for diseases that are considered rare, which is defined as any illness that affects less than 200,000 people in the US. To qualify, the drug must fulfill an unmet medical need or display potential advantages in efficacy and safety over current treatments. This designation confers a number of benefits to the recipient company, such as the waiving of certain fees, tax breaks, and a seven year period of market exclusivity if the drug ends up getting approval for public use from the FDA.

About The Trial and RNL

The phase 1 trial has a “dose-finding” component and the initiation of Cohort 6 is an important step in which a boosted dose, including a drug volume of 8.8 mL and a radiation dose of 22 millicuries (mCi), will be administered. RNL is being developed in order to deliver a very strong dose of radiation therapy directly to the affected brain tissue in a safe manner; this dose is as much as 25 times more than what is typically utilized.