It’s no secret that, in many cases, funding for rare disease research and drug development is slim; the financial burden often falls to families and patients. 40 years ago, the Orphan Drug Act was created to attempt to lessen this burden. Drugs designed for rare conditions—or conditions affecting under 200,000 people nationally—may be granted Orphan Drug designation by the FDA. This comes with a host of benefits, from fee waivers to seven years of market exclusivity upon approval, to incentivize drug development in this space. Both INB-400 and INB-410, developed for multiple forms of glioma and including newly diagnosed glioblastoma, received Orphan Drug designation in late April 2023, shares Pharmaceutical Technology.
Drug developer IN8bio describes INB-400 as a genetically modified DeltEx autologous drug created from altered gamma-delta T cells. INB-410 is the allogeneic form of drug-resistant immunotherapy (DRI) technology.
Moving forward, IN8bio plans to launch a Phase 2 clinical study to test INB-400’s safety, efficaciousness, and tolerability in people with newly diagnosed glioblastoma. If all goes well, the company will begin enrolling patients later this year.
What is Glioblastoma?
Also known as glioblastoma multiforme (GBM), glioblastoma is a rare and aggressive form of primary brain cancer. It forms from star-shaped astrocyte cells in the brain, often in the cerebrum. Even though glioblastoma is rare, it is also the most common form of primary brain cancer. Men are more likely to develop them than women. Having certain genetic disorders, or having had radiation therapy, also increases the risk.
Glioblastomas are difficult to treat. These tumors can create their own blood supply, helping them to grow and spread. Surgery, radiation, chemotherapy, and electric-field therapy are all used as treatments. Unfortunately, the prognosis is still poor; more research and drug development is needed to help patients live a longer life.
When symptoms of glioblastoma appear, they may include:
- Nausea and vomiting
- Double or blurred vision
- Impaired language
- Drowsiness
- Weakness on one side of the body
- Persistent headaches
- Changes in mood, behavior, or personality
- Difficulty thinking or speaking
- Seizures
