TTX101 for Malignant Gliomas (Including Glioblastoma!) Earns Orphan Drug Designation

 

Right now, there is an urgent need to identify and develop treatments for people with malignant gliomas, including glioblastoma. Unfortunately, there are limited effective treatment options; these cancers are often extremely aggressive and treatment-averse. To best help patients, new interventions are needed. 

Biotechnology company TargTex is currently working to address this unmet need through the development of TTX101. According to OncLive, the U.S. Food and Drug Administration granted Orphan Drug designation to TTX101 for malignant glioma. Orphan Drug designation is granted to therapies that intend to address the needs of rare conditions affecting fewer than 200,000 people nationwide. 

What is TTX101? TTX101 is a novel hydrogel-based localized therapy that provides a significant increase in drug load. Applied following tumor excision, the therapy provides active treatment as the wound heals and before the patient undergoes radiotherapy. It means to address issues with the calcium channel; when calcium channel expression is higher, it correlates to poorer survival in people living with glioblastoma. 

Researchers are currently planning to launch a Phase 1/2a clinical study for people with recurrent glioblastoma. TTX101 has so far shown promise in preclinical studies. Administered intracranially, TTX101 was able to reduce tumor size in murine models. It was also safe and well-tolerated. 

The Key Facts: Glioblastoma

Also known as: Stage IV astrocytoma

Glioblastoma is a rare, aggressive brain cancer. Symptoms often appear rapidly, and the cancer is often diagnosed in later stages despite no previous signs of brain cancer. Doctors do not know what causes glioblastoma, though they have identified certain risk factors: prior radiation therapy and certain genetic conditions. While glioblastoma often spreads throughout the brain, it does not usually spread to other areas of the body. Treatment can be ineffective, in part due to the fact that glioblastoma can create its own blood supply, thus sustaining its growth. Symptoms of this cancer may include changes in mood, behavior, or personality (such as anxiety or impulsivity), muscle weakness or paralysis, fatigue and lethargy, recurrent headaches, nausea and vomiting, seizures, problems with balance and coordination, vision abnormalities, and problems with thinking or speaking.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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