Recent Study Confirms Validity of the Therapeutic Target of an Experimental Brain Cancer Drug

According to a story from BioSpace, Oncotelic, Inc., recently announced the publication of a study in the Journal of Clinical Research in Pediatrics. This study appears to back up the therapeutic potential of the experimental product candidate OT101, which is being developed as a treatment for the rare and difficult to treat brain cancers diffuse intrinsic pontine gliome (DIPG) and glioblastoma. The drug is designed to inhibit the activity of TGF-beta 2, which has been linked to immune system suppression in these brain cancers.

About Diffuse Intrinsic Pontine Glioma

Diffuse intrinsic pontine glioma is a very rare brain tumor that is known for its poor response for treatment. It appears in the pons, the central area of the brain stem. Its locations makes the tumor inoperable via surgery. Symptoms of diffuse intrinsic pontine glioma include vision loss, difficulty swallowing, difficulty breathing, and problems with speech. Like other brain tumors, diffuse intrinsic pontine glioma is difficult to treat as many drugs cannot cross the blood-brain barrier. The tumor almost always relapses after treatment, and five year survival rate is less than one percent. To learn more about diffuse intrinsic pontine glioma, click here.

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. Symptoms of glioblastoma include personality changes, headaches, memory loss, seizures, vomiting, and nausea; patients may lose consciousness in late stages. 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.

An Effective Therapeutic Target

The results of this study are confirmation of earlier findings which revealed that TGF-beta 2 is expressed to an abnormally high degree in these brain cancers and may also play a significant role in curtailing the immune system response to these tumors. Therefore, suppressing its activity could serve as a useful immunotherapy. The research further validates the results of a phase 2 trial of OT101 in glioblastoma in which the drug was able to induce meaningful responses in patients with relapsed, refractory disease. 

OT101 has also earned Rare Pediatric Disease designation from the US Food and Drug Administration (FDA) as a treatment for diffuse intrinsic pontine glioma.


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