Have you ever heard of Orphan Drug designation? In the United States, this designation is granted by the U.S. Food and Drug Administration (FDA) to drugs or biologics intended to treat, diagnose, or prevent rare conditions. These are conditions affecting fewer than 200,000 Americans. Drug developers whose product receives this designation also earn fee waivers, tax credits, and seven years of market exclusivity upon approval. According to Targeted Oncology, DSP-0390, an investigational treatment for glioma, recently earned this designation.
DSP-0390: An Overview
Sumitomo Pharma describes DSP-0390 as:
an emopamil-binding protein (EBP) inhibitor…[which] causes an accumulation of EBP substrates zymostenol and zymosterol, which contributes to lethal autophagy in several types of cancer and efficient cellular cholesterol depletion via secondary LXR activation.
In short, DSP-0390, given orally, mediates cholesterol synthesis, attacks cancer cells, and reduces tumor growth. Sumitomo Pharma is currently evaluating DSP-0390 for glioma within a Phase 1 clinical trial. During the course of the dose-escalation study, researchers hope to evaluate:
- Safety
- Pharmacokinetic profile
- Pharmacodynamic profile
- Efficacy in reducing tumor/cancer activity
- Maximum tolerated dose
- Tolerability
- Objective response and response duration
About Glioma
Doctors and researchers are not entirely sure what causes glioma, a rare tumor which forms in the brain and spinal cord. However, risk factors include age (45-65), a family history of glioma, and radiation exposure. Although gliomas are considered to be rare, these are also one of the most common forms of primary brain cancer. This cancer forms in glial cells, which normally surround nerve cells to help protect nerve cell function. Subtypes of glioma include astrocytomas, glioblastomas, oligodendrogliomas, and ependymomas. Symptoms vary based on tumor subtype, size, and location. Potential symptoms can include:
- Nausea and vomiting
- Headache
- Confusion
- Speech difficulties
- Vision loss or other vision-related abnormalities
- Numbness and/or weakness
- Memory loss
- Cognitive decline
- Urinary incontinence
- Problems with balance and coordination
- Changes in mood, behavior, and personality
- Irritability
- Seizures (in someone who typically did not or had not had a history of seizures)
