In the past, it was difficult to incentivize the development of treatments for rare diseases, given issues with both profitability and small patient populations. However, all people are deserving of adequate and effective therapeutic options. The Orphan Drug Act, and resulting Orphan Drug designation, were designed to stimulate drug development within this space. Orphan Drug designation is granted to drugs or biologics intended to treat, prevent, or diagnose rare conditions (affecting fewer than 200,000 Americans). Benefits include fee waivers, tax credits, and seven years of market exclusivity upon approval. According to Cancer Network, the FDA recently granted Orphan Drug designation to BEA-17, an investigational treatment for glioblastoma. 

Developed by precision oncology company Beactica Therapeutics, BEA-17 is a small molecule degrader that targets LSD1 and coREST. As Beactica Therapeutics explains:

Lysine-specific demethylase 1 (LSD1) is an epigenetic enzyme that regulates expression of large number of target genes and functions as a scaffolding protein that stabilizes transcriptional complexes. Furthermore, it is frequently overexpressed in cancer where it contributes to tumor growth [and may promote] anti-tumor immunity.

So far, preclinical in vivo research has shown that BEA-17 shows promise for treating several types of cancer. It is able to penetrate the blood-brain-barrier (BBB) and has good oral availability. Of course, more research is still needed to determine the impact of BEA-17 in humans. 

About Glioblastoma

Glioblastoma, though rare, is considered the most common form of primary brain tumor. It begins in star-shaped astrocyte cells, normally in the cerebrum. Glioblastoma is highly malignant and aggressive; the tumors are able to make their own blood supply, stimulating growth and spread throughout the body. As a result, the prognosis for a glioblastoma diagnosis is poor.

Males are more likely to develop glioblastomas than females. Additional risk factors include chemical exposure, being between 45-70 years old, previous radiation therapy or certain pre-existing genetic disorders such as neurofibromatosis.

Symptoms of glioblastoma can, but do not always, include: 

• Blurred or double vision
• Persistent headaches 
• Nausea and vomiting
• Changes in mood, behavior, or personality
• Drowsiness
• Memory loss 
• Problems with balance and coordination
• Weakness on one side of the body
• Difficulty thinking or speaking
• Seizures

Treatment options are often used in combination and include chemotherapy, electric-field therapy, radiation, and surgery.