In the United States, the Food and Drug Administration (FDA) grants Orphan Drug designation to drugs or biologics which intend to treat, diagnose, or prevent rare conditions. “Rare” conditions are those affecting fewer than 200,000 people nationally. To incentive drug development within this sphere, this designation comes alongside benefits such as fee waivers, increased regulatory assistance, tax credits, and seven years of market exclusivity if the drug becomes approved. According to Cancer Network, the FDA recently granted this designation to QN-302, a therapy for pancreatic cancer. 

Developed by Qualigen Therapeutics (“Qualigen”), QN-302 uses the G4 selective transcription inhibitor platform that the company licensed from University College London. As described by Qualigen: 

The licensed technology comprises lead compound QN-302 and back-up compounds that target regulatory regions of cancer genes that down-regulate gene expression in multiple cancer pathways. Developed by Dr. Stephen Neidle and his group at UCL, the G4 binding concept is derived from 30+ years in nucleic acid research, including that of G4s, which are higher order DNA and RNA structures formed by sequences containing guanine-rich repeats.

Since G4s are highly represented in tumor cells, binding could help reduce cancer proliferation. Preclinical in vitro studies found that QN-302 was well-tolerated with no toxicological reactions. The treatment did not harm kidney or heart tissue. 

Moving forward, Qualigen hopes to submit an Investigational New Drug (IND) application to the FDA, which would allow them to launch a clinical study. 

A Brief Education: Pancreatic Cancer

Pancreatic cancer forms in the cells of the pancreas. This organ, which sits behind the lower part of the stomach, releases enzymes and hormones; these aid in digestion and blood sugar management, respectively. The majority of pancreatic cancer is exocrine, occuring in cells that line the pancreatic ducts. Pancreatic neuroendocrine tumors, which form in hormone-producing cells, are rarer. Risk factors include chronic and hereditary pancreatitis, smoking, older age, long-standing diabetes, obesity, diets high in red and processed meats, and being of African-American or Ashkenazi Jewish descent. 

Unfortunately, pancreatic cancer is asymptomatic in earlier stages. More symptoms appear as the cancer progresses. Symptoms can, but do not always, include:

• Newly onset or worsening diabetes 
• Dark-colored urine and pale stools
• Unintentional weight loss 
• Jaundice (yellowing of the skin, eyes, and mucous membranes) 
• Appetite loss
• Abdominal pain which radiates to the back
• Fatigue
• Itchy skin
• Blood clots

Treatment options include chemotherapy, radiation therapy, surgery, targeted therapy, and supportive care.