In the United States, the FDA grants Orphan Drug designation to drugs or biologics intended to treat, prevent, or diagnose rare diseases or conditions. Do you know what makes a condition considered “rare?” To be rare, a disease may only affect fewer than 200,000 Americans. Drug developers whose products receive Orphan Drug designation also gain a variety of benefits and incentives, such as fee waivers, tax credits, increased regulatory assistance, and seven years of market exclusivity upon approval. According to a news release from biopharmaceutical company Immunicum AB, its therapy ilixadencel recently received Orphan Drug designation for the treatment of gastrointestinal stromal tumors (GISTs). In the past, ilixadencel has also received Fast Track designation for this indication, as well as Orphan Drug designation for the treatment of soft tissue sarcoma (STS). 

What is Ilixadencel?

According to Immunicum, ilixadencel is a cell-based immune primer which:

uses dendritic cells sourced from healthy human donors that are specifically activated to produce significant amounts of vigorous immune stimulatory factors. By administration through intratumoral injection, these cells induce a local inflammatory reaction, leading to destruction of tumor cells, and recruitment and activation of a patient’s own dendritic cells into the tumor environment.

Following ilixadencel treatment, the patient’s dendritic cells engulf tumor cells and use that to create an anti-tumor effect in the body. Currently, Immunicum is evaluating ilixadencel for a variety of cancers and solid tumors, including liver cancer. So far, studies done have shown the treatment to be relatively safe and well-tolerated. Moving forward, Immunicum will continue to evaluate ilixadencel for GISTs in a Phase 2 clinical trial. 

Gastrointestinal Stromal Tumors (GISTs): An Overview

So what are gastrointestinal stromal tumors (GISTs)? As the name suggests, these tumors form in the gastrointestinal tract – particularly in the stomach or small intestine. However, in rarer cases, tumors may form in the esophagus, rectum, or colon. GISTs exist under the umbrella of soft tissue sarcoma (STS), a form of rare cancer. In many cases, spontaneous gene mutations cause GIST formation by causing interstitial cells of Cajal (ICCs) to grow at an over-accelerated rate. These tumors are most common in those between the ages of 40-70. Symptoms of GISTs can include:

• Abdominal pain and swelling
• Appetite loss
• Anemia (low red blood cell count)
• Nausea and vomiting (which may produce bloody vomit)
• Gastrointestinal bleeding and bloody stool
• Fatigue and general malaise
• An abdominal mass
• Unintended weight loss
• Difficulty swallowing