BOLD-100 for Gastric Cancer Granted Orphan Drug Designation


In a news release from early May 2021, biopharmaceutical company Bold Therapeutics (“Bold”) shared that its treatment BOLD-100 received Orphan Drug designation for the treatment of patients with gastric (stomach) cancer.


So what is BOLD-100? According to Bold, BOLD-100 is:

an anticancer agent being developed in combination with standard of care therapies, to improve patient outcomes in difficult to treat cancers. Previously, BOLD-100 successfully completed a Phase 1 monotherapy dose escalation study in advanced cancer patients that showed BOLD-100 was generally safe and well-tolerated with a safety profile suggesting that it could be combined with other anti-cancer therapies.

By selectively inhibiting GRP78, altering the unfolded protein response (UPR), and inducing reactive oxygen species, BOLD-100 stops DNA damage from occurring. Through this, the ruthenium-based therapy also causes cancerous cell death. Outside of gastric cancer, Bold is also exploring BOLD-100 as a potential treatment for patients with pancreatic, colon, and bile duct cancer. In fact, BOLD-100 previously received Orphan Drug designation for pancreatic cancer.

The Orphan Drug designation is granted to drugs or biologics used to treat rare diseases or conditions. Within the United States, a rare disease (or rare cancer) is considered one which affects under 200,000 American citizens. Although gastric cancer is more common in other locations, such as Asia, only 27,000 patients are diagnosed within the United States each year. Since BOLD-100 received Orphan Drug status for gastric cancer, Bold Therapeutics now reaps a specific set of benefits. These include the chance to receive accelerated approval, discounts on fees, and up to 7 years of market exclusivity (pending approval).

Stomach Cancer

Also known as gastric cancer, stomach cancer occurs when cancerous cells form in the stomach lining. Altogether, there are three layers in the stomach wall: mucosal (inner), muscularis (middle), and serosal (outer). When stomach cancer forms, it initially begins in the mucosal layer and then metastasizes outward. There are multiple forms of stomach cancer, including:

  • Carcinoid tumors. Typically, carcinoid tumors are a rare form of gastric cancer. These tumors form in hormone-producing stomach cells.
  • Stromal tumors. Much like carcinoid tumors, stromal tumors are somewhat rare. These tumors are often treated differently than other stomach cancers. Altogether, stromal tumors form in supporting connective tissue.
  • Squamous cell carcinoma, leiomyosarcoma, and small cell carcinoma. These are extremely rare forms of stomach cancer.
  • Lymphoma. Sometimes, cancers like lymphoma, which normally form in immune system tissue, can form in the stomach wall.
  • Adenocarcinoma. When you think of gastric cancer, you are most likely thinking of adenocarcinoma. This common form makes up approximately 90-95% of stomach cancer diagnoses. Adenocarcinoma forms within the mucus-producing stomach cells.

Risk factors for stomach cancer include H. pylori infection, age (65+), being male, having a family history of stomach cancer, obesity, gastrointestinal reflux disease, and having a high-sodium but low fruit/vegetable diet.

In early stages of stomach cancer, patients may not display symptoms. However, as the cancer progresses, symptoms may include:

  • Bloody stool
  • Jaundice (yellowing of the skin and eyes)
  • Fatigue
  • Unintended weight loss
  • Chronic heartburn and indigestion
  • Nausea and vomiting
  • Feeling full and/or bloated, even after eating only small amounts
  • Difficulty swallowing
  • Stomach pain
  • Anemia (low red blood cell count)

Learn more about stomach cancer here.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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