It has historically been difficult to encourage the development of therapies for patients with rare diseases. To overcome this, the Orphan Drug Act was passed in 1983. This act helped to facilitate drug development within this sphere to better assist patients. Eventually, this led to the U.S. Food and Drug Organization (FDA) granting Orphan Drug designation to drugs or biologics intended to prevent, diagnose, or treat rare conditions (those affecting fewer than 200,000 Americans). Incentives given with this designation include fee waivers, tax credits, and seven years of market exclusivity upon drug approval. Reported by Cancer Network in December 2022, the FDA granted Orphan Drug designation to AUM302 for neuroblastoma.
Aum Biosciences describes AUM302 as:
a first-in-class oral kinase inhibitor rationally designed to uniquely combine pan-PIM kinase, pan-PI3K and mTOR inhibition in a single agent. This multi-targeting ability has been engineered into a single molecule that has the ability to inhibit multiple key intracellular pathways.
Through this, Aum Biosciences believes that AUM302 would stop the proliferation of cancer cells. Additionally, by inhibiting multiple pathways, this therapy offers the potential to overcome drug resistance.
So far, preclinical studies have found that AUM302 is safe and well-tolerated. In in vitro studies, AUM302 caused the death of tumor cells and cancer cells, as well as improved chemotherapy efficacy.
Neuroblastoma is a rare cancer which forms from immature nerve cells (neuroblasts) in nerve tissue. Normally, these cells become working nerve cells. However, in neuroblastoma, a genetic mutation in neuroblasts causes uncontrolled growth and tumor formation. While these tumors can grow in the abdomen, chest, neck, or near the spine, they most commonly form in the adrenal glands. Neuroblastoma most often affects infants and children aged five or younger. Symptoms can, but do not always, include:
- A lump or mass in the neck, chest, pelvis, or abdomen
- Bone pain
- Appetite loss
- Unintended weight loss
- Diarrhea or constipation
- Anemia (low red blood cell count)
- High blood pressure
- Bulging eyes
- Dark circles around the eyes
- Painless, bluish lumps under the skin (in infants)
- Abdominal distention (in infants)
- Difficulty breathing (in infants)
Treatment options depend on the cancer’s stage, the cells involved, the age of onset, and where the tumor is located. Potential treatment options include chemotherapy, radiation therapy, immunotherapy, MIBG therapy, surgery, and stem cell transplantation.