Cancer1
Pixabay

Diagnosis of pediatric cancer is one of the worst news a family can receive. Having a young family member with cancer affects the entire family's dynamics and causes significant anxiety and distress. As a child cannot fully attend to their own medical needs, their parents or guardians have to adapt their lives around the patient's treatment schedules.

Edison Oncology, a Biopharmaceutical company that develops and commercializes new therapies targeting the fight against cancer, is developing a medication that makes the treatment of several pediatric cancers less disruptive and less costly. These cancers include but are not limited to, neuroblastoma, rhabdomyosarcoma, Ewing sarcoma, hepatoblastoma, and medulloblastoma.

The medication, Orotecan®, is a novel oral formulation of irinotecan, an intravenous drug that has demonstrated efficacy in treating various pediatric and adult cancers. It is a topoisomerase-I inhibitor approved by the FDA for the treatment of colorectal cancer and is also used 'off label' in treating several types of cancer including gastric cancers, neuroendocrine and adrenal tumors, pancreatic cancer, soft tissue sarcomas, and bone cancer.

According to Jeffrey Bacha, CEO of Edison Oncology, pediatric patients undergoing irinotecan treatment need daily infusions for five to 10 days every two weeks for several months. This means the patient must be admitted to a hospital or infusion center for 30% to 50% of their time, and a parent or guardian must accompany them. This has a huge negative impact on the patient's quality of life and drives up healthcare costs.

Physicians have tried to administer irinotecan orally and have achieved the same effect as intravenous dosing, but the medication's poor taste has led to reduced patient compliance and limited widespread adoption of oral irinotecan regimens.

Edison Oncology developed Orotecan, seeking to replicate the benefits of irinotecan but with a taste that is more palatable to patients. It is currently in a Phase 1 and 2 clinical trial that aims to evaluate its safety, tolerability, and efficacy. Several centers across the US, including Duke Children's Hospital & Health Center, Cincinnati Children's Hospital, Children's National Hospital in Washington, DC, and UCSF, are participating in the trial.

During the first cycle of treatment, each patient receives four daily doses of Orotecan and one daily dose of irinotecan taken orally to allow for pharmacokinetic comparison. During all subsequent cycles, patients receive Orotecan once daily in five-day courses, administered every 21 days.

Patients were also asked to rate the palatability of irinotecan and Orotecan on a seven-point scale. In a preliminary analysis, the former scored a unanimous 1, or "really awful", while the latter scored an average of 4.2, signifying "OK to good".

Bacha says that the interim data from the clinical trial is "promising," with initial pharmacokinetic observations supporting equivalency between the Orotecan formulation and unformulated i.v. irinotecan is given orally for both the parent compound and active metabolites SN-38 and SN-38 glucuronide.

Additionally, the data suggests that the Orotecan oral formulation is palatable to patients and is suitable to be taken at home. This represents a potentially significant improvement in the quality of life of patients and their caregivers, as well as reduced cost of treatment compared to administering irinotecan intravenously. To date, the longest at-home treatment duration conducted is 13 cycles of once-per-day oral dosing.

"Our development of Orotecan is part of our business model that pursues accelerated approval for orphan cancer indications by using established human clinical safety and efficacy data," Bacha says. "The interim data from our ongoing clinical trial is very encouraging, and we're looking forward to advancing Orotecan to pivotal clinical trials and eventually seeking regulatory approval for use to treat pediatric and adult cancers. We believe this medication has immense potential to enhance the quality of life for patients while reducing treatment costs."