Promising targeted therapy offers new hope to certain patients with advanced pancreatic cancer

A promising new targeted cancer therapy will soon be available to certain patients with advanced pancreatic cancer – from the comfort of their home. The treatment will be available through a first-of-its-kind, entirely telehealth-based cancer clinical trial at The Ohio State University Comprehensive Cancer Center – Arthur G. James Cancer Hospital and Richard J. Solove Research Institute (OSUCCC – James).

Traveling for specialized cancer treatment is often cost-prohibitive for patients experiencing cancer – particularly for rare but aggressive types like pancreatic cancer, where clinical trials can represent the most up-to-date and targeted treatment for advanced disease."

Sameek Roychowdhury, MD, PhD, a medical oncologist with the OSUCCC – James and principal investigator of the new study

Roychowdhury explains that so-called "smart drugs" open a new world of treatment options for patients with cancer. These targeted therapies are "smart" because they target only the genetic mutations contributing to cancer cell growth, delivering genomic-guided, precise treatment for each person's disease characteristics. In this case, the genetic mutation is in the fibroblast growth factor receptors, or FGFR, which are present in roughly 1% of pancreatic cancer patients.

Pancreatic cancer is a rare but often aggressive form of cancer that is diagnosed in about 64,000 people each year. The disease is slightly more common in men and often diagnosed in later, less treatable stages because its symptoms usually occur after it has spread to other parts of the body. While surgery can be curative in the earliest stages of the disease, it is rarely detected before it has spread, and approved treatment options are limited. This, said Roychowdhury, is why expanding access to targeted drug therapy clinical trials is so critical.

"There may be hundreds of gene mutations in someone's cancer. Discovering which ones are driving how the cancer behaves and treating the mutation with novel therapies is the basis of 'smart drug' – or precision cancer medicine – research," said Roychowdhury, who is also a physician scientist with the OSUCCC – James Translational Therapeutics Program. "One of the major barriers for precision oncology clinical trials is the rarity of some gene mutations -- which limits pharmaceutical company interest and feasibility."

The rise of telemedicine represents a silver lining from the COVID19 that Roychowdhury says will help overcome barriers to access for cancer clinical trials.

This new telehealth study will give patients from across the United States access to oral targeted drug therapies without having to travel to a different city. Study participants will get follow-up care with Roychowdhury via telehealth, delivered in partnership with the patient's local oncologist.

"This is a game changer for cancer clinical trials, and more importantly, patients," said Roychowdhury, who has 10 years of experience with FGFR smart drugs.

The preliminary research on FGFR that helped launch this treatment concept was supported by Gateway for Cancer Research, a nonprofit organization dedicated to exclusively funding early phase clinical trials for all types of cancer. An early adopter and innovator in decentralized oncology research, Gateway hopes to increase awareness of telemedicine-based clinical trials so patients know they may have options despite proximity to research sites.

"An informed patient is cancer's greatest enemy," said Richard J Stephenson, founder and chairman of Gateway. "It was an informed patient who was desperately seeking treatment that put the wheels in motion and paved the way for this new trial."

The new clinical trial will include partnerships with Incyte Pharmaceuticals, the Pancreatic Cancer Action Network, Foundation Medicine Inc., and Caris Life Sciences.

"By taking clinical trial treatment options directly to the patient and partnering with community oncologists across the United States, we greatly expand access to patients who need these therapies – and we are more capable of making meaningful discoveries by recruiting larger groups of patients," Roychowdhury said.

In addition to the clinical trial, Dr. Roychowdhury's team has created a registry for patients to join and support research on rare types of pancreas cancer.

This trial is expected to begin accruing patients in late 2023. To learn more about participating in the study or the registry, email [email protected]. To learn more about gastrointestinal treatment and research at the OSUCCC – James, visit cancer.osu.edu.