Biotechnology

‘Smart drug’ gives new hope to some patients with advanced pancreatic disease

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NOTE TO EDITORS: High-resolution B-rolls and images are available for download at

Credits: Ohio State University Comprehensive Cancer Center – Arthur G. James Cancer Hospital and Richard J. Solove Research Institute (OSUCCC – James)

NOTE TO EDITORS: High-resolution B-rolls and images are available for download at

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

“Travel for specialized cancer treatment is often a cost barrier for patients with cancer – especially for rare but aggressive types like pancreatic cancer, where clinical trials can represent the most advanced and targeted treatment for advanced disease,” said Sameek Roychowdhury, MD, PhDa medical oncologist with OSUCCC – James and principal investigator of the new study.

Roychowdhury explains that so-called “smart drugs” open up a whole new world of treatment options for cancer patients. These targeted therapies are “smart” because they only target genetic mutations that contribute to cancer cell growth, providing a genomically-guided, precise treatment for each person’s disease characteristics. In this case, the genetic mutation is in the fibroblast growth factor receptor, or FGFR, which is present in about 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 males and is often diagnosed in a late, less treatable stage because the symptoms usually appear after it has spread to other parts of the body. While surgery can cure it in the early stages of the disease, it is rarely detected before it has spread, and approved treatment options are limited. This, Roychowdhury said, is why expanding access to targeted drug therapy clinical trials is so important.

“There may be hundreds of gene mutations in a person’s cancer. Finding what drives how cancers behave and treating mutations with new therapies is the foundation of ‘smart drug’ research – or precision cancer treatment,” said Roychowdhury, who is also a physician scientist with OSUCCC – James Translation Therapy Program. “One of the major barriers to precision oncology clinical trials is the rarity of multiple gene mutations — which limits the interest and viability of pharmaceutical companies.”

The rise of telemedicine represents a silver lining to COVID19 which 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 targeted oral drug therapy without having to travel to another city. Study participants will receive 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 experience with the smart drug FGFR.

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

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

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

“By providing patients with direct clinical trial treatment options and partnering with community oncologists across the United States, we are greatly expanding access to patients who need these therapies – and we are better able to make meaningful discoveries by recruiting larger cohorts of patients,” Roychowdhury said.

Apart from clinical trials, Dr. Roychowdhury has created a list of patients to join and support research on a rare type of pancreatic cancer.

The trial is expected to start enrolling patients in late 2023. To learn more about participating in the study or enrollment, email (protected e-mail). To learn more about gastrointestinal medicine and the research at OSUCCC – James, visit cancer.osu.edu.

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