For 78-year-old Eileen Pratt, seeing a small lump appear above her eyebrow four years ago did not seem like a big deal. A mother of three grown children, and grandmother to seven grandchildren, she was basically in good health. But her primary care doctor told her she needed to have the lump removed and checked just to be safe.

“When the lump biopsy showed cancer, I was surprised,” said Pratt. “But it didn’t sound good because it was an aggressive cancer.” A scan showed similar tumors under her arms and five others in her head. Pratt was diagnosed with a rare, aggressive blood cancer called Peripheral T-Cell Lymphoma or PTCL. This type of lymphoma develops from mature-stage white blood cells or T-cells, and results when T-cells grow abnormally in the lymphoid tissues outside of the bone marrow, such as the lymph nodes, spleen, gastrointestinal tract, and skin.

Pratt endured two difficult years of four different treatments regimens to treat the PTCL, including chemotherapy and a stem cell transplant, but the cancer kept coming back. This is when Pratt was identified as a candidate for a clinical trial for a drug called tipifarnib at Yale Cancer Center and Smilow Cancer Hospital.

“Eileen was out of treatment options. Her cancer was progressing rapidly, so we enrolled her in the tipifarnib trial,” said Pratt’s hematologist, Francine M. Foss, MD, Professor Medicine (Hematology) and Dermatology at Yale Cancer Center and Smilow Cancer Hospital. Foss is a global expert in T-cell lymphoma and was leading the tipifarnib research, and a national phase 2 study, at Yale. “The results were very impressive right away. We treated a number of patients on this study, like Eileen, with tipifarnib and observed similar, very dramatic, positive clinical responses.”

Tipifarnib, also called Zarnestra, belongs to the family of drugs called farnesyltransferase inhibitors. It’s an older drug developed nearly twenty years ago for use in the treatment of patients with acute myeloid leukemia (AML). Importantly for patients with cancer, tipifarnib is an oral drug.

“The tipifarnib is also a game-changer concerning treatment and quality of life,” said Pratt. “Instead of grueling weekly chemotherapy, I take tipifarnib in pill form at home and I only need to come in once a month for check-ups, blood tests and to pick up the medication. That means no more long drives from my home in upstate Connecticut to the hospital in New Haven every week.” Pratt said she experiences few side effects (mainly fatigue) with tipifarnib, and her hair has even grown back since chemotherapy.

“Another important aspect of the tipifarnib trial was that we found that responders could be identified based on expression of certain molecular biomarkers,” said Foss. “In the era of precision medicine, tipifarnib is emerging as a novel targeted agent for this disease.” Foss added that it is due to the willingness of Pratt and many other patients to enter clinical trials that advancements are made, and new therapies become available for cancer patients. Findings from the tipifarnib trial were so promising, they were highlighted at the 2021 American Society of Hematology Annual Meeting & Exposition in Atlanta, Georgia.

Pratt has the distinction of being on tipifarnib longer than any other patient and she will be able to continue treatment as long as it remains effective. “I feel very fortunate to have found Dr. Foss and her team when I did. I have kids and grandkids to love!”