Mary Jo Pilat

Family tragedy fuels professor’s fight to find treatment for melanoma.

Mary Jo Pilat

Making research personal

Family tragedy fuels professor's fight to find treatment for melanoma

As a member of Stand Up to Cancer’s (SU2C)/Melanoma Research Alliance (MRA) Melanoma Dream Team, you might expect Mary Jo Pilat to drop names of celebrities she’s brushed shoulders with — after all, SU2C relies on the entertainment industry’s support to raise funds for cancer research.

But Pilat has never met those famous folks. Family members and friends who have lost their battles with cancer and melanoma patients fuel her focus on fighting the disease from a different arena — as a clinical researcher.

Pilat, a clinical assistant professor in EACPHS’s physician assistant (PA) studies program, is participating in “Using Molecularly-Guided Therapy for Patients with BRAF wild-type Metastatic Melanoma.” Led by co-principal investigators Patricia LoRusso, associate director of innovative medicine at the Yale Cancer Center, and Jeffrey Trent, president and research director of the Translational Genomics Research Institute (TGen), the six-module, $6 million project is supported by SU2C, MRA and the American Association for Cancer Research with participation from more than 20 research universities and institutions and several pharmaceutical companies. The ambitious, innovative clinical trial is evaluating cutting-edge melanoma treatments.

“Unlike chemotherapy, targeted treatments attack cancer cells by inhibiting particular growth pathways,” said Pilat. “But cancer is smart. If it grows using one pathway and we block it, it moves to a different one. We have to figure out how to stop it.”

According to the American Cancer Society, about half of all melanomas have mutations in the BRAF gene that produce an altered protein that accelerates tumor growth. However, these mutations also make patients more responsive to drugs that target the protein.

“Patients in the BRAF mutant population who use targeted therapy often see their tumors shrink or disappear,” said Pilat. “The team wondered about the people without the mutation. Do they have mutations in other genes that will help us identify different targets? If we conduct a genetic analysis on patients’ tumors, can we identify a drug to target their genetic mutation and create a better response than traditional treatment?”

The Melanoma Dream Team — which includes more than 80 researchers from across the country — focuses on expanding treatment options for patients without the BRAF mutation. Clinicians collect tumor samples from patients with BRAF wild-type melanoma and send them to TGen for extensive genomic analysis. Based on the genomic targeted therapy data, the group decides which targeted agent from their library of investigational and non-investigational drugs would be most effective. An outside medical overseer reviews decisions and shares recommendations. The study involves two-to-one randomization, which means twice as many patients receive the molecularly-guided therapy as the traditional chemotherapy. The goal is to determine which treatment produces the best response rate. As clinical project manager, Pilat presents the clinical trial to various clinical sites, synthesizes and sends information to the medical overseer for confirmation, randomizes patients, serves as a general clinical consultant and cares for patients.

The team’s pilot study results were published in the August issue of Molecular Cancer Therapeutics. Researchers hope to recruit at least 96 patients for the clinical trial over the next 18 months.

Pilat says the study is unique because experimental drugs and targeted treatments from several pharmaceutical companies are being used in one clinical trial. The ability to discuss different molecular targets and access to the drugs that hit them will help researchers determine whether they can generate a personalized plan for each patient.

“In the future, it may not matter what kind of cancer you have — what will matter is the genomic profile,” she said. “Once we know the genomic profile, we may be able to create an individualized treatment plan for that cancer.”

Pilat took a nontraditional route to her current role. After earning a bachelor’s in chemistry at Wayne State, she began working in the School of Medicine’s biochemistry department, where she became a research assistant and later earned a PhD. She was exposed to clinical trials during a postdoctoral fellowship at the University of Michigan. Although she enjoyed research, Pilat wanted a more patient-oriented career. She applied to the PA studies program. After graduation, she was offered a job at the Karmanos Cancer Institute, where she worked with LoRusso as a research PA — a position that allowed her to merge her research and patient-centric interests.

“When I became a faculty member, Dr. LoRusso asked if I was interested in continuing to work on the project. I wanted to keep collaborating with her,” Pilat said. “Being part of this is fascinating and exciting, and I have a special interest in melanoma.”

Cancer has always been Pilat’s primary focus, but her passion for melanoma is driven in part by the death of a 26-year old cousin who died from the disease and left behind a three-month old son.

“Tricia died before we knew about the BRAF mutation and these targeted therapies. I often wonder whether this treatment would have made a difference if it had been available,” she said. “Cancer treatment has vastly improved since I started. The field is growing by leaps and bounds, but there is still a lot of work to do.”

Pilat believes SU2C’s approach to combating cancer could prove successful. According to the organization’s website, nearly 950 scientists are involved in SU2C-funded research projects, and about 5,200 people are participating in more than 150 clinical trials supported by the nonprofit.

“Stand Up to Cancer is a phenomenal organization. It recruits renowned researchers and requires them to collaborate on treatments and clinical trials for different types of cancer,” Pilat said. “Programs and studies like this that bring experts together to share ideas and solve problems is the way to go.”

Back to spotlights