The timing could not have been worse.
Ellen Coopersmith learned she had a rare, advanced ovarian cancer in 2016, soon after the death of her husband. Her three children were still in high school.
“I couldn’t die — I knew I had to beat this,” Ellen says.
A kindergarten teacher living in suburban Philadelphia, she came to Memorial Sloan Kettering Cancer Center (MSK) after surgery at a local hospital. The MSK team kept the cancer at bay over the next decade with two more surgeries. She also had chemotherapy, but it didn’t work.
So when Ellen’s doctor, gynecological medical oncologist Rachel Grisham, MD, suggested she consider joining a clinical trial, she jumped at the opportunity. Dr. Grisham was investigating a new combination of targeted therapy drugs. In earlier phase 2 testing, 44% of the patients responded to this new approach.
“I was very excited when I found out I could get into the phase 3 trial,” Ellen says. She started in January 2025.
The good news came quickly. Within four months, her cancer had shrunk by 70%.
By participating in MSK research, Ellen got early access to a treatment that has dramatically decreased her tumors and changed her outlook on life.
“When you’re first diagnosed with cancer, you just stop and think, ‘OK, how many years do I have left, and what do I need to get done in those years?’ ” Ellen says. “I never thought my life would be extended like this. I could not be any luckier. Now it’s exciting to think, maybe I’ll get to meet my grandkids.”
Treating Low-Grade Serous Ovarian Cancer
Ellen was diagnosed with a low-grade serous ovarian cancer (LGSOC). LGSOC is rare, accounting for less than 10% of all cases of ovarian cancer, and is more common in younger women — especially those in their 20s or 30s.
As the words low grade suggest, LGSOC is considered less aggressive than other types of ovarian cancer, mainly because it grows more slowly.
Patients diagnosed with LGSOC tend to live longer than those with other types of ovarian cancer, even when the cancer has spread. However, as was true in Ellen’s case, it is also less likely to respond to chemotherapy. In fact, chemotherapy is effective in fewer than 1 in 20 patients with LGSOC.
The new low-grade serous ovarian cancer treatment that Ellen is receiving is a combination of two targeted drugs — avutometinib and defactinib. Based on the impressive phase 2 clinical trial results, this new therapy recently received accelerated approval from the U.S. Food and Drug Administration (FDA) for a subset of patients. Avutometinib plus defactinib now called AvmapkiTM FakzynjaTM Co-Pack and is the first treatment ever approved specifically for LGSOC.
“We expect Avmapki Fakzynja Co-Pack therapy will become the new standard of care for many LGSOC patients,” Dr. Grisham says.
How Learning From ‘Exceptional Responders’ Leads to New Treatments
The research that brought this treatment to life started more than a decade ago at MSK.
In the early 2010s, targeted therapy drugs directed at cancer-causing mutations were new and not fully understood. Curiously, in some clinical trials where the drugs failed for most patients, they worked for a small number — sometimes only one or two people out of dozens. These patients would have a complete response. Their cancer would be totally eliminated, and it would not come back.
These patients were called “exceptional responders.”
“We set out to understand how the biology of the tumors from those exceptional responders was different than other patients,” says MSK physician-scientist David Solit, MD. “Why did they survive? What could we learn that would help us use targeted drugs more effectively?”
Discovering MEK1 and FAK Proteins as Two Key LGSOC Treatment Targets
One of the first exceptional responders studied by Dr. Solit’s lab had low-grade serous ovarian cancer. Researchers, including Dr. Grisham, analyzed this patient’s tumor DNA and discovered it carried a defect in a protein called MEK1. In 2015, they published a paper in the Journal of Clinical Oncology that showed how defects in the MEK1 protein drive the growth of LGSOC.
Other labs built on their discovery and found that targeting another protein called FAK at the same time was more effective than targeting MEK1 alone. This combination approach is what has been approved by the FDA.
“This is a great example of translational research,” Dr. Solit says. “Not only are lab discoveries driving clinical care, but we’re taking what we learn from patients back to the lab for further investigation.”
And the investigation continues. One goal of the phase 3 trial in which Ellen is enrolled is to learn more about which patients are most likely to benefit from the treatment.
“We’re continuing to collect information about other genes and proteins that may be involved in this cancer,” says Dr. Grisham, who is the global leader of the ongoing phase 3 study.
Joining an MSK Clinical Trial Gave Ellen a New Outlook and Hope for the Future
Ellen says she never worried about getting an experimental therapy that wasn’t yet FDA-approved. She had complete trust in her doctors. “Dr. Grisham really knows me,” she says. “She understands the genetics of my disease, and she knows what my body needs in order to fight this.”
Ellen also credits her surgeon, Yukio Sonoda, MD, for his compassionate care over the years.
Recently retired from her teaching job to focus on her health. Ellen, now 62, is enjoying a new chapter of life. She has more free time for long hikes, comedy clubs, and dinner with friends.
“What I love the most about my doctors at MSK,” says Ellen, “is that they care about not only the quantity of life, but the quality of life that you have.”
Dr. Solit holds the Geoffrey Beene Chair.