Aly Wolff died of neuroendocrine cancer on April 22, 2013, however her courageous battle continues. Today, three years later, a new clinical trial at the University of Wisconsin Carbone Cancer Center has been approved and holds great promise in offering a new line of treatment for those with neuroendocrine tumors.

Since her diagnosis at the young age of 19, Aly’s Wolff’s dream was to find a cure for cancer. This dream inspired the formation of The Aly Wolff Foundation, Inc., a 501(c)(3) that funds UW Carbone cancer research with proceeds raised through their annual 5K run/walk: Aly’s Honky Tonk Hustle.

To date, The Aly Wolff Foundation has raised $360,700 for cancer research and human-hair wigs for patients in need. This year, the foundation hopes to bring that total to over $500,000 since its inception.

"We could not do this trial without the Wolffs and Aly's Honky Tonk Hustle," said Dr. Noelle LoConte, associate professor of medicine at the Carbone Cancer Center who specializes in gastrointestinal cancers, including neuroendocrine tumors. "We are so grateful."

The trial, which is approved but not yet open to enrollment, will treat neuroendocrine tumor (NET) patients with a new combination of two anti-cancer drugs. Both drugs have been found to be well-tolerated on their own, and the combination approach may result in another line of treatment not previously available to NET patients. The trial will focus specifically on pancreatic neuroendocrine tumors (pNET); if successful, future trials may be available for any NET patient.

For patients with metastatic pancreatic neuroendocrine tumors, current therapies are mostly temporizing measures, and most patients still succumb to their disease" said Dr. Nataliya Uboha, an assistant professor of medicine at the Carbone Cancer Center who authored the trial. "Patients need systemic therapies, and that is why we are doing this trial."

NETs of all tissue origins are relatively rare, though their incidence is on the rise. Uboha said that pNET patients whose disease has metastasized from its initial site typically begin treatment with a relatively benign course of drugs to manage their disease, but that those drugs will not work indefinitely. The next steps depend on how much disease is present and how aggressive it is, and often include targeted drug.

Aly Wolff

"Those drugs don't shrink tumors, they just keep them stable for some time," Uboha said. "If someone comes in with pain and symptoms from their disease, you want something that will shrink the tumor, so you need to put them on chemotherapy."

While chemotherapies were approved for pNET in the past, the drugs had such high toxicity profiles that their use is now limited. Retrospective studies of a combination of two drugs, temozolomide and capecitabine, have shown a response in up to 70 percent of patients and have led the National Comprehensive Cancer Network to recommend this treatment. A clinical trial is underway now to more closely study the effect of these drugs.

This new trial at UW Carbone will be looking at the combination of temozolomide and a newer drug, TAS-102, that is related to capecitabine but that has a different mechanism of action.

"TAS-102 was approved for colon cancer patients last fall and many of those patients who no longer respond to capecitabine show a response to TAS-102," Uboha said. "If this trial is successful, then we are one step closer to adding another line of therapy in treating neuroendocrine tumors."

The phase 1B trial will initially enroll patients with low or intermediate grade NET of any tissue origin to determine the tolerable dose. Then, it will enroll pNET patients from across Wisconsin and, if more patients are needed, may include partner sites through the Big Ten Cancer Research Consortium. Patients will receive the chemotherapy for one year or until the disease progresses, whichever comes first.

"This is an ambitious study, and you are helping to fund areas such as patient reimbursement for travel and lodging, which will dramatically help with accrual to the study," LoConte said about participants of Aly’s Honky Tonk Hustle. "You will also be helping to fund some of the correlate studies, meaning we will try to figure out if there are biomarkers associated with the patients who benefitted from the study."

Aly’s parents, Russ and Sheila Wolff, said they could not be more proud of what Aly has created. “This development is exactly what Aly had in mind, and it is a step toward her ultimate goal of finding a cure for this terrible disease,” they added.

The trial is sponsored by Taiho Oncology, Inc. with support from the funds raised by The Aly Wolff Foundation, Inc. Please consider registering for Aly’s Honky Tonk Hustle, taking place on May 21 in McFarland, Wisconsin.