New Hope

Pelotonia Dollars Support Innovative Clinical Trials

Clinical trials improve cancer care by demonstrating the safety and effectiveness of new treatments, examining treatment strategies and probing problems associated with therapies so refinements can be made. Here are summaries of two Pelotonia-supported clinical trials at the OSUCCC – James. To learn more about these and other trials, call The James Line at 800-293-5066 or visit

A Cancer-Killing Virus for Treating Solid Tumors in Children

OSUCCC – James researcher Timothy Cripe, MD, PhD, chief of the Division of Hematology and Oncology, and Blood and Marrow Transplantation at Nationwide Children’s Hospital, is leading a clinical trial he believes will help children with solid tumors that occur outside the brain.

The cancers include neuroblastoma, which occurs in children age 17 months on average and arises from immature nerve cells; sarcomas, or tumors of muscle and other soft tissue; and osteosarcoma, tumors that develop in bone.

“We’ve made progress in treating these types of cancers, but we’ve essentially reached the maximum benefit from surgery, chemotherapy and radiation,” Cripe says. “We need new types of therapies for these patients, particularly those with metastatic disease.”

Cripe’s phase I trial is testing the use of an oncolytic (cancer-killing) virus that selectively destroys cancer cells while doing little damage to healthy cells. The virus replicates in cancer cells and causes them to burst, killing those cells and spreading the virus to adjacent tumor cells.

In addition, studies in animals suggest that the bursting cells release cancer-cell specific molecules that stimulate the immune system to attack the tumor. The therapy therefore kills cancer cells both directly and indirectly through an immune response.

The virus is a modified herpes simplex virus type 1. A similar type of weakened virus has been approved by the U.S. Food and Drug Administration to treat melanoma in adults. “Our trial is designed to learn if this type of treatment is safe in children and young adults,” Cripe says.

The trial is open to patients aged 7 to 30 with any type of solid tumor located outside the brain and central nervous system. (The trial does not accept leukemia or lymphoma patients.)

The study has two parts. In part one, the virus is injected directly into the patients’ tumors. In part two, patients receive an infusion of the virus into a vein.

“The idea is that the bloodstream will carry the virus to metastatic tumors anywhere in the body and kill them,” Cripe says. “This is a new type of therapy for childhood solid tumors. We believe it will provide another option for treating these cancers and in the future should be able to be combined with standard treatments.

“Pelotonia is supporting the systemic testing of the virus, which is important for patients with metastatic disease,” he adds. “We are the only ones in the world using this virus systemically. We’re hoping it will open an entirely new era of cancer therapy that is more effective and safe.”

Seeking a Gentler Therapy for AML

Acute myeloid leukemia (AML) is the most common form of acute leukemia in the United States. Some 20,800 new cases were expected in 2015, along with nearly 10,500 deaths from the disease.

An early-stage clinical trial supported in part by Pelotonia funds and led by principal investigator Sumithira Vasu, MBBS, assistant professor in the Division of Hematology, is evaluating the feasibility and safety of an innovative immune therapy developed by Vasu and colleagues in the OSUCCC – James Leukemia Research Program.

The therapy is designed for older patients and people who cannot withstand the rigors of stem-cell transplantation, which is currently the most effective treatment for many cases of AML.

The need for new therapies is critical. Only about 40 percent of AML patients under age 65 achieve long-term remission. The survival rate is worse in patients 65 and older, who often develop subtypes of AML that make it harder to achieve remission and more likely to recur. Also, older patients often have other medical problems that leave them less able to tolerate chemotherapy. Hence, only about 10 percent of older AML patients are alive five years after diagnosis without an allogeneic (from a donor) transplantation.

The treatment being studied by Vasu and colleagues is carried out in four steps over 16 days. First, participants receive low doses of fludarabine, a drug that mildly suppresses the immune system. That is followed by: low doses of an anticancer drug called decitabine; an infusion of immune cells called natural killer (NK) cells that were obtained from a compatible donor; and several doses of a drug that stimulates NK-cell growth.

The researchers hypothesize that the fludarabine will help the patients’ immune system accept the NK cells, and the decitabine will make AML cells more susceptible to killing by NK cells. “Though additional clinical testing will be necessary, we believe this therapy could help more patients achieve remission,” Vasu says.

In addition to this trial, Pelotonia funds helped Vasu, William Blum, MD, and Natarajan Muthusamy, DVM, PhD, both professors in the Division of Hematology, study patient samples to help develop a novel combination regimen for patients over 60.

Using donated samples from patients who participated in clinical trials using decitabine led by Blum, the study showed that decitabine also modulates leukemia cells and makes them more susceptible to killing by NK cells and to antibodies that depend on NK cells for killing. These preclinical studies were published in the journal Blood and led to an international, multicenter trial evaluating the combination of decitabine and a novel antibody that relies on NK cells for killing AML cells.

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