Collaboration Between U of M, New York Biotech Firm Leads to Childhood Cancer Drug Trial

The mixed public-private effort with Vaccinex is aiming to bring about a treatment for intractable osteosarcoma.

Collaboration Between U of M, New York Biotech Firm Leads to Childhood Cancer Drug Trial
A public-private collaboration between University of Minnesota researchers and a clinical-stage biotech company making an experimental anti-cancer drug has resulted in a new push for an immunotherapeutic treatment against childhood bone cancer.
Vaccinex Inc., a privately-held drug discovery firm based in Rochester, New York, for the past several years has been working with Branden Moriarity, an assistant professor of pediatric hematology and researcher at the Masonic Cancer Center, University of Minnesota, on preclinical studies of how its drug candidate known as VX15 could work against osteosarcoma, the most common malignant bone tumor found in children and adolescents.
That work has now advanced to the opening in May of a Phase I clinical trial to be carried out in Minneapolis and other sites around the country headed by Dr. Emily Greengard, also an assistant professor at the Masonic Center and an expert in childhood cancers. The trial is the first ever to study the possibility of using anti-cancer immunotherapy drugs which stimulate the body’s immune system against osteosarcoma, a condition which mainly affects otherwise healthy children and teens.
The disease is an especially cruel and insidious form of cancer associated with skeletal growth. In the vast majority of cases, it has already metastasized by the time of diagnosis, usually in the lungs, resulting in intractable pain. With no known ways to detect it early, treatments for osteosarcoma are extremely limited: Chemotherapy and surgical resection are currently the standard therapies.
The plight of juvenile osteosarcoma victims was spotlighted recently with the case of Minnesota teenager Zach Sobiech, whose song “Clouds” became an internet sensation shortly before he died in 2013 at age 18. 
The collaboration with Vaccinex first took shape as a result of Moriarity’s work using a U of M-developed genetic tool to explore whether gene targets for onco-immunological drugs could be pinpointed against osteosarcoma. His lab was partially funded as the result of donations received through the Children's Cancer Research Fund, which administers Zach Sobiech's foundation.
In 2015, Moriarity and several colleagues published the results of a study in which they succeeded in identifying some possible genetic “drivers” of osteosarcoma, with one of the most likely candidates being a signaling protein known as SEMA4D. It was found to be expressed at high levels in over half of all human osteosarcomas; thus, it was theorized that slowing or inhibiting the expression of SEMA4D could help stop the growth of osteosarcoma.
Moriarty told TCB that once those results were published, it wasn’t long before he heard from Vaccinex about its drug candidate VX15.
“I had already known about their antibody targeting SEMA4D in clinical trials for other kinds of solid tumors, so I specifically mentioned it in my paper,” he said. “They saw that reference, contacted us, and offered to give us free supplies of the drug for use in a trial against osteosarcoma.
“It’s come after a long period of research in which we used a technology called the Sleeping Beauty transposon-based forward genetic screen, which was first developed at U by Perry Hackett and refined by my mentor, David Largaespada,” he said. “The upcoming trial will be focused on patients who have exhausted all other treatment options, so any kind of result showing an improvement in outcomes would be just huge for the children who suffer from this cancer.”
Vaccinex assisted the U researchers in procuring an “investigational new drug,” or IND, designation from the U.S. Food and Drug Administration needed to proceed with the upcoming trial, which is being administrated by the Children’s Oconology Group and supported by the National Cancer Institute. It will study the side effects and best dose of VX15 to see how well it works in treating 75 younger patients whose tumors have recurred after chemotherapy treatment, or who do not respond to chemo or other treatments.
Once the safe dosages are determined (after a three-year period), it is to be followed by a larger Phase II trial to determine whether or not VX15 can be effective against the disease itself.
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