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New cancer treatment in clinical trial thanks to Zach Sobiech: Teen’s legacy lives on in cancer research

Pediatric oncologists Brenda Weigel, co-director of the sarcoma program for the Masonic Cancer Center at the University of Minnesota, and Branden Moriarity, an assistant professor in the Department of Pediatrics at the Masonic Cancer Center, photographed in Minneapolis on Jan. 30, 2018, are researching osteosarcoma using money from the Zach Sobiech osteosarcoma fund. (Mary Divine / St. Paul Pioneer Press)1 / 2
17-year-old Zach Sobiech, left, plays guitar as his friend Samantha "Sammy" Brown,foreground, sings a song they wrote earlier that night called 'Star Hopping' in Lakeland, Minn., on December 3, 2012. They were recording the song at the studio called School of Music & Mayhem run by Joe Schertz, not pictured. (Ben Garvin / St. Paul Pioneer Press)2 / 2

MINNEAPOLIS — Pediatric oncologist Brenda Weigel has always had bleak news for patients diagnosed with osteosarcoma.

Treatments for the rare form of bone cancer are generally ineffective.

"We had essentially the same chemotherapy drugs for 30 years to offer, and that was it," said Weigel, co-director of the sarcoma program for the Masonic Cancer Center at the University of Minnesota.

But thanks to research paid for by the Zach Sobiech Osteosarcoma Fund, Weigel can begin to offer hope.

"I can now say, 'We actually have things that are in development that may someday change the course of this disease,' " Weigel said. "Five years ago, I couldn't say that. Ten years ago, I certainly couldn't say that. Now we have at least the promise that there may be things on the horizon, and that's huge."

Sobiech is the Lakeland teenager whose song "Clouds" became an internet sensation shortly before he died of cancer in 2013 at age 18. At his request, proceeds from the song — more than $1.5 million — have been donated to the fund, which supports osteosarcoma research at the University of Minnesota.

As a result, researchers developed a promising new therapy for osteosarcoma patients that last month went to clinical trial at the Masonic Cancer Center and at 30 other hospitals around the country.

Here's how it happened: The osteosarcoma team, which includes pediatric oncologists, epidemiologists, surgeons and experts in veterinary medicine, in 2014 discovered a causative gene of osteosarcoma for the first time. It is called semaphorin 4D (SEMA4D).

After their findings were published in the journal Nature Genetics in 2015, officials at the biotechnology company Vaccinex, based in Rochester, N.Y., contacted lead author Branden Moriarity to tell him that an experimental antibody they had developed could block the activity of SEMA4D.

"It's called VX15," said Moriarity, an assistant professor in the hematology/oncology division of the university's pediatrics department. "It turns out it does a bunch of stuff. It activates the immune system, and it can cause the immune system to kill the cancer cells."

Experiments involving VX15 and osteosarcoma have been "very successful" during laboratory experiments, Moriarity said. The antibody cured mice of the disease, "and in a dish with human cells, we were able to reduce their ability to migrate, or metastasize, and reduce their ability to grow outside of the tumor."

"So this drug will actually do two things: It will activate the immune system, and it will stop the tumor cells from growing, which could be a one-two punch to eliminate the cancer," he said.

About 600 children and adolescents are diagnosed with osteosarcoma each year in the U.S. Osteosarcoma tumors develop in bones during a growth spurt; boys are at a 20 percent greater risk of getting them.

If the tumor has spread to other tissues, the chance of survival past five years is less than 30 percent.

'Clouds' a YouTube, iTunes hit

Sobiech was diagnosed with osteosarcoma in the fall of 2009. Three years later, he wrote and performed "Clouds" as a farewell to family and friends. He died in May 2013.

A video of "Clouds" has been viewed more than 13.5 million times on YouTube, and the song reached No. 1 on the iTunes singles chart.

Beginning the year of Sobiech's death, about 5,000 people have gathered annually at the Mall of America at Christmastime to sing "Clouds" and raise money for the osteosarcoma fund.

Discoveries coming fast

The discovery of SEMA4D and the subsequent clinical trial happened in just five years, which is "pretty extraordinary" when it comes to translating novel therapies to the clinic, said David Largaespada, a mouse geneticist and professor in the U's department of genetics and cell biology.

"We hope through the basic work we did in mice, we're going to have a pipeline of potential therapies coming along," Largaespada said. "We have to be focused on this for the long haul. Five years is a long time, but if we don't keep going and keep working another five years and another five years, we won't make the progress needed. It is made more difficult because it is a rare disease."

Patients receiving immune-based treatments like VX15 don't experience side effects such as nausea, vomiting, hair loss and fatigue, Weigel said

"The drugs that we give, especially to treat osteosarcoma, have a lot of long- and short-term complications," she said. "We say, it's 'cure with a cost.' Only time will tell what the long-term side effect is of turning on the immune system, but what we know so far is these are generally very well-tolerated drugs. Most children and young adults on these types of therapies can continue with normal daily activities."

In addition to the clinical trial, researchers are working on a web-based registry called the Biology of Osteosarcoma Registry and Biobank (BOOST). Logan Spector, a pediatric cancer epidemiologist and a professor in the university's pediatrics department, is leading the effort.

BOOST participants will fill out an online survey, then receive a DNA-collection kit through the mail. Patients and their first-degree relatives — parents, siblings, children — are invited to enroll. Spector said researchers will use the samples — 200 have been collected to date — to understand why osteosarcoma occurs and how genetics influence response to treatment.

"When it comes to genetic research, there is power in numbers," Spector said. "The information could help us identify who's at risk, possibly monitor them for early signs of tumors and also just answer the question that a lot of patients and parents have, which is 'Why did this happen to me?' or 'Why did this happen to my child?' "

The research team last year applied for a $792,000 grant from the American Cancer Society and received the highest score possible, Moriarity said. "Unfortunately, due to budgetary constraints, the ACS is only able to fund a subset of outstanding grants."

The grant request is now on a list of "pay-if" grants, which depend on individual donors who want to support research that would not otherwise be funded. ACS is actively looking for a donor, he said.

"This deserves to be funded," Moriarity said in a phone interview. "Right now, 300 children a year die because of osteosarcoma. With new therapies, we could eliminate death from osteosarcoma. If this comes through, this will have vindicated Zach's goal to cure (the disease). What better way to honor his legacy?"

Zach's mom: Research keeps me connected

Laura Sobiech said her son lives on through the team's research.

"This is another way for me to stay connected to Zach," said Sobiech, the author of "Fly a Little Higher: How God Answered a Mom's Small Prayer in a Big Way." "It helps me feel like I'm not traveling away from him."

That the team has had a breakthrough is just "one of those really solid things that we can see happening," Sobiech said.

"It's not all theory," she said. "It's not just in the labs. It's out there now. It's touching children's lives, so I'm really excited about it. I've always been blown away that we get to be a part of this. We have that privilege."

Still, she admits the news is bittersweet.

"There is always that possibility that we will be part of discovering something that could have saved Zach's life," she said. "But I can't go there. Because on the flip side of that, if Zach hadn't died, his story would not have made the impact it did, and we wouldn't have the funds to find that.

"It's just kind of a vicious circle that I don't enter into," she said. "I choose to just look at the upside, which is that wasn't our story. We didn't have a cure for Zach at that time. We didn't miss anything because we tried everything that was available at that time, and now we move forward to give parents in the future more options than we had."