Osteosarcoma FAQ

What is osteosarcoma? Osteosarcoma is a type of bone cancer that begins in the cells that form bones. Osteosarcoma is most often found in the long bones — more often the legs, but sometimes the arms — but it can start in any bone. In very rare instances, it occurs in soft tissue outside the bone. Grace had a very large high-grade tumor that was growing completely unchecked or unknown to her or her family. Grace had no pain, no swelling or prior symptoms before she fractured her shoulder in 2021. 

• How is it treated? Osteosarcoma is treated with aggressive surgery to remove any cancer through amputation to get "clean margins". Surgeons cut away cancer ridden bone and tissue during surgery and send samples to a pathology lab where specialists look at tissue slides to ensure all cancer is gone. Once the cancerous bone and tissues are removed, surgeons perform a limb salvage procedure and save whatever healthy bone and tissues remain. Many children then receive a prosthetic arm, leg or hand. Once they recover from surgery, infusion chemotherapy is started. Kids get the same medicines as adults because there have been no new chemo medicines to treat osteosarcoma in over 45 years. The image above is an actual pathology slide of osteosarcoma cancer cells under a microscope (cancer cells are blue/healthy cells are pink).

• How many kids get osteosarcoma in the US? Nationally, there are about 1000 kids under the age of 20 who get osteosarcoma each year.

• How many kids have it in Nebraska? Nebraska is the #1 state in the midwest for osteosarcoma cancer in children. 70-90 children are diagnosed with pediatric cancer in Nebraska each year and those numbers are rising. Osteosarcoma is one of the hardest cancers to treat. You can find out more by visiting the Nebraska Coalition to End Childhood Cancer's webpage to learn more. Grace's doctors at UNMC are currently researching the impact of pollution and brain cancer in our state and have asked the state legislature to devote millions more in funding to continue to understand why kids in Nebraska are getting sick. When Grace was diagnosed in 2021, we were in active treatment with 12 other families. Three of those children passed away in 2023 and three more are currently in end-of-life care. 

• How did Grace get it? Since Grace's cancer was biopsied and determined to not be genetic, doctor's aren't sure how she got this aggressive type of cancer. Thought leaders in pediatric solid tumor research believe agricultural communities where herbicides and pesticides are regularly used are contaminating drinking water and making kids sick, but more research needs to be done to confirm these early findings. 

• Why doesn't infusion chemo work for Grace? Grace's cancer was genetically smarter than most patients. When she received infusion chemo, her cancer cells genetically modified to become resistant to the medication-making her cancer harder to treat.

• Why is osteosarcoma so hard to fight? All your DNA and genetic material needed to make cells for your body are made in your bone marrow. When you have an aggressive bone cancer, the bad cells start to outnumber the good. Osteosarcoma cancer cells are stronger than most soft tissue cancers and they have the ability to block the body's immune system to destroy them. These cells can also genetically modify quickly to become resistant to chemo. This cancer also spreads quickly over time. If a patient isn't diagnosed early, most osteosarcoma also spreads to the lungs. Once in the lungs, patients have less than a 25% chance of survival. Grace experienced 3 lung relapses in 15 months. She beat the odds and survived longer than most.

• Was Grace in any pain?  The VATS surgery in February 2023 was Grace's second procedure on her lungs in 12 months. Her lungs were very sore then and post-op she complained it felt like she was breathing shards of glass. Losing her lower lobe of her right lung also created complications for her. As her cancer spread to the spine and the nodules grew in her lungs, Grace's pain did increase over time but it was well managed by her hospice team.

• Why did Grace try Vactosertib? Grace was her own advocate and refused more surgeries or infusion chemo to treat new metastasis in 2023. We consulted with solid tumor specialists in Cleveland who are leading a national cancer trial for the medication. Because Grace's cancer was so aggressive, she couldn't wait for the national trial and received this breakthrough medicine in advance by approval from the FDA for "compassionate use". We hoped the medicine would weaken her the cancer cells so her body's own immune system could attack and destroy them-but it was unsuccessful for Grace. To find out more about Vactosertib, you can view the research information provided by Dr. Huang in Cleveland. 

• What is Vactosertib and what does it do? A gene called TGF beta-1 is supposed to do good things, like prevent the body from creating too much inflammation. But in kids with osteosarcoma, TGF beta-1 supports tumor growth and prevents immune cells from attacking the cancer cells, allowing osteosarcoma cells to multiply freely and travel to other places in the body. As it travels, very few immune cells successfully attack the cancer. Vactosertib aims to stop TGF beta-1 from suppressing the immune system and supporting tumor growth in patients. The initial mouse study helped MedPacto secure Orphan Drug Designation (ODD) through the Food and Drug Administration (FDA). Part of why osteosarcoma treatments haven’t changed in decades is because the disease only affects about 400 children and adolescents under the age of 20 each year in the United States. It’s typically not profitable for a pharmaceutical company to develop therapies for a disease that doesn’t affect many patients. The FDA created the ODD through the Orphan Drug Act to incentivize rare disease drug development, aiming to make it profitable for companies and ensuring that kids facing orphan diseases get new treatments. In addition to the pill’s effectiveness in preliminary studies, the drug hasn’t revealed any side effects. Compared to current treatments: which increase infection risk, cause painful mouth sores, hair loss and more–this is extremely promising news for cancer patients who have aggressive osteosarcoma cancer cells like Grace.