CLEVELAND — There's certainly something to be said for being in the right place at the right time.
Dr. Alex Huang overheard a conversation between colleagues discussing a new type of drug being tested in adult cancer patients. What really caught his attention was the fact that it virtually had no side effects.
"Very minimal side effects," he said. "You don't lose hair, you don't have sores in your mouth, you don't drop your blood counts, you don't need transfusions, you don't get nausea, vomiting, and all the other issues that typically associate with with chemotherapy and other modalities for cancer."
Huang is the co-leader of the immune oncology program at Case Western Reserve University's Comprehensive Cancer Center. Along with Dr. Kristen VanHeyst — an assistant professor in the Department of Pediatrics at Case's School of Medicine and part of the immune oncology team specializing in osteosarcoma — he saw a chance to help patients who often have little hope.
"Osteosarcoma is the most common primary aggressive malignancy of the bone in adolescents and young adults, as well as in older adults," VanHeyst explained. "Unfortunately, the prognosis for osteosarcoma overall has not changed in decades, and the standard of care has also not changed in decades. So a patient who does have disease recurrence or refractory disease or metastatic disease often has a very grim prognosis, and there are just not many treatment options available to them."
The drug that piqued their interest is called Vactosertib, made by South Korean pharmaceutical company MedPacto. It was designed to inhibit the body's TGF-beta molecules to suppress tumor progression and metastatic growth.
It's already being studied for several different types of cancer, including colorectal and gastrointestinal cancers. It's a different type of immunotherapy that is believed to not attack the actual tumor, but the space it inhabits.
"We believe it changes the tumor microenvironment — the actual environment in which the tumor lives — as opposed to directly addressing the tumor itself, to make the environment in which the tumor lives less hospitable to the tumor," VanHeyst said.
Huang told us getting the manufacturer to add osteosarcoma to the testing protocol was a team effort.
"We are able to come together with industry partners who were very willing to participate and to explore the possibility of offering a new drug to this patient population with us together," Huang recalled. "I think that partnership is unique in our ability to actually offer and create a set of data that is convincing enough — not just to our industry partner, but also the FDA — that we are able to generate sort of preclinical data to convince a variety of different stakeholders that this we might be on to something here."
The phase 1 clinical trial at UH Rainbow Babies and Children's Hospital opened this past Wednesday for osteosarcoma patients 18 years old and up. Researchers hope to recruit 48 patients, and in the spring, they expect to be able to accept patients as young as 12. The study is testing safety and dosing in this phase.
Vactosertib is an oral medication in pill form.
"Patients will take a two day, two times a day for five days, and they will have two days off," Huang described. "You do it from Monday to Friday and you take Saturday, Sunday off, and you do that repeatedly for three or four weeks."
Another benefit is that patients do not have to stay in the hospital overnight, and patients who live out of state don't need to travel long distances frequently.
While they're cautiously optimistic about the drug, they know there's several years of research ahead of them. What they also do know is in lab rats who had osteosarcoma tumors, those tumors were shrinking after three to four weeks of being on the drug, and it also stopped the spread of osteosarcoma cancer throughout the body.
While Rainbow Babies and Children's is the first open study site, it will eventually become a multi-continental, multi-institution phase 1/2 clinical trial that will take place in more than 20 hospitals throughout the United States, Europe, and South Korea.
"This trial itself is an important step because it gives an option for these patients to try," VanHeyst said. "And for those I speak with and the physicians around the country and even internationally who have reached out to us, this is a big step for them, because it offers some hope for a potential therapeutic option."
For more information on the Vactosertib for Osteosarcoma phase 1 clinical trial, email PHOCTU@uhhospitals.org or head to clinicaltrials.gov.