Once at home on palliative care, a pediatric patient with osteosarcoma enjoys an active return to school and a prolonged progression-free period while on Vactosertib monotherapy.
Oct 10, 2022
Once at home on palliative care, a pediatric patient with osteosarcoma enjoys an active return to school and a prolonged progression-free period while on Vactosertib monotherapy.
For one young boy with high-risk osteosarcoma, it seemed like treatment options for his rare cancer were exhausted. Now roughly 7 years from the time of his original diagnosis, he had experienced almost every form of treatment available for this rare cancer, but his disease had proven to be quite resistant at every turn. Diagnosed at age 5, he was at the younger end of the age spectrum for this disease, for which the average age of diagnosis is 15. The initial treatment for his cancer required 11 months of aggressive chemotherapy and surgical removal of his lower right leg, a step that is often necessary to reduce the risk of cancer recurrence. When the tumor was removed, pathologists who reviewed his tumor tissue noted a poor response to chemotherapy, a sign that he was at a considerably higher risk for his cancer coming back.
Osteosarcoma typically requires surgery, chemotherapy, and rarely radiation therapy. Surgeries in parallel with chemotherapy make up roughly 45.5% of all total treatment regimens, while drug treatment alone makes up only 15.9%. There is an urgent need for new therapies.
Roughly three years after completing all his treatment, his osteosarcoma came back, this time in his lung. Lung or ‘pulmonary’ metastatic osteosarcoma carries a very high risk of mortality for patients with this disease. Isolated tumors in the lung can be treated, but the rapid development of additional lung tumors is common. He underwent removal of this section of his lung to remove any visible cancer but one year later he suffered another relapse at another site in his lung, this time requiring radiation treatment followed by nearly a year of treatment with combinations of 3 different chemotherapy agents, an approach that ultimately halted the progression of the disease in his lung.
Osteosarcoma is a malignant cancer that develops in connective tissue such as bone, cartilage, or muscle, and is one of the most commonly reported rare cancers and accounts for 5.6% of all sarcomas. It can develop at any age, but most cases occur during childhood, early adolescence, and young adulthood, and 25~50% of patients experience lung metastasis even during chemotherapy.
Yet after fighting lung metastases for almost two years, his osteosarcoma came back again, this time in his brain. His severe symptoms, including headache and vomiting, caused an expanding tumor that was causing bleeding in his brain, requiring a craniotomy to remove the tumor, followed by radiation treatment. This brave 11-year-old continued to fight his cancer, having received treatment for this disease for more than one-half of his life. But the natural history of metastatic osteosarcoma is that it comes back, again and again. Though he enjoyed 6 months without needing any treatment, it recurred once again, this time in the gluteus muscle adjacent to the bones in his hip, requiring more surgery and more radiation. But by December of last year, just 6 months later, the disease in his hip was back. And while he would receive more palliative radiation at this site, it seemed like he had run out of options for treatment and was becoming frail from all his prior therapy. Home hospice seemed like the best option, but his doctors never stopped looking for an alternative for him.
Based on pre-clinical data, doctors at University Hospitals Rainbow Babies & Children's Hospital pursued a compassionate use approval of Vactosertib monotherapy from the FDA
The idea for this approach was the result of an intense effort by physician-scientists at UH Rainbow Babies & Children’s Hospital and Case Western Reserve University School of Medicine who conducted a preclinical trial of Vactosertib in a mouse model of human osteosarcoma that involves the spread of disease to the lung, much like in humans. In this model, Vactosertib improved the survival of mice by 100 percent, significantly restraining tumor growth and pulmonary metastasis, and completely preventing pulmonary metastasis.
Compassionate drug use is making a new and unapproved drug available to treat patients with life-threatening illnesses or incurable diseases, such as terminal-stage cancer, when no other treatments are available. It is a program that makes new and unapproved clinical drugs available to patients at no cost.
With a fighting spirit, this young man agreed to begin treatment with Vactosertib in February of this year. Remarkably, he was active and able to participate in school-related activities without any significant side effects or complications. More importantly, he enjoyed nearly 6 months with no new sites of disease. While Recent scans and a biopsy revealed the disease in his hip has persisted, he remains without any new lesions within either his brain or his lung, a potential benefit from the prolonged exposure to Vactosertib monotherapy.
According to the Reporter Ocean data, the osteosarcoma treatment market size is estimated to be valued at USD 624Million and is expected to register a CAGR of 6.09% to reach USD 791.08Million by the year 2025.