Over 310,000 people get breast cancer each year in the US. About 20% of breast cancers are caused by a protein called HER2 and are aggressive. We have developed a vaccine called WOKVAC that trains the immune system to identify and kill cancer cells that have high levels of HER2. Early results in patients show that the vaccine is safe and can create a cancer-killing immune system response. We are now conducting a patient trial where patients with HER2+ breast cancer get the vaccine along with their normal treatment before they have surgery to remove the tumor. Our goal is to have the vaccine create cancer-killing immune cells that will work together with their normal treatment to kill the cancer cells and protect the patient from the cancer for years or decades. So far, we have given the vaccine to 16 patients on this trial. The vaccine has been safe, and early results are encouraging. We are expanding the trial to 25 patients to better help us decide if other patients should get this vaccine. We are looking at how well the patients do after getting the vaccine and looking to see if the vaccine increases the number of cancer-killing cells in their tumors and blood.
This project is about helping young women, age 45 and under, who are diagnosed with breast cancer. These women often have more serious types of cancer and are diagnosed later than older women. We want to make it easier for them to learn about and join clinical trials. Clinical trials are studies that test new treatments to see if they work better than current ones.We want young women with breast cancer to get clear information and strong support when making choices about their care. Many are also dealing with big life events like having children, starting careers, or handling stress. These things can make it hard to think about joining a clinical trial. By adding a research assistant and training nurses to help, we hope to make the process easier and less confusing.This program will help young women feel more confident and informed about their treatment options. It will also help them learn about new therapies through clinical trials. By giving support and easy-to-read materials in both English and Spanish, we hope to improve their care and make their experience less stressful.
Immune therapy is a cancer treatment that turns on killer T cells to attack the tumor. It is a major advance in cancer care. As it is less damaging to healthy tissue than chemotherapy, it has fewer side effects. Most importantly, it can help patients with advanced disease who had few options before. However, many patients do not benefit from immune therapy. The reasons why are not fully understood. Cancer affects people of all ages, but it is much more common in the elderly. T cells are key to the success of immune therapy, but aged T cells do not work as well as young ones. We have discovered that a signal important for T cell function is lost as people age. The loss happens even before a tumor appears. As tumors grow, aging makes even more T cells lose this signal. Our research will test whether the loss of this signal explains why older patients do not respond to immune checkpoint therapies. We will explore ways to restore this signal to improve treatment outcomes. Through this research, we hope to make immune therapy effective for more patients, especially older adults who face the highest rates of cancer.
Funded by the Dick Vitale Pediatric Cancer Research Fund
Glioblastoma is a cancerous brain tumor and the major cause of cancer-related death in children, teens, and young adults. Standard treatments like surgery, chemotherapy, and radiation don’t work here. Our international consortium found a group of glioblastomas caused by problems with how DNA is copied. These are called replication repair deficient (RRD)-gliomas. We showed that they have many mutations and can respond after stimulating the body’s immune defenses using immunotherapy.We recently discovered three types of RRD-gliomas (RRD1-3). Each type acts differently and responds to treatment in its own way. We believe using specific treatments for each group will help patients live longer with fewer side effects. Our plans are:RRD1: These tumors have many immune cells. We will reduce use of harmful treatments like radiation.RRD2: These tumors have fewer immune cells. We will use two kinds of immunotherapy together to help the body fight the cancer.RRD3: These tumors have little immune activity. We will use immunotherapy with drugs that target special features of the tumor. These ideas are based on strong lab studies, tests in animals, and early results in patients. Now we will study how each tumor type responds differently to more precise treatments. We will track and adjust this in real time by testing tumor DNA in the fluid around the brain and spine. This project will advance research and improve care for young people with these deadly brain tumors. In the future, it will be expanded to help treat other types of deadly cancers.
Funded by the Dick Vitale Pediatric Cancer Research Fund
Many children with cancer have changes in their genes that help tumors grow. One important change is called FGFR1 N546K, which is found in about 3% of children with solid tumors, including certain brain cancers and other childhood cancers. This change makes cancer cells grow faster, but current cancer drugs do not work well against it.Our research team will search for new medicines that specifically target this genetic change. We will begin by testing 65,000 compounds to find which ones block the cancer-causing protein. The most promising compounds will then be tested in cancer cells to confirm they work in the right way. Finally, we will study how these medicines attach to the protein using detailed imaging, which will guide us in improving them further.Children with this gene change currently have very few treatment options. If we are successful, the medicines we discover could help treat not only one type of cancer but many different childhood cancers that share similar gene changes. Our ultimate goal is to give doctors new tools that help children live longer, healthier lives and to create a path toward better treatments for childhood cancer.
Funded by the Dick Vitale Pediatric Cancer Research Fund
Rhabdomyosarcoma (RMS) is a connective tissue cancer with features of skeletal muscle, and the most common soft tissue cancer of childhood. RMS can be classified as lacking or having a PAX3::FOXO1 fusion, in which part of the PAX3 protein becomes attached to part of the FOXO1 protein. This hybrid, fused protein is the driving mutation of fusion-positive RMS (FP-RMS). Survival for children with FP-RMS is less than 30%, and this has not improved in over 40 years. In fact, we have no new effective drugs for this cancer. Chemotherapies developed in the 1970s are still the best we have today. This research focuses on understanding how to block PAX3::FOXO1. However, PAX3::FOXO1 is a difficult drug target due to its complex structure. To complicate matters, at least six other fusions have recently been discovered that drive FP-RMS. Rather than being discouraged, we have leveraged this information. We have figured out that all of these seven fusions depend upon a core set of helper proteins to cause FP-RMS. In this project we will figure out the regions of the seven fusions that have common roles and that are responsible for recruiting the helper proteins. Last, we will use hi-tech chemistry to find small molecules to attach to these common regions to dissolve away the helper proteins. This will provide a platform from which to design, and in the future, clinically evaluate new drugs to block any fusion found in FP-RMS. We hope to provide targeted, less toxic treatments.
Funded by Jeffrey Vinik and the Tampa Bay Lightning in support of Hockey Fights Cancer powered by the V Foundation
White blood cells in the body are responsible for fighting disease. The disease is usually infection but the immune system can also kill the tumor in a patient with cancer. There are new forms of treatment called “immunotherapy” which increase the immune response to a tumor in a patient with cancer. This proposal is based on treatment using the white blood cells that reside within a tumor. Because they live within the tumor, they recognize the tumor as foreign, but the tumor defends itself from these cells. To tip the balance in favor of the immune system, these cells are grown outside of the body, away from the harmful effects of the tumor. They are then given back to the patient and since they are stronger, they can more easily kill the tumor. An ongoing clinical trial is testing the treatment in pediatric patients. In this proposal we will evaluate the cells that are given to these patients so we can better understand how they work and improve the treatment for future patients.
Breast cancer is the most common cancer in women. In about one out of three cases, the cancer spreads to other parts of the body. One type, called HER2-positive breast cancer, often grows faster and is harder to treat when it spreads. Current treatments have helped many people, but they do not always work and can cause serious side effects.Our project is creating a new way to both find and treat HER2-positive breast cancer. We are developing a medicine that can deliver tiny amounts of radiation straight to cancer cells. Depending on the type of radiation used, the medicine can either help doctors see the cancer with a scan or destroy it.It works like a “smart missile.” First, it can locate the cancer in the body with a special imaging test. Then, it can carry a different type of radiation to the tumor to kill the cancer cells, while leaving most healthy cells unharmed.We believe this approach could help doctors choose the right treatment for each patient, lower the chance that the cancer will come back, and cause fewer side effects than treatments like chemotherapy. If successful, this strategy could improve both the length and quality of life for people with HER2-positive breast cancer.
Funded by the Dick Vitale Pediatric Cancer Research Fund
Some brain tumours in children grow very quickly and are hard to treat. As a result, many children affected by these tumours have poor outcomes. Scientists know that changes in the DNA of tumour cells help them grow, but they are still learning how these tumour cells interact with the body’s immune system. Tumour cells are surrounded by immune cells, which can sometimes help the body fight the cancer, but in other cases, they may help the tumour grow. Researchers have worked for years to help the immune system find and kill cancer cells. While this approach has worked well for some types of cancer, it has not been effective in treating paediatric brain tumours. By studying the tumour and immune cells together, we hope to identify which types of immune cells are present, what they do, and how they interact with brain and tumour cells. Our study aims to learn how immune cells act around these tumours and how we might be able to change their behaviour to help fight the cancer. We will test whether blocking specific cell communications can help slow down tumour growth and train the immune system to recognise and attack the cancer. What we learn here could lead to new treatments that help children with these serious brain tumours live longer and healthier lives.
Funded by the Dick Vitale Pediatric Cancer Research Fund
Diffuse midline glioma is a deadly brain tumor that affects children. Radiation is the main treatment, since surgery and chemotherapy do not work well. New drugs are being tested, but they are not proven yet. To find better options, we built a new method that combines gene disruption with detailed study of brain tumors. This lets us test the role of many genes in new ways. We found genes that may help tumors respond better to treatment. Now, we will study how these genes work. Our goal is to discover new treatment combinations that can help children with glioma live longer and healthier lives.
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