2025 Archives - Page 3 of 8

John Mullinax, MD

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.

Sima Ehsani Chimeh, MD

Funded by Hooters

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.

Augusto Faria Andrade, PhD

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.

S. John Liu, MD, PhD

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.

Lillian Guenther, MD

Funded by the Dick Vitale Pediatric Cancer Research Fund

My group investigates specific features of pediatric bone tumors that allow them to survive. One cancer we are interested in is Ewing sarcoma. Ewing sarcoma is a common bone tumor in children. It is challenging to treat, particularly when the cancer has spread. We have become interested in a protein that is important for Ewing sarcoma cells. We want to understand what this protein does in cells. This will help us to kill Ewing sarcoma cells. We are also working with chemists to make new drugs to disrupt its activity. We will test these in Ewing sarcoma cells. Our hope is that these studies will eventually lead to new treatments for Ewing sarcoma.

Markus Müschen, MD, PhD

Funded by the Dick Vitale Pediatric Cancer Research Fund

Acute lymphoblastic leukemia (ALL) is the most common cancer in children. Many children survive with current treatments but when the disease recurs, it is often deadly and as such we need new treatment options for these children.In most cancers, a protein called beta-catenin is highly prevalent, making cancer cells grow faster. We thought this would be true for ALL but found the opposite. In ALL, beta-catenin is kept at very low levels because the cells quickly break it down. When it does appear, it works with a partner called LEF1 instead of its usual partner, TCF7. This pair slows cancer growth and can even make the cells die.This means ALL cells are very sensitive to beta-catenin buildup. We can use this weakness to our advantage. We have found four kinds of existing drugs, already tested in people for other diseases, which block the breakdown of beta-catenin in different ways.Our goal is to test these drugs to see which works best against ALL that does not respond to chemotherapy. Because these drugs are already known to be safe, we can move faster toward clinical testing in children. If successful, this approach could give new hope to families facing relapsed leukemia.

Evan Weber, PhD

Funded by the Dick Vitale Pediatric Cancer Research Fund

Pediatric cancer patients have greatly benefited from CAR-T cell therapy, which is a treatment that uses a child’s own T cells – a type of immune cell – to find and kill cancer. This approach has helped many children with blood cancers, but fewer than half stay cancer-free after one year. One reason for this is that sometimes CAR-T cells don’t survive long enough in the body to stop the cancer from returning. To fix this issue, we are trying to develop ways to make CAR-T cells act more like marathon runners that stay in the fight instead of sprinters that slow down too soon.My research focuses on the bone marrow, which is the part of the body where some blood cancers hide and where CAR-T cells go to fight them. We’ve found that the bone marrow sends out special signals that affect how well CAR-T cells survive. In this project, we will study those signals and use what we learn to create longer-lasting CAR-T cells. This work could ultimately make CAR-T therapies work better for kids and adults and inform other approaches to help these powerful immune cells stay active longer.

Andras Heczey, MD

Funded by the Dick Vitale Pediatric Cancer Research Fund

Special white blood cells can be engineered to fight cancer. They are engineered with a molecule call CAR. This molecule helps them kill cancer cells. The special white blood cells include T and NK cells. These cells can cure some blood cancers. They show great promise for children with solid tumors. CAR T cells often get exhausted in solid tumors. CAR T cells are currently made outside the body. This can further weaken them.Our research aims to overcome these challenges. We will target Glypican-3 (GPC3). It is a molecule found on many childhood solid tumors. We propose a new way to engineer CAR T cells inside the body (in-body). We will use special virus-like particles (LVPs). These LVPs will precisely reprogram the children’s own immune cells to fight GPC3-positive cancer. We will test “armoring” strategies of CAR T cells. This is to make these in-body generated CAR T/NK cells even more powerful and long-lasting.Our hypothesis is that in-body engineered GPC3-CAR T cells will be highly effective against cancer cells. We will first maximize the effectiveness of our LVP delivery system. Next, we will compare the different armoring strategies. We will study how they boost the survival of in-body generated CAR T cells. Finally, we will select the most potent armoring strategy. Ultimately, this research aims to bring safer, more effective CAR T-cell therapy to children with solid tumors. The findings may be applicable to other cancers in the future.

Brian Czerniecki, MD, PhD

Funded by Hooters

Breast cancer is the most common cancer type and 2nd leading cause of death by cancer for women in the US. Patients with early-stage breast cancer or DCIS can survive. But there are groups at high risk for the cancer coming back or developing invasive breast cancer (IBC). These patients are treated with surgey, radiation, and other types of therapies. While these treatments often work, recurrence and IBC are still problems. Our project aims to create a therapy using the patients’ immune system. This therapy will help the body to recognize and engage in the fight against their cancer. If successful, this therapy will be the first non-estrogen inhibitory immunotherapy. And this therapy will help prevent the cancer from coming back and prevent IBC.

Antony Michealraj Kulandai Manuvel, PhD

Funded in partnership with WWE in honor of Connor’s Cure

Supratentorial ependymoma is a rare but serious brain tumor that mostly affects children. Right now, the main treatments are surgery and radiation, but these are not always enough. Our research team is working to understand this cancer better by creating models of the tumor using samples from patients and mice. These models help us find what makes this tumor different from healthy brain cells. We’ve discovered that ST-EPN tumor cells have special dependency for their survival and growth, and we’re testing new avenue to stop the tumor from growing without harming normal cells. We also found that these tumor cells can steal more nutrients than healthy brain cells, which helps them grow faster. By using approved drugs to block this process, we hope to cut off the tumor’s energy supply. By studying real patient tumors and using mouse models, our goal is to find new and better treatments. In the future, this research could help doctors treat children with ST-EPN more safely and effectively.