Kenneth Westover, MD, PhD

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.

Chonghui Cheng, MD, PhD

Cancer vaccines are a promising new treatment that help the immune system find and destroy cancer cells. These vaccine work by teaching the body to recognize special signals, called neoantigens, that appear only on cancer cells. Most cancer vaccines today use neoantigens caused by changes in DNA. But because these changes are different for each person, it is hard to make a vaccine that works for everyone. Our research aims to develop a more widely useful cancer vaccine for triple-negative breast cancer (TNBC), a fast-growing and hard-to-treat cancer. We are studying a new type of neoantigens, that comes from a mistake in how cells process RNA. Normally, cells remove parts of RNA called introns before making proteins. But in some cancer cells, this process fails when a protein called hnRNPM is missing. As a result, the introns stay in, leading to unusual protein pieces that the immune system can recognize and attack. Our team includes experts in RNA, data science, and vaccine development. We are working together to find common neoantigens in TNBC that come from faulty introns and to make a strong mRNA cancer vaccine. We will look for the most common neoantigens made this way in TNBC and build better tools to find neoantigens that current methods miss. We will create mRNA vaccines that teach the immune system to attack these cancer signals. If this works, the vaccine could help treat TNBC and possibly other cancers that make the same neoantigens.

Cheng, Zhang, & Pollet

Andrew Brenner, MD PhD

Some cancers can spread to the fluid that surrounds the brain and spine. This is called leptomeningeal metastases, or LM. It is a serious and often deadly problem. Today, there are very few treatments that work well for this condition.Our team is studying a new treatment called Rhenium Obisbemeda (186RNL). This treatment sends tiny amounts of radiation straight into the spinal fluid, where it can kill cancer cells. Unlike standard radiation, which can hurt healthy parts of the brain, this method targets cancer cells more carefully and reduces damage to normal tissue.In our research, we are collecting samples from patients to see how their cancer and immune cells respond to this treatment over time. We are also using lab models to test whether this radiation works better when combined with other treatments—like drugs that help the body’s immune system fight cancer or block cancer cells from fixing themselves.Our goal is to find safer and more effective ways to treat LM and possibly other hard-to-treat cancers. This research could lead to better options for people with advanced cancer, giving them more time and better quality of life.

Jennifer McQuade, MD

Immunotherapy is a type of cancer treatment that helps the body’s immune system fight cancer. It has changed how we treat many cancers. But not all patients benefit from it. So, we need new ways to make this treatment work better. One area of interest is the gut microbiome. This is the group of trillions of bacteria that live in our gut. These bacteria can affect how the immune system works and how well immunotherapy works. Our research, and that of others, has shown that people who respond to immunotherapy have different gut bacteria than those who do not. Diet plays a big role in shaping the gut microbiome, as the bacteria in our gut eat what we eat. We have shown that diet is linked to how well people respond to immunotherapy. In mice, changing the diet changed both the gut bacteria and the response to treatment. Now, we are testing if diet changes can help patients who are starting immunotherapy. We want to see if we can improve their gut bacteria and boost their immune response through their diet. If this works, it could be a simple and low-cost way to help more people benefit from immunotherapy. We also found that a plant-based, high-fiber diet lowers certain bile acids in the body; these acids may weaken the patient’s immune response. In this study, we will test if these bile acids can be used as a marker of the extent to which the diet and treatment are working.

Andrew Koh, MD

Funded by the Dick Vitale Pediatric Cancer Research Fund

Immune checkpoint inhibitor therapy (ICT) is a form of cancer therapy that boosts the immune system to kill cancer cells.  ICT can help cure some adult cancers but has not been effective in children with cancer. This proposal explores whether a combination of standard cancer therapy and ICT is both safe and effective in children with solid tumors in a clinical trial.  First, we will test tumor, blood, and stool samples collected from patients in this clinical trial.  We will attempt to learn what factors determine whether a patient will respond to this combination therapy or not respond. Second, we will use mouse cancer models to test different combinations of standard cancer therapy and ICT to see which combinations work the best.   This work will help us understand if combining standard cancer treatments with ICT is both safe and effective in children with solid tumors.

Elizabeth Wasmuth, PhD

Abeloff V Scholar*

Funded by the Stuart Scott Memorial Cancer Research Fund with support from Ken Romanzi and Nancy Major Romanzi, M.D.

Our research looks at how hormone receptors play a role in cancer. These receptors are involved in prostate, breast, uterine, and ovarian cancers. Normally, they help control important functions in the body. But as people get older and hormone levels drop, these receptors can stop working properly and help cancer grow.

Even though there are treatments that block these receptors, many patients still see their advanced cancers return within two years. This happens because cancer cells find new ways to turn the receptors back on, which makes the treatments less effective.

To tackle this problem, we use advanced imaging tools, including high powered microscopes, to make 3D models of the hormone receptors. This helps us see how the receptors work and what goes wrong in cancer. We have already found new interactions at the molecular level that were not known before. With support from the V Foundation, we hope to create better drugs that target these receptors more effectively, helping to stop cancer from coming back and improve patient outcomes.

Xin Cai, MD, PhD

Cancer must change its nutrient uptake to grow. Drugs blocking cancer’s use of nutrients have been the basis of cancer therapy. However, most of these drugs work by blocking the pathways that metabolites use. They exhibit significant toxicity since they also harm normal tissues. We are looking at metabolite-targeted therapies that are less toxic. We hope the therapy will be more specific and effective as well. We don’t seek to block metabolite pathways. Instead, we target the specific metabolites that change in the tumor microenvironment. We study and harness the power of our body’s metabolites as drugs. Our work has the potential to change how we target cancer, leading to less toxic and more effective drugs. Our work will also help to diagnose cancer.

Michael Andreeff, MD, PhD

Funded by the Dick Vitale Pediatric Cancer Research Fund with support from Hockey Fights Cancer and Jeffrey Vinik

The most common cancer in children, including teenagers, is a blood cancer named leukemia. Chemotherapy is the main treatment for pediatric leukemias. Although most patients respond well, some do not, leading to poor outcomes. Chemotherapy can also have negative side effects both during treatment and for the rest of their lives.

Patients who don’t get better with chemotherapy are those that have one of most common genetic changes, the rearrangement of a gene called KMT2A (KMT2A-r). In a study at The University of Texas MD Anderson Cancer Center, patients with KMT2A-r leukemia survived for 6 months after 2 chemotherapy treatments and only 2.4 months after 3 or more treatments. Scientists are looking at new ways to treat these patients and help them live longer.

Menin inhibitors could be a good option because they target KMT2A-r leukemia and have fewer side effects than chemotherapy. But some patients with KMT2A-r leukemia can also have mutations in other proteins that don’t let the menin inhibitors work as well by themselves.

With the help of the V Foundation, Drs. Andreeff, Carter and, Cuglievan, at MD Anderson Cancer Center plan to test different combination treatments that target menin and other proteins at the same time to get better results. This can potentially help children with KMT2A-r leukemia live longer and have better lives.

Masataka Suzuki, PhD

Funded with support from the Scott Hamilton CARES Foundation

A new kind of treatment for cancer that helps people’s bodies fight off the disease has allowed some patients to live longer, healthier lives. These new treatments, however, do not work for every type of cancer or for every patient. Solid cancers, in particular, are very good at protecting themselves from these therapies. Also, these new drugs are very difficult and expensive to make and sometimes can cause dangerous side effects.

The overall goal of this proposal is to make a safer and cheaper, but just as powerful, new treatment for solid cancers. For more than ten years, I have worked to develop better, safer cancer therapies. One of these new drugs was just tested in cancer patients and some people responded really well. When patients get this therapy, it acts like a delivery truck, dropping off special instructions to the body and teaching it how to cure cancer all on its own. Because not everyone who received the therapy responded to it, we are writing better instructions so that more patients will have better results. In this proposal, we hope to test these new and improved instructions in mice and see if they can cure cancer. Overall, if this work is successful, we will have discovered a new approach to treating cancers that we can then test in humans.

Josephine Taverna, M.D.

One challenge of lung cancer treatment is that cancer cells thrive in a tumor ecosystem (or habitat) that protects them. This tumor ecosystem consists of immune cells, blood cells, connective tissue that allow lung tumors to grow and spread to organs (brain, bones, liver, lungs). We recently discovered that PD1, AXL and STAT3 signals in lung cancer serves as “on switches” that drive lung cancer growth, treatment resistance and spread to organs. More importantly, these cancer signals allow cancer cells to communicate with nearby cells for protection. We found that blocking PD1, AXL and JAK signaling can block communication between tumor and non‐cancer cells in tumorecosystem. Our research team would like to perform mouse experiments and clinical trial using drug combinations that turn off these signals and disable the tumor within its habitat, thereby preventing tumor growth and spread. This therapy could help improve survival for our patients with lung cancer.

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