Advancing the Most Promising Research

Targeting chromatin regulators of oncogenic transcription in NUP98-rearranged leukemia

This research focuses on a specific and especially aggressive type of childhood blood cancer called NUP98-rearranged acute myeloid leukemia. Children with this form of leukemia have very poor outcomes with current treatments, making it a devastating diagnosis for families and one of the most challenging childhood cancers to fight.

This cancer starts when something goes wrong with a child’s genes, creating abnormal proteins that shouldn’t exist. These rogue proteins essentially hijack the standard controls, telling cells when to grow and when to stop. In healthy cells, there’s a natural process where young blood cells gradually mature and specialize into the different types of blood cells the body needs. But these abnormal proteins keep the cancer cells stuck in an immature state, constantly multiplying instead of maturing properly. It’s like having a factory that only produces defective products and never stops running.

The research team has already had some success blocking certain protein interactions that feed this cancer. Now, they’ve identified two additional proteins, KAT6A and KAT7, that act like essential fuel sources for keeping this type of leukemia alive. Think of these proteins as power sources that the cancer cells need to survive and multiply.

What’s promising about this discovery is that drugs designed to target these specific proteins are already being tested in early clinical trials for other conditions. This means the researchers don’t have to start from scratch. They can build on existing work to figure out how to use these treatments most effectively for children with this devastating disease.

By targeting multiple protein “fuel sources” that the cancer depends on, this approach could potentially offer new hope for children with a type of leukemia that has historically been very difficult to treat.

Selective targeting of epigenetic pathways underlying drug tolerant persistence in neuroblastoma

This research tackles one of the most heartbreaking aspects of neuroblastoma. Even when doctors use the most aggressive treatments available, about 40% of children with high-risk cases will see their cancer return. And when neuroblastoma comes back, it’s almost always fatal.

The problem isn’t that the chemotherapy doesn’t work. It actually kills most of the cancer cells. But here’s what happens: a small group of especially stubborn cancer cells manages to survive even the strongest chemotherapy treatments. These survivor cells essentially go into hiding, lying dormant in the child’s body like seeds waiting for the right conditions. Eventually, these hidden cells wake up and start multiplying again, causing the cancer to return.

The research team has made an important discovery about how these survivor cells stay alive during treatment. They found that these resilient cancer cells depend on two specific proteins, called menin and MLL1, that act like protective shields, helping them survive the chemotherapy that kills other cancer cells.

The researchers’ solution is to combine the standard chemotherapy with a new type of drug called menin inhibitors. These drugs work by blocking those protective proteins, essentially removing the shields that keep the survivor cells safe. Without their protection, these previously untouchable cancer cells should become vulnerable and die along with the rest of the cancer.

What makes this approach promising is that menin inhibitors aren’t experimental. They’ve already been proven safe and effective in children with certain types of leukemia, and the FDA has recently approved them. This means the treatment could potentially be available to children with neuroblastoma relatively quickly, offering new hope for preventing the devastating relapses that currently claim so many young lives.

Targeting Proline tRNA Biogenesis as a Therapeutic Strategy in NOTCH1-Driven T-ALL

This research focuses on T-cell acute lymphoblastic leukemia (T-ALL), a type of blood cancer that affects 10-15% of children who get ALL (acute lymphoblastic leukemia). Historically, children with T-ALL have had worse outcomes than those with other forms of this cancer. For children whose T-ALL doesn’t respond to current treatments, the survival rates are very poor, and doctors currently have no other treatment options to offer these families.

The research team has discovered something important about how T-ALL cancer cells stay alive. All cells in our body need to constantly build proteins to function, like how a factory needs to manufacture products to stay in business. To build proteins, cells use special helper molecules called tRNAs that work like delivery trucks, bringing specific building blocks (amino acids) to the protein-making machinery.

The researchers found that T-ALL cancer cells have an unusual dependency: they desperately need large amounts of a specific type of delivery truck, which carries a building block called proline. Think of proline like a special type of brick needed to build certain structures. Many of the proteins that drive T-ALL cancer growth are built using lots of these proline “bricks,” so the cancer cells need an enormous supply of the delivery trucks that carry proline.

When the researchers block the production of these proline delivery trucks, something remarkable happens: the T-ALL cancer cells can’t build the proteins they need to survive, so they die. But normal, healthy cells don’t depend as heavily on these specific delivery trucks, so they remain largely unaffected.

The team plans to test a drug that blocks this proline delivery system. What’s encouraging is that this drug is already being tested safely in clinical trials for other diseases, which means it could potentially be used to treat children with T-ALL much sooner than if they were starting from scratch. This could offer new hope for families facing this challenging diagnosis, especially those whose children haven’t responded to current treatments.

Interrogation of mast cells as a high-risk biomarker in core binding factor mutated pediatric acute myeloid leukemia

This research looks at a type of blood cancer called acute myeloid leukemia that affects about 700 children each year in the US. Unfortunately, more than 30% of these children don’t survive beyond 5 years. One of the biggest problems doctors face is figuring out which kids are most likely to have their cancer come back, even among children whose cancer initially seems easier to treat; about 30% still see their cancer return within 5 years.

The researchers studied bone marrow samples (the spongy tissue inside bones where blood cells are made) from 99 children with this cancer. They found something interesting: kids with a specific type of this blood cancer had unusually high numbers of immune cells called “mast cells” in their bone marrow. Normally, mast cells make up less than 1% of the cells in bone marrow, but these children had much higher levels.

What’s concerning is that children with more of these mast cells in their bone marrow had worse outcomes, meaning they were more likely not to survive or have their cancer return. The researchers think these mast cells might actually be helping the cancer by creating a “safe space” for cancer cells to hide and grow, while also preventing the body’s natural immune system from effectively fighting the leukemia.

This discovery could help doctors better identify which children are at highest risk for poor outcomes. It might also lead to new treatments that target these challenging mast cells.

Companion Molecular Imaging for PTK7 Targeted Immunotherapies in Pediatric Solid Tumors

Scientists have created a new type of treatment called immunotherapy that works by targeting a specific protein called PTK7. This protein is found in large amounts on dangerous childhood tumors (solid masses of cancer cells), but it’s hardly found at all in healthy parts of the body, which makes it a good target for treatment.

The problem is that doctors currently have no way to tell if a child’s tumor has high levels of this PTK7 protein without doing surgery to remove a piece of the tumor for testing. This is risky and difficult for children who are already sick.

To fix this problem, the researchers are developing a new type of medical scan that works like a special camera. They’re using PET scans, a type of imaging test that’s already commonly and safely used in children with cancer, but they’re adding a “molecular probe” (think of it like a special dye) that sticks only to the PTK7 protein.

When doctors do this scan, they’ll be able to see a map of where PTK7 is located throughout the child’s entire body without any surgery. This will help them figure out which children are most likely to benefit from this PTK7-targeted treatment, and they can also use these scans to see how well the treatment works over time, all without putting the child through an invasive surgery. We are additionally excited about this research as this new way of creating molecular probes could prove to be useful in developing probes for many types of cancer, beyond PTK7 expressing tumors, so many patients in the future will likely benefit from this work.

Intranasal Delivery of Targeted Nanotherapeutics and Oncolytic Virus in Pediatric Glioma

This research focuses on a very aggressive type of brain tumor called diffuse midline glioma that mainly affects children. These tumors are devastating. Most children survive less than a year after diagnosis.

The biggest challenge with treating these brain tumors is their location. They grow in the most critical parts of the brain where surgeons can’t safely operate without causing severe damage. Making things even harder, the brain has a natural protective barrier (called the blood-brain barrier) that blocks most medicines from getting from the bloodstream into the brain. So, even when doctors give children cancer drugs through an IV, very little actually reaches the tumor.

The researchers have come up with an innovative solution: delivering treatment through the nose using simple nose drops. This might sound unusual, but there’s actually a direct pathway from the nose to the brain that can bypass that protective barrier entirely.
They’re planning to test two types of treatments delivered this way. First, they’ll use tiny particles (called nanoparticles) that carry chemotherapy drugs directly to the tumor. Second, they’ll use a specially modified virus that has been engineered to seek out and destroy only cancer cells while leaving healthy brain cells alone.

The beauty of this approach is that it’s completely non-invasive. There is no surgery, no needles, just nose drops that children can receive repeatedly as needed. This could potentially offer hope for children with these untreatable brain tumors.

Advancing Innovative and Effective Therapies for Children with Malignant Rhabdoid Tumors

Malignant rhabdoid tumors (MRT) are one of the most dangerous types of childhood cancer, and they mainly affect babies and very young children. The survival statistics are heartbreaking. Fewer than 1 out of 5 children live more than five years after diagnosis. For babies diagnosed before they’re six months old, fewer than 1 out of 10 survive to age four.

These tumors happen when children are missing a protective gene called SMARCB1. This gene normally helps keep cells healthy and functioning properly, but without it, cells can easily turn cancerous.

Scientists have discovered a promising treatment approach that combines two types of medicine: PARP inhibitors (drugs that prevent cancer cells from repairing their own damaged DNA) and temozolomide (a chemotherapy drug). When used together, these medicines can slow down tumor growth. The problem is that current PARP inhibitors cause serious and dangerous side effects in children.

The researchers are now testing two newer versions of PARP inhibitors, called AZD-5305 and AZD-9574, that have been specifically designed to be much more precise. Think of it like using a scalpel instead of a hammer: these new drugs are engineered to target and destroy only the cancer cells while leaving healthy cells alone, which should dramatically reduce the harmful side effects children experience.

This research represents new hope for families facing this devastating diagnosis. The goal is to find treatments that are both more effective at fighting these aggressive tumors and much safer for the young children who need them

METTL3-targeting ASOs and synthetic lethality approaches in pediatric neuroblastoma

Neuroblastoma is a serious type of childhood cancer that current treatments often can’t cure, especially in the most aggressive cases. What makes this cancer different from others is that it’s usually not caused by damaged genes, but rather by problems with how genes are controlled. Think of it like having healthy light switches but faulty wiring that makes them work incorrectly.

The researchers have identified a key troublemaker: a protein called METTL3 that acts like a master switch, telling genes to help tumors grow and spread. When they block this protein, the cancer slows down, and the cells start behaving more like normal, healthy cells. However, current drugs that block METTL3 have two big problems: they can harm healthy cells throughout the body, and cancer cells eventually learn to resist them.

To solve this, the research team is creating a much more precise approach. They’re developing special RNA molecules (think of them as tiny messengers) that can silence METTL3, but only inside neuroblastoma cancer cells. To deliver these messengers exactly where they need to go, they’re packaging them in microscopic particles and coating those particles with antibodies, proteins that work like GPS systems to find and attach only to cancer cells.

They’re also using CRISPR, a powerful gene-editing tool, to find other proteins that work alongside METTL3. The idea is that blocking METTL3 and these partner proteins together will be much more effective at killing cancer cells than targeting just one protein alone.

This approach could lead to treatments that are both safer for children (because they target only cancer cells) and more effective at stopping this hard-to-treat cancer, giving children and families new hope.

Novel GD2/B7-H3 Bispecific Antibody with Agonist CD40 Antibody, Epigenetic Modifier Inhibitors and Checkpoint Blockade to Improve Treatment Efficacy for High-Risk Neuroblastoma

Researchers are working on a new approach to treat neuroblastoma, a tough childhood cancer. The current primary treatment is an antibody drug that fights cancer, but it has a serious problem: it causes excruciating pain because it attacks both cancer cells and healthy nerve cells. This pain is so severe that doctors often have to give children lower doses of the medicine, which makes it less effective at fighting the cancer. Even with treatment, about 90% of children whose neuroblastoma comes back don’t survive.

The research team is developing a new “smart” antibody drug called INV724 that can tell the difference between cancer cells and nerve cells. Think of it like a guided missile that can distinguish between enemy targets and civilians. It should be able to attack the cancer while leaving healthy nerve cells alone. This could eliminate the severe pain that children currently suffer through while still effectively fighting their cancer.
But the researchers aren’t stopping there. They’re also combining this smart antibody with several other treatments that work in different ways:

• A drug that activates the immune system to better recognize and attack cancer
• Medicines that change how cancer genes work (called epigenetic modifiers)
• Drugs that remove the “brakes” from the immune system so it can fight cancer more aggressively (checkpoint blockers)

The idea is that using multiple treatments together that attack cancer from different angles will be much more effective than any single treatment alone. If this combination approach works, it could be the first truly effective treatment for children whose neuroblastoma doesn’t respond to current therapies, potentially saving many young lives while sparing them from treatment-related suffering.

Translational Strategies To Enhance B7-H3-CXCR2 CAR T Homing and Function in Sarcoma

This research focuses on pediatric sarcomas, which are aggressive cancers that develop in bones, muscles, and other soft tissues in children. These cancers have heartbreakingly low survival rates. Only 20-30% of children survive when the cancer spreads to other parts of the body or comes back after treatment. What makes this even more frustrating is that treatments for these cancers haven’t gotten much better in over 20 years, and the current treatments often cause lifelong health problems for the children who do survive.

The researchers are working on a cutting-edge treatment called CAR T cell therapy. Here’s how it works: doctors take immune cells (specifically T cells, the body’s natural cancer fighters) from the child’s own body, then genetically modify them in a laboratory to make them much better at recognizing and attacking cancer cells. Think of it like giving the immune cells a special training course and new weapons before sending them back into the body to fight cancer.

The big problem with this approach for solid tumors like sarcomas is that these specially trained immune cells have trouble actually reaching the cancer. It’s like having excellent soldiers who can’t find the battlefield.

To solve this, the research team has created enhanced CAR T cells with two special features:

1. They target a protein called B7-H3 that’s found on sarcoma cancer cells (like giving them a “wanted poster” of their target)
2. They express something called CXCR2, which acts like a GPS system that helps the immune cells follow chemical trails that tumors naturally product

This should help the modified immune cells find their way to the cancer and be more effective at destroying it once they reach it. For families who have watched their children endure years of ineffective treatments, this breakthrough could finally offer them a chance to beat this disease.

PR Domain Inhibition to Target Leukemia Stem Cells in Pediatric Acute Myeloid Leukemia

This research focuses on acute myeloid leukemia (AML), a type of blood cancer that affects 15-20% of children who develop leukemia. One of the most heartbreaking aspects of this cancer is that even when treatment initially works and the cancer seems to be gone, it often comes back later, devastating children and families with more rounds of treatments.

This happens because of special cancer cells called “leukemia stem cells.” Think of these like the roots of a weed. Even if you cut down the visible parts of the weed (destroying most of the cancer), if the roots remain underground (these stem cells hide and survive treatment), the weed will eventually grow back. These leukemia stem cells are incredibly tough and can survive chemotherapy that kills other cancer cells, which is why the cancer returns in many children.

The researchers have discovered something important: there’s a protein called PRDM16 that acts like a “master switch” or “life support system” for these stubborn stem cells. This protein essentially keeps the leukemia stem cells alive and protected during treatment.

The research team is developing a new drug that specifically targets and blocks this PRDM16 protein. The goal is to cut off the life support to these treatment-resistant stem cells, causing them to die along with the rest of the cancer. What’s promising is that this drug appears to harm only the leukemia stem cells while leaving the body’s healthy blood stem cells (which children need to make normal blood cells) completely unharmed.

This approach gets to the root cause of why cancer comes back after treatment, and because the drug specifically targets the problem cells, it could provide a much safer and more effective treatment option for children with high-risk AML. The research is advanced enough that it could potentially move to clinical trials relatively quickly, which could mean more effective treatments for children sooner rather than later.

Dual inhibition of MDM2 and tubulin for precision treatment of acute myeloid leukemia

This research focuses on acute myeloid leukemia (AML), a type of blood cancer that’s especially challenging to treat in children. Currently, fewer than 70% of children with this cancer survive five years after diagnosis, and about half of all patients don’t respond well to the chemotherapy treatments we have now. To make matters worse, these current treatments cause severe side effects that can affect children for the rest of their lives.

The researchers have made an important discovery about how this cancer works. They found that two proteins, called MDM2 and tubulin, team up inside cancer cells to help them grow and survive. Think of these proteins like partners in crime: when they work together, they make the cancer stronger and harder to defeat. This partnership is significant in about 50% of AML patients who have high levels of the MDM2 protein and tend to have worse outcomes.

The research team has identified a promising new drug called L-243 that works like a wrench thrown into the gears of this partnership. Instead of using chemotherapy that attacks all rapidly dividing cells (both healthy and cancerous), L-243 specifically targets and disrupts the connection between these two proteins. When their partnership is broken, the cancer cells can’t survive, and they die, while normal healthy cells are largely left alone.

This selectivity is what makes L-243 potentially much safer than traditional chemotherapy. It’s like using a precision tool instead of a sledgehammer. Children could potentially get effective cancer treatment without experiencing the devastating side effects that current treatments cause.

What’s encouraging is that L-243 is based on a compound already being tested in clinical trials for other types of cancer. This means the development process could move more quickly than starting completely from scratch, which could bring hope to families sooner rather than later.

Redefining residual disease detection in pediatric AML

Dr. Green Gladden is using a new technique (single-cell RNA sequencing) to better understand the risk for relapse due to the presence of low levels of cancer cells following standard treatment in children with acute myeloid leukemia (AML).

As part of this CURE-funded study, Dr. Green Gladden proposes to redefine the way leukemic response to chemotherapy is measured by not just asking, “How much disease remains?” but “What is the biology of the disease that remains?” which is evaluated by looking at the gene expression of residual leukemic cells.  By better understanding risk for relapse and improving how risk is determined and categorized, she hopes to improve outcomes for patients with AML.  Understanding the biology of residual disease will pave the way for future therapy development.

Defining determinants of a cGAS-STIGN-mediated anti-tumor inflammatory response in osteosarcoma

Dr. Young’s research aims to gain an improved understanding of how osteosarcoma avoids the body’s immune surveillance and to deliver a novel treatment paradigm for this highly aggressive pediatric cancer. Recent attempts to introduce better treatments for osteosarcoma have not been successful, and this research aims to be the basis for the development of a broad range of new immunotherapy treatments for osteosarcoma. Her laboratory has identified that activation of a biochemical pathway called STING has anti-tumor benefits in animal models and a protective effect in human disease. Therefore, they will study this pathway as an important therapeutic target. This proposal will serve as the foundation for ongoing work to translate STING-activating therapies for patients with osteosarcoma.

Defining replication stress and DNA damage as a therapeutic vulnerability in osteosarcoma

The goal of Dr. Sweet-Cordero’s research is to evaluate new combination therapies for osteosarcoma, a disease for which few improvements in therapy have been achieved in the last 40 years. In this research, his lab will utilize a unique collection of patient-derived cell lines that reflect the true state of patients’ cancers. Through previous extensive studies, the Sweet-Cordero lab identified a potent and promising combination of drugs (gemcitabine/ATRi). In this CURE-sponsored research, the lab will test this drug combination further on human and mouse models. These studies will serve as necessary evidence to support the development of possible clinical trials within the next 18-24 months.

Targeting and imaging serine metabolism in the tumor microenvironment in pediatric brain tumors

Diffuse midline gliomas (DMGs) are the deadliest form of brain cancer in children, and no effective treatments currently exist. Dr. Viswanath has shown previously that DMG tumor growth depends on serine (an amino acid crucial for cell function), and blocking serine in the tumor environment sensitizes the cancer cells to existing immunotherapy. Dr. Viswanath’s lab has also found that a new tumor imaging technique called deuterium metabolic imaging allows them to identify whether the tumor is responding to therapy quickly. In this CURE-funded study, they will determine whether reducing serine has a therapeutic effect and whether deuterium metabolic imaging can be used to monitor treatment response in mouse models of DMG. These studies will lay the foundation to bring this innovative therapeutic and imaging strategy to children with DMG in the near future.

c-MYC protein degradation in therapy-resistant pediatric leukemias

Dr. Andreeff’s research is focused on reducing the levels of c-MYC, a protein that is known to play a critical role in regulating cell growth and proliferation, in pediatric leukemias that do not respond well to traditional treatments. Dr. Andreeff proposes to investigate a novel drug candidate, GT715. His lab has already demonstrated highly promising therapeutic effects in cell and animal models, though the drug has not yet been tested in patients. Dr. Andreeff will also be working on testing another promising therapy, called venetoclax plus 5’-azacitidine or nelarabine to enhance the therapeutic effect of GT715. This experimental approach is aimed at finding alternative therapies to improve treatment outcomes in very challenging cases, and the hope is that this research will lead to early-phase clinic trials soon.

Metabolic reprogramming of the Ewing sarcoma tumor microenvironment using pramlintide to augment NK cell immunotherapy

The treatment options for children and adolescents with metastatic Ewing sarcoma are limited, and thus new strategies are needed. Dr. Kleinerman’s research aims to develop a strategy where pramlintide, a synthetic hormone involved in regulating blood sugar levels, is used to alter the metabolic conditions within the Ewing Sarcoma tumor environment. Decreasing available sugar in the tumor environment has the potential to starve the tumor cells and reduce their ability to spread. This alteration aims to improve the effectiveness of NK cell immunotherapy against Ewing Sarcoma (Natural Killer, or NK cells are a type of immune cell that can directly kill cancer cells), potentially making the tumor more susceptible to immune attack by NK cells. These studies can provide the necessary justification to rapidly move to a clinical trial.

Development of a GPC2 CAR T cell amplifying RNA vaccine

Dr. Bosse’s research aims to develop a novel therapy for neuroblastoma that involves using an RNA vaccine to boost the function of an immunotherapy involving CAR T cells engineered to target GPC2. GPC2 is a protein that is found on the surface of neuroblastoma cells, but not on healthy cells. Thus, the engineered CAR-T cells can specifically seek and destroy neuroblastoma while leaving healthy cells unscathed. If this research strategy is successful, Dr. Bosse’s lab will have developed a novel technology that will hopefully cure children with neuroblastoma and several other different types of pediatric tumors that also have high levels of GPC2.

Effective TGF-beta signaling blockade synergizes cryoablation-induced STING activation in treating refractory and metastatic sarcoma

Effective therapy for refractory or metastatic osteosarcoma, rhabdomyosarcoma, and other sarcomas remains a major challenge. Dr. Huang’s lab will combine two approaches that are believed to be promising in treating metastatic disease: cryoablation (an FDA-approved approach using extreme cold against the tumor) and the non-toxic drug Vactosertib (a-TGF-beta inhibitor). cryoablation, through mechanisms that are not completely understood, can increase the patient’s immune response against the spreading tumor. However, the response is inconsistent between patients and is often weaker than necessary. It is believed that Vactosertib can augment the immune response and work in concert with cryoablation to mount anti-tumor immune responses in patients. Dr. Huang hopes that, if successful, these CURE-funded studies can provide the preliminary data required to enter a clinical trial in the near future.

Dissecting and targeting PAK4-mediated signaling in Ewing sarcoma development and metastasis

Ewing sarcoma is the second most common bone tumor in the pediatric population, and overall survival rates have improved to approximately 70%. However, despite maximizing treatment regimens, long-term outcomes for patients with metastatic disease remain extremely poor, with overall survival rates dropping to 20-30%. Thus, finding different treatments, or combination of treatments, is essential in order to increase survival for Ewing sarcoma patients. With funding from CURE, Dr. Yustein’s lab will determine if inhibiting PAK4 (a protein that plays a role in regulating cell growth and movement ) in combination with other Ewing-relevant therapies will be effective in blocking metastasis. In addition, they will also further understand the role of PAK4 in the Ewing sarcoma cell, which can identify additional treatment options. Completion of these CURE-funded studies will impact Ewing sarcoma patients by identifying new treatment regimens that can be quickly transferred to clinical trials for high-risk disease.

Defining the druggable GLI Interactome in medulloblastoma

The most common malignant pediatric brain tumor is medulloblastoma. Although much is understood about the genetic makeup of this tumor, there are still forms of the disease that do not respond to therapy. Further, children who do respond to therapy suffer from intellectual and cognitive deficits. Therefore, we urgently need to identify new, more effective, and less-toxic drugs for the treatment of these tumors. In this CURE-funded study, Dr. Robbins will work to better understand a collection of over 200 proteins, called the GLI Complex, that work together in Medulloblastoma to drive mortality and morbidity. As part of this study, they will also identify and test drug candidates that are expected to inhibit the GLI Complex and, in so doing, stop the spread of MB. Importantly, as part of this work, they will also investigate the toxicity of the drug candidates, ensuring that any new treatments will be minimally or non-toxic to the developing brain.

Rapid Transition of B7-H3 Targeted Therapies to High-Risk Childhood AML

Dr. Meshinchi aims to use his CURE funds to rapidly develop two complementary therapies targeting B7-H3, a protein expressed in very high-risk pediatric acute myeloid leukemia (AML). The project combines short-term repurposing of an antibody-drug conjugate called Vobramitamab duocarmazine (Vobra duo), currently in clinical trials for prostate cancer, with the long-term goal of establishing immunotherapeutic cures for aggressive pediatric AML expressing B7-H3 using CAR T cells. Based on his preliminary data, Dr. Meshinchi believes that effectively targeting B7-H3 could transform outcomes for these very high-risk AML. In the very near future, Dr. Meshinchi hopes to have enough pre-clinical data to support compassionate use access to Vobra duo and will pursue clinical trials with both pediatric and adult AML patients.