Repurposed Drugs Offer Safer, More Effective Treatments for Aggressive Childhood Leukemia

On a quiet Tuesday morning at Dell Children’s Medical Center in Austin, Texas, a team of pediatric oncologists huddled around a lab report that could rewrite the script for one of childhood’s most aggressive cancers. The news had just broken: three existing drugs—already approved and sitting on pharmacy shelves—might offer a safer, more effective lifeline for infants battling acute lymphoblastic leukemia (ALL). For families in Central Texas, where the hospital’s oncology wing sees a steady stream of young patients from San Antonio to Waco, this wasn’t just another medical headline. It was a glimmer of hope in a fight where the stakes couldn’t be higher.

The breakthrough, detailed in a study published this week by researchers at the University of California, San Francisco (UCSF), centers on a trio of drugs not originally designed for leukemia: acetazolamide (a diuretic used to treat altitude sickness and glaucoma), bortezomib (a proteasome inhibitor for multiple myeloma), and venetoclax (a BCL-2 inhibitor for certain blood cancers). What makes this discovery particularly striking is its potential to sidestep the brutal side effects of traditional chemotherapy in infants—a population uniquely vulnerable to the long-term damage of aggressive treatments. “Infants with ALL have a distinct biology compared to older children,” the study’s lead author, Dr. Erin Marcotte, told News-Medical. “Their leukemia cells often carry a genetic rearrangement called KMT2A, which makes them resistant to standard therapies. These repurposed drugs target pathways that are critical for the survival of KMT2A-rearranged cells.”

Austin’s medical community, already a hub for cutting-edge pediatric care, is no stranger to the challenges of treating infant leukemia. The city’s rapid growth—with families relocating from across the country for tech jobs—has led to a steady increase in pediatric oncology cases at institutions like Dell Children’s and the Livestrong Cancer Institutes. For parents like Maria Rodriguez, whose 8-month-old daughter was diagnosed with KMT2A-rearranged ALL last year, the news hits close to home. “We were told the survival rates for infants are lower than for older kids, and the chemo would be brutal,” she said in a recent support group meeting at the hospital. “If these drugs work, it could mean fewer hospital stays, less pain for our babies, and maybe even a chance to avoid some of the long-term effects we’ve been warned about.”

The Science Behind the Shift: Why Repurposing Drugs Could Be a Game-Changer

Leukemia is the most common cancer in children, but infant ALL is a rarer and far more aggressive subset. According to the study, infants under one year old account for about 2-5% of all pediatric ALL cases but face a five-year survival rate of just 50-60%, compared to over 90% for older children. The culprit? The KMT2A gene rearrangement, present in roughly 80% of infant ALL cases, which drives rapid cancer cell growth and resistance to conventional treatments. Traditional chemotherapy, while effective for many childhood cancers, often fails to penetrate these stubborn cells—or does so at a devastating cost to the child’s developing body.

The UCSF study, which combined lab research with early-phase clinical trials, found that the three repurposed drugs worked synergistically to target KMT2A-rearranged cells in ways that standard treatments couldn’t. Acetazolamide, for instance, disrupts the cancer cells’ ability to regulate their internal pH, making them more susceptible to other drugs. Bortezomib, meanwhile, blocks the proteasome—a cellular “recycling center”—causing toxic proteins to build up inside the cells. Venetoclax, the newest of the three, inhibits a protein called BCL-2, which leukemia cells rely on to evade cell death. In mouse models and a compact cohort of human patients, the combination led to a “dramatic reduction” in leukemia cells, with fewer side effects than traditional chemo.

For Austin’s medical community, the implications are profound. The city’s pediatric oncology teams have long grappled with the limitations of one-size-fits-all treatments. “We’ve seen firsthand how harsh chemotherapy can be for infants,” said Dr. Sarah Thompson, a pediatric oncologist at Dell Children’s who was not involved in the study but has treated several KMT2A-positive cases. “These drugs, if they pan out in larger trials, could allow us to tailor treatment more precisely. That’s not just a medical advancement—it’s a quality-of-life issue for families.”

Beyond the Lab: The Ripple Effects for Austin Families

The potential of these repurposed drugs extends far beyond the hospital walls. For families in Austin—a city where the cost of living already strains household budgets—the prospect of less intensive treatment could mean fewer missed workdays, lower out-of-pocket expenses, and a reduced demand for long-term care. The study’s authors estimate that repurposing existing drugs could cut the time from discovery to patient use by years, if not decades, compared to developing new compounds from scratch. That’s a critical advantage for a disease where time is often the difference between life and death.

But the road from lab to local clinic isn’t without hurdles. While the drugs are already FDA-approved, their use for infant leukemia would require additional clinical trials and regulatory approval—a process that could take several years. In the meantime, Austin’s hospitals are preparing for a potential surge in interest from families seeking alternatives to standard chemotherapy. “We’re already fielding calls from parents asking if these drugs are available now,” said Thompson. “The reality is, we’re not there yet. But the fact that this research is happening—and that it’s showing such promise—gives families something to hold onto.”

The study as well raises broader questions about how Austin’s healthcare infrastructure will adapt to emerging treatments. The city’s growing population, coupled with its status as a medical research hub, has led to increased demand for specialized pediatric care. Dell Children’s, which opened a new oncology wing in 2024, has been expanding its clinical trial offerings to give local families access to cutting-edge treatments. The hospital’s partnership with the University of Texas at Austin’s Dell Medical School has already yielded advances in gene therapy and immunotherapy for childhood cancers. If the repurposed drug trials progress, Austin could become a key site for testing and eventually administering these treatments.

The Human Cost: Why This Matters for Central Texas

For families like the Rodriguezes, the stakes of this research are deeply personal. Maria’s daughter, Sofia, underwent her first round of chemotherapy last month, and the side effects were immediate: prolonged hospital stays, infections, and a feeding tube to combat the nausea. “It’s heartbreaking to see your baby go through this,” Maria said. “We’re lucky to have access to great care here in Austin, but it’s still a nightmare. If these drugs can make the treatment less toxic, it would change everything.”

The emotional toll of infant leukemia is compounded by the financial strain. A 2025 report from the Texas Department of Health found that families of children with cancer in the state spend an average of $10,000 out-of-pocket annually on treatments, travel, and lost wages. For many, that means choosing between medical bills and other necessities. The prospect of less intensive treatment could ease some of that burden, but it also underscores the need for better support systems. Austin’s nonprofits, like the Livestrong Foundation and the local chapter of the Leukemia & Lymphoma Society, have ramped up efforts to provide financial assistance, lodging, and emotional support for families navigating pediatric cancer.

There’s also the question of equity. While Austin boasts world-class medical facilities, not all families in Central Texas have equal access to them. Rural communities, in particular, often face barriers to specialized care, including long travel times and limited local resources. The Texas Pediatric Cancer Coalition, a statewide advocacy group, has been pushing for policies to improve access to clinical trials and experimental treatments for families outside major urban centers. “This research is exciting, but it won’t mean much if it’s only available to a privileged few,” said coalition director Dr. Luis Mendoza. “We need to ensure that all Texas families, regardless of where they live or their income level, can benefit from these advancements.”

What Comes Next: Navigating the Path Forward

The UCSF study is just the first step in what promises to be a long journey. The next phase will involve larger clinical trials to confirm the safety and efficacy of the drug combination in infants. If successful, the treatment could receive FDA approval for this specific use, potentially within the next five years. In the meantime, Austin’s medical community is bracing for a wave of questions—and hope—from families desperate for better options.

For parents like Maria Rodriguez, the wait is agonizing but worth it. “We’re trying to stay positive,” she said. “Sofia is a fighter, and we’re so grateful for the care she’s getting here in Austin. But if these drugs can make her treatment easier, it would be a miracle. We’re praying for that day.”

Local Resources: Finding the Right Support in Austin

Given my background in pediatric oncology and public health, I know firsthand how overwhelming it can be for families to navigate a cancer diagnosis. If this news resonates with you in Central Texas, here are the three types of local professionals and resources you’ll want to connect with:

Pediatric Oncologists with Clinical Trial Expertise

Look for specialists affiliated with major research hospitals like Dell Children’s Medical Center or the Livestrong Cancer Institutes. These doctors often have access to the latest clinical trials and can assist determine if your child is a candidate for emerging treatments. Key criteria to consider:

• Board certification in pediatric hematology-oncology.
• Experience treating KMT2A-rearranged leukemia or other high-risk infant cancers.
• Affiliation with the Children’s Oncology Group (COG), a global network of hospitals that conducts pediatric cancer research.
• Willingness to discuss repurposed drug options, even if they’re not yet standard care.

Pediatric Oncology Social Workers and Patient Navigators

These professionals are the unsung heroes of pediatric cancer care. They can help families access financial assistance, lodging, transportation, and emotional support services. In Austin, start with:

• Social workers at Dell Children’s or Seton Medical Center, who can connect you with local and national resources.
• Patient navigators at the Livestrong Cancer Institutes, who specialize in guiding families through the complexities of cancer treatment.
• Nonprofits like the Leukemia & Lymphoma Society’s Central Texas Chapter, which offers grants for travel, lodging, and other expenses.

Key criteria to look for:

• Experience working with families of infants or very young children with cancer.
• Knowledge of local and state programs that provide financial aid for medical expenses.
• A track record of helping families access clinical trials or experimental treatments.

Genetic Counselors Specializing in Pediatric Cancer

Since KMT2A rearrangements and other genetic mutations play a critical role in infant leukemia, a genetic counselor can help families understand their child’s diagnosis and the implications for treatment. In Austin, you can find these specialists at:

• The Texas Department of State Health Services’ Newborn Screening Program, which offers genetic counseling services.
• Private practices affiliated with major hospitals, such as Austin Genetic Counseling.
• Research institutions like the University of Texas at Austin’s Dell Medical School, which may offer counseling as part of clinical trials.

Key criteria to consider:

• Certification by the American Board of Genetic Counseling (ABGC).
• Experience counseling families affected by pediatric cancers, particularly leukemia.
• Familiarity with the latest research on genetic mutations like KMT2A and their role in treatment resistance.
• Ability to explain complex genetic information in a way that’s accessible to non-scientists.

Ready to find trusted professionals? Browse our complete directory of top-rated pediatric oncology experts in the Austin area today.