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Patients reclaim their future as gene and cell therapies rewrite cancer treatment

Patients reclaim their future as gene and cell therapies rewrite cancer treatment GenoMethods.org © genomethods.org
Patients reclaim their future as gene and cell therapies rewrite cancer treatment © genomethods.org
At The University of Kansas Cancer Center, patients with sickle cell disease and multiple myeloma are experiencing a new era of medicine—where their own cells become the treatment.

Ian Wilhite and Emily Schoeb are not waiting for the next big drug. Their own cells are the treatment. At The University of Kansas Cancer Center, this is happening now. Patients are seeing the results of gene and cell therapies in real time.

Ian has lived with sickle cell disease since he was a child. The pain was sharp and unpredictable. Some days, it made life impossible. He had to pause his legal career. Then he traveled from Texas to Kansas City for CASGEVY® gene therapy. That changed everything. "I think the best part about this treatment is that it feels like I’m getting time back," Ian says. For people with incurable blood disorders, time is everything.

As of 2026, the FDA has expanded approval of CASGEVY to include children as young as 2 years old with sickle cell disease experiencing recurrent vaso-occlusive crises or transfusion-dependent beta thalassemia.

DDW-Online

The steps are personal and complex. Blood is drawn. Stem cells are separated. The faulty gene is edited out. The fixed cells go back into Ian’s body. This process takes months. For Ian, it is already working. The pain that once ruled his days is now manageable. "It used to be so intense. It used to be daily. It could be at an eight or a nine or a 10, and I can already start to notice that it just feels like everything got turned down. It’s so much easier to deal with."

The CMS regulatory FAQ lists CASGEVY as one of only two gene therapies approved in the U.S. since December 2023 for certain sickle cell patients. CASGEVY costs $2.2 million per patient. LYFGENIA is priced at $3.1 million as of 2026. CASGEVY uses ex vivo CRISPR/Cas9 gene editing. It disables the BCL11A gene, which normally blocks fetal hemoglobin. This lowers sickle hemoglobin (HbS) and eases symptoms. But these gene therapies are only approved for the "classic" sickle cell type with two HbS alleles (HbSS). Other genotypes are not covered, as explained in a PediatricsNationwide review.

Emily Schoeb’s story is different but just as intense. She learned she had multiple myeloma right before turning 40. Her first thought was her 10-year-old daughter. The diagnosis hit hard. Standard care meant a bone marrow transplant and many drugs. Emily also joined a clinical trial for CAR T-cell therapy. Her own immune cells were reengineered to fight cancer. The result? Her two-year prognosis is now talk of a cure. Joseph McGuirk, DO, her doctor, calls CAR T-cell therapy "a stunning therapy that’s revolutionized the treatment of multiple myeloma."

The science is exact. T cells are taken out. They are changed in the lab to spot cancer. The cells are multiplied to hundreds of millions. Then they go back into the patient. Stem cell transplants are still the standard, but clinical trials at The University of Kansas Cancer Center are pushing new ground. The team is working on off-the-shelf CAR T-cell therapies. These could help more people, especially in rural areas where care is hard to reach. Unlike Emily’s custom treatment, off-the-shelf versions use donor cells, frozen and ready to go.

In April 2024, CARVYKTI (cilta-cel) received expanded FDA approval in the U.S. for use in adults with multiple myeloma after at least one prior line of therapy, including a proteasome inhibitor and immunomodulator, and who are refractory to lenalidomide.

J&J press release

Emily’s view is clear. "I’m honored. Never in a million years would I have thought that getting a cancer diagnosis would be an honor … It’s revolutionary. I feel so lucky to have been diagnosed with cancer when I did, and to have access to The University of Kansas Cancer Center … I feel so lucky and honored and excited to continue to be a part of it. Whatever I can do to help to get the news out and to spread the word."

These are not rare stories. The University of Kansas Health System is building a reputation for complex care. It has sites across Kansas and Missouri. The focus on clinical trials and advanced therapies is part of a bigger shift in cancer care. Recent reported advances in gene-edited stem cell transplants for blood cancers show this movement is growing.

One thing is clear. The old model of one-size-fits-all cancer treatment is fading. Therapies now match the patient. The University of Kansas Cancer Center is not just treating disease. It is giving people their futures back. As gene and cell therapies move from trial to standard care, patients like Ian and Emily are no longer defined by their diagnosis. They are defined by what comes next.

Elena MacLeod Clinical biotechnology and CAR-T editor GenoMethods.org
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Elena MacLeod

Elena MacLeod is Clinical Biotechnology Editor at GenoMethods, covering CAR-T, engineered cell therapies, gene therapy, clinical trials, cancer immunology and regulatory developments. Her evidence-first reporting focuses on trial design, patient populations, safety, efficacy, response durability and the limitations that determine how early clinical results should be interpreted.