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Gene and stem cell therapies are on the rise to treat leukemia and many other serious health conditions, but paying for these new therapies presents new challenges, said Dr. Steven Pearson, founder and president of the Institute for Clinical and Economic Review (ICER).
Stem cell therapies are already in common use for bone marrow cancer, and are approved for patients with acute spinal cord injury, according to Melissa Conrad Stöppler, MD, of MedicineNet. Treatments like CAR T-cells have shown rapid promise in trials and some are approved to fight blood cancers like non-Hodgkins lymphoma.
What do gene and stem cell treatments have in common? All are either single or short-term therapies, making them very different from typical drug regimens, Pearson told Managed Healthcare Executive, an industry trade magazine. Healthcare providers have fewer opportunities to charge for these services, leading to high prices. For instance the $2 million Zolgensma, used to treat spinal muscular atrophy, was labeled the "world's most expensive therapy" this summer in Forbes magazine.
But insurers are wary of these new treatments, and are unsure of their long term success, Pearson said. He said these high costs are especially burdensome for the public Medicaid health system, which cannot raise premiums on its users and must look for funding from taxes.
What do we know about these emerging treatments and their risks over time?
Stem Cells and CAR T-Cells
Stem cells can potentially repair and regenerate damaged tissue, Dr. Stöppler said. This is because these primitive cells have yet to specialize into performing a particular function. This flexibility means they can potentially develop into helpful cells whose growth can be directed by medical professionals.
"Stem cell therapy is an exciting and active field of biomedical research," she said, but potential serious long-term side effects have been identified in these therapies.
Leukemia is one condition that can be treated using either stem cells or CAR-T cells, though some CAR-T cell therapies are still in their trial phase, Dr. Stöppler said. In the case of this blood cancer, a patient's own stem cells may be removed and treated to destroy leukemia cells, or a donor's stem cells may be used.
She said these stem cell transplantations require several weeks of treatment in the hospital. Complications may develop, such as infections and bleeding, as well as a white blood cell reaction to donor tissue known as graft-versus-host disease (GVHD), which can be severe.
"GVHD can occur at any time after the transplant, even years later," Dr. Stöppler writes.
The danger of cytokine-release syndrome (CRS) is possible following CAR T-cell therapy, Dr. Stöppler said. This side effect can cause mild flu-like symptoms, but also serious problems that include nerve damage, suppressed immune function, and a condition known as tumor lysis syndrome that results when cancer cells are rapidly destroyed.
"Because CAR T-cell therapy is so new, the patients who have had this treatment have not been followed over the long term," she writes.
In addition to their currently approved uses, Dr. Stöppler said stem cells are being researched for treating a wide variety of diseases and injuries, including: