Researchers investigated whether a drug called fedratinib, currently used to treat a blood disorder, might help patients with chronic kidney disease—a serious condition where kidneys gradually stop working properly. Using computer models and real-world safety data, scientists found the drug could potentially reduce kidney scarring, but it also carries some important risks including vitamin B1 deficiency and kidney damage. The study suggests that if doctors do use this drug for kidney patients, they should use lower doses and closely monitor patients’ kidney function and vitamin levels. This research highlights the challenge of finding new treatments: drugs that help in one way can sometimes cause problems in another.
The Quick Take
- What they studied: Whether a drug called fedratinib could help treat chronic kidney disease (kidney scarring) and what safety concerns it might cause
- Who participated: The study analyzed 1,268 real-world reports of people who took fedratinib between 2019 and 2025, collected from the FDA’s adverse event reporting system
- Key finding: Computer models predicted the drug could reduce kidney scarring by blocking harmful pathways, but real-world safety data showed it actually caused kidney and urinary problems in some patients and severe vitamin B1 deficiency (1,262 times more common than expected)
- What it means for you: Fedratinib might eventually help kidney disease patients, but it’s not ready for regular use yet. If doctors consider using it, patients would need lower doses and frequent blood tests to monitor kidney function and vitamin levels. This is still experimental and requires much more research.
The Research Details
Researchers used two main approaches to study fedratinib. First, they used computer programs (like virtual drug-testing software) to predict whether the drug would be toxic and which body systems it might affect. They also mapped out how the drug interacts with different proteins in the body to understand its effects. Second, they analyzed real-world safety reports from the FDA database covering six years (2019-2025) to see what side effects people actually experienced when taking the drug. This combination of computer predictions and real-world data helps researchers understand both the potential benefits and actual harms of a medication.
This research approach is important because it combines theoretical predictions with real-world evidence. Computer models can quickly identify potential problems before large clinical trials, saving time and money. However, real-world data is crucial because drugs sometimes behave differently in actual patients than predicted. By comparing what the computer models predicted with what actually happened to patients, researchers can identify unexpected dangers and develop safer treatment strategies.
The study’s strength lies in analyzing a substantial number of real-world reports (1,268 cases) from the FDA’s official database, which captures actual patient experiences. However, the study has limitations: it’s observational rather than experimental, meaning researchers couldn’t control all variables. The computer predictions, while sophisticated, don’t always match real-world outcomes perfectly. The study doesn’t include a control group of kidney disease patients not taking the drug, making it harder to determine if all reported problems were truly caused by fedratinib. Additionally, the sample size for kidney disease patients specifically wasn’t clearly defined.
What the Results Show
Computer models predicted that fedratinib would be relatively safe for kidneys but could cause serious problems in the lungs, liver, and heart. However, real-world safety data told a different story. Analysis of FDA reports found that kidney and urinary problems occurred 1.71 times more frequently than expected in people taking fedratinib. Even more striking, vitamin B1 deficiency was reported 1,262 times more often than expected—an extraordinarily high number suggesting a strong connection between the drug and this deficiency. This contradiction between computer predictions and real-world outcomes is a critical finding that suggests the drug affects the body in ways the computer models didn’t anticipate.
The research identified how fedratinib works at the molecular level. The drug blocks several proteins (SRC, JAK2, AKT1, and EGFR) that are involved in kidney scarring. By blocking these proteins, it could theoretically reduce fibrosis—the harmful scarring that damages kidneys. However, blocking AKT1 and EGFR also appears to cause kidney injury and vitamin B1 depletion. The study noted that fedratinib carries a ‘black-box warning’ (the FDA’s strongest safety warning) for a serious brain condition called encephalopathy, which may be related to the vitamin B1 deficiency.
Fedratinib was originally developed and approved for treating myelofibrosis, a blood disorder involving bone marrow scarring. The drug’s ability to reduce scarring in blood disorders raised hopes it might work similarly in kidney disease. However, this study reveals that the drug’s effects are more complex and potentially more dangerous in kidney patients than in blood disorder patients. The findings suggest that drugs effective for one type of scarring may not be safe or effective for another, and that careful testing in each specific disease is essential.
The study has several important limitations. First, it’s based on voluntary reports to the FDA, so some side effects may be underreported while others might be overreported. Second, the study couldn’t prove that fedratinib directly caused the reported problems—only that they occurred together. Third, the computer models didn’t accurately predict the kidney and vitamin B1 problems, suggesting our current understanding of how this drug works is incomplete. Fourth, the study didn’t include detailed information about dosing, duration of treatment, or patient characteristics that might explain why some people experienced problems. Finally, this research doesn’t include controlled clinical trials, which are necessary to definitively prove safety and effectiveness.
The Bottom Line
Based on current evidence, fedratinib should NOT be used routinely for chronic kidney disease outside of research studies. If future clinical trials show promise and doctors consider using it, the research suggests: (1) Use a 50% lower dose (200 mg daily instead of 400 mg) for patients with severely reduced kidney function or those age 70 and older, (2) Monitor kidney function with blood tests regularly, (3) Check vitamin B1 levels frequently, (4) Supplement with vitamin B1 if levels drop, (5) Watch for signs of brain problems (confusion, weakness, numbness). Confidence level: MODERATE—based on observational data, not controlled trials.
This research is most relevant to: (1) Kidney disease patients and their doctors considering experimental treatments, (2) Researchers developing new kidney disease therapies, (3) Pharmaceutical companies studying fedratinib for kidney disease, (4) Regulatory agencies like the FDA evaluating drug safety. People with other conditions taking fedratinib should discuss vitamin B1 monitoring with their doctors. This research should NOT change treatment for people currently taking fedratinib for approved uses without consulting their physician.
If fedratinib were approved for kidney disease (which is not currently the case), benefits in reducing kidney scarring might take weeks to months to appear, similar to other anti-scarring treatments. However, side effects like vitamin B1 deficiency could develop within weeks. Any actual use would require ongoing monitoring indefinitely. Realistic expectations: this is early-stage research; 5-10 years of additional clinical trials would be needed before this drug could potentially be approved for kidney disease.
Want to Apply This Research?
- If a patient were enrolled in a clinical trial using fedratinib, they should track: (1) Kidney function markers (eGFR and creatinine levels from blood tests), (2) Vitamin B1 levels (from blood tests), (3) Symptoms of vitamin B1 deficiency (tingling in hands/feet, weakness, confusion), (4) Urinary symptoms (changes in urine color, frequency, or pain), (5) Neurological symptoms (dizziness, numbness, memory problems). Record test dates and results monthly.
- Users in a hypothetical fedratinib trial could use an app to: (1) Set reminders for scheduled blood tests and doctor visits, (2) Log vitamin B1 supplement doses if prescribed, (3) Record any new symptoms or side effects daily, (4) Track dietary sources of vitamin B1 (whole grains, nuts, pork, fish), (5) Maintain a medication log showing exact doses and timing, (6) Generate reports to share with their doctor at appointments.
- Long-term monitoring would require: (1) Monthly blood tests for kidney function (eGFR, creatinine) and vitamin B1 levels for the first 3-6 months, then quarterly if stable, (2) Regular doctor visits (monthly initially, then every 3 months) to review results and symptoms, (3) Annual comprehensive kidney disease assessments, (4) Continuous symptom tracking through the app with alerts for concerning changes, (5) Coordination between nephrology (kidney specialist) and primary care doctors, (6) Documentation of all side effects for safety reporting.
This research is preliminary and does not establish that fedratinib is safe or effective for treating chronic kidney disease. Fedratinib is not currently approved by the FDA for kidney disease treatment. This article is for educational purposes only and should not be used to make medical decisions. If you have chronic kidney disease, consult your nephrologist (kidney specialist) before considering any new treatments. If you are currently taking fedratinib for an approved condition, do not stop or change your dose without talking to your doctor. The findings about vitamin B1 deficiency and kidney problems are based on observational data and require confirmation through controlled clinical trials. Anyone experiencing symptoms like confusion, weakness, numbness, or changes in urination while taking fedratinib should contact their healthcare provider immediately.