Understanding how kidney tumors in Tuberous Sclerosis Complex resist treatment to create new therapies
Official title: Elucidation of Tumor Resistance Mechanisms in Tuberous Sclerosis Complex-Associated Renal Angiomyolipoma for the Design of Novel Nanotherapies
Funded at Brigham and Women's Hospital · NIH-11093992 · Data updated 2026-11-12
This study is looking at how certain kidney tumors in people with Tuberous Sclerosis Complex resist treatment, so researchers can learn more about them and find better ways to help patients.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Brigham and Women's Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | June 1, 2023 |
| Project end | May 31, 2028 |
| Funding year | 2025 |
| Award (this year) | $402,377 |
| NIH project number | 5R01CA269583-03 |
| Project ID | NIH-11093992 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Tuberous Sclerosis Complex who have developed renal angiomyolipomas.
✕Less likely to help
Patients without Tuberous Sclerosis Complex or those who do not have renal angiomyolomas may not benefit from this research.
What this research studies
This research focuses on patients with Tuberous Sclerosis Complex (TSC) who develop benign kidney tumors called renal angiomyolipomas (AML). The study aims to uncover the mechanisms behind the tumors' resistance to current treatments, particularly the drug everolimus, which only partially reduces tumor size. By using genetically engineered patient-derived stem cells to create organoids that mimic these tumors, researchers hope to better understand their biology and develop more effective therapies. This innovative approach could lead to breakthroughs in treating AML and improving patient outcomes.
Why it matters
Potential benefit: If successful, this research could lead to the development of more effective treatments for kidney tumors in Tuberous Sclerosis Complex patients.
How similar studies have gone: While there have been challenges in studying renal angiomyolipomas, this approach using patient-derived organoids is novel and has not been extensively tested in this context.
Where this research is happening
-
Brigham and Women's Hospital
Boston, United States Active
Researchers
- Principal investigator: Lemos, Dario — Brigham and Women's Hospital
- Study coordinator: Lemos, Dario
Related research
Other NIH-funded projects on the same topics.
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VIP Interneurons and Brain Circuits in Tuberous Sclerosis
A study to understand how loss of a brain gene (Tsc1) affects VIP interneurons and the frontal brain region in people and models with Tuberous Sclerosis Complex.
-
Clinical Readiness to Treat Tuberous Sclerosis–Related Neuropsychiatric Issues
A large NIH grant aims to ready treatments for the neuropsychiatric and developmental challenges that many people with tuberous sclerosis complex (TSC) face, including autism-like symptoms and sleep or sensory problems.
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Understanding why renal tumors in TSC resist therapy to guide new nano-based treatments
Researchers are studying why kidney tumors in TSC patients resist current drugs, with the goal of designing smarter, more effective treatments that could help people with TSC-related AML.
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Immune causes of kidney disease in tuberous sclerosis
The team is looking at whether immune cells and a protein called B7-H3 help kidney cysts and growths in people with tuberous sclerosis complex.
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Improving care for autism, learning, sleep, and behavior issues in tuberous sclerosis
This project develops user-friendly tools, follows patients over time, and searches for sleep, sensory, and brain-activity markers to help people with tuberous sclerosis complex who have autism, intellectual disability, or other neuropsychiatric challenges.
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Investigating the role of specific proteins in kidney disease related to Tuberous Sclerosis Complex.
This study is looking at how certain proteins affect kidney problems in people with Tuberous Sclerosis Complex, with the hope of finding better treatments that could help improve kidney health and reduce the need for long-term medication.
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.