New treatment approach for Pulmonary Lymphangioleiomyomatosis and Tuberous Sclerosis Complex
Official title: Novel Combination Therapy for Treatment and Prevention of PulmonaryLymphangioleiomyomatosis (LAM) and Tuberous Sclerosis Complex (TSC)
Funded at Biosputnik LLC · NIH-10697901 · Data updated 2026-11-12
This study is testing a new combination treatment for people with Pulmonary LAM and Tuberous Sclerosis Complex to see if it can help manage their symptoms and improve their quality of life while being safer than current options.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Biosputnik LLC NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project start | September 1, 2023 |
| Project end | August 31, 2025 |
| Funding year | 2023 |
| Award (this year) | $299,997 |
| NIH project number | 1R41HL164196-01A1 |
| Project ID | NIH-10697901 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are women of childbearing age diagnosed with Pulmonary Lymphangioleiomyomatosis or Tuberous Sclerosis Complex.
✕Less likely to help
Patients with other unrelated conditions or those who do not have LAM or TSC may not benefit from this research.
What this research studies
This research investigates a novel combination therapy aimed at treating and preventing Pulmonary Lymphangioleiomyomatosis (LAM) and Tuberous Sclerosis Complex (TSC), both of which are serious genetic conditions. The approach focuses on targeting the mTOR pathway, which is involved in the growth of benign tumors associated with these diseases. By exploring new therapeutic options that are safer and more effective than the current standard treatment, this research aims to improve patient outcomes and quality of life. Patients may be monitored for their response to the new therapy and any potential side effects.
Why it matters
Potential benefit: If successful, this research could provide safer and more effective treatment options for patients suffering from LAM and TSC.
How similar studies have gone: While there have been studies on treatments for LAM and TSC, this specific combination therapy approach is novel and has not been extensively tested.
Where this research is happening
-
Biosputnik LLC
New York, United States Active
Researchers
- Principal investigator: Pleimes, Dirk — Biosputnik LLC
- Study coordinator: Pleimes, Dirk
Related research
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Investigating the role of specific proteins in kidney disease related to Tuberous Sclerosis Complex.
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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.