How Tuberous Sclerosis tumors use tryptophan to grow

Project 3 - Filippakis

NIH-funded research University of New England · NIH-11251590

This project looks at whether blocking the breakdown of the amino acid tryptophan can slow growth of tumors in people with tuberous sclerosis complex (TSC) and lymphangioleiomyomatosis (LAM).

Quick facts

Grant typeNIH-funded research
Study typeNIH-funded research
Funding institutionUniversity of New England NIH-funded
Lab location1 site (Biddeford, United States)
Project IDNIH-11251590 on NIH RePORTER

What this research studies

From a patient perspective, researchers are studying why cells in TSC grow so much faster by looking at a cell process called macropinocytosis that lets cells gulp up nutrients. They focus on how the tryptophan-kynurenine pathway fuels that nutrient uptake in cells lacking the TSC2 gene, which overactivate mTORC1. In lab experiments with TSC2-deficient cells and related tumor models, they will block the kynurenine pathway and measure effects on nutrient uptake, cell proliferation, and tumor formation. The goal is to find whether shutting down this metabolic route can reduce tumor growth in TSC and LAM.

Who could benefit from this research

Good fit: People with tuberous sclerosis complex (TSC) or lymphangioleiomyomatosis (LAM), especially those with tumor manifestations such as angiomyolipomas, would be the most relevant candidates for related future trials or sample donation.

Not a fit: Patients whose disease is not driven by TSC1/2-related mTORC1 activation or who are not eligible for future clinical trials may not see direct benefit from this specific approach.

Why it matters

Potential benefit: If successful, this work could point to new treatments that slow or shrink TSC- and LAM-related tumors by targeting tryptophan metabolism.

How similar studies have performed: Drugs that block mTOR (like sirolimus) already help some TSC/LAM patients, but targeting macropinocytosis and the tryptophan-kynurenine pathway is a newer, preclinical strategy with promising early lab results but not yet proven in people.

Where this research is happening

Biddeford, United States

Researchers

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page below.
Conditions Bourneville Disease
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.