Targeting weaknesses in cancers that use the ALT telomere pathway

Identifying molecular vulnerabilities of ALT-dependent cancers

NIH-funded research Weill Medical Coll of Cornell Univ · NIH-11285147

Looking for ways to exploit specific molecular weak spots in cancers that maintain their chromosomes using the ALT telomere pathway, to help patients with ALT-positive tumors.

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionWeill Medical Coll of Cornell Univ NIH-funded
Lab location1 site (New York, United States)
Project IDNIH-11285147 on NIH RePORTER

What this research studies

This project studies aggressive human cancers that use an alternative lengthening of telomeres (ALT) pathway to keep growing. Researchers use engineered cell models that mimic ALT-positive tumors and a focused CRISPR gene-screening approach to find genes the cancer depends on. They have already identified candidate vulnerabilities such as the enzyme KDM2A and are exploring whether blocking those targets selectively harms ALT cancer cells. The goal is to turn those lab findings into possible drug targets that could be tested in patients down the line.

Who could benefit from this research

Good fit: Adults whose tumors are identified as ALT-positive or who have ATRX/DAXX alterations in their cancer would be the most relevant candidates for eventual therapies from this work.

Not a fit: Patients with cancers that maintain telomeres by telomerase rather than ALT, or children under the adult age range, are unlikely to benefit directly from this specific research.

Why it matters

Potential benefit: If successful, this work could lead to new targeted treatments for patients with ALT-positive cancers that currently have few options.

How similar studies have performed: Preclinical studies have pointed to some targets and vulnerabilities in ALT cells but there are currently no approved targeted therapies, so this approach is promising yet still early-stage.

Where this research is happening

New York, 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.
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.