Finding new treatments for rare diseases using existing data

Repurpose open data to discover therapeutics for understudied diseases

NIH-funded research Michigan State University · NIH-10704561

This study is looking for new treatment options for rare diseases by using existing data to see how genes in those diseases respond to different drugs, so patients might find new therapies that could help them.

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionMichigan State University NIH-funded
Lab location1 site (East Lansing, United States)
Project IDNIH-10704561 on NIH RePORTER

What this research studies

This research aims to discover new therapeutic options for rare and understudied diseases by utilizing existing open data and advanced computational methods. By analyzing gene expression profiles from disease samples and comparing them with drug-induced profiles from cancer cell lines, the researchers can predict potential new treatments. The approach involves rigorous quality control and integration of various datasets to ensure accurate results. Patients may benefit from new treatment options that are currently not available for their conditions.

Who could benefit from this research

Good fit: Ideal candidates for this research include individuals diagnosed with rare cancers such as hepatocellular carcinoma, Ewing sarcoma, or basal cell carcinoma.

Not a fit: Patients with common diseases or conditions that are well-studied and have established treatments may not benefit from this research.

Why it matters

Potential benefit: If successful, this research could lead to the identification of new therapies for rare diseases that currently have limited treatment options.

How similar studies have performed: Previous research has shown promise in using similar data-driven approaches to identify new therapeutic candidates for various diseases.

Where this research is happening

East Lansing, 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 Cancersneoplasm/cancer
Last reviewed 2026-06-09 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.