# Novel Nanomedicine-Based Therapeutic Approach For Treatment of Cancer Cachexia

> **NIH NIH R37** · OREGON STATE UNIVERSITY · 2021 · $446,372

## Abstract

Project Summary
Cachexia is a debilitating syndrome that occurs in numerous diseases including cancer and is characterized by
a significant loss of skeletal muscle mass (with or without fat loss) that cannot be reversed by nutritional support.
It most commonly occurs in gastric (85% of patients), pancreatic (83%) and lung (61%) cancers and up to 30%
of deaths in these patient populations are attributable to cachexia alone. The numerous clinical trials confirmed
that wasting of skeletal muscle mass is the hallmark of cachexia and it is responsible for shorter survival, poor
treatment outcomes, increased toxicity to chemotherapy, post-operative complications, and physical impairment.
Despite all efforts, there are currently no effective therapies to prevent muscle wasting in cachectic patients.
A multidisciplinary team of investigators with complementary expertise in drug delivery, gene therapy, and cancer
cachexia will develop an effective treatment for cachexia-associated muscle wasting. It will be based on
polymeric nanoparticles specifically designed to deliver follistatin messenger RNA (mRNA) to the liver cells after
systemic administration. The delivered mRNA will direct the cellular machinery of the liver cells to produce
follistatin, a secreted glycoprotein that is capable of increasing lean muscle mass through inhibition of myostatin
and Activin A. The last two are growth differentiation factors whose serum concentrations are increased in
cachectic states, and they play a critical role in negatively regulating muscle mass.
The research team has already developed mRNA-loaded nanoplatform that is non-toxic and elevates serum
levels of follistatin protein within 8 h after subcutaneous injection. Wild-type mice that were treated with the
nanoplatform exhibited significant increases in lean body mass. To further advance this technology, the goals
are: (i) to enhance the delivery of the already developed nanoparticles to the liver, (ii) to evaluate delivery
efficiency of the nanoparticles in mice with the humanized liver, and (iii) to validate therapeutic efficacy of the
nanoparticles in novel murine pancreatic ductal adenocarcinoma (PDAC) and lung cancer models that exhibit all
the hallmark features of human cachexia. These goals will be addressed with the following Specific Aims: 1
Evaluate the efficiency of mRNA-loaded nanoparticles following intravenous administration. 2. Test nanoplatform
in mice with the humanized liver. 3. Determine whether nanoparticle-mediated delivery of follistatin mRNA
attenuates cachexia in mouse models of PDAC and lung cancer.
At the completion of this project, the research team will produce strong evidence that the proposed therapy will
preserve muscle mass and attenuate other features of cachexia in mice with pancreatic and lung cancers. The
long-term impact of this project is the successful application of the novel treatment in medical practice to
sufficiently limit death and suffering from various cancers inclu...

## Key facts

- **NIH application ID:** 10144944
- **Project number:** 5R37CA234006-03
- **Recipient organization:** OREGON STATE UNIVERSITY
- **Principal Investigator:** Oleh Taratula
- **Activity code:** R37 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $446,372
- **Award type:** 5
- **Project period:** 2019-05-01 → 2024-04-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10144944

## Citation

> US National Institutes of Health, RePORTER application 10144944, Novel Nanomedicine-Based Therapeutic Approach For Treatment of Cancer Cachexia (5R37CA234006-03). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/10144944. Licensed CC0.

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