# Expanded Clinical Trial of Granulocyte Colony-Stimulating Factor (G-CSF) to Treat Hot Flush and Other Vasomotor Symptoms in Women with Naturally-Occurring and Surgically-Induced Menopause

> **NIH NIH R43** · MENOGENIX, INC. · 2020 · $299,831

## Abstract

One of the most common and distressing symptoms of menopause are hot flashes. Hot flashes and
related symptoms represent a significant unmet, worldwide, healthcare need, not only in women after
surgical or natural menopause, but also in breast and prostate cancer patients undergoing hormone
suppression or ablation. Hot flashes occur in 65 – 90% of women undergoing natural menopause and
approximately 70 million individuals worldwide seek therapy each year (~33% of all women aged 50-
65). A hot flash is a sudden feeling of warmth that is generally most intense over the face, neck and
chest as well as peripheral vasodilation leading to flush. The duration is variable but averages about 4
minutes. It is often accompanied by sweating. Although not life-threatening, hot flashes can have a
detrimental effect on a woman's functional ability and quality of life. The physiological mechanisms
leading to a hot flash episode are not known. Similarly, it is not known how decreasing hormone
levels that occur during the menopause transition lead to hot flashes and related symptoms. The only
FDA-approved therapies for hot flashes are hormone therapy (HT) and a low dose form of paroxetine,
an anti-depressant. HT is associated with severe health risks including heart disease, cancer, stroke
and dementia. Paroxetine use is associated with suicidal thoughts and addiction. While there are no
other FDA-approved alternatives, physicians often prescribe other anti-depressants, anti-psychotic
agents and pain medications, all of which have significant side effects. Seeking less dangerous
alternatives, many women turn to alternative therapies including nutraceuticals and acupuncture but
none of these have proven to be effective in placebo-controlled trials. An effective, non-hormonal,
non-anti-depressant, treatment for hot flashes and related vasomotor symptoms would be a welcome,
novel and useful treatment modality. MenoGeniX is repurposing a naturally occurring human protein
called G-CSF to treat hot flashes based on placebo-controlled phase 1 clinical trial data which
showed that a single administration of G-CSF can lead to a rapid and significant reduction in hot flash
frequency and severity in women with natural and surgically-induced menopause. G-CSF was well
tolerated: no serious adverse events were reported and adverse events were generally mild, of short
duration, and should not impact clinical adoption. Women with surgically-induced menopause as well
as African-American women responded especially well. In this phase 1 SBIR application, we are
requesting funding to evaluate the safety and efficacy of G-CSF administered monthly (three single
injections at 28-day intervals) to treat menopausal hot flashes. With a well-established safety record,
G-CSF may represent a valuable alternative for women who are unwilling or unable to take HT,
paroxetine, off-label anti-depressants, anti-psychotic agents, and pain medications, including opioids.

## Key facts

- **NIH application ID:** 9907820
- **Project number:** 1R43AG066538-01
- **Recipient organization:** MENOGENIX, INC.
- **Principal Investigator:** James Woodruff Emlen
- **Activity code:** R43 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $299,831
- **Award type:** 1
- **Project period:** 2020-06-01 → 2022-05-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9907820

## Citation

> US National Institutes of Health, RePORTER application 9907820, Expanded Clinical Trial of Granulocyte Colony-Stimulating Factor (G-CSF) to Treat Hot Flush and Other Vasomotor Symptoms in Women with Naturally-Occurring and Surgically-Induced Menopause (1R43AG066538-01). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/9907820. Licensed CC0.

---

*[NIH grants dataset](/datasets/nih-grants) · CC0 1.0*