Accelerating a Treatment for Children with DIPG

The Musella Foundation has partnered with FLAG Therapeutics to accelerate development of a promising new therapy called FLAG-003 for children with Diffuse Intrinsic Pontine Glioma (DIPG).

DIPG is a rare and aggressive tumor located in the brainstem. It affects children typically between ages 3 and 12 and accounts for approximately 10-15% of pediatric brain tumors. About 150-300 children in the United States are diagnosed each year.

Despite decades of research, there are currently no curative treatments. Radiation therapy may temporarily slow tumor growth, but median survival remains less than one year.

A Promising New Therapy

FLAG-003 is an innovative small-molecule therapy designed specifically for aggressive brain tumors like DIPG.

In preclinical studies the drug has demonstrated several characteristics researchers believe are critical for treating this disease:

• Ability to cross the blood-brain barrier
• Targeting of multiple tumor survival pathways
• Selective killing of cancer cells
• Activity against drug-resistant tumor cells

How FLAG-003 Works

FLAG-003 combines two powerful mechanisms in one molecule:

• Anti-angiogenic activity that blocks tumor blood supply
• Tubulin inhibition that prevents cancer cells from dividing

By attacking tumor cells through multiple mechanisms at once, FLAG-003 is designed to overwhelm the tumor while limiting toxicity to normal brain tissue.

Encouraging Early Results

Preclinical studies conducted in collaboration with major research centers including the Mayo Clinic, Dana-Farber Cancer Institute, and Duke University have shown strong anti-cancer activity. In laboratory (cancer cell lines) and animal models of both DIPG and glioblastoma (GBM), the therapy demonstrated significant anti-tumor activity and improved survival.

Funding Goal: $2.25 Million

• Raised: $320,735.00
• Goal: $2,250,000
• Remaining: $1,929,265.00

How Donations Help

Funds raised through the FLAG-Musella Accelerator Fund will be used to:

• File the Investigational New Drug (IND) application with the FDA
• Produce clinical trial drug supply
• Help launch a Phase I/II clinical trial in children with DIPG

As donations are received, these steps can move forward immediately. With an approved IND and clinical-grade drug supply available, this therapy could move rapidly into clinical trials for children who desperately need new treatment options.

Please donate using the form on the right. You may also send a check made out to "Musella Foundation" with "FLAG-Musella DIPG Fund" in the memo and mail it to:

Musella Foundation
1100 Peninsula Blvd
Hewlett, NY 11557

Call us at 888-295-4740 if you have questions. Donations are tax deductible to the full extent of the law.