Functional Precision Medicine Emergency Grant (Dr. Maggie Fader, Nicklaus Children's)

Functional Precision Medicine Emergency Grant

Recipient: Dr. Maggie Fader, Nicklaus Children's Hospital
Trial Registry: NCT05857969

Purpose

Emergency funding to keep active clinical trial open after primary funding source was lost. Trial serves patients who have exhausted standard treatment options.

Focus Areas

Personalized Tumor Testing & Treatment:

• Uses molecular profiling and functional testing to guide treatment in heavily pre-treated patients
• Patients have typically relapsed multiple times and conventional options exhausted

What Makes This Trial Special

Functional Approach (not just sequencing):

• Does not rely on DNA analysis alone
• Tests LIVE tumor cells in vitro against 120+ drugs
• Includes off-label agents (e.g., montelukast, antibiotics)
• Identifies which drugs actually kill that specific child's cancer

Speed:

• Results delivered in 10-14 days from biopsy

Flexibility:

• Open to children nationwide
• Results provide options and ideas rather than rigid trial guidelines
• Allows treatment guidance versus mandatory drug combinations

Actionable Outcomes:

• Provides data for informed treatment decisions
• While not all drugs may be insurance-covered, data informs oncologists and families

Clinical Impact Example

Patient Case - Sasha:

• Child with multiple relapses referred to hospice
• Tumor biopsied and tested through trial
• Belinostat identified as top-ranked therapy alongside Lurbinectedin
• Added to high-dose ifosfamide infusion
• Remarkable response: tumors flattened rapidly including skull metastases
• Results now guide all future chemotherapy blocks

Related Programs

Other precision medicine initiatives mentioned:

• BCC's In:Formation
• MSK/Columbia's OncoTreat and OncoTarget

References

• Clinical Trial: NCT05857969
• Pilot study published in Nature Medicine