Signatures Precision Medicine Program

“This is what we want for every patient. We shouldn’t just be waiting until a child is told they are incurable to investigate the best ways to personalize treatment.”Dr Giselle Sholler

The launch of a new clinical trial called PEDS-PLAN in September of 2015 marks a paradigm shift in childhood cancer treatment and precision medicine — the first time DNA and RNA sequencing are being done in newly diagnosed patients.

Personalized medicine… precision medicine… molecularly guided therapy (MGT)… genomics. They’re different terms for what is generally the same concept — personalizing treatment per patient based on specific genetic or molecular analysis. In childhood cancer, genomics is being used to beat cancer on a kid by kid basis.

Abstracts and Publications

Precision Medicine Trials

NMTRC012

Pediatric Precision Laboratory Advanced Neuroblastoma Therapy (PEDS-PLAN)
A prospective open label, multicenter study to evaluate the feasibility and acute toxicity of using molecularly guided therapy in combination with standard therapy followed by maintenance therapy with DFMO in subjects with newly diagnosed high risk neuroblastoma.
OPEN

NMTRC009

Molecular-Guided Therapy for Childhood Cancer
Molecular-guided therapy for the treatment of patients with relapsed and refractory childhood cancers
CLOSED

NMTRC001

Molecular Guided Therapy for Refractory or Recurrent Neuroblastoma
A Feasibility Trial Using Molecular-Guided Therapy for the Treatment of Patients with Refractory or Recurrent Neuroblastoma
CLOSED

NMTRC008

Molecular-Guided Therapy for Relapsed and Refractory Childhood Cancer
A Feasibility Trial using Molecular-Guided Therapy for the Treatment of Patients with Relapsed and Refractory Childhood Cancer
CLOSED

A Trial Using Molecular-Guided Therapy in Patients With Refractory or Recurrent Neuroblastoma

A Pilot Trial Using Molecular-Guided Therapy in Patients With Refractory or Recurrent Neuroblastoma
OPEN

History: Our Work with Precision Medicine

The image below is from a detailed blog post written at beatnb.org.