I recently completed my BS and MS in Biomedical Engineering at Northwestern University, where I worked as a student researcher for two years. My research focused on developing drug delivery systems for infectious diseases and women’s health. I worked on projects in formulation science as well as manufacturing and design engineering, in order to produce medical devices for long-acting protection against HIV.
After completing a practicum in technology commercialization consulting, where the main goal was to assess the potential profit of repurposing a drug for a rare disease, I realized I wanted to work in research. Rather than work for a company with the goal of maximizing profit, I wanted to work for a company with the goal of helping those patients that need it most. I preferred to spend my time discovering new solutions to problems driven by clinical need, rather than analyze existing options driven by profit.
Last year, I joined cc-TDI as a Biomedical Engineering Fellow and have been investigating pediatric brain tumors using synthetic model systems that more accurately represent human physiology. I hope, through my work with cc-TDI, to discover novel targets and therapies for patients with rare pediatric brain tumors.
Veterinarian by choice, expert Cancer mom by necessity. Our daughter, Carlin, was diagnosed with ETMR when she was two and a half. Until a seizure prompted an ER visit and subsequent MRI revealed a brain tumor, we had no idea Carlin was anything other than a bright, normal toddler. Our world was turned on end after her tumor resection surgery when the pathology came back as ETMR (previously called ETANTR), and we learned that she was facing dismal odds for survival. Carlin immediately began six months of the most hellish chemotherapy imaginable followed by six weeks of focal proton radiation and another year of maintenance chemo. Carlin fought through it all with sass and a smile. She was and continues to be an extraordinary tiny human. To date, she has shown no sign of recurrence with routine MRI monitoring. We are fortunate to report that she is turning six years old next week, and by all current measures is a mischievous, over-achieving, Star Wars-loving kindergartener with a smile that will light up a room.
Cancer, however, remains a dark cloud that still follows our family as we fear relapse, secondary cancers, developmental issues, and other organ damage. And we are keenly aware that Carlin is an anomaly. There are only a handful of other ETMR survivors that I am aware of. The vast majority of these children die within a year of diagnosis. My inspiration for getting involved in this research project is to raise awareness and seek a cure for a rare cancer that responds very poorly to available therapies. ETMR research deserves attention, and I'm excited to be able to partner with CC-tdi to advocate for research to help children and families affected by this disease.
The origin of "TutuTough" came about when Carlin was inpatient and chose a tutu from the hospital gift shop, which led to in-room dance parties when she felt able. The tutu became a symbol and TutuTough a slogan that Carlin embraced and our family drew strength from. Our family sported tutus and our support team posted pictures of themselves in tutus. More incredibly, people took photos of their pets (all species - dogs, cats, cows, horses) wearing tutus which helped brighten some very dark days for us. The tenacity of that tiny, bald, brown-eyed, tutu-wearing girl helped inspire and educate friends, family and strangers to the reality that is pediatric cancer. You never think you could be "that" family, until in a heartbeat of a pathology report, you are. Through the TutuTough Project, we hope to make a cure for ETMR a reality.
ETMR (previously ETANTR) is an extremely rare and aggressive pediatric brain tumor. It strikes young children, most commonly less than 4 years old. ETMR has a dismal prognosis, responding poorly to traditional chemotherapy and radiation treatments. Little is known about the genetic origins of the disease and treatment options are limited. Our research project aims to change that. We hope to advance understanding of the tumor by studying multiple ETMR cell lines and testing potential chemotherapy drugs that will specifically target ETMR growth pathways.
The Children’s Cancer Therapy Development Institute (cc-TDI)’s mission is to make ALL childhood cancers universally survivable by delivering new treatments to clinical trials through scientific discovery. Because so little is known about ETMR, we start by creating a roadmap for a cure that includes creating a registry for ETMR patients and families, generating a shareable central resource (biorepository) for cell lines and mouse models, and discovering new therapeutics (drugs) to treat ETMR. The first and most critical step is to develop new cell lines and mouse models that will provide the basis for drug screening and testing.
To do this, we will recruit 10 fresh ETMR samples for making primary cultures, doing drug screening, and preparing tissue for future avatar mouse making.
Why is this important?
ETMR is almost always fatal and treatment options are limited and frequently ineffective. We must dramatically accelerate the discovery of treatment options for this brain tumor by advancing novel drug and drug combinations to clinical trial to make ETMR universally survivable.
Who will benefit?
Children and families touched by ETMR will benefit, and we hope in a tangible period of time. Thus far there are fewer than 3 cell lines or mouse models for this rare, orphan brain tumor of childhood. Discovery starts with having enough cell lines and animal models as tools. Sharable tools. This project is building that foundation.
BudgetThis project aims to create a broader and more widely available set of primary cultures for ETMR, a foundation necessary to develop treatment options, in part by recruiting additional tissue samples. In addition to the scientific work (RNA/DNA sequencing, drug screening), the project also seeks to empower families by educating them about the current state of disease understanding and treatment options in a ’nano course’ setting, where they get to engage with the foremost experts in the field.
We are growing BT-183 from Jen Chan lab... thank you, Jen! Next steps are STR fingerprinting, DNA exome sequencing and RNA deep sequencing. Then we'll pursue chemical screens, and apply Noah's computational approach to predicting drug combinations.
The explanted PDX tumor from Champions Oncology (thank you Chris Noakes!) eventually senesced ... we will re-attempt the culture using different conditions soon.