Past Events
Our thanks to Mo Rocca for his hilarious and informative interview, to Calvin Hughes of WPLG Miami for interviewing, to our Couple of the Year Robert & Mary Simses, to the Chief of Pediatric Sarcoma Service at MSKCC Dr. Damon Reed, and to our Man of the Year Dr. Paul Meyers.
And congratulations to our flower and raffle winners!
Tal Dror Cohen, MD, is a third year pediatric fellow at Memorial Sloan-Kettering Cancer Center in New York, and the newest participant in the Margaux’s Miracle Foundation Fellowship. Her research is focused on a gene therapy strategy to enhance the persistence of antitumor efficacy of human chimeric antigen receptor (CAR)-modified NK cells for childhood cancer.
Dr. Cohen shares her thoughts on the fellowship below:
Dear Margaux’s Miracle Foundation and the family of sweet Margaux Grossman,
I would like to express my sincerest gratitude to you for establishing the Margaux Grossman Fellowship at Memorial Sloan Kettering. In learning about Margaux from the foundation website I can see she was a truly special young woman that is continuing to leave a positive lasting impression on her loved ones and on other children battling cancer.
I feel humbled and honored to receive this title and hope that together we can achieve our common goal of making miracles happen for the children with cancer who need them the most.
Thank you so much
With love,
Tal Cohen
Read Dr. Cohen’s project description here.
Our thanks to Rita Rudner for her very funny look into her life as a parent and entertainer, to Calvin Hughes of WPLG Miami for hosting, Dr. Paul Meyers for his updates from our research fellowship, to friend of the foundation Samantha Eisenstein Watson, and to our Couple of the Year, Greg & Kiki Norman.
We loved laughing and shopping with you all.
Memorial Sloan-Kettering Cancer Center has published their Year in Review for 2022. It includes an important update on the genetics of pediatric cancers.
Children with cancer are not routinely screened for inherited genetic mutations associated with the disease, but a study led by pediatric oncologist and geneticist Michael Walsh and genetic counselor Elise Fiala may change that. The team sequenced healthy cells from more than 750 children treated at MSK Kids, our pediatric program, and found that 18% had a heritable mutation that predisposes them to developing cancer — a much higher number than expected. In many cases, the mutation found had no known link to the existing cancer. This research helps fill a gap in understanding the potential connections between inherited mutations and pediatric cancer, and may prompt families to take steps to reduce the risk of future cancers.
You can read more updates from MSKCC in their Year in Review PDF.
Melanie Degliuomini, MD, is a third-year pediatric fellow in the combined Memorial Sloan Kettering Cancer Center (MSK) and NYP-Weill Cornell Medicine (WCMC) program. Dr. Degliuomini’s primary clinical and research focus has been dedicated to studying the effects of cancer treatment on platelets within the pediatric oncology population.
Under the mentorship of Dr. Gerald Soff, a distinguished clinician and researcher specializing in hematologic disorders occurring in adult cancer patients, she has been analyzing large-scale MSK pediatric institutional data regarding the prevalence and impact of chemotherapy-induced thrombocytopenia (CIT) in both the solid tumor and hematologic malignancy patient populations.
In addition to this project, under the mentorship of both Dr. Michael Ortiz and Dr. Soff, she also serves as a lead clinical investigator on a Phase II clinical trial studying the efficacy of Romiplostim use in the prevention of CIT in pediatric solid tumor patients. She has been actively involved in the ongoing interim analyses pertaining to the trial and serving as the lead in creating the protocol addendum from such analyses.
Her current research and involvement in the open clinical trial aims to provide hematologic support in order to minimize the platelet transfusion burden and delays in care associated with such myelosuppressive chemotherapy. Her knowledge and experience in clinical research has been further enhanced by the Weill Cornell Medicine Master’s Program in Clinical and Translational Investigation, which she will be completing in June 2022. She has excelled in courses tailored to advance her knowledge in the foundations of clinical trials and protocol design.
Following fellowship, Dr. Degliuomini intends to utilize the skills acquired during her training to excel as both a clinician and researcher. From her experiences in fellowship, she has acquired invaluable career development skills arising from the breadth of clinical diagnoses seen, the protected research time provided to succeed as a clinical trialist, and the relationships formed with strong, dedicated mentors.
Read Dr. Degliuomini’s CV here.
We have known for many years that Ewing’s sarcoma arises when cell division goes wrong, creating a translocation which brings together a portion of chromosome 11 with a portion of chromosome 22. The resulting translocation creates a new sequence of DNA which instructs the cell to become malignant. This mechanism of translocation is the basic mechanism which leads to cancer for dozens of sarcomas.
For over two decades we have been trying to find a way to target this abnormal sequence of DNA to reverse the process which transforms normal cells into cancer cells. The DNA sequence is buried deep inside the cell nucleus and it has been very hard to find a way to attack it. Dr. Jeffrey Toretsky found a compound that worked in the test tube, and we began a clinical trial of the investigational compound TK-216.
We have now seen a patient with Ewing sarcoma whose disease had relapsed three times, for whom chemotherapy was not working, respond to this novel compound. This is the first time we have ever successfully targeted a chromosome translocation which is responsible for the emergence of cancer. Since we presented these results at the international meeting in November, 2019, we have seen three additional patients respond to this ground breaking therapy.
Margaux’s Miracle Supporter,
MSK is dedicated to the belief that everyone, no matter who they are or what neighborhood they live in, deserves the best cancer care available. However, this goal remains unrealized for many people with cancer in historically underserved communities. To accelerate change for every person with cancer, MSK has launched the Office of Health Equity (OHE), which I lead.
The OHE is committed to improving cancer health equity through three key pillars — clinical research, patient access, and training — setting a course to advance cancer health equity at MSK and worldwide.
One of the priorities of the OHE is to develop a knowledge base focused on clinical research dedicated to improving outcomes among people with cancer regardless of their race, ethnicity, culture, gender, gender identity, sexual orientation, age, or socioeconomic status.
As a surgeon and someone working to improve care for underserved communities, I am optimistic that MSK has the expertise and drive to make a real difference in this critical issue.
Help us improve health equity for every person with cancer.
Cancer disparities exist in many forms and affect various populations differently in terms of how commonly the disease occurs, survival rates, and the type of treatments that people receive. These systemic inequalities in health and cancer care must be addressed, and we cannot do it without your support.
Thank you,
Carol L. Brown, MD, FACS
Attending Surgeon
Senior Vice President and Chief Health Equity Officer
Tara O’Donohue is a Pediatric Hematology Oncology Fellow at Memorial Sloan Kettering Cancer Center where her primary clinical and research focus is the development of early phase clinical trials using genomically targeted therapies for relapsed and refractory pediatric cancers. Throughout her fellowship training, Dr. O’Donohue has gained experience in multiple facets of early phase trial development including: preclinical hypothesis testing of novel agents in vitro and in vivo in the laboratory, completing a Master’s degree in Clinical and Translational Investigation through the Cornell Clinical and Translational Science Center, and working with a drug sponsor to develop and open a first in pediatrics, international phase 1/2 clinical trial evaluating a novel ALK/ROS1/TRK inhibitor (repotrectinib) for pediatric patients with relapsed and refractory solid tumors. Dr. O’Donohue’s work in Dr. Andrew Kung’s laboratory also provided the preclinical rationale for an investigator-initiated clinical trial evaluating repotrectinib plus conventional chemotherapy for a subset of patients with relapsed and refractory solid tumors that may benefit from augmented therapy.
Read Dr. O’Donahue’s extraordinary CV here.
Dear Margaux’s Miracle Foundation,
I am honored to be the recipient of the Margaux Grossman Fellowship at MSK Kids. On behalf of myself as a clinical and translational investigator as well as on behalf of the rest of my colleagues working to improve outcomes in childhood cancers, we are extremely grateful for your continued support of this important work.
I am currently a fourth-year fellow and clinical instructor focusing on developmental therapeutics in relapsed and refractory childhood cancers. Cancers which occur in the pediatric population are a heterogeneous group of relatively uncommon malignancies with complex and understudied biologic characteristics, often resulting in a lack of effective therapeutic options. I currently work within the Pediatric Translational Medicine Program (PTMP) at MSK Kids which is comprised of a core group of basic, translational, and clinical researchers using state-of-the-art genomic profiling platforms to develop a portfolio of precision medicine-based therapies to improve outcomes for children and young adults with all types of cancer. Within the context of this role, I work closely with laboratory investigators in the Kung Lab to facilitate expeditious preclinical validation of novel therapies in pediatric models to inform rational clinical trial development.
Based on the results of our preclinical work evaluating a novel therapy, repotrectinib, a multi-kinase inhibitor with therapeutic potential in various pediatric sarcomas, neuroblastoma, lymphoma, and central nervous system malignancies, we have worked to develop two different clinical trials. An international, industry-sponsored, first in pediatrics phase 1/2 trial evaluating repotrectinib monotherapy in relapsed and refractory pediatric cancers is currently open and accruing. Furthermore, with support provided by the Swim Across America Young Investigator Award and the Kristen Ann Carr Fellowship, as well as the drug sponsor, Turning Point Therapeutics, we have developed an investigatorinitiated, single-institution phase 1/2 trial combining repotrectinib with irinotecan and temozolomide in pediatric patients with relapsed and refractory pediatric solid tumors. This trial has been submitted to our internal institutional review board, and we anticipate opening to enrollment in the coming months. We are very excited about the therapeutic potential of repotrectinib (alone or in combination with chemotherapy) across a diverse array of pediatric malignancies based on a strong biologic rationale, promising results of preclinical studies in pediatric cancer models, as well as an acceptable safety profile when evaluated in adult patients.
On behalf of the MSK Kids community, and most importantly, our patients and their families, we wanted to extend a heart-felt thank you for your tremendous showing of support for our research program and fellows.
Sincerely,
Tara O’Donohue, MD, MS
Many thanks to Jon Meacham for his hilarious and poignant look into American politics, to Calvin Hughes of WPLG Miami for his heartfelt introduction, Dr. Paul Meyers for his hopeful updates from our research fellowship, and to our Woman of the Year, Debbi Stark.
Whenever you visit smile.amazon.com/ch/04-3643503 and make any Amazon purchase, AmazonSmile donates a portion of your purchase to Margaux's Miracle Foundation.
Consider shopping with AmazonSmile today.
