Embedded Files
Our laboratory links together basic biophysical phenomena to create new drugs for pediatric cancer. It's a creative, multidisciplinary challenge.
-Jeffrey Toretsky, MD
APRIL, 2025
Toretsky Lab Earns "Image of the Month" Recognition
The image titled "Nuclear Speckle Protein SRRM2 Dissolves During Anaphase in Ewing Sarcoma Cells" has been selected as the “Image of the Month” by Georgetown’s Institute for Soft Matter Synthesis and Metrology (I(SM)²). Olivia Oakley was responsible for the staining and imaging, David Allegakoen designed the CRISPR model, and Emma McGuire conducted the subsequent live-cell imaging.
The high-resolution imaging was captured using a Nikon spinning disk SoRa microscope. The lab continues to refine its microscopy techniques, as recent advances in oncology research highlight the potential of visualizing proteins to uncover new therapeutic opportunities.
Click here to read the full article
OCTOBER, 2024
Dr. Jeffrey Toretsky, MD, and Dr. Emre Deniz, PhD. Participated in the Annual Bellringer Fundraiser 50 Mile Bike Ride
Dr. Jeffrey Toretsky, MD, and Dr. Emre Deniz, PhD. Participated in the Annual Bellringer Fundraiser 50 Mile Bike Ride
Dr. Toretsky and Dr. Deniz geared up to ride to raise awareness and funds to fight cancer. PhD. candidate and Taryn Shaw also volunteered along the route to cheer on riders and provide support.
At least 1,166 Riders — over 200 more than last year
70 Virtual Riders
391 Volunteers
419 Student Riders
151 Teams
$4.28 million raised since 2022 (and counting — money for this year’s ride will roll in through December 31). Bellringer.org
SEPTEMBER, 2024
Georgetown Lombardi’s Jeffrey Toretsky, MD, Honored with Hyundai Hope on Wheels Endowed Chair in Pediatric Oncology
Georgetown Lombardi’s Jeffrey Toretsky, MD, Honored with Hyundai Hope on Wheels Endowed Chair in Pediatric Oncology
Hyundai Hope On Wheels established the Endowed Chair of Pediatric Oncology grant, a $3 million commitment which will ensure that future generations of researchers continue into perpetuity at leading medical institutions. The inaugural grant was awarded to Georgetown University's Lombardi Comprehensive Cancer Center and Dr. Jeffrey A. Toretsky, MD. Click here to read the full story
SEPTEMBER, 2024
Welcome Quinn Tufino to the Lab!
Welcome Quinn Tufino to the Lab!
The Toretsky Lab is pleased to announce our newest member to the team, Quinn Tufino. Quinn joins the lab from Ithaca College, Ithaca, NY, matriculating with Honors and a Biochemistry B.S., and Neuroscience Minor. Welcome Quinn!
SEPTEMBER, 2024
Dr.'s Toretsky and Üren attend AACR Special Conference in Cancer Research: Advances in Pediatric Cancer Research
Dr.'s Toretsky and Üren attend AACR Special Conference in Cancer Research: Advances in Pediatric Cancer Research
Dr. Toretsky and Dr. Üren attended the AACR Special Conference in Cancer Research: Advances in Pediatric Cancer Research hosted in Toronto, Ontario, Canada, September 5-8, 2024. Joining conference attendees at the forefront of pediatric cancer research, the this years program was developed by conference co-chairs Alejandro Gutierrez, Cynthia E. Hawkins, Andrea A. Hayes, and Gilles Vassal. With presentations on cancer predisposition, immunotherapy and immunology, precision medicine, novel early relapse detection tools, pediatric cancer disparities, emerging therapies, and new strategies for early-phase clinical trials, this conference provides a comprehensive exploration of the latest advancements.
Dr. Üren presented a well received poster focused on some of the labs most recent work lead by MD/PhD. candidate, Anna Molotokova, ROME, a novel membrane protein in vertebrates, enhances metastatic phenotype of Ewing sarcoma cells.
AUGUST, 2024
GUMC Talk Spotlights Biological Novelty: Liquid-Liquid Phase Separation
GUMC Talk Spotlights Biological Novelty: Liquid-Liquid Phase Separation
Thom Leiding, CEO and founder of Labbot, a biotechnology company, conducted a workshop on liquid-liquid phase separation (LLPS), a phenomenon thought to be the driving force for a myriad of diseases, at a Georgetown University Medical Center (GUMC) event titled “Continuous Tracking of Phase Separation and Aggregation” on Aug. 29, 2024.
LLPS refers to when a homogenous mixture spontaneously forms distinct layers, much like when oil separates from water. Scientists believe that LLPS could be occurring at a microscopic level within cells, making the cell much more dynamic than previously thought. Click here to read the full feature from The Hoya
AUGUST, 2024
Million Mile 2024 Fundraiser Campaign Kick Off at Pupatella Pizza, Arlington, VA.
Million Mile 2024 Fundraiser Campaign Kick Off at Pupatella Pizza, Arlington, VA.
Current and former members of the Toretsky and Üren research labs gathered together on Wednesday, August 28, 2024 at Pupatella Pizzeria in Arlington, Va to honor Nathan and kick off the Million Mile 2024 Fundraiser Campaign.
In 2023, Nathan's Cancer Slayers made an unbelievable impact. As a team we surpassed our fundraising goals twice over for the fourth year in a row, raising more than $144,000! Since launching Nathan's team in May 2020, we've raised an unbelievabable $448, 542 to fight childhood cancer in Nathan's honor. We are so proud of the amazing work we've accomplished, and are ready to do it again this year and continue on in Nathan's memory. We hope you'll join Nathan's team for the ALSF Million Mile challenge this September to raise awareness and critical funds to help kids defeat cancer. Just click "Join the Team" to sign up and to read Nathan's full hero story, plus an amazing video!
Thank you to Michele, Michael, and Spencer Fleming for your dedication and support!
JULY, 2024
Clinical Trial of TK216 Published in the Journal of Clinical Oncology
Clinical Trial of TK216 Published in the Journal of Clinical Oncology
Twenty-four years after Drs. Toretsky and Üren conceived of an approach to inhibit EWS::FLI1 (note newest nomenclature), the clinical trial of TK216 was published in the Journal of Clinical Oncology, the flagship journal of the American Society of Clinical Oncology. This trial had a number of exceptional responders that was remarkable for a first-in-class, first-in-human drug. Future developments, including an oral formulation are pending.
Click here to read the paper in full
JUNE, 2024
Lab Summer Picnic 2024
Lab Summer Picnic 2024
Lab members gathered at Fletchers Boathouse Cove for our annual Summer Lab Picnic! A wonderful spread of dishes were prepared by lab members who were joined by family and friends. A rousing Viking Chess tournament completed the days festivities with a winning throw from our youngest honorary lab member, Deeksha Selvanathan (daughter of Saravana Selvanathan. Thank you to everyone for your participation and amazing food and Special Thanks to Dr. Toretsky and Dr. Üren for hosting the event!
Lab members gathered at Fletchers Boathouse Cove for our annual Summer Lab Picnic! A wonderful spread of dishes were prepared by lab members who were joined by family and friends. A rousing Viking Chess tournament completed the days festivities with a winning throw from our youngest honorary lab member, Deeksha Selvanathan (daughter of Saravana Selvanathan. Thank you to everyone for your participation and amazing food and Special Thanks to Dr. Toretsky and Dr. Üren for hosting the event!
MAY, 2024
Ashley Gaffey Accepted to Doctoral Program
Ashley Gaffey Accepted to Doctoral Program
Congratulations to our Research Technician Ashley Gaffey on her acceptance to the University of Florida Graduate Program in Biomedical Sciences. Thank You for your hard work in the lab and at the bench. Best wishes and success in your new adventures!
Congratulations to our Research Technician Ashley Gaffey on her acceptance to the University of Florida Graduate Program in Biomedical Sciences. Thank You for your hard work in the lab and at the bench. Best wishes and success in your new adventures!
APRIL, 2024
Lab Improvements
Lab Improvements
The lab was temporally shut down for one week for improvements to our ventilation and fire alarm systems. Several researchers were asked to clear benches and several other preparations for the work. Thermostats were installed in offices and key areas of the lab as well as motion sensors which will help to ensure more stable working environments. Thank you to everyone for your assistance. Below you can find a few photos from the lab during the upgrade.
JANUARY, 2024
2024 New Year Social at Comet Ping Pong
2024 New Year Social at Comet Ping Pong
Members of the lab gathered after work for an evening of Pizza and Ping Pong at Comet Ping Pong in Washington, DC. Taking over the private event space with an independent and original underwater film from Dr. Toretsky and smooth moves on the court by Dr. Üren and several members demonstrated their keen hand eye coordinations. A buffet of never ending pizzas and salads were served up with a few tasty beverages. Thank you to all who could attend and Thank You to Dr. Toretsky and Dr. Üren for a wonderful evening.
NOVEMBER, 2023
Dr. Toretsky Presents at the University of Delaware
Dr. Toretsky Presents at the University of Delaware
Dr. Toretsky accepted the opportunity to present a lecture for the Graduate Student Seminar Series at the University of Delaware Department of Medical and Molecular Sciences. From Napkin to Clinical Trial: Creating a Drug for Ewing Sarcoma and Beyond discussed the rationale, discovery, and clinical trial of the first small molecule to directly target a chimeric fusion oncoprotein from ideal to clinical trial.
Dr. Toretsky accepted the opportunity to present a lecture for the Graduate Student Seminar Series at the University of Delaware Department of Medical and Molecular Sciences. From Napkin to Clinical Trial: Creating a Drug for Ewing Sarcoma and Beyond discussed the rationale, discovery, and clinical trial of the first small molecule to directly target a chimeric fusion oncoprotein from ideal to clinical trial.
OCTOBER, 2023
David Allegakoen Presents at FusOnC2
David Allegakoen Presents at FusOnC2
David Allegakoen presented a Lightening Talk during the FusOnC2 Meeting, on October 26, 2023. The presentation, A splicing reporter for screening vital splicing proteins in Ewing sarcoma, outlined current progress in generating a comprehensive EwS and hMSC transcriptome, incorporating both short and long read RNAseq data during assembly.
David Allegakoen presented a Lightening Talk during the FusOnC2 Meeting, on October 26, 2023. The presentation, A splicing reporter for screening vital splicing proteins in Ewing sarcoma, outlined current progress in generating a comprehensive EwS and hMSC transcriptome, incorporating both short and long read RNAseq data during assembly.
OCTOBER, 2023
Day Hike on the Billy Goat Trail
Day Hike on the Billy Goat Trail
Members of the lab enjoyed taking a break from the lab to enjoy a fine fall day of hiking and apicnic lunch along the Trip Billy Goat Trail.
Members of the lab enjoyed taking a break from the lab to enjoy a fine fall day of hiking and apicnic lunch along the Trip Billy Goat Trail.
SEPTEMBER, 2023
Emma McGuire and Olivia Oakley Present at Georgetown ISM Day
Emma McGuire and Olivia Oakley Present at Georgetown ISM Day
Emma McGuire and Olivia Oakley represented the Toretsky Lab while presenting posters at the Institute for Soft Matter Synthesis and Metrology, ISM Day, September 21, 2023, at Georgetown Main Campus.
Emma McGuire and Olivia Oakley represented the Toretsky Lab while presenting posters at the Institute for Soft Matter Synthesis and Metrology, ISM Day, September 21, 2023, at Georgetown Main Campus.
Emma McGuire – Evaluating ETS activity and effect of treatment in transfected cells using TK-216 compound shown to bind directly with transcription factors.
Emma McGuire – Evaluating ETS activity and effect of treatment in transfected cells using TK-216 compound shown to bind directly with transcription factors.
Olivia Oakley – Visualizing the Effect of (S)-YK-4-279 on EWS::FLI1 and its Protein Partners.
Olivia Oakley – Visualizing the Effect of (S)-YK-4-279 on EWS::FLI1 and its Protein Partners.
Georgetown’s Institute for Soft Matter Synthesis and Metrology, or I(SM)2, launched in 2011 as the result of $6.9 Million construction grant from NIST, serves to catalyze the development of fundamental principles and practical measurement tools that can be applied to soft matter synthesis and precision measurement and characterization (metrology). The Institute comprises faculty primarily from the Departments of Physics and Chemistry, Biology, as well as Oncology in the Medical School.
AUGUST, 2023
Toretsky and Uren Awarded $50,000 Research Grant at Mubadala Citi DC Open
Toretsky and Uren Awarded $50,000 Research Grant at Mubadala Citi DC Open
Together with Hyundai Hope on Wheels, local Hyundai dealerships donated $50,000.00 to Pediatric Cancer Research! Dr. Toretsky and Dr. Uren were brought to Center Court to accept the grant on behalf of Georgetown University and the Lombardi Comprehensive Cancer Center. Thank you to Hyundai Hope on Wheels, Kevin Reilly and Alexandria Hyundai!
Together with Hyundai Hope on Wheels, local Hyundai dealerships donated $50,000.00 to Pediatric Cancer Research! Dr. Toretsky and Dr. Uren were brought to Center Court to accept the grant on behalf of Georgetown University and the Lombardi Comprehensive Cancer Center. Thank you to Hyundai Hope on Wheels, Kevin Reilly and Alexandria Hyundai!
AUGUST, 2023
Million Mile 2023 Fundraiser Campaign Kick Off
Million Mile 2023 Fundraiser Campaign Kick Off
Current and former members of the Toretsky and Uren research labs gathered together on Wednesday, August 31st 2023 at Pupatella Pizzeria in Arlington, Va to honor Nathan and kick off the Million Mile 2023 Fundraiser Campaign.
In 2022, Nathan's Cancer Slayers made an unbelievable impact. Surpassing fundraising goals twice over for the third year in a row and raising nearly $122,000! Since launching Nathan's team in May 2020, we've raised an unbelievable $303,286 to fight childhood cancer in Nathan's honor. We are so proud of the amazing work we've accomplished, and are ready to do it again this year and continue on in Nathan's memory.
For more information and or to make a contribution please visit Alex’s Lemonade Stand Foundation
JUNE, 2023
7th Annual CCF Research Symposium
7th Annual CCF Research Symposium
Lab members participated and presented posters at the 7th Annual CCF Research Symposium on June 14th, 2023. The Symposium, Finding Cures through Innovative Research in our Community included a keynote presentation by Patrick J. Grohar, M.D. Ph.D., Director of Translational Research in the Center for Childhood Cancer Research, Children’s Hospital of Philadelphia
Lab members participated and presented posters at the 7th Annual CCF Research Symposium on June 14th, 2023. The Symposium, Finding Cures through Innovative Research in our Community included a keynote presentation by Patrick J. Grohar, M.D. Ph.D., Director of Translational Research in the Center for Childhood Cancer Research, Children’s Hospital of Philadelphia
For more information and video presentations please click below.
JULY, 2023
David Allegakoen appointed T32 recipient for the 2023-2024 Academic Year
David Allegakoen appointed T32 recipient for the 2023-2024 Academic Year
We are excited to announce the return of David Allegakoen to the Toretsky Lab as a Doctoral Student candidate in the Tumor Biology program. In addition, David has been appointed a T32 fellowship for the 2023-2024 academic year.
David's previous tenure in the lab includes success as a Masters Student and Research Assistant under the guidance of Dr. Aykut Uren.
In addition to tuition and stipend support, T32 trainees are eligible to receive up to $800 in travel support to scientific conferences and $4200 for other training related expenses. Congratulations David!
MAY, 2023
Summer Picnic at Rock Creek Park
Summer Picnic at Rock Creek Park
Lab members gathered at Rock Creek Park for an annual Lab Picnic! A wonderful spread of dishes were prepared by lab members who were joined by family and canine friends. A rousing Viking Chess tournament completed the days festivities. Thank you for your participation and special thanks to Dr. Toretsky and Dr. Uren for hosting the event!
Lab members gathered at Rock Creek Park for an annual Lab Picnic! A wonderful spread of dishes were prepared by lab members who were joined by family and canine friends. A rousing Viking Chess tournament completed the days festivities. Thank you for your participation and special thanks to Dr. Toretsky and Dr. Uren for hosting the event!
MARCH, 2023
Saté Indonesian Food Truck Lunch
Saté Indonesian Food Truck Lunch
The lab gathered for lunch and enjoyed selections from the local Indonesian Food Truck Lunch, Saté. Inspired by Dr. Toresky's time in Indonesia during his recent sabbatical. Dr. Toretsky shared details of his travels and visiting Dr. Sardjito General Hospital in Yogyakarta, Indonesia, as a visiting physician and to present a talk on his experience as a clinical research physician.
The lab gathered for lunch and enjoyed selections from the local Indonesian Food Truck Lunch, Saté. Inspired by Dr. Toresky's time in Indonesia during his recent sabbatical. Dr. Toretsky shared details of his travels and visiting Dr. Sardjito General Hospital in Yogyakarta, Indonesia, as a visiting physician and to present a talk on his experience as a clinical research physician.
FEBRUARY, 2023
Lab Member Draws Winning Key for a 2023 IONIC5 at Washington Auto Show
Lab Member Draws Winning Key for a 2023 IONIC5 at Washington Auto Show
Team Georgetown Lombardi, which included Jeff Petro, Emre Deniz and Alan Zwart, won this year’s Hyundai Hands on Hope contest at the Washington, DC Auto Show.
The trio secured a $60,000 pediatric cancer research grant for Georgetown Lombardi from the Washington Area Hyundai Dealers when Jeff Petro picked the key that started the contest’s Hyundai IONIQ5 SE (which he will take home). All three had successfully remained in contact with the car for 44 hours.
Runner-up Children’s National received a $40,000 research grant.
Our thanks to the generosity of the Washington Area Hyundai Dealers and Kevin Reilly, owner of Alexandria Hyundai.
More information on the 2023 contest may be found here 2023 Hands on Hope Contest and additional information about the Hyundai Hope on Wheels Foundation
JANUARY, 2023
Research Grant Award: Hyundai Grant
Research Grant Award: Hyundai Grant
The Toretsky Lab has received a $300,000.00 Research Grant from the Hyundai Hope on Wheels Foundation for the proposal, Dissecting the role that therapeutic targeting of mRNA splicing in Ewing sarcoma. The award period will span from January 2023 - December 2024.
The Toretsky Lab has received a $300,000.00 Research Grant from the Hyundai Hope on Wheels Foundation for the proposal, Dissecting the role that therapeutic targeting of mRNA splicing in Ewing sarcoma. The award period will span from January 2023 - December 2024.
AUGUST, 2022
The Toretsky Lab welcomes Emma McGuire, who joins the lab as a Research Assistant. Welcome Emma!
The Toretsky Lab welcomes Emma McGuire, who joins the lab as a Research Assistant. Welcome Emma!
JUNE 2022
The Toretsky Lab welcomes Ashley Gaffey, who joins the lab as a Research Assistant. Welcome Ashley!
The Toretsky Lab welcomes Ashley Gaffey, who joins the lab as a Research Assistant. Welcome Ashley!
JUNE 2022
Lab members attended and presented posters at the 6th Annual CCF Research Symposium held on June 8th.
Lab members attended and presented posters at the 6th Annual CCF Research Symposium held on June 8th.
MAY, 2022
Dr. Toretsky and Dr. Uren attended the AACR Special Conference on Sarcomas in Montreal, QC, Canada.
Dr. Toretsky and Dr. Uren attended the AACR Special Conference on Sarcomas in Montreal, QC, Canada.
FEBRUARY, 2022
The Toretsky Lab completed the installation of a new SpectraMax® iD3 multi-mode plate reader capable of measuring absorbance, fluorescence, and luminescence.
The Toretsky Lab completed the installation of a new SpectraMax® iD3 multi-mode plate reader capable of measuring absorbance, fluorescence, and luminescence.
JANUARY, 2022
Dr. Toretsky visited Dr. Sardjito General Hospital in Yogyakarta, Indonesia, as a visiting physician and to present a talk on his experience as a clinical research physician.
Dr. Toretsky visited Dr. Sardjito General Hospital in Yogyakarta, Indonesia, as a visiting physician and to present a talk on his experience as a clinical research physician.
DECEMBER 10, 2021
Dr. Toretsky was invited to give a talk at the Bone Cancer Research Trust for their James Ewing Century Meeting. His talk, titled "From James Ewing to a Clinical Trial: 100 years to improve survival for Ewing sarcoma patients," was given via video conference to the group in Essen, Germany.
Dr. Toretsky was invited to give a talk at the Bone Cancer Research Trust for their James Ewing Century Meeting. His talk, titled "From James Ewing to a Clinical Trial: 100 years to improve survival for Ewing sarcoma patients," was given via video conference to the group in Essen, Germany.
NOVEMBER 5, 2021
Dr. Toretsky was invited to give a keynote talk at the Australia-New Zealand Sarcoma Association. His talk, titled "From Napkin to Clinical Trial: a 20-year Quest to Improve Survival for Ewing Sarcoma Patients," was given via video conference to the group in Melbourne, Australia.
Dr. Toretsky was invited to give a keynote talk at the Australia-New Zealand Sarcoma Association. His talk, titled "From Napkin to Clinical Trial: a 20-year Quest to Improve Survival for Ewing Sarcoma Patients," was given via video conference to the group in Melbourne, Australia.
JUNE, 2021
The Toretsky Lab welcomes Olivia Oakley, who joins the lab as a Research Assistant. Welcome Olivia!
The Toretsky Lab welcomes Olivia Oakley, who joins the lab as a Research Assistant. Welcome Olivia!
MAY, 2021
Research Assistant Olivia Blumberg was named the St Baldrick's Research Fellow at Georgetown, which supported her project in the lab over the Summer of 2021. Congratulations, Olivia!
Research Assistant Olivia Blumberg was named the St Baldrick's Research Fellow at Georgetown, which supported her project in the lab over the Summer of 2021. Congratulations, Olivia!
OCTOBER, 2021
Garrett Graham graduated from Georgetown University with his PhD in Tumor Biology - his thesis titled "Cassette Exon RNA Splicing Altered in Ewing Sarcoma Is Characterized by Higher Guanine-Cytosine Content and Lower Splice Site Strength Indicative of Intron-Definition Splicing." Congratulations, Garrett!
Garrett Graham graduated from Georgetown University with his PhD in Tumor Biology - his thesis titled "Cassette Exon RNA Splicing Altered in Ewing Sarcoma Is Characterized by Higher Guanine-Cytosine Content and Lower Splice Site Strength Indicative of Intron-Definition Splicing." Congratulations, Garrett!
OCTOBER 16, 2020
Dr. Toretsky was invited to give a keynote talk at the Bone Cancer Research Trust Conference in London, UK via video conference. His talk was titled "From Napkin to Clinical Trial: Creating a Drug for Ewing Sarcoma."
Dr. Toretsky was invited to give a keynote talk at the Bone Cancer Research Trust Conference in London, UK via video conference. His talk was titled "From Napkin to Clinical Trial: Creating a Drug for Ewing Sarcoma."
JULY, 2020
R01 Funding Award! Dr. Toretsky in collaboration with researchers at The Hospital for Sick Children in Toronto, Canada have secured R01 funding with the grant Dissection of EWS-FLI1 oncogenic mechanisms and small molecule targeting. The award period will span from July 2020 - June 2025.
R01 Funding Award! Dr. Toretsky in collaboration with researchers at The Hospital for Sick Children in Toronto, Canada have secured R01 funding with the grant Dissection of EWS-FLI1 oncogenic mechanisms and small molecule targeting. The award period will span from July 2020 - June 2025.
JUNE, 2020
Jeff Petro was promoted to the position of Lab Manager! Congratulations, Jeff!
Jeff Petro was promoted to the position of Lab Manager! Congratulations, Jeff!
MAY, 2020
Lab member Jessica Haladyna graduated from the Georgetown University School of Medicine, and is now completing her Pediatric Residency Training at the Children's Hospital of Los Angeles (CHLA Class of 2023). Congratulations, Jessica!
Lab member Jessica Haladyna graduated from the Georgetown University School of Medicine, and is now completing her Pediatric Residency Training at the Children's Hospital of Los Angeles (CHLA Class of 2023). Congratulations, Jessica!
DECEMBER , 2019
Opening of Phase 1b Expansion Cohort of Clinical Trial of TK216
Opening of Phase 1b Expansion Cohort of Clinical Trial of TK216
SAN DIEGO--(BUSINESS WIRE)--Dec. 11, 2019-- Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, today announced that it has opened for enrollment a Phase 1b expansion cohort of its ongoing clinical trial evaluating TK216, a first-in-class, targeted, investigational small-molecule inhibitor of the E26 transformation-specific (ETS) family of oncoproteins, in patients with relapsed or refractory Ewing sarcoma.
The decision to open an expansion cohort was based on favorable interim results from the dose-finding cohort of the trial, including a deep and sustained clinical response reported for one of the patients treated at the current, highest exposure dose regimen, who is without evidence of Ewing sarcoma after more than eight months on study and has tolerated treatments with TK216, alone and in combination with vincristine, well. TK216 has been generally well tolerated in this trial. Dose limiting toxicities consisted of transient and manageable myelosuppression, primarily neutropenia.
The expansion cohort will further evaluate the recommended Phase 2 dose regimen of TK216 (200 mg/m2/day for 14 days) in combination with vincristine and is anticipated to enroll approximately 18 patients with relapsed or refractory Ewing sarcoma.
“We are encouraged by the initial clinical activity demonstrated by TK216 in the dose-finding portion of this clinical trial in patients with relapsed/refractory Ewing sarcoma and look forward to further evaluating the recommended Phase 2 dose regimen of TK216 in a larger number of patients with this devastating disease,” said James Breitmeyer, M.D., Ph.D., Oncternal’s President and CEO. “This first-in-class targeted investigational agent may also be applicable in other malignancies driven by ETS alterations including acute myeloid leukemia (AML) and prostate cancer, which we continue to explore in preclinical studies.” Full Press Release and Additional Information
AUGUST 12, 2019
Dr. Toretsky was invited to give a keynote talk at the Children's Cancer Therapy Development Institute in Portland Oregon at their annual Nanocourse. His talk was titled "From Idea on a Napkin to Clinical Trial: Creating a Drug for a Rare Cancer".
Dr. Toretsky was invited to give a keynote talk at the Children's Cancer Therapy Development Institute in Portland Oregon at their annual Nanocourse. His talk was titled "From Idea on a Napkin to Clinical Trial: Creating a Drug for a Rare Cancer".
SEPTEMBER, 2019
Sep. 17, 2019-- Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, today announced that Paul A. Meyers, M.D., Chief, Pediatric Sarcoma Service and Vice Chair for Clinical Affairs of Memorial Sloan Kettering Cancer Center, presented a case study of a patient with Ewing sarcoma who achieved a sustained response following treatment with Oncternal’s investigational product candidate, TK216, in an ongoing Phase 1, first-in-human clinical trial. The presentation entitled, “TK216 for the Treatment of Ewing Sarcoma,” was given at the Fall Children’s Oncology Group (COG) Meeting.
Case Study of TK216 in Ewing Sarcoma: Achieved Sustained Response
JULY, 2019
Sydney Parks Receives 2019 St. Baldrick's Summer Fellow Award
Sydney Parks Receives 2019 St. Baldrick's Summer Fellow Award
Returning summer researcher, Sydney Parks, received a 2019 St. Baldrick's Summer Fellow Award aiding to fund a summer research project. Through the St. Baldrick’s Summer Fellowship, Sydney began cloning common EWS-FLI1 (EF) mutants into a tetracycline-inducible plasmid previously generated by Garrett Graham. In the future, a library of such plasmids can be expressed in human mesenchymal stem cells (hMSCs) to investigate hMSC transformation into ES-like cells following EF expression. Sydney is currently a senior at Rice University studying Biochemistry and Cell Biology.
This grant funds an undergraduate student to complete work in pediatric oncology research for the summer. The experience may encourage them to choose childhood cancer research as a specialty. Ewing Sarcoma (ES) is an aggressive pediatric cancer.
JUNE, 2019
Toretsky Receives 4th Annual Force 3 Fellowship Award for research in Ewing sarcoma
The 35th Annual Childrens Cancer Foundation Gala was held Saturday, November 2nd, 2019 where more than 500 individuals joined CCF and Mark Viviano, as emcee, and recognized Extraordinary Families – children and their families who have endured, fought, overcome, grieved and persevered in spite of this life-changing diagnosis. Two amazing young people – Darren Leonard and Grace Callwood – were featured with their families in a video played during the program. Many other children that evening were presented with medals, recognizing the battles they have overcome with having a diagnosis of cancer.
CCF also granted awards to nine area researchers, including the 4th Annual Fore 3 Fellowship Award to Dr. Jeffrey Toretsky, MD, and to several programs and facilities making significant differences in the lives of these children, totalling nearly $1 million.
MAY 14-15, 2019
Dr. Toretsky was an invited participant and speaker at the NCI-sponsored workshop, "Liquid Phase Condensation as a Cellular Regulatory Mechanism: A State of the Science and Implications for Cancer Research." His talk was titled "EWS-FLI1, RHA Helicase A, Splicing regulators and Phase Separation: What’s in the droplet and how might it function?" For more information on Protein Phase Separation check out this review in Cell
Dr. Toretsky was an invited participant and speaker at the NCI-sponsored workshop, "Liquid Phase Condensation as a Cellular Regulatory Mechanism: A State of the Science and Implications for Cancer Research." His talk was titled "EWS-FLI1, RHA Helicase A, Splicing regulators and Phase Separation: What’s in the droplet and how might it function?" For more information on Protein Phase Separation check out this review in Cell
AUGUST, 2018
Dr. Kalliopi Tsafou, a post-doc in our laboratory, penned a brilliant review that discusses the possibilities of targeted transcription factors. Transcription factors have been called undruggable, but we did not believe it. Read our review in the Journal of Molecular Biology
Dr. Kalliopi Tsafou, a post-doc in our laboratory, penned a brilliant review that discusses the possibilities of targeted transcription factors. Transcription factors have been called undruggable, but we did not believe it. Read our review in the Journal of Molecular Biology
DECEMBER, 2017
The National Academy of Inventors (NAI) has names Dr. Toretsky to the 2017 class of fellows
The National Academy of Inventors (NAI) has names Dr. Toretsky to the 2017 class of fellows
Georgetown Lombardi’s Jeffrey Toretsky, MD, Named a National Academy of Inventors Fellow
OCTOBER, 2017
Our paper addressing synergy with YK-4-279 was featured on the cover of Science Signaling
Our paper addressing synergy with YK-4-279 was featured on the cover of Science Signaling
APRIL 23, 2017
Dr. Toretsky presented a talk titled “Targeting EWS-FLI1 in Ewing Sarcoma” at the annual American Society of Pharmacology and Experimental Therapeutics in Chicago, IL
Dr. Toretsky presented a talk titled “Targeting EWS-FLI1 in Ewing Sarcoma” at the annual American Society of Pharmacology and Experimental Therapeutics in Chicago, IL
FEB 24-26, 2017
The first Phase Separation and RNA Processing as Drivers of Cancer and Neurodegenerative Disease Conference took place in La Jolla, CA led by co-organizers Jeff Toretsky and Aaron Hosking. An NIH R13, Burroughs-Wellcome Grant, and corporate support provided funding for the meeting.
The first Phase Separation and RNA Processing as Drivers of Cancer and Neurodegenerative Disease Conference took place in La Jolla, CA led by co-organizers Jeff Toretsky and Aaron Hosking. An NIH R13, Burroughs-Wellcome Grant, and corporate support provided funding for the meeting.
FEB 6, 2017
A great article from STAT about Dr. Toretsky’s “Odyssey” to commercialize the molecule that he and the lab discovered for Ewing Sarcoma. Enjoy the full article here.
A great article from STAT about Dr. Toretsky’s “Odyssey” to commercialize the molecule that he and the lab discovered for Ewing Sarcoma. Enjoy the full article here.
SEPTEMBER, 2016
Saravana Selvanatha was promoted to Research Assistant Professor! Congratulations, Saravana!
Saravana Selvanatha was promoted to Research Assistant Professor! Congratulations, Saravana!
AUGUST 31, 2016
Congratulations to Dr. Saravana Selvanathan, PhD., for receiving a travel award to attend the 2016 Bermuda Principals Impact on Splicing Conference! The conference will take place November 8-10, 2016 on the exotic island of Bermuda! Congratulations Saravana!
Congratulations to Dr. Saravana Selvanathan, PhD., for receiving a travel award to attend the 2016 Bermuda Principals Impact on Splicing Conference! The conference will take place November 8-10, 2016 on the exotic island of Bermuda! Congratulations Saravana!
May 16, 2016
Dr. Toretsky will present a talk “Small Molecule Targeting of the Intrinsically Disordered Oncoprotein EWS-FLI1” at the Intrinsically Disordered Proteins Gordon Research Conference. The conference will be held in Les Diableretes, Switzerland, June 26 – July 1, 2016.
Dr. Toretsky will present a talk “Small Molecule Targeting of the Intrinsically Disordered Oncoprotein EWS-FLI1” at the Intrinsically Disordered Proteins Gordon Research Conference. The conference will be held in Les Diableretes, Switzerland, June 26 – July 1, 2016.
OCT 26, 2015
The 2015 Winner of the NFL Players Association Georgetown Lombardi Award, Devon Still, Visits our lab and meets Dr. Toretsky.
The 2015 Winner of the NFL Players Association Georgetown Lombardi Award, Devon Still, Visits our lab and meets Dr. Toretsky.
SEPT 16, 2015
Congratulations to Dr. Toretsky, who was honored by Hyundai Hope on Wheels at an outdoor ceremony at Georgetown Lombardi.
Congratulations to Dr. Toretsky, who was honored by Hyundai Hope on Wheels at an outdoor ceremony at Georgetown Lombardi.
SELECTED PUBLICATIONS
SELECTED PUBLICATIONS
2022
2022
Graham, Garrett & Selvanathan, Saravana & Zöllner, Stefan & Stahl, Emily & Shlien, Adam & Caplen, Natasha & Üren, Aykut & Toretsky, Jeffrey. (2022). Comprehensive profiling of mRNA splicing indicates that GC content signals altered cassette exon inclusion in Ewing sarcoma. NAR Cancer. 4. 10.1093/narcan/zcab052.
2021
2021
Zöllner SK, Amatruda JF, Bauer S, Collaud S, de Álava E, DuBois SG, Hardes J, Hartmann W, Kovar H, Metzler M, Shulman DS, Streitbürger A, Timmermann B, Toretsky JA, Uhlenbruch Y, Vieth V, Grünewald TGP, Dirksen U. Ewing Sarcoma-Diagnosis, Treatment, Clinical Challenges and Future Perspectives. J Clin Med. 2021 Apr 14;10(8):1685. DOI: 10.3390/jcm10081685. PMCID: PMC8071040. PMID: 33919988
2016
2016
Saygideger-Kont Y, Minas TZ, Jones H, Hour S, Celik H, Temel I, Han J, Atabey N, Erkizan HV, Toretsky JA, Üren A. Ezrin Enhances EGFR Signaling and Modulates Erlotinib Sensitivity in Non-Small Cell Lung Cancer Cells. Neoplasia. 2016 Feb;18(2):111-20. doi: 10.1016/j.neo.2016.01.002. PubMed PMID: 26936397.
2015
2015
Minas TZ, Han J, Javaheri T, Hong SH, Schlederer M, Saygide?er-Kont Y, Celik H, Mueller KM, Temel I, Özdemirli M, Kovar H, Erkizan HV, Toretsky J, Kenner L, Moriggl R, Üren A. YK-4-279 effectively antagonizes EWS-FLI1 induced leukemia in a transgenic mouse model. Oncotarget. 2015 Nov 10;6(35):37678-94. doi: 10.18632/oncotarget.5520. PubMed PMID: 26462019; PubMed Central PMCID: PMC4741957.
Zöllner SK, Rössig C, Toretsky JA. Synovial sarcoma is a gateway to the role of chromatin remodeling in cancer. Cancer Metastasis Rev. 2015 Sep;34(3):417-28. doi: 10.1007/s10555-015-9575-z. PubMed PMID: 26277104.
Selvanathan SP, Graham GT, Erkizan HV, Dirksen U, Natarajan TG, Dakic A, Yu S, Liu X, Paulsen MT, Ljungman ME, Wu CH, Lawlor ER, Üren A, Toretsky JA. Oncogenic fusion protein EWS-FLI1 is a network hub that regulates alternative splicing. Proc Natl Acad Sci U S A. 2015 Mar 17;112(11):E1307-16. doi: 10.1073/pnas.1500536112. Epub 2015 Mar 3. PubMed PMID: 25737553; PubMed Central PMCID: PMC4371969.
Erkizan HV, Schneider JA, Sajwan K, Graham GT, Griffin B, Chasovskikh S, Youbi SE, Kallarakal A, Chruszcz M, Padmanabhan R, Casey JL, Üren A, Toretsky JA. RNA helicase A activity is inhibited by oncogenic transcription factor EWS-FLI1.Nucleic Acids Res. 2015 Jan;43(2):1069-80. doi: 10.1093/nar/gku1328. Epub 2015 Jan 6. PubMed PMID: 25564528; PubMed Central PMCID: PMC4333382.
Celik H, Sajwan KP, Selvanathan SP, Marsh BJ, Pai AV, Kont YS, Han J, Minas TZ, Rahim S, Erkizan HV, Toretsky JA, Üren A. Ezrin Binds to DEAD-Box RNA Helicase DDX3 and Regulates Its Function and Protein Level. Mol Cell Biol. 2015 Sep;35(18):3145-62. doi: 10.1128/MCB.00332-15. Epub 2015 Jul 6. PubMed PMID: 26149384; PubMed Central PMCID: PMC4539380.
2014
2014
Toretsky JA, Wright PE. Assemblages: functional units formed by cellular phase separation. J Cell Biol. 2014 Sep 1;206(5):579-88. doi: 10.1083/jcb.201404124. Review. PubMed PMID: 25179628; PubMed Central PMCID: PMC4151146.
Shah N, Wang J, Selich-Anderson J, Graham G, Siddiqui H, Li X, Khan J, Toretsky J. PBX1 is a favorable prognostic biomarker as it modulates 13-cis retinoic acid-mediated differentiation in neuroblastoma. Clin Cancer Res. 2014 Aug 15;20(16):4400-12. doi: 10.1158/1078-0432.CCR-13-1486. Epub 2014 Jun 19. PubMed PMID: 24947929; PubMed Central PMCID: PMC4134768.
THANK YOU
THANK YOU
The Toretsky Laboratory is grateful to many philanthropies for support over the years. Our success has been built on your trust along with your ability to share our vision of using multidisciplinary research to better target cancer.
Ongoing supporters include:
Ongoing supporters include:
Children’s Cancer Foundation, Inc.
Hyundai Hope on Wheels
Previous supporters have included:
Previous supporters have included:
Burroughs Wellcome Clinical Scientist Award in Translational Research
Go 4 the Goal
Dani’s Foundation
Rutledge Foundation
CureSearch AFLAC
Make Some Noise Foundation
Liddy Shriver Sarcoma Initiative
Amschwand Sarcoma Foundation
Alex’s Lemonade Stand Foundation
Friend’s of Colin
St. Baldrick's Foundation
Alan B. Slifka Foundation
Your tax dollars supported us through these NIH grants:
Your tax dollars supported us through these NIH grants:
RC4CA156509
R01CA133662
R01CA138212
R01CA88004
R41CA102841
R01CA78843
RR022388
R01CA233619-01A1
R44CA261557-01
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