Combined Approaches by Immune Checkpoint inhibition for Hypermutant Cancers

Uri Tabori, MD

Staff Physician Haematology/OncologyThe Hospital for Sick ChildrenView Bio

Dr. Uri Tabori is a Pediatric Hematologist Oncologist at the Hospital for Sick Children and a Professor for Pediatrics and Medical Biophysics at the University of Toronto.

Dr. Tabori holds the Garron Family Chair in Childhood Cancer Research and maintains an active clinical practice in the treatment of children with cancer, focusing particularly on those with brain tumors.

He is especially interested in translating basic genomic and genetic discoveries for early detection and precision therapeutics in individuals determined to be at high-risk of developing cancers at a young age. As such, he also leads the International Replication Repair Deficiency Consortium

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Meet the Team

  • Uri Y. Tabori, MD, The Hospital for Sick Children (Canada)
  • John Maris, MD, Children’s Hospital of Philadelphia
  • Victor Velculescu, MD, PhD, Johns Hopkins University
  • Patrick Forde, MBBCh, Johns Hopkins University
  • Pamela Ohashi, PhD, University of Toronto (Canada)
  • Trevor Pugh, PhD, University of Toronto (Canada)
  • Cynthia Hawkins, MD, PhD, FRCPC, The Hospital for Sick Children (Canada)
  • Adam Shlien, PhD, The Hospital for Sick Children (Canada)
  • Eric Bouffet, MD, The Hospital for Sick Children (Canada)
  • Michael Fisher, MD, Children’s Hospital of Philadelphia
  • Daniel Morgenstern, MD, The Hospital for Sick Children (Canada)


  • Patient Advocate: Denise Bebenek
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About This SU2C Catalyst Clinical Trial

Is your child’s cancer resistant to treatment? Stand Up To Cancer (SU2C) is supporting a clinical trial that is seeking new ways to treat certain types of childhood cancers, called “hypermutant” cancers, for which there is currently no cure. The researchers believe these cancers could be very receptive to treatment with immune checkpoint inhibitors (ICI) such as nivolumab. Immunotherapy drugs like nivolumab work by “releasing the brakes” on the body’s immune system, and allowing it to fight against cancer.

This trial is seeking pediatric cancer patients who have either a hypermutant tumor or have Constitutional or Biallelic Mismatch Repair Deficiency (CMMRD or BMMRD) syndrome. Tumors will be biopsied and tested for hypermutation as part of the screening process.

This trial is seeking to:

  • Identify hypermutant cancers
  • Identify biomarkers that will predict which patients will respond to immunotherapy
  • Test the responses to nivolumab, and to other drugs combined with nivolumab

The trial will be conducted internationally, in a total of four locations in Canada, the U.S., France and Israel.

We Need You

Why Your Participation Matters

Patients who are identified as eligible for this trial will receive therapy with nivolumab for 24 months and one year of follow-up. Immune checkpoint inhibitors such as nivolumab have shown promising evidence against hypermutant cancers, including Constitutional or Biallelic Mismatch Repair Deficiency (CMMRD and BMMRD). BMMRD/CMMRD is one of the most aggressive cancer syndromes: All patients who have B/CMMRD develop multiple cancers before the age of 18. Results of this trial could lead to better treatments and improve outcomes for difficult-to-treat pediatric and adult cancers worldwide.

Key Insights for Participants


Children (12 months -18 years) with refractory or recurrent hypermutated malignant tumors, who have a current disease for which there is no curative therapy available. Includes constitutional or Biallelic Mismatch Repair Deficiency (B/CMMRD) positive patients.

Key criteria are summarized below and can be found in detail on Interested patients will need to review their medical histories with clinical trial patient coordinators before they can be accepted to participate in this trial.

Male or Female
Age Range:
12 months - 18 years
Refractory or recurrent hypermutated malignancies


Your child must have a measureable or evaluable tumor present on a scan.

Your child’s current disease must be one for which there is no known cure.

Your child must have fully recovered from the acute toxic effects of any previous anti-cancer therapy.

Your child must have adequate organ function; specifically bone marrow, renal, liver, pulmonary, and pancreatic function.

Your child is NOT eligible if he or she was previously treated with nivolumab or other anti-PD-1 or anti- PD-L1 immune checkpoint inhibitors.

Your child’s medical history CANNOT include an autoimmune disease, including autoimmune hypothyroidism; an uncontrolled infection; HIV/AIDS;

or acute or chronic hepatitis B or C. Your child is also NOT eligible if he or she requires immunosuppressive doses of corticosteroids.

More +Less -

Before beginning the trial, your child must be able to function well enough to care for him/herself most of the time without assistance, as appropriate to the child’s age.

Your daughter is NOT eligible to participate in this clinical trial if she is pregnant or is breast feeding.


You play a vital role.

Here are the locations where your child can participate in this clinical trial. If you are interested, please contact the patient coordinator at your preferred site. The patient coordinator is there to help you understand every aspect of the clinical trial process and can answer any questions.

The patient coordinator will start by reviewing your child’s medical history with you to see whether he/she meets all the criteria to participate. The coordinator will then guide you through a detailed “informed consent” document that you will be required to sign when your child enrolls in a clinical trial.

Toronto, Canada

Dr. Eric Bouffet
Dr. Daniel Morgenstern
P: +1 416-813-5055

Philadelphia, U.S.A.

Children’s Hospital of Philadelphia
Michael Fisher

Paris, France

Institute Gustave Roussy
Dr. Jacques Grill
P: +33-142114211

Tel Aviv, Israel

Tel Aviv Sourasky Medical Centre

Dr. Rina Dvir
P: +972-3-697-3494


Support for this study comes from the Bristol-Meyers Squibb Company.
Learn more about BMS +
Here are some links to organizations that provide more information about Pediatric Hypermutant Cancers, and may provide information on patient support, treatment options, and current clinical trials.
St. Baldrick's Foundation +
For additional resources, please visit +

Find out if there's a trial for you. Reach out today.

Even if you do not meet the requirements for this trial, there may be other trials for you. Get started with the SU2C Clinical Trial Finder, a free and confidential cancer clinical trial matching and referral service.
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