MSK Kids

MSK Kids, the pediatric program of Memorial Sloan Kettering Cancer Center, treats more young people facing cancer than any other hospital in the United States. Our researchers work together to make breakthroughs that improve the standard of care for childhood cancers, and MSK is one of the few places in the world where scientific discoveries can quickly and successfully be translated into improved treatments.

Pediatric Renal Cancers

Wilms Tumor

Diagnosed in about 500 children in the United States each year, Wilms tumor is the most common type of kidney cancer found in young people. Most diagnoses occur between ages 2 and 5, but the tumors can also develop in infants, older school-age children, and, very occasionally, adolescents or adults.

Selinexor Clinical Trial

Phase II Study for Relapsed Wilms Tumor

Physicians and researchers at MSK Kids have played a major role in improving survival rates for children with aggressive childhood cancers, providing real hope for these patients and their families. At MSK, our goal is to continue innovating through research, ever mindful of the need for less toxic therapies that minimize the long-term effects of cancer treatment.

Michael Ortiz, M.D., specializes in treating pediatric kidney and other rare childhood cancers. Recently, he and a team of scientists at MSK described (Coutinho, et al., Med, 2022) that targeting the protein pump Exportin 1 (XPO1), which is normally involved in coordinating intra-cellular signaling, is an unexpected weakness in Wilms tumors (WT) and malignant rhabdoid tumors (MRT).  Concurrent unpublished laboratory and clinical data in adults suggested that another rare cancer impacting adolescents, malignant peripheral nerve sheath tumors (MPNST), may also be susceptible to XPO1 inhibition. 

Selinexor is the first drug developed to block XPO1 and has now been studied in several thousand cancer patients, including multiple studies in children with cancer, so a safe dose to administer to children has been established but testing has not included WT, MRT, or MPNST.  Dr. Ortiz and his team have recently opened a multi-center phase II clinical trial of Selinexor in children, adolescents, and adults with advanced solid tumors, specifically evaluating how well Selinexor treats cases of advanced WT, MRT, MPNST, and other pediatric solid tumors that we believe may benefit from XPO1 inhibition.  The phase II trial will enroll 8 patients with very high risk relapsed WT and then potentially enroll another 13 WT patients if sufficient activity was noted in the initial group and the primary objective of the study is to evaluate the overall effectiveness of Selinexor in this very high-risk subgroup of WT.  In addition, up to 15 patients with MRT, MPNST, and other pediatric solid tumors may enroll which will provide preliminary evidence of activity in these less common pediatric solid tumors. 

Prior trials did not offer a liquid option and so young children were not able to be treated with Selinexor since they could not swallow tablets and tablets could not be crushed.  This study will incorporate a liquid suspension to provide drug access to children as young as 12 months old and will have a separate cohort enrolling up to 9 patients in order to confirm the safety and drug levels in young children (less than 6 years of age).  This trial also includes a more aggressive supportive care approach, encouraging the use of medicines to raise platelet counts which is a common but treatable side effect. 

In order to make sure this study provides the greatest benefit to each individual who enrolls, physicians will allow patients who benefit from Selinexor to undergo surgery and radiation on study (if clinically appropriate) and will permit individual patients to adjust the Selinexor dose up or down as tolerated so they can be treated at the best dose for them. Importantly, MSK’s physicians will study their patients' tumors to evaluate whether they can predict which patients will benefit from Selinexor including a novel sequencing platform called Darwin OncoTarget/OncoTreat, which has the potential to identify previously unanticipated patients who may benefit from this approach.

The proposed study was approved by the pharmaceutical sponsor, Karyopharm. Although Karyopharm will make the drug available for the trial, no additional financial support will be provided from the industry partner. Therefore, philanthropic support is necessary to continue the study.

Philanthropic funding will help to accelerate the start of the Phase II study and, eventually, allow for the study to expand beyond MSK to multiple testing sites, including institutions in Boston, Atlanta, Cincinnati, and Los Angeles, with potential to expand to other domestic and international sites. Funding would also help MSK physicians and researchers to perform correlative studies that would provide important guidance on designing and evaluating the drug.

The Impact of Philanthropy

Philanthropy drives innovation and understanding. Your partnership with MSK is crucial to continued progress in pediatric cancer research and education, and each contribution brings us closer to improving the lives of all children with cancer. Thank you for your kind consideration in supporting great steps forward.

You may also donate by check. Checks, made payable to Memorial Sloan Kettering Cancer Center, should be mailed to:

 

Memorial Sloan Kettering Cancer Center, Office of Development

Attn: Scott Morano

P.O. Box 27106

New York, NY 10087

 

We kindly ask that you indicate on the check that the gift is for Pediatric Renal Tumor Funding Priorities.

 

If you have any questions please contact Scott Morano at moranos@mskcc or 917-453-3521. 

Employer Matching Gifts - Matching gifts are a benefit offered by many companies to encourage philanthropy amongst their employees. This is an incredible way to increase your impact. For most companies, it’s as easy as completing an online or paper form and returning it to MSK. MSK confirms your gift and sends the necessary paperwork directly to your company. If you cannot find specific instructions through MSK’s search tool, https://giving.mskcc.org/matching-gifts, contact your company’s Corporate Philanthropy or Human Resources department to initiate the process.

 

Many employers also offer corporate sponsorships to organizations that their employees are interested in supporting. Contact your employer or check with your HR department to see if your employer participates in a sponsorship program that can support MSK.