Saving Kids from a Deadly Cancer

 

While our corporate vision is broad and aspirational, our strategy is more pragmatic. We intend to follow the proven path of biotech startup success by developing immunotherapy treatments for rare diseases under the Orphan Drug Act. Rare diseases often have much lower cost clinical trials due to small patient sizes, which translate to faster time to market for successful treatments.

Four decades ago, patients with rare diseases had very few treatment options. The 1983 Orphan Drug Act changed everything by providing substantial tax incentives for companies to invest in rare conditions affecting fewer than 200,000 Americans. These incentives include FDA fast tracking, market exclusivity and reduced taxes.

Ewing Sarcoma

Our initial immunotherapy development is targeted at treating Ewing Sarcoma, a rare but deadly bone and soft tissue cancer that primarily affects children and young adults and for which there is no current FDA approved treatment to prevent recurrence.

 

Facts:

  • It is the 2nd most common type of bone cancer in children
  • About 200 children and young adults are diagnosed each year in the United States
  • Survival rate of 55-65% if the disease is localized when diagnosed
  • Survival rate of 20-35% if the disease has spread when diagnosed
  • No treatment options are currently available for recurrence

 

Research and Development

We have entered into a sponsored research agreement (SRA) with the University of California at Los Angeles (UCLA), led by a team of professors and physicians who run the Ewing Sarcoma program at the UCLA Jonsson Comprehensive Cancer Center. During the term of SRA, we have the exclusive right to negotiate and license any resulting intellectual property.


We have also entered into a Letter of Intent with UCLA that gives us the exclusive right to negotiate a license to the pre-existing patent application “Methods of Treating Ewing Sarcoma.”


The objective of the program is to develop effective immunotherapies such as CAR T-cell and monoclonal antibodies for Ewing Sarcoma. This program expands on the preliminary laboratory work done at UCLA by leveraging outside commercial-grade immunotherapy development processes and services. We believe the resulting technology will be able to target other cancers such as adrenal cancer and brain cancer, as well as enabling the development of other immunotherapies, vaccines and drugs.


From 2016-2020, UCLA was ranked the #1 public university in the nation by U.S. News & World report. The Jonsson Comprehensive Cancer Center was ranked 13th and the Geffen Medical School was ranked 21st best in the country.

 

The UCLA Team

Satiro De Oliveira, MD

Principal Investigator

  • Health Sciences Associate Clinical Professor
  • Department of Pediatrics, Division of Hematology/Oncology
  • David Geffen School of Medicine at UCLA
  • UCLA Jonsson Comprehensive Cancer Center

https://www.uclahealth.org/satiro-de-oliveira

 

Noah Federman, MD

Co-Principal Investigator

  • Health Sciences Clinical Professor,
  • Departments of Pediatrics and Orthopedics
  • Medical Director, CTSI Clinical and Translational Research Center
  • Director, Pediatric Bone and Soft Tissue Sarcoma Program,
  • Jonsson Comprehensive Cancer Center
  • UCLA David Geffen School of Medicine

https://www.uclahealth.org/noah-federman

Steven J. Jonas, MD/PhD

Co-Principal Investigator

  • Assistant Professor
  • David Geffen School of Medicine at UCLA
  • Department of Pediatrics, Division of Pediatric Hematology/Oncology
  • UCLA Mattel Children's Hospital, Children’s Discovery & Innovation Institute, and the California NanoSystems Institute
  • Eli & Edythe Broad Center of Regenerative Medicine & Stem Cell Research
  • Alex’s Lemonade Stand Young Investigator & Hyundai Hope on Wheels Young Investigator/Scholar

https://uclahealth.org/mattel/steven-jonas

 

For more information about Ewing Sarcoma or to show your support, visit the Sarcoma Foundation of America.