Clinical Research Update – March 2014

Clinical Research Update

March 2014

Pediatric Cancer Program

  • Pediatric BMT: our pediatric BMT program has some of the best outcomes in the country with greater than 90% 1-year survival rates. Contributions are being used to fund two studies, a Phase I (first-in-humans) study that gives cell treatments after transplant to decrease leukemia recurrence has enrolled 25 patients, and a Phase II study (being done at only a few hospitals in U.S.) that removes harmful parts of a transplant so that half-matched family members can be used as donors has enrolled 36 patients. Both have had very good outcomes and have led to national presentations by Dr. Andy Gilman and colleagues.

  • Developmental Therapeutics: 2013 was the busiest and most successful year to date. We enrolled 15 children in early-stage protocols who would not have been able to be treated at LCH prior to the development of this program. We had 5 patients who received a first of its kind clinical trial in personalized medicine. The trials involve taking live tumor tissue, examining it for genetic abnormalities, and tailoring chemotherapy to the unique genetic signature of the tumor cell. We are one of only 6 hospitals offering this.
  • We had a child travel from St. Louis who had been offered no hope at other hospitals and our pediatric cancer specialists combined with our hepatobiliary surgeons to remove the tumor near his liver and then enroll him in the above trial. He has gone home now and remains cancer free.
  • In early 2014, LCH physicians along with an LCI radiation oncologist, completed the first in the world pediatric implant of brachytherapy mesh, containing irradiated seeds, for a 12 year old boy who had recurrent metastatic Wilm’s tumor in his chest. The boy had already received extensive radiation treatment when the cancer first occurred so this brachytherapy was the only safe form of treatment possible. The mesh was designed and customized by our physician in conjunction with a specialized vendor.
  • In 2014 we plan to pursue several investigator-initiated studies in sarcoma (the specialty of our world-renowned new director from MD Anderson, Peter Anderson), as well as lymphoma, and neuroblastoma. This research could open new treatment protocols for rare relapsed solid tumors that would be unique to our  program.


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