News & Events

A Phase II clinical trial has recently opened and has begun enrolling patients at M Health Fairview University of Minnesota Medical Center. This trial will utilize CRISPR genetic engineering to design a treatment for metastatic gastrointestinal solid tumor cancer. To date, CAR-T and other cell therapies such as natural killer cells have shown promising results in liquid tumors, but there have been no effective treatments developed for the treatment of solid tumor cancers.

Multiple sulfatase deficiency (MSD) is an extremely rare genetic condition passed along to children when both of their parents have the specific genetic mutation. Termed an ‘ultra rare’ and ‘one in a million’ condition. A team of researchers at the University of Minnesota, including Paul Orchard, MD, Professor in the Division of Pediatric Bone and Marrow Transplant & Cellular Therapy at the University of Minnesota, performed the first-ever bone marrow transplants on MSD patients in hopes it would be an effective treatment.

Connor, born in Canada, travelled to M Health Fairview Masonic Children's Hospital in Minnesota to receive care for his diagnosed rare condition, adrenoleukodystrophy (ALD). Prior to diagnosis, Connor showed no signs of the disease, but the condition eventually progressed into Cerebral ALD, one of the most serious and aggressive forms of the condition. The medical team in Minnesota, known for being experts in rare diseases in ALD care, treated Connor for his condition using an umbilical cord transplant. 

At three years old, Matt received a stem cell transplant at M Health Fairview Masonic Children Hospital's Pediatric Blood and Marrow Transplant Center. The stem cell transplant was performed to treat Matt's rare condition, Wiskott-Aldrich Syndrome. That procedure changed many aspects of his life, according to the interview conducted back in 2013, but as an adult it also prompted him to shift his focus towards giving back. 20 years later, Matt returned to the University of Minnesota Medical Center to interview for medical school, pursuing his dream to become a doctor because of his past. 

Nicholas, the son of Julie Purschke, was screened at birth for adrenoleukodystrophy (ALD), a rare genetic condition that progressively destroys the protective coating on brain neurons. His screening indicated the presence of the genetic abnormality linked to ALD, prompting his parents and doctors to conduct regular yearly tests for signs of the disease. These screenings allowed physicians to detect early brain lesions caused by ALD, leading to successful treatment with an umbilical cord transplant. This story underscores the importance of patient advocacy and the role of a dedicated physician care team in managing rare diseases.