General News

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. 

In this 2017 interview, Paul Orchard, MD, Professor in the Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy, answered questions about adrenoleukodystrophy (ALD) and the services M Health Fairview has to offer for care and treatment of this condition. Through research and innovation, Dr. Orchard and the rest of the interdisciplinary team at M Health Fairview Masonic Children's Hospital work on finding and innovating alternatives to current treatments to increase treatment success. 

In 2013, shortly after Brock Groh was born, he was diagnosed with adrenoleukodystrophy (ALD), a rare genetic disorder that later developed into cerebral ALD, a life-threatening form of the disease. Brock received a life-saving bone marrow transplant at M Health Fairview Masonic Children's Hospital. To express their appreciate for the care their son received while at M Health Fairview, Brock's family started a nonprofit, X out ALD, to donate in support of research at the hospital facility. 

In this 2019 interview, Troy Lund, MD, PhD, Professor and Fellowship Program Director in the Division of Pediatric Blood and marrow Transplantation & Cellular Therapy at the University of Minnesota, discussed rare diseases as a way to promote awareness in support of Rare Disease Day. As of 2019, there are approximately 7,000 rare diseases, affecting around 25 to 30 million Americans. While treatments for rare diseases are advancing, many conditions still lack effective treatment options.

In 2022, the Food and Drug Administration (FDA) approved a gene therapy for cerebral adrenoleukodystrophy (ALD), a rapidly progressing rare genetic neurodegenerative disease that impairs the body's ability to break down very long-chain fatty acids. This treatment, developed by a team of physicians and researchers at M Health Fairview Masonic Children’s Hospital—a nationally recognized rare disease specialty center—replaces the defective gene responsible for ALD with a functional copy. This breakthrough provides a vital treatment option for a rare condition that affects approximately one in 17,000 people

In 2021, siblings Grace and Grant Goodin received the first bone marrow transplants for Multiple Sulfatase Deficiency (MSD) at M Health Fairview Masonic Children’s Hospital. This groundbreaking treatment was the first of its kind globally, led by a team of pediatric physicians and rare disease specialists. MSD is an exceptionally rare and often fatal genetic disorder, with only about 100 cases ever diagnosed worldwide, including Grace and Grant.