Turning Research into Life-Saving Treatment for Rare Diseases

A disease is rare if it affects fewer than 200,000 people in the United States. But when all rare diseases are combined, they really aren’t that rare at all. In fact, the National Institutes of Health estimates that there are as many as 7,000 rare diseases affecting 30 million Americans.

Although the road to getting a rare disease diagnosis can be a difficult one, Nationwide Children’s Hospital is leveraging new technologies to turn research into life-saving treatments for rare diseases.

In the Good Manufacturing Practice (GMP) production facility at The Research Institute at Nationwide Children’s, specialized technicians are making materials for first-in-human clinical trials for rare diseases that have no other treatments.

An Inside Look at the Facility

The facility space is controlled tightly to reduce contamination and to meet the specific requirements set forth by the Food and Drug Administration (FDA) for anything that is going to be employed for human use.

Technicians in the GMP facility go through rigorous training, from how to gown and how to move through the hallways, to how to go through the production process and move within the clean room.

The central hallway is pressurized at a higher rate than everything else around it. This means it essentially pushes into every room – that way, technicians can keep what is happening in the processing rooms isolated from one another, to allow processing of multiple products at the same time.

The facility has a section dedicated to cell-based therapy, a growing area of research that involves altering a patient’s own cells or cells from a donor and infusing them into the patient to combat disease. There is also a section dedicated to viral vector manufacturing, a process that produces to clinical-grade vectors that are administered to patients during trials.

And because of the way the GMP facility is designed, technicians can pass samples directly out of the GMP laboratory spaces into the quality control lab. This means there isn’t added travel time or delays, and samples can be turned around immediately.

Treating Diseases with No Other Treatments

When it comes to rare diseases, finding treatments – and finding them quickly – is even more important. And researchers and technicians at the GMP facility and Nationwide Children’s are focused on the handful of children each year that are diagnosed with a particular rare disease, or the dozens of children across the world that have that rare disease.

There’s no pharmaceutical priority for the facility. Its main mission is to supply high-quality, clinical-grade products that can be tested as investigative new drugs in patients with incurable genetic diseases.

The facility also fulfills requests from external clients or outside groups who are looking for partners to help get their products into phase 1 clinical trials.