One particular problem for those with rare diseases is that the manifestation of the disease is often particular to the patient, each with their own quirks and tendencies. This makes finding treatment options difficult because it needs to be individualized– this is challenging when it means finding suitable drugs which cost millions of dollars to create in the first place.
However, the pharmaceutical company FabRx has been experimenting with a tool that may lead to just that: 3D printing of pharmaceuticals to create personalized treatments. Researchers from University College London, the University of Santiago de Compostela, and the Clinical University Hospital in Santiago de Compostela have together just successfully run the first in human clinical study using their new technology, which is a pioneer in the 3D printing world.
This was the first time automated 3D printing has been used in this individualized method in a hospital when they administered personalized doses of isoleucine on patients with Maple Syrup Urine Disease (MSUD) in Santiago de Compostela.
What is Maple Syrup Urine Disease?
Maple Syrup Urine Disease
is inherited genetic metabolic disorder in which the patients cannot process particular proteins and amino acids. Its symptoms include vomiting, lethargy, slowed development, and a sweet smell to the urine, responsible for the name. It typically becomes evident right after birth, though it varies for individuals.
Why MSUD Needs Personalized Medicine
To treat MSUD, patients need personalized doses of the relevant supplements, isoleucine and valine, depending on the concentration of isoleucine in their blood. Currently, this must be done manually in hospitals in order to tailor the treatment to the patients needs. However, this type of preparation is tedious, time consuming, and expensive for the hospitals and patients, who require this medication for their entire lives. It clearly lacks efficiency, but as of now, there are not other options. Patients cannot all take one standardized medicine, but current pharmaceutical production doesn’t lend itself to flexibility and individual needs.
For these reasons, MSUD was a prime disease to benefit from the 3D printing which can individualize medicine quickly and precisely. The necessary medicine for the month which consists of 28 printlets takes less than eight minutes to prepare, which is much more efficient than the manual method typically employed. The study found the printed tablets were more effective at making the patients blood concentrations closer to the isoleucine target values, making it more reliable than the manual method.
3D printing provides rare diseases with great potential, allowing the medication to be as personal as the disease.