Manufacturing life-saving drugs might soon be as easy as preparing instant soup.
“It’s like making ramen: just add water,” said Peter Nguyen, one of the authors of the paper published in the journal Cell by a team of bioengineers at MIT and Harvard’s Wyss Institute.
The newly-developed system—white powder in a 1.5-inch-tall tube—is “unassuming,” Nguyen said, but has been able to produce a wide range of therapeutics—from antimicrobials to vaccines.
The system is based on freeze-dried pellets that contain the chemical building blocks needed to make molecules such as therapeutic compounds. Simply adding water to the pellets can start the production reaction.
This compact, portable system might help make drugs more accessible in far-off or low-resource areas such as developing countries, war zones, and even space stations, Nguyen said.
“Rapid access to vaccines in remote regions could change lives,” said Michael Jewett, a bioengineer at Northwestern University who was not involved in the study.
To create freeze-dried reaction pellets, the bioengineers opened up cells and pulled out the innards that translate genetic instructions into molecules. Then they idled these tiny biological machines by exposing them to cold temperatures and low pressure, which removed all of the water.
In the same way, the researchers freeze-dried DNA carrying the instructions for specific therapeutic molecules. The pellets whir to life when mixed with these DNA instructions and a drop of water and start producing therapeutics within minutes.
Freeze-dried pellets were able to produce several antimicrobial compounds and more than 50 types of antibodies—molecules that can identify and disarm invaders such as viruses, bacteria, and cancer cells. The pellets were used to produce antibodies able to neutralize breast cancer cells and Clostridium difficile, a bacterium that can cause life-threatening diarrhea and inflammation of the intestine.
The pellet-derived drugs were as effective as their commercial counterparts and could be safely ingested or applied to mice’s skin, depending on the compound.
The investigators also employed the tiny biological machines to manufacture a diphtheria vaccine, which triggered an immune response in mice comparable to the one seen with traditional diphtheria shots.
Other bioengineers look at the possibility of producing pharmaceuticals on demand with optimism. “This is an important first step for providing patient-specific treatments where treatments are not available now,” Jewett said.
Today, pharmaceutical companies rely on a few centralized factories that manufacture products and dispatch them to points of care such as clinics and hospitals. However, the products have to be transported and stored at low temperatures to preserve their therapeutic activity. This makes global distribution a challenging process, especially in areas with low resources and limited access to electricity.
The researchers hope that the pellet-based system will pave the way for moving the production of drugs and vaccines from manufacturing plants to hospitals, eliminating the challenges of transportation.
Freeze-dried pellets and DNA can be stored at room temperature for one year, and possibly even longer, either in the same tube or separated. According to co-author Shimyn Slomovic, they could be used as a “virtual pharmacy.” When needed, a therapeutic molecule could be rapidly synthesized by mixing pellets with DNA and adding water without the need for refrigeration, expensive equipment, or specialized staff. “This technology has the potential to vastly lower the costs of therapeutics production and distribution,” Slomovic said.
Some scientists believe there is still a long way to go. David Shoultz, head of drug development at the global health nonprofit PATH, said in an interview to STAT that it is unlikely that these technologies will allow “instant drug” or “instant vaccine” production in the next couple of years: “It’ll probably be more like 10 or 12 years. And we’re still going to need facilities, know-how, and people to implement them.”
According to Slomovic, these concerns are understandable. “We’re creating a technology for an infrastructure that doesn’t exist yet,” he said. Centralized production and global distribution would be ineffective in extreme situations such as disease outbreaks, armed conflicts, or even missions to Mars. In these scenarios, freeze-dried pellets would represent a valid alternative. Just add water.