"Over 90% of the global population can't afford protein drugs, like insulin, due to the expense of production and the required refrigeration for storage or transportation. I am determined to challenge this scenario," says Penn Dental Medicine's Dr. Henry Daniell, who has been working to overcome these obstacles through a plant-based system to make shelf-stable drugs.
He and his colleagues are making great strides -- showing for the first time, the commercial viability of large-scale production of a low-cost drug made from whole plants. In a recent study published in Biomaterials, they confirmed efficacy of their method for FDA approval and human use, producing a drug that promotes tolerance to clotting factors, which could be taken by hemophilia patients, using freeze-dried lettuce capsules.
"This is a milestone in our field, to make a fully functional drug in plants, produce it at a large scale and in quantities sufficient for human clinical trials," Daniell said.
Daniell, professor in Penn Dental Medicine's Department of Biochemistry, is senior author on the study. It builds on previous work by Daniell using genetically modified plants to introduce a protein into the body that would teach the immune system to tolerate clotting factors that are given as a treatment for hemophilia.
Normally, 20-30% of people who get infusions of clotting factor develop antibodies against them that interfere with treatment. The earlier study successfully stopped and even reversed the production of these clotting factor inhibitors by feeding the plant-based drug to mice with hemophilia A.
That study used a tobacco plant platform to "grow" the drug. To take this approach into humans, however, Daniell's team turned to lettuce, identifying the genetic vector to introduce the therapeutic gene into the plant cell's DNA and grow the drug within the lettuce leaves, which are then freeze-dried and encapsulated.
Two different growing systems were used -- one in Daniell's greenhouse, a high-tech facility that grows the plants in soil, using natural light; the second was the Fraunhofer USA facility, which more closely replicates how a commercial pharmaceutical production facility would run, using a hydroponic system and artificial lighting. They determined they could produce 36,000 doses in just 1,000 square feet and harvest a new batch of pharmaceutical-containing lettuce every 4-6 weeks.
"This changes the way we think about delivering protein-based drugs and making them affordable to the global population," said Daniell.
For more information
Penn Dental Medicine
Beth Adams
T: +1 215-573-8224