In 2015, the FDA approved Spritam which is the first drug developed using the 3D printing technology. Previously known active substance levetiracetam is used for its manufacture, and it is used in the treatment of epilepsy. The tablets “printed” with this substance look as usual but they can almost instantly dissolve even in small portions of water.
The 3D printing technology makes it possible to develop tablets with exact content of the active substance, that is, designed for an individual patient with their individual needs. It also allows to add a variety of tastes into drugs, but it is rather a nice bonus to what such a serious technology can do.
“Nobel” RNA interference for the treatment of rare genetic diseases
In 1998, Andrew Fire and Craig Mello described the mechanism of RNA interference. This mechanism enables to control the activity of certain genes in the cells of animals, plants and fungi. In 2006, for their research, they received the Nobel prize in Physiology or Medicine. And in 2018 and 2019, the FDA approved two drugs, the effect of which is built on the same mechanism, and they are Patisiran and Givosiran.
Patisiran is intended for the treatment of familial amyloid polyneuropathy. The “mutated” form of the protein transthyretin is synthesized in people with this disease, which leads to the violations in the body function. At a certain stage Patisiran blocks the synthesis of the “wrong” protein and thus reduces these violations. Givosiran has the same principle helping to fight against another rare disease – acute renal porphyria. Probably, the mechanism of RNA interference will be used to cure some other genetic diseases.
In 2017, the FDA approved two drugs: Kymriah for the treatment of acute B-cell lymphoblastic leukaemia and Yescarta to treat certain types of lymphoma.
The drugs are developed using an individual patient’s own T-cells, one of the types of immune cells. A special gene is “inserted” in these cells that is responsible for the production of a particular protein, a chimeric antigen receptor (CAR). After that, genetically-modified T-cells are put back in the human body, where they use their new receptor to attack and destroy cancer cells. The cost of such therapy is approximately $400 thousand, it is used in cases when other methods prove ineffective.
