One of the concerns these days about self-assembling nanotechnology is that it is so advanced beyond the current drug paradigm that it becomes problematic from a regulatory point of view.
Let’s say, for example, that you have a patient admitted to an oncology ward. The doctor decides to get a biopsy to assess the nature of the tumour. The current next steps would be a microscopic analysis of the biopsy followed by chemotherapy/radiotherapy if a cancer is detected. The story is very different with advancing nanotechnology. The patient would have his biopsy taken but instead of analysis with microscopy, the tissue would be ground up and its DNA extracted. The DNA would be sequenced using nanopore technology within 5 minutes (see Oxford Nanopore Technologies for more info). The sequence would then be characterized against the patient’s healthy tissue DNA for comparison. The mutations that have arisen in the tumor are then catalogued and turned into a work order which computes a targeted smart drug for each mutation. These smart drugs are made and placed into a DNA nanoliposome and shipped to the oncology ward for that patient. All of the most pertinent mutations are addressed upon delivery and the cancer cells are forced either to self-destruct or are gene-edited to transform into non-cancerous cells. The healthy tissue around the tumor is never affected, unless it contains some residual pre-cancerous mutations which are corrected along with the cancerous cells.
Sounds great doesn’t it?
Well, what about the testing that goes into this personalized drug? Since this drug was tailored for only one person, it would be entirely useless to give to anyone else. This makes drug trials a one person show. I think I have a few answers for this.
Firstly, the smart drug delivery vehicles themselves can still be tested for toxicity without smart drugs in them. Secondly, the responsibility for any treatment would fall on the doctors that prescribe them with the caveat that the patient would need to sign a waiver for “experimental medicine”. Thirdly, molecular medicine needs to be its own specialization within the medical practice just to be able to keep up with DNA nanotechnology advancements. We are talking about doctors focusing their entire study on molecular medicine in medical school with a bit of human physiology thrown in for context. Finally, the FDA needs to recognize fundamental changes taking place in medicine and that a new personalized protocol needs to be established for ensuring that smart drugs are safe.
Certainly the old paradigm of lengthy and costly drug trials is in danger. This makes things really interesting. If the cost of sending smart drugs to market is much lower than the classic clinical trial series drug, it could mean a lot more competition in the future from smaller players.