This is a great story: At the age of six, Mila Makovec was diagnosed with a devastating neurological disorder called Batten disease. There was no known treatment for her fatal condition. Then, in a dramatic demonstration of the power of personalized medicine:
… doctors in Boston created a treatment just for her. In only eight months, they found the genetic cause of Mila’s disease, designed a drug to overcome the error, and began giving it to Mila via an injection in her spine, in what’s believed to be the first individually tailored treatment of its kind.
In January, 2018, Mila became the first person in the world to receive a drug customized for just one person. It was named Milasen. Twenty months after Mila started receiving the drug, her mother and doctors say that the personalized treatment was partly successful.
Scientific results has just been published this week:
We describe how molecular diagnosis of a rare, fatal neurodegenerative condition led to the rational design, testing, and manufacture of milasen, a splice-modulating antisense oligonucleotide drug tailored to a particular patient. (…) This study offers a possible template for the rapid development of patient-customized treatments.Kim, Jinkuk, et al. ‘Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease’. New England Journal of Medicine, vol. 0, no. 0, Oct. 2019, p. null. Taylor and Francis+NEJM, doi:10.1056/NEJMoa1813279.
This is the apex of personalising medicine: a unique drug, probably suitable only for a single patient. A pill just for you.
However, there is catch. The treatment has been funded, among others, by Mila’s Miracle Foundation. In 2017, Mila’s family set out on a mission to find a cure for our daughter Mila’s devastating condition. The cost of the treatment is in the millions of dollars, well beyond the possibilities of the vast majority. They run a successful crowdfunding campaing. The whole story closely resembles the success/failure stories about innovation I have been discussing in this blog for years. And therefore the (partial) success raises a lot of questions about the future of medicine.
Does Mila’s story show us the way? My guess is yes. We cannot and should not prevent this model from happening. It will do, it will help the winners, maybe also others. It will help us find new models for medicine. It will very likely brings a serious backlash. It will be unavoidable, because we are not prepared and we are not preparing ourselves to deal with our society’s increasing complexity.
I am not judging, only trying to anticipate. I would like to hear other ideas. What do you think?
Featured Image: TanekaBradt, Morpheus The Matrix Neo Red Pill And Blue Pill