A Chinese father took it upon himself to develop a cure for a rare genetic disease that his son was diagnosed with.
A father’s devotion: Xu Wei, a 30-year-old online entrepreneur with no prior college education, devoted his time to learning and developing medicine at home after his one-year-old son Haoyang was diagnosed with Menkes disease, reported South China Morning Post.
The disorder, which affects the cellular transport of copper, is associated with seizures, stunted growth, failure to thrive, unstable body temperature and intellectual disability, according to the National Organization for Rare Disorders.
Infants diagnosed with Menkes disease usually have a life expectancy not exceeding three years.
In his search for solutions to treat his son, Xu came across an experimental treatment for the disease in the U.S.
At the time, Cyprium Therapeutics and its licensing partner Sentynl Therapeutics were at phase one of clinical trials with CUTX-10, a copper histidine treatment for Menkes disease.
Having no access to the copper injections or alternatives left Xu with no choice but to find a way to develop a similar treatment on his own.
Taking matters into his own hands: Xu enrolled in public courses to study pharmacology and proceeded to read American research papers using translation software.
“Sometimes the translations did not make sense and I would check everything word-by-word,” Xu said. “It was complicated, but there were always some clues online.”
He found that creating the copper histidine treatment involves mixing water with copper chloride dihydrate, L-histidine and sodium hydroxide.
By taking the courses, Xu will be able to apply the correct quality control in the pharmaceutical drugs that he will eventually create.
Xu first approached a company to produce the experimental drug at its industrial plant using the research. The medicine was successfully made, proving that the concept has potential.
DIY medicine: Realizing that the agreement would be too expensive for him in the long term, Xu went on to purchase the necessary equipment worth 20,000 yuan (around $3,100) and created a lab in his own home.
After testing the copper histidine on rabbits and himself, he felt confident that it was ready for his son.
Still, Xu started Haoyang with small dosages, eventually working up to the standard dosage.
Xu then brought his son to the hospital for a check-up to make sure the treatment did not do any damage to the boy’s liver or kidneys.
Xu also went on to develop another drug, called elesclomol, which some studies suggested can also help treat Menkes disease.
His son, now aged two, remains unable to speak nor roll over on his own and can only consume liquid food.
Xu is now preparing to study genetic engineering as he believes genetic research will also help his son overcome the disease.
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