Doctor has been in remission for FIVE YEARS after testing drug on himself 

A doctor with a potentially fatal condition has been in remission for five years after testing an experimental treatment on himself.

Dr David Fajgenbaum, who works at the University of Pennsylvania, was diagnosed with an aggressive form of Castleman disease in 2010, aged 25. It triggers an abnormal overgrowth of cells of the lymph system, which can cause multiple organ failure.

His form of the condition, called idiopathic multicentric, kills around a third of patients within five years, according to figures.

But he is still alive today, after gambling on a drug called sirolimus. His technique also helped two other patients go a year-and-a-half without a flare-up.

Dr David Fajgenbaum, who works at the University of Pennsylvania, was diagnosed with an aggressive form of Castleman disease in 2010. It causes an abnormal overgrowth of cells of the lymph system, which can cause multiple organ failure

Dr David Fajgenbaum, who works at the University of Pennsylvania, was diagnosed with an aggressive form of Castleman disease in 2010. It causes an abnormal overgrowth of cells of the lymph system, which can cause multiple organ failure

His form of the condition, called idiopathic multicentric, kills around a third of patients within five years, according to figures (it is unclear who he is pictured with at the 4th Annual RARE Tribute to Champions of Hope Gala in September 2015)

His form of the condition, called idiopathic multicentric, kills around a third of patients within five years, according to figures (it is unclear who he is pictured with at the 4th Annual RARE Tribute to Champions of Hope Gala in September 2015)

Dr Fajgenbaum’s own research into the condition, prompted by his diagnosis, suggested it may work by blocking a crucial pathway.

Blood tests he had taken along the way showed a spike T cells before suffering a flare-up, which includes fatigue, fever and night sweats.

And they also revealed an increase in the levels of a protein called VEGF-A. Both can be kept under control through the PI3K/Akt/mTOR pathway.

Sirolimus, or rapamycin, works through this same mechanism, which gave Dr Fajgenbaum enough hope that it could help him.

He decided to test the drug on himself, after consulting with Dr Thomas Uldrick, senior author of the research and Dr Fajgenbaum’s physician.

And now he has reported his remission – and that of two other patients taking the same drug – in the Journal of Clinical Investigation.

Sirolimus is already available for the treatment of other conditions, particularly to prevent organ rejection after kidney transplantation.

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