It’s easy to think about treating diseases in black and white terms – we want a cure, a silver bullet to set things right, but the reality of treatment can often be quite different, more akin to trench warfare than a Hollywood ending. It can take researchers hundreds of millions of dollars and a decade of research to win a precious few hard-won months, weeks, or even days. Progress can be incremental, slow, fleeting. Sometimes the cure proves as harsh as the disease itself, leaving patients and their healthcare providers to balance the risks with the potential reward.
Twenty-four patients made such a decision to try what IFLScience! calls an “exceptionally risky but pioneering technique“ carried out at three Canadian hospitals to treat their multiple sclerosis (MS), which was documented in a landmark study in The Lancet. The participants, aged 18-50, had been given a poor prognosis and were expected to be unable to walk within 10 years.
Highly active MS is sometimes treated by hematopoietic stem cell transplantation (HSCT), which suppresses the patient’s immune system with chemotherapy before reintroducing donor stem cells into the body. This treatment went further, totally destroying the patients’ immune systems via chemotherapy before rebuilding it using early-stage stem cells that “have not developed the flaws that trigger MS,” as the BBC explained.
Twenty-three of the participants responded successfully, and showed no new signs of the disease or relapses in the 13 years since their treatments. Study researchers praised this as the “first treatment to fully halt” MS, and noted “many of the patients had substantial recovery of neurological function despite their disease’s aggressive nature.” Tragically, the last patient died as a result of the aggressive chemotherapy, highlighting the risks of the treatment. This breakthrough is no silver bullet, and is estimated to only be appropriate for 5-10% of MS patients due to the danger. Dr. Mark Freedman, a neurologist at the University of Ottawa and the coordinator of the study, told The Guardian, “I hesitate to use the c-word. A cure would be stopping all disease moving forward and repairing all damage that has occurred.”
Freedman admits that the lack of a control group and the small sample size mean that large-scale clinical studies will be required to follow-up on the results. The patients from the original study will also need to be monitored to determine the long-term effects of the treatment. Still, some participants recovered sight, balance, and the ability to walk and drive, and “six returned to work or college, five married or became engaged and two had children using banked sperm or eggs, as the aggressive treatment had made them infertile.” This gives millions suffering from MS worldwide renewed hope that their condition can not only be treated, but stopped in its tracks. Although not universally suitable, this treatment can hopefully lead the way to further breakthroughs in the years to come.