Katie Jackson desperately wants a new treatment for Huntington’s disease. Her husband died from the devastating brain disorder. And because the disease runs in families, her three children have a 50% chance of inheriting the condition. She’s pinned her hopes on a cutting-edge gene therapy from UniQure NV.
But Jackson says Huntington’s patients have no desire to meet a new demand from the US Food and Drug Administration: to enroll in a new clinical trial where some people will undergo fake brain surgery without getting UniQure’s treatment.
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“It is inconceivable to us,” said Jackson, chief executive officer of Help 4 HD International, a Huntington’s advocacy organization. “Subjecting participants to invasive procedures without the prospect of therapeutic benefit is unjustifiable.”
Performing a fake surgery would involve patients undergoing anesthesia for about 10 hours. … They would also have their head shaved and have a hole drilled into their skull without going through the bone.
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Depending on how long the trial goes, the patients in the placebo group may not be eligible to get treated because their disease might have progressed.
