NIH Hits the Brakes on Stroke Drug Trial Amidst Safety Concerns and Lab Investigations
In a plot twist that could rival a medical thriller, the National Institutes of Health (NIH) has slammed the brakes on a major human trial for a potential stroke drug, 3K3A-APC, amidst concerns over safety and alleged scientific misconduct. The drug, touted as a brain protector after strokes, now finds itself at the center of a dramatic saga involving whistleblowers, questionable data, and a suspenseful NIH investigation.
The rollercoaster began with a Science investigation that unveiled whispers of potential troubles with 3K3A-APC. Whistleblowers raised alarms about data from an earlier phase 2 trial, suggesting that rather than saving lives, the drug might be linked to increased deaths or disabilities in stroke patients. As if that weren't enough, allegations of manipulated images and data in lab studies supporting the drug's promise added an extra layer of intrigue.
NIH's response on November 16 was swift and decisive—halting the launch of the eagerly awaited phase 3 trial that was set to involve 1400 people recovering from acute ischemic strokes. The trial, funded by NIH and sponsored by ZZ Biotech, the brainchild of co-founder Berislav Zlokovic, now faces a suspenseful pause as investigations unfold.
Pooja Khatri, co-investigator of StrokeNet, the consortium overseeing the trial, admitted to Science that the NIH had indeed applied the brakes. ZZ Biotech's CEO Kent Pryor, caught in the crossfire, confirmed that the trial's startup activities were in limbo during the NIH investigation. The timeline for the probe's completion? Anyone's guess.
The potential blockbuster drug, 3K3A-APC, is a modified version of an enzyme called activated protein C (APC), developed by Zlokovic and team. Cellular and animal studies hinted at its prowess in reducing brain bleeding and cell death post-stroke, earning it a whopping $30 million commitment from NIH for the phase 3 trial.
But the whistleblowers, armed with data from the phase 2 trial, weren't singing its praises. Six deaths post-treatment with 3K3A-APC versus one in the placebo group raised eyebrows. Proponents argued statistical insignificance, but doubts lingered. The 113-page dossier delivered to NIH didn't just stop at safety concerns; it delved into decades-long suspicions of image reuse and manipulation in lab experiments, adding an extra layer of complexity to the unfolding drama.
Zlokovic, a central character in this medical drama, remains enigmatic, not responding to the crescendo of allegations. Anonymously, former members of his lab told Science tales of intimidation and pressure to align experimental findings with Zlokovic's hypotheses, painting a dark backdrop to the esteemed scientist's career.
As NIH and USC launch their own investigations, the fate of 3K3A-APC hangs in the balance. Will it emerge as the hero of stroke treatment, or will the safety concerns and lab investigations bury its potential success? As the curtain falls on this act, the medical world watches, breathless, for the next twist in this unexpected stroke saga.