A small Canadian drugmaker is in the final stages of testing technology designed to treat sepsis, the surprisingly common condition that killed boxing legend Muhammad Ali.
If data from late-stage trial is positive, Spectral Medical Inc thinks it can win approval from the U.S. Food and Drug Administration as early as next year.
The technology has been in use in Japan and parts of Europe for years but has never been approved for wide use in the United States because, in the view of the FDA, it has never been tested in trial large enough to prove its effectiveness and safety.
Sepsis, derived from the Greek word for putrefaction, occurs when the immune system goes into overdrive to fight infection or bacterial toxins, triggering a cascade of physiological changes that can lead to multiple organ failure.
The leading cause of hospitalizations in the United States, it can result from a simple cut, routine surgery or infections associated with chronic disease - Parkinson's, in Ali's case.
Sepsis costs the U.S. healthcare system about $20 billion a year, according to the Sepsis Alliance, a San Diego-based patient advocacy organization.
Infants, the elderly and those with weakened immune systems are most at risk.
In extreme cases, a dramatic drop in blood pressure can lead to life-threatening septic shock.
This is where Spectral comes in.
The Toronto company holds North American rights to a system developed by Japan's Toray Industries Inc designed to restore blood pressure and correct organ dysfunction by using an antibiotic to detoxify the blood.
Once the patient's blood has been extracted it is passed through a column to remove a type of toxin, called an endotoxin, which is believed to be a major trigger for sepsis.
The system allows you to "take the blood, clean the blood and return the blood," said Dr. Claudio Ronco, an Italian kidney specialist who has used the system extensively during more than two decades in the field.
Ronco said he has no financial ties to Spectral and has received only honorariums for speaking about his experience with the treatment at industry-sponsored symposiums embedded in scientific meetings, once by Toray.
"Even if the trial in the United States is not positive ... I will treat my patients with this device, because we have seen it works," he added.
Toray has been selling the Toraymyxin system in its home market since 1994 and through distributors in Europe since 2002. The system is also approved for use in Canada.
Still, data supporting use of the device is intensely debated, with trials conducted outside of Japan and Italy failing to prove its effectiveness, Cormark Securities analyst David Novak wrote in a research note last month.
Spectral Chief Executive Paul Walker told Reuters that a lack of major recognized clinical trials that proved efficacy beyond question had "definitely slowed adoption."
The company's late-stage U.S. data is expected by September.
In the meantime, the FDA has approved the "compassionate use" of the product in select hospitals.
To accompany the device, Spectral has created a diagnostic test that can determine whether a patient has high endotoxin levels. The FDA cleared the test in 2003 and it has been approved elsewhere since then.
About 68 percent of patients with Ali's condition - those that develop septic shock - have high endotoxin levels, said Walker, a medical doctor and co-inventor of the test.
There are no specific FDA-approved medicines for the treatment of sepsis, and there have been several high-profile clinical failures.
The only drug to win FDA endorsement - Eli Lilly & Co's Xigris - was pulled off the market in 2011, a decade after approval, when follow-up clinical trials showed its use did not reduce mortality. (bit.ly/1OnzgpK)
The U.S. Centers for Disease Control and Prevention estimates that up to half of the estimated 1 million annual cases of sepsis in the United States result in death.
Every year, about 350,000 patients in North America are diagnosed with septic shock. Even a 15 percent cut in the mortality rate would be sufficient to justify an estimated $20,000 price tag per treatment, Walker said.
Spectral - which has a market value of about $160 million but generated just $3.08 million in revenue in 2015 - hopes to capture about half of the estimated $3 billion market within five years of FDA approval, he said.
If approved, Cormark's Novak expects the product to achieve peak annual sales of more than $800 million.
(Reporting by Natalie Grover in Bengaluru; Additional reporting by Amrutha Penumudi; Editing by Robin Paxton and Ted Kerr)