MIT Stroke Drug Could Top MarketBy A. Arif Husain
An MIT-patented drug has shown evidence of potentially dominating the stroke treatment market by reducing disability after illness by as much as 50 percent.
Stroke, which is the third leading killer in the United States, presently has no reliable treatments, said Professor of Brain and Cognitive Science Richard J. Wurtman, who proposed use of the drug.
Wurtman, who is also director of the Clinical Research Center, began studying the biochemistry of the compound about 10 years ago.
The Technology Licensing Office could not predict MIT's royalty earnings on sales of the drug, although the San Francisco-based investment firm Montgomery Securities, which specializes in biotechnology, predicts $300 million in sales in its first five years.
Investigators have shown that not only did more than twice as many patients fully recover from their strokes compared with placebo trials, but also cognitive functions of patients receiving the drug were significantly improved, Wurtman said.
Since the publication of those findings by Interneuron Pharmeceuticals, which Wurtman co-founded, Wurtman and his group have been able to show how the drug is metabolized in the body and how the breakdown products are useful in the treatment of stroke.
MIT owns rights on the drug as a treatment for stroke and other brain injuries, but the patent is sublicensed with Interneuron. The company has exclusive development and commercialization privileges of the drug in the U.S. and Canada.
The compound is not yet approved for public use, but is "on a fast track through the [Food and Drug Administration]," Wurtman said.
"Until now, there has been very, very little progress in dealing with stroke and I'm very happy that this drug seems to make a difference," Wurtman said.
Drug seems to have few side effects
A stroke occurs when an area of the brain is deprived of blood supply and oxygen, either by a blood clot or due to excessive bleeding. Brain cells beyond the blockage begin to deteriorate and die, and deficiencies in brain functioning are observed.
Citicoline counteracts the effects of stroke in three ways. First, it provides the precursor molecules needed for damaged cells to repair their own membranes, which slows down deterioration and further cell death.
Second, by stimulating membrane repair, harmful by-products of the initial deterioration are cleaned up and prevented from doing more damage.
Third, the drug boosts the production of the brain chemical acetylcholine, which is thought to be involved in memory.
The leading drug, TPA, is targeted at breaking up blood clots. It is effective only if given within three hours of the onset of symptoms, and has "very, very high incidence of fatal side effects," Wurtman said.
Citicoline, which can be given up to 24 hours after a stroke, suffers only two side effects, both trivial, Wurtman said. The drug can cause dizziness, but only at radically high doses. Also, patients on the drug tended to fall down more often than those given placebo, though Wurtman attributed the effect to increased activity among the treated patients.
The results of the large-scale 259-patient clinical trial were presented last month at the annual meeting of the American Academy of Neurology in San Francisco.
In a separate study which will be printed next month in the Archives of Neurology, Paul Spiers, a visiting scientist in the Clinical Research Center, showed that citicoline improved memory in normal adults with lower than average memory abilities.