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Novel Nuclear Therapy a Test Case for Expedited FDA Approval With PM-FDA Battle

August 30, 1994

WASHINGTON (AP) _ Dr. Michael Werner stood before Congress and pleaded with U.S. regulators to let dying brain tumor victims have a shot at a stunning Japanese therapy that saved his life.

″Americans need this - fast,″ said the Wyoming physician.

Somebody at the Food and Drug Administration was listening. Just nine months after Werner paid Japan $60,000 to undergo its Boron-Neutron Capture Therapy, BNCT is emerging here.

Doctors plan to treat patients in two nuclear reactors in September, under a special FDA compassionate-use provision, and FDA regulators are actually meeting with other scientists to help speed their work.

″They agreed to waive a lot of the normal barriers,″ said Robert Zamenhof, a Massachusetts Institute of Technology physicist. ″They’re pushing it.″

It’s part of FDA’s plan to surmount its greatest criticism - long delays - by streamlining approvals for lifesaving treatments and even helping scientists gather the right data before they file the first FDA application.

″People tend to focus just on FDA time. We think we can shorten the whole time it takes to get a device through″ the pipeline, said FDA’s Dr. Susan Alpert.

Will it work? Critics are skeptical, noting that FDA is strengthening some regulations even as it claims to become more user-friendly. And immense pressure from desperate patients and their politicians forced the support only after Japan had done the procedure for 20 years.

But Zamenhof says if FDA can expedite BNCT - an incredibly complicated treatment involving new drugs, neutron beams never used on people and massive coordination with nuclear regulators - it ought to work anywhere.

BNCT was created by a U.S. doctor to treat an always lethal brain tumor called glioblastoma multiforme, which strikes 7,000 Americans a year. Doctors cluster boron inside the tumor and then pulverize it with a boron-targeted neutron beam.

But U.S. tests failed in the 1950s so the government abandoned BNCT. A Japanese medical student took the idea home, treated 130 patients and claimed a 20 percent success rate.

U.S. researchers aren’t sure the late Dr. Hiroshi Hatanaka’s success was really so high because he never documented his work scientifically. But his method excited them enough that:

-MIT in two weeks plans to radiate a melanoma patient to find a safe but potent neutron level, and then pursue the more fragile brain tumor patient.

-Long Island’s Brookhaven National Laboratory expects approval to treat its first brain tumor patient next month.

Neither reactor is optimal, so Georgia Tech is busy refitting what researchers call the best reactor for humans.

And scientists at 20 other sites, from the Idaho National Energy Laboratory to private companies like the Idaho Brain Tumor Center, are busy making plans to treat hundreds.

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