Pictures of Judah Folkman: http://www.usc.edu/hsc/info/pr/1vol3/320/folkman.html

http://www1.od.nih.gov/wals/folkman.html

Two Patients Step Forward With Encouraging Results: Folkman's Therapy Keeping Their Tumors in Check

By Susie Davidson

Advocate Correspondent

BOSTON - Who among us hasn't silently kvelled in advance, even though they know it’s way premature, about the possibility of a Jew maybe, maybe curing cancer?

That possibility might have taken a tiny step forward this past Friday, when two Boston patients successfully undergoing Endostatin therapy spoke before a panel of drug investigators in Maryland. Endostatin, the nervously-monitored, possibly breakthrough anticancer drug, was produced in Judah Folkman's Children's Hospital lab.

Nearly forty years ago, Harvard Medical School Professor of Surgery Folkman developed a treatment in response to his theory that tumors require blood vessels to thrive (a process called angiogenesis). 54 drugs are currently being tested around the world in an attempt to halt this blood supply and ideally, starve the tumors out of existence. Early lab results in mice were nothing short of astounding as tumors shrunk dramatically. 82 patients, all with end-stage, advanced cancer, have taken Endostatin since 1999, with mixed results. In Boston, four patients remain in this first stage, which has mainly been to test for side effects (none yet). Generally, Phase III is where effectiveness is on trial.

The end of the first round of testing has brought some disappointment for both patients and investors, as results have not been as spectacular as had been hoped. However, its safety has been definitively observed, especially as compared to the standard chemo-radiation barrage. And it has shown undeniable promise. Some tumors indeed stabilized (as in the case of these two patients, one who had advanced pancreatic cancer and has now outlived a dire prognosis by several months). Folkman himself lauded its record of safety over that of any other drug in his experience.

Further, the drug should be aptly viewed as therapy rather than cure, and in this light, it has resoundingly succeeded. Most cancer researchers hold as a goal a manageable stabilization of the disease, not unlike a basically livable, chronic condition.

In March, Dr. Edward Gubish, Executive Vice President of Research and Development at EntreMed, Endostatin's licenser, expressed confidence in the results thus far at the American Association for Cancer Research's annual meeting in New Orleans: "We are pleased with the Endostatin data presented at this conference….The latest data bolster Endostatin's potential as a therapeutic candidate for a wide variety of cancer patients."

What may be most exciting is the fact that as dosage has increased, results seem to have improved, with still no adverse side effects. And, Endostatin appeared for the first time to slow bloodflow to tumors, in humans, on computer images - doing just what Folkman had designed it for. Perhaps, as doctors envision, in both less sick individuals and in cohort with standard therapy, well, who knows…?

A second, more comprehensive, critical round of testing is set to commence during this summer, involving dozens of Bostonians. It will focus on the specific types of tumors which have shown good response thus far.

Folkman, a tireless, workaholic crusader for cancer research known for his extreme humility and personal attention to cases (he phoned this writer's home very late one evening on a wrong number, asking for a patient who had called about discomfort), is the very picture of dedication and resolve. Subject of a recent PBS Nova documentary, recipient of myriad medical awards and distinctions, he still leaves it to others to comment as he plows onward.

"The importance of angiogenesis is so obvious when you think about it," says Isaiah Fidler of the M. D. Anderson Cancer Center in Houston.

"The world owes Judah Folkman a debt of gratitude."