Unproven Treatment for Diabetes Draws Patients Who Won't Wait for Approval
RALPH T. KING JR.
Oct. 03, 1995
Diabetic Toby Warbet quit her secretarial job last year because of physical problems, including blurred vision and a general loss of sensation. Such was her desperation that when she heard about an unproven treatment that might help her, she decided to borrow $20,000 from relatives to pay for it.
Since the treatment _ an injection of fetal and rabbit pancreatic cells flown in from Russia _ two months ago, Ms. Warbet says her improvement has been ``subtle.'' Still, she remains hopeful. ``Even if the chances are one in a million, I was hoping I would be the one,'' says the Livingston, N.J., resident.
Some experts believe Ms. Warbet's chances may not be even that good. Loran Medical Systems Inc., which provided the treatment, claims effectiveness based largely on 1993 experimental data. But the scientist who directed that trial terms the results ``not clinically significant,'' and they haven't been published in a peer-reviewed journal. Nor does Loran tell prospective patients that it is not complying with a Food and Drug Administration advisory, contained in a letter to Loran last April, that the treatment must be approved by the agency before any injections are given.
Unorthodox medical procedures often prove irresistible to seriously ill people. Occasionally, they help in ways that accepted therapies don't. Other times, they do nothing or even go awry. But with government-funded research drying up, medical authorities say, more patients are trying experimental treatments without proven benefit or FDA approval. Especially vulnerable are the nation's 800,000 insulin-dependent, or Type 1, diabetics, who must keep an almost hourly vigil to defend against complications leading to blindness, kidney failure or amputation.
The trend raises questions about the FDA's ability to protect the public. Loran, for example, is ignoring the agency. Over the past four months, it has given one-time treatments to 24 people for a total tab of nearly $500,000, and it is planning to treat 72 more within seven months. It maintains that what it's injecting doesn't fall under the definition of a drug and thus is not subject to FDA regulation. It adds that it sent a letter to the agency arguing its case in May and hasn't received any response. The FDA says it's investigating but hasn't intervened.
``There are an extraordinary number of red flags up on this,'' says Darrell Wilson, a diabetes specialist at Stanford University Medical Center, who warned several patients not to take the treatment.
J.C. Wheeler, Loran's president, defends the treatment. ``I would not do anything that I had any inkling would injure anyone.'' Loran is trying to provide access to a therapy that is working, he says, one that has generated no profit for the company to date. ``I don't believe that we are giving anyone false hopes,'' he adds.
The idea behind pancreatic-cell injections appears promising. In Type 1 diabetics, the pancreas doesn't make enough insulin to control the body's use and storage of sugar. Add thousands of insulin-producing cells, and the body should be able to perform this function.
The chief obstacle: The immune system often disables the foreign cells. ``Past experience with well over a hundred fetal-cell transplants shows that while they may work initially, almost all fail within a year,'' says Frank Vinicor, president of the American Diabetes Association.
Loran has sidestepped skepticism in the scientific community and taken its case directly to the public. A front-page article in the Los Angeles Times last April described a clinical trial, conducted at Sansum Medical Research Foundation in Santa Barbara, Calif., in collaboration with Loran, as ``a marked improvement over previous U.S. trials.'' The breakthrough? In a technique developed by Russian researchers, the cells of human fetuses were blended with those of newborn bunnies to try to neutralize the immune response.
The story was based on a news release issued by Loran but not authorized by Sansum, says the nonprofit foundation's chief executive officer, Charles Peterson. ``It was a source of some embarrassment for us,'' Dr. Peterson says. ``The bottom line is we got (a result) that was statistically significant but not clinically significant'' enough to offer the product as a therapy. Afterward, Dr. Peterson says, he severed ties with Loran, didn't renew a contract of a Sansum staff member affiliated with Loran and abandoned the approach used in the trial.