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It takes the Food and Drug Administration an average of nearly seven years to approve promising new anti-cancer drugs. For most terminally ill patients, that’s not nearly fast enough. Now patient advocates are taking the FDA to court in an effort to force the agency to streamline its approval process.

In late July, the Washington Legal Foundation sued the FDA and the Department of Health and Human Services in U.S. District Court on behalf of the Abigail Alliance for Better Access to Developmental Drugs, a Virginia-based advocacy group for terminally ill patients. The lawsuit contends that the FDA’s tortuous drug-approval process effectively denies terminally ill cancer patients access to experimental anti-cancer drugs, thereby violating their constitutional rights.

Alliance founder Frank Burroughs named the group after his daughter Abigail, who two years ago succumbed to cancer at age 21 after trying unsuccessfully to obtain access to two experimental anti-cancer drugs. The group’s lawsuit also details the struggles faced by other Alliance patients who were urged by their physicians to try experimental drugs after traditional therapies failed. None of the Alliance patients was able to get into the very limited group who participated in the drug companies’ clinical trials.

The lawsuit calls on the FDA to give special initial approval to experimental drugs that show effectiveness and to permit their sale and distribution to patients with no other approved treatment options. The FDA was withholding comment pending review of the lawsuit.

Individuals receiving treatment for cervical cancer, prostate cancer or colorectal cancer may find significant relief from the effects of radiation-induced proctopathy by taking oral vitamin A.

Radiation-induced anal ulcers characterized by diarrhea, urgency, rectal pain, rectal bleeding and fecal incontinence may occur six months or more after irradiation of prostate and pelvic malignancies. In a double-blind placebo-controlled trial, both male and female patients with radiation-induced anal ulcers were successfully treated with oral vitamin A.* The trial group consisted of 14 males and two females with a median age of 71. Of the enrolled patients, 13 had been treated for prostate cancer, two had been treated for cervical cancer, and one had been treated for rectal cancer. Eight patients were randomized for vitamin A (8000 IU twice daily) and eight patients were randomized for placebo. After three months, seven of eight patients (88%) had a significant reduction in symptom parameters based on Fisher’s Exact Test versus two of eight patients (25%) on placebo. Five nonresponders to placebo were then given the same therapeutic dose of vitamin A, and responded favorably to treatment. The researchers concluded that the vitamin A-treated test subjects showed a significant reduction in symptoms of proctopathy as compared to placebo. Improved rectal function and decreased bleeding were attributed to the wound healing and repair properties of vitamin A.

A major study of African-American stroke patients was halted early when aspirin was found to be just as effective as the prescription antiplatelet drug ticlopidine in preventing recurrent stroke, heart attack and vascular death.

African-Americans are at approximately twice the risk of having a stroke compared to other racial groups, yet are often underrepresented in clinical trials. For this reason, medical researchers formed the African-American Antiplatelet Stroke Prevention Study (AAASPS), headquartered at Rush-Presbyterian-St. Luke’s Medical Center in Chicago. The AAASPS program involved 1,809 African-American patients at more than 60 hospitals and medical centers in the United States.

The study was designed to determine the efficacy and safety of aspirin and ticlopidine in preventing recurrent stroke and other vascular events in African-American patients who had already experienced a noncardioembolic ischemic stroke. Of the 1,809 patients, 902 randomly received 500 milligrams (mg) per day of ticlopidine, while 907 patients received 650 mg per day of aspirin. The study was double-blind, in that neither the researchers nor the participants knew which medication they were taking.

Although the study was scheduled to run until October 2003, it was halted in July 2002 by the data and safety monitoring board appointed by the National Institutes of Health when statistical analyses showed that there was less than a 1% chance of ticlopidine being significantly better than aspirin if the study were to continue to completion. The analyses did indicate a 40% to 50% likelihood of aspirin being significantly better than ticlopidine in reducing the risk of recurrent stroke if the trial continued to completion.

“The decision of the data and safety monitoring board to stop the study was based on the potential futility of ticlopidine use for the primary study outcome end point and the small likelihood, but potential for serious adverse events among ticlopidine-treated patients,” stated the researchers in their article which was published in the June 11, 2003 issue of the Journal of the American Medical Association.*

After 6.5 years of follow-up, 106 patients in the ticlopidine group experienced a subsequent stroke, while only 86 patients in the aspirin group had a stroke. In addition, nine of the ticlopidine patients had a heart attack compared to only eight of the aspirin patients. Each group experienced 18 “vascular deaths” during the study period.

“We thought that ticlopidine would substantially be better than aspirin in reducing recurrent strokes, heart attacks and death. We did not find that,” said study author Dr. Philip Gorelick of Rush Medical College in Chicago.

In addition, the ticlopidine group experienced slightly more serious and non-serious adverse events than the aspirin group (29.9% versus 28.9% overall). Of the ticlopidine patients, 3.4% developed significantly low white blood cell counts, compared to only 2.2% of patients in the aspirin group. Diarrhea was reported slightly more often in the ticlopidine group than the aspirin group (0.3% versus 0.2%). However, major gastrointestinal tract bleeding occurred more frequently among the aspirin group (0.9% versus 0.4%).

Aspirin is much less expensive than ticlopidine. A month’s supply of ticlopidine could easily exceed $100, while aspirin costs only pennies per day. In addition, blood tests must be performed every two weeks during the first three months of use of ticlopidine, adding further to the expense and inconvenience.

“Aspirin is much less expensive than other major antiplatelet agents, is readily available, easy to use, and relatively safe,” concluded the researchers. “Head-to-head comparison with other agents indicates that it may be difficult to outperform aspirin as a stroke prevention therapy in some noncardioembolic ischemic stroke patients.”

“Our data call into question the superiority of the thienopyridine ticlopidine in black non-cardioembolic ischemic stroke patients, and suggest that ticlopidine is unlikely to be superior to aspirin for prevention of recurrent stroke and major vascular events in these patients. Furthermore, ticlopidine may have a less favorable and potentially serious adverse event profile. Therefore, aspirin is a reasonable first choice prevention agent in aspirin-tolerant black patients with noncardioembolic ischemic stroke.”

— Marc Ellman, M.D.