Important Cancer News

Court Backs Experimental Drugs for Dying Patients

By Marc Kaufman
Washington Post Staff Writer
Wednesday, May 3, 2006; A09

Terminally ill patients have a constitutional right to obtain experimental drugs before the Food and Drug Administration has decided whether to approve them, a federal appeals court ruled yesterday.

Saying that dying patients have a basic "right of self-preservation," the court held that drugs that have passed the first phase of FDA review -- which determines whether a product is safe -- should be made available if they might save someone's life.

The 2 to 1 decision by the U.S. Court of Appeals for the District of Columbia Circuit overturned a lower court's ruling. The judges sent the case back to the district court for a full hearing and possibly a trial.

The case was brought by the Abigail Alliance for Better Access to Developmental Drugs -- which advocates making experimental drugs available earlier to dying patients -- and was argued by the Washington Legal Foundation, a public interest law firm that has frequently challenged regulatory agencies.

The appeals court found that "barring a terminally ill patient from the use of a potentially life saving treatment impinges on this right of self-preservation." It also agreed with the alliance that the Supreme Court's 1990 decision establishing a "right to die" in the case of Nancy Cruzan, a brain-dead Missouri woman who was ultimately disconnected from life support, applied in reverse in this case.

"If there is a protected liberty interest in self-determination that includes a right to refuse life-sustaining treatment, even though this will hasten death, then the same liberty interest must include the complementary right of access to potentially life-sustaining medication, in light of the explicit protection accorded 'life,' " Judge Judith W. Rogers wrote.

Judge Thomas B. Griffith dissented, saying that the ruling goes against the "expressed rule" of Congress and the president. "The majority's approach injects courts into unknown questions of science and medicine," he wrote.

In its opposition to the challenge, the FDA had said that the agency already has programs that make potentially lifesaving drugs available before final approval. In addition, it said that allowing large numbers of patients to take unapproved drugs could put many at unacceptable risk, even if they are terminally ill.

"We remain sympathetic to the desire of terminally ill patients to gain access to experimental treatments when they have exhausted other therapeutic options, and are exploring a number of new efforts to improve how we make investigational drugs available through expanded access programs," said Scott Gottlieb, deputy commissioner for medical and scientific affairs.

The Arlington-based Abigail Alliance was founded in 2001 by Frank Burroughs, whose 21-year-old daughter died of cancer. The family had tried to enroll her in a number of clinical trials of promising cancer medications but was rebuffed, and Burroughs decided to continue his fight to expand access to experimental drugs after her death. He said several of the potentially lifesaving drugs that his group sought to make available while they were still under regulatory review went on to be approved and widely used.

Burroughs said yesterday that he hopes the FDA will work with his group and others to make unapproved drugs more easily available to dying patients.

The alliance has sought access to medications that have passed Phase I of the FDA review, which generally involves studies of 20 to 80 patients and results in a determination of whether the drug is safe enough for further human testing. Phase II trials determine whether a drug is effective, and Phase III studies involve larger groups and give a better sense of whether a drug is safe and useful.

© 2006 The Washington Post Company

-- Update September 2004 --

Burzynski Research Institute Receives Orphan Drug Designation for Antineoplastons A10 and AS2-1 for Treatment of Brain Stem Glioma

Houston, TX, September 7, 2004 – Burzynski Research Institute (BRI) (OTCBB:BZYR) announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for its drug candidates Antineoplastons A10 and AS2-1 for the treatment of brain stem glioma.

The FDA’s orphan drug program is intended to encourage research, development and approval of products for diseases that affect fewer than 200,000 patients in the United States per year and provide a significant therapeutic advantage over existing treatments. If Antineoplastons A10 and AS2-1 receive approval from the FDA, orphan drug designation will provide Antineoplastons A10 and AS2-1 with seven years of market exclusivity following such regulatory approval. The designation also enables BRI to apply for clinical research funding, tax credits on clinical research and development expenses, a potential waiver of user fees associated with filing the marketing application and assistance from the Office of Orphan Product Development (OOPD) in guiding the drug through the regulatory approval process.

"We are pleased to receive the FDA's orphan drug designation for Antineoplastons A10 and AS2-1, an important step toward bringing this treatment to market" said Luke E. Lawson, Ph.D., Director of Regulatory Affairs of BRI. "Therapeutic options for brain stem glioma patients are rather limited. We are committed to accelerating, wherever possible, our efforts to help address the unmet medical need of these patients worldwide."

Burzynski Research Institute is a biopharmaceutical company committed to developing medicines for serious and life threatening diseases from genomics and naturally occurring compounds. Research and development efforts are focused on antineoplastons, autoimmune disease and HIV.

Dr. Robert Burdick Review

Seattle-area oncologist Dr. Robert Burdick reviewed the records of several of Dr. Burzynski's patients with brain tumors. His report states that the responses seen can only be attributed to treatment with antineoplastons.

Publications by Independent Scientists and Dr. Burzynski and Associates

Clinical Trials Data

- - - Results of Brain Tumor Trials - - -

(as of November 2001)

Over 100 clinical trials using different chemotherapy regimens have been conducted on recurrent brain tumors. The general consensus is that such tumors cannot be cured by chemotherapy, and the response rate is only modest. Complete responses are almost never seen, and the total response rate usually includes stable disease. Even though some of these studies report significant responses, the durations of improvement are short. Ultimately, almost all of these patients die from their brain tumors. Patients who are being treated with antineoplaston therapy often are the exception.

Antineoplastons have been used to treat neoplastic disease since the 1970s. Patients can tolerate administration of antineoplastons very well, for as long as approximately 10 years in one case.

The Burzynski Research Institute currently sponsors 72 different Phase II clinical trials with Antineoplastons A10 and AS2-1 in different forms of cancer. Five of these trials have reached the final point and proved antincancer activity in various types of malignant brain tumors.

CAN-1: Results in 35 Evaluable of 43 Total Patients

Complete response (CR) 25.7% Progressive disease 20%

Partial response (PR) 22.9%

Stable disease (SD) 31.4%

CR + PR (objective response) 48.6%

CR + PR + SD 80%

The largest group of patients was diagnosed with glioblastoma multiforme and the second largest with anaplastic astrocytoma.

BT-9: Phase II Study of Primary Brain Tumors in 13 Evaluable of 20 Patients

Complete response (CR) 23.1% Progressive disease 7.7%

Partial response (PR) 30.8%

Stable disease (SD) 38.5%

CR + PR (objective response) 53.8%

CR + PR + SD 92.3%

Most of the patients were diagnosed with astrocytoma.

BT-11: Phase II Study of Brain Stem Gliomas in 18 Evaluable of 25 Patients

Complete response (CR) 16.7% Progressive disease 33.3%

Partial response (PR) 22.2%

Stable disease (SD) 27.8%

CR + PR (objective response) 38.9%

CR + PR + SD 66.7%

The patients treated were diagnosed with brain stem glioma.

BT-13: Phase II Study in Children with Low-Grade Astrocytoma in 8 Evaluable of 9 Patients

Complete response (CR) 37.5% Progressive disease 0%

Partial response (PR) 25.0%

Stable disease (SD) 37.5%

CR + PR (objective response) 62.5%

CR + PR + SD 100%

The patients treated were children diagnosed with astrocytoma.

BT-15: Phase II Study in Adult Patients with Anaplastic Astrocytoma in 16 Evaluable of 23 Patients

Complete response (CR) 12.5% Progressive disease 37.5%

Partial response (PR) 12.5%

Stable disease (SD) 37.5%

CR + PR (objective response) 25.0%

CR + PR + SD 62.5%

The patients treated were diagnosed with anaplastic astrocytoma which did not respond to radiation therapy and/or chemotherapy.

BT-18: Phase II Study of Mixed Gliomas in 11 Evaluable of 14 Patients

Complete response (CR) 27.3% Progressive disease 45.4%

Partial response (PR) 9.1%

Stable disease (SD) 18.2%

CR + PR (objective response) 36.4%

CR + PR + SD 54.6%

The patients treated were diagnosed with mixed glioma.

Adverse Reactions to High-Dose Treatment

Almost all patients experience increased urine output and slight thirst. The treatment usually is free from adverse reactions or is associated with mild side effects. Moderate side effects (Grade 2 by NCI criteria) include fluid retention, hypernatremia, hypocalcemia, hypokalemia, hypomagnesemia, nausea and vomiting, elevation of SGPT, leukopenia, allergic skin rash, fever, chills, headache, tinnitus and decreased hearing, decreased and blurred vision.

Serious adverse reactions (Grades 3 and 4 by NCI criteria) were observed in only a small number of cases and included high serum sodium levels (0.9%); low calcium levels (0.6%); low magnesium levels (0.3%); low potassium levels (0.3%); elevations of serum bilirubin (0.3%), SGOT (0.2%), and SGPT (0.3%); and thrombocytopenia (1.1%). It is suspected that in many cases, neurologic toxicity, visual toxicity, and ototoxicity resulted from the brain tumors. Serious thrombocytopenia occurred in only one patient who received combination chemotherapy 6 weeks before administration of antineoplastons, which could have contributed to bone marrow suppression. Generally, adverse reactions were fully reversible.

NOTE: Most patients who come to Dr. Burzynski already have failed at least one type of therapy and many have failed more than one type. Many of these patients are considered to be in the final stages of cancer and have no remaining conventional treatment options available to them.