U.S. President Barack Obama Proclaims September 2013 As National Ovarian Cancer Awareness Month — What Should You Know?

Yesterday, U.S. President Barack Obama designated September 2013 as National Ovarian Cancer Awareness Month. “… During National Ovarian Cancer Awareness Month, we lend our support to everyone touched by this disease, we remember those we have lost, and we strengthen our resolve to better prevent, detect, treat, and ultimately defeat ovarian cancer…. This month, we extend a hand to all women battling ovarian cancer. We pledge our support to them, to their families, and to the goal of defeating this disease. …”

Yesterday, U.S. President Barack Obama designated September 2013 as National Ovarian Cancer Awareness Month. The Presidential Proclamation is reproduced in full below.

During National Ovarian Cancer Awareness Month, Libby’s H*O*P*E*™ will honor the women who have lost their lives to the disease, support those who are currently battling the disease, and celebrate with those who have beaten the disease. This month, medical doctors, research scientists, and ovarian cancer advocates renew their commitment to develop a reliable early screening test, improve current treatments, discover new groundbreaking therapies, and ultimately, defeat the most lethal gynecologic cancer.

Let us begin this month with several important facts relating to ovarian cancer. Please take time to review these facts — they may save your life or that of a loved one.

Ovarian Cancer Facts

Lethality. Ovarian cancer causes more deaths than any other cancer of the female reproductive system.

Statistics. In 2013, the American Cancer Society (ACS) estimates that there will be approximately 22,240 new ovarian cancer cases diagnosed in the U.S. ACS estimates that 14,030 U.S. women will die from the disease, or about 38 women per day or 1-to-2 women every hour. This loss of life is equivalent to 28 Boeing 747 jumbo jet crashes with no survivors — every year.

Signs & Symptoms. Ovarian cancer is not a “silent” disease; it is a “subtle” disease. Recent studies indicate that women with ovarian cancer are more like to experience four persistent, nonspecific symptoms as compared with women in the general population, such as (i) bloating, (ii) pelvic or abdominal pain, (iii) difficulty eating or feeling full quickly, or (iv) urinary urgency or frequency. Women who experience such symptoms daily for more than a few weeks should seek prompt medical evaluation. Note: Several other symptoms have been commonly reported by women with ovarian cancer. These symptoms include fatigue, indigestion, back pain, pain with intercourse, constipation and menstrual irregularities. However, these additional symptoms are not as useful in identifying ovarian cancer because they are also found in equal frequency in women within the general population who do not have the disease.

Age. Although the median age of a woman with ovarian cancer at initial diagnosis is 63, the disease cancer can afflict adolescent, young adult, and mature women. Ovarian cancer does not discriminate based upon age.

Prevention. Pregnancy, breastfeeding, long-term use of oral contraceptives, and tubal ligation reduce the risk of developing ovarian cancer.

Risk Factors.

• BRCA Gene Mutations. Women who have had breast cancer, or who have a family history of breast cancer or ovarian cancer may have increased risk. Women who test positive for inherited mutations in the BRCA-1 or BRCA-2 gene have an increased lifetime risk of breast and ovarian cancer. A women can inherit a mutated BRCA gene from her mother or father. Women of Ashkenazi (Eastern European) Jewish ancestry are at higher risk (1 out of 40) for inherited BRCA gene mutations. Studies suggest that preventive surgery to remove the ovaries and fallopian tubes in women possessing BRCA gene mutations can decrease the risk of ovarian cancer.

• Lynch Syndrome. An inherited genetic condition called “hereditary nonpolyposis colorectal cancer” (also called “Lynch syndrome“), which significantly increases the risk of colon/rectal cancer (and also increases the risk of other types of cancers such as endometrial (uterine), stomach, breast, small bowel (intestinal), pancreatic, urinary tract, liver, kidney, and bile duct cancers), also increases ovarian cancer risk.

• Hormone Therapy. The use of estrogen alone menopausal hormone therapy may increase ovarian cancer risk. The longer estrogen alone replacement therapy is used, the greater the risk may be. The increased risk is less certain for women taking both estrogen and progesterone, although a large 2009 Danish study involving over 900,000 women suggests that combination hormone therapy may increase risk. Because some health benefits have been identified with hormone replacement therapy, a women should seek her doctor’s advice regarding risk verses benefit based on her specific factual case.

• Smoking. Smoking has been linked to an increase in mucinous epithelial ovarian cancer.

Early Detection. There is no reliable screening test for the detection of early stage ovarian cancer. Pelvic examination only occasionally detects ovarian cancer, generally when the disease is advanced. A Pap smear cannot detect ovarian cancer. However, the combination of a thorough pelvic exam, transvaginal ultrasound, and a blood test for the tumor marker CA-125 may be offered to women who are at high risk of ovarian cancer and to women who have persistent, unexplained symptoms like those listed above. This early detection strategy has shown promise in a 2013 University of Texas M.D. Anderson Cancer Center early detection study involving over 4,000 women. Importantly, another large ovarian cancer screening trial that is using similar early detection methods is under way in the United Kingdom, with results expected in 2015. The U.K. study is called “UKCTOCS” (UK Collaborative Trial of Ovarian Cancer Screening) and involves over 200,000 women aged 50-74 years.

Treatment.

• Treatment includes surgery and usually chemotherapy.

• Surgery usually includes removal of one or both ovaries and fallopian tubes (salpingo-oophorectomy), the uterus (hysterectomy), and the omentum (fatty tissue attached to some of the organs in the belly), along with biopsies of the peritoneum (lining of the abdominal cavity) and peritoneal cavity fluid.

• In younger women with very early stage tumors who wish to have children, removal of only the involved ovary and fallopian tube may be possible.

• Among patients with early ovarian cancer, complete surgical staging has been associated with better outcomes.

• For women with advanced disease, surgically removing all abdominal metastases larger than one centimeter (debulking) enhances the effect of chemotherapy and helps improve survival.

• For women with stage III ovarian cancer that has been optimally debulked, studies have shown that chemotherapy administered both intravenously and directly into the abdomen (intraperitoneally) improves survival.

• Studies have also found that ovarian cancer patients whose surgeries are performed by a board-certified gynecologic oncologist at a high quality/high surgical volume medical facility have more successful outcomes. Such medical facilities include National Cancer Institute-Designated Cancer Centers and member institutions of the National Comprehensive Cancer Network.

• Patients can enter clinical trials at the start of, during the course of, and even after, their ovarian cancer treatment(s).

• New types of treatment are being tested in ovarian and solid tumor clinical trials, including “biological therapy” and “targeted therapy.” For example, these types of treatment can exploit biological/molecular characteristics unique to an ovarian cancer patient’s specific tumor classification, or better “train” the patient’s own immune system to identify and attack her tumor cells, without harming normal cells.

Survival. 

• If diagnosed at the localized stage, the 5-year ovarian cancer survival rate is 92%; however, only about 15% of all cases are detected at an early stage, usually fortuitously during another medical procedure. The majority of cases (61%) are diagnosed at a distant or later stage of the disease.

• Overall, the 1-, 5-, and 10-year relative survival of ovarian cancer patients is 75%, 44%, and 34%, respectively.

• The 10-year relative survival rate for all disease stages combined is only 38%.

• Relative survival varies by age; women younger than 65 are twice as likely to survive 5 years (56%) following diagnosis as compared to women 65 and older (27%).

Help Spread the Word To “B-E-A-T” Ovarian Cancer

Please help us “B-E-A-T” ovarian cancer by spreading the word about the early warning signs & symptoms of the disease throughout the month of September.

B = bloating that is persistent and does not come and go

E = eating less and feeling fuller

A =abdominal or pelvic pain

T = trouble with urination (urgency or frequency)

Women who have these symptoms almost daily for more than a few weeks should see their doctor. Prompt medical evaluation may lead to detection at the earliest possible stage of the disease. As noted above, early stage diagnosis is associated with an improved prognosis.

__________________________________________________________

The White House

Office of the Press Secretary

For Immediate Release August 30, 2013

BY THE PRESIDENT OF THE UNITED STATES OF AMERICA

A PROCLAMATION

Each September, America calls attention to a deadly disease that affects thousands of women across our country. This year, over 22,000 women will develop ovarian cancer, and more than half that number of women will die of this disease. During National Ovarian Cancer Awareness Month, we lend our support to everyone touched by this disease, we remember those we have lost, and we strengthen our resolve to better prevent, detect, treat, and ultimately defeat ovarian cancer.

Because ovarian cancer often goes undetected until advanced stages, increasing awareness of risk factors is critical to fighting this disease. Chances of developing ovarian cancer are greater in women who are middle-aged or older, women with a family history of breast or ovarian cancer, and those who have had certain types of cancer in the past. I encourage all women, especially those at increased risk, to talk to their doctors. For more information, visit www.Cancer.gov.

My Administration is investing in research to improve our understanding of ovarian cancer and develop better methods for diagnosis and treatment. As we continue to implement the Affordable Care Act, women with ovarian cancer will receive increased access to health care options, protections, and benefits. Thanks to this law, insurance companies can no longer set lifetime dollar limits on coverage or cancel coverage because of errors on paperwork. By 2014, the health care law will ban insurers from setting restrictive annual caps on benefits and from charging women higher rates simply because of their gender. Additionally, insurance companies will be prohibited from denying coverage or charging higher premiums to patients with pre-existing conditions, including ovarian cancer.

This month, we extend a hand to all women battling ovarian cancer. We pledge our support to them, to their families, and to the goal of defeating this disease.

NOW, THEREFORE, I, BARACK OBAMA, President of the United States of America, by virtue of the authority vested in me by the Constitution and the laws of the United States, do hereby proclaim September 2013 as National Ovarian Cancer Awareness Month. I call upon citizens, government agencies, organizations, health care providers, and research institutions to raise ovarian cancer awareness and continue helping Americans live longer, healthier lives. I also urge women across our country to talk to their health care providers and learn more about this disease.

IN WITNESS WHEREOF, I have hereunto set my hand this thirtieth day of August, in the year of our Lord two thousand thirteen, and of the Independence of the United States of America the two hundred and thirty-eighth.

BARACK OBAMA

__________________________________________________________

Sources:

• Cancer Facts & Figures 2013. Atlanta: American Cancer Society; 2013 [PDF file].

• Presidential Proclamation — National Ovarian Cancer Awareness Month, 2013, Office of the Press Secretary, The White House, August 30, 2013.

Ovarian Cancer Survivor Seeks “Compassionate Use” Drug Exemption From BioMarin to Save Her Life

Andrea Sloan, a 7-year survivor of stage 3c ovarian cancer, is seeking a “compassionate use” exemption from pharmaceutical company BioMarin to save her life. Sloan is scheduled to start treatment at the M.D. Anderson Cancer Center on September 5 and would like to obtain access to the “PARP 1/2 inhibitor” drug known as “BMN 673” by that time. A robust grassroots campaign in support of Sloan has emerged on social media and Change.org in an effort to get an affirmative response from BioMarin.

Andrea Sloan, a 7-year, stage 3c survivor of ovarian cancer, is seeking a “compassionate use” investigational cancer drug exemption from pharmaceutical company BioMarin to save her life. Sloan, the executive director of a non-profit that advocates for survivors of domestic violence and abuse, now finds herself forced to publicly advocate for herself in a last chance effort to get the cancer treatment she needs.

Drugs that are being tested but have not yet been approved by the U.S. Food and Drug Administration (FDA) are called “investigational drugs.” These drugs are generally available only to people who are taking part in a clinical trial. The FDA “Expanded Access” protocol — sometimes referred to as a “compassionate use” exemption — involves the use of an investigational drug outside of a clinical trial to treat a patient with a serious or immediately life-threatening disease or condition, who has no comparable or satisfactory alternative treatment options.

FDA regulations allow access to investigational drugs for treatment purposes on a case-by-case basis for an individual patient, or for intermediate-size groups of patients with similar treatment needs who otherwise do not qualify to participate in a clinical trial. Before an investigational drug can qualify for compassionate use, the patient’s physician, the FDA, and the drug manufacturer must approve such use.

Unfortunately, drug manufacturers may not always be willing or able to provide access to a drug outside of their clinical trials. By law, drug companies are not required to make their drug available through the FDA expanded access protocol, or to make more of a drug for that purpose.

Andrea Sloan, a 7-year survivor of stage 3c ovarian cancer, is seeking a compassionate use exemption from pharmaceutical company BioMarin to save her life. Sloan is scheduled to start treatment at the University of Texas M.D. Anderson Cancer Center on September 5, and she would like to obtain access to the “PARP 1/2 inhibitor” drug known as “BMN 673” by that time. A robust grassroots campaign in support of Sloan has emerged on social media and Change.org in an effort to get an affirmative response from BioMarin.

“BioMarin’s BMN 673 offers me the best chance at a long life,” said Sloan. “My doctors and the FDA agree that I am an excellent candidate for this drug and meet the criteria for compassionate use exemption. However, BioMarin’s lack of a policy on compassionate use is preventing me from gaining access to the drug I need to save my life. I respectfully implore them to reconsider and make the ethical decision to help me.” [Emphasis added]

Sloan has endured two full rounds of chemotherapy, five surgeries, and a stem cell transplant. While her cancer remains responsive to treatment, her bone marrow can no longer tolerate traditional therapies. Her world-class oncology team at M.D. Anderson believes that BioMarin’s PARP inhibitor BMN 673, which is currently being tested in a phase I solid tumor clinical trial, is the best option for Sloan’s BRCA-1 gene-mutated form of ovarian cancer.

Unfortunately, Sloan hit a barrier in gaining access to BMN 673. Further enrollment of ovarian cancer patients in the phase I solid tumor trial is now closed, and the publicly announced portion of the trial that will be entering phase III testing is only open to BRCA gene-mutated breast (but not ovarian) cancer patients. Therefore, Sloan is left with the compassionate use exemption as her only option to access the drug she needs to fight her cancer. Based on FDA requirements, Sloan qualifies for the compassionate use exemption. Data emerging from the phase I BMN 673 study suggest that the drug is a safe and effective treatment option for patients with BRCA gene-mutated ovarian cancer.

Moreover, on August 16, 2013, BioMarin announced that its medical study abstract, entitled “PARP inhibition with BMN 673 in ovarian and breast cancer patients with deleterious mutations of BRCA1 and BRCA2,” has been selected as a “late breaking” abstract by the 17th ECCO — 38th ESMO — 32nd ESTRO European Cancer Congress, which will be held from September 27 through October 1, 2013 in Amsterdam, The Netherlands. BioMarin’s oral presentation at the European Cancer Congress (scheduled for September 29, 2013) will include data presented from 28 ovarian cancer patients with deleterious germline (inherited) BRCA gene mutations, including 17 patients from the phase I BMN 673 trial dose escalation cohort (range 100 µg to 1100 µg) and 11 patients from the dose expansion cohort. Presumably, the most recent ovarian cancer patient data to be presented at the European Cancer Congress will expand upon the positive data presented by the company at the 2013 Annual Meeting of the American Society of Clinical Oncology, which indicate that positive RECIST (“Response Evaluation Criteria in Solid Tumors“) and/or CA-125 ovarian cancer patient responses occurred at BMN 673 drug doses ≥ 100 µg/d in 11 out of 17 (64%) BRCA gene-mutated ovarian and peritoneal cancer patients. The positive RECIST medical imaging findings and the CA-125 blood test results highlight the promising effectiveness of BMN 673, albeit among a small group of ovarian cancer patients.

BMN 673 is a targeted therapy designed to disrupt the tumor without traditional chemotherapy drug side effects, thereby making it an optimal treatment for Sloan.

Despite Sloan’s best efforts, she has been unable to convince BioMarin to allow compassionate use of BMN 673. BioMarin, according to Sloan, has not been cooperative, merely citing their lack of a policy on the issue. Sloan, the executive director of a non-profit that advocates for survivors of domestic violence and abuse, now finds herself forced to publicly advocate for herself in a last chance effort to save her life. Sloan is committed to advocating for meaningful reform on this topic and hopes BioMarin will lead by example in starting a national dialogue.

If you would like to help Andrea Sloan obtain compassionate use of BMN 673, please click on this picture and sign her petition at Change.org.

For those interested in supporting Andrea Sloan, please sign her petition on Change.org that urges BioMarin to grant her a compassionate use investigational cancer drug exemption for BMN 673. Also, you can follow Andrea on Twitter at @andi_sloan.

Update:

Yesterday, BioMarin issued a statement to KXAN, an Austin, Texas local affilate of NBC, in response to a in-depth news story that KXAN aired tonight regarding Andrea Sloan’s dire situation. Effectively, BioMarin rejected Andrea Sloan’s request for compassionate use of BMN 673, although its statement was worded as a general drug “expanded access” policy explanation.

BioMarin acknowledged that it allowed preapproved expanded access to one of its investigational drugs which completed phase III clinical testing last year. In terms of general guidelines for its expanded access programs, the company stated:

“We implement these [expanded access] programs when we have sufficient scientific evidence to support both the safety and the efficacy of a product for an indication. Additionally, we implement these programs only when we can ensure that access will be provided equitably, ensuring that the process is appropriately blinded, and when we are confident that the expanded access will not inhibit our clinical trial plans or clinical trials for a disease generally.”

In terms of Andrea Sloan’s specific case, BioMarin stated that it does not comment on the status of individual patients. Apparently in a refusal to grant expanded access to any preapproved patient who requests compassionate use of BMN 673 prior to completion of phase III drug testing, the company stated:

“… However, we note that, although the current data [for BMN 673] that we have looks promising, there is no data at this point to support anything beyond dosing and some preliminary safety. It is too early to know if the experimental therapy is safe or effective, or will even prolong life, until we conduct the appropriate Phase 3 trials. The data that we have is from an ongoing early stage clinical trial, and it is the first trial that we have ever done with this therapy in humans.”

Accordingly, it appears that BioMarin’s current expanded access policy for its investigational drugs, such as BMN 673, will only extend to drugs that have already completed phase III clinical trial testing.

Sources:

• Ovarian Cancer Survivor Andrea Sloan Seeks Compassionate Use Exemption From BioMarin to Save Her Life, Press Release, Digital Journal, August 29, 2013.

• BioMarin Provides BMN 673 Program Update, BioMarin Pharmaceutical Inc, Press Release, July 25, 2013.

• De Bono JS et. al. First-in-human trial of novel oral PARP inhibitor BMN 673 in patients with solid tumors. J Clin Oncol 31, 2013 (suppl; abstr 2580).

• Shen Y. et al.  BMN 673, a novel and highly potent PARP-1/2 inhibitor for the treatment of human cancers with DNA repair deficiency. Clin Cancer Res. 2013 Jul 23. PMID: 23881923 [Epub ahead of print]

• A Phase 1, First in Human, Single-arm, Open-label Study of Once a Day, Orally Administered BMN 673 in Patients With Advanced or Recurrent Solid Tumors. ClinicalTrials.gov Identifier: NCT01286987.

• Advocate for others needs help in a fight for her life, by Shannon Wolfson & Joe Ellis, In-Depth Investigation, KXAN News, August 29, 2013.

• Statement From BioMarin on Expanded Access, August 28, 2013.

• BioMarin Announces Oral Presentation of BMN 673 Most Recent Data on Breast and Ovarian Cancers at the European Cancer Congress 2013, Press Release, August 16, 2013.