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.

“Smile, Open Your Eyes, Love and Go On.”

Today marks the 2nd anniversary of Libby’s death from ovarian cancer at the age of 26. Although the family healing process continues, we celebrate Libby’s life formally on this day to honor her memory, and remind ourselves that life is precious and should not be taken for granted.

Today marks the 2nd anniversary of Libby’s death from ovarian cancer at the age of 26. Although the family healing process continues, we celebrate Libby’s life formally on this day to honor her memory, and remind ourselves that life is precious and should not be taken for granted.  This day also reminds us that there is a considerable amount of work yet to be done in raising ovarian cancer awareness and finding a reliable screening test, and ultimately a cure, for this unforgiving disease.

As reported by the American Cancer Society earlier this month, the estimated number of newly diagnosed ovarian cancer cases and related deaths in the U.S. during 2010 will be 21,880 and 13,850, respectively.  Simply stated, a U.S. woman will die every 38 minutes from ovarian cancer in 2010. Cancer Research U.K. also reported this month that the 10-year ovarian cancer survival rate nearly doubled since the 1970s. Unfortunately, this much heralded statistical “doubling” represents an increase of the long-term ovarian cancer survival rate from 18% to only 35%. Ovarian cancer still remains the most lethal gynecologic cancer in women. I know that if Libby were alive today, she would say, “we must do better.”

Although the vast majority of visitors to this website never knew Libby, it is because of her that Libby’s H*O*P*E*™ was created and shared with the general public. What began as a family website used to exchange ovarian cancer and cancer-related information within the family during Libby’s illness, has rapidly become a global information resource for ovarian cancer survivors and their families after her death. It is my greatest hope that Libby would be proud of the following accomplishments achieved over the past two years, which are dedicated to her memory:

• Created Libby’s H*O*P*E*™ mission statement to be carried out by a future nonprofit, tax-exempt organization.

• Generated approximately 145,000 website visitors, from 60 countries around the world.

• Generated 5% to 10% of daily website visitors from major U.S. and international cancer centers and elite academic institutions actively engaged in cancer research.

• Established social media networks through Facebook, Twitter, Vodpod, YouTube, Friendfeed, and SocialVibe, which are used to disseminate important ovarian cancer awareness information, as well as stories of hope.

• Established a website library containing over 500 videos relating to ovarian cancer and cancer-related topics.

• Responded to approximately 700 ovarian cancer survivor (and family) general informational inquiries, which were answered within 96 hours of website posting or email receipt.

• Created Vox Populi website article features which provide the general public with a better understanding of how ovarian cancer impacts the daily life of a woman diagnosed with the disease and her family. These stories have been well-received by our readers as a source of inspiration and hope.

• Presented at the 2010 National Ovarian Cancer Coalition (NOCC) 6th Annual Women’s Health Expo, held on March 20th in the greater Washington, D.C. area.  The presentation was entitled, A Patient Advocate’s Perspective on the Importance of Ovarian Cancer Awareness and Helpful Online Resources.

• Highlighted in the Eyes on Advocacy section of the 2010 University of Washington Tumor Vaccine Group (UWTVG) quarterly (Winter) newsletter entitled, TVG Focus. The UWTVG is headed by Mary L. (Nora) Disis, M.D., a Professor of Medicine and Adjunct Professor of Pathology and Obstetrics & Gynecology at the University of Washington, and a Member of the Fred Hutchinson Cancer Research Center. Dr. Disis is a world-renowned cancer immunologist.

• Established a new working relationship with Women’s Oncology Research & Dialogue (WORD), a non-profit, tax-exempt organization dedicated to raising gynecological cancer awareness.  To promote this new relationship, WORD recently shot a video of Paul Cacciatore, the Libby’s H*O*P*E* founder.  In the video, Paul addresses the genesis of the website, the Libby’s H*O*P*E* mission statement, and why it is important for all women to educate themselves about the early warning signs of ovarian cancer.  WORD will be launching a new website before the end of 2010, and it is anticipated that this video will appear on both the WORD and Libby’s H*O*P*E* websites at that time.

• Established a new relationship with elite cancer researchers working on behalf of the British Columbia Cancer Agency.

“Remember Me”

Based upon instructions from Her Majesty Queen Elizabeth II (“Her Majesty”), a poem entitled, She Is Gone, was recited at the Queen Mother’s funeral, which was held in Westminster Abbey on April 9, 2002. The poem recitation sparked a glut of media interest because of its simple, upbeat nature – and mystery author, who was credited in the service program as “Anon” [i.e., Anonymous].  Apparently, Her Majesty found the poem while leafing through old memorial service books and she chose it to be read at her mother’s funeral, where it struck a chord with millions of mourners.

After the conclusion of the Queen Mother’s funeral, the BBC, The Times, and other U.K. media outlets took great effort to identify the author, with attributions going to, among others, Immanuel Kant and Joyce Grenfell. Eventually, it was discovered that the true author was Mr. David Harkins, who wrote the poem in 1981 while working at a bakery.  Mr. Harkins, who now works as an artist selling paintings over the Internet, said he “couldn’t believe his eyes” when he saw his poetry published in several newspapers after the funeral.

Quite shocked by all of the media attention, David Harkins sent the original manuscript of the poem to Prince Charles (of Wales), and St. James’s Palace replied thanking Mr. Harkins for explaining its origin. As it turned out, the poem was originally written by David Harkins in homage to an unrequited love. Mr. Harkins recalled: “I was 23 when I first met Anne Lloyd, my inspiration for the poem I called Remember Me.”  The reply received by David Harkins from the Prince of Wales’s office stated: “I have no doubt that it [Remember Me] will be reproduced on many occasions over the years to come. The Prince of Wales has asked me to send you his very best wishes.”

I chose to include Remember Me as part of our tribute to Libby for two reasons.  First, the poem is instructive as to how Libby would want all of us to continue on with our lives, energized by our loving memories of her.  Second, Libby would no doubt find great joy and humor in the fact that a talented baker from a small U.K. town became famous worldwide for his literary prowess rather than his pastries. The full text of Remember Me is provided below.

Remember Me

You can shed tears that she is gone

Or you can smile because she has lived

You can close your eyes and pray that she will come back

Or you can open your eyes and see all that she has left

Your heart can be empty because you can’t see her

Or you can be full of the love that you shared

You can turn your back on tomorrow and live yesterday

Or you can be happy for tomorrow because of yesterday

You can remember her and only that she is gone

Or you can cherish her memory and let it live on

You can cry and close your mind, be empty and turn your back

Or you can do what she would want: smile, open your eyes, love and go on.

— written by David Harkins, Silloth, Cumbria, U.K. (1981)

Did You Ever Hear An Angel Sing?

The inspirational story of Rhema Marvanne provides further proof that it is possible to “smile, open your eyes, love and go on,” after the death of a family member from ovarian cancer. It is difficult to believe that the life lesson highlighted by this touching story is provided to us through the example of a 7-year old child, albeit it a very talented one.

Rhema Marvanne was born on September 15, 2002.  Rhema lives with her father Teton Voraritskul, and a family pet dog named, “Mojo.” According to her father, Rhema began singing at the same time she began talking.

Rhema’s mother, Wendi Marvanne Voraritskul, loved Rhema with all of her heart. Wendi was diagnosed with ovarian cancer when Rhema was just 3 years old. Succumbing to the disease three years later, Wendi Marvanne died at the age of 36 on November 8, 2008.  According to Teton Voraritskul, most of Rhema’s memories with her mom were pleasant ones, but revolved around surgery, multiple chemotherapy treatments, sickness and struggle. Wendi was a strong believer in God and never complained about or questioned God during her illness. Teton explains that Wendi always encouraged those around her, even in the midst of her cancer battle. During Wendi’s final months, Rhema and Teton took care of her. Rhema spent almost every hour with her mother. When asked what her greatest accomplishment was prior to her death, Wendi simply replied, “Rhema.” A YouTube video featuring Wendi and her original songs is provided below. You can learn more about Wendi Marvanne’s life and music at www.jchouseofmercy.org.

It was less than a year ago that Rhema recorded her first song, Amazing Grace, and quickly became an Internet singing sensation. She obtains great inspiration from her mother, Wendi.  It is Rhema’s greatest hope to make her mother proud, both as a singer and as a “servant of God,” which is why she also performs for church congregations, non-profit organizations, charities, hospitals and special events.

Already a supremely talented singer, recording artist, and actor, Rhema counts the following among her recent accomplishments:

• Appeared on the Maury Povich show episode entitled, Maury’s Most Talented Kids 2010.

• Appeared on International News, via Supreme Master TV – Global Satellite, which first aired on June 16, 2010 in 44 countries (in 60 different languages).

• Recorded in Nashville with Gary Chapman and created her 1st music video featuring the song Amazing Grace.

• Recorded the song entitled The Prayer with Terry White and musicians from the Nashville Symphony, and is currently working on a Christmas album.

• Acted in 1st featured film entitled Machine Gun Preacher, a Lionsgate production set to release in Fall 2011. The movie Machine Gun Preacher is based upon the true story of Sam Childers (portrayed by well-known actor Gerard Butler), a drug-dealing biker who finds religion and dedicates his life to helping Sudanese children escape the Lord’s Resistance Army (LRA) in Africa. Childers founded the Angels of East Africa, a children’s village located in southern Sudan, for the children he saves from the LRA.

• Appeared on Daystar and Trinity Broadcasting television networks.

During her free time Rhema enjoys playing with her friends and dolls, and loves to watch movies. The best description of Rhema is provided by her father:

…The best way to describe Rhema is that she has a beautiful heart and soul. She is sweet, kind, caring and most importantly pure in heart. Most people who have dealt with or are currently dealing with cancer, disease, challenges, etc…..see hope and inspiration in Rhema. The little girl who should have been scared or harmed by seeing her mother suffer and gone, is strong and perfect. I see Rhema as a cancer survivor. She gives me hope for goodness in mankind. God gave her a beautiful heart and the voice of an angel. Most people that hear her sing can not deny that God does speak through a child. Her voice touches people’s hearts.

Whenever we remember Libby, or any woman who lost her battle to ovarian cancer, we should follow Rhema’s example and heed the call to action set forth in the last line of the poem Remember Me, ” … smile, open your eyes, love and go on.”

Libby, we will always love you and keep your memory alive in our hearts and minds.

How Can You Help?

To support Libby’s H*O*P*E*™, you can make a donation ($10 minimum) through our Facebook Cause page.  All donations made to the Libby’s H*O*P*E*™ Facebook cause are designated for the benefit of the Ovarian Cancer Research Fund (OCRF). OCRF is one of the largest U.S. private, non-profit organizations dedicated to finding an early detection test, and ultimately a cure, for ovarian cancer.

If you are not a Facebook member, you can still make a donation through the Libby’s H*O*P*E*™ Facebook donation page (no membership or registration required).

If you are unable to donate, you can nevertheless support OCRF without any out-of-pocket cost by clicking on our “SocialVibe” widget that appears on the website homepage right sidebar, or by using our designated SocialVibe website. For each reader that clicks on the SocialVibe widget (or goes to our designated SocialVibe website), and watches the video presented and/or answers the question(s) listed, our current SocialVibe sponsor will donate money to OCRF on your behalf for ovarian cancer research. It’s fast & it’s free!

Special Thanks:

We would like to extend special thanks to Teton Voraritskul for allowing us to feature Rhema’s story and music videos, as well as the video of Wendi’s life.  To learn more about Rhema Marvanne and her music, go to www.RhemaMarvanne.com. Rhema’s songs are sold on iTunes®, Amazon.com, and RhemaMarvanne.com.

Sources:

• Jemal A., Siegel R., Xu J. et. al. Cancer Statistics, 2010.  CA Cancer J Clin. 2010 Jul 7. [Epub ahead of print] [PMID: 20610543].

• Long-term survival from once-deadly cancers doubles, Press Release, Cancer Research U.K., July 12, 2010.

• Remember Me, written by David Harkins, Silloth, Cumbria, U.K., PoeticExpressions.co.uk.

• I wrote the Queen Mum’s funeral poem, written by Chris Musson, News & Star.

• David Harkins website (http://www.davidharkins.co.uk/).

• Rhema Marvanne website (http://www.RhemaMarvanne.com).

_________________________________________

*“Vox Populi,” a Latin phrase that means “voice of the people,” is a term often used in broadcast journalism to describe an interview of the “man on the street.”

In the spirit of Vox Populi, Libby’s H*O*P*E*™ searches online for original writings and visual media created by ovarian cancer survivors, survivors’ family members, cancer advocates, journalists, and health care professionals, which address one or more aspects of ovarian cancer within the context of daily life. The written and visual media features that we discover run the gamut; sometimes poignant, sometimes educational, sometimes touching, sometimes comedic, but always honest. The Vox Populi feature may take the form of an essay, editorial, poem, letter, story, song or video picture montage.

It is our hope that the Vox Populi feature will allow our readers to obtain, in some small way, a better understanding of how ovarian cancer impacts the life of a woman diagnosed with the disease and her family. We invite all readers to submit, or bring to our attention, original writings and visual media suitable for publication as Vox Populi features.

Do You Twitter? Tell Libby’s H*O*P*E* What You’re Doing!

Libby’s H*O*P*E*™ recently added a feed from its Twitter account (http://www.twitter.com/libbyshope) to the homepage left sidebar.  I know what you are thinking.  What in the heck is Twitter?

Twitter is a social networking  service that allows its users to send and read other users’ updates (known as “tweets”), which are text-based messages that cannot exceed 140 characters. Updates are displayed on the user’s profile page and delivered to other users who have signed up to receive them. Senders can restrict delivery to those in their circle of friends (delivery to everyone being the default). In addition, users can receive message updates via the Twitter website, mobile phone, Blackberry, etc.  It’s easy!  Set up a Twitter account and choose to follow “libbyshope.”  At this point, you will receive all Libby’s H*O*P*E*™ weblog updates no matter where you are located. You can perform a general search on the Twitter website for any topic (e.g., “cancer”) and you will find organizations and individuals who share your interest in that topic.

Twitter also allows you to send messages of 140 characters or less to Libby’s H*O*P*E*™.  In those messages, you can add web links to relevant news or medical articles and even include pictures. If you attend a cancer conference,  local hometown educational seminar or a fundraising event, you can send us brief comments (and pictures) directly from those events in real time.  Or, if you just feel like telling us how you are doing, that’s ok too!  Twitter allows cancer survivors to stay in touch with one through the communication of  valuable information and advice.

Currently, we are streaming Libby’s H*O*P*E*™ outbound twitter messages to our weblog (left homepage sidebar), but if the interest is high enough, we will stream all messages received and sent by Libby’s H*O*P*E*™ on the weblog.  By way of example, Libby’s H*O*P*E*™ is currently following the American Cancer Society, Association of Cancer Online Resources (ACOR), Cure Magazine, Lance Armstrong and several other cancer survivors and advocates.

The addition of Twitter to the Libby’s H*O*P*E*™ weblog is simply our way of allowing you to easily follow us regardless of your location.  More importantly, we hope to hear from many of you through this technology in the near future.  We provide below a video that describes “Twitter in plain English” and several additional links that will allow you to better understand Twitter.

Twitter in Plain English

Additional Resources:

• Help Resources/Getting Started, Twitter.com.

• Applications, Twitter.com. (we use twhirl as a desktop widget).

• Tiny URL.com (application allows you to shorten any web address for inclusion in a Twitter message, thereby allowing you to minimize usage of the 140 character twitter message limit.

• TwitPic.com (allows you to share photos on Twitter).

• What is Twitter?, Tweeternet.com