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Clinical Trials for Hereditary Cancer: Where the Rubber Meets the Road

December 12, 2012

Clinical Trials for Hereditary Cancer: Where the Rubber Meets the Road

This blog is a call to action! Please read on, and then post, blog, tweet, retweet, and share about this issue so that we can assure that hereditary cancer research continues!

The call for more research is a constant theme for all diseases including cancer, and sometimes it’s easy to get frustrated by the slow pace of progress. The multistep process from discovery to FDA approval is often long and doesn’t always end in success. But research is necessary to assure that new treatments work as well or better than current standard-of-care. For this to happen, studies must recruit enough people to prove that the agents work. This is particularly critical for research that focuses on a small specific population like people with a BRCA mutation.

PARP inhibitor research is a prime example. I first heard about PARP inhibitors at the 2005 ASCO annual meeting. In her plenary address on advances in hereditary cancer, Dr. Barbara Weber from the University of Pennsylvania mentioned targeted agents (PARP inhibitors) that were designed to exploit weaknesses of cancer cells in people with BRCA mutations. This was exciting news! I was hopeful that this could be the beginning of personalized therapy for people in our community. From that moment on, I vowed to do whatever it took to learn about, share with our community, and promote the studies to determine whether these drugs worked.

Early small clinical trials of PARP inhibitors were promising, but delays and road-blocks affected development of larger research studies. Some of the roadblocks had to do with study design; others involved dosing or side effects as researchers determined the most effective combinations of PARP inhibitors with other anticancer agents. Despite these issues, enthusiasm continues for the potential of these drugs in people with BRCA mutations. Yet, eight years later, there are still no FDA-approved PARP inhibitors and people are still dying of hereditary cancers!

FORCE has continued to advocate for further research on PARP inhibitors, petitioning scientists, the FDA, and pharmaceutical companies to address the road-blocks and challenges and to facilitate the research and find answers for hereditary cancer. After eight long years, our pleas and efforts have been rewarded. Several PARP inhibitor studies are now recruiting, including a large, Phase II study on PARP inhibitors for women with BRCA-associated advanced breast cancer. Our participation in this research is critical. Unless enough people participate, these studies will not continue. If enrollment falls short, the next time scientists have an idea for treating or preventing hereditary cancer, they may decide that the BRCA community is too difficult to research, and fewer studies will be designed for us. That would be tragic considering how many members of our community develop and succumb to cancer.

This is where the rubber meets the road!

We have worked long and tirelessly to advocate for this research. Now that we have it, we cannot afford to turn a deaf ear. At this moment, the fate of hereditary cancer treatment research rests with each of us. Although most of the current studies are open only to women with advanced cancer, even if that doesn’t describe you, perhaps you know someone who fits that description. If PARP inhibitors work for advanced hereditary cancer, the next step will be tests to see if they also work for earlier cancers.

Here is what you can do to help:

  • Get involved. Consider enrolling in a study if you are eligible, and share information about PARP inhibitor research with everyone that you know. Post it prominently on your social media pages, share it with your online or in-person support group, discuss it with your local media, and write or blog about why hereditary cancer research is important. Please remember to share your efforts with us. Email us,  post on FB or the FORCE message boards about ways you have spread the word about this important research.
  • Stay tuned to FORCE to learn of new available studies. We will be updating this page in the upcoming weeks with new featured studies so check back often.
  • Support FORCE with a donation to help us continue our important work to advocate and recruit for research specific to hereditary cancer

We must participate in and promote hereditary cancer clinical trials and other studies if we and future generations are to realize more effective treatment and prevention for hereditary cancers.

Posted in: Research
Tags: BRCA , Breast Cancer , Hereditary Cancer Research , BRCA1 , BRCA2 , FDA , Clinical Trials , PARP Inhibitor

32 Comments

December 12, 2012

KG says:
Has this study been promoted by Army of Women?

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December 13, 2012

facingourrisk says:
I am uncertain who is promoting the study. This is research of great interest to the HBOC community which is why we are highlighting it and asking our community to share this on all the places where they post.

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December 13, 2012

hopeforheather says:
Do you know if other women who are high-risk (but not BRCA +) are eligible?

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December 13, 2012

facingourrisk says:
The phase II study mentioned is for women with mutations, but some of the studies on our featured research page are open to other populations. Each clinical trial has it's own eligibility criteria.

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December 21, 2012

Denise latarski says:
How do I find out more?

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December 21, 2012

facingourrisk says:
Hi Denise, Visit our Featured Studies page at: https://www.facingourrisk.org/information_research/featured_studies.php If you have further questions about a particular study you can contact the study coordinator if one is listed. Feel free to contact us at: info@facingourrisk.org.

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December 15, 2012

Wayne Mccafferty says:
what about men with a mutated BRCA2 gene ?

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December 16, 2012

facingourrisk says:
Hi Wayne, If you check out the Featured Research Page there is a link to PARP inhibitor studies enrolling for pancreatic, prostate, and melanoma. I hope this is helpful!

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December 17, 2012

Hereditary Cancer Research « Lee Asbell says:
[...] https://www.facingourrisk.org/2012/12/12/clinical-trials-for-hereditary-cancer-where-the-rubber... [...]

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December 19, 2012

Margaret Snow says:
There are so many reasons to support these trials. Certainly we all know whether our friend relatives, neighbor's cancer is HER+, estrogen sensitive etc. We are on the cusp of needing to know whether every young woman is BRCA positive or not. If this research works, then BRCA status of patient and tumor will be a necessary part of the workup.

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December 24, 2012

Sharon Jack says:
Speaking as someone who is benefitting from parp inhibitor research I heartily endorse this post. I have been on the BMN673 parp inhibitor trial since March (nine months now) and it has reduced my cancer by over 90%. My CA125 went from 204 down to 10. I am BRCA1 with ovarian cancer - first diagnosed 2C in April 2009, recurrence in July 2010 and again in Dec 2011. I chose to go on this trial rather than have more chemotherapy. I have loved being on this new drug - I have had no nasty side effects - just slight queasiness for the first month. My bloods have been excellent and my energy levels good. Being on a trial does involve many hospital visits for monitoring but for me it is so worth it. Whatever happens in the future, I have had a year without having to have chemotherapy and where I have felt really well, been able to work all through, exercise, swim and go away on holidays! I must point out however this trial has not been successful for all the patients that have tried it but that it why we need more research to find out exactly why and which patients do benefit. Happy to answer any questions anyone might have about parp inhibitors or being on a trial.

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January 6, 2013

April Wang says:
Hi, Sharon: Thank you so much for sharing your experience with parp inhibitor. My sister was first diagnosed with 3C in Feb. 2009. She is BRCA1 with ovarian cancer. She had two recurrences and two more chemotherapies after the first chemotherapy in 2009. Her heart and blood vine are damaged by Chemo. It's hard for her to have more chemotherapy. After reading your post, I see hope and think she might be a good candidate for BMN673 parp inhibitor trial. However, she lives in China. I am not sure if there is any possibility for her to get involved with the trial. Do you know where I can find more information about this trial? My email address: jejo_wang@yahoo.com. I am looking forward to hearing back from you! Thanks, April Wang

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January 7, 2013

Lynne says:
go to clinicaltrials.gov and type into the search box : ovarian cancer AND china

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January 9, 2013

April says:
Thank you very much, Lynne.

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January 8, 2013

Sharon Jack says:
Hi April So sorry to hear about your sister. You can find more information about the BMN673 trial at: http://clinicaltrials.gov/show/NCT01286987 however it is currently only available in the UK and the US. As Lynne said you can look on the website for studies that are available in China and see if there is anything suitable for your sister. I must also add that since writing my previous post the BMN673 drug has stopped working for me and I have come off the trial. However it gave me a fantastic year without chemotherapy and where I have been able to work, exercise, holiday and socialise so I am grateful to have been able to take part in the trial. wishing you all the best Sharon

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December 26, 2012

Clinical trials & research | Saying NOPE to Breast Cancer says:
[...] FORCE’s blog post about clinical trials for hereditary cancer: Clinical Trials for Hereditary Cancer: Where the Rubber Meets the Road. If you are eligible, consider enrolling in a study. Find a list of current studies on [...]

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January 4, 2013

Amy says:
I have been enrolled in a study since October 2012 in Chicago taking the part inhibitor any-888. I was diagnosed with breast cancer in 2001 and 2009. I found out in September it has metastasized to my chest wall and a lymph node under my collar bone. I had my first scans after 9 weeks on meds and the mass (7cmx4cm) is stable. Didn't shrink but didn't grow which is good. Biggest size effect of my medication abt-888 is nausea/vomiting but have been controlling it with scope patch.

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January 4, 2013

Amy says:
Oops should have mentioned I do have the brca 1 mutation and tumor is triple negative. I started the abt-888 on my 37th birthday.

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January 4, 2013

facingourrisk says:
Thanks for sharing your experience. Please take care. Much love, Sue

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January 4, 2013

Mary G says:
Sue, The eligibility requirements for this study will exclude virtually all women who have already had some treatment for metastatic breast cancer. I have been living with metastatic BC for 19 months and carry the BRCA 2 gene. I have already had a taxane and a platinum-based drug, so I am ineligible to enroll in the trial. The only women who will be eligible will be the newly diagnosed or those women "lucky" enough to have been successfully treated with hormonals, the same group that already lives the longest with MBC. I am very discouraged but still hope to get a PARP drug through a Phase III trial or by petitioning the FDA for a compassionate use allowance. You seem to be blaming the victims when you say funding might evaporate if we don't step up. You are asking a very sick group of terminal cancer patients to pick up a ball and run with it. The truth is, clinical trials involve extended stays far from home for those of us not living near major research centers. They add the stress of regular travel to our lives and rob us of precious time at home with our families. I almost died in early August waiting for a PARP trial to open up at MD Anderson, probably this very trial. Carboplatin has temporarily saved my life but has excluded me from participating in the trial. The decision to start carboplatin, knowing it would exclude me from the trial, was heart wrenching for me and my doctor, but I was close to heart and liver failure on the drug we were using to buy time until the trial opened, and we figured being dead would exclude me as well. I love the work you do with FORCE and am so grateful my daughter and I have you as a resource, but the tone of this plea for participants has really turned me off. I look forward to your response. Mary Gugich

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January 5, 2013

facingourrisk says:
Hi Mary, Thank you for your post and for highlighting many of the challenges that all clinical trials present. It is particularly hard to hear about clinical trials specifically designed for our community and hear that the eligibility or lack of access to an enrolling facility is keeping motivated people from being able to participate. I'm sorry that you inferred any blaming of the victim into this post. My words are a call to action for our community. Many times people who are not in treatment don't learn about clinical trials but so many in our community know someone who might qualify. Raising awareness is our best defence in assuring that clinical trials for hereditary cancer continue. In 2012, the large GOG study 8199 which was a continuation of GOG-199, looking for early detection and markers for ovarian cancer closed due to lack of participation. I only found out about the slow recruitment too late, and despite our best efforts, the study closed. This is not the fault of our community at all but the lack of awareness and participation impacts us all. That is why as a small community that is a subset of a larger breaast and ovarian cancer community, people dealing with HBOC need to unite across our differences and promote and advocate for more research and resources and awareness not just of HBOC but of any clinical trials specifically for HBOC. I'm hoping that every person who reads this blog will help us spread the word so that we can be certain that the PARP inhibitor studies accrue and we can finally learn if they work for HBOC cancers. We have been told that this particular breast cancer study has not been recruiting the numbers needed for us to get answers. Thus the call to action. Unfortunately, when I looked at the study on http://www.clinicaltrials.gov, the listing wasn't up-to-date and many of the centers that are already recruiting were listed as not being active yet. This is a big problem. We contacted as many as the centers as we could and included the contact information for the open studies on the study page: https://www.facingourrisk.org/information_research/NCT01506609.php . It's certainly a problem if people who are motivated and interested in a study go to a trusted source and the listing says that the study isn't active yet. This is an information issue that impacts our ability to get these studies completed. You raise several other excellent points about barriers to clinical trials, including the fact that many people need to travel at great expense to participate. Some studies, (such as at NIH) do help with travel costs but there are still issues with lost wages from traveling, child care, many barriers that make it even harder to complete the studies. And then there are the eligibility requirements that leave many very motivated people ineligible for a study. It is a frustrating and upsetting barrier, and I am so sorry to hear that you do not qualify for the study. I have heard that the veliparib study may allow "expanded access" for those who do not qualify to participate in the research to receive compassionate use access to the drug. I hope that you are successful in your efforts to get access to veliparib, please keep me posted! If this drug shows efficacy and we can get FDA approval, that will greatly expand our entire community's access to these promising drugs! So let's keep spreading the word and telling everyone we can about the studies that are open. Over the course of the next few weeks we will be adding more content with information on open PARP inhibitor studies. It is encouraging to me that more studies are continuing to open up, and if we can get the studies filled through word-of-mouth promotion, then we will finally know if PARP inhibitors improve survival for hereditary cancers. Much love, Sue

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January 4, 2013

says:
I'm on the Olaparib trial at National Institutes of Health/National Cancer Institute. The oral PARPi was paired with carboplatin for the first 8 cycles, Jan-July. My lung mets have shrunk about 80%!

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January 4, 2013

Lynne says:
brca 1 positive with 15 weeks of carbo/taxal and olaparib. 4 small tumors in liver and 6 in lymph nodes when I started. All tumors were less than 1" in diameter when I began treatment. At this point I have one tumor left (2.1cm X .7cm). But this is my 7th treatment of chemotherapy in 5 years.

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January 5, 2013

Mary G says:
Lynne- that's great! I wish you continued success. Where are you receiving your treatment? Had you been treated with a taxane or platinum chemo before? I would love to find a trial that would accept me!

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January 5, 2013

Susan says:
I'm BRCA2+ with Stage IIIC ovarian cancer. Have been on a trial using Veliparib (ABT-888) in combination with standard chemo for the first 6 cycles. I completed chemo in July 2012 and have been in remission since then taking Veliparib only. I'm hopeful that Veliparib continues to keep me in remission. I have not experienced any side effects from Veliparib. Once chemo ended in July, all side effects went away I must admit that I enrolled in the trial for selfish reasons. Because of my BRCA+ status, both of my oncologists felt a PARP inhibitor was the best option for me and the trial was the only way to get this drug. So it seems that it is a win-win situation whereby it's helping me and I'm also helping the cause. While participating in the trial has been very time-consuming and cumbersome, I'm very grateful to be able to get this drug.

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January 9, 2013

Julie says:
I am participating in the G9927 trial (studying parp inhibitor ABT888 with Carboplatin and Doxorubicin) at Hopkins . I am BRCA1+ with IIIC ovarian cancer. In 2010 did seven cycles of Carbo, Taxol, and Avastin - was on Avastin for another year, and had a recurrence in June 2012. Have had four cycles with this study on parp inhibitors, with two more to go. My CA125 is down to 7 and my last scan looked clear. I was under the impression that once I finish my 6th cycle I would no longer have access to the ABT-888, after reading your message, I'm hopeful that maybe I can still have access to the parp inhibitor. Like you, I haven't had any side effects from the ABT888 alone. So nice compared to chemo.

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February 2, 2013

Susan says:
Julie, I've been told that I can remain on Veliparib indefinitely unless there is evidence of disease progression. Have you checked about whether this also applies to your trial as we know that different trials have different rules.

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February 4, 2013

says:
I did check, and unfortunately with the trial that I'm in, I won't be able to get the parp inhibitor once my last treatment is over. I have to admit I'm nervous about another recurrence. Hopkins has a study now for a vaccination for ovarian cancer. You need to have had two recurrences to participate, but it would be amazing if they could figure out a way to stop it before it starts! Thanks for your response, and good luck.

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February 4, 2013

Lynne says:
I tried to get into a clinical trial using a PARP inhibitor since the fall of 2009 . Unfortunately. Reseachers don't want a person that has been treated more than two times, at the most three with chemotherapy agents. Fortunately for me, Dr. Rivkin, of the Swedish Medical Center Cancer institute, designed a trial that did not put a limit on the number

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February 4, 2013

Lynne says:
Continue : of previous treatments. Dr. Rivkin based his enrollment on over all health. After 5 years and 6 different regimens , almost all with carboplatin and taxol, I am responding very well to Olaparib, Carboplatin , and taxol. If you have been heavily treated with chemotherapy, check out Dr. Rivkin's trial at clinicaltrials.gov . Type in Olaparib in the search box. His trial is currently listed as #10 and untitled: "Phase 1b study of Olaparib plus weekly Carboplatin and Paclitaxel on Relapsed Ovarian Cancer."

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February 4, 2013

facingourrisk says:
Here is a link to the study: http://clinicaltrials.gov/ct2/show/NCT01650376?term=NCT01650376

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June 10, 2020

New Year’s Then (20 Years Ago) and Now - Facing Our Risk of Cancer Empowered - Facing Our Risk of Cancer Empowered says:
[…] promising agents known as PARP inhibitors. This marked the beginning of our continuous efforts to overcome barriers and help recruit patients from our community into relevant clinical trials enrolling patients with […]

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