BY LINDSAY RAY | SEPTEMBER 30, 2013
Ahead of schedule, the Food and Drug Administration (FDA) approved Perjeta (pertuzumab) to treat patients with early-stage breast cancer prior to surgery (neoadjuvant) on Sept. 30. This comes on the heels of a unanimous vote from the Oncology Drugs Advisory Committee to recommend the drug for approval. Perjeta was approved just last year to treat patients with metastatic HER-2 positive breast cancer.
Perjeta is the first drug approved for the neodjuvant treatment of cancer and provides another treatment option for patients at high risk of a breast cancer recurrence, metastasis or of dying from the disease. Perjeta is still intended for HER-2 positive patients, but has now been extended to include those with inflammatory, locally advanced or early-stage breast cancer who are at high risk. Perjeta is to be used in combination with Herceptin (trastuzumab) and chemotherapy, and depending on the combination used, chemotherapy can be used after surgery as well. Herceptin, however, should be continued after surgery to complete a year of treatment.
Perjeta was approved under the FDA's accelerated approval program, which provides patients access to drugs for serious conditions while further ongoing trials are conducted. The drug was approved based on a phase 2 trial in which 417 participants were randomly assigned to receive one of four treatment combinations prior to surgery (given every three weeks for four cycles of treatment): Herceptin and docetaxel; Perjeta, Herceptin and docetaxel; Perjeta and Herceptin; or Perjeta and docetaxel. Thirty-nine percent of those receiving the Perjeta, Herceptin and docetaxel combination achieved a complete pathologic response, which means there was no detectable invasive cancer in the breast or lymph nodes. Only about 21 percent of those who received Herceptin plus docetaxel achieved a complete pathologic response. Perjeta is also the first drug approved based on a pathologic complete response instead of traditional endpoints, such as survival data and progression-free survival.
A large, phase 3 trial, called APHINITY, is ongoing to confirm these results and also examine the long-term effects of Perjeta. Approximately 4,800 participants with HER-2 positive breast cancer who have received prior breast cancer surgery and are at high risk for cancer recurrence are enrolled. Results from this trial are expected in 2016. Perjeta is still being further explored for treating breast cancer as well as other HER-2 positive cancers, colorectal and gastric cancers.
When asked for a comment, Lisa Carey, medical director of the UNC Breast Center in Chapel Hill, N.C., said that it is important that the FDA is recognizing the need for more flexible and nimble approaches. "This is a good one since the neoadjuvant trial was well designed and completed," she says. "The adjuvant trials, which will define this in terms of more clinically meaningful results, are finished collecting patients and just need to mature. So this is a good proof-of-principle for using the neoadjuvant approach as an intermediate."
However, some questions still need to be answered. Carey explains: "The questions center on the use of such a short duration of Perjeta since the augmentation of pathologic complete response with Herceptin was associated with improved outcome but with a year of therapy, not weeks. Also how can the heterogeneity of HER2+ disease impact response to single or dual HER2-targeting, and how can we get smarter since this is now an extremely expensive approach that needs a better selection strategy."
As Carey noted,the Perjeta-Herceptin combination will also carry a hefty price tag--it is expected to cost between $27,000-$49,000 for 9-18 weeks of therapy. However, there are patient assistance programs in place that might be able to help off-set the price.
Common side effects for participants receiving the Perjeta-Herceptin-docetaxel combination included hair loss, a decrease in white blood cells (neutropenia) and nausea. Other side effects included allergic reactions, hypersensitivity and decreased cardiac function.
For more information, visit Perjeta.com or call 855-737-5382.
For more information on HER-2 positive breast cancer visit: "Research Unravels New Ways to Treat HER2-Positive Breast Cancer".
Editor's Note: This blog was updated on Oct. 1.RELATED POSTS
BY LINDSAY RAY | MAY 8, 2013
I sat in on a session about genetics and biomarkers at the annual Oncology Nursing Society Congress in April. After the session was over, the nurse next to me turned and said, "This is the future." And that really was my feeling as well. What can sound like a lot of scientific jargon is actually really important to understanding how not only cancer works but also how our bodies function.
We have come a long way in our understanding of the inner workings of cells. April 14 was the tenth anniversary of completing the map of the human genome. With that basic road map, we've been able to ramp up research efforts into genetics and genomics. In 2009, there were about 285 genome-wide association studies; recently, there were more than 7,000. But what exactly are genetics and genomics? And what do they mean for cancer patients?
DNA contains all of our genes. The genome then is the entirety of that genetic information. Let's think of DNA as a book. The genome would be like the table of contents telling you what's in the book. And each gene would be like a chapter, complete with its own more detailed information. Genetics is the study of a single gene. It would be like intensely studying a chapter in the book.
Genomics is the study of several genes. So you'd look at multiple chapters in the book and see how they can improve your knowledge overall and relative to each other. There are ways that researchers can drill down even further and look at much more detailed genetic information as well. But while we have this book and table of contents, this wonderful resource of information, we still can't read all of it. Better understanding this information is what drives research.
In cancer research, scientists are mostly concerned with genetic mutations. The gene's purpose is often to produce necessary proteins. When a mutation occurs, and there are many kinds of mutations, the cell can no longer produce these proteins correctly. Genetic mutations can either be hereditary and passed on from family member to family member, or they can develop after we're born and as we grow older, changes called somatic mutations.
This is where biomarkers come in. A biomarker is any sort of biological molecule that can provide us with certain information about diseases and treatments. It can be found in the blood or tissue, for example, and it can even be a change in a gene. Some of the main uses for biomarkers in cancer help us try to find cancer early (screening), determine how treatable or "bad" a cancer is (prognosis), figure out if a treatment will work for a particular patient (predicting) or see if a treatment is working (monitoring).
For example, two of the most well-known genetic biomarkers are in breast cancer--the BRCA1 and BRCA2 gene mutations. These genes give us prognosis information--women with these mutations are at an increased risk of ovarian and breast cancers.
An example of a biomarker that tells us slightly different information is the epidermal growth factor receptor (EGFR) in lung cancer. Some mutations to this receptor might mean that the tumor will respond well to certain tyrosine kinase inhibitors, whereas another mutation in the same receptor means that the tumor might actually resist these types of drugs.
These are only a few examples of the types of biomarkers for cancer, and this information only scratches the surface of what we're learning from genetics, genomics and biomarkers. As research moves forward, we get a little bit closer to reading more and more of our genetic material and understanding its implications in terms of better outcomes for cancer patients.RELATED POSTS
BY LINDSAY RAY | APRIL 26, 2013
As I spend more time with oncology nurses, I continue to be in awe of them. I walked over to the conference this morning with a wonderful nurse who was just completely excited about learning more information and being able to take this information back to Indiana to better help her patients. That eager, helpful spirit permeates the vibrant atmosphere at the Oncology Nursing Society Conference. Nurses view themselves as partners with their patients during the cancer experience. And that is evident in the compassion and empathy they demonstrate for their patients.
For example, I attended an informal discussion about the challenges young adult cancer patients face. Nurses had gathered to talk about what exactly defines a young adult, the isolation young adults can feel by being treated in patient unit usually filled with older adults. And the topics of sexuality and infertility became a main focus. Nurses worked on identifying reasons why they might not bring up the topic of sexuality with their young adult patients--nurses might not know how to answer the questions, there could be an age barrier, they're unsure when's the right time to bring up the topic, etc. Figuring out the barriers to having this particular conversation is just one step closer to making this topic more open to patients.
But a recurring theme I have heard from nurses is that it's not only their responsibility to teach their patients, but they also have the opportunity to listen and learn from their patients. Working together patients and nurses can make treatment and quality of life during cancer better.RELATED POSTS
BY LINDSAY RAY | APRIL 25, 2013
Although I've worked with CURE for a few years, this was my first opportunity to actually attend our Extraordinary Healer award event. I've always heard that this event is special, but there's a difference between knowing an event is special and actually being there to experience that special moment. No words can quite capture the feeling that was in the room. And I'm rarely at a loss for what to say--words are kind of my job. But I can try to capture some small amount of what happened this evening.
It was moving to listen to honorary mistress of ceremonies Marlee Maitlin describe the many obstacles she's had to overcome to achieve her dreams of being an actress and the people who helped to shape who she is along the way. As she so thoughtfully pointed out, oncology nurses often serve as the people who help shape cancer patients lives--they help to heal, but also help patients find their own gifts and overcome challenges both personal and medical.
And while I had read the nominating essays, it was different to actually hear these words from the patients themselves. To stare into the nurses' appreciative faces. But mostly what made it special was that it was a shared experience. There were people to share in the laughter and the tears, people who know all about the circumstances being described.
So thank you to all the oncology nurses who were able to share this experience with us. And thanks to all the oncology nurses and patients and families who have shared so much of themselves and their experiences with us over the years.RELATED POSTS
BY LINDSAY RAY | FEBRUARY 22, 2013
Today, Kadcyla (ado-trastuzumab emtansine, or T-DM1) received a long-awaited approval from the Food and Drug Administration (FDA) to treat patients with metastatic, HER2-positive breast cancer who have previously been treated with Herceptin (trastuzumab) or taxanes, a class of drugs that includes paclitaxel and docetaxel.
Kadcyla belongs to a new class of drugs called antibody-drug conjugates and works by fusing the powerful chemotherapy drug emtansine to the antibody Herceptin, which targets HER2 receptors on cancer cells.
The approval was based on results from the phase 3 EMILIA trial, which enrolled 991 patients who had previously been treated with Herceptin and a taxane-based chemotherapy. Patients were randomly assigned to either receive Kadcyla, or the current standard--a combination of Xeloda (capecitabine) and lapatinib. Those in the Kadcyla arm had a median overall survival of 30.9 months compared with 25.1 months for those in the combination arm. Patients receiving Kadcyla also had improved median progression-free survival: 9.6 months compared with 6.4. Furthermore, patients on Kadcyla experienced fewer severe side effects (grade 3 or higher) than those on Xeloda and lapatinib (43.1 percent compared with 59.2 percent).
Common side effects included low platelet count, nausea, fatigue and joint or muscle pain. This drug also carries a boxed warning (the highest warning the FDA can give) advising doctors and patients that in some instances, this drug can cause liver toxicity, heart toxicity and death. It can also lead to severe birth defects.
The drug is expected to be available within two weeks, and the estimated cost will be $9,800 a month, or $94,000 for a course of treatment. Genentech, the drug's manufacturer, is planning to begin patient assistance programs to help patients that might not be able to afford Kadcyla.RELATED POSTS
BY LINDSAY RAY | FEBRUARY 11, 2013
The Food and Drug Administration approved Pomalyst (pomalidomide) on Feb. 8 to treat patients with multiple myeloma who have progressed during or within 60 days after receiving at least two therapies, such as Revlimid (lenalidomide) or Velcade (bortezomib).
Pomalyst is a derivative of Thalomid (thalidomide), another drug used to treat multiple myeloma and in the same family of drugs as Revlimid. Called immunomodulating agents, these drugs incite the immune system to attack the myeloma cells.
The approval is based on a phase 2 clinical trial in which patients received either Pomalyst and low-dose dexemethasone, a type of steroid, or Pomalyst alone. In those on Pomalyst alone, 7.4 percent had their cancer completely or partially disappear compared with 29.2 percent for those receiving the combination. This response lasted for more than 7 months in the combination arm.
Pomalyst also performed well in a phase 3 clinical trial in which patients received either Pomalyst and low-dose dexemethasone, a type of steroid, or high-dose dexemethasone alone. Those in the Pomalyst combination arm lived 7.7 weeks longer without their disease progressing than those on the steroid alone. The median for overall survival had not yet been reached for those receiving Pomalyst when the data were presented at the American Society of Hematology annual meeting in December.
Common side effects included low white and red blood cell counts, fever and infections. The drug also includes a boxed warning (the highest warning the FDA can give) that pregnant women should not take the drug as it causes serious side effects, such as blood clots and birth defects. Therefore, prescribing physicians must be certified with the Pomalyst Risk Evaluation and Management Strategy (REMS) program.
For more information, visit pomalyst.com or call 800-931-8691.RELATED POSTS
BY LINDSAY RAY | JANUARY 31, 2013
On Jan. 23, the FDA approved another version of second-line therapy for treating patients with metastatic colorectal cancer. Avastin was first approved for use as a first-line therapy in colorectal cancer in 2004, and Avastin with 5-FU based-chemotherapy was approved as a second-line option in 2006. This most recent indication allows Avastin (bevacizumab) to be used as a second-line therapy in conjunction with fluoropyrimidine-combination chemotherapy and either irinotecan or oxaliplatin. Fluoropyrimidines include Xeloda (capecitabine) and floxuridine, as well as 5-FU (fluorouracil). Avastin cannot be used as adjuvant therapy (additional treatment after the primary therapy).
Approval is based on a phase 3 trial with 820 patients with metastatic colorectal cancer who had progressed during or following treatment with Avastin-based chemotherapy. Patients were randomly assigned to "cross-over" chemotherapy plus Avastin or cross-over chemo alone. (Cross-over chemotherapy means that patients who had previously been treated with oxaliplatin-based chemo received irinotecan-based chemo in the study, and vice versa.)
Patients in the Avastin arm had a median overall survival of 11.2 months compared with 9.8 months for those in the chemotherapy-only arm. Progression-free survival was also improved by a median of 1.7 months for those in the Avastin arm.
The side effects in this study were similar to those seen in previous studies, which include fatigue, diarrhea and mouth sores.
Avastin can cost $88,000 per year and is also approved for treating advanced renal cell carcinoma, glioblastoma and non-squamous, non-small cell lung cancer.
For more information, visit avastin.com or call 877-428-2784.RELATED POSTS
BY LINDSAY RAY | NOVEMBER 1, 2012
As many of us are coming down off the Halloween candy sugar rush, others of us are gearing up for another event--Movember. Men are encouraged to "change the face of men's health" by quite literally changing their faces and letting their facial hair grow for an entire month.
The goal is to raise awareness and funds for all men's health issues, especially prostate and testicular cancers. Prostate cancer is the most common cancer for American men after skin cancer and will affect 1 man in 6 during his lifetime. Testicular cancer is rarer but often found in younger men.
Actor Nick Offerman, perhaps best known for his sweet 'stache and playing the beloved Ron Swanson on Parks and Recreation offers his tips on growing a mustache to be proud of.
Or if you can't grow a mustache, like me, but want to show your support, you can always add a mustache to a photo or take a photo with a mustache.
To learn more about Movember, visit movember.com or if you would like more information on prostate cancer, check out CURE's recent A Patient's Guide to Prostate Cancer, Second Edition.RELATED POSTS
BY LINDSAY RAY | OCTOBER 29, 2012
The Food and Drug Administration has approved the second drug in two months for treating chronic myeloid leukemia (CML). On Oct. 26, Synribo (omacetaxine mepesuccinate) was approved to treat adult patients whose CML has progressed after treatment with at least two tyrosine kinase inhibitors (TKIs), such as Gleevec (imatinib), Sprycel (dasatinib) and Tasigna (nilotinib). The previous drug approval came in September for the tyrosine kinase inhibitor Bosulif (bosutinib).
When disease progresses after treatment with multiple TKIs, it can be more difficult to treat and options become more limited. There are some mutations that don't respond well to TKIs. Synribo works by blocking certain proteins that promote cancer cell growth and provides another option after TKI treatment.
Synribo's effectiveness was established in trials with a cohort of patients whose disease had progressed despite treatment with TKIs, and all patients received Synribo. In the group of 76 chronic phase CML patients, effectiveness was determined by a reduction in the number of cells that have the Philadelphia chromosome genetic mutation, which most CML cells have. After an average of 3.5 months of treatment, 14 of the 76 patients had a response that lasted for a median of 12.5 months. For those patients in the accelerated phase, effectiveness was measured through a normalization of white blood counts or absence of leukemia. Five out of the 35 patients had a response in an average of 2.3 months, and the response lasted for a median of 4.7 months.
Synribo is injected under the skin twice a day for 14 days over the course of a 28-day cycle. Once the white blood cell count normalizes, Synribo is then given twice a day for seven consecutive days for a 28-day cycle, and the patient continues with Synribo as long as he or she has a clinical benefit. Common side effects include low platelet, red and white blood cell counts; diarrhea; nausea and fatigue.
For more information, please visit synribo.com or call 800-896-5855.RELATED POSTS
BY LINDSAY RAY | SEPTEMBER 27, 2012
After the approval of Zaltrap (ziv-aflibercept) for colorectal cancer in August, there's more good news for metastatic colorectal cancer patients. This morning, the Food and Drug Administration announced that it had approved Stivarga (regorafenib) for patients with metastatic colorectal cancer (mCRC) who have progressed after trying other therapies. The FDA had granted the drug a priority review in late June, which guarantees a drug will be reviewed within six months.
Stivarga works as a multi-kinase inhibitor, which means it targets several different pathways that can promote tumor growth, including the vascular endothelial growth factor receptor (VEGF), which signals tumor blood vessel growth, and c-KIT, an oncogene that also helps cancer growth. Patients take the drug orally, once-a-day in four week cycles (three weeks on, one week off before starting the therapy again).
The approval is based on the phase 3 CORRECT trial, which randomly assigned 760 mCRC patients who had prior treatment to either receive Stivarga or placebo. All patients in the trial received best supportive care. Stivarga was shown to extend median overall survival to 6.4 months compared with the 5 months for those in the placebo arm. The drug also delayed tumor growth by two months compared with the 1.7 months for those on placebo.
Common side effects include fatigue, hand-foot syndrome and diarrhea. The drug also carries a warning that severe and fatal liver toxicity can sometimes occur.
Stivarga has already been made available to some patients through an extended access program. Regorafenib is also being considered for FDA approval for patients with gastrointestinal stromal tumors (GIST).
Be sure to check out CURE's feature on colorectal cancer in our Winter issue.RELATED POSTS