BY ELIZABETH WHITTINGTON | DECEMBER 6, 2013
Pancreatic cancer, while hard to treat, is gaining awareness, research dollars and inspirational advocates. Learn more about how we're gaining on this aggressive cancer in the Winter issue of CURE.
BY ELIZABETH WHITTINGTON | NOVEMBER 26, 2013
Xalkori (crizotinib), a lung cancer drug that was approved in 2011 under the Food and Drug Administration's accelerated approval, has passed the last hurdle to regular approval. On Nov. 20, the FDA gave the ruling after a phase 3 clinical study confirmed that the drug works in patients who have metastatic cancer with a particular genetic mutation called ALK and who had progressed on platinum-based chemotherapy. Specifically, Xalkori works in lung cancers that are driven by a mutation in the anaplastic lymphoma kinase (ALK) gene, which occurs in about 5 percent of non-small cell lung cancers.
Accelerated approval is a conditional approval the FDA grants for drugs that show a significant improvement before a trial is completed. It can be based on survival, delayed disease progression or another endpoint that was pre-determined before the trial began. Efficacy of the drug must be verified in a later study, either a phase 3 or 4 trial before it can be given a regular approval.
Previous results, which led to the accelerated approval, were based on two single-arm studies, meaning all patients in the study received the investigational drug rather than being randomly assigned to receive the investigational drug or a standard of care. These studies showed Xalkori improved objective response rates, meaning the tumors shrunk with Xalkori in 50 percent and 61 percent of the time in the two studies, respectively.
This new approval is based on results that showed the drug delayed disease progression for 7.7 months compared to chemotherapy, which only kept the cancer from growing for 3 months. Side effects included vomiting, diarrhea, constipation, fatigue and vision impairments.
Many accelerated approvals later receive regular approval. A paper examining accelerated approvals and post-marketing studies found that from Dec. 11, 1992 to July 1, 2010, the FDA granted 35 oncology products accelerated approval, which accounted for 47 new indications. Clinical benefit was later confirmed in subsequent studies for 26 of the 47 new indications. The average time between accelerated approval and regular approval was more than four years, but ranged anywhere from less than a year to more than 12 years. During that time, three indications were revoked or had a restricted distribution due to follow-up studies.
To better understand this newest approval for Xalkori, we asked the FDA a few questions:
1. What is the significance of the regular approval for Xalkori?
• Xalkori received a broad indication for the treatment of ALK-positive metastatic non-small cell lung cancer (NSCLC) in 2011 because this magnitude of high response rates had not been observed with standard approved treatments for lung cancer. Because of this, we didn't want to require patients to progress on chemotherapy before enabling access to a drug that appeared to provide a significant benefit over the standard of care. Xalkori's accelerated approval in 2011 was based on response rates in single-arm trials, and its regular approval is based on a post-marketing trial that provides confirmation of the clinical benefit based on a randomized study against chemotherapy.
2. What does it mean to patients?
• Xalkori's regular approval highlights an evolving paradigm of very rapid drug development, where we are able to get drugs to the patients who need them most at an accelerated pace. Practically speaking for NSCLC patients, Xalkori's regular approval gives access to a therapy that demonstrates superior progression-free survival by about five months and a reduction in the relative risk of progression by about 50 percent. The post-marketing trial provided supporting evidence of higher response rates – 65 percent of tumors shrunk versus 20 percent in those receiving chemotherapy. The randomized trial also provided additional safety data, which helped the FDA determine that its risk-benefit was acceptable.
You can read more about the regular approval at fda.gov.
You can also read more about accelerated approval and other routes to FDA approval in CURE's article "A Primer on How Faster Approval Works."
We'll be featuring advancements in lung cancer in an upcoming issue of CURE, so stay tuned for more updates.RELATED POSTS
BY ELIZABETH WHITTINGTON | NOVEMBER 19, 2013
The stomach cancer community has seen incremental progress over the past year, but the next advancement in treatment research appears to be ramucirumab, a drug that works by cutting off the blood supply to a tumor.
Phase 3 study results, published last month in The Lancet, show that patients with gastric or gastroesophageal junction cancers that had progressed on first-line chemotherapy lived longer on the drug than on placebo (5.2 months versus 3.8 months). It also delayed disease progression. Side effects included high blood pressure and abdominal pain.
Based on these results from the REGARD study, the Food and Drug Administration granted ramucirumab priority review on Oct. 23, which means the agency will make a decision in six months. Typically, it could take up to 10 months for review without the designation.
Laura Goff, a gastrointestinal oncologist at Vanderbilt-Ingram Cancer Center in Nashville, Tenn., says it's likely the drug will be approved because the study showed a small but real improvement in survival in patients taking the drug compared with supportive care, such as pain medication and anti-emetics.
"Helping patients live longer is a very real benefit. However, the question of how much longer is what often spurs debate. On average, the group taking the drug lived about a month and a half longer than the group not taking the drug. Therefore, the treatment group did better, but not a lot better," she says. "The good thing is that there were very few side effects."
Currently, there is no approved treatment once the cancer has progressed on initial therapy, which means the investigational drug fulfills an unmet need--a requirement for the priority review designation. The FDA seems to be on a roll lately, so a decision may come even earlier. However, Goff says there are other chemotherapies that have been shown to be helpful in this setting.
"Going forward, in order to recommend to my patients, I will need to see what the FDA approval looks like. Also, since this drug is a monoclonal antibody, I would anticipate that it would be very expensive," she says.
While the REGARD study looked at ramucirumab used alone, the phase 3 RAINBOW trial is combining ramucirumab with paclitaxel to hopefully further extend survival without increasing side effects. "I am very interested in seeing the results of this agent when given in combination with chemotherapy to see what magnitude of benefit is achieved," Goff concluded.
The drug is also being examined in lung, colorectal and liver cancers.
Update: This blog was updated on Nov. 19 to include an email interview with gastrointestinal oncologist Laura Goff.RELATED POSTS
BY ELIZABETH WHITTINGTON | OCTOBER 3, 2013
The National Brain Tumor Society Summit kicks off today in Cambridge, Mass. While it may be a little late for most of us to attend, there is a live broadcast Friday morning at 8:30 EST. The webcast will include an update on the state of pediatric brain tumor research. If you can't make it, the recording, along with a recap of the entire summit, will be online in a few weeks.
You can find more information at braintumor.org/about-us/nbts-summit.
The National Brain Tumor Society is the combined organization of the National Brain Tumor Foundation and the Brain Tumor Society. The two groups came together in 2008. The organization has provided millions of dollars worth of grants to research groups, including pediatric brain tumor research and initiated the Defeat GBM Research Collaborative, which aims to double the five-year survival rate of patients with glioblastoma multiforme.RELATED POSTS
BY ELIZABETH WHITTINGTON | SEPTEMBER 30, 2013
With enrollment "opening day" of the health insurance exchanges happening on Oct. 1, there is a lot of confusion, misinformation and information overload. Here are a few resources to help you sort it out and get organized.
> The Cancer Support Community and the Association of Community Cancer Centers, along with 16 other cancer and patient advocacy organizations, produced the Cancer Insurance Checklist. This worksheet was created to help people--with either a history of cancer or high risk of cancer--shop for insurance coverage in their individual state's health insurance exchange. The website also includes a list of resources and a glossary.
> The American Cancer Society and its advocacy arm, ACS Cancer Action Network, have put together a fact sheet of tools and information for patients and survivors to help them navigate the Affordable Health Care Act. You can find it at acscan.org/healthcare/learn.
> The LIVESTRONG Foundation has produced a five-step guide to insurance enrollment. It is also partnering with the advocacy group Young Invincibles and the social action news site RYOT to reach the young adult population and encourage them to get insurance coverage. On Oct. 2, the three groups will host an in-person and live Google+ Hangout session geared toward helping cancer survivors navigate healthcare enrollment.
> Naturally, the homepage for WhiteHouse.gov has recently been filled with information on the healthcare law, including a short video of "What Obamacare Means for You," infographics and a summary of healthcare choices and premiums expected in several states (whitehouse.gov/healthreform/map). Healthcare.gov also provides information on the marketplace, along with a tool to determine if a user would qualify for lower insurance costs.
> NPR.org has a great assortment of articles that explains many of the current and upcoming healthcare law pieces.
Do you know of any other tools that help cancer patients and survivors navigate the insurance process? If so, please share!RELATED POSTS
BY ELIZABETH WHITTINGTON | SEPTEMBER 11, 2013
In the fall issue of CURE, we look at how patients can prevent common treatment-related side effects, including pharmaceutical strategies and integrative therapies.
We asked our Facebook fans, "Did integrative therapy approaches help you in dealing with side effects from cancer or treatment?"
We received many responses, and they were all across the board.
From yoga and acupuncture to laughing and music therapy, patients and survivors shared what integrative therapies helped them deal with side effects and the psychosocial issues in dealing with cancer and its effects. Many large cancer centers and hospitals offer integrative therapies, and several now have their own dedicated departments and staff.
However, not everyone was quick to answer. Some patients said it was hard to find integrative therapies, especially if they were not offered at the location they received their cancer treatment. If integrative therapies aren't offered at your clinic, there are several non-profit groups that offer support, information and services.
Here are a few tips to find integrative therapy options:
1. Talk with a dietitian. Foods that boost immunity, lower fatigue and have the right amount of calories may help reduce certain side effects. Many insurance plans and Medicare cover nutrition services if it is prescribed by your physician. You can get a referral from your medical team. You can also find registered dietitians at eatright.org. Use your ZIP code and refine your search to "expertise: oncology, cancer nutrition."
2. Ask your medical team and fellow survivors about fitness programs geared toward survivors, including those at gyms, yoga studios and other local hospitals. LIVESTRONG at the YMCA is a twelve-week program that helps survivors increase their health and fitness, but also aims to reduce the severity of side effects. The program is available at certain YMCAs across the country.
3. Investigate whether your insurance company offers discounts or coverage on health programs, acupuncture, massage or other non-traditional therapies.
4. Reach out to local support groups and non-profits that may offer integrative therapies. Organizations such as the Cancer Support Community offer onsite support and classes, including yoga and meditation.
Where did you find your integrative therapy services? Did it help alleviate side effects?RELATED POSTS
BY ELIZABETH WHITTINGTON | SEPTEMBER 6, 2013
Because of the hard-to-treat nature of pancreatic cancer even small gains are cause to take notice, which is why the approval of Abraxane (paclitaxel) is welcome news. Abraxane is the first treatment to be approved for pancreatic cancer in nearly eight years.
Results of the MPACT study, a phase 3 international trial that that examined Abraxane and gemcitabine, found the combo extended overall survival by more than 7 weeks when compared with gemcitabine alone. It also improved one-year survival from 22 percent to 35 percent. Those results helped the Food and Drug Administration decide to approve the regimen for first-line therapy for metastatic disease. The drug was priority review approval back in May and a decision was expected later this month.
Abraxane is a form of Taxol that is encased in a protein and administered intravenously. This formulation helps reduce severe allergic reaction, and the protein may make it easier for paclitaxel to reach cancer cells than traditional Taxol. Side effects of the combination includes fatigue and peripheral neuropathy.
It's believed that Abraxane helps target the stroma, a matrix of cells and molecules that are tightly knit together around the cancer. If Abraxane can weaken the stroma, gemcitabine may have a better chance in targeting the cancer cells.
Abraxane is already approved for advanced lung and breast cancers.RELATED POSTS
BY ELIZABETH WHITTINGTON | AUGUST 15, 2013
It's pretty low to fake cancer to receive money, sympathy or attention. But CNN is reporting that an oncologist in Michigan is being charged with fraud for telling people they had cancer when they didn't and profiting from the unnecessary treatments, among other complaints. He's being accused of pocketing about $35 million over two years in false Medicare claims.
Employees of the clinic, including another oncologist and nurses, have stepped forward with claims of abuse and neglect (complaint), but it breaks my heart to know this may have gone on for so long. Did it really take two years to finally charge this man? He's being officially accused of healthcare fraud, but reading over the witness accounts, I suspect there will be many more charges and lawsuits to come.
Some of the charges he's being accused of include:
-Administering unnecessary chemotherapy to patients in remission
-Deliberate misdiagnosis of patients to justify giving expensive treatment
-Administering chemotherapy to end-of-life patients who would not benefit
-Deliberate misdiagnosis of patients to justify testing
-Deliberate misdiagnosis of anemia to justify treatments
For many patients, they must now wait for their medical records to be returned from the FBI and begin the search for another doctor. We talk about the dangers of overtreatment and overscreening, but my goodness...I'm just floored.RELATED POSTS
BY ELIZABETH WHITTINGTON | JULY 30, 2013
We had an inkling it was coming, but that doesn't minimize the fact that this is big news.
The U.S. Preventive Services Task Force (USPSTF) is recommending high-risk individuals be screened for lung cancer. Yes, the same panel of experts that recommended we take a step back from screening mammography and the prostate-specific antigen (PSA) test because the harms may outweigh the benefit for some individuals.
That's why this is big news. With the USPSTF's recommendation (currently in a draft version), it's ultimately saying that screening for lung cancer in this population will save lives--about 20,000 a year. And there have been studies to attest to this fact, namely The National Lung Screening Trial. (You can read more about the trial in "Good News to Catch a Bad Disease" from CURE, 2010.)
The last update the USPSTF made regarding lung cancer screening was in 2004, when it concluded that there was insufficient evidence to recommend for or against screening. Nearly 10 years later, the panel's recommendation is backed by a large, randomized clinical trial.
The USPSTF is recommending low-dose computed tomography (CT) scans annually for adults aged 55 to 79 years old who have no signs or symptoms of lung cancer but are at high risk because of their current or former smoking history. High risk is defined as a history of heavy smoking and either a current smoker or someone who quit within the past 15 years. The Task Force defines "heavy smoking" as essentially smoking a pack of cigarettes every day for 30 years (or two packs for 15 years, and so on). You can read about the meaning of the USPSTF's recommendation here.
The upside: Based on previous studies, 14 to 20 percent of lung cancer deaths could be averted (about one life saved for every 320 people screened). The downside: The increased number of false-positives (where the CT scan shows cancer when there is none) and radiation exposure (although minimal) from the imaging scan. This is why individuals who do not fall into the high-risk category should not be screened--because the risks (radiation, overtreatment, anxiety) may outweigh the benefits. The USPSTF has shared the full draft of its recommendations for public review. You can find it here. Public comments will be gathered until Aug. 26.
Several medical organizations have already developed guidelines recommending lung cancer screening, including the American Society of Clinical Oncology and the American Thoracic Society. The American Cancer Society issued new guidelines earlier this year that also called for high-risk individuals (aged 55 to 74 years) to be screened for lung cancer with low-dose CT scans annually.
With an official recommendation from the USPSTF, it is assumed that Medicare and private insurance companies will begin to cover these routine screenings for high-risk individuals, as noted in the Affordable Care Act.
And if you're worried about the cost of screening these individuals, consider this: A report from Health Affairs in 2012 noted that the cost of screening will be much less than the healthcare costs associated with treating advanced lung cancer. The cost per life-year saved would be less than $20,000, which is actually more cost-efficient than mammography.
Plus there is also a suggestion that undergoing lung cancer screening may help smoking cessation rates. You can't beat that.
Update: Talking with lung oncologist and CURE advisory board member, Dr. Mohammad Jahanzeb, he has high hopes for the recommendation's effect.
"[It] will no doubt result in saving thousands of lives (by my estimate, about 15,000 lives per year in the United States alone)," he wrote in an email. "It will perhaps save a lot of patients from expensive, yet non-curative treatments for advanced stage lung cancer."
Updated July 31, 2013RELATED POSTS
BY ELIZABETH WHITTINGTON | JULY 20, 2013
A research team, which includes members from Vanderbilt University and University of Louisville, is exploring the attitudes of heart health, stress level and lifestyle factors in cancer patients prior to their diagnosis, during treatment and now as a cancer survivor. Your input will help influence how healthcare providers address the unmet needs in cancer patients as they transition to survivorship.
If you are a cancer survivor and have five minutes – please share your opinion by taking the following brief survey. All responses from a short survey will be collected in anonymous fashion.
Hopefully, the researchers will share their results with us when they are available!RELATED POSTS