Why regulation matters ... but also flexibility

BY ELIZABETH WHITTINGTON | FEBRUARY 9, 2012

Margaret Hamburg, MD, the Commissioner of the FDA released the statement below marking the 50 years from when thalidomide became a headline in the U.S. It's an interesting history lesson, although I'm a little disappointed she didn't mention that thalidomide later became a treatment (and springboard for other treatments) for myeloma - which is much more common than leprosy.

I also want to draw your attention to one of her closing statements:

Going forward, smart regulation requires regulatory flexibility that responds to changing situations, new information and new challenges. It also demands that we advance regulatory science: the knowledge and tools necessary for the meaningful and timely review of products for safety, efficacy, quality and performance.

The FDA has made some decisions lately that does require flexibility, including the Avastin decision in metastatic breast cancer. There have been some bumps along the way for sure, but as long as the agency remains flexible and allows itself to evolve with science and the increasingly loud voice of the public, it will be interesting to see where this takes us.

It's also worth noting that the agency approved several notable cancer treatments in 2011. And we're well on our way in 2012, as a few new treatments have already been approved this year ahead a schedule. Here's hoping for promising treatments and even some cures.

50 Years after Thalidomide: Why Regulation Matters
Posted on February 7, 2012 by FDA Voice

By: Margaret Hamburg, M.D.

Fifty years ago, the vigilance of FDA medical officer Dr. Frances Kelsey prevented a public health tragedy of enormous proportion by ensuring that the sedative thalidomide was never approved in the United States. As many remember, in the early 1960's, reports were coming in from around the world of countless women who were giving birth to children with extremely deformed limbs and other severe birth defects. They had taken thalidomide. Although it was being used in many countries, Dr. Kelsey discovered that it hadn't even been tested on pregnant animals.

Dr. Kelsey's reaction to thalidomide exemplifies the FDA's mission: protecting and promoting the health of the American people, using science for regulatory decision-making.

Now I know that in some circles regulation is viewed as a roadblock to innovation and economic growth. But in actuality, when done right, regulation isn't a roadblock; it's the actual pathway to achieving real and lasting innovation.

Smart, science-based regulation instills consumer confidence in products and treatments. It levels the playing field for businesses. It decreases the threat of litigation. It prevents recalls that threaten industry reputation and consumer trust, not to mention levying huge preventable costs on individual companies and entire industries. And it spurs industry to excellence.

The tragedy of thalidomide led to changes that strengthened both the regulatory and scientific environment for medical product development and review.

In response to the public uproar, in 1962 Congress enacted the Kefauver-Harris amendments to the Federal Food, Drug and Cosmetic Act. Thanks to these new amendments, manufacturers had to prove that a drug was not only safe, but also effective. Approvals had to be based on sound science. Companies had to monitor safety reports that emerged postmarket and adhere to good manufacturing practices that would lead to consistently safe products. And there were new protections for patients.

The amendments not only benefited patients, they helped industry, raising scientific standards that eventually ushered in today's sophisticated, science-based life sciences industry.

For the very first time, many companies put in place research and development programs, including the design and implementation of controlled clinical trials. Major therapeutic breakthroughs resulted, including the use of beta blockers in patients after a heart attack and angiotensin-converting enzyme inhibiters to improve survival in patients with heart failure. All of these were good news for public health and for corporate bottom lines. The best drugs and treatments rose to the top, not simply those that were most heavily marketed.

The Harris-Kefauver Amendments created a culture of quality and innovation that laid the foundation for our current regulatory environment which fosters a domestic pharmaceutical industry that is second to none.

Going forward, smart regulation requires regulatory flexibility that responds to changing situations, new information and new challenges. It also demands that we advance regulatory science: the knowledge and tools necessary for the meaningful and timely review of products for safety, efficacy, quality and performance.

Thalidomide, once again, is a good example. It came back on the U.S. market in 1998 after data showed it was safe and effective to treat a complication of leprosy. In an appropriate balancing of benefit and risk, FDA required strong safety monitoring and a strict dispensing plan before approving the drug.

Regulation such as this requires a strong, robust FDA, one endowed with the necessary resources to ensure smart, sound, science-based regulation.

• More trail mix
• When the cancer comes back
• Two new drugs approved, one pulled from the pipeline
• Emerging treatment option for myeloma presented at hematology meeting
• The big news from the American Society of Hematology

Dating Teleconference Today

BY LINDSAY RAY | FEBRUARY 8, 2012

Dating can be a daunting task. When you first meet someone, there is the awkward dance of getting to know one another. Adding in a cancer diagnosis can make the footing even trickier. Figuring out when and how to disclose your cancer information is a question many patients and survivors struggle with, and there's no one-size-fits-all answer.

There's also a certain amount of fear for both the patient/survivor and the person he or she is dating. A recent commenter on the blog "Would You Date a Cancer Survivor?" wrote:

I'm trying to decide whether or not to date someone with cancer. [...]I am a caregiver for two aging parents, one who has alzheimer's and three progressive, fatal and untreatable medical conditions. We have no other family member living on this continent to help with my parents and I'm not up to taking on being a caregiver for and losing a boyfriend as well. I know little about cancer and survival rates or what someone with cancer goes through during treatment so I really have no idea of what I'd be getting myself into where I to continue dating him. Any thoughts or advice would be appreciated.

To help patients and survivors navigate the dating scene, the Young Survival Coalition and Bright Pink are hosting a teleconference about romance and the issues that surround it, such as self image, confidence and intimacy, tonight at 8 p.m. ET. The speakers leading the discussion will be Jean Rowe, LCSW, OSW-C, and breast cancer survivor Jamie Pleva.

To RSVP, email your full name to RSVP@BeBRightPink.org

To find out more information, visit: brightpink.org/event-020812-teleconference.

• Make a difference
• For the Cure to a broken heart
• An update on the Cancer Genome Atlas for breast cancer
• Video blog: Test could spare some DCIS patients from radiation
• Video blog: HER2-targeted agents

Making every breath matter

BY ELIZABETH WHITTINGTON | FEBRUARY 7, 2012

As the nation prepared for Super Bowl Sunday, many were introduced to the story of Keasha Draft, the newlywed wife of retired NFL linebacker Chris Draft.

A beautiful love story, except that Keasha was diagnosed with lung cancer last year. She was 37 years old. Five weeks after their wedding, she passed away on Dec. 27. The ESPN video is below.

"She courageously faced lung cancer, showing us all with every breath that we all need to hold onto life and love with both hands for as long as we can." --The Chris Draft Family Foundation

• Women and lung cancer: Time to turn the tide
• The Great American Smokeout
• I'm not dead yet!
• Xalkori approved for non-small cell lung cancer
• Drug approvals: Are we in a new age yet?

More trail mix

BY SUZANNE LINDLEY | FEBRUARY 7, 2012

Ever hear of trail mix? Those of you who know me know that it is one of my favorite snacks; especially the kind with lots of chocolate!! The simple snack is a combination of dried fruit, nuts and chocolate. It was made as a quick treat to be taken along on the trail. I pick through my trail mix and really enjoy some of it and then there are some pieces that I don't like (the raisins) just get left in the bag.

Those of you who don't know me have probably near heard scans results and trail mix mentioned in the same sentence. You may have never before considered the similarities ... that with both there can be bits that are really, really good and other random pieces that are easier not to digest. Once again I'm experiencing a "trail mix" kind of scan. A few of the tiny tumors in my lungs have disappeared. One of the larger tumors continues to show stability. These are both reasons for celebration. Areas in my spine light up and one lung lesion showed a growth spurt. In spite of lights and spurts, this a positive scan for me with more chocolate than raisins! So, just like with trail mix I'm going to enjoy the good stuff.

Thanks to all of you - my friends who have sent prayers and thoughts, emails and phone calls. I'm sending all the good stuff in this mix of life back to you - continued HOPE for good scans, great procedures, easy recoveries, many magical moments and wonderful memories ahead.

Enjoy a happy day today!
Suzanne

Suzanne Lindley has been living with metastatic colorectal cancer since 1998. She is the founder of YES! Beat Liver Tumors, an organization for individuals living with metastatic liver tumors, and an advocate for Fight Colorectal Cancer. She is also a regular guest blogger for CURE.

• Why regulation matters ... but also flexibility
• When the cancer comes back
• Two new drugs approved, one pulled from the pipeline
• Emerging treatment option for myeloma presented at hematology meeting
• The big news from the American Society of Hematology

Make a difference

BY ELIZABETH WHITTINGTON | FEBRUARY 6, 2012

Today, the breast cancer talk wasn't of Komen or Planned Parenthood or pink handguns.

Feb. 6 was the day that Rachel Moro and Susan Niebur, breast cancer survivors in every sense of the word, died.

Rachel had been living with metastatic breast cancer and blogged at The Cancer Culture Chronicles as @ccchronicles. Her profile statement is especially meaningful today: "It's time to move beyond pink ribbons and messages of "breast cancer awareness," and start agitating for real and meaningful action in the fight to eradicate this disease for good."

Susan had lived with inflammatory breast cancer, her fourth bout with cancer.

Both were prominent bloggers. Rachel's death was sudden. Susan had been sick for a while. Both chronicling their journey and supporting others in their online communities of women dealing with breast cancer, either as newly diagnosed, survivors or those living with metastases.

As I was reading reactions of others on Twitter about their deaths, a colleague noted that Susan had been interviewed for a story in the Spring issue--one that focused on how to help children cope with a parent's diagnosis. This was a perfect fit for Susan, who blogged under the name @WhyMommy at Toddler Planet: The joy of life after cancer. My hope is that the article will be just one more piece of a lasting legacy of helping others cope with this horrible disease.

Susan's family noted her passing on her blog today and wrote: "In lieu of flowers, please consider furthering Susan's legacy through a contribution to the Inflammatory Breast Cancer Research Foundation. Or please choose to make a difference somewhere, anywhere, to anyone."

Make a difference.

These women did. Unfortunately, sadly, there will be other women who will fill the void left by Rachel and Susan...women with cancer, or those who will be diagnosed, who will become a voice for others to draw strength and inspiration from. We need them. Just like we need Rachel and Susan.

Those wanting to remember Rachel and Susan tonight can join the #bcsm Twitter chat tonight at 9 pm ET/6 pm PT.

• Dating Teleconference Today
• For the Cure to a broken heart
• An update on the Cancer Genome Atlas for breast cancer
• Video blog: Test could spare some DCIS patients from radiation
• Video blog: HER2-targeted agents

When the cancer comes back

BY GUEST BLOGGER | FEBRUARY 3, 2012

As my sister drove me to the hospital on a wintery night after my leukemia returned for the second time, I said over and over, "I'm never going to see my grandchildren. I'm never going to see my grandchildren."

I thought I was at the end of the line. I had already had three bone marrow transplants, each preceded by intensive chemotherapy, when my doctor told me that I had relapsed again.

On the night of Dec. 21, 2008, I had felt a little better after feeling sick for several days. My daughter and I were making cookies. Then I fell to the floor.

After she helped get me to bed, I took my temperature and discovered that I had a fever. I called Dr. Edwin Alyea, my physician at the Dana-Farber Cancer Institute, and he said he was sorry to tell me on the phone, but the pathology report on my latest bone marrow biopsy showed that I had relapsed.

He said he would understand if I didn't want to go through treatment again, but if I wanted to proceed, he had an idea for a new regimen.

I wanted to live. I wanted to see my three children, 16, 19 and 23, continue growing up into the wonderful adults I knew they would be.

I wanted to walk my Labrador retriever, play tennis, run a road race and return to my job as a newspaper reporter.

Dr. Alyea had said to go to the emergency room and get admitted, and then he would come see me and there would be a plan. It turns out I had pneumonia, so they had to treat that before they did anything else.

I was first diagnosed with acute myeloid leukemia in 2003 after feeling extremely tired while running a 10-kilometer road race near my home in South Hadley, Mass. Thinking I was probably anemic, not eating right or training poorly, I went to my internist. He said my blood counts were abnormal and sent me for a bone marrow biopsy. I soon learned that I had AML, a fast-moving cancer of the blood.

The "What, me?" response was pretty strong. I ate well and exercised, I didn't smoke and I was slender. But I had to accept it when, within about a week, I found myself in a bed at Boston's Brigham and Women's Hospital, about 90 miles from home. Under the care of doctors from Dana-Farber, I received three rounds of in-patient chemotherapy, with rest periods in between at home, and then my first bone marrow transplant.

It was an autologous transplant, meaning they used my own new, clean stem cells, removed after two rounds of chemotherapy and then returned to me in a rescue mission after a third and powerful round basically cleared out my bone marrow.

I was in remission, but my first Dana-Farber doctor, Daniel J. DeAngelo, told me that remission is not cure. He said that after two years you break out the Champagne, but only after five years can you use the word cure.

After two, then three-and-a-half years passed and normalcy wrapped its arms around me, I got another shock. The leukemia was back. I learned this just after I played in, and won, a doubles match at a tennis tournament.

"Leukemia is curable," DeAngelo said. "We'll get you back on your feet."

"I am on my feet," I thought to myself as I left his office. And then I burst into tears.

This time I would get an allogenic transplant, with stem cells coming from a donor. After the leukemia cells are killed by chemotherapy and healthy donor cells fill your bone marrow, the donor cells patrol your body to fight off any leukemia that might try to sneak back in.

But after six months, I learned a new term, graft failure: The donor cells had packed up and left, leaving my bone marrow almost empty. The cause was uncertain, and the donor was a good match who agreed to try again. After more chemotherapy, I had transplant No. 3.

Six months later, I had that second relapse.

There were so many things to worry about that a nurse who called me Nervous Nellie told me over and over, "Don't worry, they'll figure it out." And as you will see, the incredible doctors did just that.

I had a new donor and a new chemotherapy regimen consisting of three drugs. One of them, Atgam, is made from rabbit serum, and the nurses called it shake and bake, which is exactly what I did while I received it intravenously.

The transplant, on Jan. 31, 2009, went smoothly, but a few weeks later, I developed a severe blood infection, went into kidney failure and lapsed into a coma. One night, it was touch and go. My ex-husband brought my daughter and told my sons to come quickly and to bring their dark suits. Dr. Alyea met with them and said that there were many things wrong with me, but they would tackle them one by one.

And somehow I struggled to the surface, confused, scared and unable to speak. My legs were swollen like tree trunks, and I needed two nurses to turn me over. The nurses, who ranged from kind and gentle to kind and commanding, helped me pull through.

I regained my voice when a nurse nicknamed Big Red asked me, "What's my name?"

"Lisa," I answered in a grainy whisper.

"Say it loud!" she said.

It took all of my strength to say, "Lisa, Lisa, Lisa!"

But from then on I could speak.

After extended sessions of dialysis, my kidneys returned to normal. I was in bed for more than a month. When I got up, slowly and needing oxygen at first just to sit on the edge of the bed, I had to learn how to walk again. Total time in the hospital: three and a half months.

Recovery has been long and slow. Because you are like a baby with no immune system, during the first year you can't go into crowded places, and when you do go anywhere, you need to wear a mask and gloves. When I got back to walking, I was so wobbly I was like a Gumby doll. One day I fell over backwards, hitting my head on the pavement and earning a trip to the emergency room.

Two years after my transplant, I met my donor, Denise. Donors come from all over the world, but in an example of one degree of separation, Denise lives in New Jersey and is in a book group with one of my friends. Like all donors, she did an incredibly generous thing.

We hugged and grew teary as I thanked her multiple times and she thanked me for giving her a chance to save a life.

Now I am pretty much back to myself, despite graft-versus-host disease, a common complication after transplant in which the "graft" recognizes the "host" as foreign and attacks it. I don't have a bad case, but I do take big handfuls of pills and visit Dana-Farber for frequent check-ups.

I watched my daughter graduate from high school and go to college. I was at my middle son's college graduation and shared my older son's joy when he told me that he gave his wonderful girlfriend an engagement ring.

I rejoined my tennis team and, with my longtime doubles partner, won my first match back, an incredible thrill. I worked back up to running, which took a while, because when I first tried, my feet felt like they were made of lead. The Saturday after Thanksgiving this year, my son and I joined some 3,000 other runners in a scenic six-mile crossrace called the Talking Turkey.

I couldn't go back to work fulltime, but I have been doing freelance writing.

I am left with this question: How do you deal with it when you know that the same bus can hit you twice? You worry that all sorts of things – mainly fatigue - can signal a relapse. I talk to myself. "Maybe you're tired because you just played two hours of tennis." Oh, right.

The passage of time helps. So does hitting the ball on the sweet spot, or doing yoga, or feeling my feet hit the ground when I run, or sitting on the couch watching TV with whichever child is home, or watching the dog lie in the sun at my feet while I write or read. I laugh a lot. Sometimes when I roll over in bed, I flash back to when I couldn't do it myself, and I am so grateful to do that simple thing that I never would have thought about before.

I try to take it one step at a time, appreciating all that I have and looking forward to the good things.

Born and raised in New York, Ronni Gordon lives in South Hadley, Mass., where she raised her three children, Ben, 26; Joe, 22; and Katie, 19. She is a graduate of Vassar College with a master's degree in journalism from Boston University. She spent most of her career in daily journalism as a features writer at the Republican, in Springfield, Mass., and has been published in The New York Times, The Philadelphia Inquirer and elsewhere. She now spends her time freelancing, writing her blog (runnerwrites.blogspot.com) and working on her tennis game.

• Why regulation matters ... but also flexibility
• More trail mix
• Two new drugs approved, one pulled from the pipeline
• Emerging treatment option for myeloma presented at hematology meeting
• The big news from the American Society of Hematology

For the Cure to a broken heart

BY KATHY LATOUR | FEBRUARY 3, 2012

First a recap. For the past four days we have been bombarded with information about the decision by Susan G. Komen for the Cure to remove funding from 18 Planned Parenthood sites, using the reasoning that they don't fund any organization under investigation. Well, within about 12hours it became clear that the decision came straight from the office of Karen Handel the new Komen Vice President of Policy and a former Georgia office holder and gubernatorial candidate who is anti-abortion and has even gone so far as to say she is anti-Planned Parenthood.

Now there is enough evidence to confirm that the decision to stop funding organizations under investigation was made in December specifically to stop funding Planned Parenthood, and again it was Handel that drove the decision. Komen has been down the Planned Parenthood path a number of times in the past decade and had made what everyone thought was a final decision–-it would be funded. But that was before someone recommended Karen Handel as the best person for the job of Vice President of Policy.

That person is known in the ranks of Komen as someone who mixes a particularly powerful batch of Kool-Aid that Nancy Brinker has become addicted to.

So where are we? Komen stands by its decision, but now Nancy Brinker goes on television to say it's a policy change to give money to direct service organizations, not pass through groups, which means they want to give the money to the groups that do the mammograms and Planned Parenthood evidently doesn't do that, they must send women out and then pay for it from Komen funds. So why the switch?

Personally, I think Nancy considered what their decision would mean for poor women in Dallas who go to Parkland Hospital, which, I am fairly sure gets Komen funds for its screening program and has been under investigation for two years – and found guilty, by the way, in areas that don't pertain to screening. And no one thought about this before? Komen is in a mess and this is where the broken hearts come in.

At the first headline, I blew it off. Komen has been a target before, a lot. They are big, they raise a lot of money, and the well over 100 affiliates raise money through the Race for the Cure, 75 percent of which is kept local for their own screening, education and treatment needs. This money reaches the very poorest of the poor and the women who work tirelessly for Komen affiliates make a real and tangible difference in the lives of these women. These are the salt of the earth of the American heartland where women are proud to take care of each other and dedicate thousands of hours a year to do just that.

Speaking around the country at Komen events, I have met hundreds of Komen volunteers and I would be proud to call any of them a friend. I have never asked anyone if she was a Democrat or Republican or if she was for or against abortion.

With the 25 percent the national foundation receives they fund numerous other projects including basic research, and as a 25-year and 7-year survivor, and a woman who lost her mother to breast cancer, I count on research to be sure my daughter doesn't have to face this disease, and if she does, she lives through it.

I have been president of the board of two nonprofits funded by Komen, one, the Bridge Network, provides direct support to women diagnosed with breast cancer who have no insurance. The other was Gilda's Club North Texas, now Cancer Support Community. These programs would have had a much harder time without Komen.

But what has really broken my heart this week is talking to the women who built the foundation, some of whom spent upwards of 20 years committed to this cause. The woman who created the policy office at Komen, a survivor herself, spent endless hours in the halls of Congress during her 10-year tenure, which ended in 2008. She worked both sides of the aisle to overcome any idea that Komen was politically driven. She also convinced the leaders of our country that this organization was about more than pink ribbons, it was about the power of the vote. She organized advocacy groups before we understood what advocacy was about. For 10 years she grew the office, only to watch the newest occupant, who has been with Komen nine months, bring it down in two days.

These were strong women who wouldn't take no for an answer, just the way Nancy Brinker wouldn't when she started the organization as a promise to her sister. There are some of the "old guard" left, women who took Komen to the heights in the nonprofit world to make it what it was on Monday of this week: An organization committed to ending breast cancer that was built on integrity and very decidedly non political – because breast cancer doesn't care what you believe, it is an equal opportunity killer. There is no place for politics at Komen.

There are many of us who fear that Komen will not be able to recover from this in part because of the way it has been handled. If I had been Karen Handel, I would have offered my resignation immediately, and I was hoping she would have the honor to do that. Then I saw the retweet she posted, "Just like pro-abortion group to turn cancer orgs decision into a political bomb to throw. Cry me a freaking river" and feared the worse. This woman has no grace, no courage and an ego that doesn't care if she brings down in nine months what it took others 30 years to build.

Resign Karen. You owe it to a lot of people.

1:27 p.m. Nancy Brinker has made a statement that includes the following

"We will continue to fund existing grants, including those of Planned Parenthood, and preserve their eligiblity to apply for future grants," Nancy G. Brinker, the agency's ambassador, said in a statement.

So Handel is still there and to shut down the firestorm, PP can apply. This does not mean they will be funded.

• Dating Teleconference Today
• Make a difference
• An update on the Cancer Genome Atlas for breast cancer
• Video blog: Test could spare some DCIS patients from radiation
• Video blog: HER2-targeted agents

After a dry spell, metastatic prostate cancer sees dramatic improvement with new therapies

BY ELIZABETH WHITTINGTON | FEBRUARY 1, 2012

Prostate cancer has seen its share of progress over the past few years, including several new drugs that have been approved for patients with metastatic disease. On Tuesday, results of two clinical studies were announced that foreshadow additional therapies that will help further extend survival and improve quality of life.

While prostate cancer is one of the most common cancers, it has a 99 percent five-year survival rate, with most patients diagnosed at an early stage. But for more than 28,000 men this year, prostate cancer will be fatal. For many of these patients, those with metastatic prostate cancer to the bones or men whose cancer doesn't respond to standard hormonal treatment, Alpharadin and MDV3100 could be very useful.

Alpharadin (radium-223) is an intravenous drug that uses alpha particles (a type of ionizing radiation) to kill cancer cells. MDV3100, a new form of hormone therapy, is a pill that works by preventing the tumor cells from using testosterone that can spur cancer growth. The two drugs work very differently and both improve survival, which led some researchers to suggest that combining these agents with other therapies could produce a synergistic response.

In the Alpharadin trial (called ALSYMPCA), 922 prostate cancer patients with bone metastases were randomized two to one to Alpharadin or placebo, with all patients receiving best supportive care. Because the most common site of prostate cancer metastasis is typically bone tissue, targeting a drug to delay or attack cancer at the bone seems like a smart idea. That's what Alpharadin does--it targets the cancer cells that have invaded the bone tissue. It's not a cure, but it did prolong survival by 30 percent (14 months versus 11.2 months) and delayed the time to a patient's first skeletal-related event, such as a spinal cord compression or bone fracture by a median of 5.2 months (13.6 months versus 8.4 months). The side effects of the drug are surprisingly minimal--a slight increase in neutropenia and diarrhea.

The AFFIRM trial, which involved MDV3100, also randomized patients two to one with the experimental treatment versus placebo. The trial included 1,199 patients with metastatic prostate cancer that had progressed after treatment with docetaxel and other hormonal therapies.

The drug increased survival by a median of 4.8 months over placebo (18.4 months versus 13.6 months). Howard Scher, MD, chief of genitourinary oncology at Memorial Sloan-Kettering Cancer Center in New York and co-lead investigator of the AFFIRM study, also noted that more patients treated with the drug experienced tumor shrinkage and more than a 50 percent decline in PSA levels. The drug also delayed cancer growth by five months. Side effects, which included diarrhea, fatigue and hot flashes, were well-tolerated and had similar rates of toxicity to the placebo arm.

Nicholas Vogelzang, MD, chair and medical director of the Developmental Therapeutics Committee of US Oncology, who moderated the press briefing on Tuesday, called the results unprecedented. "This will definitely change the way we take care of patients every day in the office."

He may not have to wait long. MDV3100 has been submitted to the Food and Drug Administration. And both MDV3100 and Alpharadin have been granted fast-track status, which means they will likely have a quicker-than-usual review by the agency.

• Zytiga approved for metastatic prostate cancer

Two new drugs approved, one pulled from the pipeline

BY LINDSAY RAY | JANUARY 31, 2012

Skin Cancer

Ahead of its March 8 deadline, the Food and Drug Administration (FDA) approved Erivedge (vismodegib, GDC-0449) on Jan. 30 for patients with locally advanced basal cell carcinoma (BCC) that can't be treated with surgery or radiation or who have metastatic BCC, making Erivdege the first drug approved by the FDA for metastatic BCC. In another first, Erivedge is the first drug approved that works by inhibiting the Hedgehog pathway, which is involved in controlling cancer cell division and active in most BCCs. The drug is a pill taken once a day.

BCC, along with squamous cell carcinoma, is a non-melanoma skin cancer and one of the most common types of skin cancer, with an estimated 3.5 million cases diagnosed each year. Advanced BCC is rare, however, and disfiguring, which is why the drug fills an unmet need in this patient population. Last April, we covered some of the research about Erivedge (then known as GDC-0449) and even the first patient to try the drug, which you can read about here.

The approval in based on a multicenter trial with 96 patients. Of those with metastatic BCC, 30 percent had partial tumor shrinkage, and of those with locally advanced BCC, 43 percent had partial or complete tumor shrinkage.

Side effects include nausea, fatigue, hair loss, diarrhea, changes in taste and weight loss. Some serious side effects may occur, so a boxed warning will be included on the drug alerting physicians to potential risk of death or birth defects for an unborn baby. Doctors are required to verify pregnancy status before starting treatment.

A month's supply should cost $7,500, and Genentech (the drug's manufacturer) estimates a treatment course will last 10 months, totaling $75,000. The Patient Action Network Foundation has announced they will now offer co-payment assistance up to $7,500 per year for out-of-pocket expenses. To see the eligibility guidelines and for more information about the program, visit the Patient Action Network Foundation.

This drug should be available in one to two weeks, per the manufacturer.

For more information about Erivedge, visit www.erivedge.com.

Kidney Cancer

Just a few days before, on Jan. 27, the FDA also approved Inlyta (axitinib) for patients with advanced renal cell carcinoma (kidney cancer) who haven't responded to previous treatment.

Inlyta targets the vascular endothelial growth factor (VEGF) pathway, which is important to the development of new blood vessels in tumors, which helps tumors grow. The approval follows a 723-patient trial in which patients on Inlyta had a median progression-free survival of 6.7 months compared with the 4.7 months on the standard treatment, Nexavar (sorafenib).

Inlyta is a pill taken twice daily and is expected to cost around $8,900 per month. Side effects include diarrhea, fatigue, high blood pressure, decreased appetite and nausea. Because it can cause high blood pressure, individuals with this condition should have it controlled before taking Inlyta. Also, sometimes serious bleeding problems can occur, so patients with untreated brain tumors or gastrointestinal bleeding should not take Inlyta.

For more details, visit www.inlyta.com.

Non-Hodgkin Lymphoma

Finally, pixantrone was pulled from the FDA approval pipeline earlier this week. In 2010, the FDA rejected the drug, but Cell Therapeutics (its manufacturer) appealed and resubmitted the drug for approval as a treatment for non-Hodgkin lymphoma patients who no longer responded to other therapies. An FDA advisory meeting was set for Feb. 9, with a possible approval in April, but the manufacturer pulled the application to allow the company more time to prepare for the review. It plans to resubmit later this year.

• Why regulation matters ... but also flexibility
• More trail mix
• When the cancer comes back
• Emerging treatment option for myeloma presented at hematology meeting
• The big news from the American Society of Hematology

Cancer screening rates low

BY LENA HUANG | JANUARY 31, 2012

The percentage of Americans getting screened for cancer is below national targets with lower rates in the Asian and Hispanic populations, says the Centers for Disease Control and Prevention (CDC) and the National Cancer Institute in a study released last Friday.

The study revealed that in 2010 breast cancer screening rates were 72.4 percent, which is below the target of 81 percent set by Healthy People 2020, a government initiative to set benchmarks to measure the impact of prevention activities. Cervical cancer screening was at 83 percent compared with the benchmark of 93 percent; and colorectal cancer screening was at 58.6 percent lower than the target of 70.5 percent.

According to the report, screening rates in the Asian population were "significantly lower" at 64.1 percent for breast cancer, 75.4 percent for cervical cancer and 46.9 percent for colorectal cancer. Hispanics were less likely to be screened for cervical cancer (78.7 percent) and colorectal cancer (46.5 percent) compared with non-Hispanics at 83.8 percent and 59.9 percent, respectively.

In a statement accompanying the study, lead author Sallyann Coleman King, MD, said, "It is troubling to see that not all Americans are getting the recommended cancer screenings and that disparities continue to persist for certain populations. Screening can find breast, cervical, and colorectal cancers at an early stage when treatment is more effective." King, who is also an epidemic intelligence service officer in the CDC's division of cancer prevention, added, "We must continue to monitor cancer screening rates to improve the health of all Americans."

While financial costs may be a barrier to some in obtaining screening, the report also notes programs that can help. The CDC's National Breast and Cervical Cancer Early Detection Program provides access to free or low-cost screening and diagnostic services to underserved women across the country. The CDC's Colorectal Cancer Control Program offers access to screening to underserved men and women in 25 states. In addition, reducing financial barriers to preventive care is an aim of the Affordable Care Act. Under the act, breast, cervical and colorectal cancer screening is covered free under Medicare and new health insurance plans.

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