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BY LINDSAY RAY | APRIL 27, 2012
For decades, there hasn't been a new drug approved for sarcoma, but on April 26, the Food and Drug Administration approved Votrient (pazopanib) for patients with advanced soft tissue sarcoma who have received at least one prior chemotherapy treatment. However, the drug is not for patients who have adipocytic soft tissue sarcoma and gastrointestinal stromal tumors (GIST). Votrient was previously approved in 2009 for advanced renal cell carcinoma.
There are two main types of sarcomas--those that develop in the bones and those that develop in the soft tissues of the body, such as fats, muscles, nerves and more. Soft tissue sarcomas can occur anywhere in the body, but most develop in the arms or legs. While there are around 50 different subtypes, only about 11,000 cases are diagnosed each year, making this a rare tumor-type. Votrient is a tyrosine kinase inhibitor that works by inhibiting proteins that either help with the growth of new blood vessels that feeds these tumors or tell the tumor cells to grow and divide. It's a tablet taken once a day, without food, so either an hour before or two hours after a meal.
The drug was approved based on a phase 3 randomized, multicenter trial in which 369 patients were assigned to either receive Votrient or a placebo. Those in the Votrient arm had a median of 4.6 months progression free survival compared with the 1.6 months for those on placebo. Trending to improvement over the interim results announced at last year's meeting of the American Society of Clinical Oncology, median overall survival was still insignificant: 12.6 months for those on Votrient and 10.7 months for patients on placebo.
Common side effects included diarrhea, nausea, vomiting, decreased appetite and weight loss, hypertension, hair and skin changes, tumor pain, musculoskeletal pain, headache, shortness of breath, taste changes and fatigue, among others. Votrient also carries a boxed warning for the potential risk of liver damage, which can sometimes be fatal, so patients should be monitored for liver function. Other serious side effects include hemorrhage, gastrointestinal perforation, perforated lung and thrombotic events, such as a heart attack.
Other drugs in development for sarcomas include ridaforolimus, which was rejected by the same advisory panel that recommended Votrient for approval, ombrabulin, brivanib and Yondelis.
For more information, visit gsk.com call 888-825-5249.
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BY GUEST BLOGGER | APRIL 12, 2012
CURE invited Dennis M. Abbott, DDS, founder and CEO of Dental Oncology Professionals of North Texas, to explain the risks and management of dry mouth during cancer treatment.
"Sometimes my mouth gets so dry that I wake up with my tongue stuck to the roof of my mouth. It's been so bad that I've had to get a drink of water to get it unstuck!" - B.D., Mesquite, TX
Dry mouth. Xerostomia. Hyposalivation. Cotton mouth. Call it what you will...but very few people really understand what a severely dry mouth is all about better than someone battling cancer.
Dry mouth is a common unwanted companion for many oncology treatments. For patients undergoing chemotherapy, xerostomia is a pharmacological side effect of the cytotoxic drugs used to combat the cancer. In head and neck radiation therapy, hyposalivation is a direct effect of ionizing radiation administration on the salivary glands. At best, dry mouth is annoying; but in severe cases, the potential effects of xerostomia on teeth and soft tissues of the mouth can be devastating for years.
The story begins with spit, or saliva. Under normal conditions, the average human produces about one liter of saliva per day. Saliva functions as a protector of the oral cavity. It keeps the tissue moist. It neutralizes the acidic by-products of intraoral bacteria. It begins the digestion process, by moistening what we eat and breaking down starchy foods. It lubricates the moving parts of the mouth allowing us to smile and speak. In short, saliva is a big deal...and it is greatly missed when it's gone!
A loss of saliva can lead to a host of problems: difficulty chewing or swallowing; changes in taste; nutritional compromise; intolerance to oral medications, such as pills and capsules; increased susceptibility to dental decay; higher risk for oral infections; increased likelihood of injury to oral tissues; and an inability to wear dentures or partials.
Often, patients find the consequences of dry mouth annoying; while sometimes, they can be devastating. Some may even become emotionally depressed after not being able to carry on with what had previously been daily routine activities such as eating and tasting food.
From a dental health perspective, severe dry mouth can be very damaging to the teeth and increase the risk of intraoral infections. Teeth in a dry mouth are especially susceptible to decay at the gum line. A cavity at this location can be especially problematic since decay does not have to travel far to infect the center of the tooth, leading to a dental abscess. Likewise, a patient with diminished saliva has an increased risk for intraoral bacterial, viral or fungal infections that can become a systemic health problem if the patient has mouth sores, as in mucositis.
The solution comes by first identifying the problem. Like many areas in medicine, there are several ways to manage dry mouth. A dental oncologist, a dentist that specializes in oral medicine as it relates to cancer care, can help decide which is right. Treatment can range from systemic medication to mouth rinses or topically applied intraoral gels. A neutral rinse can be made by combining 1/4 teaspoon salt, 1/4 teaspoon baking soda and 1 quart water. This simple mouth rinse can be used to moisturize the mouth by following the directions to swish and spit. Again, a dental oncologist can determine which method of management is best for you.
Fluoride is an essential element for management of dry mouth. Carrier trays for localized delivery of fluoride make it possible to get the tooth-strengthening gel right where it needs to be. Patients with dry mouth must commit to meticulous oral hygiene including brushing and flossing two to three times a day, regular use of prescription-strength fluoride, and professional dental cleanings at least once every three months. When dental restorations are required, the dentist can even choose a fluoridecontaining filling material.
Food choices often change when dry mouth is a factor. Frequent consumption of highly acidic foods should be avoided as this can be harmful to tooth enamel and increase the risk of decay. Foods that are high in sugar and sticky foods must also be controlled. When these foods are enjoyed, a proper dental hygiene regimen should immediately follow to minimize the time these damaging foods have contact with the teeth.
Understanding the risk and seeing dry mouth as more than just an inconvenience is a big part of the battle. Knowing there are healthcare professionals who understand the struggle and can help manage not only the xerostomia but also the treat any infection or pain that might arise should encourage patients facing dry mouth to ask questions and seek help. So, when spit doesn't happen...call your dentist or dental oncologist.
Dennis M. Abbott, DDS, is the founder and CEO of Dental Oncology Professionals of North Texas, an oral medicine practice dedicated to meeting the unique dental and oral health needs of patients battling cancer. In addition to private practice, he is a member of the dental oncology medical staff at Baylor Charles A. Sammons Cancer Center and Baylor University Medical Center in Dallas. Dr. Abbott has conducted studies focusing on bisphosphonate-related osteonecrosis of the jaw and xerostomia in patients with cancer.
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BY KATHY LATOUR | APRIL 4, 2012
On April 12, we are going to have our first ever Facebook chat with Dennis Abbott, DDS, the founder and CEO of Dental Oncology Professionals of North Texas, an oral medicine practice dedicated to meeting the unique dental and oral health needs of patients battling cancer.
I met Dr. Abbott when a former student of mine looked me up to tell me he was doing public relations for a dentist who specialized in oncology. I was intrigued, and it was great to hear from my former student, Norberto, who graduated in the early '90s and had worked tirelessly since then for nonprofit organizations as he worked to make the world a better place. After doing some research with Dr. Abbott and others in the field, we decided to do a story on oncology dentistry and had a big response from our readers. We had a number of readers say something along the lines of "It's about time you talked about dental issues."
My interest came from the fact that I suffered a lot from oral mucositis during chemotherapy.
Oral mucositis is that nasty side effect that causes your mouth to fill with little blisters. My mom used to call them canker sores, and it felt like I had a mouth full of them. The only thing that had any impact on it at all was liquid novacaine, which I kept in my mouth as much as I could. When I tried to eat it was impossible, and talking was out of the question, which for me was really hard. I couldn't sleep because I had to spit out the novacaine, and I would wake myself up and not be able to go back to sleep.
When talking to Dr. Abbott about mucositis before the story, he told me about how he treats it now and just the compassion in his voice made me feel that today's cancer patients were getting lots of care that we didn't get in the '80s when they didn't know what to do other than try to kill the pain.
Some patients face losing their teeth and others have bad teeth going into treatment, which can impact their health directly.
To participate inCURE 's Facebook chat and to hear what Dr. Abbott has to say join us on April 12 at 12 p.m. CT (1 p.m. ET/ 10 a.m. PT). You can RSVP for the event here.
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BY KATHERINE LAGOMARSINO | FEBRUARY 22, 2012
Some 31,000 units of preservative-free methotrexate, equivalent to a month's supply of the drug for the entire nation, are currently on their way to hospitals around the country, said Michael Ball, CEO of the drug manufacturer Hospira, in a Food and Drug Administration (FDA) press conference on Tuesday.
"Next week," said Ball, "we'll release 34,000 vials, another month's worth. By mid-March, we'll release another 55,000 vials."
Hospira is one among a handful of drug manufacturers picking up the slack for the nation-wide shortage of this life-preserving drug used for the treatment of acute lymphoblastic leukemia (ALL), the most common type of childhood cancer that is 90 percent curable with regular injections of methotrexate.
Last Friday, APP, another drug manufacturer, received FDA approval to produce preservative-free methotrexate and will begin supplying clinicians in the next four to six weeks. Two other manufacturers, Mylan Pharmaceuticals and Sandoz, have also agreed to ramp up production.
The FDA also announced in yesterday's briefing that they have found a substitute for Doxil (doxorubicin) a drug used to treat various forms of ovarian and lung cancers. Lipodux, produced by manufacturer Sun Pharma Global FZE, will temporarily replace Doxil, which has been in short supply since July.
While this news certainly comes as a relief to parents, healthcare providers, and of course, the patients whose very survival depends on methotrexate or Doxil, many say this situation is sure to happen again. "The system is fragile," said Peter C. Adamson, MD, the current chair of the Children's Oncology Group, who sat on the panel at yesterday's briefing. "For many of these drugs, there may be a sole provider. There are potential future crises that are waiting to happen."
In October, President Obama issued an executive order instructing drug manufacturers to report potential shortages to the FDA in a timely fashion so the agency has time to find other drug alternatives. In addition, the FDA said it was working on developing even tougher legislation that would create permanent solutions for these recurring drug shortages, although the agency admits these shortages stem from complex issues that include the problem of demand outpacing supply, particularly when it comes to generic drugs that have lower profit margins than brand name drugs, as well as other economic and legal issues.
Adamson concluded the nearly hour-long briefing with a comment that perhaps resonates most with the general public. "I understand that passing legislation is a complex and difficult process, but no more difficult than curing a child with cancer."
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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.
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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.
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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.
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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.
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BY GUEST BLOGGER | JANUARY 6, 2012
CURE invited Diane Gambill, PhD, a CURE advisory board member, to share her thoughts on advancements in myeloma highlighted at the 2011 annual meeting of the American Society of Hematology.
I have blogged about the 'next generation' proteasome inhibitor carfilzomib and will likely have a chance to do so again as this interesting class of drugs expands. The proteasome inhibitors are a favorite of mine because they target a very interesting complex of proteins inside cells that was discovered in the 1990s. The proteasome helps the cell get rid of proteins that are made incorrectly or that get damaged once they are already made. Since myeloma cells grow rapidly and are actively making lots of proteins, they accumulate damaged proteins more quickly than normal cells. If cells cannot get rid of damaged proteins, they will die.
This explains, at least in part, why the proteasome inhibitor currently available in the clinic, Velcade (bortezomib), is effective in treating myeloma. Velcade and the immunomodulator Revlimid (lenalidomide) have become standard therapies for myeloma; unfortunately, not all myeloma cells are killed by standard treatments. Some myeloma cells are naturally resistant to treatment (referred to as treatment refractory) while others become resistant during treatment. Several studies are under way or completed that suggest carfilzomib might be active in myeloma that is refractory to treatment or becomes resistant during the course of treatment.
Two phase 2 trials have been conducted to study the efficacy of single-agent carfilzomib in treatment refractory or resistant myeloma. The PS0-171-003-A1 trial was reported at ASCO in 2011 [Proteasome inhibitors in myeloma: The next generation] and showed that 24% of patients who had previously received Velcade or Revlimid responded to carfilzomib. These responses were long lasting and there was a relatively low incidence of adverse events, including peripheral neuropathy (1% grade 3/4). Grade 3/4 peripheral neuropathy, which is considered moderate to severe neuropathy, occurs in about 8% of patients treated with Velcade for relapsed/refractory myeloma.
The second phase 2 trial with final results reported at ASH 2011, PX-171-004, showed similar results. This trial examined response to carfilzomib in patients who had relapsed or refractory disease but had not previously received Velcade. The response rates in this trial ranged from 42% to 52% depending on the dose of carfilzomib (20 mg/m2 or 27 mg/m2) with the higher response rate achieved with the higher dose. Responses were durable with a median time to disease progression of 8.3 months and 13.1 months at the low and high dose, respectively. This is an important result because the majority of patients had previously received an average of two prior therapies including Revlimid and/or stem cell transplant. The safety profile was similar to that seen in the PX-171-003-A1 trial.
One question frequently asked about carfilzomib is whether the decreased rate of adverse events is 'real.' In other words, do these statistics have any meaning for individual patients? The answer appears to be yes. A pooled analysis of overall safety from these phase 2 studies was also reported at ASH 2011. The pooled analysis showed that only 10% of the 526 patients on these studies required a dose reduction because of side effects. Eighteen percent of patients were able to stay on the treatment for at least 12 cycles. With regard to peripheral neuropathy, only five patients (1%) required dose reduction or discontinued therapy.
The ability of patients to tolerate their therapy for multiple cycles is important in achieving a response. Preliminary results of a continuation study suggest there are no cumulative long-term effects of single-agent carfilzomib.
Based on the results of PX-171-003-A1, the FDA has granted a standard review for consideration of approval of carfilzomib. This means a decision on approval will likely occur in the fall of 2012. A word about phase 2 trials, though: We have seen many instances where phase 2 results are not substantiated in randomized phase 3 trials. Even if approved on the basis of the phase 2 trial, the FDA will likely require a phase 3 study to confirm the phase 2 results. Phase 3 trials are under way in Europe and the U.S. The European trial, FOCUS, is evaluating carfilzomib versus best supportive care in patients who have had three or more prior therapies. The U.S. trial, ASPIRE, is assessing combination therapy with Revlimid/dexamethasone with and without carfilzomib in patients who have received one to three prior therapies.
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BY ELIZABETH WHITTINGTON | JANUARY 2, 2012
There were several big studies that were presented at the 2011 annual meeting of the American Society of Hematology, including many early phase studies in lymphoma, leukemia and myeloma.
One of the surprising studies that came out included a phase 3 study of a drug that was withdrawn from the market last year called Mylotarg. It appears that it may have a second life after researchers tweaked the dose for older patients with acute myeloid leukemia.
Mylotarg, also known as gemtuzumab ozogamicin, was approved back in 2000 for relapsed AML, but was withdrawn when follow-up studies showed the drug did not improve response rates and also increased the risk of a rare, but life-threatening liver complication called VOD (veno-occlusive liver disease).
A French group tested the drug in a phase 3 trial using a modified dosing regimen that was easier on patients, which reduced side effects and significantly improved survival. Newly diagnosed patients aged 50 to 70 years who received standard chemotherapy with 3 mg/m2 three times a week lived longer than patients on chemotherapy alone (19.2 months versus 34 months).
"With the lower dose, we have less toxicity ... and we have more efficacy," says Sylvie Castaigne, MD, lead investigator of the ALFA study. She said this recent study included fewer deaths from VOD than in the past studies, but that patients also experienced low platelet counts, however that side effect was manageable.
Several ongoing trials with Mylotarg may show the drug still has a place in AML treatment. The drug's maker, Pfizer, is waiting for additional study results before determining whether to resubmit the drug to the FDA for approval.
"At this point, we are trying to better understand the data out there in hopes that they indeed look as good as we would like them to look," says Mark Shapiro, MD, PhD, a senior director of global medical affairs with the drug company. "Pfizer is definitely interested, the issue is what can we do with this data."
Because the drug has been withdrawn, it is typically not available to most patients. However, it is still being studied in clinical trials. Additionally, there are other studies looking at a second-generation drug called inotuzumab ozogamicin.
You can read more from ASH from Dr. Anas Younes (Studies highlight progress in lymphoma, pose more questions) and other stories on lymphoma, myeloma and leukemia in our News section.
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