BY GUEST BLOGGER | DECEMBER 28, 2013
It's not happiness that brings you gratitude, it's gratitude that brings you happiness. Today I am a very happy girl. As I sat in the Louis Armstrong Airport waiting for my delayed flight home to Philadelphia from the American Society of Hematology's Annual Meeting (#ASH13 on social media) I am reflecting back on my experience. I have a lot for which I am grateful.
I am grateful for the educational grant that enabled the International Myeloma Foundation (myeloma.org) to bring 12 patient advocates from across the United States to the American Society of Hematology's Annual Meeting (hematology.org).
I am grateful that IMF asked me to be one of those patient advocates this year and report from #ASH13.
I am grateful for the progress that has been made in treating myeloma in the last decade; because of these advances I am well enough to travel alone halfway across the country five years post-diagnosis.
I am grateful for the many discussions I had with my fellow advocates during our time in New Orleans. These conversations both formal and informal enlightened me on treatment options, clinical trials, side effect management, advocacy and the best place to get a muffuletta.
I am grateful for all the researchers that are bringing new treatment protocols and drugs to clinical trial. The passion I felt during the presentations of medical research abstracts and at the satellite events held throughout the conference was contagious.
I am grateful to everyone who has helped fund research, from the individual patient who held a grassroots bake sale to all who lobbied Washington to restore government funds for medical research.
I am grateful to my fellow myeloma warriors who have participated in clinical trials; without them we wouldn't have made any progress in our war against myeloma or have research abstracts to present.
I am grateful for the education the IMF has provided me, from the new patient resource packet I received in the mail days after my diagnosis to the ongoing educational webcasts. The IMF hosted 3 live webcasts from #ASH13 and there are several recorded #ASH13 interviews posted on their website.
I am grateful for social media that has allowed me both to learn from others and share what I learned through Twitter - @MyelomaTeacher #ASH13IMF (I was in the top 5 influencers at #ASH13), IMF blogs, and a Patient Power interview.
I am grateful for my family and friends who continue to support me in my efforts to be a patient advocate.
This list could go on and on.
I plan on using what I learned to help others at my local in-person support group, in the several online communities of which I am a member, in my mentoring of newly diagnosed patients, by participating in myeloma related webcasts, and through continued engagement in social media.
Remember to use the following IMF link to get all the myeloma highlights from #ASH13.
Goodbye New Orleans, but thanks for the memories!
Cynthia Chmielewski, a retired educator, is a patient advocate and mentor, a patient services volunteer, and a "life-long learner" who lives in New Jersey. She is an advocate for IMF and a five-year survivor of myeloma. This blog was originally published at myeloma.org. You can find her on twitter at @MyelomaTeacher.RELATED POSTS
BY GUEST BLOGGER | MARCH 27, 2013
The first thing he asked should have tipped me off.
"Did you come alone?"
It was a sunny morning in July almost five years ago. The next four words my hematologist said will forever be embedded in my memory: You have multiple myeloma.
Like so many before me, the moment of my diagnosis was the first time I'd ever heard the word. As a teacher, I did what I often encouraged my students to do when they didn't recognize a word – I asked him to spell it out for me. When I told my family, they immediately thought I had been diagnosed with skin cancer.
But myeloma is not melanoma. Myeloma (also multiple myeloma) is a blood cancer. At the time, the only blood cancers I had been aware of were leukemia and lymphoma. The only blood cancer organization I'd ever heard of was the Leukemia and Lymphoma Society. Using deductive reasoning, I figured that since the word "myeloma" wasn't in the organization's name, then myeloma couldn't be a cancer. Some people call it wishful thinking. Others might call it denial.
Prior to my diagnosis, I'd been suffering from excruciating pain. I'd been having trouble with fatigue, and often felt myself forgetting things – like whether or not I recorded my students' grades before handing back their assignments.
My orthopedic doctor treated me with pain medication and physical therapy for two years before discovering that compression fractures in my spine were the source of my pain. I was scheduled for surgery to have the fractures repaired, but the surgeon required medical clearance from my primary care physician, who in turn referred me to a hematologist when my blood work revealed I had severe anemia.
That's how I ended up sitting across from a hematologist on a hot summer day, learning about my cancer.
Knowing what I know now about myeloma, it's frightening to remember the roundabout road I traveled to get my diagnosis. See, anemia and bone pain are two of the four common symptoms of myeloma. The other two are renal insufficiency and high calcium levels in the blood (which can cause confusion). If I had been aware of these symptoms and their connection to myeloma, I might have questioned why two years of physical therapy and pain medication were not working.
This is why I believe it is so important to promote myeloma awareness. I'm grateful that the International Myeloma Foundation has designated March as Myeloma Awareness Month and initiated their "Tell One Person" campaign to help spread life-saving information about a disease that, despite its being the second most common blood cancer, often goes untreated for years because of a lack of awareness.
This year, an estimated 22,350 adults in the United States will learn about myeloma when they receive their diagnosis. To help them know they are not alone, I have been doing my part by using social media to reach out to the myeloma community. I am @MyelomaTeacher on Twitter, where I post facts about myeloma symptoms, treatment and other information that benefits myeloma patients and their caregivers.
I keep going back to the realization that if I had known about the CRAB criteria (C-calcium, R-renal insufficiency, A-anemia, and B-bone involvement) I would have saved myself two years of debilitating pain and confusion.
If I can help one person avoid making the same mistake I did, and recognize their symptoms early on, it will make all the difference in the world.
Cynthia Chmielewski, a retired educator, is a patient advocate and mentor, a patient services volunteer, and a "life-long learner" who lives in New Jersey.RELATED POSTS
BY GUEST BLOGGER | MARCH 19, 2013
Michael and I lived a traditional American lifestyle: he worked full time in the manufacturing industry while I stayed at home to raise our two young children. Our youngest was just two years old when Michael began experiencing serious back pain. We never imagined that the symptoms would mark the beginning of Michael's lifelong battle against cancer.
In 2000, at the age of 36, Michael was diagnosed with multiple myeloma, a cancer of cells in the bone marrow that affects production of red cells, white cells and stem cells, and can damage bone. Myeloma is also a cancer--as he was told by doctors--for which there was no cause or cure, and which had on average a five-year survival rate.
A five-year survival rate?
When we heard that, our first thoughts were for our children. Mikey was barely walking and Allison was only seven years old. We were devastated, but Michael was not willing to accept the idea that he wouldn't be there to watch them grow up.
Thirteen years later, Michel, the kids and I look back with gratitude that my stubborn husband refused to believe the doctors' prognosis. Allison is now a sophomore in college and just turned 20, and Mikey is already in high school. We are defying the odds 13 years later, and none of us takes a millisecond of life for granted.
One of the ways we show our gratitude is by taking every opportunity to share what we've learned to help others who are struggling with the disease. Educating patients and doctors about myeloma, a disease that is growing in numbers and affecting increasingly younger people, has become our mission--particularly during March, which is Myeloma Awareness Month.
Like most patients at the time of diagnosis, neither Michael nor I had ever heard of myeloma. Fortunately, the International Myeloma Foundation (IMF) was there to help us find expert doctors and learn about the latest treatments and clinical trials of new drugs. The IMF also inspired us to reach out to others in our community who were struggling with myeloma and establish the first myeloma support group in our home state of Connecticut. Today we enjoy hosting monthly meetings with our fellow myeloma patients and caregivers--learning, sharing and celebrating our personal milestones as a group.
There have been some very scary moments since myeloma entered our lives. After an operation left Michael paralyzed for two months, he needed to re-learn to stand, walk and perform routine things again as simple as putting on his socks and shoes each morning, and was forced to stop working. I cannot tell you how much I admire his strength. He never gave up, and when the time came, he was able to recover enough to help coach Mikey's baseball team.
Though it was Michael who had to suffer through the radiation treatment to shrink a large plasma cell tumor that had destroyed two-thirds of his sacrum, a successful stem cell transplant, and an operation that resulted in irreparable nerve damage in both his legs--we faced the successes and setbacks together as a family.
Then--as is frequently the case with myeloma patients--Michael's disease returned. This time, after much debate, his doctors at New York-Presbyterian Hospital/Weill Cornell Medical Center settled on a course of treatment (a combination of Revlimid and dexamethasone) that worked.
By 2006, Michael had attained complete remission. In the summer of 2007, our family embarked on an educational outreach project called the Myeloma Mobile.
With Mikey, Allison and the dog and cat in tow, our family traveled across the country, covering over 12,000 miles, and shared our knowledge and experiences with other patients searching for help and hope. It was an eye-opening experience, not to mention a wonderful time of togetherness. Have you ever parallel-parked a 35-foot RV? I have.
For the past several months, Michael and I have been actively involved with the IMF's Myeloma Awareness Month outreach programs. Our hometown of Prospect has officially declared March as Myeloma Awareness Month along with more than 40 cities and towns; six counties; three states and the entire country of Barbados that have issued similar proclamations. (Barbados? Maybe we'd better go investigate!)
The future for myeloma patients doesn't look nearly as bleak as it did when Michael was first diagnosed. In the past 10 years, the Food and Drug Administration has approved eight new treatment therapies for multiple myeloma. People are living longer and with better quality of life with the disease.
Michael is an example of that. Our family leads an active life and we are encouraged that his remission will last a long time. In fact, Michael is also a musician and he wrote a song for our children entitled "I'm Not Leaving."
I think that pretty much says it all, doesn't it?
Robin Tuohy lives in Connecticut and is Senior Director of Support Groups for the International Myeloma Foundation. Before that, she worked as a paralegal for over 10 years at a Fortune 500 company. She and her husband Michael have been married for 23 years and "face all that life brings us together--both the blessings and the challenges."RELATED POSTS
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.
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.RELATED POSTS
BY GUEST BLOGGER | DECEMBER 30, 2011
CURE asked Anas Younes, MD, professor in the department of lymphoma/myeloma, division of cancer medicine at M.D. Anderson Cancer Center in Houston, and a CURE advisory board member, for his thoughts on the lymphoma advancements announced at this year's American Society of Hematology.
I finally got to blog on what is news at this year's ASH meeting in San Diego. Almost 20,000 people attended the meeting, and more than 1,000 studies were presented on hematologic malignancies and benign hematologic disorders. But for lymphoma, I think the most important studies are the following three. That is not to say that other studies are not significant, but I think the following ones have a more relevant impact on the treatment of lymphoma. I will explain why.
A Phase III Randomized Intergroup Trial (SWOG S0016) of CHOP Chemotherapy Plus Rituximab Vs. CHOP Chemotherapy Plus Iodine-131-Tositumomab for the Treatment of Newly Diagnosed Follicular Non-Hodgkin's Lymphoma We all know that radioimmunotherapy (RIT) is active therapy for lymphoma. Two drugs (Iodine-131-Tositumomab (Bexxar) and ibritumomab tiuxetan (Zevalin) are already approved by the FDA for the treatment of relapsed follicular and indolent lymphoma. But in clinical practice, the use of RIT has been relatively modest compared to other newly approved drugs. Despite the documented safety of these agents, there is still a perception among oncologists and patients that these agents are not as safe as other drugs. Many of us wanted to see whether the use of RIT may improve patients' survival, which has not been shown in a randomized study. If so, such data would certainly re-energize the RIT field. This phase 3 randomized study was positioned to answer such a question.
Unfortunately, there was no difference between RCHOP and CHOP followed by Bexxar. I am sure many experts will try to dissect the data in many different ways, including whether this data is applicable to all RITs or just Bexxar. Should we do another randomized study with Zevalin? May be with rituximab maintenance? Should we include rituximab in both treatment arms as part of the induction? But at the end of the day, this trial as good as it is, will not help move RIT forward. With the current competitive environment that includes many new promising agents that have minimal side effects, I think the use of RIT, as we currently know it, will continue to decline.
Frontline Therapy with Brentuximab Vedotin Combined with ABVD or AVD in Patients with Newly Diagnosed Advanced Stage Hodgkin Lymphoma Brentuximab vedotin (or SGN-35) has produces 75 percent response rate in heavily pre-treated patients with relapsed Hodgkin lymphoma (HL). So, it makes sense to move it up front and test in newly diagnosed patients whose tumors are less resistant to therapy. Because HL is highly curable with chemotherapy, such as ABVD, it is unethical to test brentuximab alone in these patients. Instead, it makes more sense to add it to ABVD hoping the combined brentuximab + ABVD will be more effective. However, to prove the point, multiple steps needed to be taken. The first step was the core of this clinical trial: Is it safe to combine brentuximab with ABVD, and what is the optimal dose that we can use in such combination? Because ABVD is the standard regimen, it is kept intact while the doses of brentuximab are escalated to maximum dose.
During the conduct of this study, an increase in the incidence of lung complications were observed, which were similar to bleomicin toxicity: shortness of breath, dry cough and lung infiltrate. This toxicity was reversible in 9 of 10 patients with simple measures that included discontinuation of bleomycin and administration of steroids. But because bleomycin is the weakest drug in the ABVD regimen, and usually associated with unpredictable lung toxicity, it was eliminated to generate a brentuximab + AVD combination. At the time of the study presentation, brentuximab + AVD was not associated with any lung toxicity.
What was presented at ASH and was not in the printed abstract is the results of interim PET scan results after two cycles of therapy. With ABVD alone, we typically see 20 percent to 30 percent of the patients continue to have PET positive scans after 2 cycles, which usually correlate with bad prognosis. In contrast, after 2 cycles of brentuximab-based therapy (with ABVD or AVD), only 3 percent of the patients had positive PET. So these results look very encouraging, but of course we need more time and longer follow up to find out what this all means. Obviously, the ultimate test will be to compare the standard ABVD with the new regimen brentuximab + AVD in a randomized trial. This study is planned for the end of 2012. The outcome of such trial may indeed change the standard of care for patients with HL.
The Bruton's Tyrosine Kinase (BTK) Inhibitor PCI-32765 Induces Durable Responses in Relapsed or Refractory (R/R) Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma (CLL/SLL): Follow-up of a Phase Ib/II Study and The Bruton's Tyrosine Kinase Inhibitor PCI-32765 Is Highly Active As Single-Agent Therapy in Previously-Treated Mantle Cell Lymphoma (MCL): Preliminary Results of a Phase II Trial There is a lot of buzz about the promising clinical results with the oral small molecule inhibitor PCI-32765, which inhibits an enzyme called Bruton kinase. Last year, there was emerging data from a phase 1 study that also got a lot of attention. This year, the early results are now confirmed in a larger number of patients with CLL and MCL, both are practically incurable lymphoid malignancies.
In CLL, a 70 percent response rate was seen in 27 patients treated with 420 mg daily. This single-agent activity data is so impressive, so a randomized phase 3 study is being planned.
In MCL, results from an ongoing phase 2 study reported a response rate of 67 percent, which is also very impressive for a single agent in this disease. If this data continues to hold as more patients are enrolled, a phase 3 randomized study in relapsed MCL will be the obvious next step. There is a clear need for new agents for patients with MCL, and PCI-32765 may as well what we all have been waiting for!RELATED POSTS