Love and Gratitude to my marathon runners and Man-of-the-Year

I need to take the time to send a ton of love and gratitude to three young men in my life: my son Rocky, and his childhood friends Tyler Breskin and Adrian Gastelum. They did a ton of fundraising for leukemia and lymphoma research the past few months. 

Rocky and Tyler ran the San Diego Rock’n’Roll Marathon on Team La Verne, and were third place and eleventh place respectively in the United States in fundraising. Rocky and Tyler dedicated their efforts in honor of me.

Adrian just got second place last night for Phoenix Man-of-the Year raising funding for research. Adrian dedicated his fundraising in honor of his brother, who died of leukemia, and me. Wonderful, wonderful men.

Tyler and Rocky at the finish line.

Adrian

MY LIFE since diagnosis

This is a concept map of my life since I was diagnosed with high-risk leukemia a few years ago. Each cancer patient faces emotional, physical, and financial tolls, as well as trying to cognitively put their hands around what is happening to their body and what can be done.  In my case, there were no real successful options, until I entered a clinical study.

I now have a cognitive understanding of the cancer. I am dealing with the physical and emotional toll. I believe the financial toll is manageable for now. After 2 1/2 years of weekly depreciating insurance interviews and filling out questionnaires and providing evidence of disease, I thought I could get on with my life. But now I have been randomly selected (LUCKY ME) to participate in another detailed insurance questionnaire about my medical condition. Here we go again...

And now I find out that the drug that is working for me will probably cost $150,000 per year... I am so happy I have a demented sense of humor!

                                                                   -- Dr. La Verne

Dr. Sharman's Post: How expensive are new drugs?

I just got through reading Dr. Sharman's blog post and wanted to share it with you. It is a continuation of my conversation about the out-of-control costs of cancer drugs. I will let his words speak to you...

http://www.cll-nhl.com/2013/05/how-expensive-are-new-drugs.html?utm_source=feedburner&utm_medium=email&utm_campaign=Feed%3A+DrSharmansCllNhlBlog+%28Dr.+Sharman%26%2339%3Bs+CLL+%26amp%3B+NHL+Blog%29

Paying to Survive: The Cost of Leukemia Drugs

My husband Carl and I have worked hard all of our lives. We both went to college, earned a living, raised a family, contributed to society, and hoped to leave the world a better place because we were here. Cancer reared its ugly head when we least expected it, and it took a financial and emotional toll on us. Becoming a proactive patient has helped me to personally deal with my diagnosis of 17p deleted leukemia.

As you all know, I have just completed nine months in a clinical trial with an experimental drug Ibrutinib, which will not cure the leukemia, but seems to be keeping it at bay, until a cure can be found. Hurrah! It looks like the FDA will approve of the drug in a year or so. This will be my lifeline and has changed my chances for survival. Hurrah! Now the next obstacle in our lives… How much am I going to have to pay to survive?

Here is a quote from the Blood Journal Report, which was prepublished April 25, 2013:

“Of the 12 drugs approved by the FDA for various cancer indications in 2012, 11 were priced above $100,000 per year. Cancer drug prices have almost doubled from a decade ago, from an average of $5,000 per month to more than $10,000 per month.”

If the cost of Ibrutinib follows in suit with today’s cost of Imatinib, more commonly known as Gleevac, which is a drug for chronic myeloid leukemia, the drug company will probably price it about $100,000 per year. Hmmm… My husband is retired. I am not working now. I don’t believe we have an extra $100,000 per year laying around to pay for my survival. I wonder if my insurance will help me pay for this? We are not alone in this situation. I guess we will cross that road when we get there.

All I can say is “Thank God for compassionate and sensible physicians.” Dr. Hagop Kantarjian from M.D. Anderson in Houston is taking the lead with a paper in the Blood Journal Report:

“The Price of Drugs for Chronic Myeloid Leukemia (CML); A Reflection of the Unsustainable Prices of Cancer Drugs: From the Perspective of a Large Group of CML Experts.”

Dr. Kantarjian states:

“As a group of more than 100 experts in chronic myeloid leukemia (CML), we draw attention to the high prices of cancer drugs, with the particular focus on the prices of approved tyrosine kinase inhibitors for the treatment of CML. This editorial addresses the multiple factors involved in cancer drug pricing, their impact on individual patients and healthcare policies, and argues for the need to lower the prices of cancer drugs to allow more patients to afford them and to maintain sound long-term healthcare policies.”

Blood Journal Report full article:

http://bloodjournal.hematologylibrary.org/content/early/2013/04/23/blood-2013-03-490003.full.pdf

The New York Times article, published April 25, 2013, states that physicians in more than 15 countries on more than five continents have joined together to suggest “… that charging high prices for a medicine needed to keep someone alive is profiteering, akin to jacking up the prices of essential goods after a natural disaster.”

New York Times full article

http://www.nytimes.com/2013/04/26/business/cancer-physicians-attack-high-drug-costs.html?_r=1

Stay tuned for the next adventure of “Dr. La Verne’s Awesome Adventure: Slaying the Leukemia Dragon.”

Dedicated to Dr. Mohammed Farooqui

This painting is on display at Banner Hospital for the next few months. When it is returned to me, I will give it to Dr. Farooqui as a gift of appreciation.

Novel Antibody Directed at Chronic Lymphocytic Leukemia

By Anna Azvolinsky, PhD¹ | April 3, 2013

¹Freelance Science Writer and Cancer Network Contributor.

Researchers have identified a novel monoclonal antibody directly targeted against a receptor found in abundance on chronic lymphocytic leukemia (CLL) cells, but not normal B cells. The humanized antibody can directly kill CLL cells, according to Thomas Kipps, MD, PhD, professor of medicine and deputy director for research at the University of California, San Diego Moores Cancer Center, and colleagues. The results of the study are published in the online edition of Proceedings of the National Academy of Sciences.

In contrast to normal B cells, CLL cells express a high level of CD44, a cell-surface glycoprotein receptor. CD44 is thought to mediate one of the important survival signals for leukemia cells. CLL cells receive survival signals from its tumor environment, including cells that are present in the lymph nodes and the bone marrow of CLL patients. Previous work from Kipps and colleagues has shown that CLL cells can undergo drug-induced or spontaneous cell death when removed from a patient and cultured in the laboratory. Because the RG7356 antibody induces cell death of the CLL cells by binding to CD44, the drug is a potential new therapy for treatment of at least a subset of CLL patients.

Ibrutinib on a Fast Tract for FDA Approval for 17p Deleted Patients

EXTRA! EXTRA!

April 8, 2013 it was announced that the FDA granted breakthrough therapy designation for ibrutinib (Janssen and Pharmacyclics) as a monotherapy for patients with CLL/SLL (chronic lymphocytic leukemia or small lymphocytic lymphoma) with deletion of the short arm of chromosome 17. This genetic mutation is associated with poor prognosis and is one of the worst prognostic factors in patients with CLL. This is what I have.

This is the third breakthrough designation the FDA granted for ibrutinib an investigational Bruton’s tyrosine kinase inhibitor. One was as a monotherapy for previously treated patients with relapsed or refractory mantle cell lymphoma, and the other was as a monotherapy for patients with Waldenström’s macroglobulinemia.

The breakthrough therapy designation was created in 2012. It enables the development and review of drugs shown in preclinical studies to offer potentially substantial improvements over existing therapies for patients with serious or life-threatening diseases to be expedited.

Patients like me with 17p deletion generally do not respond well to chemoimmunotherapy, and are limited on their treatment options. In my case, because I am bi-racial, I have less than a 1% chance of finding a bone marrow donor match. Ibrutinib has the potential to improve the outcome in this serious and life-threatening disease, and is much less risky than a bone marrow transplant.

A Phase II global study of ibrutinib in patients with CLL deletion 17p, will be available this year and Pharmacyclics plans to enroll 111 patients worldwide. The study called “RESONATE™ -17” is a single-arm, open-label, multi-center trial using ibrutinib as a monotherapy in previously-treated patients who have deletion 17p, who did not respond or who relapsed (a high unmet need population). The purpose of the study will be to determine overall response rate.

Those of you following my blog know that I have just completed my 9-month milestone as “a little white laboratory mouse” in the clinical trial with ibrutinib. Dr. Adrian Wiestner, M.D., PhD, of the National Institutes of Health is the lead investigator in the clinical study. Dr. Wiestner just presented the findings from this clinical trial at the American Association for Cancer Research (AACR) in Washington, D.C. Here is an excerpt from Pharmacyclics’ press release from April 8, 2013. Please note that this is a fast track to FDA approval, and applies to 17p deleted CLL/SLL patients like me. :-)

Phase II Ibrutinib Monotherapy Study Shows CLL Patients Achieved Rapid and Sustainable Disease Control Irrespective of Age or High Risk Prognostic Factor

SUNNYVALE, Calif., April 8, 2013 /PRNewswire/ -- Pharmacyclics, Inc. (Nasdaq: PCYC) today announced results from a Phase II trial of the investigational oral agent ibrutinib which demonstrated rapid and sustained disease control as a monotherapy in untreated, relapsed and refractory chronic lymphocytic leukemia (CLL) patients, irrespective of characteristics that predict poor outcomes to chemoimmunotherapy.

This study was discussed at today's American Association for Cancer Research (AACR) annual meeting in Washington, DC together in addition to 8 other presentations covering advances in clinical and pre-clinical research with ibrutinib. The Phase II study, which was sponsored by the National Heart, Lung and Blood Institute, included an analysis of two CLL patient cohorts: the elderly, above 65 years of age, (n=24, of which 8 were treatment naive) and the high risk genetic group with a deletion of chromosome 17p (del 17p) (n=29, of which 15 were treatment naive). Many elderly patients with CLL are unable to tolerate aggressive therapies. Patients with deletion of chromosome 17p typically are poor responders to chemoimmunotherapy and have limited treatment options with no standard of care defined. Of all CLL patients enrolled in this trial, 72% had been characterized as Rai stage 3-4, indicating an advance stage, high-risk patient population.

The results of the study were presented by lead investigator Adrian Wiestner, M.D., PhD, Hematology Branch, NHLBI, National Institutes of Health. "Ibrutinib was highly efficacious as a single agent in patients with untreated, relapsed and unresponsive CLL, irrespective of their del 17p status," Dr. Wiestner said. "Responses in this study appear to be durable, and results indicate the drug is effective against the disease in lymph nodes, spleen and bone marrow. This is important because existing therapies often fail to effectively eliminate cancer cells in these tissue sites. Targeted therapy for CLL is becoming a reality, and this new approach may greatly improve the lives of patients with this disease."

The study also evaluated in vivo effects of ibrutinib using blood and tissue samples collected before and during treatment. Ibrutinib demonstrated rapid and sustained disease control in blood, lymph nodes, spleen and bone marrow. After 6 months, 95 percent of patients experienced a reduction in lymph node size and all showed reduction in spleen enlargement, with a median reduction of 55 percent. In 26 patients, for whom a bone marrow biopsy was done, tumor infiltration decreased by 82 percent.

The Progression Free Survival probability for these patients at 12 months was estimated to be 94 percent. Most adverse events were mild and manageable and included diarrhea, fatigue and rash, severe events occurred in less than 13 percent of patients. 

"We are very pleased with the continued research that is being demonstrated at AACR across 9 different presentations. These advances show the potential breadth of the ibrutinib program and provide further opportunities for us to pursue. We are grateful for the continued support of our collaborators, investigators, patients and shareholders," said Bob Duggan, CEO and chairman of the board.

Ibrutinib has been designated as a FDA Breakthrough Therapy in CLL patients with deletion 17p, based on data from completed Phase I/II clinical studies, where ibrutinib as a monotherapy was used to treat patients with this disease. Ibrutinib has the potential to improve the outcome in this serious and life-threatening disease, and may provide a substantial improvement over existing therapies for this indication.