Storming Capitol Hill: LLS Leads the Charge for Cures and Access

DECLARE IT! Cancer ends with me.

Blood cancers are the #3 leading causes of cancer death in the United States – right behind #1 cancers of the respiratory system (including lung cancer), and #2 cancers of the digestive system (including colorectal cancer), according to the American Cancer Society. Breast cancer is #4.

Since the Leukemia & Lymphoma Society (LLS) was formed, it has invested more than $1 billion in blood cancer research and is motivated to ensure patient access to these innovative therapies. What many people are not aware of is that blood cancer research is a gateway to cures for other cancers. Multi-drug chemotherapy and stem cell transplantation began as blood cancer therapies. Almost half of all cancer drugs newly approved by the US Food & Drug Administration (FDA) in the past dozen years were first approved for blood cancers.

LLS is a leader in a unique approach to research and fundraising. The non-profit forms partnerships with biotechnology and pharmaceutical companies, the National Institutes of Health, universities, and medical centers to discover innovative medical therapies and to move them to market.

I am proud to be an LLS patient advocate. Last week over 500 LLS patient advocates from all around the country stormed Capitol Hill lobbying for two health care bills (HR 460 the Patients’ Access to Treatments Act and S 1365/HR 2827 the Medicare Part D Beneficiary Appeals Fairness Act) to improve access to innovative therapies for patients. These bills address Medicare and private commercial insurance plans. The bill that was passed in Arizona addressed individual insurance plans and small group insurance. Both U.S. Congress bills when passed will make “specialty tier” drugs affordable.

We went through training workshops and formed teams to deliver a message to our legislators in the U.S. Congress. My team consisted of LLS staff, Board members, leukemia physician, patient (me). NOTE: I will be posting more about these wonderful team members in my next blog.

We met with the offices of two senators and five representatives:

(1) Rep. Kyrsten Sinema (Arizona District 9) (D)

(2) Sen. Jeff Flake (R)

(3) Rep. Ann Kirkpatrick (D)

(4) Office of Sen. John McCain (R)

(5) Office of Rep. Paul Gosar (R)

(6) Office of Raul Grijalva (D)

(7) Office of Rep. Trent Franks (R)

Another Arizona team met with several other representatives.

We did not even get pushback from the insurance companies when they realized that passing the bills would increase premium costs about $3 more per year. Not having access to these drugs would mean additional hospitalization costs, etc.

To say that these two bills are personal is an understatement. They directly affect my particular situation. If I do not have access after the clinical trial, I will not be able to afford the cost. The cancer will progress.

I will keep a very close watch on the voting of these bills.

State of Arizona passes the Fair Access to Cancer Treatment House Bill 2078

Arizona's Governor Brewer signed HB 2078 into law Wednesday, April 30, 2014, officially enacting the FACT Act (HB 2078) -- Fair Access to Cancer Treatment. 

Arizona Republic article:
In spite of the fact that there are some little factual snafus about my particular situation (i.e. my diagnosis), the overall message is good.
http://www.azcentral.com/story/news/politics/2014/04/18/bill-broaden-coverage-chemo-pills/7856915/

Bill Briefing:
http://www.accc-cancer.org/ossn_network/AZ/pdf/TACOS-advocacy-oralparity-FACTBriefing.pdf

Leukemia & Lymphoma Society Celgene speech

Dear friends and family:

I gave this speech in front of about 200 Celgene employees in Phoenix, Arizona. They manufacture cancer drugs. I thought I would share it with you.

Celgene Speech

Dr. La Verne Abe Harris

Phoenix Biotechnology Company

Thursday, April 24, 2014

Hello. My name is La Verne. To my university students I am Dr. Harris. To my colleagues I am Dr. La Verne. To my children's friends I am Mama La Verne. To my 13 grandchildren I am Grandma. And to my husband Carl I am affectionately known as Honey Bear.

I am your friend, your sister, your mother, your colleague.

I have cancer and I am living on borrowed time.

Believe me, I did not want this job.

When I was diagnosed with blood cancer I told my doctor that I did not have time for this and this was a major inconvenience in my life.

I found out I had a leukemia with a chromosome deletion of 17p, which means a poor prognosis. I was told by a leukemia expert that I had two to three years to live and that unfortunately I was chemo-resistant and had less than a 1% chance of finding a bone marrow donor, because I am half Japanese and half German. Apparently there are not too many of them in the world. In other words, “La Verne, I’m so sorry.”

For some reason I was not devastated. I was sad, but calm inside. I believed that I was not going to die yet. I knew I was in for the battle of my life with the Dragon. You see, I am not afraid to die. And even more, I am not afraid to live.

I tried to get a handle on the cancer in which I had been diagnosed at the age of 57 at the height of my career as a Computer Graphics Technology professor. I tried to get my hands around possible solutions, because that’s what I did – helped technologists and engineers learn how to be creative thinkers. I was committed to find possibilities – if not for me, then for someone else who follows me.

I left my career at Purdue University to connect with blood cancer experts and educate myself. I was on a mission to save my life. At the time I was horrified to find that not much had been done in cancer research with those with 17p deletions. I knew not to read any medical research older than five to 10 years or I would already see myself as dead. Finding a clinical trial was my only hope.

I read and talked to experts about upcoming future cancer treatments – immunotherapy and monoclonal antibodies, and oral treatments. I found a clinical trial with an experimental oral cancer targeted treatment. I felt good about this one. I did not qualify for the clinical trial because of my age. Researchers wanted someone 65 or older. As a researcher, I understand demographic limitations. But it was at that moment I so wanted a fake ID card that some of my college students had!

Time was ticking away. Thank God I was so resilient, because three years after my diagnosis I qualified for two clinical trials – one at MD Anderson in Houston and one at the National Institutes of Health in Bethesda, Maryland. I selected the one in NIH because the one drug I wanted to take was being tested instead of a combination of two. I did not want the extra variable.

I have been in a clinical trial for 21 months. I am not in remission yet, but I am better off than I was when I began and I am headed in the right direction.  This drug will not cure me, but it will hold the cancer at bay until a cure hopefully can be found. I do have some side effects, such as wavy hair, brittle nails, fatigue, and cramping. But I am here. I am alive and still a force to be reckoned with. LOL.

This year the FDA will be approving this experimental drug PCI-32765 (aka ibrutinib under the branding of Imbruvica). Good news, right? This means cancer patients have access to this experimental drug, because after all, cancer treatment is cancer treatment; right?

Not so.

The way the laws are written today in Arizona and in the federal government, a cancer patient can pay $30 copay for chemotherapy administered intravenously. If a cancer patient gets cancer treatment administered orally or injected, the laws do not apply. In my particular case once the FDA approves of the drug, it will cost me $8,000 per month. I will not be able to afford it.

The drug is my lifeline. Without it the cancer progresses.

You see oral cancer treatment was not invented years ago when the laws were written. Unless the laws are revised, the insurance companies are not legally bound to cover the cancer treatment.

The Fair Access to Cancer Treatment (FACT) is just that. It legally makes insurance companies be fair to all cancer patients. What does it matter that cancer treatment is given orally, injected, or intravenously? Cancer treatment is cancer treatment.

I recently gave my testimony to the Arizona Legislature House of Representatives. I called out the lobbyist by name who spoke before me and told him that the bill needs to be passed “because my life is worth it!”

HB2078 is on the final leg of approval in Arizona, but the governor has to sign it into law. Right after this town meeting, log into the Leukemia & Lymphoma Society website LLS.org and tell the governor to sign the bill now! I will pass around the direct URL to patient advocacy.

http://advocacy.lls.org/p/dia/action3/common/public/?action_KEY=10794

The second week of May my son Rocky Harris and I will be flying to Washington DC to talk to the US Congress about Fair Access to Cancer Treatment so that the laws will also apply to those people 65 and older. The health of your company and the health of my body depend on it.

Let me end by telling you that I am here alive today because of people like you, who believe in medical research and believe in their innovations. Love and gratitude to all of you at Celgene!

Safety information on the use of IMBRUVICA

Thank you to my blood sister Anne for forwarding this information to me:

"Piece of my Heart-Part 2" By La Verne Abe Harris

IMBRUVICA (aka ibrutinib, pci-32765) is FDA-approved for the treatment of:

• Patients with mantle cell lymphoma (MCL) who have received at least one prior therapy.

• Patients with chronic lymphocytic leukemia (CLL) 

who have received at least one prior therapy.

The FDA's accelerated approval of these indications was based on the overall response rate of patients in the Phase II clinical trials of PCYC-1102 and PCYC-1104. 

The following safety information is described in the package insert for the use of IMBRUVICA in patients with mantle cell lymphoma who have received at least one prior therapy or chronic lymphocytic leukemia who have received at least one prior therapy:

IMPORTANT SAFETY INFORMATION 
WARNINGS AND PRECAUTIONS

Hemorrhage – Five percent of patients with MCL and 6% of patients with CLL had Grade 3 or higher bleeding events (subdural hematoma, ecchymoses, gastrointestinal bleeding, and hematuria). Overall, bleeding events including bruising of any grade occurred in 48% of patients with MCL treated with 560 mg daily and 63% of patients with CLL treated at 420 mg daily.

The mechanism for the bleeding events is not well understood.  IMBRUVICA may increase the risk of hemorrhage in patients receiving antiplatelet or anticoagulant therapies. Consider the benefit-risk of withholding IMBRUVICA for at least 3 to 7 days pre and post-surgery depending upon the type of surgery and the risk of bleeding.

Infections - Fatal and non-fatal infections have occurred with IMBRUVICA therapy. At least 25% of patients with MCL and 35% of patients with CLL had infections Grade 3 or greater NCI Common Terminology Criteria for Adverse Events (CTCAE). Monitor patients for fever and infections and evaluate promptly.

Myelosuppression - Treatment-emergent Grade 3 or 4 cytopenias were reported in 41% of patients with MCL and 35% of patients with CLL. These included neutropenia (29%), thrombocytopenia (17%) and anemia (9%) in patients with MCL and neutropenia (27%) and thrombocytopenia (10%) in patients with CLL. Monitor complete blood counts monthly.

Renal Toxicity - Fatal and serious cases of renal failure have occurred with IMBRUVICA therapy. Treatment-emergent increases in creatinine levels up to 1.5 times the upper limit of normal occurred in 67% of patients with MCL and 23% of patients with CLL. Increases in creatinine 1.5 to 3 times the upper limit of normal occurred in 9% of patients with MCL and 4% of patients with CLL. Periodically monitor creatinine levels. Maintain hydration.

Second Primary Malignancies - Other malignancies have occurred in 5% of patients with MCL and 10% of patients with CLL who have been treated with IMBRUVICA. Four percent of patients with MCL, had skin cancers, and 1% had other carcinomas. Eight percent of patients with CLL had skin cancers and 2% had other carcinomas.

Embryo-Fetal Toxicity - Based on findings in animals, IMBRUVICA can cause fetal harm when administered to a pregnant woman. Advise women to avoid becoming pregnant while taking IMBRUVICA. If this drug is used during pregnancy or if the patient becomes pregnant while taking this drug, the patient should be apprised of the potential hazard to a fetus.

ADVERSE REACTIONS –

MCL: The most commonly occurring adverse reactions (≥20%) in the clinical trial were thrombocytopenia*, diarrhea (51%), neutropenia*, anemia*, fatigue (41%), musculoskeletal pain (37%), peripheral edema (35%), upper respiratory tract infection (34%), nausea (31%), bruising (30%), dyspnea (27%), constipation (25%), rash (25%), abdominal pain (24%), vomiting (23%), and decreased appetite (21%).

*Treatment-emergent decreases (all grades) of platelets (57%), neutrophils (47%) and hemoglobin (41%) were based on laboratory measurements and adverse reactions.

The most common Grade 3 or 4 non-hematological adverse reactions (≥5%) were pneumonia (7%), abdominal pain (5%), atrial fibrillation (5.4%), diarrhea (5%), fatigue (5%), and skin infections (5%).  Treatment-emergent Grade 3 or 4 cytopenias were reported in 41% of patients. Ten patients (9%) discontinued treatment due to adverse reactions in the trial (N=111).

The most frequent adverse reaction leading to treatment discontinuation was subdural hematoma (1.8%). Adverse reactions leading to dose reduction occurred in 14% of patients.

CLL: The most commonly occurring adverse reactions (≥ 20%) in the clinical trial were thrombocytopenia*, diarrhea (63%), bruising (54%), neutropenia*, anemia*, upper respiratory tract infection (48%), fatigue (31%), musculoskeletal pain (27%), rash (27%), pyrexia (25%), constipation (23%), peripheral edema (23%), arthralgia (23%), nausea (21%), stomatitis (21%), sinusitis (21%), and dizziness (21%).

*Treatment-emergent decreases (all grades) of platelets (71%), neutrophils (54%) and hemoglobin (44%) were based on laboratory measurements per IWCLL criteria and adverse reactions.

The most common Grade 3 or 4 non-hematological adverse reactions (≥ 5%) were pneumonia (8%), hypertension (8%), atrial fibrillation (6.3%), sinusitis (6%), skin infection (6%), dehydration (6.4%), and musculoskeletal pain (6%). Treatment-emergent Grade 3 or 4 cytopenias were reported in 35% of patients.

Five patients (10%) discontinued treatment due to adverse reactions in the trial (N=48). These included 3 patients (6%) with infections and 2 patients (4%) with subdural hematomas. Adverse reactions leading to dose reduction occurred in 13% of patients.

DRUG INTERACTIONS

CYP3A Inhibitors - Avoid concomitant administration with strong or moderate inhibitors of CYP3A. If a moderate CYP3A inhibitor must be used, reduce the IMBRUVICA dose.

CYP3A Inducers - Avoid co-administration with strong CYP3A inducers.

SPECIAL POPULATIONS - Hepatic Impairment - Avoid use in patients with baseline hepatic impairment.

For the full prescribing information, visit http://www.imbruvica.com/downloads/Prescribing_Information.pdf

About IMBRUVICA

IMBRUVICA is indicated for the treatment of patients with mantle cell lymphoma or chronic lymphocytic leukemia who have received at least one prior therapy.1 For more information about IMBRUVICA, including the full prescribing information, please visit www.IMBRUVICA.com. IMBRUVICA is a first in class, oral therapy and is a new agent that inhibits a protein called Bruton's tyrosine kinase (BTK).1 BTK is a key signaling molecule of the B-cell receptor signaling complex that plays an important role in the survival and spread of malignant B cells.8,9,10 IMBRUVICA blocks signals that tell malignant B cells to multiply and spread uncontrollably.1,11 It is one of the first medicines to file for FDA approval via the new Breakthrough Therapy Designation pathway, enabling Pharmacyclics to rapidly bring this medicine to patients in need.

To date, 11 Phase III trials have been initiated with ibrutinib and a total of 42 trials are currently registered on www.clinicaltrials.gov. Janssen and Pharmacyclics entered a collaboration and license agreement in December 2011 to co-develop and co-commercialize IMBRUVICA.

About Pharmacyclics

Pharmacyclics® is a biopharmaceutical company focused on developing and commercializing innovative small-molecule drugs for the treatment of cancer and immune mediated diseases. Our mission and goal is to build a viable biopharmaceutical company that designs, develops and commercializes novel therapies intended to improve quality of life, increase duration of life and resolve serious unmet medical healthcare needs; and to identify and control promising product candidates based on scientific development and administrational expertise, develop our products in a rapid, cost-efficient manner and pursue commercialization and/or development partners when and where appropriate.

Pharmacyclics markets IMBRUVICA and has three product candidates in clinical development and several preclinical molecules in lead optimization. The company is committed to high standards of ethics, scientific rigor, and operational efficiency as it moves each of these programs to viable commercialization.

Pharmacyclics is headquartered in Sunnyvale, California and is listed on NASDAQ under the symbol PCYC. To learn more about how Pharmacyclics advances science to improve human healthcare visit us at www.pharmacyclics.com.

NOTE: This announcement may contain forward-looking statements made in reliance upon the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, including statements, among others, relating to our future capital requirements, including our expected liquidity position and timing of the receipt of certain milestone payments, and the sufficiency of our current assets to meet these requirements, our future results of operations, our expectations for and timing of ongoing or future clinical trials and regulatory approvals for any of our product candidates, and our plans, objectives, expectations and intentions. Because these statements apply to future events, they are subject to risks and uncertainties. When used in this announcement, the words "anticipate", "believe", "estimate", "expect", "expectation", "goal", "should", "would", "project", "plan", "predict", "intend", "target" and similar expressions are intended to identify such forward-looking statements. These forward-looking statements are based on information currently available to us and are subject to a number of risks, uncertainties and other factors that could cause our actual results, performance, expected liquidity or achievements to differ materially from those projected in, or implied by, these forward-looking statements. Factors that may cause such a difference include, without limitation, our need for substantial additional financing and the availability and terms of any such financing, the safety and/or efficacy results of clinical trials of our product candidates, our failure to obtain regulatory approvals or comply with ongoing governmental regulation, our ability to commercialize, manufacture and achieve market acceptance of any of our product candidates, for which we rely heavily on collaboration with third parties, and our ability to protect and enforce our intellectual property rights and to operate without infringing upon the proprietary rights of third parties. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, performance or achievements and no assurance can be given that the actual results will be consistent with these forward-looking statements. For more information about the risks and uncertainties that may affect our results, please see the Risk Factors section of our filings with the Securities and Exchange Commission, including our transition report on Form 10-K for the six month period ended December 31, 2012 and quarterly reports on Form 10-Q. We do not intend to update any of the forward-looking statements after the date of this announcement to conform these statements to actual results, to changes in management's expectations or otherwise, except as may be required by law.

Dr. Byrd serves as national principal investigator of this Pharmacyclics-sponsored clinical study. He has served as an unpaid advisor to both Pharmacyclics and Janssen in developing the compound ibrutinib. Byrd does not have a financial interest in either company.