Professor Jennifer L. Aaker speaking about National Marrow Donor Program at #med2

I registered for the Be The Match Registry in 2009 when I was moved by a story on Facebook about a young woman named Jodie Gee. There is a tremendous need for Asians to register on the Be The Match Registry. The National Marrow Donor Program® (NMDP) is a nonprofit organization dedicated to creating an opportunity for all patients to receive the bone marrow or umbilical cord blood transplant they need, when they need it. I encourage you to register for the Be The Match Registry. You can request a cheek swab kit here.

Medicine 2.0 kicked off this morning with this:

Professor Jennifer L. Aaker shared a moving story about two south-east Asians (Sameer and Vinay) who were diagnosed with leukemia and who required a bone marrow transplant to survive. She spoke about the power of social media and about social change. How do you influence the community in a way that revolutionizes the world? To learn the answer,  I encourage you to read her book titled, "The Dragonfly Effect."

Congrats to my wife Ellen for finishing the Philly marathon today!

I'm very proud of my wife Ellen who finished her first marathon today! It was the Philadelphia marathon and she ran it to support the Leukemia and Lymphoma Society (Team in Training). Congrats Ellen!

Mylotarg (gemtuzumab ozogamicin) removed from the market

Here it is from the FDA:

FDA: Pfizer Voluntarily Withdraws Cancer Treatment Mylotarg from U.S. Market

Pfizer Inc. today announced the voluntary withdrawal from the U.S. market of the drug Mylotarg (gemtuzumab ozogamicin) for patients with acute myeloid leukemia (AML), a bone marrow cancer. The company took the action at the request of the U.S. Food and Drug Administration after results from a recent clinical trial raised new concerns about the product’s safety and the drug failed to demonstrate clinical benefit to patients enrolled in trials.

Optimal treatment for CML (Chronic Myeloid Leukemia)

What's the optimal treatment for CML (Chronic Myeloid Leukemia)?  In the New England Journal of Medicine (NEJM), there were a few interesting articles this week that caught my attention:

• Dasatinib versus Imatinib in Newly Diagnosed Chronic-Phase Chronic Myeloid Leukemia
• Nilotinib versus Imatinib for Newly Diagnosed Chronic Myeloid Leukemia

Both of these studies were comparing imatinib (Gleevec) to newer drugs (dasatinib or Sprycel and nilotinib or Tasigna). In both cases, the newer agent appeared to result in better clinical responses (see conclusions below). If this is now the case, where does imatinib fall into treatment regimens for CML?  Who's going to want to receive Gleevec for their CML?

Conclusions: Dasatinib, administered once daily, as compared with imatinib, administered once daily, induced significantly higher and faster rates of complete cytogenetic response and major molecular response. Since achieving complete cytogenetic response within 12 months has been associated with better long-term, progression-free survival, dasatinib may improve the long-term outcomes among patients with newly diagnosed chronic-phase CML.

Conclusions: Nilotinib at a dose of either 300 mg or 400 mg twice daily was superior to imatinib in patients with newly diagnosed chronic-phase Philadelphia chromosome–positive CML.

Arzerra (ofatumumab) for CLL approved by the FDA

FDA NEWS RELEASE

For Immediate Release: Oct. 26, 2009

Media Inquiries: Karen Riley, 301-796-4674, karen.riley@fda.hhs.gov
Consumer Inquiries: 888-INFO-FDA

FDA Approves New Treatment for Chronic Lymphocytic Leukemia

The U.S. Food and Drug Administration today approved Arzerra (ofatumumab) for patients with chronic lymphocytic leukemia (CLL), a slowly progressing cancer of the blood and bone marrow.

Arzerra is approved for patients with CLL whose cancer is no longer being controlled by other forms of chemotherapy.

CLL primarily affects people older than 50 and arises from a group of white blood cells known as B-cells that are part of the body’s immune system. Each year, about 16,000 people are diagnosed with CLL and about 4,400 people die from the disease.

Arzerra is a monoclonal antibody, a type of biotechnology product. Antibodies that occur in nature are produced by the immune system in response to invaders. Arzerra binds to a specific protein found on the surface of both normal and malignant B cells, making the cells more susceptible to immune system attack.

The product was approved under the FDA’s accelerated approval process, which allows earlier approval of drugs that meet unmet medical needs. Products may receive accelerated approval based on a surrogate endpoint, such as a reduction in the size of the tumor or decrease in the number of cancerous white cells or in an enlarged spleen or lymph nodes. These indirect measures for clinical outcomes are considered reasonably likely to predict that the drug will allow patients to live longer or with fewer side effects of a disease.

“The approval of Arzerra illustrates FDA's commitment to using the accelerated approval process to approve drugs for patients who have limited therapeutic options,” said Richard Pazdur, M.D., director of the Office of Oncology Drug Products in the FDA's Center for Drug Evaluation and Research.

The accelerated approval process requires further study of the drug. The manufacturer is currently conducting a clinical trial in CLL patients to confirm that the addition of Arzerra to standard chemotherapy delays the progression of the disease.

Arzerra's effectiveness was evaluated in 59 patients with CLL whose disease no longer responded to the available therapies.

The product’s safety was evaluated in 181 patients in two studies in patients with cancer. Common side effects included a decrease in normal white blood cells, pneumonia, fever, cough, diarrhea, lower red blood cell counts, fatigue, shortness of breath, rash, nausea, bronchitis and upper respiratory tract infections.

The most serious side effects of Arzerra are increased chance of infections, including progressive multifocal leukoencephalopathy (PML), a brain infection that is generally fatal. Patients at high risk for Hepatitis B should be screened before being treated with Arzerra. Patients with evidence of inactive hepatitis should be monitored for re-activation of the infection during and after completing treatment.

Arzerra is manufactured by London-based GlaxoSmithKline.

Heart transplant cancer patient running the Ironman World Championship

Wow! This story just makes you want to go to the gym and jump on a treadmill. Heart transplant recipient and cancer survivor Kyle Garlett will compete in October's Ironman World Championship! What an amazing success story of someone who defeated a serious illness.

• Kyle was diagnosed with Hodgkin's lymphoma as a high school senior in 1989.
• He developed leukemia as a result of his earlier chemotherapy.
• He received a heart transplant in 2006.
• Now he's running marathons and competing in triathlon.
  

Isn't it amazing what some people accomplish in their lives? I also really admire Lance Armstrong who battled testicular cancer and won multiple consecutive victories in the Tour de France. Time for me to go to the gym!

Read the amazing CNN story here.

Arzerra (ofatumumab) appears promising for CLL

Arzerra (ofatumumab) appears promising for chronic lymphocytic leukemia (CLL). Ofatumumab (formerly known as HuMax-CD20) is a targeted therapy. It is actually a human monoclonal antibody that targets a distinct antibody binding site (the small loop epitope) of the CD20 molecule on the cell membrane of B cells. It is currently under development for the treatment of CLL and has also shown potential in treating follicular non-Hodgkin’s lymphoma, diffuse large B cell lymphoma, rheumatoid arthritis and relapsing remitting multiple sclerosis. Is it available yet? No. The FDA is currently still reviewing this biologic agent. Here's a snippet from the June GSK press release:

GlaxoSmithKline and Genmab A/S today announced that the United States Food and Drug Administration (FDA) informed the companies that the agency has extended the action date for the ofatumumab BLA application by three months.

The FDA has been slow recently, don't you think? Better safe than sorry? Or, is the FDA being overly-cautious? We don't want to see another Raptiva incident again, do we? Raptiva was voluntarily withdrawn in April by Genentech because of an increased risk of progressive multifocal leukoencephalopathy (PML), a rare and usually fatal disease of the central nervous system. It's difficult to find such rare adverse effects until the drug gets used by the public, so I don't think anyone is faulting either the FDA or Genentech.

Well, let's see what happens with ofatumumab.

Are You Willing to Save a Life?

If you are, then please register to donate your bone marrow to save a life. There's a person named Nick Glasgow who has a Facebook page about his fight against acute leukemia (to find this page, search for "SAVE NICK GLASGOW" after you log into Facebook. He's in need of a bone marrow transplant and you could be the one to save his life. Given his unique ethnic (he's 1/4 Japanese and 3/4 Caucasian), it will be very difficult for him to find a match. Advances in medical technology and drug development have led to some incredible cancer therapies, but a bone marrow transplant still remains the only cure for many diseases.

I was deeply moved by a story on Facebook about a woman named Jodie Gee who was also in need of a bone marrow transplant. Her story inspired me to register with the Asian American Donor Program at AADP.org. Here's what I had to do to register: order a registration kit (which was free through the AADP); sign several forms; swab the inside of your mouth; and send the packet in the mail (pre-stamped). It only takes a few minutes of your time and you'll never be forced to donate if you're on the registry.