Sunday, November 15, 2009

When Bad Things Happen to Famous People

In a press release issued earlier this week, former NBA star and actor Kareem Abdul-Jabbar announced that he has been diagnosed with leukemia. Specifically, he has chronic myeloid leukemia (CML). Abdul-Jabbar went on to say that his disease can be managed by taking daily oral medication and he expects to live a long healthy life.

With leukemia? How is that possible?

Abdul-Jabbar has benefited from one of the first and most exciting applications of the translational research I have blogged about in the past.

Before 2000, CML was treated with a combination of a chemotherapy drug called cytarabine and another drug called alpha-interferon. Patients treated with this combination usually responded well, but none were cured. The only curative treatment was a bone marrow transplant, and in adults, bone marrow transplantation carries a significant risk of death.

So how will Kareem Abdul-Jabbar lead a long healthy life by just taking a daily oral medication?

The answer lies in the molecular biology of CML. It shows how basic research, undertaken for no reason other than to understand the biology of cancer, can lead to unexpected therapeutic breakthroughs. Such research can transform a disease like CML from one that kills without a bone marrow transplant into one that is managed just like high blood pressure.

Here’s how it happened:

Working in Chicago in the 1970’s, Janet Rowley discovered that the “Philadelphia Chromosome,” an abnormal chromosome seen only in the leukemia cells of patients with CML, is actually composed of pieces of two different chromosomes. Her idea, that so-called chromosome translocations could result in the creation of new genes that can cause disease, was heretical at the time, but is now a part of the standard dogma of oncology. She was rewarded with a Lasker Award (often called the “American Nobel”) in 1998.

Subsequent research demonstrated that the Philadelphia Chromosome instructs the leukemia cells to make a new enzyme (called a tyrosine kinase) that causes the leukemia.

Researchers led by Brian Druker eventually developed a drug that blocks the activity of the tyrosine kinase that results from the Philadelphia chromosome. This drug, called imatinib (the trade name is Gleevec), is a pill that, taken daily, kills CML leukemia cells. This work was also rewarded with a Lasker Award in 2009.

The net result of the work of Dr. Janet Rowley, Dr. Brian Druker, and a host of others is that CML has been transformed from a disease curable only by bone marrow transplantation, into a disease that can be managed as a chronic condition, by taking pills every day. An incredible change over a short period of time!

This is the model of basic and translational research those of us in the field emulate, and the prime example of targeted therapy for cancer.

Work that started out as very basic science, motivated only by a desire to understand biology, has led to a transformative new medicine, and now Kareem Abdul-Jabbar (and many people far less famous than him) should be able to lead a healthy, active life taking daily oral medication, despite having leukemia.

Related Post:
When Translational Research Really Translates


Kristen's mom said...

Do you know any children with down syndrome that have had a successful bone marrow transplant? My daughter is now fighting leukemia for the 4th time. When she was 3 she was diagnosed with ALL, she relapsed after being off treatment for 18 months. She was cancer free for 12 years. She was diagnosed with AML last Septenber,and after being off treatment for 7 months, this past week she relapsed. We were told that her only chance was a bone marrow transplant but that they were extremely risky for a child with down syndrome. And first she has to reach a full remission, which might be a litlle tough. I can find no information of a child with down syndrome have a BMT.

Doctor David said...

Hi Kristen's mom,

Our group successfully transplanted a little boy with Down syndrome and relapsed AML a couple of years ago. So it can be done!

Web design firms said...

I recently came across your blog and have been reading along. I thought I would leave my first comment. I dont know what to say except that I have enjoyed reading. Nice blog. I will keep visiting this blog very often.

Anonymous said...

I'm curious as to what a year of Gleevec would cost if someone didn't have insurance and had CML.

StorytellERdoc said...

Nice medical sites. I just started my own blog, and I found your's searching through the other mediblogs. Well written.

Stop by.

Anonymous said...

I work at Fox Chase Cancer Center, formerly the Institute for Cancer Research where the Philadelphia chromosome was discovered. It is also home to Dr Meg von Mehren who you probably know and who was a major player in the Gleevec clinical trials. It is a wonderful place to work, but we don't treat kids. We leave that to the experts at CHOP.

lymphoma leukemia said...

I would like get more information about this, thanks for sharing this information...

levitra cialis said...

I did knew about Kareem's condition, the treatment seems to be very good and modern, lets hope that he can live more.

PinkLeaf said...

Hi Dr. David, my mother has had CML for 6 years and thank God, she is now in remission. Thanks for the lucid explanation of CML's biology and how Gleevec works!

Anonymous said...

My husband was on Gleevec for 2 1/2 years and it stopped working. He had been in a "complete molecular response." He then started on Sprycel and it caused many side effects, including fluid in chest cavity. Doctor started him on Iclusig (Ponatinib) 3 months ago and he was feeling great. A trip to his cardiologist yesterday showed a high heart rate since he's been on Iclusig. Iclusig can cause heart problems.He had his mitral valve replaced with a bovine valve 8 yrs ago and he has a pacemaker so his heart is monitored so that's how they know his heart rate has been up since he started on Iclusig. He's running out of options for his CML.