Lupus, an autoimmune disease, [recently] turned up on the front page of the Wall Street Journal (WSJ). It cropped up, also, on the first page of the New York Times business section, and elsewhere. Scientific American published a nice online review just now. The reason is that the FDA has approved a new monoclonal antibody for treatment of this condition.
The drug belimumab (Benlysta), targets a molecule called BlyS (B-lymphocyte Stimulator). The newspapers uniformly emphasize that this drug marks some sort of triumph for Human Genome Sciences, a biotech company that first reported on BlyS in the journal Science way back in 1999. BlyS triggers B cells to produce antibodies that in patients with lupus tend to bind and destroy their own cells’ needed machinery, causing various joint, lung, liver, kidney, brain, blood vessel and other sometimes life-threatening problems. So if and when Benlysta works, it probably does so by blocking aberrant autoimmune B-cell activity.
The newspapers don’t give a lot of details on the drug’s effectiveness, except that it appears to help roughly one in 11 patients, and the main benefit may be that some lupus patients on Benlysta can reduce their use of steroids, which have long-term and toxic effects on many organs. The most recent major medical publication on a trial on the drug came out in the Lancet two weeks ago.
Some reported caveats are that the drug has not been adequately tested or approved for patients with severe kidney or neurological manifestations of the disease, and that its activity, marginal as it is, appears to be less in patients of African heritage based on trials completed thus far. Additional trials are in the works.
The drug is expensive, to the updated tune of $35,000 per year. According to the WSJ: “Estimates of how many Americans are affected range from 161,000 to 1.5 million.” (How’s that for a wide ballpark figure? Likely a function of how hard it is to define and establish diagnosis for this disease, which anticipates how hard it will be to measure this drug’s effects — see below.) The same WSJ piece says analysts expect the drug to become a blockbuster, with annual sales eventually topping $1 billion. Read more »
*This blog post was originally published at Medical Lessons*
Maybe you read the other day in The New York Times that the pharmaceutical industry has a problem. Big blockbuster drugs like Lipitor are going off patent and the industry leaders don’t have new blockbusters showing promise to replace them. So the big companies search for little companies with new discoveries and they consider buying them. Industry observers think the days of $5 billion-a-year drugs to lower cholesterol or control diabetes may be past for awhile, and the companies will have smaller hits with new compounds for autoimmune conditions and cancer.
When I saw my oncologist for a checkup yesterday — the news was good — we chatted about the article and the trend toward “niche science.” We welcomed it. We didn’t think — from our perspective — the world needed yet another drug to lower cholesterol. We need unique products to fight illnesses that remain daunting, some where there are no effective drugs at all. For example, my daughter has suffered for years from what seems to be an autoimmune condition called eosinophilic gastroenteritis (EGID). Her stomach gets inflamed with her own eosinophil cells. They would normally be marshaled to fight a parasite in her GI tract but in this case, there’s nothing to attack. So the cells make trouble on the lining of the stomach and cause pain and scarring. Right now, there’s no “magic bullet” to turn off these cells. My hope is some pharma scientists will come up with something to fill this unmet need.
In the waiting room before I saw my doctor at the cancer center in Seattle I overheard a woman on the phone speaking about her husband’s new diagnosis of pancreatic cancer. I was sitting at a patient education computer station nearby. When she was finished I introduced myself and showed her some webpages to give her education and hope: pancan.org and our Patient Power programs about the disease. She was grateful. I did tell her — and she already knew — that there was no miracle drug for pancreatic cancer and that it was a usually-fatal condition. But that there were exceptions and, hopefully, her husband would be one. Of course, wouldn’t an effective medicine be best? Read more »
*This blog post was originally published at Andrew's Blog*
The New York Times has a series called “Patient Voices” which gives insights from the patients with the disease, physical and emotional changes in their lives, and accommodations made. The most recent series is on patients with alopecia (hair loss).
“The Voices of Alopecia” by Tara Parker-Pope (July 6, 2010):
This week, Patient Voices explores alopecia, an autoimmune disease that leads to a few bald patches to the loss of every hair on a person’s body.
To hear what it’s like to live with alopecia, listen to the Patient Voices audio slideshow that features adults, children and their parents who are coping with the condition.
Listen to these seven people tell what it’s like to live with alopecia:
– Matt Kelly, 43, lost his hair at age 38 over a 6 week time span.
– Jennifer DeFreece, 29, developed alopecia totalis as a child.
– Margaret Staib, 42, an artist with three daughters.
– Rafi Wasselman, 16, says his best medicine is his collection of caps.
– Maureen McGettigan, 47, began losing her hair at age 16.
– Annie Kazmi, 33, tells her daughter Noori’s story. Then Noori tells her own. Read more »
*This blog post was originally published at Suture for a Living*
Daniel Wallace, MD
The annual American College of Rheumatology conference was held last week in San Francisco. I had the chance to interview Dr. Daniel Wallace, a world expert in lupus (systemic lupus erythematosis) management, to tell me about the latest advances in the treatment of this disease.
Dr. Wallace is currently a Clinical Professor of Medicine at the David Geffen School of Medicine at UCLA. His clinical practice is based at Cedars-Sinai, where he is involved in the care of 2,000 lupus patients, the largest practice of its kind in the United States. The Wallace Rheumatic Disease Research Center currently runs over 30 clinical trials for patients with lupus and other rheumatic diseases. Dr. Wallace is the author of 6 medical textbooks, 15 book chapters, and over 200 medical publications.
**Listen to the podcast of our interview here**
Dr. Val: What is Lupus?
Dr. Wallace: Lupus is what happens when the body becomes allergic to itself. It’s the opposite of cancer and AIDS. There are probably about 1 million people living with lupus in the United States. Ninety percent of them are women, and 90% develop lupus during their reproductive years.
Dr. Val: Historically speaking, what has treatment been like for patients with lupus, and how has that changed over the years? Read more »