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Rituximab Update

In September of 2012 I posted a blog about a biologic, Rituximab.  Some researchers had hoped this medication would prove to be an effective treatment for SLE.  Unfortunately, Rituximab  did not live up to expectations. However, the drug has been used to treat SLE  in rescue situations.  As such, it is tried when other options have failed. In many of these cases, Rituximab has been effective, especially when used along with  other drugs.
Even though clinical trials of Rituximab have not yielded promising results, investigation continues to see if it might be useful as part of a regimen with other lupus treatments.  As  experience with Rituximab accumulates–in doctors’ practices and in trials–data  is collected about potential side effects.  It is important for physicians and patients to be alert to these so that prompt action may be taken, if necessary.
A 2015 Hindawi Journal article reports a case of early-onset neutropenia and thrombocytopenia that was associated with the administration of Rituximab. The patient’s treating physicians entertained the possibility that these conditions may not have been caused by Rituximab but may have been a manifestation of SLE.  This analysis was rejected because the patient’s clinical profile did not support the conclusion.
This case of early-onset neutropenia and thrombocytopenia associated with Rituximab is unusual, if not unique. The authors of the Hindawi article contemplate the possibility that the side effects  may have also occurred in other cases but were not detected.
In any event, both  neutropenia and thrombocytopenia were transient in this case.  The  conditions cleared up 12 days after Rituximab therapy stopped.
risk of neutropenia is infection; a risk of thrombocytopenia is bleeding.
One thing that should be remembered as side effects of Rituximab are discussed: the medicine is used for very sick people, people who may not have many other options. Side effects will likely not be a reason to avoid treatment, but being aware of those side effects may help to keep the patient safe while treatment proceeds.
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Accelerating Medicines Partnership

The US government, Big Pharma and non-profit organizations have agreed to work together in a novel approach to finding effective treatments for lupus and other diseases. Called AMP (Accelerating Medicines Partnership), the collaboration would involve pooling research and information in the early stages.  All “partners” in the project would be able to learn from the work of others in the group.  The emphasis would be on finding biomarkers that would aid in the development of targeting drugs.

AMP grew out of the frustration of patients and doctors.  Access to effective drugs for devastating diseases, such as lupus and Alzheimer’s, is woefully limited.  Progress in finding new treatments has been slow. One of the reasons for this, some have suggested, is that competing firms doing research often duplicate the efforts of each and thus waste valuable time.  If all the research results could be pooled and both private and public resources dedicated to exploiting those results, progress might be realized.

The unique aspect of AMP is that although competing private companies would pool information on the early end of research efforts, at some point competing companies would separate and develop their own products. These, then, could be sold for profit. Pooling information would no longer be part of the process.

While AMP seems to offer great potential for advances in the treatment of certain diseases (the list is limited to lupus, Alzheimer’s, rheumatoid arthritis and diabetes), there are some researchers who question the approach.  Dr. Michael D. Lockshin, at the Hospital for Special Surgery in New York, wonders if the AMP approach for lupus is too narrow in focus.  Dr. Lockshin explains that lupus is a disease of many manifestations. He believes that effective treatments for lupus may emerge from AMP but that to eradicate the disease completely,  “...you will need to go into other fields.”

AMP is projected to continue for five years.  Results are scheduled to be reviewed regularly. Whatever questions may be raised about narrow focus or eventual profits reaped, one thing is certain: millions of people will benefit if this collaboration yields effective treatment for even one of the targeted diseases.

Lupus and Clinical Trials


 By A. G. Moore 11/23/2013
 Melingue_Jenner
 
 Edward Jenner injecting James Phipps, his gardener's
 son, with cowpox
 Lithograph by Gaston Melengue
 Wikimedia Commons, public domain, Copyright expired

Sixty years ago, when Flannery O’Connor learned she had lupus, one medication was available to treat her disease: hydrocortisone. Twenty years before her diagnosis, Edward O’Connor, her father, learned that he had lupus.  For him there was no treatment. Times have changed since Flannery and her father battled lupus. Today the list of treatment options is long. These options exist because researchers have worked tirelessly to solve the lupus puzzle and because they were aided in their efforts by the participation of countless lupus patients in clinical trials. While much of the work researchers do takes place in the laboratory, the proving ground for their investment is the clinical trial. Currently there are clinical trials underway that are exploring new theories in lupus therapy. One of these theories has been described by Dr. Timothy Niewold, who is a scientist affiliated with the Mayo Clinic. Dr. Niewold is investigating the role of interferon in lupus. There is clear evidence that in some people levels of interferon rise with disease activity. In certain instances, lupus has actually been precipitated by administration of interferon (for an unrelated illness). With the apparent association between interferon levels and lupus, it has been suggested that decreasing interferon production might temper symptoms of the disease. Drugs are being designed that are supposed to do just that. Targeting interferon is one example of the trend in lupus treatment to zero in on specific components of the immune response–rather than suppressing the entire immune system. When Flannery O’Connor was prescribed hydrocortisone, the drug was effective because it stopped her immune system from attacking her organs. However, shutting down the whole immune system is a rather blunt way of dealing with autoimmunity. It’s kind of like trying to weed the front lawn with a scythe; maybe the job gets done, but a whole lot of grass and flowers are sacrificed in the process. Shutting down the immune system can have devastating consequences. It is hoped that by targeting specific actors in the system, treatment may not only be more effective but may have less dramatic side effects. One of the interferon-targeting drugs currently under investigation is sifalimumab. Dr. Niewold (of the Mayo Clinic) explains that an important aspect of research on interferon-targeting medications is understanding how this cytokine acts in people from different ethnic backgrounds. As sifalimumab goes through its different trials, it is becoming clear that interferon does not have equal significance for all ethnic groups. For example, interferon activation in African American SLE patients is dependent on the presence of autoantibodies; this is not true for European Americans. Dr. Niewold states: “This heterogeneity may be clinically important, as therapeutics targeting this pathway are being developed.” Targeting specific aspects of the immune system is the theory behind the development of other lupus therapies. B and T cells, for example, are known to be very aggressive in active lupus. Belimumab , currently prescribed for certain types of lupus, targets B cells and Abatacept, which is an RA drug under consideration for lupus, targets T cells. Both drugs are currently in clinical trials. Clinical trials are essential if new therapies are to come on the market. They also may be of help to those who volunteer as subjects, because there is possibly the opportunity to receive novel and effective treatment. However, every person who volunteers as a subject should be clear about the goals of the study for which they have been recruited. Not every study has as a primary endpoint improvement in participants’ disease status. Some studies examine other aspects of drug development. Volunteers should never lose sight of the fact that participation may carry with it significant risks. One research paper I came across illustrates the importance of being informed before becoming a volunteer. This paper gives the results of a clinical trial in which sifalimumab was studied. The primary purpose of the trial is described: “..to evaluate the safety and tolerability of multiple doses of intravenous (IV) sifalimumab in patients with moderate-to-severe SLE.” Secondary objectives of the trial are also described: “..to evaluate the PK (Pharmacokinetics) and immunogenicity” of the drug. Only as a third and almost incidental objective is observation of the effect sifalimumab has on disease activity. The research paper describes this third objective in the following words: “…measurements of disease activity…were included only as an exploratory end point… ” The clinical trial described by this research paper has obvious significance for lupus treatment; finding out dose tolerance and learning about adverse side effects are important pieces in the development of a possibly valuable lupus intervention. However, these are findings that have general importance and likely will not immediately benefit study participants. It does not seem that those who conducted the study expected subjects in the clinical trial to experience sustained improvement in disease status; that was not the study’s defined prime objective. This leads me to repeat the following: if you are considering being a volunteer in a clinical trial, make sure you understand what it is that the researchers hope to learn from that trial. Does the trial offer participants an opportunity to reap the benefits of new lupus treatment, or are participants merely being mined, as it were, for information that may contribute some day to a good lupus intervention? Know that there are risks associated with participation. In the study described above, for example, some participants did die. Whether or not these deaths were attributable to sifalimumab is not certain, though, as the paper states, for at least two of the deaths, “… a potential role of the drug in contributing to the infection cannot be excluded.” Without clinical trials there cannot be new drugs to treat lupus. Volunteers for these trials are essential if the trials are to succeed. However, if you choose to become a subject, be clear about what it is you are submitting to. And then, if you are satisfied with the risk/reward ratio, go for it. You and everyone else with lupus may benefit.

Barbara Enright

(Excerpt from These Are the Faces of Lupus )

Block 16 in Glitter Gulch, at the beginning of the twentieth century, was home to brothels and gambling parlors.  Local residents recall that prostitutes in scanty attire used to sit on porches and wait for their clients to call. Glitter Gulch, and Block 16, were both testaments to the fact that through much of Las Vegas’ history, the city owed its economic existence to the patronage of men – men who worked the gold and silver mines in one century and men who constructed Hoover Dam in the next.

By the time Benny Binion built the Horseshoe Casino on a plot that abutted Block 16, the seedy gaming parlors and brothels had been bulldozed and converted to parking lots.  Not that Benny would have objected to the brothels, or any other income-producing enterprise.  Legend has it that he had fled to Vegas from Dallas back in ’46 because the Chicago mob and local politicians muscled him out of town. The way some tell the story, Benny left behind in Dallas a legacy of multiple murders, bootlegging and organized gambling rackets.

When Benny Binion set up business in Glitter Gulch, Vegas was a pretty open town, but not open enough for Benny.  He didn’t like the $50 limits that were traditionally placed on wagers, so he raised the limit to $500, and then he removed the limit altogether, (on first bets). Binion’s Horseshoe Casino became the first establishment to offer a no limit game.  It was also the first casino to put down carpet (instead of sawdust) on its floors.

If Benny was anything, he was an original.  Thus, in 1970 he began a new tradition, one which today has grown to attract draws participants and spectators from around the globe.  It was Benny who hosted the first World Series of Poker tournament.  While the tournament’s million dollar pot was originally the main draw, now the prestige earned by winning the title and the tournament’s gold bracelets are at least as coveted as the cash prize.

Today, Benny Binion and the Horseshoe Casino no longer sponsort the World Series of Poker.  Benny died in 1989, and a few years later a corporate conglomerate (Harrah’s) bought the rights to the tournament.  Before Benny departed from the poker scene, however, he pulled off at least one more “first”: the Women’s Tournament in the World Series of Poker. In 1986, Barbara Enright became the first Women’s Tournament champion and the first female to claim the tournament’s coveted gold bracelet.  Nine years later, she won her second Women’s Tournament, and her second gold bracelet.  

Barbara did not rest on her laurels.  She continued to enter open (mixed gender) competitions, until, in 1996 she won the World Series Pot Limit Hold ‘em Tournament and, to the surprise of almost everyone (except herself) made it to the finals table in the Main Event – the only woman ever to do so.

In the shadow of block 16, where women were once sold for as little as a dollar and where they whiled their days awaiting the pleasure of men, Barbara Enright played with the best of them – and won. By the time she received her third trophy bracelet in 1996, Barbara had been diagnosed with lupus for twenty years.

Barbara Enright was born on August 19, 1949 in Los Angeles California.  In her youth, she was licensed as a cosmetologist and worked at a number of jobs: cocktail waitress, bartender, and stylist for Hollywood celebrities.   In the 1970’s she tried her hand at poker in the Gardenia California card rooms, where she did so well that she eventually quit her other jobs and dedicated herself to poker.

Barbara asserts that she has never played a “woman’s” game.  She comes at her opponents with decidedly “unfeminine” aggression.  Enright explains her success in the traditionally male-dominated poker room:  she simply states that she is aggressive and that if a woman is too “soft” in tournament play she cannot succeed.  But if she asserts herself and defies the expectations of the men sitting at the table with her, she can do “very well.”

On July 6, 2007, Barbara Enright was inducted into the Poker Hall of Fame, the first woman ever to be invited into that select academy.  As of 2007, it was estimated that her tournament winnings exceeded $1,200,000 , more than any other woman in tournament history (at that time).

Today she lives in Hollywood California with Max Shapiro, a poker player and a columnist for Card Player Magazine.

Barbara once said that her dream was for there to be world peace and free medical coverage for every person. Perhaps the content of this dream was influenced by her experience with lupus. While most of her bios refer to the fact that Barbara has lupus, her challenge with the disease has remained private. Some writers refer to the disease as almost a footnote in her life; many writers do not refer to it at all.  It seems that the kind of lupus Barbara lives with and the treatment she receives for it, are very different from the lupus that took Inday Ba’s life.* Since her diagnosis in 1976, Barbara has raised a child, managed a successful career in an exotic profession and earned a reputation for being smart, friendly and focused.

In the storied saloons of Glitter Gulch, where ladies were historically dispatched like grains of sand from the vast and unforgiving Mojave desert, Barbara Enright made her mark.  A combination of raw talent, hardscrabble determination and a dollop of luck helped her to secure a place in the history of Las Vegas and the world of professional poker.

* Actress who died or Lupus at the age of 32

What Is Lupus

What is Lupus?

By A. G. Moore

Autoimmune disease: auto, from the Greek, means self (as in automobile: a self-moving vehicle). An autoimmune reaction, in which the body essentially makes itself sick, can be as simple as a rash induced by contact with poison ivy, or a full-blown case of systemic lupus.

The analogy to a car is helpful here: a car (automobile) operates automatically only in the sense that someone turns it on, turns it off and directs its path. However, from time to time reports appear in the newspapers of cars that are out of control—cars that somehow accelerate on their own or fail to respond to instructions to stop, or turn. These automobiles, instead of being dutiful and useful servants, become dangerous agents. This is sort of what happens in an autoimmune disease.

The immune system exists to protect the body. A complex set of interactions go into operation when the body is under assault. The system includes the ability to surround and consume an alien entity (such as bacteria). The trigger—or ignition, if comparing it to a car—for the immune system to go into action is an external threat. In a healthy individual, when the threat is removed the immune system becomes quiet.

However, when autoimmune disease takes hold, the immune system goes haywire, like a car out of control. It starts attacking things that aren’t foreign because it sees healthy tissue as an enemy. In lupus, the attacking immune cells can go after just about anything—the lungs, heart, blood, brain, etc.

The mystery of why this happens is only vaguely understood. Some of the triggers that set off the autoimmune attack have been identified—Espstein-Barr and UV exposure, for example. But on the whole, the autoimmune syndrome that is at the heart of lupus remains a mystery. Once this mystery is solved, then a cure, or even a way of preventing the disease, may be found.

Right now, medicine can offer people who have lupus only one thing: possible control of their symptoms. In most cases this is achievable, though sometimes at great cost.

Lupus can be very resistant to treatment. Since it is still a mystery and since the remedies for the disease are diverse and variably effective, it makes sense that someone with lupus find the smartest, most skilled physician available to treat their disease. Only a doctor with insight, experience and top-notch training should be enlisted in the war against lupus, an obstinate and potentially fatal autoimmune illness.

Reference:

What is Lupus?http://www.health.ny.gov/diseases/conditions/lupus/fa

What is Lupus?http://www.lupusinternational.com/About-Lupus-1-1/What-is-Lupus-.aspx

What is Lupus?http://www.lupusalliance.org/content.asp?id=8

What is Lupus?http://www.lupus.org/webmodules/webarticlesnet/templates/new_learnunderstanding.aspx?articleid=2232&zoneid=523