Tag Archives: treatment

Many times when seeing a physician for pemphigus or pemphigoid they are quick to prescribe a systemic treatment that will hopefully help you reach remission. This can be a good thing. However, sometimes the obvious may be overlooked.  For example, if you are in pain,  having trouble eating or swallowing, your clothes are sticking to your lesions, the blisters on your scalp make bathing and showering difficult, or perhaps you are having chronic nosebleeds. These symptoms can be managed with topical treatments, but they are often forgotten. There are different options available for different body locations in many different strengths. Be candid with your doctor and let them know where you are having disease activity and how severe it is. Although, ultimately, the systemic treatment is going to make the difference in the long run.  Topical treatment can help relieve many of your symptoms along the way!

If you’re not sure which medications to ask for or their strengths, just “Ask a Coach”!

Remember, when you need us we are in your corner!

664715_11160870-pillRare diseases, including several autoimmune disorders, are getting more attention from drug-makers, according to a new report by the Pharmaceutical Research and Manufacturers of America (PhRMA), a consortium of 36 US-based pharmaceutical and biotechnology companies. In 2012 alone, 13 drugs for orphan diseases (“orphan drugs”) were approved by the Food and Drug Administration (FDA). Approximately 452 medicines and vaccines are in development for the nearly 7,000 orphan diseases worldwide.

orphan diseases are defined as diseases with fewer than 200,000 patients. In total, however, across the nearly 7,000 orphan diseases, 30 million people in the US, or about 10% of the population, are affected by an orphan disease. The pemphigus and pemphigoid (P/P) diseases are considered “ultra-orphan” diseases because they are extremely rare. It is estimated there are only about 50,000 new P/P cases each year worldwide, with only a few thousand of those being in the US.

Rare diseases tend to be more complex than common diseases, meaning that there are a number of factors that combine to cause disease. In the case of P/P, while there seem to be genetic risk factors, how these contribute, singularly or in combination, and to what extent the environment (like diet and other conditions that are present) also contributes is not well understood.

Somewhat fortuitously, complex diseases represent the next great frontier for drug developers. Having tapped into the ‘simpler’ diseases, making great strides in treatment of conditions like high cholesterol, these ‘low-hanging fruit’, as drug-makers like to call them, have been consumed. It is truly a time of paradigm-shifting mentality among drug makers.

That said, the costs of developing new medicines is extremely high, so companies must make their choices wisely. If we were to calculate the amount that pharmaceutical and biotechnology companies spend on research and development yearly and compare that to the number of drugs that are approved for clinical use by the FDA each year, the cost per successful drug is a staggering $1.2 billion. It’s not difficult to imagine, then, why companies aimed at developing new drugs are most interested in those that can recoup these huge costs — for instance, by developing drugs for very common conditions and risk factors such as diabetes and high cholesterol. As well, given the complex nature of rare diseases, they are not necessarily among the ‘low-hanging fruit’ that some diseases represent.

To incentivize companies to prioritize new drugs for rare conditions, they may apply for orphan drug status through the FDA, a result of passage of the Orphan Drug Act (ODA) of 1983. With this status, a drug receives seven years of market exclusivity. Market exclusivity is particularly appealing to companies developing drugs because the seven-year exclusivity period differs from laws applicable to other drugs in that it does not begin until the drug is approved by the FDA approval.

The ODA is considered a resounding success. Since its inception, there have been more than 400 medicines approved for a total of 447 orphan diseases. As well, there are hundreds of new medicines in development, including an impressive list available in the PhRMA 2013 report (phrma.org/sites/default/files/pdf/Rare_Diseases_2013.pdf).

While not all of the 452 orphan drugs in development will be approved for patient use, this is certainly a lot of activity. A search of the list included within the PhRMA report, as well as a search of clinicaltrials.gov (that lists all clinical trials in progress), shows a handful of drugs in testing for conditions related to or directed at P/P.

There are 18 new orphan drugs in phase I-III trials (there are three phases of clinical trials and drugs must pass all of them, indicating reasonable levels of safety and meaningful efficacy-effectiveness in treating the condition) that are indicated for autoimmune disorders.

New drugs are not the only source of treatment for disease. Another source is to use an existing drug, developed for another condition, for a different indication. Such is the case with Rituxan® (rituximab), which was originally developed for Non-Hodgkin’s lymphoma. In that disease, B cells of the immune system bearing a marker called CD20 (thus the name CD20+ B cells) have gone awry.

Since P/P shares this hallmark, Rituxan® has been successfully used ‘off-label’ for P/P. It is an antibody-based drug, which requires it to be injected into the patient. In general, any drug that acts as a suppressor of the immune system (immunosuppressant) is a potential candidate for treating a range of auto-immune conditions, including P/P. CellCept® (mycophenolate mofetil), another immune system suppressor that was developed for transplant patients to help prevent the body’s rejection of the ‘foreign’ organ, has recently been approved for use in P/P.

Besides the high cost of developing new drugs, companies that seek treatments for orphan diseases face difficulty in finding enough patients to participate. Indeed, patients tend to be dispersed geographically and may include small children. Physicians and patients who are interested in participating in trials or gaining more information should visit clinicaltrials.org.

Within the P/P community, the IPPF is also a great resource for learning about clinical trials. Members of our medical advisory board serve as investigators on trials and being in our patient database could lead to a company reaching out to you about participating in a trial.

For instance, among the new drugs aimed at treating P/P, drug-maker Novartis is studying VAY736, an antibody-based drug aimed at another B cell marker called BAFF-R. The study is in a very early stage and should be recruiting patients soon.

The time is ripe for development of new drugs for complex orphan diseases. The surge in new medicines in the first 30 years since the ODA should accelerate as less ‘low-hanging fruit’ exist for companies developing new drugs.

A new study in mice where researchers replicated a rare type of immune cell in the lab and then infused it back into the body, is raising hope for a new treatment for severe autoimmune diseases such as multiple sclerosis and rheumatoid arthritis.

The researchers, from Duke University Medical Center in the US, write about their work on a type of B cell, in a paper that was published online in Nature at the weekend.

B Cells

B cells are immune cells that create antibodies to attack unwanted pathogens like bacteria and viruses.

The type that the researchers on this study focused on are known as regulatory B cells or B10, after interleukin-10 (IL-10), a cell-signalling protein that the cells use.

B10 cells help control immune response and limit autoimmunity, which is where the immune system attacks the body’s own healthy tissue as if it were an unwanted pathogen.

Although there aren’t many of them, B10 cells play a key role in controlling inflammation: they limit normal immune response during inflammation, thus averting damage to healthy tissue.

Regulating Immune Response Is a Highly Controlled Process

Study author Thomas F. Tedder is a professor of immunology at Duke. He says in a statement that we are only just beginning to understand these recently discovered B10 cells.

He says these regulatory B cells are important because they “make sure an immune response doesn’t get carried away, resulting in autoimmunity or pathology”.

“This study shows for the first time that there is a highly controlled process that determines when and where these cells produce IL-10,” he adds.

What they Did

For their study, Tedder and colleagues used mice to study how B10 cells produce IL-10. For IL-10 production to start, the B10 cells have to interact with T cells, which are involved in switching on the immune system.

They found B10 cells only react to certain antigens. They found that binding to these antigens makes the B10 cells turn off some of the T cells (when they come across the same antigen). This stops the immune system from harming healthy tissue.

This was a new insight into the function of B10 cells that spurred the researchers to see if they could take this further: what if it were possible to use this cellular control mechanism to regulate immune responses, particularly in respect of autoimmunity?

Replicating Large Numbers Outside the Body

B10 cells however are not common, they are extremely rare. So Tedder and colleagues had to find a way to make a ready supply of them outside the body.

They found a way to isolate the B10 cells without damaging their ability to control the immune responses. And they found a way to replicate them in large numbers, as Tedder explains:

“Normal B cells usually die quickly when cultured, but we have learned how to expand their numbers by about 25,000-fold.”

“However, the rare B10 cells in the cultures expand their numbers by four-million-fold, which is remarkable. Now, we can take the B10 cells from one mouse and increase them in culture over nine days to where we can effectively treat 8,000 mice with autoimmune disease,” he adds.

Influencing Autoimmunity

The next stage was to try out the new B10 cells: could they influence autoimmunity sufficiently to affect disease symptoms?

They found when they introduced a small number of B10 cells into mice bred to have a disease similar to multiple sclerosis, their symptoms lessened significantly.

“B10 cells will only shut off what they are programmed to shut off,” explains Tedder.

If you have rheumatoid arthritis, you would want cells that would only go after your rheumatoid arthritis,” he adds.


He and his colleagues suggest their work shows there is potential to remove regulatory cells, replicate them in their millions, and put them back in the body of a person with an autoimmune disease and it will effectively “shut down the disease”, as Tedder describes it:

“This may also treat transplanted organ rejection,” he adds.

The researchers call for more studies to learn how to replicate human B10 cells, and find out how they behave in humans.

Autoimmune diseases are complex, so making a single therapy that targets several diseases without causing immunosuppression is not easy, Tedder explains.

“Here, we’re hoping to take what Mother Nature has already created, improve on it by expanding the cells outside of the body, and then put them back in to let Mother Nature go back to work,” he says.

Grants from the National Institutes of Health, the Lymphoma Research Foundation, and the Division of Intramural Research, National Heart, Lung, and Blood Institute, NIH, helped pay for the study.

Article from: http://www.medicalnewstoday.com/articles/251507.php

Written by Catharine Paddock PhD
Copyright: Medical News Today

Pemphigus vulgaris (PV) is an autoimmune disease in which the body’s immune system develops antibodies to two of its own proteins, the desmogleins DSG1 and DSG3 that help maintain the integrity of the skin. The immune attack causes painful blisters on the skin and mucus membranes that can lead to infections. Current therapies are geared towards suppressing the entire immune system, but this is problematic as it causes many side effects and leaves the patient vulnerable to infection.

To identify better therapeutic targets, researchers at the Institute for Research in Biomedicine in Bellinzona, Switzerland, identified the portions of DSG1 and DSG3 that are targeted by antibodies. In the study, published this month in the Journal of Clinical Investigation, Antonio Lanzavecchia and colleagues collected immune cells from PV patients and isolated the antibodies to determine which ones were involved in PV. By studying the antibodies, they were able to identify regions of DSG3 that are the primary target of the immune sysm. These findings could help with new ways to diagnose and treat PV.

Full article available at: http://www.medicalnewstoday.com/releases/249883.php

Erythema multiforme (EM) is an uncommon, immune-mediated disorder that presents with cutaneous or mucosal lesions or both. In herpes simplex virus (HSV)–associated EM, the findings are thought to result from cell-mediated immune reaction against viral antigen-positive cells that contain the HSV DNA polymerase gene (pol ). The target lesion, with concentric zones of color change, represents the characteristic cutaneous finding seen in this disorder. Although EM can be induced by various factors, HSV infection continues to be the most common inciting factor. Histopathologic testing and other laboratory investigations may be used to confirm the diagnosis of EM and to differentiate it from other clinical imitators. Imitators of EM include urticaria, Stevens-Johnson syndrome, fixed drug eruption, bullous pemphigoid, paraneoplastic pemphigus, Sweet’s syndrome, Rowell’s syndrome, polymorphus light eruption, and cutaneous small-vessel vasculitis. Because disease severity and mucosal involvement differ among patients, treatment should be tailored to each patient, with careful consideration of treatment risk vs benefit. Mild cutaneous involvement of EM can be managed primarily with a goal of achieving symptomatic improvement; however, patients with HSV-associated recurrent EM and idiopathic recurrent EM require treatment with antiviral prophylaxis. Inpatient hospitalization may be required for patients with severe mucosal involvement that causes poor oral intake and subsequent fluid and electrolyte imbalance. With this review, we strive to provide guidance to the practicing dermatologist in the evaluation and treatment of a patient with EM.


This report describes the clinical presentations and treatment responses of three children with PV, as confirmed according to histology and indirect immunofluorescence studies. In all three cases, oral prednisone used in conjunction with mycophenolate mofetil (MMF) resulted in complete clinical remission, during which all pharmacotherapy was successfully discontinued. Resolution of the skin and mucosal blistering tended to occur quickly with prednisone, and after initiation of treatment with MMF, discontinuation of all pharmacotherapy was achieved within a range of 10 to 30 months in the three patients. One patient experienced a recurrence of genital lesions 19 months after discontinuation of therapy, but the condition remitted within 2 weeks with topical corticosteroid therapy. At the time of this report, the duration of complete remission ranged from 6 to 19 months. In summary, combination therapy with prednisone and MMF for pediatric PV appears to be a safe and effective approach that is associated with durable remission.