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Bullous Pemphigoid: 3 Things You Need to Know

Updated on February 28, 2016

Rare skin disease affecting 30,000 Americans receives special attention for Rare Disease Day.

Today, February 29th, marks international Rare Disease Day. Held on the last day of February each year, Rare Disease Day seeks to educate policy makers, researchers, health professionals and the public alike about uncommon medical conditions. According to the National Organization for Rare Disorders (NORD), there exist 7,000 identified rare diseases, many of which lack FDA-approved drug treatment.

One such uncommon disease that has garnered particular attention on this year’s Rare Disease Day is bullous pemphigoid (BP). In an effort to raise awareness for this severe and disabling skin condition, The International Pemphigus & Pemphigoid Foundation has announced its support for Rare Disease Day and hopes to push education about BP to the forefront.

According to NORD, a disease is considered rare if it afflicts fewer than 200,000 individuals in the United States. Bullous pemphigoid affects 30,000 Americans and is an autoimmune skin disease that manifests with itchy skin blisters, particularly on the thighs, armpits or abdomen, which break open and can leave painful areas of raw skin. The disease will typically wax and wane over a period of years and require chronic meditation to control the symptoms.

Although the condition is generally found in patients over the age of 65, the large generation of young people today that will age simultaneously and grow into a substantial elderly population will likely increase the number of people living with BP. In light of Rare Disease Day and bullous pemphigoid potentially on the rise, here are three things you need to know about this potentially severe condition.

We don’t know what causes bullous pemphigoid.

In an autoimmune disease, antibodies that are supposed to ward off foreign invaders and fight against threats to a person’s health are instead mysteriously produced to combat normal bodily functions. In other words, the body attacks itself and begins antagonizing its healthy organs. In the case of bullous pemphigoid, antibodies are produced against the outer and immediately underlying layers of skin, resulting in blisters that form in between the two layers. While researchers have suggested that exposure to UV light and the intake of certain drugs may contribute to the onset of bullous pemphigoid, like most autoimmune diseases, the true cause is unknown.

Diagnosis is difficult and treatment can lead to severe side effects.

Because bullous pemphigoid is so uncommon and can be difficult to diagnose, patients often have to visit several doctors before receiving the correct diagnosis. This means that people are living with BP’s uncomfortable side effects for long periods of time without relief from the symptoms. Even once the disorder is detected, the most common treatment plan involves oral corticosteroids that have numerous undesirable consequences.

“Many patients require high doses of drugs that put them at risk for toxicities and serious side effects that may be disabling, rarely even fatal, in this elderly population,” said Dr. Neil Korman, Professor of Dermatology at University Hospitals Case Medical Center in Cleveland, Ohio. Creams can also be inconvenient and inadequate for those who have widespread rashes across their body. Finally, extended steroid intake weakens bones and the immune system, ultimately expediting mortality.

New research is brightening the future for BP patients.

In an effort to address the need for a better bullous pemphigoid treatment, researchers are testing a new biological drug called bertilimumab. By blocking the activity of a pro-inflammatory protein known as eotaxin-1, bertilimumab is expected to relieve clinical symptoms and inflammation in diseases such as ulcerative colitis, Crohn’s disease and atopic dermatitis, all with minimal side effects. Most notably, it can reduce the blisters that plague sufferers of bullous pemphigoid.

Immune Pharmaceuticals, Inc. has received FDA approval to test bertilimumab in clinical trials in bullous pemphigoid. “We have received strong interest from several top academic centers to enroll patients in our phase II bullous pemphigoid clinical trial,” said Dr. Monica Luchi, MD, Chief Medical Officer at Immune. “We anticipate data in 2016 which will support further development.”

During the ongoing phase II of bertilimumab clinical testing, Immune will administer the drug to a group of 10-15 patients and closely monitor the medication’s efficacy and side effects. Results from the study and an Orphan Drug Designation from the FDA are anticipated in 2016. Pending results of the phase II study and regulatory feedback, clinical development will continue towards supporting registration and marketing approval.

Despite the challenges in diagnosing and treating this rare disease, patients with bullous pemphigoid can be optimistic. With companies such as Immune Pharmaceuticals committed to finding an answer, these patients and their relatives can look to the future with hope.

****Makena Owens contributed to this article.

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