FDA Approves Drug to Treat Rare Autoimmune Disease

The U.S. Federal Drug Administration (FDA) has approved a drug manufactured by ChemoCentryx to treat a rare group of autoimmune diseases, called anti-neutrophil cytoplasmic autoantibody-associated vasculitis, or ANCA-AAV for short. According to ANCA Vasculitis News, ANCA-AAV causes inflammation and damage to the body’s small blood vessels. This inflammation is the result of antibodies that bind to certain cells of the immune system, called neutrophils, and overly activate them.

Since small blood vessels are found throughout the body, ANCA-AAV causes a variety of symptoms, including:

  • Poor kidney function, leading to kidney failure
  • Severe respiratory problems, including shortness of breath, hoarse voice, cough with blood or mucus, and chest pain
  • Neurological symptoms, including tingling, burning, numbness, and weakness
  • Central nervous system (CNS) involvement, such as headaches, cognitive impairment, and memory deficits. In severe cases, seizures, paralysis or loss of consciousness may also result.
  • Ear, nose, and throat (ENT) symptoms, like sinusitis, nasal discharge, rhinitis, and tinnitus (ringing in the ears)
  • Eye symptoms, including eye pain, vision impairment, and vision loss
  • Joint pain, muscle pain, and muscle loss
  • Skin lesions, including rashes, sores, ulcers, bumps, and bleeding underneath the skin
  • Digestive problems, like vomiting, diarrhea, nausea, abdominal pain, and blood in one’s stool
  • And more…

Due to the numerous symptoms ANCA-AAV causes, it can often be fatal for patients; in fact, ChemoCentryx states that the first year mortality rate for patients living with the disease is between 11 and 18 percent. Current ANCA-AAV treatments on the market include steroids and immunosuppressant medications which compromise the body’s ability to fight off infections. That’s why having a new treatment on the market that works via a different mechanism may be a saving grace for many ANCA-AAV patients.

The new drug, called avacopan, will be sold by ChemoCentryx under the brand name Tavneos. It works by blocking a protein called C5a receptor that is responsible for causing numerous inflammatory diseases. The drug’s wholesale price will be an astronomical $150,000-$200,000 per patient per year. However, it could be the life-saving treatment that the 40,000 ANCA-AAV patients in the US need.

The drug is currently being tested for use on other conditions as well, including the autoimmune skin disease Hidradenitis Suppurativa (HS). The drug has already received approval for commercialization in Japan, and is expected to be approved in Europe by the end of 2021.

Martina, a 29-year-old mother-of-two from Stuttgart, Germany, describes what it was like being diagnosed with this challenging autoimmune condition:

“It began rather unspectacularly, with flu-like symptoms, fever, headaches, and joint pain. So anyone would think, ‘It’s just a normal flu.’ Then it got worse over the course of a week and I went to the hospital.”

She was eventually diagnosed with ANCA-AAV after undergoing a series of blood tests. Unfortunately, she had to give up her career as an educator in the process, since she didn’t have a strong enough immune system to be interacting with children while taking steroids and immunosuppressants to control the disease. Patients like Martina are the ones that could potentially stand to benefit from new drug therapies like avacopan.

Martina, 29, appears in an ad to raise awareness for vasculitis conditions.

To find resources for those living with ANCA-AAV and their caretakers, visit: https://www.myancavasculitis.com/living-with-aav/

Christina Applegate Reveals Multiple Sclerosis Diagnosis

Actress Christina Applegate has revealed that she has MS, a neurological autoimmune disease. Photo courtesy of Mike Coppola via CNN.

49-year-old actress Christina Applegate revealed on Twitter this week that she has been diagnosed with multiple sclerosis (MS), an autoimmune disease affecting the central nervous system. Applegate says she was diagnosed “a few months ago” after experiencing symptoms of the disease.

Commenting on her diagnosis, she said: “It’s been a strange journey. But I have been so supported by people that I know who also have this condition. It’s been a tough road…but as we all know, the road keeps going.”

According to John Hopkins Medicine, multiple sclerosis occurs when the immune system attacks nerve fibers and the myelin sheath – a fatty substance which insulates healthy nerve fibers – in the brain and spinal cord. This attack causes inflammation, which destroys nerve cell processes and myelin, altering electrical messages in the brain.

There are different types of MS, the most common of which is relapsing-remitting multiple sclerosis, which affects 90% of those diagnosed. Symptoms of a multiple sclerosis relapse include: fatigue, numbness, tingling, blurred vision, unsteady gait, and weakness.

Worldwide, more than 2.3 million people live with MS, including almost 1 million adults in the United States alone, according to the National MS Society. The neurological autoimmune disease can be disabling, although the MS Society states that the majority of people with the condition do not become severely disabled. Two-thirds of people who have MS remain able to walk, though they may need a mobility aid, such as a cane, and some will use a scooter or wheelchair because of fatigue, weakness, balance problems, or to assist with conserving energy. 

Since coming out as newly diagnosed with MS, Applegate has received an outpouring of support from fans and other celebrities with the disease. Fellow actress Selma Blair, who co-starred with Applegate in a romantic comedy in 2002 and also has multiple sclerosis, tweeted: “Loving you always. Always here. As are our kids. Beating us up with love.” Talk show host Montel Williams, who also has MS, also tweeted his support: “We have MS – it will never have us unless we let it. Tara and I are sending hope and light your way.”

MS isn’t the first health battle Applegate has faced. In 2008, the star revealed that she had had a double mastectomy after testing posting for the BRCA gene, pre-disposing her to breast cancer. Facing her new MS diagnosis, Applegate has requested “privacy…as I go through this.”

3 BioTech Companies You Can Invest In to Fund Autoimmune Disease Research

At Autoimmune Warrior, we believe that scientific research and development holds the key to unlocking new, innovative treatments and ultimately, a cure for autoimmune disease. In today’s blog post, we’ll explore three different biotechnology companies that are pioneering research about autoimmune diseases.

Why should companies research autoimmune diseases?

According to the American Autoimmune Diseases & Related Disorders Association (AARDA), there are over 100 different types of autoimmune diseases affecting 50 million people in the US alone. This demonstrates that autoimmune diseases are one of the most prevalent conditions nationwide. Furthermore, the AARDA reports that autoimmunity is one of the top 10 leading causes for death among American women. These figures show the high impact that medical research could have on autoimmune patients.

There is, of course, a financial incentive for biotech companies as well. A Research & Markets report indicated that as of 2017, the global autoimmune disease therapeutics market was estimated to be worth over US$109 billion. This figure was projected to grow to US$153 billion by 2025. Part of this growth has been attributed to the rise in autoimmune diseases among the general population and specific groups; although it’s been argued that medical professionals are becoming more aware of autoimmune conditions, and therefore, are simply getting better at diagnosing patients.

How can I help fund research & development?

If you or someone you love suffers from an autoimmune condition, you’ll know how important it is to find effective treatment options. As a result, you may consider investing your hard-earned dollars in companies that are pioneering autoimmune disease research. Below are three companies that I have personally researched that are contributing to this cause.

1. Landos Biopharma

Landos Biopharma is a Virginia-based company started by former Virginia Tech inflammation & immunology professor Josep Bassaganya-Riera, PhD. Landos is considered to be clinical-stage biopharmaceutical company focused on the discovery and development of oral therapeutics for patients with autoimmune diseases. In particular, the company is developing therapeutics for those with autoimmune diseases of the gastrointestinal tract, such as ulcerative colitis (UC) and Crohn’s disease.

Landos, which was founded in 2017, is a publicly-listed company on the NASDAQ stock exchange under the ticker symbol LABP. As of writing, the stock sits at just above US$12 per share.

2. UCB

UCB is a Brussels, Belgium-based multinational company, with a long history of research and development in the area of immunology. Some of the company’s autoimmune disease research areas include: rheumatoid arthritis, psoriasis, Crohn’s disease, lupus, and myasthenia gravis (MG). The company actively works with clinics worldwide to recruit for clinical studies with autoimmune patients; some of the studies they are actively recruiting for at the time of writing (June 2021) include patients with hidradenitis suppurativa, lupus, psoriasis and myasthenia gravis.

UCB is a 90-year-old company, and is publicly listed on the EBR stock exchange under the ticker symbol UCB. As of writing, the stock sits at just above 85 euros per share.

3. Abbvie

Abbvie is a Chicago-based multinational company that was spun off from Abbott Laboratories. Abbvie has been striving to advance the standard of care in rheumatology for more than 20 years. The company says that they are focused on developing therapeutics for patients with chronic diseases, which is said to account for 75 percent of all healthcare costs. Some of the company’s autoimmune research areas include: rheumatoid arthritis, systemic lupus erythematosus, psoriasis and multiple sclerosis. The company has already developed a number of well-known anti-inflammatory treatments, including HUMIRA (adalimumab).

Abbvie was spun-off in 2013, and is a publicly-listed company on the NYSE under the ticker symbol ABBV. As of writing, the stock sits at just above US$114 per share.

Would you consider investing in these biotech companies? Why or why not? Let us know in the comments below!

Disclaimer: This blog post is not intended to provide financial advice, but to raise awareness about companies conducting research & development towards advancing autoimmune disease therapeutics. Always consult with your physician before beginning a new treatment plan.