5-Year-Old Diagnosed with Rare Autoimmune Disease to Undergo Bone Marrow Transplant

Paige Neale is a lively 5-year-old girl living with a rare and debilitating autoimmune disease. Image courtesy of Fox News.

5-year-old Paige Neale of Maryland has an autoimmune disease so rare, scientists believe that there are only 100 people on the planet who have ever had it.

The disease is called lipopolysaccharide-responsive and beige-like anchor protein deficiency, or LRBA deficiency for short. The autoimmune disease causes Paige’s body to attack her joints and internal organs, like her stomach and lungs. Paige was diagnosed with the rare autoimmune disease when her parents noticed that she was getting sick a lot more often than her fellow peers her age, including suffering from swollen joints and various GI issues.

Commenting on his daughter’s diagnosis, Mike Neale said: “It’s such a tough diagnosis because you don’t know what to expect…what laid ahead for her, and what do we do next?”

According to the National Institute of Allergy and Infectious Diseases, the disease is caused by a genetic mutation in the LRBA gene. Patients with the condition are highly susceptible to infections, especially of the upper respiratory tract. LRBA deficiency can also cause various symptoms, including poor blood clotting, anemia, weakness, fatigue, joint pain, stiffness, and eye inflammation (uveitis). It can also make patients more susceptible to developing other autoimmune conditions, such as vitiligo, psoriasis, and type 1 diabetes, which cause additional symptoms as well.

For her part, Paige was also diagnosed with the autoimmune condition juvenile rheumatoid arthritis in addition to LRBA deficiency. To keep her alive, Paige receives bi-weekly shots that cost thousands of dollars, but a new procedure is giving Paige’s family hope for the future. Their little girl is to receive a bone marrow transplant in April from her 3-year-old sister, who happens to be a 100% match.

“Right now you look at her, and she looks like a completely healthy five-year-old girl,” said her father Mike. He continued, “We tend to forget how sick she was leading up to that, so we question ourselves: Should we be putting her through this bone marrow transplant that is going to destroy her immune system and make her really sick?”

If the procedure is successful, however, Paige will no longer need to get bi-weekly injections, and she’ll be able to go on to lead a normal life – an end result that her father says is the ultimate goal. Paige enjoys skiing and horseback riding – activities that have given her and her family a sense of normalcy during the COVID-19 pandemic, and throughout her vigorous treatment regimen.

To follow along with Paige’s heartwarming story, check out the Pray 4 Paige Facebook Group.

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.

Study Reveals Increased ADHD Risk in Children Born to Mothers with Autoimmune Disease

Australian researchers have found a potential link between ADHD in children and maternal autoimmune disease. Image courtesy of Kids’ Health.

An Australian study has found a potential link between autoimmune disease and attention deficit hyperactivity disorder (ADHD).

The study took place over a decade, from 2000 to 2010, following more than 63,000 children born at full-term in New South Wales, Australia. Study author Timothy Nielsen, a PhD candidate at the University of Sydney, said that they were able to identify 12,610 mothers who had one or more of 35 common autoimmune diseases, such as type 1 diabetes, celiac disease, Crohn’s, psoriasis, multiple sclerosis, lupus, Sjogren’s or rheumatoid arthritis, to name a few. The children were identified as having a diagnosis of ADHD, or a prescription for stimulants.

The study also included a meta-analysis of existing research on this topic. The combined results of the longitudinal study and the meta-analysis found that when the mother had a diagnosis of any autoimmune disease, [this was] associated with a higher risk of ADHD in their child at later ages.

While researchers don’t know the exact reason why women with autoimmune disorders are more likely to have children with ADHD, researchers do have a hypothesis. It’s believed that maternal autoantibodies, which attack the mother’s own tissues, cross the placenta into the unborn fetus during pregnancy. Inflammatory molecules, therefore, could potentially do the same. These molecules could, in turn, alter fetal brain development, either by altering epigenetic markers, which turn certain genes on or off, or by impacting the function and formation of synapses, which allow nerve cells to communicate.

Nielsen explained, “These changes may lead directly to ADHD symptoms, or they may make the child more vulnerable to environmental risk factors.” He continued, “Our team is currently working on research into the causal mechanisms that underlie the association between autoimmune disease and ADHD, which may shed light on whether the severity of disease, symptoms, use of medications or other inflammatory factors modifies the risk of ADHD.”

This is the first study that explores the correlation between maternal autoimmune disease and the risk of ADHD in children. Other research has shown a link between autoimmune disease in mothers and other neurodevelopmental disorders, such as autism, obsessive-compulsive disorder (OCD), tics and Tourette’s syndrome.

Read the original study published in JAMA Pediatrics here: Association of Maternal Autoimmune Disease with ADHD in Children.

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