Experimental Immunotherapy Puts Lupus into Remission for Young Patient

20-year-old lupus patient Thu-Thao received an experimental treatment which put her symptoms into remission. Story via Autoimmune Warrior.
Thu-Thao (center) received an experimental treatment called CAR-T which put her lupus symptoms into remission.

Thu-Thao was 16 years old when she was diagnosed with systemic lupus erythematosus, a debilitating autoimmune disease that causes a myriad of symptoms, including organ damage, joint pain, fatigue, brain fog, and more. Thu-Thao’s main lupus symptoms were severe joint pain, heart palpitations, kidney issues, hair loss, and skin rashes. She faced life-threatening complications, and as a result, had to drop out of playing sports.

After being diagnosed with lupus, Thu-Thao received a number of conventional treatments over the course of four years, including the anti-malarial drug hydroxychloroquine (the generic for Plaquenil), steroids, biologics, and immunosuppressants. However, none of these treatments were effective and her joint pain and skin problems continued to worsen.

In March 2021, at 20 years of age, Thu-Thao received an experimental immunotherapy called chimeric antigen receptor T-cell, or CAR-T for short. This immunotherapy is typically used on cancer patients, specifically those experiencing aggressive forms of leukemia or lymphoma. This therapy reprograms destructive immune cells in the patient’s body, allowing them to recognize and destroy tumors. 

However, B-cells (the target of the therapy) are also heavily implicated in lupus, in which they create antibodies that directly target double-stranded DNA. The researchers theorized that they could use CAR-T therapy to decrease B-cell numbers in the body, resulting in fewer circulating autoantibodies that cause lupus symptoms. 

Following the therapy, Thu-Thao’s CAR-T cell numbers rapidly increased and remained circulating in her system. The B-cells and autoantibodies in her body—thought to be the cause of the autoimmune symptoms—then began to rapidly deplete as well. Just six months after the treatment, Thu-Thao is in remission from her lupus symptoms, and has returned to playing sports.

20-year-old Thu-Thao is finally experiencing relief from her debilitating lupus symptoms, four years after being diagnosed.

“I can finally breathe properly and sleep through the night, and I no longer have any water retention, and the redness in my face has disappeared. My hair is also growing much more densely,” said Thu-Thao. She is also no longer experiencing heart palpitations: her heart rate dropped from an average of 115 to 130 beats per minute to 80 beats per minute.

The scientists at Universitätsklinikum Erlangen, the German university where the CAR-T treatment was administered, are pleased to see positive preliminary results in a patient with lupus.

“We see this as a milestone in the therapy of autoimmune diseases,” the scientists commented. They are now planning a clinical study with CAR-T cells in more patients with autoimmune diseases.

To read more about this new immunotherapy and the research being done at the Universitätsklinikum Erlangen, read the full article.

9/11 Survivors May Be At Greater Risk of Developing Autoimmune Diseases

Jennifer Waddleton, 51, is suffering from an autoimmune disease after serving as a 9/11 first responder. Image courtesy of NBC news.

Jennifer Waddleton, 31, was working as a paramedic in emergency medical services when she was called to ground zero in New York City on September 11, 2001, after the devastating terrorist attacks on the twin towers. Waddleton is among an estimated 400,000 people who were exposed to toxic debris after the collapse of the towers.

At the time, Waddleton didn’t realize the impact that responding to the event had had on her physical and mental health. Now, however, things are different. She can barely stand for more than 30 minutes at a time or tolerate sunlight. She has brain lesions, her hair is falling out, and her teeth are deteriorating.

“My body is failing me at 51,” said Waddleton, who was diagnosed with cancer, chronic acid reflux, sinus issues, and post-traumatic stress disorder (PTSD). But Waddleton began to experience other symptoms that couldn’t be explained by these diagnoses, including crippling fatigue, chronic migraines, and difficulty swallowing. She knew something wasn’t right.

“In the back of my head, I always knew,” she said. “But everyone was like: ‘No, there’s nothing wrong with you. It’s all in your head. You need sleep, you work crazy hours. Stop complaining’.”

Despite dealing with medical gaslighting for years, Waddleton eventually had kidney failure, and doctors couldn’t deny her poor health any longer. She was diagnosed with systemic lupus erythematosus (SLE) in 2012, 11 years after responding to 9/11. Lupus occurs when the body’s own immune system attacks and damages its organs and tissues.

Before being diagnosed, Waddleton was concerned that her troubling symptoms were somehow related to her experience as a 9/11 responder, and if there were others out there experiencing the same thing. According to several research studies, Waddleton’s concerns are valid; autoimmune diseases do appear to be on the rise among 9/11 victims and first responders alike.

Autoimmune diseases may have been triggered among 9/11 victims as a result of exposure to toxic dust at the scene. Crystalline silica, a construction mineral and major component of the debris, is a noted risk factor for autoimmune disorders. Other chemicals found on-site, like organic hydrocarbon solvents and asbestos, have also been associated with immune dysfunction. A 2015 study found that for every month a first responder worked on the World Trade Center site, the risk of developing an autoimmune disease rose by 13%. A 2019 study based on over 43,000 World Trade Center Health Registry participants found that first responders with intense exposure to the toxic dust were almost twice as likely to develop systemic autoimmune diseases. The most frequently diagnosed autoimmune conditions were rheumatoid arthritis, Sjogren’s syndrome, lupus, myositis, mixed connective tissue disease, and scleroderma.

The same 2019 study also purported that PTSD may also be responsible for triggering autoimmune disorders among 9/11 victims and first responders. This confirms other research on the connection between chronic stress, adverse childhood experiences (ACEs), and autoimmune disease.

Many victims of 9/11 can have their health insurance covered or receive a financial payout from the September 11th Victim Compensation Fund and the World Trade Center Health Program. However, autoimmune diseases are not acknowledged by the compensation fund nor the health program. This means that those who suffer from autoimmune diseases are ineligible for free health care, and cannot receive compensation for their suffering. Most of the covered conditions on the list include acute injuries, lung conditions, cancer, and mental health issues.

Multiple petitions among 9/11 victims have requested to have autoimmune diseases added to the list of covered conditions, to no avail; the federal government has cited lack of sufficient evidence proving the link between autoimmunity and exposures from 9/11. Another issue is that autoimmune diseases may have a genetic component, making it even more difficult to prove that the development of these conditions was caused by exposures during 9/11, and not the patients’ own genetic makeup.

So for now, first responders like Waddleton will have to wait until the research catches up. Waddleton manages a Facebook group for 9/11 emergency responders who have suffered from autoimmune diseases after the event, and has seen first-hand the effects that it’s had on these patients.

“It’s incredibly frustrating,” she said. “They left everyone else hanging. This wasn’t supposed to be my life.”

To read more about this story, visit the NBC news website.

Berkeley Alumni Create Startup Focused on Autoimmune Disease Therapies

Geo Guillen, Marco Lobba, and Matthew Francis, the co-founders of autoimmune disease biotechnology company Catena Biosciences. Image courtesy of Berkeley News.

Marco Lobba was pursuing his PhD in Chemistry at UC Berkeley when he and his lab partners made a discovery. He had been studying the modification of proteins when he happened upon a technique called “oxidative coupling,” which modifies proteins so that they can be fused together. He and his partners also found that the enzyme tyrosinase could be used to make oxidative coupling much faster and more efficient. Tyrosinase is a naturally-occurring enzyme, found in fruits and vegetables, and is responsible for turning apples and avocadoes brown as they ripen.

The accelerated oxidative coupling method could be used to fuse proteins together, faster and more selectively, than any other method currently in use. This opens the door to treating autoimmune diseases, which attack the body by convincing a person’s antibodies to attack their own healthy cells. Using this discovery, scientists can attach ‘safe’ signals to healthy cells, helping the body’s immune system identify its own cells and refrain from attacking them.

“Think of it almost like Pavlov’s dogs,” explains Lobba. “Or tricking children into eating their vegetables by covering them in cheese,” he elaborated. “If you present the immune system with something it likes — at the same time as something it is attacking — it starts to associate that target as a good thing.”

Lobba presented his discovery during a course on entrepreneurship at UC Berkeley’s Haas School of Business. During the presentation, fellow classmate Geo Guillen saw how passionate he was about his research, and the value of his discovery in the treatment of autoimmune disease. It was this purpose that drove the pair to work together alongside Berkeley Chemistry professor, Matthew Francis, to co-found a startup called Catena Biosciences, focused on making autoimmune disease therapies.

Their startup launched remotely during the COVID-19 pandemic, at a time when biotechnology and pharmaceutical companies have come into focus for the role their organizations play in helping to keep our communities healthy and thriving. The startup has been valued at $10 million for its innovative technology and ground-breaking research.

Guillen commented on his company’s founding, saying: “We identified that the autoimmune market is one that is particularly ripe for disruption because a lot of the approaches to treating autoimmune disease focus on the symptoms, instead of the root cause. It’s a pretty large, untapped market.”

Catena Biosciences is aiming to conduct pre-clinical trials by the end of August 2021, which will test the impact of their therapeutics on autoimmune disease reactions in patients. Next month, the company will be looking to raise more funds for their startup to help them commercialize the treatment. The founders’ hope is that they can have a positive impact on those living with autoimmune diseases, like multiple sclerosis, lupus, and Type 1 diabetes.

The company has been awarded the 2021 Berkeley Big Ideas Award for their entrepreneurial endeavors. To learn more about Catena Biosciences, read about the company on the Berkeley News blog.