15% of myasthenia gravis patients see little benefit from existing treatments

Myasthenia gravis (MG) is a “chronic autoimmune neuromuscular disease” that causes muscle weakness due to autoantibodies against proteins in the neuromuscular junction, the connection between the nerves and muscles.

There is a broad spectrum of patients with myasthenia gravis. Some patients can live a normal life because their autoantibodies are well suppressed by medication alone. Others must undergo surgery to remove the thymus gland, a degenerated immune organ, to control the disease.

Yet others are in a life-threatening situation called “myasthenia gravis crisis,” in which the respiratory muscles are paralyzed due to the inability to suppress autoantibodies despite medication and surgery.

Even when it is not life-threatening, the autoantibodies are not suppressed well with steroids and immunosuppressive drugs. Their muscles do not move according to the brain's will, so patients are often forced to leave their jobs or live in seclusion out of despair, or quit their jobs and live a secluded life out of despair, because they see themselves trapped inside their bodies due to their illness

Although myasthenia gravis can occur at any age, it is most common in people between the ages of 30 and 60.

Not having the proper care for people with myasthenia gravis means that it's not just one individual who falls apart, but a whole family and a whole society that can be shaken. And it's not just the patient who suffers when a myasthenia gravis crisis requires prolonged treatment in an intensive care unit. The entire family, including parents, siblings, spouses, and children, suffers and faces financial burdens that will eventually come back to haunt society.

All this explains why it's so important to ensure that any severe, treatable disease is properly treated. Myasthenia gravis is one of those diseases that can be improved.

The recent approval of expensive new drugs in Korea, including complement inhibitors such as ravulizumab and zilucoplan, and neonatal Fc receptor inhibitors like rozanolixizumab and efgartigimod, which directly target the mechanisms of myasthenia gravis exacerbation, has brought hope to patients with myasthenia gravis who previously had no new treatment options.

The problem is reimbursement. None of these drugs are yet reimbursed, which means they are not available to people with MG. who do not respond to existing drugs. On April 19 this year, “Korea Myasthenia Gravis” was officially founded to address this treatment obstacle actively.

“About 15 percent (about 1,500 people) out of the total number of patients (about 10,000 people) with myasthenia gravis cannot be treated well with existing medications,” said Jung Chan-hee, 48, the first president of the organization, emphasizing that “we need to create an environment where these patients can suffer less.”

Jung says this for a reason. In 2011, he was diagnosed with myasthenia gravis at a regional hospital after five months of treatment for ptosis, a drooping eyelid, and in 2013, double vision was added to his treatment. He could continue his work by managing his medication until 2014, when his breathing became increasingly labored, and he quit his job. He was told repeatedly by his doctor that he could die if he had a myasthenia gravis crisis, when breathing becomes difficult.

When even brushing his teeth left him short of breath and unable to spit, he took a taxi and walked into the emergency room. Upon admission, his blood oxygen saturation (normally 95 to 100 percent) plunged despite oxygen therapy. The last time he saw a reading of 17 percent, he fell unconscious and woke up the following year in 2015. After three months in the intensive care unit, he woke up. He was rushed by ambulance to the National Medical Center, where he met his current physician, Dr. Jeong Yeon-kyeong.

He was introduced to Dr. Jeong by Professor Sunwoo Il-nam of the Department of Neurology at Severance Hospital, but his condition was beyond words. He had just woken up from the intensive care unit, was in a confused state of delirium, and his weight had dropped from about 65 kilograms before his myasthenia gravis crisis to less than 40 kilograms. His muscle strength was so weak that he couldn't even hold his neck, and he had a tube up his nose to feed himself, a tube down his throat to provide oxygen, and a urine line.

He recovered quickly under the care of Dr. Jeong, and now he's a different person, one who wouldn't be labeled as a “myasthenia gravis patient” unless he identified himself. In most rare diseases that cause muscle weakness, it's difficult to reverse the damage once it has occurred. However, people with myasthenia gravis have a chance to recover even if their bodies are destroyed to the point of losing the use of their limbs. Jung is a living testimony to this, and he attributes his recovery to “proper treatment for myasthenia gravis and hard rehabilitation.”

The thymoma was visible on his CT scan when he was first diagnosed, but the local hospital missed it. Surgery to remove the thymus gland is the standard treatment for people with myasthenia gravis who have thymoma. Nowadays, thymectomy is used as a treatment for patients with drug-refractory myasthenia gravis caused by acetylcholine receptor (AChR) autoantibodies, even if they don't have a thymoma. After changing his medication, he recovered quickly and walked home with support after six weeks of hospitalization. In 2016, he also had a thymectomy.

The myasthenia gravis crisis never returned. Of course, he has not been without events that have exacerbated his MG symptoms or caused health problems, such as mental stress or side effects from his medication. However, he has been able to continue his treatment with his existing medication and rejoin the workforce. This experience led him to post a phone number on an online site to share his experience with others who have myasthenia gravis, as he didn't want them to face the same problems he did.

This led him to become the secretary general of Korea Myasthenia Gravis. This led to running the Myasthenia Gravis Naver Cafe and a band. This experience led him to the problem of people with myasthenia gravis who have difficulty getting treatment. He felt that there was a need for an organization that could provide financial support to patients who could not receive proper treatment because they could not afford it, and that there was a need for an organization that could help patients who could not get out of their homes because they could not benefit from the new drugs, even though there were new drugs approved in Korea.

Jung thought he could solve this problem by allying with the Korean Organization for Rare Diseases. By joining the larger groups, Korea Myasthenia Gravis could receive information about its various medical support programs, which can then be used to benefit patients who are members of Korea Myasthenia Gravis.

"We also realized that by joining forces with the Korean Organization for Rare Diseases, we would have a stronger voice on the issue of reimbursement for new drugs for myasthenia gravis,” Jung said.

Targeting December of last year, he had prepared to establish the Korea Myasthenia Gravis and planned to visit the National Assembly along with the Korean Organization for Rare Diseases in December to make his voice heard, but everything was put on hold when he was suddenly hospitalized due to a side effect of his medication called electrolyte imbalance, which can cause dangerous situations. This year, however, the Korean Myasthenia Gravis could take its first steps under the Korean Organization for Rare Diseases and already has 130 members, according to Jung.

“What people with myasthenia gravis want most is an improved treatment environment," Jung said in an interview with Korea Biomedical Review. “Patients who don't respond well to existing treatments have a hard time with daily life. Some patients are in a coma, some have poor breathing, and some have good breathing but have difficulty eating. I hope that the new drugs can be supplied quickly so that these patients can be treated better and return to society, and I will do everything I can to help them."