The past year has been renowned for inspiring medical and health advances, from new research into multiple sclerosis and lupus, to innovative treatments for hair loss and obesity.
The year 2022 has seen several life-changing developments that are already making a difference.
- Pig organ transplantation
Over the past year and a half, researchers have made significant advances in the science of animal-to-human transplantation or xenotransplantation. They have shown that organs taken from pigs genetically modified to more closely resemble humans can appear in the human body without acute rejection. In January, a University of Alabama team became the first to document the transplantation of two kidneys into a brain-dead patient.
This is only the first step toward making xenotransplantation a viable solution to the chronic shortage of donor organs, but there have been recent setbacks. In March 2022, a terminally ill man who became the first living recipient of a modified pig heart died unexpectedly two months after surgery, possibly due to an underlying infection transmitted through the pig heart.
- A new era of hair loss treatment
For several years, some scientists have been investigating whether a class of drugs already used to treat autoimmune diseases, known as JAK inhibitors, could also help with a certain type of hair loss known as alopecia areata. In June, the Food and Drug Administration finally confirmed the results of this work, approving baricitinib as the first JAK inhibitor to treat severe alopecia.
Alopecia areata is thought to affect up to 7 million Americans, and while some people may benefit from existing treatments, there have been no specific drugs so far that address the root cause of the disease (an overactive immune system). People with a severe form of alopecia areata can lose all body and scalp hair. In clinical trials, baricitinib was found to restore a significant amount of hair in about a third of these patients taking the highest dose.
- Disclosing the underlying cause of multiple sclerosis
Multiple sclerosis is a devastating and still poorly understood neurological disease that is estimated to affect about 1 million people in the United States. In January, a large group of scientists published a study that appears to have confirmed the underlying cause of multiple sclerosis: an infection caused by the Epstein-Barr virus.
According to U.S. military medical records, a team of scientists has shown that infection with the Epstein-Barr virus significantly increases the risk of developing MS years later. Only a small percentage of people infected with EBV develop MS, so there are probably other predisposing factors. However, other experts agreed with the group’s findings, which may point to new effective treatments or preventive measures against this debilitating disease, such as the EBV vaccine.
- Drug-producing contact lenses
In March, the FDA approved the first contact lenses that not only help improve vision but also actively grow drugs directly into the eye. The lenses were developed by Johnson & Johnson and are sold as part of the Acuvue brand of disposable contact lenses. They make a commonly used antihistamine called ketotifen, which can help prevent or cure itchy eyes from allergies for up to 12 hours.
The doctors hope that this technology could soon be used to treat more serious eye conditions such as cataracts and glaucoma.
- Possible cure for lupus
In September, scientists from Germany published early research that could herald a new era in the treatment of lupus. In a small study, they found that lupus patients treated for cancer showed sustained remission of the disease for up to 17 months.
This treatment is called CAR T-cell therapy, and it works by modifying a patient’s T-cells in the lab to better target certain cancers and then injecting them back into the body. The researchers hypothesized that these modified T cells could also be targeted against the faulty immune cells that produce the autoantibodies responsible for lupus.
All five patients in the study showed improvement in symptoms after CAR T-cell therapy, as well as complete disappearance of lupus-associated autoantibodies.
- Gene therapy for hemophilia
In November, the FDA approved a first-of-its-kind treatment for hemophilia B, a rare genetic disorder that deprives humans of the ability to properly clot blood. The treatment, now called Hemgenix, is a gene therapy that provides a functional copy of the gene that is disrupted in hemophilia B patients.
Developed by CSL Behring, Hemgenix will not be cheap. The drug is expected to cost $3.5 million per patient. But its creators expect the drug to pay off because it should reduce the frequency of needing expensive infusions of the missing clotting factor. Other gene-based treatments for hemophilia A and B may also soon be on the market.
- 7.Polypill success
Alone, not all medical breakthroughs are necessarily expensive. In August, a three-in-one combination pill for heart disease, also known as polypill, underwent the largest trial.
In a large multinational study among heart attack survivors, polypill, containing aspirin, a common statin known as atorvastatin, and the ACE inhibitor ramipril, showed more heart attacks, strokes, and other cardiovascular events than the standard treatment.
The findings will pave the way for more widespread use of polypills developed by Ferrer and approved in the EU and some other countries under the name Trinomia.
- Treating a deadly genetic disease in utero
In November, doctors in the U.S. and Canada reported a major medical milestone. They were able to begin treating a patient with a deadly genetic disease while she was still in the womb, apparently helping to prevent the deadly complications that killed her siblings.
The patient, a girl named Ayla, was diagnosed with Pompe disease, a rare disease that prevents people from breaking down glycogen in the body. People with early-onset Pompe disease often die or get serious developmental problems within a few years, even if standard treatment is administered soon after birth. But researchers at the University of California, San Francisco, began a small trial to see if the treatment given even earlier in the womb could produce better results, and Ayla was their first patient.
This strategy has shown positive results.
- Cancer-fighting viruses
In September, scientists released the results of the first phase of a clinical trial that may herald a new direction in cancer treatment based on enlisting viruses to fight on our behalf.
The drug is called RP2 and is a genetically modified strain of herpes simplex virus 1, which causes most cases of oral herpes in humans. Developed by Replimune, RP2 is designed to both selectively kill cancer cells and enhance the immune system’s ability to target and neutralize cancer. Of 39 patients with advanced cancer who were treated with RP2 alone or in combination with immunotherapy, 10 patients’ cancerous tumors stopped growing or shrank, and one patient even went into complete remission that lasted at least 15 months.
- Surgical treatment for obesity
In May, Eli Lilly announced the results of a phase III trial of its type 2 diabetes and obesity drug, tirzepatide. People who received the highest doses of the drug lost up to 22% of their baseline body weight, well above the average weight loss seen in those who received standard diet and exercise recommendations. The results of tirzepatide were even greater than those of Novo Nordisk’s analogous drug Wegovy, approved for the treatment of obesity last year and close to the average weight loss seen in the most effective bariatric surgeries.
Tirzepatide has since been approved by the FDA as a treatment for type 2 diabetes (marketed under the trade name Mounjaro).
