The Scan by 2 Minute Medicine® is a pop-culture medical newsletter and exclusive benefit for 2 Minute Medicine Plus subscribers.
We begin with a discussion about a new gene therapy to treat sickle cell disease. Then we take a closer look at how lupus can affect motherhood. After that, we examine short- and long-term effects of hurricanes on health. Finally, we discuss Rafael Nadal’s recent retirement and consequences of sport injuries.
One-Stop Shop for Sickle Cell Disease:
The Story: Kendric Cromer, a 12-year-old boy from Washington, D.C., became the first person outside of clinical trials to receive Lyfgenia, a one-time gene therapy to treat sickle cell disease.
Kendric Cromer, a 12-year-old boy from Washington, D.C., spent many days of his childhood in the hospital, with pain so severe even morphine and oxycodone could not control it. Fortunately, he may be on the path towards reclaiming his childhood and enjoying ordinary activities like basketball, swimming, and playing in the cold – pain-free.
What is Sickle Cell Disease?
Kendric is one of 100, 000 people in the United States and 8 million worldwide with sickle cell disease (SCD), an inherited blood disorder that disproportionately affects people of African descent. In SCD, mutations create abnormal hemoglobin molecules, a protein essential for red blood cells (RBC). Consequently, “sickle cell” RBCs adhere to each other and to blood vessel endothelium, disrupting microcirculation and causing vascular occlusion and subsequent tissue infarction. This can cause extreme pain and high mortality events such as acute chest syndrome, where the pulmonary vasculature is occluded. Unfortunately, even ordinary activities such as basketball and playing in the cold can trigger sickle cells to adhere. Tionne Watkins, a 4-time Grammy Awards winner, spent 3 days unconscious in the intensive care unit after breastfeeding her newborn for the first time. Ramon Reed, an American actor, has undergone 3 laser eye surgeries to repair complications of sickle cells restricting blood flow in his eye vessels. Unfortunately, SCD has no cure, only temporary symptom relief.
How does Lyfgenia work?
A new therapy developed by Bluebird Bio, Lyfgenia, may be the breakthrough that provides lasting improvement. Lyfgenia is a one-time gene therapy made from the patient’s blood stem cells that induces the body to produce normal, functional copies of hemoglobin. In its clinical trials, Lyfgenia prevented vaso-occlusive events and severe vaso-occlusive events in 88% and 94%, respectively, of patients 6-18 months after treatment.
What does the future hold?
However, two barriers prevent Lyfgenia from being widely available. First, price: this therapy costs $3.1 million, one of the highest prices ever for a treatment. Second, the process involves stem cell collection, modification, chemotherapy, and a lengthy hospital stay to monitor for complications. All in all, this procedure can take months and there are only a few hospitals around the world that have the facilities and personnel to do so. In fact, Bluebird estimates they can only treat 85-105 patients per year. In the meantime, Kendric remains an inspiration for the hopeful. He aspires to be a geneticist; Lyfgenia may just offer him the life to fulfill those dreams.
Lupus and Losing Motherhood:
Selena Gomez, the 32-year-old American singer, has been candid about her struggles with lupus since her diagnosis in 2013. She has shared her journey through chemotherapy and a kidney transplant to address complications, as well as her experience with psychological side effects such as depression and anxiety. Recently, Selena shared that she can’t carry her own children, partly due to this chronic disease that affects 1.5 million Americans and at least 5 million people around the world.
There are 4 types of lupus, with systemic lupus erythematosus (SLE) being the most common. It is a multisystem autoimmune disease that predominantly affects women of childbearing age. It can disturb any organ, although the skin, lungs, kidneys, and cardiovascular system are commonly involved. Although women with lupus can still carry children, the disease itself and the drugs used to manage it increase the risk of maternal and fetal complications. For example, immunosuppressants dampen the autoimmune response seen in SLE but commonly used immunosuppressants such as cyclophosphamide may cause ovarian failure, and consequently, infertility. Pregnancy can also cause SLE flare-ups which increase adverse pregnancy outcomes such as fetal loss, pre-eclampsia, and thrombosis.
Lupus awareness is gaining traction worldwide thanks to the efforts of Selena and other celebrities with the disease such as Halsey and Nick Cannon. The Lupus Research Alliance, the largest non-profit funder of lupus research worldwide raised millions at their New York City Walk with Us to Cure Lupus event on October 19. Although the exact etiology of lupus remains unsolved, increasing awareness and continued research offer hope that this illness will one day be cured.
Hurricane Danger:
Hurricane Milton, a Category 5 hurricane with maximum sustained wind speeds of 120 mph and up to 18 inches of rain, made landfall in Florida on October 9. As of October 17, the storm has cost 24 lives and caused damages of over $30 billion. The tremendous rainfall and accompanying storm surge is historically the leading cause of hurricane-related deaths. However, hurricanes cause short- and long-term effects on medical infrastructure, public health, and individual socio-psycho-environmental stress.
Damage to agriculture and manufacturing facilities releases chemicals and pathogens into the environment, increasing the risk of infectious diseases. Flood damage to homes increases mold growth and leads to occupant exposure when they return. A 2016 study found increased respiratory symptoms in children were associated with mold exposure after Hurricane Katrina. Furthermore, power outages and flooding interrupt healthcare delivery as they force hospitals, pharmacies, dialysis clinics, and other healthcare facilities to close. Hurricanes increase rates of acute illness and exacerbate existing conditions as patients with chronic conditions are unable to properly manage their conditions. Finally, studies have shown hurricane exposure elevates levels of post-traumatic stress disorder, depression, and anxiety.
The 2024 Atlantic hurricane season has already claimed the lives of over 300 people. However, Florida continues to enjoy heavy migration, with around one million new residents in 2023 and 2022. Natural disasters don’t pick and choose, and celebrities such as Tom Cruise, an American actor, and Stephen King, an American author, own properties in Florida and braced for Hurricane Milton. Other unaffected celebrities such as Taylor Swift, an American singer, and actress and actor couple Blake Lively and Ryan Reynolds have contributed millions towards the Hurricane Milton relief fund.
A Tennis Legend Retires:
Rafael Nadal, the 38-year-old professional tennis player from Spain, announced on October 10 that he will retire at the end of the year, cementing his legacy as one of the greatest players ever. He will depart the sport with 22 Majors championships (including 14 French Open titles and a career French Open record of 112-4!!), good for second all-time. This is even more impressive considering his career has been plagued with injuries and surgeries. From chronic tendinitis to fractures to an abdominal tear, Nadal’s injuries have cost him nearly as many Majors (16) as he’s won.
There is no doubt sports are beneficial for an individual’s health. A large systematic review in 2019 found physical activity decreases atherosclerotic disease, cardiovascular disease, and all-cause mortality. However, injuries, particularly in one’s youth, and poor rehabilitation post-injury can lead to chronic, negative health outcomes. Fractures that disturb physeal growth may cause long-term disability through angular deformity, altered joint mechanics, and limb length discrepancy. MRI studies of young athletes in sports that place intense physical loads on the spine, such as diving, have shown an increased proportion of thoracolumbar spine abnormalities. Contact sports such as football may pose an even greater risk through head trauma. A systematic review found multiple concussions may be a risk factor for future cognitive impairment and mental health problems.
While Rafael Nadal enjoyed a 23-year career, other professional athletes haven’t been as lucky. Greg Oden, the first overall pack in the 2007 NBA draft only played in 105 of a possible 574 games over a 7-year career due to chronic knee injuries. While Shohei Ohtani remains one of the best players in baseball, the once-in-a-generation superstar hasn’t pitched since the 2022-2023 MLB season after a second Tommy John surgery to repair his torn ulnar collateral ligament. Physical activity is necessary for normal growth in children and healthy living for everyone. However, it is equally important to avoid overuse, recognize injuries early, and manage them appropriately.
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