Alma's Gene Therapy Treatment in China Shows Positive Progress After Swedish Denial
Alma, a three-year-old with MLD, shows improvements after gene therapy in China, despite initial treatment denial in Sweden.
Key Points
- • Alma was denied gene therapy in Sweden due to ethical concerns.
- • Family raised about 2 million SEK for treatment in China.
- • After nine months, Alma shows stabilization and small motor improvements.
- • Family criticizes lack of transparency from the Swedish Treatment Council.
Alma, a three-year-old girl from Karlstad, Sweden, faced a critical setback in her battle with metachromatic leukodystrophy (MLD) when Swedish healthcare authorities denied her the gene therapy treatment known as Libmeldy. After being told that the treatment was deemed unethical due to her already exhibiting symptoms, traveling to China for alternative treatment became the family's only option. Following a five-month wait that included her diagnosis and subsequent treatment denial, Alma's father, Juan Macall, expressed frustration at the potential irreversible damage caused by the delays, stating, "The tough decisions could have been avoided if there had been access to treatment earlier."
In a remarkable turn of events, nine months after receiving the gene therapy in China, Alma has exhibited signs of improvement, with health reports indicating that her condition has stabilized and she has made notable progress in her motor skills. This positive outcome stands in stark contrast to the grim prognosis provided by Swedish medical authorities. The family engaged in a crowdfunding effort, successfully raising approximately 2 million SEK to facilitate their journey to China.
Alma's family has not only felt the weight of medical challenges but also the pressure of bureaucratic decisions made by the Swedish Treatment Council. Critically, they have expressed concerns regarding the lack of transparency surrounding the council's decision-making process, voicing that they received no substantial scientific rationale behind the denial. Juan Macall highlighted the disconnect between the six individuals who made decisions against Alma's treatment and the over 3,000 supporters who contributed to her crowdfunding campaign, prompting him to say, "While a handful decided on my daughter's fate, thousands rallied to give her a chance."
As Alma continues to recover, her family's journey highlights significant challenges within the Swedish healthcare system regarding access to innovative treatments, restating the pressing need for policy improvement in cases involving rare diseases. Their ongoing story serves as a poignant reminder of how timely medical interventions can significantly alter health trajectories for children facing life-threatening conditions.