According to a story from Buzzfeed News, the ongoing coronavirus/COVID-19 pandemic has put the brakes on critical rare disease research all over the globe, ranging from early, highly experimental studies to large-scale trials that involve hundreds of rare disease patients. Unfortunately, the delays are putting the lives of patients in jeopardy in some cases.
Maxwell Freed
Maxwell Freed is a three year old boy living with a very rare disorder known as SLC6A1. Despite the fact that there are only about 50 known patients, progress was being made on studying the disorder. Much of the research efforts were being funded largely thanks to the diligent efforts of his parents. At the current pace of development, the Freed’s were hoping that he would undergo a trial testing an experimental treatment in a year or so. But the pandemic has ground the project completely to a halt. Before long, progression of the disease will prevent him from ever experiencing the potential benefits of treatment. It will be too late.
AJ Henwood
Meanwhile, Nicole Henwood of NF2 BioSolutions says that the pandemic may also seal the fate of her son AJ as well. As a patient living with neurofibromatosis type 2, he deals with tumors on spine and nerves, as well as others that have appeared on the blood vessels in his brain, threatening his survival. NF2 BioSolutions was in the early stages of researching and developing a potential gene therapy for the disease, but the states where this research was happening are still under lock down. The problem is exacerbated by the fact that a huge swathe of medical funding has now shifted towards studying the coronavirus as the scientific community desperately scrambles to develop a vaccine and find a worthwhile treatment.
COVID-19: Time is Essential
Some organizations have shifted to new strategies for fundraising, such as through online game tournaments (since participants don’t have to leave home) instead of basketball or baseball tournaments. However, many families are stuck waiting for things to return to normal while rare disease patients see disease progression. In this field of research and medicine, time is as precious as life itself, and now it’s slipping away.
