SIMPATHIC
Tiny TIM
Tim is waiting for a cure
Tim is suffering from a rare neurological disease. Just like other patients with rare neurometabolic and neuromuscular disorders, Tim is waiting for medication to cure him, or at least ease his symptoms. Medication development is costly and time-consuming: 12 years at an average cost of 1.2 billion euros before entering the market is no exception. The scarcity of patients with rare diseases like Tim’s impacts the process: trials and studies can take much longer with a lower anticipated return on investment. There simply are less patients that will buy the drug compared to more prevalent diseases. Yet patients like Tim – or Tina or Them – need medication now. What if we were to do things radically differently to speed up the process and repurpose existing medication tailored to Tim’s symptoms?
Disclaimer: Although we use ‘Tim’ and ‘he’ to refer to an imaginary patient to represent all patients impacted by SIMPATHIC, these patients will not all be men. The word ‘Tim’ or ‘he’ should be read as including both men, women (Tiny Tina) and individuals who do not identify themselves as either (Tiny Them).
Research consortium introduces a Tiny-Tim
That is the aim of the SIMPATHIC consortium. We are developing a new approach to expedite the development of existing drugs for groups of patients with rare neurological, neurometabolic and neuromuscular disorders like Tim. From a tube of Tim’s blood or a small piece of his skin, we will obtain Tim’s cells and convert them into one type of brain cells. Let’s call this collection of cells a Tiny-Tim. This group of converted cells is not a clone of Tim, yet a cellular model of one type of brain cells (or an organoid consisting of different types of brain cells) that can be used to test several drugs at the time using highly automated procedures. We aim to test a single drug to cure or ease common symptoms of multiple diseases. For not only Tim can get the best medication he so desperately needs, but this medication might also be suitable for other patients suffering from different neurological diseases with similar symptoms. The Tiny-Tim combined with the highly automated procedures enable the researchers to expedite the matching-making process between the repurposed medication and the impact on disease symptoms.