Between a groundbreaking medical research achievement and its implementation in clinical practice often pass not only years, but decades. Time that people with a serious disease do not have - especially not if it is a rare disease. These diseases, most of which are genetically determined, usually break out in childhood and often lead to irreparable cognitive and physical damage or death within a few years.
It therefore makes sense to create institutions that are concerned with making basic research usable for patients as quickly as possible. The Berlin Institute of Health is one such center, headed by Professor Christopher Baum. As a "physician who wanted to do research," he was already involved in molecular genetics and its applicability for therapies in the 1990s. This specialization led him more or less automatically to rare diseases: "If there's anywhere you can intervene in the development of a disease, it's in genetic diseases," says Baum.
What sounds ingenious from the point of view of the affected person, that a defective gene can be replaced by a healthy one and thus cure a disease, is in practice a highly complex process. First, because of the sheer volume of potentially affected genes and thus different ailments. "The biggest challenge with rare diseases is their enormous diversity," Baum says, noting that 6,000 to 8,000 have been defined so far, but the physician expects the number to increase in the coming years. "That's where you first have to find scientists to work on it."
On the other hand, there is a need for targeted networking of researchers and physicians working on patients. "We believe that knowledge can only become applications at all if you work together in large networks, if you are prepared to go beyond the boundaries of your own laboratory," Baum explains. Not only to test therapeutic approaches, but also because of the technical complexity involved in establishing treatment options in clinical practice: "Bringing something into application is so multifaceted that you have to take many different parallel steps. If you worked through them one after the other, it would take far too long. If you can coordinate multiple researchers, you can shorten the gap from knowledge gain to application by years, creating hope for new therapies faster."
Another important aspect, in Baum's view, is far too often overlooked to this day, the interaction with industry. "In fact, there is no therapy at all that can come into use without the involvement of private industry. In the end, you always need biotech or pharma, and that's in private hands. That's where entrepreneurship is needed, far-sighted thinking, a willingness to take risks, but also just a lot of technical know-how." So you can't turn research into health without broad alliances.
