By Iris Jacobs, MD
It’s difficult for most of us to fully appreciate what it’s like to have a rare disease caused by a deficient or defective protein. Traditionally, patients with certain types of protein deficiency or defect had no option other than to be intravenously injected two to three times per week with conventional therapies.
While frequent injections (prophylaxis) may prevent the onset of symptoms in most instances, this approach is a burden for patients who lead busy lives. It is also the main reason that routine prophylaxis is often delayed in young children. In addition, some symptoms can cause progressive and in certain patients debilitating damage.
At CSL Behring, innovation is a long-standing tradition, and since our start 100 years ago, we have continually sought to deliver on our promise to address unmet medical needs and enhance treatments for our patients. Our scientists were the first to use pasteurization, and in 1981 we introduced the first pasteurized coagulation factor product that transformed how the industry produced plasma products.
In keeping with this tradition, scientists at CSL Behring undertook the challenge to develop a better treatment option for patients with certain medical disorders that would allow for prolonged dosing intervals while protecting against progressive and debilitating damage. Because of its long physiological half-life of approximately 20 days, we found recombinant albumin to be an ideal genetic fusion partner for some of our therapeutic proteins.
After years of basic research and development and a robust global clinical program involving five studies, CSL Behring successfully developed a recombinant fusion protein that, when linked with coagulation Factor IX, is used to treat congenital FIX deficiency. Currently, multiple clinical trials are ongoing to test a recombinant Factor VIIa albumin fusion protein for treating congenital hemophilia with inhibitor and factor VII deficiency.
The clinical effect of this fusion protein, depending on the chosen dose and the patient’s clinical profile, makes it easier for patients to plan their prophylaxis. As fusion protein requires fewer injections, its use may also enhance compliance with prophylaxis therapy and facilitate starting routine prophylaxis in young children with a certain medical disorder without implantation of central venous access devices, which are often necessary for home injection by parents.
Often, patients with certain disorders are advised to dose in the morning in order to get the most coverage out of their therapy. However for most people, mornings are a hectic time of day. Trying to inject a child and get out the door to school or work on time can be difficult for families. Using an albumin fusion protein may solve this common challenge, as it provides prolonged coverage and does not need to be dosed in the mornings.
This flexibility can benefit patients across all age groups. Collectively, these clinically meaningful benefits are a major contribution to patient care. The treatment paradigm allows patients with a particular condition to live a more normal life while requiring less frequent dosing.
As with any medicine, a large number of people played a major role in the development of the recombinant fusion protein. Dedicated efforts include those of my colleague Dr. Debra Bensen-Kennedy, who provided invaluable leadership and guidance. In addition, the study team, including investigators from all over the world, helped make this innovative biotherapy possible. Of course, we also sincerely appreciate those patients who trusted us and participated in the clinical program.
For patients with unique circumstances that require individualized care, innovations such as recombinant fusion proteins translate into relative freedom from their disease, which is a life-changing experience. Or, as one young patient put it, “I didn’t want to be told, ‘no’ or ‘you can’t,’” something many patients with a rare disease hear from a young age. Perhaps, with CSL Behring’s innovative therapies, this patient and others like him will have a future of endless, unrestricted opportunities.
Dr. Iris Jacobs is CSL Behring’s Senior Global Clinical Program Director in Clinical R&D, Clinical Lead for PROLONG-9FP clinical program