Children’s Hospital & Research Center Oakland have signed a master collaboration agreement with Bayer Healthcare to research hemoglobin disorders like Sickle Cell Disease (SCD) and thalassemia. Scientists based at Bayer HealthCare’s U.S. Innovation Center in San Francisco’s Mission Bay will work with CHORI Senior Scientist and Hematology/Oncology Director Elliott Vichinsky, MD, CHORI Senior Scientist and Co-Director of the Red Blood Cell Laboratory Frans Kuypers, PhD, and other CHORI investigators to utilize promising research approaches to help identify potential new therapies for SCD and thalassemia.
“Children’s Hospital & Research Center Oakland has long been committed to helping this particular patient population,” says Dr. Vichinsky. “With Bayer’s proven expertise in drug development we can help to bring new treatments to the benefit of these patients in a timely manner.”
SCD and thalassemia are genetic blood disorders caused by alternations in red blood cells that decrease their ability to circulate oxygen and nutrients throughout the body. About five percent of the world's population suffers from one of these disorders, and in the US, SCD and thalassemia effect as many as 100, 000 individuals. These blood disorders come with a host of complications caused by the red blood cell alterations, from organ failure to pulmonary hypertension. Currently, however, there are very few treatment options, and the only cure for SCD or thalassemia is stem cell transplantation.
As Dr. Kuypers explains, "The problem with SCD and thalassemia is that every patient is different. Since blood cells are affected, virtually all parts of the body are affected but the degree of these manifestations will be different for different patients. A drug that works for one aspect in one patient may not be that effective in another patient."
“If you are trying to find a drug that works for all aspects in all patients in this population, you could spend your whole life looking for that perfect drug. So we have to find specific approaches for individual patients.”
As a result, there has been little progress to date in the development of SCD- and thalassemia-specific drugs. Add to the equation the rarity of the conditions and the costliness of drug development. Partnering with Bayer HealthCare, however, provides the unique opportunity for progress by utilizing drugs that have been developed, or are in development, for other conditions with symptoms that overlap those of SCD and thalassemia.
“You can treat SCD or thalassemia with drugs that go directly after the changes in the red blood cells, or, in this case, you can go after the harmful effects that those red blood cell changes cause using drugs that have been developed for other conditions,” says Dr. Kuypers. “Whether you are treating the cause of the symptoms or the symptoms themselves in this case doesn’t matter – either way you provide relief to the patients.”
Taking advantage of drugs already manufactured or under development for other conditions means the safety and efficacy of the drugs may already be known, or that the cost of the studies to determine the safety profiles can be leveraged by the fact that there is already an established user for the drug with a different condition.
"We believe that the expertise of Children's Hospital researchers' in sickle cell disease and thalassemia combined with Bayer's expertise in developing therapies for blood diseases will pave the way for innovative research projects that will help improve our understanding in this area and can lead to new treatment options for patients suffering from these rare diseases," says Prof. Dr. Andreas Busch, member of the Bayer HealthCare Executive Committee and Head of Global Drug Discovery. "We believe such collaborations between industry and academia can speed up the development of innovative treatments in areas with high unmet medical need."
Renowned for having one of the best clinical research centers for SCD and thalassemia in the nation, Children's Hospital & Research Center Oakland makes a perfect partnership choice.
"We are uniquely positioned with a clinic that can work directly with the lab to produce translational research studies," says Dr. Kuypers. "We see patients, we can use mouse models, we are the CA state reference lab for hemoglobinopathies. This unique combination creates the ideal environment to explore different treatment approaches in this patient population."