Support Of Ukrainian Children With Rare Diseases

”Their Lives Depend On The Continuity Of Care”

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Since the invasion of Ukraine began, millions of people have left the country. Among them are also children who need very special support – children with rare diseases. Dr. Krystof Bankiewicz, Chief Scientific Officer for Neurology at AskBio, a wholly owned subsidiary of Bayer AG, and Chief Executive Officer of the Columbus Children’s Foundation (CCF), is a world-renowned expert in neuro-restorative medicine. It was clear to him that the support of the CCF – a nonprofit organization striving to ensure that all kids with ultra-rare genetic conditions are afforded access to state-of-the-art care – is urgently needed. 

In Poland, CCF is now working to make medical treatment immediately available to Ukrainian refugees in urgent need of help, specifically focusing on helping children with rare diseases and their families. We spoke to Dr. Krystof Bankiewicz about the work of his team.


Dr. Bankiewicz, you are not only a pioneer of gene therapies for neurological disorders – you also are CEO of the Columbus Children’s Foundation (CCF). Can you tell us more about the Foundation? 


The Columbus Children’s Foundation began in Europe in 2017 with a mission to ensure that children with ultra-rare disorders are not left behind. 


Through a collaboration between Columbus Venture Partners, Asklepios Biopharmaceutical, and Viralgen, the Foundation has since expanded throughout the US and abroad. Recognizing the potential for gene therapy to solve many of the ultra-rare genetic disorders these special children face, we harness our combined network of scientific experts, technology resources, and affordable manufacturing capacities. 


Together, we are focused on providing financial support to accelerate the advancement of gene therapy clinical trials for ultra-rare genetic diseases and alleviating the burden for children and families to access life-altering clinical trials around the world.


Please tell us about the Foundation’s efforts to care for Ukrainian refugee children with ultra-rare diseases and the current situation they face.


I have a close connection to Poland, and have many friends, family members and colleagues there who are witnessing an unprecedented influx of Ukrainian refugees seeking shelter, medical care and safety. Through these relationships and the advanced rare disease gene therapy work I do with the Interventional NeuroTherapy Center at Brodno Hospital in Warsaw, I know first-hand that many of the hospitals and patient organizations in Poland are trying to provide medical care to a significant inflow of patients while also trying to serve the many children with rare genetic diseases who have very specific medical and nutritional needs.


On-the-ground efforts are taking place to ensure that these refugee children are treated properly with no delay and at no cost. Their  lives depend on the continuity of care; but tragically, in many cases, these children were taken from their homes quickly without their critical daily life equipment, food and medicines, and have no belongings when arriving in Poland.


To give you a few concrete examples of our work:  we are helping provide care and necessities to 10-year-old Nazar and 8-year-old Sergei, who both have Duchenne Muscular Dystrophy and can no longer walk. They fled Ukraine with their families without any medications, mobility supports, or any additional clothing or possessions. Further, we are helping to provide financial support to 18 children with a rare metabolic disorder called Phenylketonuria (PKU) who fled Ukraine without the life-sustaining food and medicines that ensure their medical stability. The food for these kids is highly specialized and delivered at a cost of $50,000 over the next three months of care. 


The CCF is also supporting 12 Ukrainian families with children who have Down Syndrome and need assistance with housing, finances, legal support, and items for everyday care. The daily situation is very difficult for these families and more arrive in Poland every day.


How can people support the CCF’s efforts in Poland?  


CCF is raising funds to ensure these children – and others – have their medical, housing and specialized nutrition needs fulfilled. Along with our Spain-based sister foundation,‎ Fundación Columbus, we have identified a number of local organizations in Poland directly working to help refugee children with rare diseases. We have stepped in to help coordinate fundraising efforts, and through the generous contributions of quick-acting donors, we have already begun providing financial assistance to these organizations.


If people are moved to help these children, you can contribute on our targeted account for Ukrainian Children found at Ukrainian Children – Columbus Children’s Foundation.


We evaluate all requests to ensure we use funds for the greatest and most immediate humanitarian effect. Contributions are focused on organizations that directly support transports and access to medicines for children still in Ukraine, or those that aid Ukrainian refugee children with rare disorders who have fled to Poland who need very specific medical care. 


What is most challenging about advancing treatments and enabling access for children with ultra-rare diseases?


The preclinical and clinical development behind these gene therapy treatments is very challenging and, thus, the cost for these treatments is expensive. Many of the 7,000 known rare diseases have very small patient populations. Pharmaceutical and biotech companies usually focus on larger indications. 

So, this is where CCF steps in. We use a non-profit approach to bring together translational expertise, manufacturing, and patient support to accelerate ultra-rare disease programs to ensure children are not left behind when science and medicine can put a curative treatment in reach. We rely on partnerships with industry, academia, and the patient community to help secure philanthropic funding for these programs which seek to ensure equitable access to children around the world to ensure the barriers to receiving care are reduced or eliminated. 


AskBio is exploring new advances in gene therapy. Which disease areas are you focusing on? 


Our pipeline is the result of patient-focused innovation, rigorous clinical standards, and research collaborations that drive a promising portfolio of gene therapies. We continue to advance therapeutics to bring hope to patients who suffer from neuromuscular, central nervous system, cardiovascular, metabolic, and other disorders.


Central nervous system genetic disorders can be some of the most complex diseases, since many develop later in life. Scientists and research teams at AskBio are collaborating with the world’s leading neuroscientists and medical professionals on CNS disorders such as Huntington’s disease, Friedreich ataxia and Parkinson’s disease. Furthermore, AskBio scientists were among the first to introduce gene therapies for various forms of muscular dystrophy. As the first for-profit organization to receive a grant from the Muscular Dystrophy Association (MDA) in 2004, AskBio’s quest continues to solve the most complex neuromuscular conditions.


And a personal question: You have seen many children with serious and often untreatable diseases – how do you cope with this?


The disorders we see are often devastating. Parents are warriors for their children who have significant and emergent medical needs with a degenerative prognosis. Honesty, humility and understanding is needed when collaborating with parents of these children as they are simply trying to ensure that the moments they have with their child are as good as possible, as they seek to ensure their child gets the care they need and deserve. However, I am renewed by the promise of gene therapy for many of these monogenic genetic diseases and through our clinical trials we are seeing miraculous results first-hand showing that we can dramatically change the lives of these children for the better. I am hopeful that through our experiences and learning we can deliver results like this for hundreds and thousands of other children.