Interventional NeuroTherapy Center

Brodno Hospital

Poland

AADCd Gene Therapy Treatment: Poland

Target Area Substantia Nigra Pars Compacta & Ventral Tegmental

(based on GT approach developed under NHI grant, approved by the FDA, 2 US IRB's and constitute experimental treatment to save lives of ADAC deficient children.

Click on the Flag for more information on our sister page: Gene Therapy Trials: USA...

Interventional NeuroTherapy Center, Brodno Hospital, Warsaw, Poland:

This is the first Interventional Neurology Center (INC) to open in Europe and the second in the world. Interventional. Doctors at the Centre perform extremely precise brain surgery operations with "live" imaging.

The only other such facility exists at the University of California at San Francisco, where Polish neurosurgeon Professor Krystof Bankiewicz works. It's sister facility (Poland) has been established as a result of his efforts, with grants mainly from the European Regional Development Fund.

Bródno Hospital VP Wiesław Malicki said that the Interventional Neurology Center had been equipped with the most advanced hybrid room in Europe. It will be used to perform various types of treatments and diagnostic tests. Patients will come from across our continent, and specialists from around the world will operate and treat them.

Professor Miroslaw Ząbek wants to use intraoperative neuronavigation for gene therapy, which can be helpful in the treatment of Parkinson's disease. It is essential to perform the operation in real time because it requires great precision.

"In order to achieve a therapeutic effect, we must introduce viruses containing the appropriate DNA fragment precisely into a specific region of the brain" - said Professor Ząbek.

They build into brain cells and produce neurotransmitters, the deficiency of which causes Parkinson's disease. However, it must be produced exactly where it is needed.

The first such operations were performed in San Francisco by Professor Krystof Bankiewicz, a graduate of the Medical Academy in Kraków. Both Professor Bankiewicz and Professor Zabek, who is one of the most famous neurosurgeons in Poland, have worked together for over 20 years.

Professor Ząbek believes that gene therapy can be used to cure all diseases of the brain, which are caused by a substance deficiency.

"The progress in modern medicine is so large that today we can effectively treat people who not so long ago would die" - said the Speaker of the Senate Witold Karczewski.

Article by Science in Poland PAP

Professor Krystof Bankiewicz MD PhD

Professor Miroslaw Zabek Chair of Neurosurgery Department Neurotherapy Center, Brodno Hospital, Warsaw, Poland

Many thanks to Professor Miroslaw Zabek, the support from the Brondo Hospital in Warsaw and from the Govenors Office of the Masovian State who have made the treatment of AADCd children with Gene Therapy in the EU is possible.

All patients treated in Warsaw have shown clinical improvement.

Title: “Neuroterapi Center - a new gene therapy for the treatment of brain diseases” (Bankiewicz introduction to his polish clinic/lab)

Published: 4/3/2016

Country: Poland

AADCd patients receive Gene Therapy in Europe

LANDMARK moment as Romain, aged 16, from the France, has now received AAV2-hAADC Gene Therapy Treatment in Poland. He is the 3rd young ADULT to receive the pioneering brain surgery.

Teenagers and young adult AADCd sufferers often develop bone deformities, such as scoliosis and contractures, as a consequence of many years of suffering repeated oculogyric crises (OGC’s). Assuming our children survive into adulthood, at some stage they will likely need corrective surgery, offering only short term relief, as their OGC’s will continue and so the cycle will begin again. However, Gene Therapy, is proving to lead to a cessation of OGC’s, which is hugely SIGNIFICANT to future corrective surgeries that these young adults may need. Gene Therapy not only makes them strong enough to withstand such surgery, it also means that it will be a long-term sustainable solution, as the OGC’s are no longer a cause for concern.

So far Paul, Julia, Romain, Yusuf, Huzaifah, Marcus, Swathi, Hallie, Haia, Rian, Aimar, Kacper, Ania, Irai and Ted have all received this potentially lifesaving treatment. This now makes a total of 15 AADCd sufferers receiving this pioneering brain surgery in EUROPE courtesy of Professor Krystof Bankiewicz and his incredible medical team, with more children scheduled in the coming months...

This complex brain surgery, which places this missing AADC enzyme back into the dopaminergic cells via the Substantia Nigra and Ventral Tegmental Area of the brain, has already been performed on 7 x AADCd children at UCSF in the USA, between Jan 2017-Dec 2018, with some potentially incredible ‘life-changing’ results. This treatment has the potential to set the benchmark for many other neurometabolic diseases with a single gene defect and we are very proud to be part of this important journey, which could drastically change the landscape in how we treat many RARE diseases.

We are truly grateful for the extraordinary work carried out by Professor Krystof Bankiewicz and his team and for their dedication to our children. Their commitment to treating our children is exceptional but their pledge to making this affordable through the Columbus Children’s Foundation should not be underestimated.

Kacper : first AADCd patient to receive Gene Therapy in Europe February 2019

Image of Kacper's brain scan during Gene Therapy treatment

"Children have a chance for a new, a better life. I would like for all children with this disease to be able to receive GT treatment and live as close to a normal life as possible, so they are not chained to strollers or beds"

Ela (Kacper's mum)