Treatments for childhood cancer might first conjure up images of chemotherapy, radiotherapy or surgery. But so-called such as CAR-T cell therapy are fast gaining ground. They work by changing a child’s cells in the lab to equip them better to fight cancer. For example, immunotherapy uses the body’s own defense mechanisms to kill cancer cells.
‘Drug-based therapy often comes with debilitating side effects, whereas cell therapies have the potential to be much smarter forms of treatment,’ says Adeel Saleem, who started at the Princess Máxima Center in September. ‘It’s a relatively new development, but it’s rapidly advancing. We’re seeing the promise of cell therapies come true for many children with cancer.’
In-house cell manufacturing facility
To help further unlock the promise of these new treatments, the Princess Máxima Center, is building a dedicated cell therapy facility for manufacturing advanced therapy cell-based products. In the facility, specialist scientists will work to develop and manufacture cell therapies ready to be given to children with cancer. According to Saleem, a fundamental reason for building an in-house facility is that childhood cancers are rare, and for some forms there are no alternative commercially available cell-based treatments. ‘Manufacturing cell therapies locally helps us meet that smaller-scale demand and focus on the clinical needs of the individual child. It will also speed up translational research and help bring new developments to patients more quickly.’
The cell therapy facility will be host to a specialist team of people to safeguard the quality and production of advanced cell therapies. As quality and safety is also of the utmost importance in preparing conventional medicines, the facility is linked to the pharmacy at the Princess Máxima Center. ‘Biological therapies are different from ‘regular’ medicines, as they are living cells. That makes them less predictable than pharmaceuticals,’ Saleem explains. ‘That’s why we must follow stringent regulations when manufacturing cell products for therapy.'
A bridge between research and therapy
Since 2019, one form of biological therapy, CAR-T cell therapy, has been a standard part of treatment for children with acute lymphoblastic leukemia (ALL) for whom other therapies no longer work. At the Princess Máxima Center, scientists aim to make CAR-T work for more children with ALL. They are also looking at ways of making CAR-T and similar cell-based therapies available for children with other forms of cancer.
‘I see my role as a bridge between different groups that want to translate research into a clinical therapy,’ says Saleem. ‘For a large part, we’ll be doing translational work. We will look at new candidate cell therapies that research groups in the Máxima Center come up with in the so-called pre-clinical phase. In our cell therapy facility, we will then optimise, scale up and prepare them for translational into a clinical therapy in line with EU standards, so the cells are ready to be given back to children in the form of cancer treatment.’
From the outside, the new sixth and seventh floors on top of the research building are already taking shape. An essential task for Saleem in his new position as head of the cell therapy facility is to finalize the plans for the interior. ‘To create a future-proof facility, we need to anticipate: what technology and expertise we will need in five or ten years. Cell therapies hold enormous promise for childhood cancer. It’s very special to have joined the Princess Máxima Center and to be pioneers in such a rapidly developing field.’