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TSCure Research

The next-generation of drugs

The TSA is funding new
research to create a next-

generation drug that could
improve the lives of people
with TSC. We caught up with
Kacper Rogala of the

Whitehead Institute in
Boston, USA, who is carrying What do you do outside of the lab?
out the work.
I absolutely love playing handball as a goalkeeper. The ball is often
thrown at you from five to nine metres, with speeds exceeding
60mph. Cat-like instincts and next-level agility are a must if you
want to do well in this game.
Can you give us a summary of your work? What issues are you trying to solve for TSC I can get really competitive playing handball and always give
patients? 100% on the court. During my time at University College London,
My work is concerned with structural biology and drug
we came second in the British University Handball
discovery. These two disciplines are very closely linked Protein affected by genetic mutations in TSC patients is
and together they can make a real impact for TSC unable to regulate mTOR, so the growth in cells Championships. Then, while at Oxford, we won that title twice in a
row – a feat that has never been accomplished by any other
patients. The full title of my research project is Towards impacted by those mutations is completely out of
specific mTORC1 inhibitors capable of rescuing TSC control. We have drugs on the market that can curb university team.
loss-of-function. the activity of mTOR, but there are two classes of mTOR I am also an experienced yacht skipper and regularly cruise the
in cells and those drugs hit both of those. North, Baltic and Mediterranean
Seas with a group of
So, when you take drugs such as rapamycin over long
friends.
periods of time, you potentially fix the abnormal
growth problems of those cells, but also affect useful Now is the time for
processes controlled by the other class of mTOR us to explore the
molecules, including the balance of sugar levels. This, American waters
of course, is not ideal, because it increases your risk of and perhaps
developing other disorders, including diabetes. attempt the
challenge of
How are you trying to rectify this? crossing the

Work from our lab, and from others around the globe, Atlantic!
has revealed the differences between the two mTOR
classes. Having this knowledge, it is now possible to
work towards creating medicines that will act only on
the mTOR class involved in TSC and not on the other.
We want to be able to develop a next-generation drug
that alleviates all symptoms of TSC and which patients
can take daily without having to worry about Read a full version of this interview at
developing other conditions.
www.tuberous-sclerosis.org

20 Summer 2018 TSA SCAN 21