Thursday, February 15, 2024 – A 13-year-old boy from Belgium has become the first in the world to be cured of a deadly brain cancer.
Lucas Jemeljanova at the age of 6 was diagnosed
with diffuse intrinsic pontine glioma (DIPG), a very rare and highly aggressive
brain tumor that kills 98 percent of sufferers within five years.
According to Mail Online, he was randomly assigned to
receive everolimus, a type of chemotherapy drug, in a clinical trial, which is
used to treat kidney, pancreas, breast, and brain cancer but has not been used
successfully to treat DIPG.
His parents, Cedric and Olesja, took him
to France to be one of the first enrolled in the BIOMEDE trial, which
was testing potential new drugs for DIPG.
Everolimus works by blocking mTOR, a protein that helps
cancer cells divide and grow and produce new blood vessels. This stops or slows
down the growth of the cancer by preventing the cancer cells from reproducing
and by decreasing the blood supply to the tumor cells.
Doctors were afraid to stop the treatment regiment until a
year and a half ago, by which point it turned out Lucas had stopped taking the
drugs anyway.
Dr Grill said: 'I didn’t know when to stop, or how, because
there was no reference in the world.'
'Over a series of MRI scans, I watched as the tumor
completely disappeared,' his doctor, Jacques Grill, head of the brain tumor
program at the Gustave Roussy cancer center in Paris, told AFP.
It was revealed that seven other children in the trial have
been considered 'long responders' after they had no relapses for three years
after their diagnosis, but only Lucas's tumor totally disappeared.
The reason for some children responding to the drugs while
others did not is probably because of 'biological particularities' of their
tumors, Dr Grill said.
'Lucas's tumor had an extremely rare mutation which we
believe made its cells far more sensitive to the drug,' he said.
Roughly 300 children a year are diagnosed with DIPG,
according to the Dana-Farber Cancer Institute. After diagnosis, the median
survival is nine months.
DIPG typically found in children between ages five and nine.
This type of tumor is located at the base of the brain and
the top of the spine, but it is not known what causes them.
The tumor presses on the area of the brain called the pons,
which is responsible for a number of critical bodily functions such as
breathing, sleeping, and blood pressure.
Over time, the tumor affects heartbeat, breathing,
swallowing, eyesight, and balance.
Some of the first symptoms of the tumor are problems with
eye movement, facial weakness, difficulty walking, strange limb movements, and
problems with balance.
Lucas was diagnosed aged six after he fell ill in the summer
holidays.
He could not walk straight, had difficulty peeing, would
pass out, and suffered nose bleeds.
The researchers are now trying to reproduce the difference
seen in Lucas's cells.
'Lucas is believed to have had a particular form of the
disease.
'We must understand what and why to succeed in medically
reproducing in other patients what happened naturally with him,' Dr Grill said.
The researchers are looking at the genetic abnormalities of
patients' tumors while also making tumor 'organoids', an artificially grown
mass of cells that resembles an organ.
The team wants to replicate his genetic differences in the
organoids to see if tumors can be destroyed like Lucas's was.
'The next step will be to find a drug that has the same
effect on tumor cells as these cellular changes,' said Marie-Anne Debily, a
researcher supervising the lab work.
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