News From Us

 Mila was an incurable Batten patient until doctors developed a special medicine for her.  Mila Makovec’s doctors accomplished what was previously thought impossible: They soon developed a medicine from scratch for this girl with a terminal brain disease.  Mila’s doctors, who have a fatal and incurable Batten patient, mapped her entire gene.

 Examining the differences in Mila’s genes, doctors found the genetic mutation that caused her disease.  So they started working on a drug they thought could cure the disease.  Mila’s cells and the drug tested in animals received approval from the US Food and Drug Administration.  While it normally takes around 10 years to develop a drug, Mila’s doctors accomplished this in less than a year.  Thanks to this, Mila now has fewer seizures.

Cancer immunotherapy also entered a new era this year.  Now doctors use the patient’s own immune system to fight cancer.  Half of the melanoma patients who were thought to be incurable 10 years ago are now cured.  Previously, only 1 in 20 patients with advanced melanomas survived for five years.  Most died within months.  Thanks to this came a new approach to cancer.  Seven-year-old Charlotte Stevenson of Belfast is among the first patients to use a new type of drug that has “revolutionized” cancer treatment.  These drugs only target tumors, no matter where they are in the body.  larotrectinib is the first drug approved in Europe and has the ability to detect genetic disorders.

 Isabelle Carnell Holdaway’s life was saved by an experimental cocktail of viruses.  The young girl’s body was being attacked by a deadly and insurmountable bacterium.  Doctors put the probability of survival at 1 percent.  Large, black and purulent lesions formed on his skin, where the bacteria lived.

 When he was taken to intensive care, his liver began to deplete and bacterial colonies spread throughout his body.  But doctors at Great Ormond Street Hospital used an untested bacteriophage treatment.  This treatment used viruses to kill bacteria.  Bacteriophage has never been a mainstream therapy and has lagged behind with the development of easier-to-use antibiotics.  But today, this treatment method has been applied again due to the emergence of antibiotic-resistant bacteria.  This method, which saved Isabella’s life, may enable more people to cling to life in the future.