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First results
At Madeux and in the other low dose patient, urine sugar levels rose 9% on average after four months. Muenzer said that it was difficult to know if it was a significant change. Little is known about the biology of these compounds, including whether they fluctuate during the day or before or after meals.
A liver biopsy on a patient who received a low dose of treatment revealed no evidence of genetic modification, but Sangamo scientists said this dose was well below the level at which these signs had been detected in primate research.
Two other patients received an average dose corresponding to twice that of the first two patients. Their GAG levels decreased by 51% after four months, on average. Two of the main types of these sugars that accumulate in the tissues decreased by 32% and 61% respectively.
It is unclear whether such declines can improve patient health or slow the progression of the disease.
"This is not evidence that this therapy is successful, but that these patients have had enough genes to provide them with the enzyme they need for the rest of their lives," Muenzer said.
But he said that an important goal was achieved: the treatment seems safe. There were two serious side effects – one patient was hospitalized for bronchitis and another for an irregular heartbeat – but these were judged due to their disease and pre-existing conditions, not to gene therapy.
Blood tests did not detect the missing enzyme. The company's scientists said it could be due to the fact that everything that was manufactured was quickly used by the cells rather than entering the bloodstream – an explanation accepted by outside experts. What matters, they said, was the result of enzymatic activity, the fall of sugars.
NEXT STEPS
Two other patients received the highest dose tested – 10 times the initial dose – for a total of six patients included in the study. The next step is to start removing patients from the weekly enzymatic treatments they received to see if gene therapy has changed their bodies to make enough.
More results are expected at a medical meeting in February.
"We need to see sustained levels to make it practical. If it only works for six months, it's not very beneficial, "said Muenzer. "Time will tell."
In an interview at his Arizona home last month, Madeux, 45, told The Associated Press that he had volunteered for the study in the hope to be able to stop the weekly three-hour enzymatic infusions, but also to help future generations. with the disease.
"I'm old and having hunters did a lot of damage to my body," said Madeux. "I am rather lucky to have lived so long."
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