Page 28 - ARUBA TODAY

A28 SCIENCE
Thursday 7 december 2017

Gene therapy shows promise against blood-clotting disease

By MARILYNN MARCHIONE might not pass it on as leaders work for or have Konduras, 58, a machinist therapy, neither brother
AP Chief Medical Writer they divide, so the benefits stock in Spark. treated in March. has needed clotting factor
Gene therapy has freed 10 may wane over time, but Dr. Matthew Porteus of Before, even small amounts treatment.
men from nearly all symp- they’ve lasted eight years Stanford University, who of exertion would cause tiny The therapy is still experi-
toms of hemophilia for a in other tests in people and wrote a commentary in muscle tears and bleeding mental and its eventual
year so far, in a study that up to 12 years so far in dogs. the journal, called the re- problems requiring clotting cost is unknown, but clot-
fuels hopes that a one-time ting factor treatment costs
treatment can give long- about $200,000 per patient
lasting help and perhaps per year, Porteus said.
even cure the blood dis- Another gene therapy,
ease. from BioMarin Pharmaceu-
Hemophilia almost always tical for a different form of
strikes males and is caused hemophilia, also showed
by lack of a gene that promise in a different study.
makes a protein needed Thirteen patients have
for blood to clot. Small cuts been treated and have
or bruises can be life-threat- had a big drop in bleeding
ening, and many people episodes and clotting fac-
need treatments once or tor treatments, study lead-
more a week to prevent se- ers report. One-year results
rious bleeding. will be given at an Ameri-
The therapy supplies the can Society of Hematology
missing gene, using a virus conference that starts Sat-
that’s been modified so it urday.
won’t cause illness but fer- Other companies are work-
ries the DNA instructions to ing on hemophilia treat-
liver cells, which use them ments; Sangamo Thera-
to make the clotting fac- peutics is testing traditional
tor. The treatment is given gene therapy and gene
through an IV. editing approaches.q
In a study published
Wednesday by the New
England Journal of Medi-
cine, all 10 men given the This Saturday, Dec. 2, 2017 photo provided by Jay Konduros, left, shows him and his brother, Bill,
therapy now make clot- at Jay’s home in Cambridge, Ontario, Canada.
ting factor in the normal Associated Press
range. Bleeding episodes “The hope is that this would sults “striking” and said, “I factor treatment.
were reduced from about be a one-time treatment” think we’re definitely on the “Even something as innoc-
one a month before gene to fix the problem, said the road” to a cure. uous as reaching over your
therapy to less than one a study leader, Dr. Lindsey It feels like one to Canadi- head to get something out
year. Nine of the 10 no lon- George of Children’s Hos- ans Jay and Bill Konduras, of a closet, or reaching
ger need clotting factor pital of Philadelphia. brothers who live an hour’s down to tie a shoe” could
treatments, and the 10th Spark Therapeutics, the drive outside Toronto who trigger trouble, Bill Konduras
needs far fewer of them. Philadelphia-based com- were in the study. said.
There were no serious side pany that makes the treat- “It’s pretty magical,” said Six years ago, he nearly lost
effects. ment, and Pfizer, which Jay Konduras, 53, who runs his leg after a motorcycle
Follow-up is still short — a now is working with Spark a bakery and was treated crash tore open an artery;
year on average. Some on it, paid for the study, in June 2016. he spent nearly a month in
cells with the new gene and some of the study “Life-changing,” said Bill the hospital. Since the gene