Witam,

na grupie dyskusyjnej dla rodziców dzieci z ARPKD (postać recesywna, grupa
A...@y...com) pojawił się optymistyczny post na temat możliwości
zahamowania rozwoju torbieli w przypadku wielotorbielowatego zwyrodnienia
nerek (PKD) lekiem OPC31260, który, testowany dotychczas na zwierzętach,
wykazał takie oto obiecujące właściwości. (post wklejam poniżej, a ponadto
http
://w
ww.n
atur
e.com/cgi-taf/DynaPage.taf?file=/nm/journal/vaop/ncu
rrent/abs/nm935.html)

Ponieważ jestem laikiem, matką dziecka podejrzewanego o jedną z postaci tej
choroby, chciałam zapytać, czy ten optymizm jest uzasadniony, czy
rzeczywiście jest to przełom, a także co z tego dla nas w Polsce i kiedy
może wynikać.

Będę wdzięczna za opinie i informacje od nefrologów/zainteresowanych na
grupę lub konto: m...@y...co.uk . Z góry dziękuję.

Poniżej post. Przepraszam, że długi i mam nadzieję, że nie naruszam zanadto
netykiety wklejając całość, ale zależy mi na Państwa opinii:

>From: "Keith Marks" [...]
>Reply-To: A...@y...com
>To: A...@y...com
>Subject: [ARPKD] Exciting news regarding PKD Research
>Date: Tue, 23 Sep 2003 12:11:01 -0400
[...]

>DATE: September 22, 2003
>
>FROM: Dan Larson, President & CEO
>
>
>The following discovery was embargoed for release until yesterday [Sunday
>afternoon], so now I can freely share this good news with those who - by
>virtue of your "time, talent and treasure" - provide the leadership and
>support it takes to make the PKD Foundation able to pursue our vital,
>life-saving mission.
>
>The press release herein references cutting-edge work done by our great
>friend and world renowned scientist/clinician - Dr. Torres [et al] at the
>Mayo Clinic, and Vince Gattone, Ph.D., a longtime friend and PKD researcher
>at the Indiana University School of Medicine. Their work creates notable
>optimism for a soon treatment to slow...perhaps stop, the progression of
>human PKD.
>
>This is great news...but as notable as this is...already in the works is
>yet
>another, perhaps even more significant discovery. This potential
>breakthrough is one I've been sworn not to divulge...yet if progress
>continues as it has so far, I hope to be able to tell you some more
>terrific
>news, soon.
>
>I rejoice in this progress and in the "help and hope" it represents for
>countless PKD families, worldwide, for whom we're privileged to serve.
>
>
>RESEACHERS IDENTIFY NOVEL THERAPY FOR WORLD'S MOST COMMON LIFE-THREATENING
>GENETIC DISEASE
>. Discovery Could Benefit Millions Suffering from Polycystic Kidney
>Disease
>Rochester, Minn. (September 22, 2003) - Researchers at the Mayo Clinic and
>Indiana University, utilizing an existing drug already used on humans to
>treat irregular fluid retention, found it also retards cyst production and
>disease progression in polycystic kidney disease (PKD), the world's most
>common life threatening genetic disease. PKD causes cysts to form in both
>kidneys hindering their ability to filter toxins from the blood; kidney
>failure results in the majority of cases. Mayo researchers using a drug
>called OPC31260, determined it inhibits the production of a hormone called
>"cyclic AMP" by blocking a hormone receptor found almost exclusively on the
>cells from which the cysts develop. They discovered this drug reduces the
>size of kidney cysts in laboratory animals that have the identical form of
>PKD as found in humans, and with very little toxicity.
>
>These findings are likely to pave the way for clinical trials of this drug
>in people with PKD; it generates great optimism for a soon therapy to stop
>the progression of PKD. Future studies will determine how rapidly it could
>be used to treat those with PKD. The findings of the study will be
>reported
>in the October issue of Nature Medicine The PKD Foundation, the National
>Institutes of Health and the Mayo Foundation provided grants that led to
>this promising discovery.
>
>PKD affects 1 in 500 American men, women and children irrespective of age,
>race, gender, ethnicity, geographic location or socio-economic status; an
>estimated 12.5 million people, worldwide, have PKD. More Americans have
>PKD
>than the combined number of those who have cystic fibrosis, sickle cell
>anemia, hemophilia, muscular dystrophy, Down's syndrome, or Huntington's
>disease. There is currently no treatment or cure for PKD.
>
>PKD Foundation president and CEO, Dan Larson says, "This may well the most
>promising discovery since we found the PKD genes; this breakthrough
>predicts
>a much brighter future than otherwise probable - for countless PKD
>sufferers, worldwide. If we can slow...even stop the progression of PKD,
>we
>will prolong life, avoid pain, suffering and many premature deaths for
>those
>with PKD - while saving billions of dollars for taxpayers and freeing-up
>thousands of spots on the kidney transplant waiting list."
>