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Clinical trial on SCLS

Barney Message
25 Aug 2020, 05:17 PM

HansDeWit Message
27 Aug 2020, 12:07 PM

I read this article and I think this is very interesting and hopeful BUT as far as I can judge it will take still some long time before there are results.  On the other hand let us be hopeful for a efficient treatment of SCLS!!!

Arielbatt Message
28 Aug 2020, 02:00 AM

Although as they say it is long term, it seems very hopeful.  2020 has not been a year of good news for the community and this hope fills me with joy.  If these things begin, with a laboratory that puts resources, and a team behind like the NIH, it is because they have many indications that they are on the right track.  Dr. Druey is one of the 2 people who knows the most about our syndrome, that he and his team handle this gives us an added possibility.  And if it does not come to fruition, it will surely help them learn something more about our rare disease.

aporzeca Message
30 Aug 2020, 12:02 AM

Having corresponded with Dr. Druey at NIH, I can provide context for this news.  First, to my knowledge, nobody besides Dr. Druey and his team at NIH, and some colleagues he has partnered with in various medical schools, are carrying out biomedical research on SCLS -- or what is best described as primary capillary leak syndrome, because SCLS is not the consequence or side effect of any other bodily illness.  It is a stand-alone phenomenon.

However, there is ongoing research on capillary leaks that are precipitated by other illnesses, like sepsis, certain autoimmune diseases, differentiation syndrome, engraftment syndrome, hemophagocytic lymphohistiocytosis, ovarian hyperstimulation syndrome, viral hemorrhagic fevers, and snakebite and ricin poisoning.  Moreover, there is new research on capillary leaks caused by chemotherapy medications gemcitabine and tagraxofusp, as well as certain interleukins and monoclonal antibodies.  In other words, there is research money and effort going to areas related to secondary capillary leak syndrome, because they affect many more people than those of us who experience SCLS -- cancer patients, in particular.  Therefore, I have been very hopeful that some of that research would provide new insights for Druey and team.

And this announcement is the first example.  Here is a Korean biotech company, PharmAbcine, whose bread-and-butter business is developing new cancer antibody drugs, and they came up with something (a novel Tie2-activating antibody) which apparently inhibits blood vessel leakiness in mouse tumor models.  When they heard that Dr. Druey has also been doing research on capillary leakage using not just mice but also endothelial cells from SCLS patients, they entered into a contract with NIH to jointly determine whether PharmAbcine's new creation might also inhibit vascular leakage using human cells grown from patients with primary capillary leaks.

This is all research that will be carried out in laboratory conditions, so no human trials are envisioned at this point.  If anybody experiencing an episode of SCLS in the United States would like to contribute to this and other research efforts, please have your family or other caregivers request that your doctors make arrangements to deliver your episode blood samples -- namely, the first blood draws upon your arrival to the hospital -- to Dr. Druey via overnight delivery under conditions that he will gladly spell out (e.g., chilled but not frozen blood).  Episode blood cannot otherwise be obtained, and since most of us are not having episodes anymore, it is in very short supply for use in experiments of the kind envisioned in this instance.