9
Aug

JDRF Research symposium, part 2

Aaaaand... part 2!

 

 

Dr Jan Dutz (UBC, Professor): stopping beta cell.destruction by dampening or blocking the autoimmune response

Jan Dutz; rheumatologist, dermatologist.

Psoriasis: for years researchers thoug hr this was a skin disease. Turns out it is an autoimmune diseases. Drugs and treatments use antibodies

Drug marketed for psoriasis followed patients treated with ustekinumab. Bc of the narrow therapeutic index , it is very effective at one specific thing. Thses drugs are safer than immunomodulators that used to be commonly used. Instead, these drugs block signals between "intruders" and other cells.

T1D study started about 5 years ago? Found that it was safe, no notable side effects.

Study was not designed to prove that ustekinumab ... was effective ijn t1d tmnt. Only designed to prove it was safe. As this safety study was in progress, the drug was also being studied for use in IBD and Crohn's.

Follow-up study will be starting within 6 months. If used early in disease, can we prevent islet cell loss in patients with T1D?

Next up:

Dr. James Shapiro: Human islet transplantation to reverse diabetes

Islet transplantation is considered fourth-line therapy bc of need for immuosuppressants.

Barrier to islet transplantation:

Shortage of donors!

Dr Shapiro is an incredible speaker. He packed an overwhelming amount of information into his 15-minute talk, most of which is too complex to summarize!

Dr Tim Kieffir, UBC prof:

Stem cells are amenable to large scale production.

Cured some mice:

Note my skepticism. While mice are an important part of testing any therapy, I can't bring myself to get excited about any researcher's ability to cure a NOD mouse.

Last presenter was Liz Ann Gillham-Eisen, Director, office of policy & Intn'l cooperation, Health Canada

What is regenerative medicine? Medicine to augment, replace, repair, or regenerate.

Latest federal budget had an emphasis on innovation in treatment technologies.

Investment into developing regulations:

Cell therapy meets the definition of a drug and is therefore regulated by health Canada. Most work in the "cell therapy" field is in the Investigational Cells category:

(CTO: Cells, tissues, & Organs)

CTO regulates homologous use, not autologous use (bc of risks associated with donors).

In keeping with true Canadian government standards, we of course have the obligatory translation on the next screen:

Wrapping up with the panel discussion and q&a with the second round of speakers:

Dr Shapiro answering a question on accessibility and affordability:

Cost is a major barrier. Small programs exist in Vancouver, Montreal, & Toronto, but these therapies are not widely available. Dr Shapiro noted that after Alberta Health cut off funding for non-Albertans, the remaining provinces also refused, which led to a reduction in patients accessing the treatments.

Dr Kowalski talked about JDRF International's fight for accessible insulin and tech treatments. JDRF International's focus here has been in the US, but they are branching out to Canada, Australia.

Federal government emphasized their commitment to drug affordability/accessibility. Takeaway from Liz's comment- the government is in the right space for this kind of progress; now it just needs to be brought forward.

Closing remarks came from Dr Nancy Tout, T1 parent and a JDRF Research Information volunteer, and Dr. Kowalski.

Dr Tout- the future is devices, ccell therapies, cure.

Dr Kowalski: outcomes beyond a1c. Time in range, quality of life metrics, etc. The benefits of hybrid closed-loop? Sleep, waking up in range. Huge important advances are coming in quality of life- what about devices that improve that but with no noticeable difference in a1c? Next hurdles would be highlighting the benefits of these systems.

The future is now.

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