Transcript
Narrator:
The Diabetes Research Institute presents a series of reports on the latest progress in cure-focused research – promising discoveries aimed at restoring natural insulin production in those living with diabetes.
Reporter:
In searching for a cure for diabetes, researchers often work closely with pharmaceutical companies, to obtain to new drugs.
But sometimes, the drug companies’ priorities are in conflict with researchers’. That can slow progress. And, researchers can be denied access to drugs that are promising – but deemed not commercially viable.
As a result, the Diabetes Research Institute has undertaken its own Drug Discovery Initiative. Leading the effort is Peter Buchwald, Ph.D, an expert in drug design and development with extensive experience in the pharmaceutical industry.
Dr. Buchwald and his team are focused on developing safer alternatives to the powerful anti-rejection drugs that current islet transplant recipients must take.
When a person receives a transplant, his or her body’s immune system sees the cells as “foreign” and wants to reject them.
So, right now, the recipient must take anti-rejection drugs, for life. Those drugs – called
“immunosuppressants” -- suppress the patient’s entire immune system – exposing him or her to other infections.
Also: the drugs are toxic and could have harmful side effects. They could even play a role in weakening the transplanted cells.
One approach is to develop a drug which will “turn off” only the part of the immune system that attacks the transplanted cells.
Dr. Buchwald says this would allow the body to accept the islets, and allow the rest of the immune system to do its job.
Buchwald:
“So it will act only specifically on the immune response directed to the islets and not to the others, and hopefully will avoid the side effects that are there with many other currently used immunosuppressive agents.”
Reporter:
Buchwald says scientists know what specific part of the immune system attacks the transplanted cells.
Buchwald:
"It's well known what the usual immune response is.”
Reporter:
Researchers at the DRI not only know the target – they have successfully hit it. But, they used an antibody that is no longer available.
Buchwald:
“It was a single drug that worked in the long run. It avoided rejection for a very long time. We knew it works. What we are trying to do now is a different molecule. It worked with antibodies. We are trying to do it now with small molecules.”
Reporter:
Buchwald says the DRI’s new focus – on small molecules – has advantages over the use of antibodies.
Buchwald:
“Antibodies have their own problems. They are large proteins when they are administered to humans. They tend to cause unwanted foreign body reactions so many antibodies are difficult to handle and you can have often very severe allergic reactions to antibodies. Small molecules are much, much easier to develop. The chemistry involved is much easier, the formulation involved, they are much easier to administer, to achieve. So the development is much easier. The question is whether we can find a small molecule to replace this antibody. Because they tend to be less specific than antibodies.”
Reporter:
As Buchwald and the DRI search for the right small molecules, they’re also looking at another approach: to deliver anti-rejection drugs not to the entire body, but only in a tiny device, placed under the skin or in another part in the body, where the transplanted islets are.
Buchwald:
“If we can manage to have immunosuppression then only within this device, that would be a great advantage because first of all we need a much, much smaller dose. We don’t have to use the drug all over the body. It only has to be in the very small device, which is like thousands, or even less portion volume of the whole body. And somehow infuse it just locally straight in this device.”
Reporter:
Even if some of the drug leaves the device, he says it would be at a low concentration or might be engineered to become inactive quickly. The key: to prevent the drugs from entering – and damaging -- the recipient’s entire system.
Buchwald cautions that both approaches – targeting a specific part of the immune system with small molecules and localized immunosuppression -- are in their early stages. And that, in either case, it might take a combination of drugs – not just one -- to do the trick.
But, if either approach is successful, it’s possible the problem of life-long anti-rejection drugs would be solved.
Narrator:
This has been a production of the Diabetes Research Institute Foundation.
For more information, or to show your support for the Diabetes Research Institute, call 1-800-321-3437.
You also may donate online at diabetesresearch.org.