Tracy Frech, M.D., a rheumatologist at the University of Utah, talks about a skin disease that has no cure, but there is a new drug that is showing promise.
Interview conducted by Ivanhoe Broadcast News in March 2017.
Tell me a little bit about the trial.
Dr. Frech: At the University of Utah, we offer many systemic sclerosis trials to try to improve outcomes in our patients. Several of the trials target skin involvement. This most recent trial sponsored by Corbus examined skin thickening as an add-on therapy. Clinical trials in general will give a placebo versus active therapy in a blinded manner. As physicians we don’t really like that because that means our patients may be not getting a treatment. The reason that’s an acceptable trial design in scleroderma is because no drug has even been proven to work. If patients have tried lots of drugs that haven’t worked the idea is either the drug or a placebo in a clinical trial is an appropriate next step. In this trial we added on this product and we favorably saw an improvement in skin outcomes in the patients that were being treated with this add-on therapy.
Is it helping internally as well or just the skin?
Dr. Frech: We don’t know yet and especially at our center I only can comment on the patients that I’m treating. I’ve been favorably impressed with lots of the different outcomes. Certainly the skin looks like it’s very much improved in the patients on drug.
Carolyn seems to think it’s helping her on the inside as well.
Dr. Frech: That’s the feedback I got from patients. That’s really my hope. The way, of course, clinical trials are designed they have to be powered and have large numbers of patients to show affect. What I most care as a health care provider is that my patients feel better and I’ve been favorably impressed with feedback I have gotten from the patients in the open label extension.
Is it difficult for a trial like this to get large numbers? I’m thinking that this disease is not that common.
Dr. Frech: You’re absolutely right. Scleroderma is a rare disease. We actually had a lot of interest at our scleroderma center in enrolling in this particular trial due to the add-on nature of the design and its mechanism of action.
How does the drug work?
Dr. Frech: I’ll back up a little and talk about the pathophysiology of scleroderma. Scleroderma is a disease where the blood vessels in the body send the wrong message to surrounding tissue to lay down scar tissue. When scar tissue is laid down in different organs that subsequently triggers immune system dysfunction. Most of the rheumatologic drugs that we use target the immune system so it’s more of a downstream affect. Whereas this drug is really affecting the interface of how those immune cells talk to the vasculature of blood vessels. Not only does it have anti-fibrotic affects or anti-scarring affects but also probably improves perfusion or blood flow to the different organs so it has a dual affect. Most importantly unlike our immunosuppressant medications that tell the immune system to settle down so it puts patients at risk for infection this drug doesn’t target the immune system in that way and so we don’t put our patients at risk for infection, which is particularly important if patients have open sores on their skin or have a predisposition to pneumonia because of sub-adequate respiratory status.
It sounds like it’s a communication disrupter?
Dr. Frech: Yes, that’s a good way to put it.
It’s the end of Phase II so Phase II is over?
Dr. Frech: Yes.
Now what?
Dr. Frech: The next stage will be a Phase III trial looking at larger patient numbers which is exciting. I have not received the details on the next phase but what I hope it would do is expand the patient population and look at other outcome measures so, perhaps seeing its affect in the gastrointestinal tract or lungs.
With this particular trial then you’re not measuring internally you’re looking specifically at skin?
Dr. Frech: The nice thing is the primary outcome is looking at skin improvement but also secondary outcomes are looked at and those include looking at improvement in lung function as well as patient reported outcomes which capture a lot of the general patient impressions.
Phase III is going to be expanded at what you’re looking at plus a bigger population?
Dr. Frech: That would be the hope and again the details of the Phase III Trial haven’t been released. Looking at safety outcomes of course is important in a Phase III expanded population.
When might Phase III start?
Dr. Frech: That is a good question. I have filled out a confidentiality agreement so I can get the information of the trial. After that there’s an investigator meeting and the timing of the trial is released. I would imagine in the very near future.
When?
Dr. Frech: In the next several months would be hopeful. Most of the time the greatly limiting step is the institutional review board – just to make sure it’s safe and that the data is reviewed. Once different sites look at the protocol and, of course we really want to participate, we hope to be up and running. I would imagine within six months would be my goal.
Are you one of many centers or are you the only center?
Dr. Frech: We are one of many centers. The reason with a rare disease we have so many patients as we’re one of the largest centers within a five state area. Clinical trials allow to take care of patients who may not have insurance that is covered at the University of Utah. It really allows anyone with scleroderma who meets the criteria for participation come to the center and get treated.
What haven’t I asked you about the trial or the disease or Carolyn that you think needs to be out there?
Dr. Frech: I would mostly say that, and you met Carolyn, and you probably can see why I am dedicated to the treatment and research of scleroderma, we need to do a better job for our patients. Generally speaking rheumatologists suppress the immune system, if that causing harm or could put our patients at risk. We absolutely are obligated to find better therapeutics for our patients. I’m just very grateful to be at a center that was allowed to participate in this trial because of its positive effects. I’m really looking forward to the Phase III that will increase the number of patients that we can treat and get a better efficacy and safety signal.
She says she’s taking the drug on open label now is she eligible to participate in Phase III or does she now want to?
Dr. Frech: That is a great question. With the open label she gets drugs. In the Phase III, is it still blinded so she probably wouldn’t want to go off the open-label in order to participate. Most of the time patients who do the Phase II when they’re in open label extension are not allowed to participate in Phase III. But, again I haven’t seen the details of the next trial clinical trial.
END OF INTERVIEW
This information is intended for additional research purposes only. It is not to be used as a prescription or advice from Ivanhoe Broadcast News, Inc. or any medical professional interviewed. Ivanhoe Broadcast News, Inc. assumes no responsibility for the depth or accuracy of physician statements. Procedures or medicines apply to different people and medical factors; always consult your physician on medical matters.
If you would like more information, please contact:
Suzanne Winchester
801-581-3102
Suzanne.winchester@hsc.utah.edu
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