Unmet Needs for Hemophilia Treatment


Peter L. Salgo, MD: What are the unmet needs?

Christopher E. Walsh, MD, PhD A treatment that is a cure, in effect.

Peter L. Salgo, MD: A cure?

Christopher E. Walsh, MD, PhD Yes.

Peter L. Salgo, MD: A genetic treatment.

Christopher E. Walsh, MD, PhD A genetic cure.

Peter L. Salgo, MD: Somebody who was not making factor VIII with this treatment is now making factor VIII. We’re done, go home.

Christopher E. Walsh, MD, PhD Right.

Peter L. Salgo, MD: It will put you out of business.

Christopher E. Walsh, MD, PhD Pretty much. I would love to be out business. There are plenty of other diseases to work on.

Peter L. Salgo, MD: What’s your best guess on this, going down the line?

Christopher E. Walsh, MD, PhD I would say there will be a gene therapy product available in 2 to 3 years.

Peter L. Salgo, MD: Stop, 2 to 3 years?

Christopher E. Walsh, MD, PhD Yes, yes.

Peter L. Salgo, MD: You just keep stopping me. There’s progress going on. It’s amazing, these are quantum leaps.

Christopher E. Walsh, MD, PhD Yes.

Peter L. Salgo, MD: This is disruption upon disruption upon disruption.

Christopher E. Walsh, MD, PhD Yes.

Peter L. Salgo, MD: Let’s get rid of hemophilia. You’re telling me in 2 to 3 years, we can.

Christopher E. Walsh, MD, PhD Correct.

Peter L. Salgo, MD: How certain are you?

Christopher E. Walsh, MD, PhD Pretty certain. The certainty is really that I know there will be drugs that will be approved, viruses that will be approved. That is a certainty. How long they last, what side effects come from them, if they could they cause cancer, and what they are doing are all other issues. Now, they may be approved in 2 or 3 years. Whether patients will avail themselves to those drugs is a totally different question.

Peter L. Salgo, MD: What are the unmet needs?

Robert F. Sidonio Jr, MD, MSc: We’ve only talked about hemophilia A, right? We haven’t talked about hemophilia B. They’re looking at patients with hemophilia A and saying, “I know that they have a higher rate of inhibitors, but now they have options that the hemophilia B community doesn’t have.” There is a new drug called concizumab, another ‘ab’, and it’s in clinical trials that will work both in hemophilia A and B. That’s a significant unmet need.

Those patients with hemophilia B and inhibitors are incredibly difficult to treat. The options aren’t good, so they need a therapy that works the same as on the hemophilia A side. Thankfully, the companies are seeing that and saying, “We’re going to answer that.” But with gene therapy, there are success stories on both sides, which is great.

Peter L. Salgo, MD: That’s exciting.

Robert F. Sidonio Jr, MD, MSc: Yes. And then, there are the chronic pain issues. We’re talking about young children. We have these older adults who have a lot of issues and just like in the community with opiates, we have to do a better job of handling pain because we can’t use things like NSAIDs [nonsteroidal anti-inflammatory drugs] in our hemophiliacs safely.

Peter L. Salgo, MD: What about patients? We’ve talked about this from the physician’s side. What are patient concerns that we probably haven’t met?

Robert F. Sidonio Jr, MD, MSc: I give a lot of talks at patient advocacy meetings, and for me it’s helpful because people will tell you what they think about it. I go to those meetings and I see what those unmet needs are. Inhibitor management is one big thing that we’re addressing, obviously making gene therapy available, and coverage as well. These patients used to have lifetime caps that they were meeting before they became adults. You see these T-shirts that say, “I’m not pre-existing, I’m existing.” We need to make sure we protect those patients because they have a pre-existing condition. We need to make sure they’re covered and that we’re not forgetting about them as soon as they turn 18.

Peter L. Salgo, MD: It seems to me that for somebody born with this genetic issue, going forward—let’s say born in the next 5 years—the landscape is completely transformed.

Christopher E. Walsh, MD, PhD Absolutely.

Peter L. Salgo, MD: You’re dealing with quality of life issues for people who’ve been receiving older treatments and who have seen the consequences of incomplete treatment.

Christopher E. Walsh, MD, PhD That’s correct. The grandparents of any child who’s born from now on will have borne the brunt of the technology that was available at their time, including the problems of hepatitis and HIV [human immunodeficiency virus]. Their grandchildren will probably live in an age where hopefully, we won’t see inhibitors. The treatment paradigm is much easier, and patients won’t have any major significant bleeding episodes. Eventually, this will lead toward cure down the line.

Peter L. Salgo, MD: If you have any last thoughts to share with our audience, what would they be?

Robert F. Sidonio Jr, MD, MSc: I think it’s an exciting time. We don’t want to get complacent because we have these new therapies. We still need to address the issues that are in front of us about inhibitor eradication, looking at therapies, and making sure we don’t develop inhibitors in these new patients. We need to do a better job. I think it’s important that these patients still follow up with their hemophilia treatment centers, and we are still diligent in addressing all these issues.

Peter L. Salgo, MD: It’s just been great talking to you. I want to thank all of you for joining us as well. I hope you found this MD Magazine® Peer Exchange discussion to be useful and informative. I’m Dr. Peter Salgo, and I’ll see you next time.

Transcript edited for clarity.

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