MDA 2024 to Focus on Patient Experience in Gene Therapy, Nonviral Vectors, and More

Donald Wood, PhD

The 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, set to be held on March 3-6, 2024, in Orlando, Florida, will continue a tradition of bringing together clinical experts, researchers, advocates, and companies to discuss and present on the biggest current topics and concerns in the field.

CGTLive spoke with Donald S. Wood, PhD, president and chief executive officer, MDA, to learn more about the importance of the upcoming conference in the field and some of the tracks that will be the focus of this year’s conference. He touched on tracks highlighting gene therapy and the patient experience, navigating conversations with patients, nonviral vectors for gene therapy delivery, and insurance and reimbursement for gene therapy.

CGTLive: Why is this a can’t-miss conference in the neuromuscular world?

Donald Wood, PhD: This is a unique conference. It's the biggest of its kind in the world, and the world leaders in neuromuscular disease, development of treatments, therapeutics, genetic medicine, all of that will be there. We anticipate having over 2000 attendees, from probably 20 to 25 countries here, and they will learn the latest information on what is one of the most exciting areas in medicine right now. Neuromuscular diseases are among the leaders in advancing genetic medicine. And this conference will identify and bring together all the leaders in genetic medicine, from those who are working on the science of it to those who are treating families and patients. And we'll be talking about not just the disease mechanisms, but the complexity of treating patients. Basically, if you're treating patients or studying, treatments, etc. You've got to be at this conference. This is where all the information and the people leading these fields will be for 4 days in Orlando.

Tell us about the conference tracks dealing with patient experience in gene therapy.

Wood: We have probably thousands of patients now who are engaged in gene therapy, if you count all the clinical trials that are underway, and we're learning as we go. Virtually every day is a new experience, since this is such a new area of medicine. What these patients are going to feel like, what's going to happen. Another complexity is that most of the neuromuscular diseases are progressive. So you're adding a gene therapy to someone, maybe who is just starting to see some symptoms, and maybe adding gene therapy to someone who is already in a wheelchair with pronounced disabilities. And how do you design a clinical trial in a progressive disease? We are working with the FDA already in designing new ways to approach clinical trials, and that information will be here.

Another aspect is getting ready to treat patients with a genetic disease; it is very complex. We now know that in order to be effective health care providers, you have to be in a multidisciplinary clinic. At this conference, you'll be able to talk to a variety of health care providers and learn about their experiences in treating people who have diseases without treatments, and then people who can now receive treatment, and those that have a disease at different levels of treatment. So, with all that complexity, we also have a track on navigating conversations with patients. What you tell them to expect when you don't necessarily know what to expect, when it's brand new, even if it's an FDA approved treatment, each case is somewhat different. 

Tell us about the tracks addressing common issues with gene therapies.

Wood: We will have a very in-depth look at gene replacement therapies. There are problems with them. And the biggest problem that we face right now is theviral vector for most of these gene therapies. There are patients that are going to be in clinical trials, where the therapy isn't going to work very well, they leave the trial, but they can never go back in a trial with a viral vector. We now have an enormous amount of experience with that. But we're looking at new vectors that aren't viral vectors. And that is a tremendous area of effort and research.

A very practical concern that patients and physicians and healthcare givers have alike is reimbursement. This is a whole new area for insurance. Insurance companies have never really focused on folks with genetic disorders, let alone people with genetic disorders who are now getting treatments that can save their lives or improve their disease. And they complain that the cost is high. Well, these are brand new areas of treatments, and you don't have over 5 decades of treatments like you do for antibiotics for bacterial infections, so the costs are high right now. They'll come down over time, but for insurance companies to deny coverage, which many of them do, creates a problem for the physician in the health care system and for the patients. So, you're going to hear about some of those problems and how the MDA IS addressing them at a very high level. We're at the congressional level, helping develop legislation to provide for that and working behind the scenes with insurance companies. We have been successful, in a few cases, in getting reluctant insurance companies to cover, but it doesn't work every time and we still need a lot of effort in that vein.

This transcript has been edited for clarity.

Click here to register for the upcoming 2024 MDA Conference.