The Future For Hemophilia
Today we’re going to talk about the future for Hemophilia. But before we get into where we’re going, let’s take a look at where we’ve been. I’ll use myself as an example for this. I was born in 1983, and shortly before I turned one, I was diagnosed with Hemophilia. The first product I was put on was a product called Cryoprecipitate. Now, Cryoprecipitate is fresh human plasma. It was administered through an IV drip bag and, every time I had a bleed, I had a bleed I had to go down to the ER and get the Cryo to stop bleeding. So, the E.R. was my second home as a kid.
Next, we were introduced to home infusion. When we were doing home infusion, we were using factor with human plasma. My mom used to infuse me at home, so it became a lot easier. Then, we were introduced to recombinant factor without human plasma. That product, for me, was Helixate. I was on Helixate for many years. Then, in 2014, I was one of the first people to get on a new product called Eloctate. Eloctate is extended half-life factor, so instead of infusing three to four times a week on Helixate, I can infuse two times a week on Eloctate. I’ve been on Eloctate ever since.
Then, just recently, we were all introduced to anew breakthrough product called Hemlibra. Hemlibra is a subcutaneous product, so you can administer it in the fat of your arm, fat in your belly, fat in your leg. You can do that once a week. There are currently 2,500 patients currently on Hemlibra. So that is the most recent breakthrough we’ve had in the Hemophilia community. Then, we have talks about gene therapy. Now, I’ve heard about gene therapy for about a decade now. But just recently there’s a race for the pharmaceutical companies to get a product out on the market. So we are monitoring that fairly closely.
Before I get into gene therapy, let me get into the recent developments for factor. Bioverativ is developing a new product. I call it the extended, extended half-life factor, so you’ll be able to infuse once every 10 to 14 days, depending on how long it stays in your system. I’m currently on trial for this. So if it all goes good, I’ll be one of the first people to get on this new factor. So again, you’ll be able to infuse once every 10 to 14 days, depending on how long it stays in your system. We will definitely keep you updated on that.
Now, let’s go back to Hemlibra. I’ve been getting a lot of emails, a lot of messages regarding my videos regarding Hemlibra, people wondering what I think about it. I think people with inhibitors should stay on Hemlibra. Anyone else should get off Hemlibra. I think years and years of Hemlibra use is going to destroy your possibility for gene therapy. So only people with inhibitors should stay on Hemlibra. Everyone else should get off Hemlibra. That’s just my personal opinion. Also, I think the marketing from Genentech regarding Hemlibra has been sleazy. They don’t post the reported deaths on Hemlibra.com where they’re getting all the patient traffic. They post it on another website and they’re trying to bury the information. I think that’s really bad for them. Every today, I checked Hemlibra.com. They still haven’t posted it. We sent a letter to them regarding this. I think it’s bad business and if I were the patient I wouldn’t be on it.
Now, moving on to gene therapy. Gene therapy is not a cure for Hemophilia. There has been a lot of patients in the trials who have developed an antibody to the gene therapy. So is this a cure? No, if it doesn’t work for everyone it’s not a cure of Hemophilia. To further my argument, if I get a seven-figure injection to make me stop bleeding it’s not going to cure my damaged joints, inflammation, chronic pain, the lost range of motion. It’s not going to fix that. So is this a cure for Hemophilia? No, it’s not a cure for Hemophilia. There will never be a cure for Hemophilia, ever. There will always be Hemophiliacs roaming this Earth until the end of time. It’s not good business to cure patients. These pharmaceutical companies have investors. shareholders and a full board. These are companies. Curing patients is not good for business. Money is like oxygen. If you don’t have it, you’re dead. So these companies are going to develop a product that’s going to keep you coming back. The repeat business is always good business. So I think you’re going to see short-term gene therapy hit the market before you even see a one-time injection. Short-term therapy should be hitting the market within two to five years. You’re going to see short-term gene therapy where you’ll get the injection. It will stay in your system and then over time, you’ll have to come back and get another injection so you can stop bleeding. Analysts estimate gene therapy represents a $20 billion revenue opportunity in the US alone if all 20,000 patients here were treated.