Doctors Hoping Pill Form Of ALS Drug Will Be Less Intense Treatment For Patients – CBS Pittsburgh

PITTSBURGH (KDKA) Doctors are trying to make an FDA-approved drug treatment for Amyotrophic Lateral Sclerosis, or ALS, much less intense for both patients and caregivers.

I was having trouble picking up a fork. I was dropping things, Wanda Donahue, who has had ALS, or Lou Gehrigs disease, since 2017, said.

The disease has affected her arms and legs primarily.

I was just walking down the sidewalk, walking anywhere, and I fall, she said.

Shortly after her diagnosis, the FDA approved Radicava, the first new drug for ALS in two decades.

Radicava neutralizes free radicals. These unstable atoms may play a role in ALS through damage to nerve cells.

Wandas doctor asked if she would like to try it.

I said, Ill try anything, doctor. And Ive been on it ever since, she said.

Wanda gets the drug intravenously.

She puts the needle in my port, Wanda said. Sit there for 30 minutes, wait for the bag to finish, and we put the other bag on, and sit there and let it finish, and she unhooks me, and the next morning, we start off with the same thing. For 10 days.

Shell get it two weeks on, two weeks off for the rest of her life.

Ive heard of people being on it, and dont want to deal with the hook up and sitting still, Wanda said.

Because the medicine requires a lot of time and effort to give, some patients asked for a different way to get the drug. But there was no other way until now.

The company has now developed an oral form. And very soon, were going to start these trials. Its not going to be a placebo trial. Every patient will be getting the medication, says Dr. Sandeep Rana, an ALS specialist at Allegheny General Hospital.

The trial is 48 weeks long. The drug will be cycled similarly to IV Radicava two weeks on, two weeks off.

Researchers will be checking for safety and effectiveness, with labs, breathing tests, and a functional rating scale. They hope to enroll 10 patients at AGH.

I hope it is successful. It would really be a big advancement in my view, says Dr. Rana. Oral is so much more convenient, and less expensive way to go.

But Wanda says shes not interested in participating in the trial. Her ALS is holding steady with little progression on the IV medication, and she says she has more energy now.

Im happy with the way the IV is working for me. Because a pill may not work for me. The pill may not do what the IV does for me. Im not willing to give that up. In my book, its keeping me alive. I got two grandkids I want to see graduate. And thats whats keeping me going.

Wanda knows the drug is not a cure, but she just hopes it keeps her going as long as possible.

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Doctors Hoping Pill Form Of ALS Drug Will Be Less Intense Treatment For Patients - CBS Pittsburgh