Parkinson's Gene Therapy Study Begins

[newyawker99]

Researchers slipped billions of copies of a gene into the brain of a Parkinson's disease patient Monday, marking the first attempt to test gene therapy in a person with that disease.

The patient, 55-year-old Nathan Klein of Port Washington, N.Y., said he was "feeling fine" in a telephone interview a few hours after the surgery.

Klein said the disease gave him a tremor in his right arm, and made him shuffle and have to catch himself sometimes from falling. Medicines offer some relief, he said, but when he heard about the new study he decided to volunteer.

With the disease, "you don't want to go out," Klein said. "It's embarrassing when you're shaking a lot and you walk like an old man."


Parkinsons

[newyawker99]

1. kick

:kick:

[Heddi]

2. I really hope this works

I know there had been problems with gene therapy in some younger boys (I believe they had leukemia, but not sure) where they ended up dying---pretty much put that leg of gene therapy on hold.

Hopefully, this being an older person with a more...developed immune system, etc, this will go much better than it did with the young boys.

So many people suffer from Parkinson's Dx, and it seems that people are getting it younger and younger (same with Alzheimers Dx).

I wish this man, and all other study subjects, the absolute best.

[newyawker99]

3. I agree Heddi. I wish him well too.

[frustrated_lefty]

4. Different gene delivery vehicle.

The kids in the ADA trial were treated with a retroviral vector. The hazard, as we've since learned, is that retroviral vectors integrate semi-randomly into the host chromosome, which has the potential to turn "on" a cancer-causing gene or turn "off" a tumor suppressor gene. It was impossible for scientists to predict how frequently such an event would occur in human studies. Regrettably, it appears to occur much more frequently than might be hoped.

The vector being used in the Parkinson's trial is based on adeno-associated virus. Instead of integrating willy-nilly at random locations within the patient's chromosome like a retrovirus, AAV integrates within a single, known location which is NOT in any way related with cancer.

In any event, the concerns in the present study are the potential for the patient to have an adverse immune response to the vector itself, or, alternatively, that the therapeutic gene being delivered is inadequate to cure the disease. Keep your fingers crossed.

[NickB79]

5. I always found that amazing

That there was such an uproar when children, afflicted with a lethal genetic disorder, are cured of their previously incurable disorder but come down with cancer. I'm sorry because this will sound cruel if taken the wrong way, but it seems that if I had the choice between an incurable genetic disorder or leukemia, I would chose leukemia because at least there's a potential cure for that. It's not like the treatment made them any worse off; how much worse can you get when you have a lethal disease?