Ok we have questions don't we?

GregD · 07-28-2007, 10:02 AM

First off, My condolences to the family of the patient that died. Your loved one is a hero and their life should be celebrated as such. Thank You.

Now, as usual the media has sensationalized this death as a failure. Not true. The fact that the patient lived for some time after the surgery makes it a success. So much knowledge was gained and will be gained that hopefully a correction can be made and another death won't happen.
As with any medical treatment there are risks. With most medications and surgerys are prescribed or done with this thought in mind, the benifits outweigh the risks. Let's ask Carylon and Dottie if they were informed to the risks involved in this procedure? If they were informed, did they agree to continue with the procedure? Did these ladies know what they were getting into by doing this procedure? How many other deaths have been associated with this procedure? Was it infact the procedure that caused the death?
There are just too many questions to be answered to jump in with both feet and say this was a failure.
Hats off to Carylon, Dottie, and all the others that volunteered to do this. You are heros in my book.

GregD

aftermathman · 07-28-2007, 11:10 AM

we still don't know the cause of death or whether it was anything to do with the trial.

To quote: "The clinical course that this individual experienced has, to our knowledge, never been seen as a consequence of exposure to adeno-associated viral (AAV) vectors or naturally occurring AAV".

IMHO it is too early to talk of corrections and trial success or failure.

Brings home the bravery of all clinical trial participants.

Neil.

paula_w · 07-28-2007, 11:37 AM

It is too early, this is true. it's in all the news alerts, and ignoring it didn't seem right either with trial participants in gene therapy right on the forum, so I posted for discussion. Carolyn is fine, her internet is down we discovered and she didn't know a thing about it.

A group of us is attempting to write a patient tool for trial participants, to take with them for the trial investigators and sponsors to fill out. They are informed of the risks and sign an Informed Consent, but more in writing is needed from the patient perspective, including more financial responsibility from the sponsor or investigator. All input is valuable and I think it's important for us all to learn from this trial step by step.

As for the participants, I'm so very grateful and proud of them.

paula

indigogo · 07-28-2007, 11:49 AM

To me the story is more about "the story" - how many reports are calling "doom" to the procedure right off the bat without all of the information and an investigation yet to be done.

It's also a story about the clinical trials process, informed consent, and the risks inherent and necessary for research to move forward.

The significance of patients willing to step forward only increases given this type of media scrutiny.

Dottie · 07-28-2007, 12:03 PM

Greg -

This was the first time I've been involved in a trial of any kind so I was a little nervous at the beginning. That vanished by the way the presenting of the protocal was handled. After agreeing to take part in the trial we went through the 23 page document word by word, sentence by sentence -asking questions, making comments, whatever. Each page was initialed and dated.
At all times we were reminded that we could drop out at any time, including ibefore surgery would begin.

The protocal starts with Purpose and Background, goes to Risks and Discomforts (llsting 22 ttems) and a further list of potential complications relatng to surgery ending with the actual Consent. I 'm sure someone who has been involved with other trials might find they wanted more informaiton but I felt informed.
I hope this answers your questions.

Dottie

jeanb · 07-28-2007, 04:07 PM

The death of the trial participant is tragic. I suspect he joined the trial with great hopes that this experimental treatment would help his arthritis. And I know our hearts go out to his family.

This is a brutal reminder that clinical trials are experiments on humans. Every trial participant takes a risk on behalf of others. Some risks are greater than others, but they are all risks nonetheless. But this is the only system we have for getting new drugs or treatments through the research pipeline.

Unfortunately new promising Parkinson’s treatments involve brain surgery of some kind. I doubt I am alone in wishing there were a different way. So the risks are great due to surgery and also due to new treatments – like gene therapy.

Will the death of this individual keep others from joining trials? Perhaps or perhaps not. For PWP who have never participated in a clinical trial, it’s one more reason to add to the list not to sign up. But for the 1% of PWP who participate in clinical trials in the US, I doubt this will deter them.

Howardh · 07-28-2007, 05:49 PM

we know the precise cause of death of this great American pioneer that died while participating in a gene therapy trial, the jury remains out on the likely cause of death. 500 taking part in this GT trial...seems to me to be a relative success so far. I see the glass as half full...the New York Times, it seems sees the glass as half empty...bit like the English Rugby Team for the Rugby World Cup......gidday Neil

Meanwhile Neurologix www.neurologix.net is pumping along with its magnificent Gene Therapy program, as are others. Every time I log onto their web site the news is always moving forward. One contributor mentioned there were 152 PD research programs going on globally as we speak, according to their neurologist, and we haven,t even started on other great American hero's Christopher and Dana Reeve's, Nancy Regan's, MJ Fox's, and 73 percent of the US population's ultimate research program EMBRYONIC Stem Cell Research....the mother of all research.

It's all good in my book.

GO HARD................SCIENCE

vlhperry · 07-28-2007, 10:35 PM

Quote:

Originally Posted by paula_w

from NYTimes today:

July 27, 2007

Patient in Experimental Gene Therapy Study Dies, F.D.A. Says

By DENISE GRADY and ANDREW POLLACK
A patient has died in a study of an experimental gene therapy, the Food and Drug Administration reported yesterday. The agency said it was investigating the death to determine whether the treatment was to blame.
The case could be another setback for gene therapy, a field with a troubled history and numerous treatment failures, including the death of a teenager in 1999 in an experiment.
The new therapy being tested, made by Targeted Genetics of Seattle, is a virus-based product injected directly in the joints in hope of relieving active inflammatory arthritis. That chronic condition can affect multiple joints and organs, and it is quite different from the wear-and-tear arthritis that commonly occurs with aging.The patient became ill soon after receiving a second injection, the drug agency said. The date is not exactly clear, but the illness was recognized as a “serious adverse event” last Friday, and the agency immediately suspended the study. That means no more injections can be given. The patient died on Tuesday.
Targeted Genetics and the drug agency said they would not describe the patient’s symptoms or the manner of death until the investigation had been completed. The company and the agency emphasized that it was not known whether the treatment had a role or the death was a coincidence. The agency said the timing was cause for concern.
H. Stewart Parker, chief executive of Targeted Genetics, said, “The patient was dosed one dose and had no issues and came back several months later and coincident with the dosing had an S.A.E.”
S.A.E. is an abbreviation for serious adverse event.
Ms. Parker said the company had used the same type of virus to carry various genes into more than 500 patients and had not seen similar problems. “These patients are all on several different medications,” she said. “They certainly have disease characteristics.”
The drug agency said it was reviewing all other studies using the same virus, which is called an adeno-associated virus, though it had not heard of similar problems.
Dr. Theodore Friedmann, a professor of pediatrics and head of the gene therapy program at the University of California, San Diego, said the A.A.V. system used in the experiment was widely regarded as safe.
But, Dr. Friedmann added, “We’ll probably come to learn it does some harmful things in some settings.”

__._,_.___

Please explain to me why my post is "off topic?" Jean and Thelma, my post encouraged research of adult stem cells. Your post, Jean, and Thelma's "Thanks for This" make no sense. Arrian has arthritis, the patient who died had arthritis. Arrian was to receive her treatment in Seattle. The patient who died was in Seattle receiving treatment for arthritis. The information from the Director was added but was not the primary message. My entire first paragraph was about a young girl with her whole life before her and is counting on stem cell/gene therapy to save her life. The little girl wants to dance but can rarely leave her wheelchair for fear of breaking a bone. Everyone on this site is so pro-science and have so little thought for anything but themselves and finding a cure for Parkinson's that they haven't the time to learn of a young girl who more than likely will never know the thrill of her first dance.

Leah McLean's Blog: A hope for healing

It's tough to see a little girl suffering from a disease that's taken an obvious toll. But 10-year-old Arrian Madden has a way of making you smile even through her own hardship. She has Juvenile Rheumatoid Arthritis, or JRA. She's had it since she was six months old. The disease makes her immune system attack her. She's in and out of the hospital with infections a couple times every year. Her bones and joints hurt almost all the time.

I had the opportunity to meet her and her family a couple weeks ago and they blew me away. Arrian was sitting in a wheel chair when we first met. But she got up to say hi. She can walk around but has to be careful not to overdue it or she could injure herself because of the JRA. She told me she wants to take ballet lessons but knows it would be too much on her fragile body.

A stem cell transplant offered in Seattle is bringing new hope for her family. They had planned to start the procedure at the end of July but a new infection is slowing them down. Arrian is in the hospital in a drug-induced coma right now. She has an infection in her kidneys and lungs. Her family is taping our news story so she can see it when she's feeling better. It will likely be months before she's strong enough to do the stem cell transplant. But her doctor says if she can have the procedure it could make all her symptoms go away. It's possible she would start to grow and develop like other children her age once if she can get off all the medication.

This family is struggling with all the challenges. They could use all the help they can get. They set up a fund called Arrian's Angels at TCF bank and they have a Caring Bridge sight set up so people can check in on her progress. We wish them all the best.

What are you advocating for? Science or humanity? I am ashamed to know any of you who have been so heartless not to offer some words of hope to this young friend of mine by at least signing her guestbook and offering some words of comfort or encouragement for a young girl who has given so much.
If advocacy is not taking into account the humanity of all then count me out.

Vicky

indigogo · 07-28-2007, 10:53 PM

Vicky -

Arrian sounds like a very brave girl, and I have every hope and pray that she will be able to get the stem cell transplant.

She is not the patient involved in the clinical trial who died. That trial involved 127 adults only. It did not include a stem cell transplant; it was gene therapy in the form of viral injections.

I have just as much admiration for those arthritis sufferers who volunteer their lives and health to participate in a clinical trial as I do for those who have Parkinson's. All are pioneers; all of them advance the science for everyone.

vlhperry · 07-29-2007, 08:24 AM

Dear Indigogo,

But Arriano is also an experimental subject. Arriana, if able to overcome her overwhelming struggle of fighting off two infections, be the first child to have this surgery. She is also opening doors for young children everywhere suffering this disease. Her family has assurred me of this.

Why is it I have to prove to everyone on this site of the value of the life of this young girl? How is it "OT" to mention anouther hero and request support without proving this is an appropriate thread to post it on. You are the most stubborn people I have ever met, but I will not lose hope that you will stop advocating (this is a support board to my knowledge, not an advocacy only board) long enough to send your prayers and support to a young girl who has her whole life ahead of her.

Vicky

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