Re: Stem Cell Therapy for ALS in Italy

[Guest guest]

Do you think that stem cell therapy could also be used for PLS since

it, too, is a degenerative motor neuron disease?

edith

> 1: Lancet. 2004 Jul 10;364(9429):200-2. Related Articles, Links

>

>

> Stem-cell therapy for amyotrophic lateral sclerosis.

>

> Silani V, Cova L, Corbo M, Ciammola A, Polli E.

>

> Department of Neurology and Laboratory of Neuroscience, Dino

Ferrari

> Centre, University of Milan Medical School-IRCCS Istituto

Auxologico

> Italiano, Milano, Italy. Silanivincenzo@s...

>

> CONTEXT: With the lack of effective drug treatments for

amyotrophic

> lateral sclerosis (ALS), and compelling preclinical data, stem-

cell

> research has highlighted this disease as a candidate for stem-cell

> treatment. Stem-cell transplantation is an attractive strategy for

> neurological diseases and early successes in animal models of

> neurodegnerative disease generated optimism about restoring

function

> or delaying degeneration in human beings. The restricted potential

of

> adult stem cells has been challenged over the past 5 years by

reports

> on their ability to acquire new unexpected fates beyond their

> embryonic lineage (transdifferentiation). Therefore, autologous or

> allogeneic stem cells, undifferentiated or transdifferentiated and

> manipulated epigenetically or genetically, could be a candidate

> source for local or systemic cell-therapies in ALS. STARTING

POINT:

> Albert Clement and colleagues (Science 2003; 302: 113-17) showed

that

> in SOD1G93A chimeric mice, motorneuron degeneration requires

damage

> from mutant SOD1 acting in non-neuronal cells. Wild-type non-

neuronal

> (glial) cells could delay degeneration and extend survival of

mutant-

> expressing motorneurons. Letizia Mazzini and colleagues (Amyotroph

> Lateral Scler Other Motor Neuron Disord 2003; 4: 158-61) injected

> autologous bone-marrow-derived stem cells into the spinal cord of

> seven ALS patients. These investigators reported that the

procedure

> had a reasonable margin of clinical safety. WHERE NEXT? The

success

> of cell-replacement therapy in ALS will depend a lot on

preclinical

> evidence, because of the complexity and precision of the pattern

of

> connectivity that needs to be restored in degenerating

motoneurons.

> Stem-cell therapy will need to be used with other drugs or

> treatments, such as antioxidants and/or infusion of trophic

molecules.

>

> PMID: 15246734 [PubMed - in process]

[Guest guest]

> Do you think that stem cell therapy could also be used for PLS

since

> it, too, is a degenerative motor neuron disease?

> edith

Edith,

I believe it will be a long time before stem cell therapy will be

used on PLS because of several reasons:

1. Our neurons that are in need of therapy are in an area of the

brain that is very hard to reach. Any method know as of this time

is risky at best.

2. The chance of getting a study done on us is very difficult due to

the rarity of our disease and the inability to definitively diagnose

PLS.

3. Due to the invasive nature and risks involved, the first trials

will be on those that have very little to lose. We have a lot to

lose! If you are at a late stage of disease you might chose to try

it, but most are not in that category, so it would be really hard to

find enough PLSers that didn't have much to lose. I tried a drug

therapy for my liver back in 2000 and I had rapid (within 2 weeks)

loss of my speech and strength. I was bedridden and unable to

communicate for several months. I would not take the risk of doing

that again. Period.

4. The stem cell therapy that is offered by some of the companies

now does not have a delivery method that is effective. It is really

a hit and miss. They inject a ton of cells into you and hope (and

pray) that some of them will get to the right area and do some

good. But it has been proven that while the new cells might work

for a short period of time, they eventually (and very quickly) stop

working and die. There isn't a case out there that has been

successful for any period of time. The ALS-TDF even injected the

new cells directly into the spinal cord of several ALS patients and

they saw no long term improvement. It wasn't worth it so they

didn't opt for doing it again. If it did work, there would be so

many ALSers getting the treatment it would make your head spin.

These folks are desperate for anything. It just doesn't work yet.

It will one day, but not today.

Thomson

[Guest guest]

> Do you think that stem cell therapy could also be used for PLS

since

> it, too, is a degenerative motor neuron disease?

> edith

Edith,

I believe it will be a long time before stem cell therapy will be

used on PLS because of several reasons:

1. Our neurons that are in need of therapy are in an area of the

brain that is very hard to reach. Any method know as of this time

is risky at best.

2. The chance of getting a study done on us is very difficult due to

the rarity of our disease and the inability to definitively diagnose

PLS.

3. Due to the invasive nature and risks involved, the first trials

will be on those that have very little to lose. We have a lot to

lose! If you are at a late stage of disease you might chose to try

it, but most are not in that category, so it would be really hard to

find enough PLSers that didn't have much to lose. I tried a drug

therapy for my liver back in 2000 and I had rapid (within 2 weeks)

loss of my speech and strength. I was bedridden and unable to

communicate for several months. I would not take the risk of doing

that again. Period.

4. The stem cell therapy that is offered by some of the companies

now does not have a delivery method that is effective. It is really

a hit and miss. They inject a ton of cells into you and hope (and

pray) that some of them will get to the right area and do some

good. But it has been proven that while the new cells might work

for a short period of time, they eventually (and very quickly) stop

working and die. There isn't a case out there that has been

successful for any period of time. The ALS-TDF even injected the

new cells directly into the spinal cord of several ALS patients and

they saw no long term improvement. It wasn't worth it so they

didn't opt for doing it again. If it did work, there would be so

many ALSers getting the treatment it would make your head spin.

These folks are desperate for anything. It just doesn't work yet.

It will one day, but not today.

Thomson

[Guest guest]

> HI,

>

> AS THINGS STAND RIGHT NOW JENNIFER IS RIGHT.

> BUT WE STILL NEED THE RESEARCH TO CONTINUE SO THAT ALS PATIENTS

DOWN THE

> LINE HAVE A FIGHTING CHANCE.

Bonnie,

You are absolutely right! I really encourage everyone that is able

to help support ALS research as well as PLS research. Not only will

we benefit from ALS research, we'll also help (hopefully) some very

dear friends like Don and Bonnie.

I also support the local ALSA as they are very inclusive of PLSers

in their support. Let's face it, our chances of having a local PLS

support chapter are slim to none, so we need ALSA. And they need

us.

Thomson