Bone Marrow

[Janie H]

Has anyone heard about testing being done for bone marrow transplant for ALS, Alzheimers,Cerebral Palsy? I am hearing of this for the first time and wonder if anyone knows more, I am in the initial stages and this could be hopeful for many of us.

 

[AfraidButNotAlone]

I read that they are doing studies in Seoul. I don't know if there are other places or anything else regarding the studies.

 

[Barbie]

I have never heard of bone marrow transplants for Pals. stem cell, but not bone marrow. isn't bone marrow transplants used in cancer treatment?

 

[AfraidButNotAlone]

From nih . gov
"Abstract


Patients with amyotrophic lateral sclerosis (ALS) experience progressive and irreversible paralysis as a result of the continued loss of motor neurons, which leads to death in less than five years. To date, there is no treatment that can change the progression of this disease. Bone marrow stem cells have shown neural regenerative and neural repairing properties. Specifically, our group showed in a murine model of the disease that these cells, when injected in the spinal cord, can rescue motor neurons through the secretion of GDNF. Based on these results, we designed a phase I/II clinical trial for the purpose of demonstrating the viability of the intraspinal injection of autologous bone marrow mononuclear cells in patients with bulbar onset ALS, with an evolution between 6 and 36 months, with a forced vital capacity (FVC) 50% and T90 29%. This article describes the technique for extracting 60 mL of bone marrow used for the intervention, processing it by density gradient, and the neurosurgical technique used for implanting it. After 6 months of follow-up, the few adverse events reported in the first seven patients included seem to show that the procedure is safe and viable. Most of these patients, including two with a rapid deterioration, have stabilized the progression of their FVC and the neurologic scales measured. The data obtained so for seem to justify the design of new trials more oriented toward the efficacy of the procedure."

 

[AfraidButNotAlone]

Abstract


The objective of this article is to assess the safety of intraspinal infusion of autologous bone marrow mononuclear cells (BMNCs) and, ultimately, to look for histopathological signs of cellular neurotrophism in amyotrophic lateral sclerosis (ALS) patients. We conducted an open single arm phase I trial. After 6 months observation, autologous BMNCs were infused into the posterior spinal cord funiculus. Safety was the primary endpoint and was defined as the absence of serious transplant-related adverse events. In addition, forced vital capacity (FVC), ALS-functional rating scale (ALS-FRS), Medical Research Council scale for assessment of muscle power (MRC), and Norris scales were assessed 6 and 3 months prior to the transplant and quarterly afterward for 1 year. Pathological studies were performed in case of death. Eleven patients were included. We did not observe any severe transplant-related adverse event, but there were 43 nonsevere events. Twenty-two (51%) resolved in ≤2 weeks and only four were still present at the end of follow-up. All were common terminology criteria for adverse events grade ≤2. No acceleration in the rate of decline of FVC, ALS-FRS, Norris, or MRC scales was observed. Four patients died on days 359, 378, 808, and 1,058 post-transplant for reasons unrelated to the procedure. Spinal cord pathological analysis showed a greater number of motoneurons in the treated segments compared with the untreated segments (4.2 ± 0.8 motoneurons per section [mns per sect] and 0.9 ± 0.3 mns per sect, respectively). In the treated segments, motoneurons were surrounded by CD90+ cells and did not show degenerative ubiquitin deposits. This clinical trial confirms not only the safety of intraspinal infusion of autologous BMNC in ALS patients but also provides evidence strongly suggesting their neurotrophic activity.

 

[Barbie]

I am not a doctor (I just play one) so does this say it did not show any improvement? where was this test done?

 

[jacquie]

I saw this article on this research today. Here is the link:

ALS Stem Cell Trial in Israel Is ?Accelerated? | MDA/ALS Newsmagazine

 

[AfraidButNotAlone]

You got me

 

[BrianD]

Post #4 is from Dec 2010 article from clinical trial in Spain. Post #5 is a June 2012 from the same group. They are still recruiting for the trial I think based on clinicaltrials d o t gov.

These articles are talking about stem cells derived from the bone marrow. They take out some marrow, sort out the cells, collect the good stem cells, and inject into the spinal column. The abstracts say it seemed to help protect the motor neurons.

If you search "amyotrophic bone marrow " you will find 3 clinical trials in Spain, Korea, and Israel. And 3 dealing with injection methods somewhere.

 

[NotALS!]

I have monoclonal gammopathy, a precusor to bone marrow cancer, as well as ALS. I also have periperal neuropathy. Rituximab is sometimes used as a treatment. I'm currently on IVIG and its helped alot. I just thought it was an interesting coincidence.

 

[AfraidButNotAlone]

Here is a copy of an email I received from Dr. Kim about the use of adult stem cells and bone marrow transplant for treatment of ALS:


I'm pleased that you had been interested in our "Bone marrow mesenchymal stem cell therapy for ALS patients".

As you know, we are one of the most advanced groups for intractable neurologic diseases with various treatment protocols.



First of all, stem cell therapy for ALS had not been used commercially, yet. Only chance to treatment with stem cell is to participate in clinical trials.

At present, we had been received IND and stem cell therapy is on going in phase 1/2 “Autologous Bone marrow mesenchymal stem cell therapy for ALS patients” clinical trial. (Information about our clinical trial is in Home - ClinicalTrials.gov)

We had finished the enrollment of patients in phase 1 & 2 clinical trial in Nov, 2012.(So there is no chance to participate in this clinical trial)

Also, we had started enrollment of patients in phase 1 “HLA-haplo matched Allogenic Bone marrow mesenchymal stem cell therapy for ALS patients” (Also, Information about our clinical trial is in Home - ClinicalTrials.gov)





These 2 clinical trials are supported from Korean NIH, therefore criteria of enrollment is very strict to be a candidate of this trial, patients should be in early stage of disease, no evidences of malignancy and other combined disease, and any transmittable disease, etc. Therefore, we have to evaluate each patient very carefully for the proper selection of candidate in these clinical trials.

All the expenses within our clinical trial including screening, enrollment, treatment related cost will be provided, but, unfortunately, the expenses to evaluate fitness of patient for our clinical trial is not included.



Whole period will be taken for 8~9 months(lead-in period for 3 months, treatment period for 2 months, follow up period for 3 months), and 3 times admissions (1 time for BM aspiration & 2 times for stem cell injection) and total 9 out patient clinic visiting will be needed.



In my opinion, there are several problems in participation of clinical trial as oversea patients.

As an enrolled patients, you should visit our hospital several times according to an our schedule during clinical period.

We will provide stem cells and procedure related cost which I mentioned before, but we could not provide the expenses of flight, accommodation, etc. related with your travel to Korea.

And, because of strict inclusion criteria, we could not confirm that your patient could participate in our new trial.

It means your patient might not be enrolled after examination for fitness for our clinical trial.



So, I thought that it would be better you would manage with stem cell after we finished our first clinical trial and we received New Drug Approval. ( I expect it would be in late half of 2013)



I’m sorry for not giving you good news.

Please feel free to contact us with any subjects,

I hope I can assist you.



Thank you.



Best regards,




Seung Hyun Kim M.D. PhD

Department of Neurology

Hanyang University Hospital

Director, Cell Therapy Center for Intractable Neurological Disorders

 

[AfraidButNotAlone]

Another copy minus the links:



I'm pleased that you had been interested in our "Bone marrow mesenchymal stem cell therapy for ALS patients".

As you know, we are one of the most advanced groups for intractable neurologic diseases with various treatment protocols.



First of all, stem cell therapy for ALS had not been used commercially, yet. Only chance to treatment with stem cell is to participate in clinical trials.

At present, we had been received IND and stem cell therapy is on going in phase 1/2 “Autologous Bone marrow mesenchymal stem cell therapy for ALS patients” clinical trial.

We had finished the enrollment of patients in phase 1 & 2 clinical trial in Nov, 2012.(So there is no chance to participate in this clinical trial)

Also, we had started enrollment of patients in phase 1 “HLA-haplo matched Allogenic Bone marrow mesenchymal stem cell therapy for ALS patients”





These 2 clinical trials are supported from Korean NIH, therefore criteria of enrollment is very strict to be a candidate of this trial, patients should be in early stage of disease, no evidences of malignancy and other combined disease, and any transmittable disease, etc. Therefore, we have to evaluate each patient very carefully for the proper selection of candidate in these clinical trials.

All the expenses within our clinical trial including screening, enrollment, treatment related cost will be provided, but, unfortunately, the expenses to evaluate fitness of patient for our clinical trial is not included.



Whole period will be taken for 8~9 months(lead-in period for 3 months, treatment period for 2 months, follow up period for 3 months), and 3 times admissions (1 time for BM aspiration & 2 times for stem cell injection) and total 9 out patient clinic visiting will be needed.



In my opinion, there are several problems in participation of clinical trial as oversea patients.

As an enrolled patients, you should visit our hospital several times according to an our schedule during clinical period.

We will provide stem cells and procedure related cost which I mentioned before, but we could not provide the expenses of flight, accommodation, etc. related with your travel to Korea.

And, because of strict inclusion criteria, we could not confirm that your patient could participate in our new trial.

It means your patient might not be enrolled after examination for fitness for our clinical trial.



So, I thought that it would be better you would manage with stem cell after we finished our first clinical trial and we received New Drug Approval. ( I expect it would be in late half of 2013)



I’m sorry for not giving you good news.

Please feel free to contact us with any subjects,

I hope I can assist you.



Thank you.



Best regards,





Seung Hyun Kim M.D. PhD

Department of Neurology

Hanyang University Hospital

Director, Cell Therapy Center for Intractable Neurological Disorders

 

[AfraidButNotAlone]

As far as I am concerned, this is still good news! If this can prove effective or at least give direction, this is GREAT news

 

[Janie H]

We need to stay on top of this, late 2013 is coming soon

 

[AfraidButNotAlone]

I am EXCESSIVELY happy that they are using adult stem cells