Autologous Stem Cell Transplants in ALS: New Hope and Preliminary Findings

 Joanne P. Shelby-Klein, BSN RN

Amyotrophic Lateral Sclerosis (ALS) is also known as Lou Gehrigs Disease, named after the famous baseball player who died from the disease. People may also be familiar with this disease from hearing about Steven Hawking, the famous physicist.

What exactly is ALS?

ALS belongs to a group of rare neurological diseases.  It affects nerve cells (motor neurons) responsible for controlling the functions of voluntary muscle.

Right now, there is no cure for ALS and no effective treatment that can stop, or reverse the advancement of the disease.

Diagnosis of ALS

A few of the tests are given below:

  1.  electromyography (EMG) and
  2. Nerve Conduction Velocity tests to determine how the nerves and muscles are working
  3. Blood and urine analysis to look for abnormal proteins and heavy metals.
  4. MRI’s are also done to look for lesions.
  5. More invasive tests are also necessary including a
    1. spinal tap
    2. myeogram, or
    3. injection of dye into the cervical spine,
    4. muscle and nerve biopsies, and
    5. a thorough neurological exam to rule out all other causes of the patients symptoms.

These tests and exams should be completed and evaluated by a neurologist who is a specialist in caring for patients with ALS and sometimes second opinions are recommended.

Management of ALS Patient

Once a person is diagnosed with ALS, they will require routine lifelong care by a neurology team, who has expertise in caring for ALS patients. Part of the routine care is an ongoing evaluation using the ALSFRS known as the ALS Functional Rating Scale. This test is performed by the doctor to estimate how impaired the patients normal daily functioning actually is. The test is quite easy to administer. Twelve common tasks are looked at and rated on a scale from 0 to 5. If the person scores a zero it means they cannot do the task. A score of 5 means they can do the task normally. All 12 tasks are then added together to give a total score. A score of 0 is the worst and a score of 48 means all functioning is normal. This test is done initially and then with each doctor visit with all results compared to the previous tests.

Can ALS be cured?

There is no known cure for ALS at this time. It is a chronic illness that gets progressively worse over time.

  1. Patients lose their ability to move without assistance
  2. Eventually are unable to chew or swallow and become unable to breathe on their own

A company in Israel called BrainStorm Cell Therapeutics is currently involved in a Phase I/II Clinical trial that uses autologous (patients own) bone marrow stem cells. They released preliminary findings that were hopeful. According to their website, this company is working on the first of its kind adult stem cell treatment that is produced from the patient’s own bone marrow cells and used for the treatment of ALS. The treatment is patented in Israel and the United States as NurOwn.

The preliminary report shows that NurOwn was given to over 30 patients with ALS in Israel in 2014. The trial was designed to test the safety and effectiveness of the medication and was supervised by Dr. Dimitrios Karussis of the Department of Neurology at Hadassah Medical Center in Jerusalem. Patients received a combination of Intramuscular (IM) and Intrathecal (into the Spinal Column) injections, as a single dose, and followed for six months after the stem cell transplantation. The report showed 12 patients, who had three or more months follow up, 92% showed a slowing in disease progression or actual improvement on the ALS Functional Rating Scale administered during routine visits. This stabilization or improvement occurred with just one dose of NurOwn. The patients all tolerated the injection and encountered no risks or side effects. It is important to know that the initial patients in the study all had early stage or Progressive ALS.

These findings caused the United States Food and Drug Administration to list NurOwn as an Orphan Drug and put it on a fast track, or rapid process, to approve for use in patients with ALS. As a result of this, the United States is able to begin a Phase 2 Clinical trial to further test the safety and effectiveness of the drug and to see if multiple doses of NurOwn would be beneficial. Currently three centers are involved in the Phase 2 study, the University of Massachusetts School of Medicine at Massachusetts General Hospital in Boston and Worchester and the Mayo Clinic in Minnesota. A total of 48 patients will be enrolled in the study.

The actual study title is “Phase 2 Randomized, Double Blind, Placebo, Controlled Multicenter Study of Autologous MSC-NTF Cells in Patients with ALS. (NurOwn)” ( ,2015). MSC-NTF stands for Mesenchymal Stem Cells – Neuro Trophic Factor. Mesenchymal stem cells are usually found in the bone marrow and can be retrieved from there using a needle biopsy. NeuroTrophic Factors are a specialized family of proteins that form, maintain and grow neurons for both the central nervous system and the peripheral nervous system. The medication was given the name NurOwn and will deliver the very potent NeuroTrophic Factors directly to the sites of nerve damage. This study will evaluate the safety and effectiveness of NurOwn in the 48 enrolled patients and help determine if this new or novel approach to treating ALS is as safe and effective as was demonstrated in the initial studies performed in Israel. The Clinical Study began enrolling patients in May 2014 and will continue until April 2016.

As in all Clinical Trials or Studies, the research team will look for specific outcome measures or goals. Patients will either receive a Placebo or the actual NurOwn Treatment. Both the patient and study team will not know who received the placebo or the NurOwn. Therefor all patients in the study will be evaluated as follows:

  • The number of patients who have adverse events or bad reactions to the treatment at the time of all study visits, numbers 1- 10.
  • Any changes in the slope of ALS Functional Rating Scale from the pre-treatment/transplant period to the post- transplant period over a 24 week period of time.
  • Any changes in FVC ( Forced Volume Capacity of the lungs) slopes from pre-transplant to 24 weeks. FVC is a measurement of the volume of air exhaled after a full inhale. In patients with ALS this number can be mildly to severely abnormal.

Who is Eligible to Participate in the Study

  • Men and women between the ages of 18 to 75 years of age.
  • Patients must have laboratory support that indicates a probable or definite diagnosis of ALS as defined by the standard criteria used to diagnosis ALS.
  • The starting point of ALS is considered to be the first reported occurrence of weakness, spasticity (leg is very rigid or tight with an exaggerated knee jerk) or bulbar symptoms such as difficulty speaking , swallowing, or breathing. The symptoms must have started more than 12 months prior to enrollment but not lasted more than 24 months.
  • Current symptoms must include leg and arm weakness.
  • The patients ALS Functional Rating Scale Score must be greater than or equal to 30 at the time of the initial screening visit.
  • Upright Slow Vital Capacity (measure of lung capacity) of greater than 65% of results for same gender, height and age at the time of the screening visit.
  • Taking a stable dose of Riluzole for a minimum of 30 days at time of enrollment or have not been taking it for at least 30 days.
  • Must be able understand the consent forms and provide a signature on the consent forms.
  • Must be able to read, understand and follow all study procedures including attending all study visits.
  • Must consent to a lumbar puncture, spinal tap.
  • Women of child bearing age must agree to consistently use 2 separate forms for birth control methods and not become pregnant during the study. They must also agree to notify the study team if they should become pregnant.
  • A pregnancy test will be completed one week prior to the bone marrow aspiration of the stem cells.
  • Males must also consistently use 2 separate forms of birth control during the course of treatment.
  • Have never received a Stem Cell treatment of any kind
  • Never had an immune system disorder including leukemia, lymphoma or related disorders
  • Never been treated by whole body radiation.
  • No history of cancer for 5 years prior to enrolling in the study.
  • Not taken medication to suppress the immune system within 4 weeks of the screening visit.
  • Never had a hip fracture.
  • No history of a spinal curvature or a severe spinal curvature.
  • Not be using either non-invasive or invasive assistance to breathe
  • Not using a feeding tube for nutrition.

Once a patient is accepted into the study, they will either be assigned to receive the investigative NurOwn treatment or a placebo. Neither the patient nor the study researchers will know who is getting the actual treatment or the placebo. That will be controlled by computer and the NurOwn Manufacturer. Both sets of patients will undergo the same procedures and followed in exactly the same way.

  • Patients will be followed for three months after enrollment before receiving either the placebo or the study drug.
  • During the 3 month pre-transplant period the patient will undergo a bone marrow aspiration (using a long needle and relatively painless) to harvest some bone marrow. This bone marrow will be used to isolate the Mesenchymal Stem Cells so they can expand and grow to the needed numbers of cells. Once the cells have expanded they will be worked with so they can put out the needed Neuro Trophic Factors.
  • After three months, the patient will receive two injections, one into the muscle (Intramuscular) and one into the spinal column (Intrathecal). These two sites are used as they are the target ALS affected neuron sites.
  • Patients will be monitored on a specific, periodic schedule using the ALS Functional Rating Scale as well as physical and clinical examination to determine any side effects, adverse reactions and if stabilization and or improvement in symptoms ,such as breathing, and disease progress has occurred. The study will last approximately 9 months.

Three Medical Centers in the United States are currently approved to conduct the study and are enrolling patients. 2 of the facilities are located in Massachusetts and are affiliated with the University of Massachusetts School of Medicine at Massachusetts General Hospital in Boston and Worchester. The Mayo Clinic in Minnesota is the third facility The NurOwn to be used in the study will be manufactured at the Dana-Farber Cancer Institute’s O’Reilly Cell Manipulation Core Facility and will be responsible for dispensing the product as needed at the three facilities.

While the report of the original study and the approval of the larger phase 2 study in the United States, is preliminary, it does offer some hope for those who daily deal with the diagnosis of ALS. Currently there are more than 12,000 people with the diagnosis of ALS in the United States alone, for an incidence rate of 3.9 for 100,000 people. To be able to stabilize patients, stop progression of the disease and give those affected a better quality of life is a worthwhile goal. Researchers are hoping that NurOwn will be the next step in achieving that goal.

References for articles



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