Phase-II clinical trials on a promising Israeli-developed treatment to halt the decline of people with the devastating neurological disease amytrophic lateral sclerosis – using stem cells taken from the patients themselves (autologous) – will soon begin in Massachusetts.

The US Food and Drug Administration announced on Monday that trials of cultured and modified adult stem cells made by Petah Tikva’s Brain- Storm Cell Therapeutics may proceed. The use of adult stem cells for the progressive, fatal disorder – known also as Lou Gehrig’s disease – was first introduced by Prof. Dimitrios Karousis, a senior Greek-born neurologist at Hadassah-University Medical Center in Jerusalem’s Ein Kerem. Earlier clinical trials showed that the stem cell treatment was well tolerated and safe.

The randomized, double- blind, placebo-controlled and multi-center trial will be launched initially at the Massachusetts General Hospital in Boston and the University of Massachusetts Memorial Hospital in Worcester, and a bit later at the Mayo Clinic in Minnesota.

The adult stem cells will be manufactured after being extracted from ALS patients’ bone marrow at Boston’s Dana-Farber Cancer Institute’s Connell and O’Reilly cell manipulation core facility. The clinical trial will include giving some patients a harmless placebo, but if patients who receive the stem cells improve, those who received the placebo will also probably get the treatment.

The trial, including the transplant by injection of the stem cells into the spinal fluid of 48 male and female ALS sufferers, aged 18 to 75, and follow-up and evaluation, will take about a year, Karousis told The Jerusalem Post. The neurologist has been treating a number of ALS patients at Hadassah for some seven years.

“Today’s announcement represents a significant milestone BrainStorm has achieved to date,” said BrainStorm president Chaim Lebovits. “More importantly, in our view, it has positive implications for the entire ALS community, indicating that the FDA recognizes the significant clinical potential of transplantation with our autologous, differentiated mesenchymal stem cells. We are excited to begin the US trial and are optimistic that the Phase II data will confirm and build upon the positive indications of clinical benefit that we observed in earlier studies.”

The team, which included Hadassah neurology department head Prof. Tamir Ben-Hur, started work in 2007. “Three years later, we joined Brain- Storm, which combined the cells with hormones, including growth factors, to improve the outcomes,” said Karousis.

“Approval for use on patients was given by the Health Ministry in 2011. Ten of the 15 patients in the Hadassah trials responded or stabilized, and the disease was halted, with their breathing improved. About three of them even showed that the disease had receded, with them improving dramatically. However, the treatment is not permanent; the injection probably has to be repeated after several months.”

Karousis noted that he has injected stem cells, with promising results, also into victims of the progressive form of another neurological disorder, multiple sclerosis (MS) – which until then has had no treatment at all and usually causes a quick decline.

The modified cells are created by removing large numbers of adult stem cells from their bone marrow and multiplying them in culture. The process of preparing hundreds of millions of adult stem cells over the period of a month is expensive, as clean rooms are needed to prevent infection. ALS is an “orphan disease” affecting a limited number of patients – perhaps only tens of thousands in the world and 500 in Israel – and there is no other significant treatment.

If the phase-2 trials prove successful, Karousis expects that the FDA will then approve their use on patients not included in experimentation. The neurologist will go to the US to advise in the performance of the clinical trials. Eventually, the technique could be considered for other diseases of neurological degeneration such as Parkinson’s, Alzheimer’s and stroke, he suggested.

“Since we began at Hadassah, there has developed much awareness in ALS and adult stem cells. There are now 300 studies in the field around the world. It was our idea to inject the modified cells into the spinal fluid,” explained Karousis, who is also chairman of the 200-member Israel Society for Neuroimmunology.

“Following successful completion of the technology transfer process for BrainStorm’s NurOwn cells, we are looking forward to collaborating on this multi-center study as the cell production facility for the Massachusetts clinical sites,” added Prof. Jerome Ritz of the department of internal medicine at Harvard Medical School and director of the Connell O’Reilly cell manipulation lab at Dana-Farber.

In healthy people, motor neurons reach from the brain to the spinal cord and from the spinal cord to the muscles throughout the body. In ALS, there is progressive degeneration of the motor neurons, eventually leading to death. With voluntary muscle action progressively affected, patients in the later stages of the disease may become totally paralyzed; but in most cases, the patients’ mental faculties are not affected.

In MS, the insulation on nerves is destroyed by the patients’ own immune system, causing a “shorting” of the electrical messages and thereby affecting many neurological functions. There appear to be more MS sufferers, because MS patients tend to live much longer, some for 30 years or more, while the life expectancy of an ALS patient averages between two to five years from the time of diagnosis.

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