Bone marrow stem cells – the transplantation of such dopamine-producing cells in rodent models of PD resulted in the amelioration of their Parkinsonian symptoms. Brainstorm Cell Therapeutics has developed a novel stem cell therapy to treat Parkinson’s Disease – using a patient’s own bone marrow stem cells to produce the missing chemical that enables restoration of motor movement.
The process – which successfully alleviated symptoms of Parkinson’s in rats – will be tested on monkeys next year, with human clinical trials scheduled for the following year.
About 1.5 million Americans have been diagnosed with Parkinson’s, a chronic progressive neurodegenerative disease. Parkinson’s affects those brain cells responsible for production of dopamine, the neurotransmitter that directs motor movement. Insufficient dopamine levels result in tremor, rigidity, slowness of movement and impaired balance.
The Tel Aviv-based BrainStorm uses adult stem cells to repair neurological damage. Developed at Tel Aviv University, the company’s propriety technology – NurOwn – has been proven capable of generating neuron-like cells derived from human bone marrow. The cells produce dopamine which can them be implanted into the PD patients.
NurOwn was developed by Prof. Eldad Melamed, Head of Neurology of the Rabin Medical Center and member of the Michael J. Fox Foundation for Parkinson’s Research, together with Tel Aviv University cell biologist Dr. Daniel Offen and Dr. Yosef Levy.
In June, 2004, BrainStorm acquired the exclusive worldwide rights to commercialize NurOwn technology through a licensing agreement with Ramot, the technology transfer company of TAU.
Along with these scientists, BrainStorm’s team of 12 researchers is working to further develop this technology. Taking bone marrow cells from rodents and human donations, the researchers demonstrated expansion of the number of cells and their differentiation into functional neural cells.
“The breakthrough here is that, first of all, our source material is bone marrow. Second, they showed not only neuronal markers and electro-physical functions, but the in-vitro expression and release of dopamine,” BrainStorm’s President and CEO, Dr Yaffa Beck told ISRAEL21c.
Perhaps most impressive, transplantation of such dopamine-producing cells in rodent models of PD resulted in the amelioration of their Parkinsonian symptoms. This is “proof of the feasibility of these cells,” says Beck, who has over 20 years experience in the pharmaceutical industry and business development.
Beck said that the tests have succeeded in showing that the cells stay in the brain, producing dopamine for up to four months.
Pointing to the advantages of using adult stem cells derived from bone marrow, Beck explains that the therapy can be autologous – meaning it can employ the patient’s own bone marrow.
“Using the patient’s own bone marrow to supply dopamine-producing cells circumvents problems of immunity and compatibility, so it is safer than using cells from an outside source,” says Beck.
She said that the advantage of adult bone marrow cells is that they can be taken from the patient, so there is no rejection or infection. The body identifies the cells as belonging to the patient.
“This is precisely our innovation, compared with other stem cell research companies. We’re talking about adult stem cells,” she told Globes.
In addition, she explains, such therapy is not fraught with the ethical issues or risk of cancer sometimes associated with embryonic stem cell therapies.
While anti-Parkinson’s drugs exist, they all have significant drawbacks. “There are drugs that replace dopamine itself, but their effects are limited,” says Beck. “The concentrations that reach the brain are not consistent, so dosages must be steadily increased, causing side effects and ‘resistance’. After few years, patients often stop responding altogether.”
As cell treatment involves implantation of cells in the brain, it would obviate the need for drugs. “In place of patients’ degenerating cells, we put in new functional cells,” says Beck, adding that this promises to be a long-term treatment.
“It’s important to distinguish between drugs for treating Parkinson’s and stem cell treatments for it,” she told Globes. “We hope that our treatment will be like a full cure. I want to be cautious, and not cause misleading over-expectations, but our hope is that a person will not need further treatment for a very long time, in contrast to other drugs. It’s possible to compare it with bone marrow transplants for cancer patients.”
Why is BrainStorm, which aims to address a wide range of neurodegenerative diseases, initially focusing on Parkinson’s Disease?
“Parkinson’s is one of the few neurodegenerative diseases that has a clearly defined deficiency, not unlike diabetes,” says Beck. “In Parkinson’s, we need dopamine-producing cells. With other diseases, it is not so apparent which cells are required, so it will take longer to develop an appropriate therapy for them.”
Having successfully demonstrated this therapy on rodent models of PD, Beck says the company plans to develop similar solutions for such illnesses as Multiple Sclerosis (MS), Alzheimer’s Disease (AD), Amyotropic Lateral Sclerosis (ALS) and other terminal neurodegenerative diseases
Israeli stem cell companies, says Beck, are in a unique position within the world research arena.
“Some of the world’s best stem cell scientists are in Israel, primarily in the area of embryonic stem cell research,” she says. “In this field, the right people have come together at the right time – and more than that, we’re Jewish,” she adds, referring to the Jewish tradition in which embryos are not considered to be human beings until they are born. This is in sharp contrast to the Christian view, which commonly holds that personhood starts at the moment of conception.
“This different way of looking at the embryo has helped advance stem cell research,” says Beck. “There has been far less of a debate regarding its ethics.”
Beck also praises the recently established Consortium Bereshit for Cell Therapy, a $15 to $20 million project of the Israel Ministry and Trade that brings together researchers from academia and industry to develop stem cell-based therapies.
“There is a nice critical mass of university researchers and companies in the Consortium. Now it’s a matter of grabbing the opportunity, providing as many incentives as possible and moving ahead in this important area.”