There may be a breakthrough stem cell-based treatment for Parkinson's disease on the horizon. BrainStorm Cell Therapeutics has published papers in academic journals discussing its ability to derive dopamine producing neurons from stem cells. Now they have improved their list of potential Parkinson's disease treatments. Last month, scientists from the company announced that they had successfully:
- Taken adult stem cells from human bone marrow.
- Multiplied the sample into millions of cells.
- Used the stem cells to develop cells that produce neutrophic factors called astrocytes.
- Transplanted the new astrocytes into the brain of a healthy monkey.
- Shown evidence that the astrocytes were able to live and function in the monkey's brain.
Now BrainStorm Cell Therapeutics wants its stem cell treatments for Parkinson's disease to be the first to enter a human clinical trial. Chief Medical Advisor, Eldad Melamed, M.D., hopes those trials can be underway within a year.
Melamed hopes the science he has been developing will become a major therapy to Parkinson's disease patients. The therapy he and his colleagues have been hoping to test in clinical tials would produce helper cells called astrocytes. "We thought we'd like to move ahead and produce another type of cell from the bone marrow of adult humans," Melamed said. "And those cells that regenerated produce neurotrophic factors."
He explained that we all have and need astrocytes to produce trophic factors, or chemicals that keep neurons alive and healthy. Pointing out that neurons do not divide or rejuvenate, Melamed says that neurons need help to survive, and he credits trophic factors, such as GDNF, for having the capability of maintaining a neuron's life for up to 120 years.
That is where last month's announcement comes in. "We have produced adult human neurotraphic factor producing cells in our laboratory, flew them to Spain, and transplanted them into monkeys," Melamed said. "This was done just to see how the monkeys tolerate the cells for safety reasons."
Now Melamed and his colleagues have begun the process of designing a clinical trial that will satisfy the U.S. Food and Drug Administration (FDA), and test the new stem-cell science. The company has completed a preliminary meeting with representatives of the FDA Office of Cellular, Tissues and Gene Therapies, to discuss BrainStorm's preclinical research program in Parkinson's Disease. If the FDA approves their trial, they will be exploring the uncharted waters of stem cell-derived treatments in human brains. It is a leap for science and has potential to be a miracle for Parkinson's disease patients.
"We are extremely pleased with the collegial reception afforded by the FDA staff," said Melamed. "Because our scientific direction is based upon autologous (i.e. derived from the patient to be treated) adult stem cells, several of the well-known concerns that FDA has in the past expressed regarding stem cell therapies were much less of a factor in our conversation."
Eldad Melamed, M.D. is a world-renowned expert in the field of neurodegenerative diseases, particularly Parkinson’s disease. Prof. Melamed has served as head of the Neurology Department at the Rabin Medical Center and Tel Aviv University since 1987. He has specialized in neurology, holding senior positions at the Hebrew University (Jerusalem), Bispebjerg Hospital (Copenhagen), National Hospital (London), and the Laboratory of Neuroendocrine Regulation (Massachusetts). He is a past president of the Israel Neurological Association and former director of the National Parkinson Foundation (USA). Prof. Melamed is a member of the Scientific Committee of the Michael J Fox Foundation for Parkinson's Research.
