Adult Stem Cells Treat Heart Failure

In 2007, Thomas Clegg was diagnosed with dilated cardiomyopathy, a condition in which the left ventricle of the heart becomes abnormally enlarged. At that time, his ejection fraction – a measure of the heart’s efficiency at pumping blood – was around 9 (nine) percent, whereas a normal ejection fraction is considered to be between 50 and 65%, or higher.

As Mr. Clegg describes, "I was one of the lucky ones. I was still working 8 hours a day. Some people who have 30% ejection fractions can’t walk across the room."

One of his physicians, Dr. Kevin Lisman, informed Mr. Clegg that his best option would be a heart transplant. However, precisely because of the fact that Mr. Clegg was still able to work and remain physically mobile throughout the day, he was very low on the list of recipients, since other people who were in worse condition took precendence.

Then, another option was offered to Mr. Clegg: autologous adult stem cell therapy. Mr. Clegg eagerly accepted the option. According to Dr. Brian Bruckner, who conducted the adult stem cell procedure at the Methodist DeBakey Heart and Vascular Center in Houston, "He didn’t really have a lot of options. Injecting cells directly is kind of a novel idea. We know they help, but we’d like to know more about that mechanism." As Mr. Clegg adds, "My wife and I just wanted to see what was going on. At this point, I would have had to get worse to get a transplant."

In an FDA-approved, Phase II, double-blind, placebo-controlled, multi-center clinical trial in which approximately 40 patients throughout the country participated, Mr. Clegg was the first to receive the adult stem cell treatment, which consisted of treating Mr. Clegg with his own (autolgous) adult stem cells that were harvested from his own bone marrow via his hip. After the doctors in Houston removed 50 milliliters of Mr. Clegg’s bone marrow, it was shipped to Aastrom Biosciences in Ann Arbor, Michigan where the adult stem cells were isolated, expanded and returned to Mr. Clegg’s doctors, who administered the cells directly into Mr. Clegg’s heart.

According to Aastrom CEO George Dunbar, "Lifesaving is where we wanted to focus our efforts. It helps with what nature already does with these mixed cell populations."

Now, nine months after receiving the autologous adult stem cell treatment, Mr. Clegg’s ejection fraction has increased to 30%.

Mr. Clegg has nothing but the highest praise for the medical team in Houston, especially his nurses. As he emphatically states, "They’re my guardian angels. What they’re trying to do is back the clock up. I honestly feel like this has added some time."

New Adult Stem Cell Facility Opens in Maryland

RNL Biostar Inc., a subsidiary of the Seoul-based company RNL Bio Inc., has been based in Rockville, Maryland since 2006. Now, in addition to its facility at the Technology Development Center, it plans to add a new facility in Germantown, worth $6 million, to which the company also plans to add 50 newly hired employees over the next 3 to 5 years. The new positions will include manufacturing technicians and lab personnel in addition to administrative personnel. According to Donna Lee, director of business development at RNL, "There will be pretty big growth spurts because of the amount of work we have."

The new RNL facility will be converted from an already existing structure, which currently occupies nearly 10,000 square feet and comprises a combination of labs and office suites. RNL Biostar projects that its facilities will occupy 20,000 square feet by 2014.

Currently RNL is in Phase II clinical trials for the testing of its proprietary adult stem cell products for the treatment of osteoarthritis and Buerger’s Disease, which is an inflammatory and blood clotting condition. Additionally, the company has received FDA authorization to commence clinical trials with its adult stem cell product in the treatment of spinal cord injury, which are scheduled to start at the end of the year.

RNL’s proprietary products are formulated from adult stem cells that are harvested from adipose (fat) tissue. According to Ms. Lee, "In Maryland, the fat samples come to our lab here first. People send in their fat and we extract stem cells out of the fat and ship them in nitrogen tanks to Korea, where cells are cultured and multiply. We have helped a lot of people with rheumatoid arthritis with a one-time IV injection. It’s really amazing to watch."

However, the U.S. citizens who donated their own adipose-derived adult stem cells must travel to China or Japan for the injections, since such treatments are not allowed in the U.S., due to a severely antiquated FDA.

RNL has also developed a stem-cell-based hand cream.

Olympic Medalist Endorses Cord Blood Bank

Shannon Miller, a 7-time Olympic Medalist and 9-time World Medalist, has signed a formal endorsement with StemCyte, one of the leading cord blood banks in the world. According to the terms of the agreement, Ms. Miller will be featured in a series of public awareness and advertising campaigns for the company.

In addition to her accomplishments as an Olympic gymnast, Ms. Miller is also a lawyer, having graduated in 2007 from Boston College Law School. She and her husband, John Falconetti, are expecting their first child in November of this year.

According to Ms. Miller, "As a former Olympic gold medalist, I’ve always sought out the best and healthiest decisions in life. As my husband and I have begun planning our family’s future, we have been thoroughly investigating the finest decisions for our baby. Cord blood banking was a no-brainer for us, and StemCyte was the obvious choice for where we wanted to store our baby’s cord blood."

In a statement made by Calvin Cole, vice president of operations in North America for StemCyte, "We were thrilled when Shannon approached StemCyte. We felt that as a world class athlete, expecting mom and role model for millions around the world, she was a perfect fit for StemCyte. As a decorated athlete who knows what it takes to be the best in the world, we’re thrilled that she clearly recognized StemCyte as the world’s best choice for parents and their cord blood banking decisions."

Headquartered in California and New Jersey, StemCyte also has facilities in India and Taiwan. As stated in their press release, StemCyte has supplied over 1,000 cord blood products for over 40 life-threatening diseases to over 200 leading worldwide transplant centers. StemCyte is also involved in the development of new umbilical cord-blood-based therapies, and is supporting the largest clinical study for the use of unrelated cord blood transplantation in the treatment of thalassemia, one of the most common genetic diseases in the world. Additionally, StemCyte is also supporting trials investigating regenerative spinal cord therapies. StemCyte has been contracted by the U.S. Department of HHS (Health and Human Services) to help establish a public National Cord Blood Inventory for unrelated transplant units.

Four-Year Prison Term Sought in South Korea for Stem Cell Fraud

South Korean prosecutors have told a Seoul court that Hwang Woo-suk should receive a four-year prison sentence for the scientific fraud that he committed in 2004 and 2005.

In addition to violation of bioethics laws, Hwang is also being charged with misappropriation of 2.8 billion won (approximately 2.25 million USD) in state funds. Although Hwang has apologized for his fraudulent reports, he has denied personal culpability, claiming that he was misled by junior researchers in his laboratory. Nevertheless, as one of the prosecutors explains, "The disappointment felt by the Korean people is enormous."

Formerly considered to be one of the world’s leading experts in the field of stem cell research, Hwang suffered a catastrophic fall from the prestige that he had previously enjoyed when he was found, in November of 2005, to have fraudulently reported the successful cloning of human embryonic stem cells. Specifically, he had authored two articles which were published in the highly respected journal Science in 2004 and 2005, and which initially catapulted Hwang into the international spotlight for his reports of the world’s first successful creation of human embryonic stem cells by cloning. The articles were later editorially retracted, however, when they were discovered to contain fabricated data, and Hwang quickly fell from fame to infamy. He subsequently confessed to charges of fraud, and was "indicted on embezzelment and bioethics law violations linked to faked stem cell research", according to media reports at the time. Additionally, he was expelled from the Seoul National University which had previously employed him, and the S. Korean government rescinded its financial support of his research and banned him from conducting further experiments on human cloning. To this day, the scandal has impacted S. Korean national law, which brought a formal halt to all experimentation on human cloning until this year. As Dr. Oh Il-hwan, a bioethics professor at the Catholic University Medical School in Korea, explains, "Hwang’s fall discouraged the government from supporting stem cell studies. It also meant that researchers in the field were the objects of scorn." Nevertheless, an investigational team at Seoul National University did verify that Hwang’s team had successfully produced the world’s first cloned dog, an Afghan hound named “Snuppy”.

Hwang is not without his supporters, who have staged high-profile, emotional rallies in the court, imploring a verdict that will allow Hwang a second chance. According to Lee Bong-gu, a lawyer for Hwang, "The prosecutors are trying to tear apart Hwang’s precious scientific evidence."

In 2006, with financial backing from his supporters, Hwang founded Sooam Biotech Research Foundation, which specializes in the cloning of animals and has produced several cloned dogs.

Hwang’s trial has been dragging on for three years and is predicted to continue into a fourth year, having been bogged down by technical testimony from numerous scientists who have testified about Hwang’s research. It is not known when a final verdict might be decreed.

Skin Cells Turned into Retinal Cells

Researchers at the University of Wisconsin at Madison are once again in the news headlines. This time, they have reprogrammed skin cells into retinal cells. The implications of such an accomplishment offer new hope to millions of people who suffer from vision problems.

Led by Drs. David Gamm and Jason Meyer, the team of researchers took ordinary fibroblasts and de-differentiated them into a more primitive state known as iPS (induced pluripotent stem) cells, a procedure which is becoming increasingly common in research laboratories throughout the world. The iPS cells, which resemble embryonic stem cells in their pluripotency, were then re-differentiated into retinal cells. Additionally, the scientists also compared the differentiation of iPS cells into retinal cells with the differentiation of embryonic stem cells into retinal cells, and both processes appeared to progress through the same stages over the same amount of time. Both types of cells developed first into neural cells, which then differentiated further into cells of the primitive eye field, which in turn differentiated further into the more specialized cells of the retina, which is the light-sensitive, image-forming membrane on the inside of the eye, without which vision is not possible. The final cells included retinal pigment epithelium cells as well as photoreceptor cells, both of which are commonly lost in diseases of the eye such as retinitis pigmentosa and age-related macular degeneration, the latter of which is estimated to afflict approximately 30 million people around the world, and which in the U.S. is the leading cause of blindness in people 55 years of age and older.

As Dr. Gamm explains, "If you understand and follow every stage of building a photoreceptor, you’re more confident because you’ve observed the assembly line. We can understand the disease better and see if there are ways to protect the cells or slow down the disorder." It is still not yet known, however, if these new retinal cells can actually function in vivo in the same manner as naturally occurring retinal cells. As Dr. Meyer adds, "That is among the next projects we’re working on."

First pioneered in 2007, simultaneously by Dr. James Thomson of the UW-Madison as well as by Dr. Shinya Yamanaka at Kyoto University in Japan, iPS cell technology is now considered to be one of the most promising fields in regenerative medicine. With the same pluripotency as embryonic stem cells, but without any of the ethical dilemmas and controversies, iPS cells seem to be able to do anything and everything that embryonic stem cells can do.

According to Dr. Robert Lanza, chief scientific officer at Advanced Cell Technology, who did not participate in the current study but has conducted similar research, "This is an important paper. It underscores the enormous potential of iPS cells, not only for treating diseases such as blindness, but as an important tool for understanding eye development. I think it will only be a matter of time before these iPS-derived cells are used in the clinic to help restore vision in patients."

Indeed, according to Dr. Gamm, who believes that clinical trials for retinal cell replacement could be conducted within the next 5 years, "That’s not pie in the sky anymore. I couldn’t have said that, even two years ago."

India Boasts Profitable Adult Stem Cell Market

In sharp contrast to the regulatory setbacks experience in other nations, such as the U.S., India offers a commercially and legally attractive environment for adult stem cell entrepreneurs.

At last count, at least 15 new firms are scheduled to enter the Indian market, and 4 already existing firms are planning major expansion. In the latter category are Reliance Life Sciences, LifeCell International, Regenerative Medical Services, and Cryo-Save India. Though each of these companies is already supplying cell cultures to select hospitals, they are all planning even further expansion.

According to the Indian Council for Medical Research (ICMR), the 15 new firms who are scheduled to enter India’s market include private commercial ventures as well as academic research institutions. Additionally, a number of organizations are conducting clinical trials with adult stem cells in India, including but not limited to Stempeutics, the LV Prasad Eye Institute, the Nichi-In Centre for Regenerative Medicine, the Centre for Cellular and Molecular Biology, the National Centre for Cell Science, and Manipal Hospital in Bangalore, among others. According to the ICMR, at least 60 adult stem cell clinical trials are currently underway in India, most of which involve autologous (in which the donor and recipient are the same person) adult stem cells, and all of which are approved by the Drug Controller General of India. The entire field of adult stem cell therapy has enjoyed a new boost in India as a result of the recent decision by the Indian government to allow the patenting of micro-organisms and related laboratory processes.

According to Mayur Abhaya, executive director of LifeCell International, based in Chennai, the greatest therapeutic value thus far has been found in the type of adult stem cell known as mesenchymal stem cells (MSCs), which are already being used by doctors in hospitals in India to treat a wide variety of ailments, which include diabetes, heart disease, stroke, arthritis, multiple sclerosis, spinal cord injury, and blood disorders, to name a few.

LifeCell, which is currently the leading company in India for the storage of umbilical cord blood stem cells, has already banked cord-blood-derived stem cells for more than 12,000 clients. The company, which is also collaborating with Cryo-Cell in the U.S., is now planning to expand into the collection and storage of adult stem cells derived from bone marrow aspirate. Additionally, LifeCell has also begun clinical trials with Harvest Therapeutics in the treatment of critical limb ischemia with adult stem cells, and is planning further expansion into the storage of menstrual blood, now known to be a rich source of highly potent adult stem cells. As Mayur Abhaya further explains, "A few more clinical trials are being planned in other disease settings, apart from the proposed collaboration with Cryo-Cell to launch the menstrual blood stem cell banking service in India."

With a population of approximately 1.2 billion people, India ranks as the second most populous nation on earth, and the world’s most populous democracy. Second only to China, whose population is estimated at 1.3 billion people, India actually offers a larger and freer commercial market than can be found in China, which is still formally Communist and is therefore still highly restricted in the nature and extent of its commerce. It is hardly surprising, therefore, that entrepreneurs of all types eventually cast a serious eye toward India. Especially with the outdated federal regulatory laws that still govern medical research in other countries, such as the U.S., it is only logical and to be expected that adult stem cell companies would discover in India a welcoming atmosphere in which they are able to thrive.

New NIH Director Emphasizes Practical Science

A highly accomplished geneticist who is not afraid to express his religious convictions, Dr. Francis Collins assures the public that his focus as the new director of the National Institutes of Health (NIH) will be on science, not religion.

As Dr. Collins publically stated today, "The NIH director needs to focus on science. I have no religious agenda for the NIH."

In his first interview before formally greeting the employees of the $40-billion agency that he has been appointed to direct, Dr. Collins added, "Here we are at a circumstance where I think our country is seeking maybe to redefine our image a bit in the world, from being the soldier to the world to being perhaps the doctor to the world. I’d like to see that happen."

President Obama announced the appointment of Dr. Collins as the new director of NIH on July 8 of this year, a decision which was met with mixed reactions from the media. Although Dr. Collins enjoys a widely respected, sterling reputation among scientists, less-informed critics in the media were concerned, at least initially, that the religious views which Dr. Collins espouses may interfere with an objective and impartial scientific approach to directing NIH. Among other responses, an article in U.S. News & World Report at the time was entitled "Obama Names an Evangelical to Lead the NIH", claiming that "Obama this week picked a Bible-believing, loud, and proud evangelical Christian to head the National Institutes of Health." Today, however, Dr. Collins may have put such concerns to rest.

Although Dr. Collins has been described as an "evangelical Christian who has defended the inherent compatibility of science and religious beliefs", it is clear that any personal religious views to which Dr. Collins might ascribe have certainly not interfered with his scientific objectivity in the past, and he has already directed a number of very high-profile scientific projects. Several other media sources offered more scientifically based assessments of his appointment, such as the Wall Street Journal which simply reported that, "Obama Plans to Name Renowned Geneticist to Head NIH."

Dr. Collins earned a B.S. in chemistry from the University of Virginia in 1970, a Ph.D. in physical chemistry from Yale in 1974, and an M.D. from the University of North Carolina at Chapel Hill in 1977. He first distinguished himself at the University of Michigan where he gained recognition for his pioneering work in positional cloning, a type of genetic screening and gene identification technique. In 1993 he accepted an invitation to succeed Dr. James Watson (Nobel laureate and co-discoverer with Dr. Francis Crick of the helical structure of DNA) as director of the National Human Genome Research Institute (NHGRI), one of 27 institutes within NIH, which he directed from 1993 to 2008. During this time Dr. Collins also became director of the Human Genome Project, an international research project which resulted in 2003 in the successful mapping of the genetic sequences of 3.1 billion chemical base pairs in the human DNA. Along with Craig Venter, founder and former president of Celera Genomics as well as the founder of The Institute for Genomic Research, Dr. Collins was awarded the "Biography of the Year" award in 2000 by the A&E Network, and U.S. News & World Report together with the Harvard Center for Public Leadership named both Collins and Venter "America’s Best Leaders" in 2005.

In 2006 Dr. Collins authored the book, "The Language of God: A Scientist Presents Evidence for Belief", in which he describes scientific discoveries as an "opportunity to worship". Dr. Collins awknowledges that he has been highly influenced by C.S. Lewis, especially by the book "Mere Christianity" to which Collins attributes his conversion to Christianity at the age of 27. Prior to his appointment as the new director of NIH, Dr. Collins has most recently served as president of The BioLogos Foundation, which he founded in 2009, the mission of which is to "contribute to the public voice that represents the harmony of science and faith."

Dr. Collins has received numerous awards and honors throughout his career, including election into the National Academy of Sciences and the Institute of Medicine. Additionally, he is the recipient of the Presidential Medal of Freedom, the highest civilian honor awarded by the President of United States.

As President Obama himself stated when he made the official announcement on July 8, "My administration is committed to promoting scientific integrity and pioneering scientific research, and I am confident that Dr. Francis Collins will lead the NIH to achieve these goals."

This year the NIH will have $40 billion at its disposal for the funding of medical research, including that which is to be conducted on human embryonic stem cells.

UC-Davis Receives Adult Stem Cell Grants

The California Institute for Regenerative Medicine (CIRM), the state legislative stem cell agency, has announced today the awarding of two separate multi-million-dollar, multi-year grants to the University of California at Davis for the R&D of adult stem cell therapies in the treatment of neurodegenerative diseases as well as inherited blood and metabolic disorders.

The two grants include $2.7 million to be devoted to the development of a treatment for Huntington’s disease using the type of adult stem cell known as mesenchymal stem cells (MSCs), and a separate grant worth $4.2 million to develop a safe treatment of infants in utero who may be predisposed to inherited blood cell disorders such as sickle cell anemia and thalassemia, among others.

According to Dr. Jan Nolta, director of the Stem Cell Program at UC-Davis and a principal investigator for the neurodegenerative diseases grant, "Today’s award from CIRM gives us a terrific boost because it opens the door to developing an innovative and effective therapeutic tool for victims of neurodegenerative disorders like Huntington’s disease. Diseases of the brain are incredibly challenging to treat. What we hope to do is create a stem-cell-based treatment that can deliver therapeutic molecules directly into damged cells in the brain."

In the U.S., Huntington’s disease occurs with a frequency of one person per every 10,000 people. Dr. Nolta has been working with MSCs for the past 20 years, during which time the cells have demonstrated their vast therapeutic versatility. Under the new grant, Dr. Nolta and her colleagues intend to re-engineer the MSCs to deliver siRNA (small interfering ribonucleic acid) to damaged areas of the brain, where the siRNA can prevent the mutant proteins that are characteristic of Huntington’s disease from being produced by cells. Hoping to exploit the natural "search and rescue" capacity of MSCs to target damaged regions of the brain, Dr. Nolta expects that such a therapy will preclude the need for systemic infusion of cell-based therapies into the CNS (central nervous system).

According to Judy Roberson, president of the Northern California chapter of the Huntington’s Disease Society of America, "This is the best news. Families like mine have been waiting for an intervention for Huntington’s since the gene was located 16 years ago. My husband and his brother and thousands of others died waiting for something, anything. Now, thanks to CIRM support and the research at UC-Davis, there’s real hope for the first time."

In the second grant, a team of UC-Davis researchers led by Dr. Alice Tarantal will be exploring an in utero model to assess the viability of transplanted stem cells in the treatment of inherited blood disorders, before irreversible damage from the diseases has occurred. Professor of pediatrics and associate director of the UC-Davis Stem Cell Program, Dr. Tarantal explains, "Our team is poised to address crucial bottlenecks that have delayed these therapies from reaching our youngest patients. We greatly appreciate the support provided by CIRM to help us work toward these goals. Infants with congenital blood diseases can be diagnosed before they are born. We hope it may be possible to treat these young patients early and thus avoid the many problems such diseases can cause."

With more than 125 scientists and physicians on its faculty, UC-Davis is currently in the process of constructing a new Institute for Regenerative Cures in Sacramento, a facility that is supported by the CIRM and which will comprise 90,000 square-feet of state-of-the-art research laboratories and clinics.

Adult Stem Cells Treat Parkinson’s

At Jaslok Hospital in Mumbai, 55-year-old Bhawarlal Jain has received autologous adult stem cell therapy for the treatment of Parkinson’s disease. Mr. Jain has suffered from advanced symptoms of the disease for 6 years, which included spondylitis – inflammation of the vertebrae – and other joint pain since 2004. According to Jain, "I managed my business until my speech became so impaired that I had to repeat everything at least four times to be understood."

Then Mr. Jain came to Jaslok Hospital in February of this year, where a new clinical trial was being conducted in which ten Parkinson’s patients were being enrolled for autologous (in which the donor and recipient are the same person) adult stem cell therapy. In addition to enrolling in the study, Mr. Jain also became the first volunteer.

As he explains, "When I heard I was going to be the first human to be treated using stem cell transplant, I was excited and scared. I had told my family that I may never return." Ten days after receiving the stem cells, however, he is no longer afraid, and any doubts or trepidation that he may have had have now been replaced with optimism. As his wife, Sukhi, explains, "I was scared initially but now I am happy that we opted for this operation." As Dr. Pettarusp Wadia, a neurologist at Jaslok Hospital, adds, "All we did was tell him about the procedure and he volunteered himself."

Specifically, the procedure utilizes the type of adult stem cell known as mesenchymal stem cells (MSCs), which are harvested from each patient’s own bone marrow via the hip, and then returned to the patient therapeutically after being processed in the laboratory. According to Dr. Paresh Doshi, head of the team who performed the eight-hour-long procedure, "We chose the stem cells from the marrow of the patient’s hip bone as these cells are readily isolated. They can expand in culture and the body can accept them easily. Small quantities of these cells were injected at an interval of every one millimeter in his brain."

Although Mr. Jain is already showing significant improvement, he will continue to be monitored for the next 18 months.

Watching Adult Stem Cells Repair the Brain

Adult stem cells are consistently proving to offer a viable and efficacious therapy for a number of diseases and injuries, especially in the treatment of neurodegenerative diseases such as Alzheimer’s, Parkinson’s, and Huntington’s disease, among others. Now, researchers have visually observed, in real time, the regenerative power of adult stem cells in a new animal model of Huntington’s disease.

Led by Dr. Yoram Cohen of the School of Chemistry at Tel Aviv University (TAU) in Israel, scientists used mesenchymal stem cells (MSCs) derived from bone marrow to treat animals in whom the disease had been artificially induced. In a novel approach to the study, Dr. Cohen and his colleagues also utilized "in-vivo MRI" (magnetic resonance imaging) to track the progress of the MSCs in the brains of the animals. The cinematic result clearly demonstrated the natural ability of the MSCs to target damaged or unhealthy tissue and repair the degeneration.

As Dr. Cohen explains, "By monitoring the motion of these cells, you get information about how viable they are, and how they can benefit the tissue. We have been able to prove that these stem cells travel within the brain, and only travel where they are needed. They read the chemical signaling of the tissue, which indicates areas of stress. And then they go and try to repair the situation."

The in-vivo MRI was conducted at the Strauss Centre for Computational Neuro-Imaging, where Dr. Cohen and his colleagues were able to observe, in real time, the activity of the live, active MSCs, which had been labeled with magnetic iron oxide nanoparticles, thereby allowing the MSCs to be tracked throughout the brain.

As Dr. Cohen further explains, "Cells that go toward a certain position that needs to be rescued are the best indirect proof that they are live and viable. If they can migrate toward the target, they are alive and can read chemical signaling." Additionally, he adds, "Bone marrow-derived MSCs bypass ethical and production complications, and in the long run, the cells are less likely to be rejected because they come from the patients themselves. This means you don’t need immunosuppressant therapy."

In actuality, since MSCs are "immune privileged", "universal donor" cells, they do not pose any risk of immune rejection even when they are not derived from the same patient to whom the cells are administered. In other words, there is no risk of immune rejection even when MSCs are administered allogeneically (in which the donor and recipient are not the same person), therefore autologous (in which the donor and recipient are the same person) administration is unnecessary, as is immunosuppressant therapy even when the MSCs are administered allogeneically.

Nevertheless, the results of the study offer further concrete evidence for both the natural "homing" ability as well as the powerful regenerative capacities of MSCs, especially in the treatment of neurodegenerative diseases for which conventional medicine currently offers no known treatment or cure.