ISCO Reports that Their Parthenogenetic Neural Stem Cells Improve Brain Function In Rodents with Traumatic Brain Injuries


International Stem Cell Corporation (OTCQB:ISCO) announced that the company’s proprietary ISC-hpNSC readily expandable neural stem cells improved cognitive performance and motor coordination in laboratory afflicted with traumatic brain injuries. ISC-hpNSCs consists of a highly pure population of neural stem cells derived from human parthenogenetic stem cells.

This preclinical study was conducted by scientists at the University of South Florida Morsani College of Medicine. The study examined rodents that had suffered from controlled cortical impact injury (rather well-known to be an established model of traumatic brain injury model).

The University of South Florida researchers divided their laboratory animals into four different cohorts. One group was treated with vehicle (the buffer in which the stem cells were delivered). This group of animals were the control group for this experiment. The next three groups were treated with ISC-hpNSCs, but the animals were given these cells in three different ways. Interestingly, laboratory animals that had received injections of ISC-hpNSCs showed the highest levels of improvements in cognitive performance and motor coordination when compared to those animals injected with only vehicle. Improvements in cognitive tests in animals transplanted with ISC-hpNSCs appeared only a few days after implantation.

ISCO’s new traumatic brain injury program will use the same cellular product (ISC-hpNSC) as their ongoing Parkinson’s disease program, which is presently in clinical trials. The safety data from the Parkinson’s disease trial can be used for future trials in patients with traumatic brain injuries.

Cell banks of ISC-hpNSCs were made under so-called “Good Manufacturing Practices,” which means that they are clean enough to be used in human patients. All of these stem cells have been extensively tested for sterility, purity, identity and safety. These extensive preclinical studies conducted during the development of the Parkinson’s disease program nicely demonstrate the safety of ISC-hpNSCs, even at high doses.

There is no approved treatment for traumatic brain injuries, and these injuries can cause long-term neurological disability. However, transplantation of neural stem cells may improve some of the symptoms of traumatic brain injury. Over 1.7 million people in North America suffer annually from traumatic brain injury, with associated medical costs exceeding $70 billion. According to the World Health Organization, the global incidence for traumatic brain injury is approximately 10 million people annually.

Preclinical studies in rodents and non-human primates have shown improvement in Parkinson’s disease symptoms and increase in brain dopamine levels following the intracranial administration of ISC-hpNSCs.

Mesenchymal Precursor Cells Reduce Cardiac Scar in Heart Failure Patients


Heart failure is a life-limiting condition that affects over 40 million patients worldwide. Fortunately, people who suffer from heart disease now may have new hope. A new study suggests that damaged tissue can be regenerated by means of a stem cell treatment that was injected into the heart during surgery.

This small-scale study was published in the Journal of Cardiovascular Translational Research. It treated and then followed 11 patients who, during coronary artery bypass graft surgery, had stem cells directly injected into their heart muscle near the site of the tissue scars that had resulted from previous heart attacks.

The most common cause of heart failure is “Ischemic cardiomyopathy” or ICM. ICM occurs when the heart has enlarged to such a degree that the vasculature can no longer supply the heart with adequate blood. ICM can also result from multiple sites of blockage in the coronary arteries of the heart that prevent adequate circulation in the heart.

In this study, researchers delivered a novel stem cell mesenchymal precursor cell type (iMP) during coronary artery bypass surgery (CABG) in patients with ICM whose ejection fractions were below 40%. The iMP cells are derived from what seem to be very young mesenchymal stem cells that lack the typical cell-surface proteins of mesenchymal stem cells. The cells have the ability to form a variety of mesodermal-derived tissues. Also, these cells suppress immunological rejection by the patient’s body, and therefore, they can be implanted into a patient’s body, even though their tissue types do not match. Therefore, these cells can not only be expanded in culture, but can also potentially differentiate into heart-based cell types, including heart muscle and blood vessels.

This study was a phase IIa safety study that was NOT placebo-controlled, double-blinded. It enrolled 11 patients, all of whom underwent scintigraphy imaging (SPECT) before their surgery. SPECT is an effect means to detect “hibernating myocardium” that does not properly contract. Hibernating myocardium is not suitable for iMP implantation.

During the CABG surgery, iMP cells were implanted in the heart muscle (intramyocardially). Stem cells were injected into predefined areas that were viable and close to infarct areas that usually showed poor vascularization. Such areas, because of their poor vascularization could not be treated with grafting because of their poor target vessel quality.

After surgery, SPECT imaging was used to identify changes in scar area. Fortunately, Intramyocardial implantation of iMP cells with CABG was safe. The huge surprise came with the reduction of the heart scar. Subjects showed a 40% reduction in the size of scarred tissue. Remember that heart scars form after a heart attack, and can increase the chances of further heart failure. This scarring, however, was previously thought to be permanent and irreversible. The patients also showed improved myocardial contractility and perfusion of nonrevascularized areas of the heart in addition to significant reduction in left ventricular scar area at 12 months after treatment.

“Quite frankly it was a big surprise to find the area of scar in the damaged heart got smaller,” said Prof Stephen Westaby from John Radcliffe hospital in Oxford, who undertook the research at AHEPA university hospital in Thessaloniki, Greece, with Kryiakos Anastasiadis and Polychronis Antonitsis.

Clinical improvement was correlated with significant improvements in quality of life at 6 months after the treatment all patients.

Jeremy Pearson, the associate medical director at the British Heart Foundation (BHF), said: “This very small study suggests that targeted injection into the heart of carefully prepared cells from a healthy donor during bypass surgery, is safe. It is difficult to be sure that the cells had a beneficial effect because all patients were undergoing bypass surgery at the same time, which would usually improve heart function.

“A controlled trial with substantially more patients is needed to determine whether injection of these types of cells proves any more effective than previous attempts to improve heart function in this way, which have so far largely failed.”

Dr. Westaby noted that improvements in the health of their patients were partly a result of the heart bypass surgery. However, he added that the next study would include a control group who will undergo CABG but not receive stem cell treatment, in order to measure exactly what impact the treatment has.

“These patients came out of heart failure partly due to the bypass grafts of course, but we think it was partly due to the fact that they had a smaller area of scar [as a result of the stem cell treatment]. Certainly this finding of scar being reduced is quite fascinating,” he said.

These results suggest that the delivery of iMP cells can induce regeneration of heart muscle and other heart tissues in patients with ischemic heart failure.

This paper was published: Anastasiadis, K., Antonitsis, P., Westaby, S. et al. J. of Cardiovasc. Trans. Res. (2016) 9: 202. doi:10.1007/s12265-016-9686-0.

SanBio, Inc Moves Forward With Clinical Stem Cell Trial for Traumatic Brain Injury in Japan


Traumatic brain injuries can result from a variety of causes, ranging from car accidents, falls, occupational hazards, and sports injuries. The cause of traumatic brain injury (TBI) differs from that of ischemic stroke, but many of the clinical manifestations are somewhat similar (motor deficits). Such injuries can cause lifelong motor deficits, and there are currently no approved medicines for the treatment of persistent disability from traumatic brain injury.

SanBio, Inc., has completed the regulatory requirements to conduct a clinical trial using their proprietary SB623 regenerative cell therapy to treat patients who suffer from TBI. The obligatory 30-day review period of clinical trial notification by the Japanese Pharmaceuticals and Medical Devices Agency (PMDA) was completed on March 7, 2016. No safety concerns were voiced, and the trial can proceed.

SanBio’s clinical trial is entitled “Stem cell therapy for traumatic brain injury” or STEMTRA, and it will study the safety and efficacy of SB623 cell therapy in treating patients who suffer from chronic motor impairments following a TBI.

Enrollment in this clinical trial started in the United States in October, 2015. The trial will include clinical sites and patients in Japan and will enroll ~52 patients. The enrollment of Japanese patients is expected to accelerate the overall enrollment of human subjects.

SanBio spokesperson, Damien Bates, the Chief Medical Officer and Head of Medical Research at SanBio, said: “SanBio’s regenerative cell medicine, SB623, has improved outcomes in patients with persistent motor deficits due to ischemic stroke, and our preclinical data suggest that it may also help TBI patients.  This is the first global Phase 2 clinical trial for TBI allogeneic stem cells, and the approval to conduct the trial in Japan, as well as in the United States, brings us one step closer to determining SB623’s efficacy for treatment whose who suffer from the effects of traumatic brain injury.”

SB623 are modified mesenchymal stem cells that transiently express a modified human Notch1 gene that only contains the intracellular domain of the Notch1 protein. This activated gene drives mesenchymal stem cells to form a cell type that habitually supports neural cells and promotes their health, survival, and healing.  When administered into damaged neural tissue, SB623 reverses neural damage. Since SB623 cells are allogeneic (from a donor), a single donor’s cells can be used to treat many patients. In cell culture and animal models, SB623 cells restore function to damaged neurons associated with stroke, traumatic brain injury, retinal diseases, and Parkinson’s disease. SB623 cells function by promoting the body’s natural regenerative process.

SanBio recently completed a US-based Phase 1/2a clinical trial for SB623 in patients with chronic motor impairments six months to five years following an ischemic stroke. The results of this trial demonstrated that SB623 can improve motor function following a stroke. On the strength of these results, SanBio initiated a Phase 2b randomized, double-blind, clinical trial of 156 subjects began enrollment in December 2015.  This trial is entitled ACTIsSIMA (“Allogeneic Cell Therapy for Ischemic Stroke to Improve Motor Abilities”).

Since the therapeutic mechanism of action of SB623 cells and the proposed route of administration are similar in the two trials (the stroke and TBI trials), the results of the TBI trial should be similar to those of the stroke trial.

The Japanese regulatory agencies grant marketing approval for regenerative medicines earlier countries as a result of an amendment to the Pharmaceutical Affairs Law in 2014. This particular amendment defined regenerative medicine products as a new category in addition to conventional drugs and medical devices, and the conditional and term-limited accelerated approval system for regenerative medicine products has started.

Two regenerative medicine products have already gained marketing approval under this new system, and the government-led industrialization of regenerative medicine products has gradually been realized.

SanBio has begun the preparation of clinical trial facilities in Japan and expects the launch of the clinical trial in 2016. the company hopes to market the medicine in Japan by taking advantage of the accelerated approval system.

Skin Cell to Eye Transplantation Successful


A presentation at the annual meeting of the Association for Research in Vision and Ophthalmology in Seattle, Washington has reported the safe transplantation of stem cells derived from a patient’s skin to the back of the eye in an effort to restore vision. The subject for this research project suffered from advanced wet age-related macular degeneration that did not respond to current standard treatments.

A small skin biopsy from the patient’s arm was collected and reprogrammed into induced pluripotent stem cells (iPSCs). The iPSCs were then differentiated into retinal pigmented epithelial (RPE) cells, which were transplanted into the patient’s eye. The transplanted cells survived without any adverse events for over a year and resulted in slightly, though significantly, improved vision.

iPSCs are adult cells that have been reprogrammed to an embryonic stem cell-like state, which can then be differentiated into any cell type found in the body.

Abstract Title: #3769: Transplantation of Autologous induced Pluripotent Stem Cell-Derived Retinal Pigment Epithelium Cell Sheets for Exudative Age Related Macular Degeneration: A Pilot Clinical Study by Yasuo Kurimoto and others from the laboratory of Masayo Takahashi’s laboratory at the RIKEN Center for Developmental Biology in Kobe, Japan.

Unfortunately, this clinical trial has been suspended because iPSCs made from other patients proved to possess too many genetic abnormalities.  Therefore, Takahashi and her colleagues have decided that allogeneic iPSCs differentiated into RPEs will probably do a better job than the patient’s own skin cell-derived iPSCs.

Positive Results from Phase 2 Study in Spinal Cord Injury


Stem Cells, Inc., has released the six-month results from cohort I of an ongoing Phase 2 clinical trial of human neural stem cells for the treatment of chronic cervical spinal cord injuries. The data displayed significant improvements in muscle strength had occurred in five of the six patients treated. Of these five patients, four of them also showed improved performance on functional tasks that assesses dexterity and fine motor skills. Furthermore, these four patients improved in the level of spinal cord injury according to the classification system provided by the International Standards for Neurological Classification of Spinal Cord Injury or ISNCSCI.

Stem Cells, Inc., expects to release their detailed final 12-month results on this first open-cohort later this quarter.

Chief medical officer, Stephen Huhn, presented these data at the American Spinal Injury Association annual meeting in Philadelphia, on Friday, April 15.  Dr. Huhn also believes that the interim results are very encouraging and reason to be quite hopeful.

“The emerging data continue to be very encouraging,” said Dr. Huhn. “We believe that these types of motor changes will improve the independence and quality of life of patients and are the first demonstration that a cellular therapy has the ability to impact recovery in chronic spinal cord injury. We currently have thirteen sites in the United States and Canada that are actively recruiting patients. We have enrolled and randomized 19 of the 40 total patients in the statistically powered, single-blind, randomized controlled, Cohort II. We are projecting to complete enrollment by the end of September so that we can have final results in 2017.”

The present Phase 2 clinical trial is a multi-center enterprise that includes physicians and scientists at 13 different sites in the united States and Canada. Incidentally, these sites are presently actively recruiting patients.

Stem Cells, Inc., has enrolled and randomized 19 of the 40 total patients in this statistically powered, single-blind, randomized controlled, cohort II.

The Phase 2 study, “Study of Human Central Nervous System (CNS) Stem Cell Transplantation in Cervical Spinal Cord Injury,” will determine the safety and efficacy of transplanting the company’s proprietary human neural stem cells (HuCNS-SC cells) into patients with traumatic injury of the cervical region of the spinal cord.

Cohort I is an open label dose-ranging cohort in six AIS-A or AIS-B subjects. For those of you not familiar with the American Spinal Injury Impairment Scale (ASI A-E scale), here is a summary of the classification scheme:

ASI – A = Complete paralysis; No sensory or motor function is preserved in the sacral segments S4-5.
ASI – B = Sensory Incomplete; Sensory but not motor function is preserved below the neurological level and includes the sacral segments S4-5 (light touch or pin prick at S4-5 or deep anal pressure) AND no motor function is preserved more than three levels below the motor level on either side of the body.
ASI – C = Motor Incomplete; Motor function is preserved below the neurological level**, and more than half of key muscle functions below the neurological level of injury (NLI) have a muscle grade less than 3 (Grades 0-2).
ASI – D = Motor Incomplete; Motor function is preserved below
the neurological level**, and at least half (half or more) of key muscle functions below the NLI have a muscle grade > 3.
ASI – E = Normal; If sensation and motor function as tested with the ISNCSCI are graded as normal in all segments, and the patient had prior deficits, then the AIS grade is E. Someone without an initial SCI does not receive an AIS grade.
Cohort II is a randomized, controlled, single-blinded cohort in forty AIS-B subjects. Cohort III, which will only be conducted at the discretion of the sponsor, is an open-label arm that involves six AIS-C subjects.
The primary efficacy outcome will focus on changes in the upper extremity strength as measured in the hands, arms, and shoulders.  This trial will enroll up to 52 subjects.
StemCells, Inc. has demonstrated the safety and efficacy of their HuCNS-SC cell in preclinical studies in laboratory rodents.  Additional Phase I studies yielded positive human safety data.  Furthermore, completed and ongoing clinical studies in which its proprietary HuCNS-SC cells have been transplanted directly into all three components of the central nervous system: the brain, the spinal cord and the retina of the eye, have further demonstrated the safety of HuCNS SC cells in human patients.
StemCells, Inc. clinicians and scientists believe that HuCNS-SC cells may have broad therapeutic application for many diseases and disorders of the CNS. Because the transplanted HuCNS-SC cells have been shown to engraft and survive long-term, there is the possibility of a durable clinical effect following a single transplantation.
The HuCNS-SC platform technology is a highly purified composition of human neural stem cells (tissue-derived or “adult” stem cells). Manufactured under cGMP standards, the Company’s HuCNS-SC cells are purified, expanded in culture, cryopreserved, and then stored as banks of cells, ready to be made into individual patient doses when needed.

Insulin-Secreting Beta Cells from Human Fat


In a study led by Martin Fussenegger of ETH Zurich, stem cells extracted from the fat of a 50-year-old test subject were transformed into mature, insulin-secreting pancreatic beta cells.

Fussenegger and his colleagues isolated stem cells from the fat of a 50-year-old man and used these cells to make induced pluripotent stem cells (iPSCs). These iPSCs were then differentiated into pancreatic progenitor cells and then into insulin-secreting beta cells but means of a “genetic software” approach.

Genetic software refers to the complex synthetic network of genes required to differentiate pancreatic progenitor cells into insulin-secreting beta cells. In particular, three genes, all of which expression transcription factors, Ngn3, Pdx1, and MafA, are particularly crucial for beta cell differentiation.

Fussenegger and his team designed a a protocol that would express within these fat-based stem cells the precise concentration and combination of these transcription factors. This feature is quite important because the concentration of these factors changes during the differentiation process. For example, MafA is not present at the start of beta cells maturation, but appears on day four on the final data of maturation when its concentration rises precipitously. The concentration of Ngn3 rises and then falls and the levels of Pdx1 rise at the beginning and towards the end of maturation.

The Zurich team used ingenious genetic tools to reproduce these vicissitudes of gene expression as precisely as possible. By doing so, they were able to differentiate the iPSC-derived pancreatic progenitor cells into insulin-secreting beta cells.

This work was published in Nature Communications 7, doi:10.1038/ncomms11247.

The fact that Fussenegger’s team was able to use a synthetic gene network to form mature beta cells from adult stem cells is a genuine breakthrough. The genetic network approach also seems to work better than the traditional technique of adding various chemicals and growth factors to cultures cells. “It’s not only really hard to add just the right quantities of these components (growth factors) at just the right time, it’s also inefficient and impossible to scale up,” said Fussenegger.

This new process can successfully transform three out of four fat stem cells into beta cells. Also the beta cells made with this method have the same microscopic appearance of natural beta cells in that they contain internal granules full of insulin. They also secrete insulin in response to increased blood glucose concentrations. Unfortunately the amount of insulin made by these cells is lower than that made by natural beta cells.

Pancreatic islet transplants have been performed in diabetic patients, but such transplantations also require treatment with potent antirejection drugs that have potent side effects.

“With our beta cells, there would likely be no need for this action (administering antitransplantation drugs), since we can make them using endogenous cell material taken from the patient’s own body. This is why our work is of such interest in the treatment of diabetes,” said Fussenegger.

Fussenegger and his group have made these beta cells in the laboratory, but they have yet to transplant them into a diabetic patient. However, the success of this synthetic genetic software technology might also be useful in the reprogramming of adult cells into other types of cells that are useful for therapeutic purposes.

Mesenchymal Stem Cells Secreting Brain-Derived Neurotrophic Factor Delay The Effects of Huntington’s Disease in Mice


Jan Nolta and her colleagues at the Stem Cell Program and Institute for Regenerative Cures at UC Davis have published a remarkable paper in the journal Molecular Therapy regarding Huntington’s disease and a potential stem cell-based strategy to delay the ravages of this disease.

Huntington’s disease (HD) is an inherited neurodegenerative disease. It is inherited as an autosomal dominant disease, which means that someone need only inherit one copy of the disease-causing allele of the HTT gene to have this disease. HD is characterized by progressive cell death in the brain, particularly in a portion of the brain known as the striatum and by widespread brain atrophy.

The portion of the brain known as the striatum lies underneath the surface of the forebrain (subcortical) and it receives neural inputs from the cerebral cortex and is the primary source of neural inputs to the basal ganglia system. The basal ganglia system (BGS) is located underneath the surface of the brain but even deeper within the cerebral hemispheres. The BGS is part of the corpus striatum, it consists of the subthalamic nucleus and the substantia nigra. The BGS help with voluntary motor control, procedural learning relating to routine behaviors. otherwise known as “habits,” eye movements, and cognitive, and emotional functions. The ventral striatum is very important in addiction because it is the reward center on consists of the nucleus accumbens, olfactory tubercle, and islands of Calleja.

This is a transverse section of the striatum from a structural MR image. The striatum, in red, includes the caudate nucleus (top), the putamen (right), and, when including the term 'corpus' striatum, the globus pallidus (lower left).
This is a transverse section of the striatum from a structural MR image. The striatum, in red, includes the caudate nucleus (top), the putamen (right), and, when including the term ‘corpus’ striatum, the globus pallidus (lower left).

HD takes its largest toll on the striatum, which affects voluntary movement, routine behaviors, and personality. Disturbances of both involuntary and voluntary movements occur in individuals with HD. Chorea, an involuntary movement disorder consisting of nonrepetitive, non-periodic jerking of limbs, face, or trunk, is the major sign of the disease. Chorea is present in more than 90% of individuals, increasing during the first ten years. The choreic movements are continuously present during waking hours, cannot be suppressed voluntarily, and are worsened by stress. HD patients show impaired voluntary motor function early on and show a clumsiness in common daily activities.

With advancing disease duration, other involuntary movements such as slowness of movement (bradykinesia), rigidity, and involuntary muscle contractions that cause repetitive or twisting movements (dystonia) occur.  Eye movement becomes progressively worse. So-called “gaze fixation” is observed in ~75% of symptomatic individuals.  Unclear speech occurs early and Swallowing difficulties occur later.

Animal models of HD used in the past have injected molecules into the brain that kill off striatal cells and mimic at least some of the characteristics of HD in laboratory animals. Unfortunately, such a model system is fat too clean, since implanted cells tend to survive perfectly well. However the brains of HD patients are like unto a toxic waste dumps and implanted cells are quickly killed off. Therefore, a better animal model system was required, and it came in the form of R6/2 and YAC128 mice. R6/2 mice have a part of the human HTT gene that has 150 CAG triplets, and show the characteristic cell death in the striatum and behavioral deficits. The only problem with this mouse strain is that the neurodegenerative decline is very rapid rather than slow and progressive. YAC128 mice have a full-length copy of the HTT gene and show a slower, more progressive neurological decline that more closely approximates the human clinical condition.

In this paper from the Nolta laboratory, they used a growth factor that is known to decrease precipitously in HD brains; a growth factor called Brain-Derived Neurotrophic Factor (BDNF). BDNF is known to mediate the survival and function of striatal neurons and the reduction of BDNF in the brains of HD patients correlates with the onset of symptoms and the greater the reduction in BDNF, the greater the severity of the disease (see Her LS & Goldstein LS, J Neurosci 2008; 28, 13662-13676).

However injecting BDNF into the brain is problematic, since the protein has a very short half-life. Delivering the growth factor by means of genetically engineered viruses shows promise, but most of the viral vectors used in such experiments are recognized by the immune system as foreign invaders. Therefore, Nolta and her colleagues decided to genetically engineer mesenchymal stem cells (MSCs) to overexpress BDNF and implant these cells into the brains of R6/2 and YAC128 mice.

MSCs have an added advantage over viral vectors: these cells migrate to damaged areas where they can exert their healing properties (see Olson SD et al., Mol Neurobiol 45; 2012: 87-98).

Nolta and her coworkers actually tested human MSCs in HD model mice. After completing all the necessary control experiments to ensure that their isolated and engineered MSCs were secreting BDNF, Nolta and others implanted them into the brains of R6/2 and YAC128 mice.

HD mice show greater anxiety, which is manifested in a so-called “open field assay” by not remaining the center of the field. The control HD mice did not stay long in the center of the open field, but the normal mice did. The MSC-BDNF-implanted mice spend far more time in the center of the field. Mind you, not as much as wild-type mice, but significantly more than their HD counterparts.

Next the volume of the striata of these mice were determined and compared to the normal mice. While all the HD mice showed shrinking of the striatum, the MSC-NDNF-implanted YAC128 mice show significantly less shrinking of the striatum. Then the degree of neurogenesis (formation of new neurons) was measured in normal, HD, HD + implanted MSCs, and HS + MSC-BDNF mice. This experiment measures the degree of healing that is occurring in the brain. The brain from HD + MSC and HD + MSC-BDNF mice showed significantly more new brain cell growth. This is probably the reason for the delayed onset of symptoms and the delayed shrinking of the striatum.

Finally, Nolta and others measured the lifespans of the R6/2 mice and compared them with R6/2 mice that had been implanted with MSCs-BDNF. Animals transplanted with the MSCs that made the most BDNF lived 15% longer than the nontreated R6/2 mice.

MSCs have been shown in several experiments to promote neuronal growth, decrease cell death and decrease inflammation through the secretion of trophic factors. MSCs can modify the toxic environment that is part of the brain of an HD patient and help damaged tissue out by inducing neural regeneration and protection (see Crigler L, et al., Experimental neurology, 198; 2–6, 54-64; Kassis I, et al., Archives of Neurology 65; 2008: 753-761).

The downside of using MSCs that they will only survive in the brain for a few months. However, several studies have shown that the benefits of modified MSC implantation persist after the MSCs are gone, since the neural reconstruction wrought by the secreted BDNF stay after the MSCs have died off (see Arregui L, et al., Cell Mol Neurobiol 31; 2011: 1229-1243 and many others).

At best this treatment would delay the ravages of HD, but delaying this disease might very well be the first step towards a cure. Hopefully, clinical trials will not be fat behind.