Reclaiming the voice by speaking with intent

February 22, 2019    By ROBBY JOHNSON

Graduate student Kirstin White leads vocal exercises for Parkinson’s patients in the UB Speech-Language and Hearing Clinic’s newest program, The LOUD Crowd®  Photo: Douglas Levere

The sounds of “may-me-my-mo-moo” thunder through a room in UB’s Biomedical Education Building on a recent Wednesday afternoon. Following that comes the fierce projection of a piercing “ah” sound.

Luckily, no one is hurt; these roaring shouts are coming from individuals with Parkinson’s disease who are patients of the UB Speech-Language and Hearing Clinic’s newest program, The LOUD Crowd®. These vocal warmups are helping these men from losing their voice to the degenerative condition.

Headed by Dona Hue Ritter-Schmidt, speech-language pathologist and clinical associate professor in the Department of Communicative Disorders and Sciences, The LOUD Crowd® is the second part of a speech therapy program offered by the clinic that helps Parkinson’s patients regain and maintain effective communication. The purpose of The LOUD Crowd® is to maintain the progress patients make through the SPEAK OUT!® program, which focuses on regaining the strength of the patient’s voice.

The programs are made possible through the teachings of the late Daniel Boone, a world-renowned speech-language pathologist and voice expert, and through a grant from the Parkinson Voice Project, a non-profit organization founded by CEO Samantha Elendary that is dedicated to preserving the voices of individuals with Parkinson’s and other neurological disorders through speech therapy. Boone found that restoring and maintaining the voices of Parkinson’s patients was most effective when emphasis was placed on converting speech from an automatic, extrapyramidal function to an intentional, pyramidal function.

Dona Hue Ritter-Schmidt (at the far end of the table) leads the group through vocal exercises. Photo: Douglas Levere

With Parkinson’s patients, they have a lack of, or a significant reduction in dopamine — which helps neurons to send messages to other neurons for movement,” says Ritter-Schmidt. “When somebody speaks purposefully, they use the pyramidal system. That system uses a more direct route and bypasses a lot of the areas that depend on dopamine. (These patients are now able) to execute movements stronger and more powerfully.

“We used to work with patients in slowing the rate of speech, being more precise and improving the loudness, but what we also know in Parkinson’s is that patients can concentrate better on one thing. By speaking with intent, the message travels directly down to the areas that are producing the movement, and they get a better bang for their buck in terms of speaking.”

The SPEAK OUT!® & LOUD Crowd® grant from Parkinson Voice Project was awarded to nearly 100 universities, clinics and hospitals throughout the country. The UB Speech-Language and Hearing Clinic is the only provider of The LOUD Crowd® program in Western New York.

The grant funded Ritter-Schmidt’s travel to a training symposium organized by the Parkinson Voice Project, as well as provided 12 LOUD Crowd® workbooks so she could administer the programs.

Ritter-Schmidt explains that one of the most significant aspects of the grant is that UB will now be a vital resource in expanding the program, with the clinic helping to train and establish SPEAK OUT!® and LOUD Crowd® programs in other area hospitals and clinics. Graduate students in the Department of Communicative Disorders and Sciences are also getting firsthand training in the program, further expanding its reach for the future.

Most importantly, The LOUD Crowd® is producing results that weren’t being achieved in the past.

“I can’t say enough about this program,” Ritter-Schmidt says. “For me professionally — and I’ve been at the university for 45 years — this is really, really great to see.

“I used to provide ‘LSVT’(Lee Silverman Voice Therapy) therapy, but the focus of that is to speak loudly. The focus of this is speak with intent and the volume comes from that. We found that people could hit the target and go through the program, but then there was very little carry over,” she notes. “It’s amazing where they have come from, where their voices are low and trailing off, to what they’re doing now (with The LOUD Crowd®).”

Many of the patients of The LOUD Crowd® have been with the Speech-Language and Hearing Clinic for more than five years and can attest to that improvement.

“This program is so much more successful than the last one,” says Chris Hipp, a patient in the program. “You can see it in the behavior in people and their body language; it just works.”

Paul Markwart reads the lyrics of "New York, New York." Photo: Douglas Levere

I’ve noticed from people who’ve been here that their voice gets stronger and that’s pretty neat,” says Paul Markwart.

“It’s ultimately good for us, but great for UB. It’s a neat symbiosis and the objective has always been to speak loudly and to project. This class has been a further defining of speaking with intent,” Markwart says. “If you are reading a story and just reading it loudly, it wouldn’t have any life to it, but if you are doing it with intent, the sounds of the story vary in texture and what you hear.”

Jim Eagan practices a vocal exercise. Photo: Douglas Levere

Not only have the patients seen a significant change in their lives because of the way they communicate, but they’re establishing friendships with people who are going through similar challenges. Bill Marx and Jim Eagan talk about the camaraderie they feel in the group, as everyone is doing things together outside of the clinic. 

Marx says that he’s proudly become “the group’s Uber driver,” while Eagan says he’s so much more outgoing because of the program. “Sometimes people with Parkinson’s tend to withdraw a little bit,” says Eagan. “For me, I was always a little quiet and shy, but this has allowed me to come out of my shell a little bit. Now I’m more outgoing.”

“This group of guys is so terrific because they’re kind of like a little fraternity,” says Ritter-Schmidt. “A lot of them are involved in boxing, dance and yoga. They take advantage of a lot of what’s out there for Parkinson’s patients.”

For these patients, The LOUD Crowd® has been a rousing success, and for people like Marx, the enjoyment he’s getting from the program is giving him a much more positive outlook.

“Parkinson’s is a debilitating disease, but I don’t look at it as a disease; I have an inconvenience,” he says. 

For more information about the SPEAK OUT!® and LOUD Crowd® programs, visit the programs’ UB website.

http://www.buffalo.edu/ubnow/stories/2019/02/loud-crowd.html

Dr. Roach: Concerns about contamination in stem cell treatment

February 22, 2019

Dr. Keith Roach

Dear Dr. Roach: I am a 76-year-old male who’s thinking of having fetal stem cell therapy for Parkinson’s disease. Please tell me your feelings and offer advice on this subject matter? -- S.C.

Answer: I am frequently asked about stem cell treatment, and my answer is usually that the theory makes sense and preliminary results are encouraging, but further research is necessary before I (by nature, a conservative physician) feel comfortable advising it for various conditions, including Parkinson’s disease.

However, recent reports from the Centers for Disease Control and Prevention have made me even more concerned -- this time, about infection. In December, the Food and Drug Administration issued a warning about stem cell treatment from umbilical cord blood. Twelve patients became ill with infections from E. coli and other fecal bacteria after injection, and the bacteria were found in unopened vials of the stem cells. The FDA plans to increase inspection of stem cell operators. There have been other adverse events reported, such as loss of eyesight (after injections into the eye) and growth of a spinal tumor.

I do not recommend stem cell treatment at this time, apart from its use in combination with chemotherapy for cancer or immune system diseases. More data is needed to prove its effectiveness, and better procedures are necessary to reduce infection risk.

Dear Dr. Roach: I am an 81-year-old woman, in very good health, with the exception of glaucoma and macular degeneration, both of which are under treatment. So far, I can drive in the daytime and I read the newspaper with the help of a magnifying glass and eyeglasses.

I’ve heard of hypoxia therapy to increase red blood cells and provide general health benefits. Do you know anything about it? I’ve found it easy to do for a brief span of time, a few times a day, or a 20-minute session. Breathing in through the nose, then out through the nose, expelling as much air as possible, and then holding my breath for 12 seconds or so.

Is it considered safe and effective for any health condition, or is it just another health fad? -- E.M.

Answer: I was unable to find reliable information supporting the use of this type of breathing for any specific condition or to increase either red blood cells or general health benefits. I have two concerns about it:

The first is physiology. It is true that having persistently low oxygen levels will increase erythropoietin, which is a hormone promoting red blood cell production. This is why people who smoke or have chronic lung disease sometimes have higher-than-normal blood counts. However, I doubt that your oxygen level will drop out of normal after 12 seconds of not breathing. Having experimented with a pulse oximeter, which gives immediate results on oxygen level, I know it takes a while -- as long as a minute of not breathing -- to get the oxygen level to go down. Further, even if your oxygen level does go down, I don’t know if the purported benefit is worth the cost of damage done while having low oxygen.

Secondly, blood cell levels are highly regulated to be optimum in healthy people. Getting more is not necessarily better (athletes who have taken erythropoietin to improve athletic performance have died from stroke due to the blood actually being too thick). If your blood counts are low, it may indicate a serious problem requiring evaluation.

Readers may email questions to ToYourGoodHealth@med.cornell.edu or send mail to 628 Virginia Dr., Orlando, FL 32803. 2019 North America Syndicate Inc.

https://www.lubbockonline.com/news/20190222/dr-roach-concerns-about-contamination-in-stem-cell-treatment

Parkinson’s disease mutation may trigger astrocyte-driven neural alpha-synucleinopathy

February 21, 2019   Kurt Samson

The most common genetic mutation in Parkinson’s disease may cause a breakdown in the ability of astrocytes to clear out toxic alpha-synuclein and the transfer of the excess protein to neighboring ventral midbrain dopaminergic neurons (vmDANs), causing their death, researchers from Spain reported in the January 10 issue of Stem Cell Reports.

Using induced pluripotent stem cell-derived (iPSCs) astrocytes and neurons from patients with the LRRK2 mutation, the authors discovered the defect is a non-cell autonomous deleteri-ous phenotype that, when co-cultured with healthy wild-type iPSC-derived neurons, reduced their number, suggest-ing some form of pathological ‘cross talk’ between the two cell types.

Astrocytes developed dysfunctional chaperone-mediated autophagy (CMA) and impaired macroautophagy, two intracellular “housekeeping” processes whereby proteins are selectively targeted for degradation.

“Our findings show that astrocytes are altered and accumulate toxic proteins, which in turn are passed on to neurons, ultimately causing their death,” explained Angel Raya, MD, PhD, co-senior author and director of the Center of Regenerative Medicine, in Barcelona, Spain.

“We found that patients have disruption in several cellular degradation path-ways, and intervention on these pathways might be a potential therapy, especially because astrocytes are con-nected to the vascular system,” he toldNeurology Today.

Co-author Antonella Consiglio, PhD, an investigator at the University of Barcelona’s Institute of Biomedicine, said more research is needed to determine whether additional factors might also contribute to neuronal cell death.

Potential molecular therapies target-ing the pathways might prove beneficial, she said, noting that by chemically enhancing CMA, they were able to pro-tect cells by promoting alpha-synuclein clearance.

“Some drugs that increase the activity of the degradation pathways that we found were altered in our studies are cur-rently in pre-clinical development,” Dr.Consiglio told Neurology Today. “We tried one of them and found that the treatment was efficient at reverting signs of the dis-ease, at least on cells in the laboratory.”

Study Design

The researchers used iPSCs from three PD patients who carried mutations in the LRRK2 gene to grow astrocytes and neu-rons, which were then co-cultured with healthy midbrain neurons from two con-trol patients. Structural changes associated with neurodegeneration and neuron loss developed in control neurons exposed to the mutated astrocytes after approximately one month.

Although it is also possible that PD astrocytes interfered with differentiation and/or maturation of vmDAN progeni-tors from the stem cells, the researchers noted that they used vmDAN differenti-ated cultures at 35 days, past the point when they might have matured into other types of cells. Moreover, there was a progressive decline and accumulation of alpha-synuclein over time only in healthy neurons co-cultured with PD astrocytes, and not in those co-cultured with control cells, they said.

That a CMA activator at least partially restored normal alpha-synuclein activity indicated that upregulation was still pos-sible and sufficient to return affected neurons to near normal levels. However, because the researchers were unable to completely restore them suggests that other non-alpha synuclelin-related fac-tors secreted by PD astrocytes might play a role, Dr. Raya said.

“Interestingly, the treatment with [CMA] activator not only cleared out alpha-synuclein in astrocytes, but also in vmDANs, partially restoring neuron survival and decreasing the number of TH-positive cells with a degenerative morphology.”

Dr. Raya said the team’s next goal is to investigate whether the findings also apply to patients with sporadic forms of the disease. The investigators are already looking into other mechanisms by which PD astrocytes might pass the disease to neurons.

Expert Commentary

Neurology professor Serge Przedborski, MD, PhD, chief of the movement disorders division and co-director of the Center for Motor Neuron Biology and Disease at Columbia University Irving

“some drugs that
increase the activity
of the degradation
pathways that we
found were altered
in our studies are
currently in pre-clinical
development. we tried
one of them and found
that the treatment was
efficient at reverting
signs of the disease, at
least on cells in the
laboratory.”

—dr. antonella consiglio

Medical Center, told Neurology Today that as in other neurodegenerative disorders such as amyotrophic lateral sclerosis, the study found that non-neuronal cells can contribute to the demise of neurons. However, he noted, much more work is needed to better clarify and confirm the reported findings.

“While the study provides an exciting proof-of principle, several points deserve clarification and call for additional stud-ies,” he said. “For instance, further demonstration is necessary to establish whether or not the decline in the number of vmDANs truly reflects death, as the authors concluded.”

“It would have been more convincing to see data for actual cell death markers rather than vmDANs counts since phe-notypic dopaminergic markers such as tyrosine hydroxylase can be readily downregulated in response to stress, giv-ing rise to a false interpretation of neu-ron loss,” Dr. Przedborski told Neurology Today.

He said that he remains uncertain about whether the mutant astrocytes exerted a deleterious effect on their neighboring vmDANs by way of a toxic phenomenon, as the authors maintained, or by merely proviidiing beneficial factors necessary to the well-being and survival of vmDANs.

“While this question can be readily clarified with future studies, it would have been crucial to have settled this issue since, to accept the pathogenic scenario proposed by the authors, one must first agree with the notion that mutant astrocytes are toxic,” he said.

“Lastly, the type of molecular mechanism targeted, autophagy, is so fundamental and a hub for so many factors, that it is difficult to conclude with certainty whether the improvement the authors report is truly or solely related to changes in alpha-synuclein turnover.”

Commenting on the study, Alice Chen-Plotkin, MD, an associate professor of neurology at the University of Pennsylvania Perelman School of Medicine, said: “I think that the key thing about this study is that it builds on a body of literature that is emerging that suggests alpha-synuclein protein can spread from cell to cell, and may occur between neurons and other types of cells,” she said.

Dr. Chen-Plotkin directs the Molecular Integration in Neurological

“i think that the key
thing about this study
is that it builds on a
body of literature that is
emerging that suggests
alpha-synuclein protein
can spread from cell
to cell, and may occur
between neurons and
other types of cells.”

—dr. alice chenplotkin

Diagnosis (MIND) Initiative at the School of Medicine, a cross-departmental program aimed at characterizing neurological disease patients at the DNA and molecular levels in order to improve clinical care and accelerate therapeutic development.

https://journals.lww.com/neurotodayonline/Fulltext/2019/02210/Parkinson_s_Disease_Mutation_May_Trigger.5.aspx

Next 20 Years Expected to Bring ‘Message of Hope’ to Parkinson’s Patients, Review Study Finds

FEBRUARY 22, 2019  BY CATARINA SILVA IN NEWS.

Discoveries into molecular mechanisms, risk factors — especially genetic — and advances in potential and repurposed therapies for Parkinson’s disease over the last 20 years are reason to believe that major breakthroughs await the next two decades, a review article by two researchers states.

The review article, “Therapies to Slow, Stop, or Reverse Parkinson’s Disease” was published in a supplement of the Journal of Parkinson’s Disease.

The development of better laboratory models, especially animal models that capture the slowly progressive nature of Parkinson’s, together with data resulting from scientific research and early clinical trials “strongly justifies sending this message of hope,” the authors write, explaining that the mechanisms underlying this neurodegenerative disease are gradually being deciphered.

The researchers, Tom Foltynie at University College London and J. William Langston at Stanford University, highlighted possible therapies that are most likely to emerge as disease-modifying treatments for Parkinson’s, despite the considerable challenges that remain in bringing a treatment successfully through a clinical study.

Based on the knowledge that mutations in the LRRK2 gene are one of the most common genetic causes of Parkinson’s disease, researchers have focused on therapies that can inhibit (block) LRRK2. But these efforts have been hindered by lung complications (lung toxicity) in primates exposed to inhibitor candidates, and scientists are exploring more selective ways of delivering such medications to avoid toxicity.

Questions also remain as to whether the brain is the prime target for LRRK2 activity, with some evidence pointing to the gut as well.

Treatments targeting the GBA gene, which encodes an enzyme called beta-glucocerebrosidase, may be relevant for people with sporadic forms of the disease in whom low levels of beta-glucocerebrosidase have been observed. This enzyme plays an important role in the mobilization and processing of alpha synuclein, which is low in GBAmutation carriers.

Ambroxol, an approved treatment for respiratory diseases associated with sticky or excessive mucus, is known to boost beta-glucocerebrosidase activity. However, it remains to be determined if Parkinson’s patients can tolerate the dose required for this therapy to reach the central nervous system. Other molecules that work in the body in ways similar to Ambroxol have been identified.

Since most available Parkinson’s therapies aim to ease motor symptoms, targeting non-motor features like cognition, speech, gait, balance difficulties and autonomic failure (or problems with regulating blood pressure and other process controlled by the autonomic nervous system) is important, given that many of these may precede motor onset. This could allow treatments to be started earlier, possibly delaying or preventing the onset of motor symptoms.

One approach to slowing disease progression gaining interest is that of “repurposing” medications already approved for diseases other than Parkinson’s. Preclinical studies found that type 2 diabetes medications — scientifically known as glucagon-like peptide 1 (GLP-1) receptor agonists — protect against alpha-synuclein-induced neurodegeneration. Various ongoing Phase 2 trials are assessing the effect of various GLP-1 receptor agonists (liraglutide, lixisenatide and semaglutide ) in Parkinson’s disease patients — NCT03659682, NCT03439943, NCT02953665). Plans for a Phase 3 trial of exenatide, another GLP-1 agonist, are underway.

Medicines used to treat primary biliary cirrhosis (an autoimmune disease of the liver; ursodeoxycholic acid), chronic myelocytic leukemia (nilotinib) and asthma (salbutamoland clenbuterol) also hold promise for Parkinson’s as they seem to contribute to nerve cell survival, eliminate toxic alpha-synuclein buildup, and modulate alpha-synuclein production, respectively.

Various studies have linked alpha-synuclein-induced neuroinflammation to Parkinson’s disease. As such, immunomodulatory therapies can be a treatment option. Evidence suggests a person’s immune system can react to toxic forms of alpha-synuclein and trigger an inflammatory reaction, which can speed disease progression. Azathioprine and sargramostim, both immunomodulatory medications, are being considered as potential candidates for slowing Parkinson’s progression.

A link between metabolism products generated by gut bacteria and brain inflammation has also been identified, and scientists might look to manipulate the gut microbiome — the trillions of microorganisms and their genetic material that live in the intestinal tract — in Parkinson’s patients, study the effects of such manipulation on the neurodegeneration process.

Lastly, the authors highlighted the possible use of nanoparticles in the disease context, as these molecules have been shown to block the formation of toxic alpha-synuclein clusters and actively work against their aggregation. In theory, nanotechnology might hold the potential to accurately target Parkinson’s-related neuropathology.

“We now have better understanding of the processes involved in PD [Parkinson’s disease] degeneration and can therefore have greater confidence that laboratory data and positive results from early clinical trials will ultimately translate to therapies that slow down PD progression,” Foltynie and Langston said in a news release.

“There are currently no drugs that have been proven to slow down PD progression. Demonstrating that one or several of the candidate approaches is successful will lead to a frameshift in patient care,” they added. “Useful cooperation and coordination between investigators around the globe are significantly accelerating the path towards discovering agents that may slow, stop, or even reverse the progression of PD.”

Their review concluded by stressing the possible importance of combination treatments in future clinical trials.

“It is tempting to speculate that the future patient may be recruited into research reminiscent of the current state of play in HIV/cancer fields, e.g., where following genotyping/ microbiome testing, they are either given the curative enzyme corrective therapy or randomised to receive combination therapies rather than any/each of these alone,” they wrote.

https://parkinsonsnewstoday.com/2019/02/22/next-20-years-expected-to-bring-message-of-hope-to-parkinsons-patients-review-study-finds/

Holding On to Happiness, but Not Too Tightly

  FEBRUARY 22, 2019 Dr. CBY DR. C IN COLUMNS

Life, liberty, and the pursuit of happiness. The H in the CHRONDI Creed refers to happiness. Happiness can be an elusive thing when battling a chronic disease like Parkinson’s. So many things can get in the way of experiencing happiness: pain, deep fatigue, irritability, the time consumed by the disease, and grief accompanying things stolen by the disease. Trying to hold on to even small moments of happiness is challenging. However, it is possible to experience moments of happiness in the face of chronic disease if one trains the brain to hold the moment gently — not too tightly.

Happiness is a state of mind and includes a broad range of phenomena, such as gratitude, inspiration, accomplishment, beauty, awe, laughter, compassion, tranquility, joy, love, exhilaration, ecstasy, and bliss. The experience of happiness can have a connection to one (or several) of these phenomena. Before you finish reading this column, let’s take a mental excursion together.

Visualize in your mind the last time you were happy and try to feel how you felt at that time. Try to hold the moment gently. Pause now to do that before reading on.

Were any of the above phenomena part of your memory? Remembering happiness is helpful in reminding us what it felt like and of what the experience may look like again. It can help us to see it in the smallest of moments throughout our lives. It is not a practice of grasping after happiness. Happiness is like a butterfly flitting from flower to flower. We take in the beauty and the rich, sensual experience and hold it gently in our mind. If we were to grasp the butterfly, we would destroy the experience.

Gently holding happiness without grasping is tied to a compassionate way of being. So much of our unhappiness is tied to grasping, to misperceptions, objectification, and poor communication in relationships. The practice of compassion is about experiencing the needs of others and then moving beyond suffering to a place of well-being. It is a shift in perception and out of suffering. Walking the path of the compassionate warrior is filled with happiness experiences accompanied by the knowledge of empathy, shifting perceptions, and shared well-being. Scrooge in Charles Dickens’ “A Christmas Carol” wasn’t happy until he experienced a shift in perception and became compassionate.

I don’t expect to experience happiness all the time. That’s just too unrealistic for where I am in my personal development as a compassionate warrior battling a chronic disease. I seek small moments each day, not by grasping for them but by looking for them, like looking at the butterfly, and then gently holding the moment in my mind. Then, I am very grateful for that moment and not sad when it naturally fades into the next experience as part of the day. This feeling of happiness is not induced by drugs or alcohol (which bring fake happiness and negative consequences). It is a happiness that comes from the practice of allowing the mind to experience both the large and small moments of happiness. I do my best to begin and end each day with a confirmation (a mantra or a prayer) of specific gratitude — not a statement of general gratitude but one aimed at something specific in my life. Gratitude is a way of holding the door open for those happiness moments.

Perhaps happiness brain training can be very helpful for those suffering from PD because of the link to dopamine production. I haven’t seen any research on this, but I find the practice to be quite helpful. What do you think? Are there methods you use to bring happiness into your life? Share them in the comments. Let’s pool together a collection of happiness tools for our readers.

***

Note: Parkinson’s News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Parkinson’s News Today or its parent company, BioNews Services, and are intended to spark discussion about issues pertaining to Parkinson’s disease.

https://parkinsonsnewstoday.com/2019/02/22/holding-on-happiness-but-not-too-tightly/

Plasma Infusions from Young Donors Carry Risks, No Benefit, FDA Warns Plasma Infusions from Young Donors Carry Risks

 FEBRUARY 22, 2019        BY JOSE MARQUES LOPES, PHD

The U.S. Food and Drug Administration (FDA) is warning consumers, physicians, and health care providers that infusions of plasma from young donors for the treatment of normal aging or diseases such as Parkinson’s or Alzheimer’s are not approved and have not undergone the agency’s rigorous testing for efficacy and safety.

As a result, such a method — also used for multiple sclerosis, heart disease, or post-traumatic stress disorder — should not be regarded as safe or effective.

“There is no proven clinical benefit of infusion of plasma from young donors to cure, mitigate, treat, or prevent these conditions, and there are risks associated with the use of any plasma product,” read a statement from FDA Commissioner Scott Gottlieb, MD, and Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research.

The benefits of plasma, the liquid portion of the blood, are well-known, particularly in trauma or for abnormal blood clotting. The FDA-recognized Circular of Information for the Use of Human Blood and Blood Components details indications for safe and effective plasma use, where the treatment’s benefits outweigh its risks. But even in such cases plasma infusion still has risks, such as allergies and transfusion-related circulatory overload, resulting in pulmonary edema (swelling) and difficulty  breathing.

Besides a lack of clinical evidence, the FDA’s concerns also include the lack of data on appropriate dosing for the indications for which these treatments are being marketed in several states. Also, reports indicate that the infusion of large volumes of plasma may lead to infections and cardiovascular problems.

The agency cautions that plasma infusions for indications other than those approved should be done by a qualified investigator or sponsor with an active investigational new drug (IND) application with the agency. Clinical trials must be performed under an IND to make sure the treatment is safe.

“We strongly discourage consumers from pursuing this therapy outside of clinical trials under appropriate institutional review board and regulatory oversight,” Gottlieb and Marks stated. “We support sound, scientific research and regulation of medical treatments.”

They added, “We’re concerned that some patients are being preyed upon by unscrupulous actors touting treatments of plasma from young as cures and remedies.” The statement mentioned reports of clinics charging thousands of dollars for therapies with unproven benefits, with potential harmful effects and which have not been guided by well-controlled clinical studies.

Also, promoting plasma in such cases could discourage patients from being treated with appropriate treatments that may be available, the agency said.

The FDA is considering taking “regulatory and enforcement actions against companies that abuse the trust of patients” and jeopardize their health” by using uncontrolled manufacturing processes or  promoting therapies with no evidence of safety or efficacy.

If considering the use of plasma infusions for unapproved indications, the FDA strongly urges patients to consult with their health care providers to confirm whether the agency has reviewed the treatment. Patients should ask the clinical investigator for the IND number and a copy of the FDA-issued communication acknowledging the IND.

Patients and physicians should report any adverse events associated with plasma administration to the FDA’s MedWatch program. The agency continues to monitor this issue, along with state and local health departments, as well as blood establishments.

https://parkinsonsnewstoday.com/2019/02/22/plasma-infusions-from-young-donors-carry-risks-no-benefit-fda-warns/

Renew! Retreat! Tampa, Florida

Partner Event:

 Renew! Retreat 
March 15th-16th, 2019
 Tampa, FL
 

The PMDAlliance is conducting a special event in March for care partners and adult children of people with Parkinson's Disease and other Movement Disorders. The retreat features special sessions for care partners and adult children while people with Parkinson's simultaneously participate in a program designed just for them. Hear from experts, connect with others walking in your shoes, and relax, recharge and renew yourself! Please see the flyer for more information! To register for this event visit www.pmdalliance.org or call 800-256-0966!

https://gallery.mailchimp.com/463e42f4c0e0bb1f7f5817957/files/f0f4e41f-ad83-4bba-8795-41cc8511ee89/Tampa_Renew_Retreat_March_2019_v2.pdf

https://www.pmdalliance.org

FoxFeed Blog:15 Projects Studying the GBA Gene Receive Nearly $3 Million in Total Funding

 Posted by  Maggie McGuire Kuhl,    February 20, 2019

Funded projects aim to define, measure and treat Parkinson's through the GBA pathway. This illustration shows a binding site for one potential treatment. (Image via Rheostat Therapeutics)

The Michael J. Fox Foundation for Parkinson's Research (MJFF) and The Silverstein Foundation for Parkinson's with GBA today announced nearly $3 million in grants to studies investigating glucocerebrosidase beta acid (GBA).

Mutations in the GBA gene are the most common genetic risk factors for Parkinson's, affecting about 10 percent of the more than 6 million people estimated to have the disease. The projects selected through this joint funding program aim to better understand the effects of GBA mutations -- and the role of GBA more generally -- and advance treatments against this target.

Read more on the selected projects in a press release.

"Defining the GBA pathway and its role in disease, including in patients without a GBA mutation, could point to new therapeutic approaches that may slow or stop Parkinson's," said MJFF CEO Todd Sherer, PhD. "This partnership with the Silverstein Foundation streamlined the grant process to more quickly direct funding to these promising projects, speeding their efforts to help Parkinson's patients."

Silverstein Foundation Founder Jonathan Silverstein said, "We are very pleased with the collaboration with The Michael J. Fox Foundation and feel confident that the projects chosen will significantly add to the library of knowledge around GBA and propel new treatments for people living with Parkinson's and, perhaps, individuals at risk for the disease."

Interested in participating in genetic research? MJFF's online clinical study Fox Insight is accelerating breakthroughs by capturing the experiences of people with and without Parkinson's. Through a collaboration with consumer genetics company 23andMe, people with Parkinson's who join Fox Insight can access the 23andMe Health + Ancestry Service at no cost and add their genetic information to the study. Register at foxinsight.org.

https://www.michaeljfox.org/foundation/news-detail.php?15-projects-studying-the-gba-gene-receive-nearly-million-in-total-funding