Crissy Field was filled with vibrant colors and massive energy for the 10th annual Moving Day San Francisco event on Saturday, May 4th. Even with the rain and wind, community members showed up to demonstrate their commitment to “moving to end Parkinson’s.”
Moving Day is an annual fundraising walk that unites people living with Parkinson’s Disease, their caregivers, and loved ones to raise awareness about Parkinson’s disease and embrace the power of exercise. This celebration encourages the Parkinson’s Disease community to move, which has been proven to help manage symptoms related to the disease.
The California Institute for Regenerative Medicine (CIRM) was present at the event, engaging with attendees to share information about its commitment to continuing to fund cell and gene therapy research to find treatments for Parkinson’s Disease.
Community members of all ages stopped by the CIRM booth to learn more about us and our impact on Parkinson’s Disease. Information shared included highlights about Aspen Neuroscience and Kenai Therapeutics‘ clinical trials and work being done by BrainXell Therapeutics. We also shared information with younger attendees through coloring pages and word searches.
The event was an amazing demonstration of a community’s dedication to coming together, rain or shine, to show their support for a common cause, in this case, Parkinson’s Disease. The rain did not dampen the energy, giving us a positive boost to continue spreading the message about CIRM and what we are doing to support the Parkinson’s community.
See more photos from the Parkinson’s Moving Day event in the gallery below. To learn more about CIRM’s investments in Parkinson’s Disease research, explore this page on our website. To learn more about the Parkinson’s Moving Day event, visit their official website.
The California Institute for Regenerative Medicine (CIRM) has approved an $8 million award to Aspen Neuroscience to advance an investigational stem cell-derived dopaminergic neuron replacement therapy for Parkinson’s disease (PD).
This individualized potential therapy is being explored in a First in Human Phase 1/2a clinical trial for patients with moderate to advanced PD.
Affecting more than one million Americans, PD is a devastating neurodegenerative disorder that causes walking and motor problems, as well as impaired balance and coordination. Existing therapies alleviate symptoms but do not treat the disease, leading to a significant unmet medical need for those suffering from this chronic condition.
“We would like to thank CIRM for their support of this program to investigate an autologous cell therapy for Parkinson’s disease, which is a very personalized condition,” said Damien McDevitt, PhD, President and CEO of Aspen Neuroscience, Inc.
“This first-in-human trial is the culmination of many years of work by a remarkable team of researchers and clinicians. Our approach to provide patients with their own dopamine neuron cells has the potential to impact the entire field of neurodegenerative disorders.”
This clinical trial aims to evaluate the safety, tolerability, and preliminary efficacy of this one-time therapy that, if successful, would eliminate the need for daily medications.
“This clinical award represents a significant step forward in the treatment landscape of Parkinson’s disease by advancing individualized therapy that has the potential to restore motor function in those impacted by this devastating condition,” said Dr. Abla Creasey, PhD, Vice President of Therapeutics Development at CIRM.
April is Parkinson’s Awareness Month and the California Institute for Regenerative Medicine (CIRM) is joining the campaign to #Take6ForPD—take 6 minutes to raise Parkinson’s awareness and highlight our commitment to funding research for the disease.
In addition to highlighting our commitment to funding research for Parkinson’s Disease, we’re also giving patients and patient advocates the opportunity to share their stories and experiences.
In this blog, CIRM Board Member David Higgins shares why spreading awareness of Parkinson’s is important to him.
Dr. David R. Higginsgrew up in West Virginia with four siblings. He earned his Ph.D. in molecular biology and genetics from the University of Rochester, followed by a postdoctoral fellowship at the National Cancer Institute before moving to San Diego in 1990 to work in biotech.
In December 2011, Higgins was diagnosed with Parkinson’s. Since then, he has become an advocate for people with Parkinson’s and their caregivers. He uses his personal experiences to guide this advocacy work, focused on improving quality of life issues through education, support, training, networking and promoting increased research funding to find a cure.
He currently serves as the Parkinson’s patient advocate member of CIRM’s governing board.
What is your personal connection to Parkinson’s Disease (PD)?
For me, Parkinson’s disease (PD) is a family legacy. My maternal grandmother suffered from PD and participated in some of the earliest Levodopa clinical trials held in the late 1960s.
In early 2014 my mother died with Lewy Body Dementia. My maternal uncle and great uncle also suffered from PD. In December 2011, I was diagnosed with PD completing the Parkinson’s Trifecta: care partner, scientist, and patient.
Additionally, in my biotech career, I worked for a company that is developing a gene therapy product to treat PD. This product currently is in human clinical trials in Europe.
What do you want people to know about PD?
PD is the second most common neurodegenerative disease, behind only Alzheimer’s disease in prevalence and with a rapidly growing incidence. Currently there are about 1 million people in the US diagnosed with PD.
PD is unique in that we know a lot about what the underlaying defect is. And there are likely several factors that influence whether you are going to develop PD. The most common model is a genetic predisposition to PD that exists in some people. If a person with a predisposition to PD is exposed to certain environmental factors, then development of the disease follows.
However, not every person who follows this pattern will develop PD. There is a familial form of PD that accounts for about 10% of the diagnosed cases. These tend to manifest earlier in life — in the patient’s 20s rather than the usual 60s.
There are a number of human genes known to be involved. However, identification of all of the so-called PD genes is not exhaustive. For example, there are about 80+ mutations that have been associated with the development of PD in humans. So clearly the situation is more complex.
Why is it important to you to raise awareness about PD?
It is critical that the stake holders, PD patients, California citizens, understand the impact that new treatments and even a cure for PD could have:
Social — Quality of life, decrease pain and suffering
Medical — Decrease huge medical costs to society to support and care for chronic illnesses
Economic — Positive impact on the economy by maintaining and increasing productivity of a sector of society that is at its peak productivity
Diagnosis — Fast accurate differential diagnoses of PD and related movement disorders such as MSA and SMA.
Higgins on a recent scuba diving trip
Why is it important to continue investing in all PD research and treatments — such as those that improve certain symptoms — and not just research that aims at finding a cure?
The goal in PD research as well as any new therapy is that new and effective treatments are just as important—and likely more within reach—than the elusive Holy Grail cure. Effective treatments that slow progression or work to treat chronic symptoms are incredibly beneficial to patients and the kinds of treatments that patients ask about most.
Why is it important that an agency like CIRM continues to invest in regenerative medicine research and treatment for PD?
Basic research and clinical development both need funding to understand the disease better, as well as to design testing in humans. Developing new therapeutics for PD and the research effort that supports it will require a huge multi-discipline collaborative effort and the financial funding to enable it. CIRM has the resources to put together the collaborative effort needed and is uniquely positioned to build appropriate collaborations needed.
To date, CIRM has invested more than $60 million in helping research for Parkinson’s progress from a basic or Discovery level through clinical trials. To learn more about CIRM’s investments in finding treatments for the disease, visit this page on our website.
Parkinson’s disease (PD) is a neurodegenerative disease that affects nearly 1 million people in the United States and more than 10 million people around the world. In California, more than 106,000 people live with PD.
Symptoms of PD include tremors, slow movement, muscle rigidity, balance issues and lack of facial expressions. Parkinson’s disease occurs when the neurons or nerve cells in the portion of the brain that controls movement die off. These neurons send signals by releasing a chemical called dopamine and are referred to as dopaminergic neurons.
Parkinson’s is the second-most common neurodegenerative disease after Alzheimer’s disease. No cure exists for the disease.
CIRM’s Commitment to Funding Parkinson’s Research
CIRM remains committed to funding regenerative medicine research—including stem cell and gene therapy research—for PD treatments to improve quality of life for patients.
To date, CIRM has invested more than $60 million in helping research for PD progress from a basic or Discovery level through clinical trials.
This year, CIRM awarded $4 million to a late-stage preclinical project by Ryne Bio aiming to improve treatment for idiopathic PD. In the proposed stem cell therapy, investigators at Ryne Bio are aiming to deliver dopamine-producing cells to replace the lost neurons to the brain of PD patients to restore or improve motor function.
“Receiving a Parkinson’s diagnosis is a difficult and life-changing event,” says Ryne Bio CEO Nick Manusos. “This Parkinson’s Awareness Month, we honor patients and their loved ones by reflecting on the clinical progress made so far, and looking forward to a brighter, healthier future.”
Other regenerative medicine approaches CIRM has funded to target PD involve understanding the disease and looking for new drugs to treat it.
CIRM grantees have taken skin cells from people with Parkinson’s disease, reprogrammed them back to an embryonic-like state, turning them into the kind of stem cell that can be transformed into any other cell in the body, then coaxing those cells to become dopaminergic neurons that are lost to the disease.
Those cells showed signs of the disease in the lab dish and were distinctly different from the same cells created from people without PD.
Another approach involves using CRISPR gene editing technology to reduce the levels of a toxic protein called alpha synuclein, which builds up in the dopaminergic brain cells affected by PD.
Overall, CIRM has awarded more than 30 grants aiming to better understand the disease and find regenerative medicine-based treatments.
“CIRM’s commitment to funding research for Parkinson’s is unwavering,” says CIRM President and CEO Maria Millan. “Our goal is to fund the most promising regenerative medicine research to support finding the best treatments to fight this devastating disease that affects millions.”
CIRM’s Task Force on Neuroscience & Medicine
As part of its Strategic Plan, CIRM has also launched a Task Force on Neuroscience and Medicine to set a plan for the $1.5 billion allocated for the support of research and the development of treatments for diseases and conditions of the brain and central nervous system. These include Parkinson’s, Alzheimer’s, stroke, dementia, epilepsy and other diseases and conditions.
The Task Force will host public meetings to gather community input to identify potentially high-impact opportunities in basic neuroscience, neurodegenerative disease, neuropsychiatric disease, neurodevelopment, and normal brain aging.
The goal of the task force is to provide final recommendations to CIRM and its governing board within six months of inception.
To see a listing of upcoming and past Task Force meetings,visit our website.
PD is characterized by a loss of dopamine producing neurons that result in motor symptoms, such as dyskinesias (involuntary, erratic, writhing movements of the face, arms, legs or trunk) and non-motor effects such as dementia, depression and sleep disorders.
PD is the second-most common neurodegenerative disease after Alzheimer’s disease affecting approximately 1 million people in the U.S. In California, it is estimated that 116,900 people live with PD, representing the highest number of people with the disease in the country.
At its early stages, PD can be treated with medication such as Levodopa to treat symptoms but these become less effective as the disease progresses.
The proposed stem cell therapy in this project offers the potential to restore dopamine neurons, which play a role in many important body functions, including movement and memory.
Investigators at Ryne Bio are aiming to deliver dopamine producing cells to replace the lost neurons to the brain of Parkinson’s disease patients to restore/improve motor function.
The current grant is being funded to conduct Investigational New Drug (IND) enabling, nonclinical safety studies per the US Food and Drug Administration (FDA) Guidance. The IND is the authorization needed to begin a clinical trial in Parkinson’s patients.
CIRM has a vested interest in seeing this therapy succeed. To date, CIRM has invested more than $59 million in helping research for Parkinson’s disease progress from a basic or Discovery level through clinical trials.
While stem cell and gene therapy research has advanced dramatically in recent years, there are still many unknowns and many questions remaining about how best to use these approaches in developing therapies. That’s why the governing Board of the California Institute for Regenerative Medicine (CIRM) today approved investing almost $25 million in 19 projects in early stage or Discovery research.
The awards are from CIRM’s DISC2 Quest program, which supports the discovery of promising new stem cell-based and gene therapy technologies that could be translated to enable broad use and ultimately, improve patient care.
“Every therapy that helps save lives or change lives begins with a researcher asking a simple question, “What if?”, says Dr. Maria T. Millan, the President and CEO of CIRM. “Our Quest awards reflect the need to keep supporting early stage research, to gain a deeper understanding of stem cells work and how we can best tap into that potential to advance the field.”
Dr. Judy Shizuru at Stanford University was awarded $1.34 million to develop a safer, less-toxic form of bone marrow or hematopoietic stem cell transplant (HCT). HCT is the only proven cure for many forms of blood disorders that affect people of all ages, sexes, and races worldwide. However, current methods involve the use of chemotherapy or radiation to destroy the patient’s own unhealthy blood stem cells and make room for the new, healthy ones. This approach is toxic and complex and can only be performed by specialized teams in major medical centers, making access particularly difficult for poor and underserved communities.
Dr. Shizuru proposes developing an antibody that can direct the patient’s own immune cells to kill diseased blood stem cells. This would make stem cell transplant safer and more effective for the treatment of many life-threatening blood disorders, and more accessible for people in rural or remote parts of the country.
Lili Yang UCLA Broad Stem Cell Research Center: Photo courtesy Reed Hutchinson PhotoGraphics
Dr. Lili Yang at UCLA was awarded $1.4 million to develop an off-the-shelf cell therapy for ovarian cancer, which causes more deaths than any other cancer of the female reproductive system.
Dr. Yang is using immune system cells, called invariant natural killer T cells (iNKT) to attack cancer cells. However, these iNKT cells are only found in small numbers in the blood so current approaches involve taking those cells from the patient and, in the lab, modifying them to increase their numbers and strength before transplanting them back into the patient. This is both time consuming and expensive, and the patient’s own iNKT cells may have been damaged by the cancer, reducing the likelihood of success.
In this new study Dr. Yang will use healthy donor cord blood cells and, through genetic engineering, turn them into the specific form of iNKT cell therapy targeting ovarian cancer. This DISC2 award will support the development of these cells and do the necessary testing and studies to advance it to the translational stage.
Timothy Hoey and Tenaya Therapeutics Inc. have been awarded $1.2 million to test a gene therapy approach to replace heart cells damaged by a heart attack.
Heart disease is the leading cause of death in the U.S. with the highest incidence among African Americans. It’s caused by damage or death of functional heart muscle cells, usually due to heart attack. Because these heart muscle cells are unable to regenerate the damage is permanent. Dr. Hoey’s team is developing a gene therapy that can be injected into patients and turn their cardiac fibroblasts, cells that can contribute to scar tissue, into functioning heart muscle cells, replacing those damaged by the heart attack.
A colony of iPSCs from a Parkinson’s patient (left) and dopaminergic neurons made from these iPSCs (right) to model PD. (Image credit: Jeanne Loring)
A new study that used adult blood stem cells to create replacement brain nerve cells appears to help rats with Parkinson’s.
In Parkinson’s, the disease attacks brain nerve cells that produce a chemical called dopamine. The lack of dopamine produces a variety of symptoms including physical tremors, depression, anxiety, insomnia and memory problems. There is no cure and while there are some effective treatments they tend to wear off over time.
In this study, researchers at Arizona State University took blood cells from humans and, using the iPSC method, changed those into dopamine-producing neurons. They then cultured those cells in the lab before implanting them in the brains of rats which had Parkinson’s-like symptoms.
They found that rats given cells that had been cultured in the lab for 17 days survived in greater numbers and seemed to be better at growing new connections in their brains, compared to rats given cells that had been cultured for 24 or 37 days.
In addition, those rats given larger doses of the cells experienced a complete reversal of their symptoms, compared to rats given smaller doses.
In a news release, study co-author Dr. Jeffrey Kordower, said: “We cannot be more excited by the opportunity to help individuals who suffer from [a] genetic form of Parkinson’s disease, but the lessons learned from this trial will also directly impact patients who suffer from sporadic, or non-genetic forms of this disease.”
The study, published in the journal npj Regenerative Medicine, says this approach might also help people suffering from other neurological diseases like Alzheimer’s or Huntington’s disease.
Neurodegenerative diseases impact millions of people worldwide with the risk of being affected by one of these diseases increasing as you get older. For many of these diseases, there are very few treatments available to patients. As life expectancy increases and the population continues to age, it is crucial to try and find treatments that can potentially slow the progression of these diseases or cure them entirely. This is one of the reasons why CIRM has committed directing around $1.5 billion in funding over the next few years to research related to neurological disorders.
One of the most common neurodegenerative diseases is Parkinson’s Disease (PD), a movement disorder that affects one million people in the U.S alone and leads to shaking, stiffness, insomnia, fatigue, and problems with walking, balance, and coordination. It is caused by the breakdown and death of dopaminergic neurons, special nerve cells in the brain responsible for the production of dopamine, a chemical messenger that is crucial for normal brain activity.
A recent study published in Nature Medicine has shown improved motor function and growth of neurons over a two year period in monkeys modeling PD. The study was conducted by Su-Chun Zhang, M.D., Ph.D. and his team at the University of Wisconsin using induced pluripotent stem cells (iPSCs), a kind of stem cell that can become virtually any type of cell that can be made from skin cells. The hope is that these results can pave the way for starting human clinical trials.
In order to replicate PD in humans, the team injected 10 adult monkeys with a neurotoxin that produces PD like symptoms. As a result of this, all 10 monkeys developed slow movements, imbalances, tremors, and impaired coordination in the hand on the opposite side of the injection. Additionally, scans revealed that on the injected side, monkeys lost most brain activity involving dopamine in two key brain areas. The team then waited three years after injecting the neurotoxin before administering the therapy, during which time the monkeys’ symptoms persisted.
To generate iPSC lines, the team obtained skin cells from five of the monkeys. The iPSCs were then turned into dopamine neural progenitor cells, which have the ability to create dopamine. These newly created cells were then administered into the brains of the five monkeys, with each monkey receiving a treatment derived from their own skin cells. A sixth iPSC line from a donor monkey was used for the remaining five monkeys to see how the treatment would work if it was not derived from their own skin cells.
The results showed that the monkeys that received the treatment derived from their own skin cells recovered. These animals moved more, moved faster, and were nimbler than before the treatment. They gained the ability to grasp treats, use all four limbs for walking, and climb their cages with ease and increased agility. However, the monkeys that received iPSCs derived from a donor did not recover. Their symptoms remained unchanged or worsened compared to before the treatment.
In a news article, Zhang emphasizes how he and his team are proceeding with a treatment derived from one’s own cells (autologous) vs. one from a donor (allogeneic).
“I initially wanted to do allogeneic transplants in patients because the autologous approach is too expensive. However, after seeing [our] data, I changed my mind. I want to go with the autologous first… because I feel the chance of success is really, really high.”
At first glance Lauren Miller Rogen and Dr. David Higgins seem an unlikely pair. She’s an actor, writer, director and has worked with some of the biggest names in Hollywood. He has a doctorate in molecular biology and genetics and has worked at some of the most well-known companies in biotech. But together they make a great team.
Lauren and David are both on the CIRM Board. She’s a patient advocate for Alzheimer’s and the driving force (with her husband Seth) of HFC (Hilarity for Charity), which has raised millions of dollars to help families battling the disease and to educate young people about the condition. It’s also made a lot of people laugh along the way. David is a patient advocate for Parkinson’s and has been instrumental is creating support groups that help patients and families cope with the disease.
Two voices, one message, watch out for predatory stem cell clinics
Last week two new papers came out echoing each other about the dangers of bogus “therapies” being offered by predatory stem cell clinics and the risks they pose to patients.
The first was from the Pew Charitable Trusts entitled: ‘Harms Linked to Unapproved Stem Cell Interventions Highlight Need for Greater FDA Enforcement’ with a subtitle: ‘Unproven regenerative medical products have led to infections, disabilities, and deaths.’
That pretty much says everything you need to know about the report, and in pretty stark terms; need for greater FDA enforcement and infections, disabilities and deaths.
Just two days later, as if in response to the call for greater enforcement, the Food and Drug Administration (FDA) came out with its own paper titled: ‘Important Patient and Consumer Information About Regenerative Medicine Therapies.’ Like the Pew report the FDA’s paper highlighted the dangers of unproven and unapproved “therapies” saying it “has received reports of blindness, tumor formation, infections, and more… due to the use of these unapproved products.”
The FDA runs down a list of diseases and conditions that predatory clinics claim they can cure without any evidence that what they offer is even safe, let alone effective. It says Regenerative Medicine therapies have not been approved for the treatment of:
Arthritis, osteoarthritis, rheumatism, hip pain, knee pain or shoulder pain.
Blindness or vision loss, autism, chronic pain or fatigue.
Neurological conditions like Alzheimer’s and Parkinson’s.
Heart disease, lung disease or stroke.
The FDA says it has warned clinics offering these “therapies” to stop or face the risk of legal action, and it warns consumers: “Please know that if you are being charged for these products or offered these products outside of a clinical trial, you are likely being deceived and offered a product illegally.”
It tells consumers if you are offered one of these therapies – often at great personal cost running into the thousands, even tens of thousands of dollars – you should contact the FDA at ocod@fda.hhs.gov.
The Pew report highlights just how dangerous these “therapies” are for patients. They did a deep dive into health records and found that between 2004 and September 2020 there were more than 360 reported cases of patients experiencing serious side effects from a clinic that offered unproven and unapproved stem cell procedures.
Those side effects include 20 deaths as well as serious and even lifelong disabilities such as:
Partial or complete blindness (9).
Paraplegia (1).
Pulmonary embolism (6).
Heart attack (5).
Tumors, lesions, or other growths (16).
Organ damage or failure in several cases that resulted in death.
More than one hundred of the patients identified had to be hospitalized.
The most common type of procedures these patients were given were stem cells taken from their own body and then injected into their eye, spine, hip, shoulder, or knee. The second most common was stem cells from a donor that were then injected.
The Pew report cites the case of one California-based stem cell company that sold products manufactured without proper safety measures, “including a failure to properly screen for communicable diseases such as HIV and hepatitis B and C.” Those products led to at least 13 people being hospitalized due to serious bacterial infection in Texas, Arizona, Kansas, and Florida.
Shocking as these statistics are, the report says this is probably a gross under count of actual harm caused by the bogus clinics. It says the clinics themselves rarely report adverse events and many patients don’t report them either, unless they are so serious that they require medical intervention.
The Pew report concludes by saying the FDA needs more resources so it can more effectively act against these clinics and shut them down when necessary. It says the agency needs to encourage doctors and patients to report any unexpected side effects, saying: “devising effective strategies to collect more real-world evidence of harm can help the agency in its efforts to curb the growth of this unregulated market and ensure that the regenerative medicine field develops into one that clinicians and patients can trust and safely access.”
We completely support both reports and will continue to work with the FDA and anyone else opposed to these predatory clinics. You can read more here about what we have been doing to oppose these clinics, and here is information that will help inform your decision if you are thinking about taking part in a stem cell clinical trial but are not sure if it’s a legitimate one.
The Stem Cellar 