blogstemcell

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Why is stem cell therapy at the forefront of regenerative medicine?

Stem cell treatment has the ability to rid people of regenerative disorders among many other diseases and illnesses. This is mainly due to its ability to convert into specialized cell types and replace damaged cells in the body. There is a growing interest in this field of medical science due to a few reasons,

In the process of watching stem cells grow into cells in heart muscles, bones, nerves and other organs/tissues, there is a possibility for doctors and researchers  to better understand how diseases and its conditions develop.

Stem cells can transform into specific cells which can then be used to regenerate and repair the damaged or diseased tissues in people. People who have spinal cord injuries, type 1 diabetes, amyotrophic lateral sclerosis, Parkinson’s disease, heart diseases, cancer, osteoarthritis can benefit from stem cell treatments. Stem cells have the ability to turn into new tissue for its use in regenerative medicine.

Using certain types of stem cells can help in order to test various drugs for safety before using investigational drugs in people. This usually can have a direct impact on drug development for cardiac toxicity testing.

Techniques like nerve cell generation to test a new drug for a nerve disease like Parkinson's continue to be studied.

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What diseases can be cured with stem cell treatment?

You can consider stem cells to be the raw materials of the human body, from these cells other cells with specialized function can be created! Favorable conditions in the body or a laboratory help stem cells divide to form more cells called daughter cells, which further turn into new stem cells or become differentiated specialized cells with a specific function like,

Blood cells

Brain cells

Heart muscle cells

Bone cells

The reason stem cells are extraordinary in its behaviors is because there are no other cell types that have the ability to produce or transform into other cell types. This makes Stem cell treatment a growing and effective means to treat various types of diseases! Up till now stem cell treatment has effectively been put into practice to treat diseases like leukemia, lymphoma, neuroblastoma, and multiple myeloma by using adult stem cells or umbilical cord stem cells to replace cells damaged by chemotherapy or disease. Stem cell treatments are also increasingly being researched upon and clinical trials are being done to test its viability to treat Parkinson’s disease and other diseases like,

Cardiomyopathy

Cerebral Palsy

Psoriasis

Testicular tumor

HIV

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What are Induced Pluripotent Stem cells and how do they work?

IPC’s(Induced Pluripotent stem cells) are known to be used for therapeutic uses, including the famous stem cell treatment for Parkinson’s disease. A striking feature of these stem cells include their ability to be made from any living person to allow immunity matching to any patient which will eliminate the need for immunosuppression when used in cell therapy.  Autologous are immune matched cells while allogeneic are unmatched cells. Why these are usually successfully used for stem cell treatments is because they can be used to model any disease, thus making it viable to treat symptoms of Parkinson’s. These stem cells help treat PD(Parkinson’s disease) by replacing and renewing the dopamine producing cells which are lost to the disease.

 Essentially, the skin cells/blood cells derived from adults are used to convert into IPC’s, this process is called reprogramming and is forced by by viruses to produce required proteins involved in pluripotency. A process called differentiation helps cells change their nature from generalized cells to specialized kinds of cells. In case this process is taking place in the laboratory, it can be accomplished by exposing stem cells to cues (hormones, growth factors, chemicals, substrates, small molecules, etc.) that recapitulate the stimuli they would normally be exposed to in the developing embryo. By being exposed to proper differentiation cues and significant time in the culture dish, they will mature into the desired cell type, and in this case  it would turn into the dopamine producing cells. Because of this detailed procedure , stem cell treatments are better used as treatment for PD.

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How are stem cells collected?

Stem cells can be sourced from the body itself.The human body has adult stem cells also known as somatic stem cells in the brain, blood, skeleton muscle, skin, heart, liver, bone marrow and other organs as well.These usually become active when the body has had an injury or disease and they create new cells needed for that particular organ to help restore the normal functioning.Stem cells can also be sourced from embryos, it is present in the amniotic fluid while the fetus is in the womb.The stem cells can be collected by extraction of samples from the particular adult tissue, for example,  blood stem cells can be isolated from bone marrow or blood from the umbilical cord.They are then grown in a controlled externally cultured environment where they will divide and grow.The process of the Stem cell treatment begins with Skin punch biopsies that are taken from Parkinson’s disease patients to isolate skin cells which are then reprogrammed to make iPSCs that are immune matched (autologous) to the patient.These iPSCs are differentiated into dopamine producing neurons and then the dopamine producing neurons are transplanted back into the patient to reset the clock on Parkinson’s disease.

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How many treatments are needed for stem cells and how does stem cells work?

Stem cell treatment uses stem cells to replace the dying dopamine producing cells in the brain which are responsible for body movement.The reason stem cells are being used is because they have the ability to differentiate into other cells as well, like in the case of Parkinson’s, introducing and transplanting stem cells help substitute for the dopaminergic cells that are lost to the disease. The number of stem cell treatments required is usually dependent on the nature of treatment in individual patients, whether treating a disease, or injury or preventing a genetic ailment. It also depends on the type of stem cell treatment required, for example, it could be  a transplant that requires surgery or a transfusion to inject the stem cells. Typically treatment can last upto a year or more depending on the disease,condition and response to treatment in the individual. Patients can experience relief within a few weeks in some cases.Usually the stem cells injected will keep producing new cells for upto a year and many patients do not require multiple treatment.

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How do stem cells work and how many treatments are needed?

Stem cell treatment uses stem cells to replace the dying dopamine producing cells in the brain which are responsible for body movement.The reason stem cells are being used is because they have the ability to differentiate into other cells as well, like in the case of Parkinson’s, introducing and transplanting stem cells help substitute for the dopaminergic cells that are lost to the disease. The number of stem cell treatments required is usually dependent on the nature of treatment in individual patients, whether treating a disease, or injury or preventing a genetic ailment. It also depends on the type of stem cell treatment required, for example, it could be  a transplant that requires surgery or a transfusion to inject the stem cells. Typically treatment can last upto a year or more depending on the disease,condition and response to treatment in the individual. Patients can experience relief within a few weeks in some cases.Usually the stem cells injected will keep producing new cells for upto a year and many patients do not require multiple treatment.

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Did Michael J Fox have Stem cell therapy?

The popular Canadian Actor was on the frontline for his unfortunate history with Parkinson’s disease and was known to have been assessed at the mild-late stage of Parkinson’s. He long argued that embryonic stem cells have the ability to cure diseases like Parkinson’s, however he switched from that to also relying on other relevant research.

How  to  integrate transplanted cells into the brain and developing on this aspect is something that the Michael J Fox Foundation has been working on. While he has been put on medication and undergone brain surgery for the thalamus(brain stimulation surgery), there is no evidence suggesting that he underwent a stem cell therapy. However ,he is a heavy supporter of the effects that stem cell treatment can have  on people suffering from Parkinson’s disease.

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Where do we stand with stem cell therapy for Parkinson’s disease in 2020

As of today, stem cell treatment stands at a good place as treatment for Parkinson’s disease. While it helps reduce symptoms and prolong your life, it has not yet been proved to cure the disease fully. Plenty of clinical trials have been done and are continuing to be done in the eventuality that stem cell treatment can grow to become more  gainful in the fight against Parkinson’s. The unlimited supply of dopaminergic neurons that stem cells provide  is what enhances and optimizes its benefits. Stem cells have showed significant benefits following transplantation in animal models of Parkinson’s disease. Stem cell therapy is a viable treatment option for Parkinson’s Disease since the Mesenchymal stem cells have the ability to target and reduce inflammation which help neurological conditions get better to a certain extent. Anywhere between $8000 - $30,000 USD is the cost of a stem cell therapy in 2020.

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How can stem cells help repair the brain?

Stem cells used in stem cell treatment are used as treatment for multiple types of health issues and helps in easing the symptoms of the disease which in turn helps prolong your life. Research suggests that damaged brain tissue can be repaired by exogenous stem cells while the anti inflammatory properties helps with speedy recovery. All this promotes better neurological function in people with brain damage. A Lot of other diseases like Parkinson’s and Alzheimer's have no proper cure but various types of treatments exist among which stem cell therapy is quite common and known to be effective. Neural stem cell therapy often helps increase the success rate of clinical trials which people having Parkinson’s disease, spinal cord injuries or other neurological conditions might volunteer for. So, yes, Stem cells do indeed help repair the brain by replacing damaged tissue and producing anti inflammatory fluids leading to healthier neurological functions thereby repairing the brain.

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Objective Realized

Late 2018, Summit's objective was realized. That objective was to enable Dr. Loring's and Dr. Bratt-Leal's autologous dopamine neuron replacement therapy for Parkinson's disease research project to move from bench to bedside as effectively and as quickly as possible. Summits board and everyone on the research team clearly understood that Parkinson's disease (PD) is a progressive neurodegenerative disease and that "time" is a critical concern for those with PD.

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The cost of completing the research along with the cost of the clinical trial process would have been tens of millions of dollars. Raising funds of that magnitude  would have taken a lot longer than all involved were ready to accept.

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Summit successfully supported and promoted this brilliant body of science which resulted in the founding a for-profit biotechnology company in late 2018. The iPSC-based therapy has been transferred to the for-profit biotechnology company, Aspen Neuroscience, Inc. and is now on an accelerated path to FDA-approval to begin clinical trials. Although Summit no longer directly supports the scientific development of the therapy, Summit continues to:

Monitor the progress of the iPSC (induced pluripotent stem cell-based therapy).

Provide informational updates.

Maintains a collaborative relationship with Aspen Neuroscience.

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What's Next?

In 2019, Summit has entered Chapter 2 of its existence. A new Board has been formed, once again, with dedicated professionals. Summit's new mission is to support, educate and raise awareness about the development of today's and tomorrow's evidence-based regenerative medical therapies focused on PD and other neurodegenerative disorders. It is our goal to empower patients and physicians with information to elevate their understanding in order to manage their care.

​Drs. Loring and Bratt-Leal continue as scientific advisors for Summit.

We share information via our website and newsletter relevant to PD, the cell-based therapy managed by Aspen Neuroscience and, in general, regenerative medicine.

We continue to fund and support iPSC and evidence-based research that will benefit PD patients and provide a better understanding model of PD in the hopes of early detection and prevention. Such as:Zero-Gravity Research - ​Stem Cells In Space Alliance.

Research studies. i.e., University of Cardiff, U.K. (Completed May, 2019.)

Fellowships & Internships

Continuing Outreach Programs.

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AUTOLOGOUS Dopamine Neuron Replacement Therapy

There is great deal of research being performed around the world for Parkinson’s disease (PD). Summit scientists are taking a very straightforward approach, developing a cell therapy to replace the specific type of nerve cells that are lost as the disease progresses, dopamine-producing neurons.

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Nobel Prize Medicine

Until the discovery of induced pluripotent stem cells (iPSCs), embryonic stem cells were commonly used by researchers because embryonic cells can become any cell in the body.  Dr. Shinya Yamanaka of Japan discovered a method by which he could transform any cell in the body back into its formative state: A stem cell. These transformed stem cells are called: iPSCs. Dr. Yamanaka was awarded the Nobel Prize in Medicine in 2012.

DNA Match

Many studies have been done comparing embryonic and iPSCs. The consensus concludes iPSCs behave exactly like embryonic stem cells. But, there is a bonus with iPSCs - they are sourced from a patient's own cells therefore removing any ethical issues and being sourced from the patient - the DNA matches. "...we believe that our cell lines show great promise without the need for immunesuppression," says Dr. Bratt-Leal, Director for Research & Development at the Parkinson's project.

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Skin Cells on a Remarkable Journey...

For this project, scientists ave chosen to use skin cells. Skin cells are abundant and easily accessed. Small biopsies from a group of Parkinson’s patients are undergoing a remarkable journey—the transformation from simple skin cells into iPSCs  and from there, the iPSCs are reprogrammed to become dopamine-producing neurons. These autologous (patient-specific) new dopamine neurons will be used to reverse the progressive symptoms of Parkinson’s disease. All cell lines are genetically screened multiple times throughout the process for efficacy.

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Who is Summit?

In 2011, Summit for Stem Cell was founded because of a desire from Parkinson’s patients for a stem cell-based therapy for their disease. Summit started as a small group of friends raising money for a cause they believed in and grew into a large organization with supporters from all around the world.

2015, Summit formed advisory board and, in 2017, became a 501(c)(3) organization. Throughout all, efforts continued to fund, promote and support the work in Dr. Jeanne Loring’s lab efforts to develop an autologous neuron replacement program for Parkinson’s disease. And, eventually, Summit opened its own lab to support the research.

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Parkinson’s Disease — The statistics are staggering…

Over 1 million Americans struggle with Parkinson’s disease (PD) and approximately 60,000 new cases are diagnosed each year.

On average, 60 new cases of PD diagnosed every year.

An estimated 7–10 million live with PD worldwide.

By the time of diagnosis, over 50% of the neurons that make dopamine have already been lost. Earlier detection is not currently possible and no FDA-approved therapy exists to replace or stop further loss of dopamine-producing neurons once a diagnosis is made.

More people have Parkinson’s disease than multiple sclerosis (MS), Lou Gehrig’s (amyotrophic lateral schlerosis, ALS) and muscular dystrophy (MD) — combined.​

6+ million Americans have a form of a neurodegenerative disorder with Alzheimers disease at the top of the list, followed by Parkinson’s disease​.

If you add spinal cord injuries, that brings the estimated ​total of afflicted Americans with a neurodegenerative condition to near 7 million Americans.

This reality needs to change.

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NEURON REPLACEMENT THERAPY

There is great deal of research being performed around the world for Parkinson’s disease (PD). Summit scientists are taking a very straightforward approach, developing a cell therapy to replace the specific type of nerve cells that are lost as the disease progresses, dopamine-producing neurons.

Nobel Prize Medicine

Until the discovery of induced pluripotent stem cells (iPSCs), embryonic stem cells were commonly used by researchers because embryonic cells can become any cell in the body. Dr. Shinya Yamanaka of Japan discovered a method by which he could transform any cell in the body back into its formative state: A stem cell. These transformed stem cells are called: iPSCs. Dr. Yamanaka was awarded the Nobel Prize in Medicine in 2012.

DNA Match

Many studies have been done comparing embryonic and iPSCs. The consensus concludes iPSCs behave exactly like embryonic stem cells. But, there is a bonus with iPSCs — they are sourced from a patient’s own cells therefore removing any ethical issues and being sourced from the patient — the DNA matches. “…we believe that our cell lines show great promise without the need for immunesuppression,” says Dr. Bratt-Leal, Director for Research & Development at the Parkinson’s project.

​Skin Cells on a Remarkable Journey…

For this project, scientists ave chosen to use skin cells. Skin cells are abundant and easily accessed. Small biopsies from a group of Parkinson’s patients are undergoing a remarkable journey — the transformation from simple skin cells into iPSCs and from there, the iPSCs are reprogrammed to become dopamine-producing neurons. These autologous (patient-specific) new dopamine neurons will be used to reverse the progressive symptoms of Parkinson’s disease. All cell lines are genetically screened multiple times throughout the process for efficacy.

Pluripotent Means “Many Powers”

“In scientific terms, stem cells are unspecialized cells that can regenerate and are able to convert into cells with specialized functions,” says Dr. Loring. “A pluripotent stem cell is a very special type of stem cell. The word pluripotent means many powers, and these cells have the power to develop into every type of cell in the body. We are turning the iPSCs into dopamine neurons in a culture dish.”

Parkinson’s disease has been selected by Dr. Loring because the disease needs only one cell-type to be replaced. Work is already underway for more complex diseases and conditions that could benefit from this brilliant body of science.

Types of Stem Cells

There are several types of stem cells. They all have, to some extent, the ability to self-renew, but some stem cell types have limitations to form the body’s cell types, while other stem cell types have far more extensive potential.

Science successfully transferred

Summit, a 501(c)(3) not-for-profit organization, successfully supported and promoted the body of science resulting in the establishment of a for-profit biotechnology company that is attracting the funding necessary for completing the therapy’s journey from bench to bedside as quickly and safely as possible. Having transferred the science successfully, Summit will continue to monitor the therapy’s development and provide information to those interested via our news letter and through email notification.

Therapy on accelerated pace…

With the quick infusion of funding, the scientific team is bustling in the lab. New scientists have been added to the team. Many of which worked previously with Drs. Loring and Bratt-Leal and are familiar with the cell replacement therapy. This familiarity is great because training-time is kept to a minimum leading to maintaining an accelerated pace. This is important to all of us. We are all cognizant of the fact that time is of great concern to those with Parkinson’s disease.

Nobel Prize Method

I Robust methods for turning these cells into dopamine neurons have been developed. We recently filed a request for the FDA to review the preclinical research plan, in a meeting called a “pre-pre-IND” consultation. This meeting helps the scientist to plan the research that will be necessary for the application for an IND (investigational new drug) that, when approved by the FDA, will allow this treatment to be used in patients. Pending approval from FDA and Scripps Institutional Review Board (IRB) the dopamine-producing neurons will be transplanted into patients using a procedure that is similar to that being used today for implanting electrodes for deep brain stimulation. Once the pilot project is complete, the goal is to open this therapy to a multicenter clinical trial​

Global Leader

This therapy is the current global leader in the development of an autologous dopamine neuron replacement therapy for PD. There are similar research projects going on around the world. The research for this therapy began almost 8 years ago and with the focus of each cell line being “personalized” — genetically checked, DNA-matching brand new dopamine neurons for each individual patient. Drs. Loring and Bratt-Leal feel this is the best approach. Currently, the estimated national average annual cost of care in the United States is estimated at $25 billion per year. (Source: Parkinson’s News Today.)

DNA Match = No Immunosuppressants!

There is excitement about the patient-specific approach. A patient’s own cells as the source for creating induced pluripotent stem cells that are then manipulated to become dopamine neurons. Patients who initially donated their skin cells will eventually receive their own cells back again — with their own DNA, just in a different cellular form. This shoulc eliminate the need for immunosuppression.

A study in the lab was recently completed. In the study, patient cell lines were used to see if their immune system would reject their own (autologous) dopamine-producing neurons. DNA-matching iPSC-based dopamine neurons placed together with their DNA matching antibodies…The results were unanimous: No rejection from any . (And, yes. When the cell lines were not autologous — there was rejection. This gives strong indication against the use of immunosuppressants should be unecessary.

We are so close to a Victory Over Parkinson’s!!!

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Summit’s Mission realized!

Global-leading research project launched. Now a hastened path to complete the necessary final studies to begin FDA approved clinical trials for ​Autologous dopamine neuron replacement therapy for Parkinson’s disease. In an effort to complete the journey from bench to bedside as quickly as possible, Summit transferred Drs. Jeanne Loring’s and Andres Bratt-Leal’s brilliant body of science to a privately owned company created specifically to hasten the highly anticipated research project for Parkinson’s disease towards FDA-approved clinical trials.

The cell-based therapy could potentially turn the clock back on Parkinson’s disease by replacing the lost dopamine neurons with patient-specific (DNA-matching) dopamine neurons.

The renowned scientific team is completing the last few studies needed to begin FDA-approved clinical trials.

​For more information on the cell-based therapy for Parkinson’s disease click here:​ https://www.summitforstemcell.org/

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OUR FOCUS

Mission

Summit for Stem Cell Foundation’s mission is to support, educate and raise awareness about the development of today's and tomorrow's evidence-based regenerative medical therapies focused on Parkinson's disease and other neurodegenerative disorders. It is our goal to empower patients and physicians with information to elevate their understanding in order to manage their care.

Our Focus

Summit is dedicated to supporting regenerative medicine through these three main objectives:

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Our Story...

Who is Summit?

In 2011, Summit for Stem Cell was founded in because of a desire from Parkinson’s patients for a stem cell-based therapy. Summit started as a small group of friends raising money for a cause they believed in and grew into a large organization with supporters all around the world.

In 2015, Summit formed advisory board and in 2017, became a 501(c)(3) organization. Throughout all, efforts continued to fund, promote and support the work in Jeanne Loring’s lab at The Scripps Research Institute to develop an autologous neuron replacement program. And, eventually, Summit opened its own lab to support the research.

Early 2019, after having completed its original goal, a new Board of Directors has been formed with the same dedication and work-ethic Summit is known for. A new Mission and Vision for Summit is in focus. Summit Board is focusing on the needs of patients with Parkinson's disease and other neurodegenerative disorders.