Boosting immune system cells could offer a new approach to treating Lou Gehrig’s disease

ALS

Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, is one of those conditions that a lot of people know about but don’t know a lot about. If they are fortunate it will stay that way. ALS is a nasty neurodegenerative disease that attacks motor neurons, the cells in the brain and spinal cord that control muscle movement. As the disease progresses the individual loses their ability to walk, talk, eat, move and eventually to breathe. There are no effective treatments and no cure. But now research out of Texas is offering at least a glimmer of hope.

Dr. Stanley Appel, a neurologist at the Houston Methodist Neurological Institute noticed that many of the ALS patients he was treating had low levels of regulatory T cells, also known as Tregs. Tregs play a key role in our immune system, suppressing the action of molecules that cause inflammation and also helping prevent autoimmune disease.

In an article on Health News Digest Appel said:

Stanley Appel

Dr. Stanley Appel: Photo courtesy Australasian MND Symposium

“We found that many of our ALS patients not only had low levels of Tregs, but also that their Tregs were not functioning properly. We believed that improving the number and function of Tregs in these patients would affect how their disease progressed.”

And so that’s what he and his team did. They worked with M.D. Anderson Cancer Center’s Stem Cell Transplantation and Cellular Therapy program on a first-in-human clinical trial. They took blood from three people with different stages of ALS, separated the red and white blood cells, and returned the red blood cells to the patient. They then separated the Tregs from the white blood cells, increased their number in the lab, and then reinfused them into the patients, in a series of eight injections over the course of several months.

Their study, which appears in the journal Neurology,® Neuroimmunology & Neuroinflammation, found that the therapy appears to be safe without any serious side effects.

Jason Thonhoff, the lead author of the study, says the therapy also appeared to help slow the progression of the disease a little.

“A person has approximately 150 million Tregs circulating in their blood at any given time. Each dose of Tregs given to the patients in this study resulted in about a 30 to 40 percent increase over normal levels. Slowing of disease progression was observed during each round of four Treg infusions.”

Once the infusions stopped the disease progression resumed so clearly this is not a cure, but it does at least suggest that keeping Tregs at a healthy, high-functioning level may help slow down ALS.

CIRM is funding two clinical trials targeting ALS. One is a Phase 1 clinical trial with Clive Svendsen’s team at Cedars-Sinai Medical Center, the other is a Phase 3 project with Brainstorm Cell Therapeutics.

Using biological “codes” to generate neurons in a dish

BrainWavesInvestigators at the Scripps Research Institute are making brain waves in the field of neuroscience. Until now, neuroscience research has largely relied on a variety of animal models to understand the complexities of various brain or neuronal diseases. While beneficial for many reasons, animal models do not always allow scientists to understand the precise mechanism of neuronal dysfunction, and studies done in animals can often be difficult to translate to humans. The work done by Kristin Baldwin’s group, however, is revolutionizing this field by trying to re-create this complexity in a dish.

One of the primary hurdles that scientists have had to overcome in studying neuronal diseases, is the impressive diversity of neuronal cell types that exist. The exact number of neuronal subtypes is unknown, but scientists estimate the number to be in the hundreds.

While neurons have many similarities, such as the ability to receive and send information via chemical cues, they are also distinctly specialized. For example, some neurons are involved in sensing the external environment, whereas others may be involved in helping our muscles move. Effective medical treatment for neuronal diseases is contingent on scientists being able to understand how and why specific neuronal subtypes do not function properly.

In a study in the journal Nature, partially funded by CIRM, the scientists used pairs of transcription factors (proteins that affect gene expression and cell identity), to turn skin stem cells into neurons. These cells both physically looked like neurons and exhibited characteristic neuronal properties, such as action potential generation (the ability to conduct electrical impulses). Surprisingly, the team also found that they were able to generate neurons that had unique and specialized features based on the transcription factors pairs used.

The ability to create neuronal diversity using this method indicates that this protocol could be used to recapitulate neuronal diversity outside of the body. In a press release, Dr. Baldwin states:

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Kristin Baldwin, PhD

“Now we can be better genome detectives. Building up a database of these codes [transcription factors] and the types of neurons they produce can help us directly link genomic studies of human brain disease to a molecular understanding of what goes wrong with neurons, which is the key to finding and targeting treatments.”

These findings provide an exciting and promising tool to more effectively study the complexities of neuronal disease. The investigators of this study have made their results available on a free platform called BioGPS in the hopes that multiple labs will delve into the wealth of information they have opened up. Hopefully, this system will lead to more rapid drug discovery for disease like autism and Alzheimer’s

CIRM applauds FDA crackdown on stem cell clinics that “peddle unapproved treatments.”

FDA

CIRM is commending the US Food and Drug Administration (FDA) for its action against two stem cell clinics offering unapproved therapies.

On Wednesday, the FDA filed two complaints in federal court seeking a permanent injunction against California Stem Cell Treatment Center Inc. and US Stem Cell Clinic LLC. of Sunrise, Florida. The FDA says the clinics are marketing stem cell products without FDA approval and are not complying with current good manufacturing practice requirements.

“We strongly support the FDA’s strong stance to seek judicial action to stop these  clinics from marketing unproven therapies that pose a threat to the safety of patients” says Maria T. Millan, M.D., CIRM’s President and CEO. “We agree with FDA Commissioner Dr. Scott Gottlieb’s statement that these ‘bad actors leverage the scientific promise of this field to peddle unapproved treatments that put patients’ health at risk.’”

In his statement yesterday, Dr. Gottlieb denounced the clinics saying they are exploiting patients and causing some of them “serious and permanent harm.”

“In the two cases filed today, the clinics and their leadership have continued to disregard the law and more importantly, patient safety. We cannot allow unproven products that exploit the hope of patients and their loved ones. We support sound, scientific research and regulation of cell-based regenerative medicine, and the FDA has advanced a comprehensive policy framework to promote the approval of regenerative medicine products. But at the same time, the FDA will continue to take enforcement actions against clinics that abuse the trust of patients and endanger their health.”

At CIRM, we believe it is critically important for participants in stem cell treatments to be fully informed about the nature of the therapy they are receiving, including whether it is approved by the FDA. Last year we partnered with California State Senator Ed Hernandez to pass Senate Bill No. 512, which required all clinics offering unproven stem cell therapies to post notices warning patients they were getting a therapy that was not approved by the FDA.

The Stem Cell Agency has taken several other actions to protect people seeking legitimate stem cell therapies.

  • All the clinical trials we consider for funding must already have an active Investigational New Drug (IND) status with the FDA and go through a rigorous scientific review by leading experts.
  • All CIRM-funded trials must adhere to strict regulatory standards and safety monitoring.
  • We have created the CIRM Alpha Stem Cell Clinics, a network of six top California medical centers that specialize in delivering patient-centered stem cell clinical trials that meet the highest standards of care and research.
  • CIRM provides access to information on all the clinical trials it supports.

“Through its funding mechanism, active partnership and infrastructure programs, CIRM has shepherded 48 FDA regulated, scientifically sound, rigorously reviewed promising stem cell and regenerative medicine projects into clinical trials,” says Dr. Millan. “Some of these treatment protocols have already started to show preliminary signs of benefit for debilitating and life-threatening disorders. We are committed to doing all we can, in partnership with patients, the research community and with the FDA, to develop transformative treatments for patients with unmet medical needs while adhering to the highest standards to protect the health and safety of patients and the public.”

To help people make informed decisions we have created an infographic and video that detail the information people need to know, and the questions they should ask, before they agree to participate in a clinical trial or get a stem cell therapy.

 

 

‘Ask The Expert’ on Facebook Live about the power of stem cells to reverse damage caused by a stroke.

facebook-live-brand-awarenessIt’s not often you get a chance to ask a world class stem cell expert a question about their work, and how it might help you or someone you love. But on Thursday, May 31 you can do just that.

CIRM is hosting a special ‘Ask the Expert’ event on Facebook Live. The topic is Strokes and Stem Cells. Just head over to our Facebook Page on May 31st from noon till 1pm PST to experience it live. You can also re-watch the event any time after the broadcast has ended from our Facebook videos page.

Steinberg

We will be joined by Dr. Gary Steinberg, chair of neurosurgery at Stanford University, who will talk to us about his work in helping reverse the damage caused by a stroke, even for people who experienced a brain attack several years ago.

CIRM Senior Science Officer, Dr. Lila Collins, will talk about other stem cell research targeting stroke, its promise and some of the problems that still need to be overcome.

You will have a chance to ask questions of both our experts, either live on the day or by sending us questions in advance at info@cirm.ca.gov.

We’ll post reminders on Facebook so make sure to follow us. But for now, mark the date and time on your diary and please feel free to share this information with anyone you think might be interested.

It promises to be a fascinating event.

 

 

Stem Cell Agency’s supporting role in advancing research for rare diseases

Orchard

The recent agreement transferring GSK’s rare disease gene therapies to Orchard Therapeutics was good news for both companies and for the patients who are hoping this research could lead to new treatments, even cures, for some rare diseases. It was also good news for CIRM, which played a key role in helping Orchard grow to the point where this deal was possible.

In a news releaseMaria Millan, CIRM’s President & CEO, said:

“At CIRM, our value proposition is centered around our ability to advance the field of regenerative medicine in many different ways. Our funding and partnership has enabled the smooth transfer of Dr. Kohn’s technology from the academic to the industry setting while conducting this important pivotal clinical trial. With our help, Orchard was able to attract more outside investment and now it is able to grow its pipeline utilizing this platform gene therapy approach.”

Under the deal, GSK not only transfers its rare disease gene therapy portfolio to Orchard, it also becomes a shareholder in the company with a 19.9 percent equity stake. GSK is also eligible to receive royalties and commercial milestone payments. This agreement is both a recognition of Orchard’s expertise in this area, and the financial potential of developing treatments for rare conditions.

Dr. Millan says it’s further proof that the agency’s impact on the field of regenerative medicine extends far beyond the funding it offers companies like Orchard.

“Accelerating stem cell therapies to patients with unmet medical needs involves a lot more than just funding research; it involves supporting the research at every stage and creating partnerships to help it fulfill its potential. We invest when others are not ready to take a chance on a promising but early stage project. That early support not only helps the scientists get the data they need to show their work has potential, but it also takes some of the risk out of investments by venture capitalists or larger pharmaceutical companies.”

CIRM’s early support helped UCLA’s Don Kohn, MD, develop a stem cell therapy for severe combined immunodeficiency (SCID). This therapy is now Orchard’s lead program in ADA-SCID, OTL-101.

Sohel Talib, CIRM’s Associate Director Therapeutics and Industry Alliance, says this approach has transformed the lives of dozens of children born with this usually fatal immune disorder.

“This gene correction approach for severe combined immunodeficiency (SCID) has already transformed the lives of dozens of children treated in early trials and CIRM is pleased to be a partner on the confirmatory trial for this transformative treatment for patients born with this fatal immune disorder.”

Dr. Donald B. Kohn UCLA MIMG BSCRC Faculty 180118Dr. Kohn, now a member of Orchard’s scientific advisory board, said:

“CIRM funding has been essential to the overall success of my work, supporting me in navigating the complex regulatory steps of drug development, including interactions with FDA and toxicology studies that enhanced and helped drive the ADA-SCID clinical trial.”

CIRM funding has allowed Orchard Therapeutics to expand its technical operations footprint in California, which now includes facilities in Foster City and Menlo Park, bringing new jobs and generating taxes for the state and local community.

Mark Rothera, Orchard’s President and CEO, commented:

“The partnership with CIRM has been an important catalyst in the continued growth of Orchard Therapeutics as a leading company transforming the lives of patients with rare diseases through innovative gene therapies. The funding and advice from CIRM allowed Orchard to accelerate the development of OTL-101 and to build a manufacturing platform to support our development pipeline which includes 5 clinical and additional preclinical programs for potentially transformative gene therapies”.

Since CIRM was created by the voters of California the Agency has been able to use its support for research to leverage an additional $1.9 billion in funds for California. That money comes in the form of co-funding from companies to support their own projects, partnerships between outside investors or industry groups with CIRM-funded companies to help advance research, and additional funding that companies are able to attract to a project because of CIRM funding.

New CIRM Alpha Stem Cell Clinic offers HOPE for boys with deadly disease

UC Davis Institute for Regenerative Cures

For people battling Duchenne Muscular Dystrophy (DMD), a rare and fatal genetic disorder that slowly destroys muscles, hope has often been in short supply. There is no cure and treatments are limited. But now a new clinical trial at the site of one of the newest CIRM Alpha Stem Cell Clinic Network members could change that.

The HOPE-2 clinical trial has treated its first patient at UC Davis Medical Center, inaugurating the institution’s Alpha Stem Cell Clinic. The clinic is part of a CIRM-created network of top California medical centers that specialize in delivering stem cell clinical trials to patients. The key to the Network’s success is the ability to accelerate the delivery of treatments to patients through partnerships with patients, medical providers and clinical trial sponsors.

UC Davis is one of five medical centers that now make up the network (the others are UC San Francisco, UCLA/UC Irvine, UC San Diego and City of Hope).

Jan NoltaIn a news release, Jan Nolta, the director of the UC Davis Institute for Regenerative Cures, says the UC Davis Alpha Clinic is well equipped to move promising therapies out of the lab and into clinical trials and people.

“We have the full range of resource experts in regenerative medicine, from the cellular to the clinical trials level. We’re also excited about the prospect of being able to link with other Alpha Stem Cell Clinics around the state to help speed the process of testing and refining treatments so we can get therapies to patients in need.”

The news of this first patient is a cause for double celebration at CIRM. The trial is run by Capricor and CIRM funded the first phase of this work. You can read the story of Caleb Sizemore, who took part in that trial or watch this video of him talking about his fight.

When the CIRM Board approved funding for the UC Davis Alpha Clinic in October of 2017, Abla Creasey, CIRM’s Vice President for Therapeutics and Strategic Infrastructure, said:

“The Alpha Clinics are a one-of-a-kind network that gives patients access to the highest quality stem cell trials for a breadth of diseases including cancer, diabetes, heart disease and spinal cord injury. Expanding our network will allow more patients to participate in stem cell trials and will advance the development of stem cell treatments that could help or possibly cure patients.”

The UC Davis Alpha Clinic provides a one-stop shop for delivering stem cell therapies, gene therapies and immunotherapies, as well as conducting follow-up visits. It’s this type of CIRM-funded infrastructure that helps steer potential clinical trial participants away from illegitimate, unproven and potentially harmful fee-for-service stem cell treatments.

The DMD trial is the first of what we are confident will be many high-quality trials at the Clinic, bringing promising stem cell therapies to patients with unmet medical needs.

 

Livers skip stem cells, build missing structures from scratch via direct cell identity conversion

Stem cells…eh, who needs them anyway?!

That’s what you might be thinking after today, at least for some forms of liver disease. That’s because a team of researchers from UCSF and Cincinnati Children’s Hospital Medical Center just published results in Nature showing liver cells can directly change identity, or transdifferentiate, in order to build, from scratch, structures missing due to disease.

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The liver contains a network of tubes called bile ducts that carry fat-digesting bile to the small intestine via the gallbladder.
Image: National Cancer Inst.

The extraordinary regenerative power of the liver in animals is well-documented. A human liver, for instance, can fully regrow from just 25% of its original mass. That’s thanks to the hepatocyte, the main type of liver cell, that has the ability to replenish pre-existing tissue lost due to disease or injury. What hasn’t been as clear cut, is whether the hepatocyte has the capacity to change identity and build functional liver structures from scratch that never developed in the first place due to genetic disorders.

To examine that possibility, the study – funded in part by CIRM – focused on an inherited liver disease called Alagille syndrome which is caused by abnormal bile ducts. Produced by the liver, bile helps digest fats in our diet. It travels from the liver via bile ducts – tree branch-like tube structures in the liver – to the gallbladder, where it’s stored before moving on to the small intestine. In Alagille syndrome, the bile ducts are fewer in number, narrower in size or altogether missing. As a result, the bile builds up in the liver causing scarring and severe damage. Nearly half of all those with Alagille syndrome, require a liver transplant, usually in childhood.

The research team mimicked the symptoms of Alagille syndrome in mice by genetically engineering the animals to lack cholangiocytes, the cells that form bile ducts. Sure enough, liver damage from bile buildup was observed in these mice at birth due to the missing bile duct structures, also called the biliary tree. However, 90% of the mice survived and eventually formed a functional biliary tree. The team went on to show, for the first time, that the hepatocytes had converted en masse into cholangiocytes and created the wholly new bile ducts.

liver cell switching

Mice that mimic Alagille syndrome are born without the branches of the biliary tree, an important “plumbing system” in the liver (A), but show a near-normal biliary system as adults (B). To build the missing branches, liver cells switch identity and form tubes, shown in green, that connect to the trunk of the biliary tree, shown in blue (C). Image: Cincinnati Children’s

The underlying molecular mechanisms of this process were further examined. The researchers showed that the lack of a particular gene activity pathway due to the absence of cholangiocytes during development causes a replacement pathway, stimulated by a protein called TGF-beta, to kick into gear. As a result, the hepatocytes convert into cholangiocytes and form bile ducts. To make a direct connection with the human form of the disease, the researchers found evidence that TGF-beta is active in the liver samples of some patients but not in the livers from healthy individuals.

With this Alagille syndrome mouse model in hand, the researchers want to identify which transcription factors – proteins that bind DNA and regulate gene activity – are involved in changing the liver cells into bile duct cells. Holger Willenbring, MD, PhD, a senior author and CIRM grantee, explained the rationale behind this approach in a press release:

willenbring photo

Holger Willenbring

“Using transcription factors to make bile ducts from hepatocytes has potential as a safe and effective therapy. With our finding that an entire biliary system can be ‘retrofitted’ in the mouse liver, I am encouraged that this eventually will work in patients.”

So rather than developing a stem cell-based therapy in the lab which is then transplanted into a patient, this approach would rely on stimulating the regenerative capacity of liver cells that are already inside the body. And if it eventually works in patients with Alagille syndrome, which only affects 1 in 30,000, it’s possible it could be applied to other liver diseases as well.

Therapies Targeting Cancer, Deadly Immune Disorder and Life-Threatening Blood Condition Get Almost $32 Million Boost from CIRM Board

An innovative therapy that uses a patient’s own immune system to attack cancer stem cells is one of three new clinical trials approved for funding by CIRM’s Governing Board.

Researchers at the Stanford University School of Medicine were awarded $11.9 million to test their Chimeric Antigen Receptor (CAR) T Cell Therapy in patients with B cell leukemias who have relapsed or are not responding after standard treatments, such as chemotherapy.CDR774647-750Researchers take a patient’s own T cells (a type of immune cell) and genetically re-engineer them to recognize two target proteins on the surface of cancer cells, triggering their destruction. In addition, some of the T cells will form memory stem cells that will survive for years and continue to survey the body, killing any new or surviving cancer cells.

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Maria T. Millan

“When a patient is told that their cancer has returned it can be devastating news,” says Maria T. Millan, MD, President & CEO of CIRM. “CAR T cell therapy is an exciting and promising new approach that offers us a way to help patients fight back against a relapse, using their own cells to target and destroy the cancer.”

 

 

Sangamo-logoThe CIRM Board also approved $8 million for Sangamo Therapeutics, Inc. to test a new therapy for beta-thalassemia, a severe form of anemia (lack of healthy red blood cells) caused by mutations in the beta hemoglobin gene. Patients with this genetic disorder require frequent blood transfusions for survival and have a life expectancy of only 30-50 years. The Sangamo team will take a patient’s own blood stem cells and, using a gene-editing technology called zinc finger nuclease (ZFN), turn on a different hemoglobin gene (gamma hemoglobin) that can functionally substitute for the mutant gene. The modified blood stem cells will be given back to the patient, where they will give rise to functional red blood cells, and potentially eliminate the need for chronic transfusions and its associated complications.

UCSFvs1_bl_a_master_brand@2xThe third clinical trial approved is a $12 million grant to UC San Francisco for a treatment to restore the defective immune system of children born with severe combined immunodeficiency (SCID), a genetic blood disorder in which even a mild infection can be fatal. This condition is also called “bubble baby disease” because in the past children were kept inside sterile plastic bubbles to protect them from infection. This trial will focus on SCID patients who have mutations in a gene called Artemis, the most difficult form of SCID to treat using a standard bone marrow transplant from a healthy donor. The team will genetically modify the patient’s own blood stem cells with a functional copy of Artemis, with the goal of creating a functional immune system.

CIRM has funded two other clinical trials targeting different approaches to different forms of SCID. In one, carried out by UCLA and Orchard Therapeutics, 50 children have been treated and all 50 are considered functionally cured.

This brings the number of clinical trials funded by CIRM to 48, 42 of which are active. There are 11 other projects in the clinical trial stage where CIRM funded the early stage research.

CIRM President/CEO Presenting at Vatican Conference Targeting Cures for Deadly Diseases

It’s not often you get invited to a meeting of some of the leading scientists, ethicists, philosophers and faith leaders in the world so when the call comes in it’s an easy one to answer. Particularly when the call is from the Vatican.

View of Basilica di San Pietro in Vatican, Rome, Italy

Maria T. Millan, MD, President and CEO of the California Institute for Regenerative Medicine (CIRM), will be part of a panel discussion at the Fourth International Vatican Conference at Vatican City, Rome. The conference, titled: Unite To Cure: How Science, Technology and 21st Century Medicine Will Impact Culture and Society, runs from April 26-28 in Rome.

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Maria T. Millan, MD

“It is a tremendous honor to be part of this historic event,” says Dr. Millan. “CIRM funds the science and development of transformative cell and gene therapies for patients with unmet medical needs so it will be important to be part of the global conversation during such a propitious time in the history of medicine.”

“We’re thrilled to bring together the world’s best scientists, doctors, ethicists and leaders of faith, business, government and philanthropy to this extraordinary global event at The Vatican,” says Dr. Robin Smith, President of The Cura Foundation, the event organizer in partnership with the Vatican. “It’s a Davos for health care, and over the course of three days we will rally the world around a very simple idea — that tomorrow’s cures are just around the corner, and by uniting together and understanding the challenges that lie ahead, we can speed the delivery of cures and foster great hope for patients all over the world suffering from deadly diseases and dangerous medical conditions.”

Dr. Millan will be part of a panel discussion titled, Public Private Partnerships to Accelerate Discoveries. The panel will be moderated by award-winning medical journalist Max Gomez, PhD., and will include David Mazzo, PhD, CEO of Caladrius Biosciences, and David Pearce, PhD, the Executive VP for Research at Sanford Health.

The topic for this panel is particularly well suited for CIRM, an agency that is devoted to accelerating stem cell treatments to patients with unmet medical needs. CIRM has funded over 800 projects and over 45 novel stem cell and regenerative medicine clinical trials. It delivers a predictable and expedited funding mechanism, an active partnership and advisory role, strategic infrastructure, involvement of key opinion leaders and patient representatives and an industry alliance program, all to increase the chances of success for its programs and for the patients who would benefit.

To learn more about Unite To Cure: The Fourth International Vatican Conference, please visit: http://vaticanconference2018.com. Or, you can follow the event on Twitter @CuraFdn and on Facebook at facebook.com/TheCuraFoundation, and join the conversation with #UnitetoCure.

The Story of a South African Bubble Boy and a Gene Therapy That Gave Him His Life Back

Ayaan Isaacs, health24

Ayaan Isaacs was born in South Africa on March 4th, 2016 as a seemingly healthy baby. But only a few days in to life, he contracted a life-threatening liver infection. He thankfully survived, only to have the doctors discover a few weeks later that he had something much more troubling – a rare disease that left him without a functioning immune system.

Ayaan was diagnosed with X-linked severe combined immunodeficiency (SCID), which is often referred to as ‘bubble baby’ disease because patients are extremely susceptible to infection and must live in sterile environments. SCID patients can be cured with a blood stem cell transplant if they have a genetically matched donor. Unfortunately for Ayaan, only a partially matched donor was available, which doesn’t guarantee a positive outcome.

Ayaan’s parents were desperate for an alternative treatment to save Ayaan’s life. It was at this point that they learned about a clinical trial at St. Jude Children’s Research hospital in Memphis, Tennessee. The trial is treating SCID patients with a stem cell gene therapy that aims to give them a new functioning immune system. The therapy involves extracting the patient’s blood-forming stem cells and genetically correcting the mutation that causes SCID. The corrected blood stem cells are then transplanted back into the patient where they rebuild a healthy immune system.

Ayaan was able to enroll in the trial, and he was the first child in Africa to receive this life-saving gene therapy treatment. Ayaan’s journey with bubble boy disease was featured by South Africa’s health24 earlier this year. In the article, his mom Shamma Sheik talked about the hope that this gene therapy treatment brought to their family.

“No child should have to die just because they are unable to find a donor. Gene therapy offered Ayaan a chance at life that he ordinarily would not have had. I was fortunate to have found an alternative therapy that is working and already showing remarkable results. We are mindful that this is still an experimental treatment and there are complications that can arise; however, I am very optimistic that he will return to South Africa with a functioning immune system.”

Carte Blanche, an investigative journalism program in South Africa, did a feature video of Ayaan in February. Although the video is no longer available on their website, it did reveal that four months after Ayaan’s treatment, his condition started to improve suggesting that the treatment was potentially working.

We’ve written previously about another young boy named Ronnie who was diagnosed with X-linked SCID days after he was born. Ronnie also received the St. Jude stem cell gene therapy in a CIRM-funded clinical trial at the UCSF Benioff Children’s Hospital. Ronnie was treated when he was six months old and just celebrated his first birthday as a healthy, vibrant kid thanks to this trial. You can hear more about Ronnie’s moving story from his dad, Pawash Priyank, in the video below.

Our hope is that powerful stories like Ayaan’s and Ronnie’s will raise awareness about SCID and the promising potential of stem cell gene therapies to cure patients of this life-threatening immune disease.

Ronnie and his parents celebrating his 1st birthday. (Photo courtesy of Pawash Priyank)


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