California’s Stem Cell Agency Accelerates Treatments to Patients

The following article is an Op Ed that appeared in today’s print version of the San Francisco Chronicle

SanFranChronicle_Web

Biotechnology was born in California in the 1970s based on the discovery out of one of its universities and California is responsible for an industry that has impacted the lives of billions of people worldwide. In 2004, the voters of California approved Proposition 71, creating the California Institute for Regenerative Medicine and setting the state on the path to becoming a global leader in stem cell research. Today the therapies resulting from the institute’s work are not just changing lives, they are already saving lives.

Lives like Evie Vaccaro, who is alive today because of a treatment CIRM is funding. Vaccaro was born with SCID, also known as “bubble baby disease,” an immune disorder that often kills babies in their first two years. Vaccaro and dozens of other babies were given stem cell treatments thanks to the institute. All are showing improvement; some are now several years past treatment and considered cured.

An accident left Jake Javier from Danville paralyzed from the chest down on the eve of his high school graduation. Javier was treated in a CIRM-funded clinical trial. Today he has regained the use of his arms and hands, is driving a car and is a sophomore at Cal Poly San Luis Obispo. Five other patients treated at the same time as Javier have all experienced improvements meaning that instead of needing round-the-clock care, they can lead independent lives.

A study by the Tufts Center for the Study of Drug Development estimated it takes at least 10 years and $2.6 billion to develop one successful drug. In 14 years, and with just $3 billion, CIRM has funded 1,000 different projects, enrolled 900 patients, and supported 49 different clinical trials targeting diseases such as cancer, kidney failure and leukemia. Four of these programs have received an expedited designation by the U.S. Food and Drug Administration, meaning they could get faster approval to help more patients

We have created a network of world class medical clinics that have expertise in delivering treatments to patients. The CIRM Alpha Clinics offer treatments based on solid science, unlike the unlicensed clinics sprouting up around California that peddle unproven and potentially harmful therapies that cost patients thousands of dollars.

CIRM has:

  • Supported the creation of 12 stem-cell research facilities in California
  • Attracted hundreds of top-tier researchers to California
  • Trained a new generation of stem-cell scientists
  • Brought clinical trials to California — for example, one targeting ALS or Lou Gehrig’s disease
  • Deployed rigorous scientific standards and support so our programs have a “seal of approval” to attract $2.7 billion in additional investments from industry and other sources.

We recently have partnered with the National Institutes of Health to break down barriers and speed up the approval process to bring curative treatments to patients with Sickle Cell Disease.

Have we achieved all we wanted to? Of course not. The first decade of CIRM’s life was laying the groundwork, developing the knowledge and expertise and refining processes so that we can truly accelerate progress. As a leader in this burgeoning field of regenerative medicine, CIRM needs to continue its mission of accelerating stem-cell treatments to patients with unmet medical needs.

Dr. Maria T. Millan is President and CEO and Jonathan Thomas, JD, PhD, is the Board Chairman of the California Institute of Regenerative Medicine. 

 

 

Support cells have different roles in blood stem cell maintenance before and after stress

How-Stem-Cells-Act-When-Stressed-Versus-When-At-Rest

Expression of pleiotrophin (green) in bone marrow blood vessels (red) and stromal cells (white) in normal mice (left), and in mice 24 hours after irradiation (right). UCLA Broad Stem Cell Research Center/Cell Stem Cell

A new study published in the journal Cell Stem Cell, reveals how different types of cells in the bone marrow are responsible for supporting blood stem cell maintenance before and after injury.

It was already well known in the field that two different cell types, namely endothelial cells (which line blood vessels) and stromal cells (which make up connective tissue, or tissue that provides structural support for any organ), are responsible for maintaining the population of blood stem cells in the bone marrow. However, how these cells and the molecules they secrete impact blood stem cell development and maintenance is not well understood.

Hematopoietic stem cells are responsible for generating the multiple different types of cells found in blood, from our oxygen carrying red blood cells to the many different types of white blood cells that make up our immune system.

Dr. John Chute’s group at UCLA had previously discovered that a molecule called pleiotrophin, or PTN, is important for promoting self-renewal of the blood stem cell population. They did not, however, understand which cells secrete this molecule and when.

To answer this question, the scientists developed mouse models that did not produce PTN in different types of bone marrow cells, such as endothelial cells and stromal cells. Surprisingly, they saw that the inability of stromal cells to produce PTN decreased the blood stem cell population, but deletion of PTN in endothelial cells did not affect the blood stem cell niche.

Even more interestingly, the researchers found that in animals that were subjected to an environmental stressor, in this case, radiation, the result was reversed: endothelial cell PTN was necessary for blood stem cell renewal, whereas stromal cell PTN was not. While an important part of the knowledge base for blood stem cell biology, the reason for this switch in PTN secretion at times of homeostasis and disease is still unknown.

As Dr. Chute states in a press release, this result could have important implications for cancer treatments such as radiation:

“It may be possible to administer modified, recombinant versions of pleiotrophin to patients to accelerate blood cell regeneration. This strategy also may apply to patients undergoing bone marrow transplants.”

Another important consideration to take away from this work is that animal models developed in the laboratory should take into account the possibility that blood stem cell maintenance and regeneration is distinctly controlled under healthy and disease state. In other words, cellular function in one state is not always indicative of its role in another state.

This work was partially funded by a CIRM Leadership Award.

 

 

Stem Cell Roundup: Knowing the nose, stem cell stress and cell fate math.

The Stem Cellar’s Image of the Week.
Our favorite image this week, comes to us from researchers at Washington University School of Medicine in St. Louis. Looking like a psychedelic Rorschach test, the fluorescence microscopy depicts mouse olfactory epithelium (in green), a sheet of tissue that develops in the nose. The team identified a new stem cell type that controls the growth of this tissue. New insights from the study of these cells could help the team better understand why some animals, like dogs, have a far superior sense of smell than humans.

MouseOlfactoryEpithelium-700x467

Peering into the nasal cavity of a mouse. Olfactory epithelium is indicated by green. Image credit: Lu Yang, Washington University School of Medicine in St. Louis.

A Washington U. press release provides more details about this fascinating study which appears in Developmental Cell.

How stress affects blood-forming stem cells.
Stress affects all of us in different ways. Some people handle it well. Some crack up and become nervous wrecks. So, perhaps it shouldn’t come as a huge surprise that stress also affects some stem cells. What is a pleasant surprise is that knowing this could help people undergoing cancer therapy or bone marrow transplants.

First a bit of background. Hematopoietic, or blood-forming stem cells (HSCs) come from bone marrow and are supported by other cells that secrete growth factors, including one called pleiotrophin or PTN. While researchers knew PTN was present in bone marrow they weren’t sure precisely what role it played.

So, researchers at UCLA set out to discover what PTN did.

In a CIRM-funded study they took mice that lacked PTN in endothelial cells – these line the blood vessels – or in their stromal cells – which make up the connective tissue. They found that a lack of PTN in stromal cells caused a lack of blood stem cells, but a lack of PTN in endothelial cells had no impact.

Chute Combo w Barrier 800x533

Expression of pleiotrophin (green) in bone marrow blood vessels (red) and stromal cells (white) is shown in normal mice (left) and in mice at 24 hours following irradiation (right). Image credit: UCLA

However, as Dr. John Chute explained in a news release, when they stressed the cells, by exposing them to radiation, they found something very different:

“The surprising finding was that pleiotrophin from stromal cells was not necessary for blood stem cell regeneration following irradiation — but pleiotrophin from endothelial cells was necessary.”

In other words, during normal times the stem cells rely on PTN from stromal cells, but after stress they depend on PTN from endothelial cells.

Dr. Chute says, because treatments like chemotherapy and radiation deplete bone marrow stem cells, this finding could have real-world implications for patients.

“These therapies for cancer patients suppress our blood cell systems over time. It may be possible to administer modified, recombinant versions of pleiotrophin to patients to accelerate blood cell regeneration. This strategy also may apply to patients undergoing bone marrow transplants.”

The study appears in the journal Cell Stem Cell.

Predicting the fate of cells with math
Researchers at Harvard Medical School and the Karolinska Institutet in Sweden reported this week that they have devised a mathematical model that can predict the fate of stem cells in the brain.

It may sound like science-fiction but the accomplished the feat by tracking changes in messenger RNA (mRNA), the genetic molecule that translates our DNA code into instructions for building proteins. As a brain stem cell begins specializing into specific cell types, hundreds of genes get turns on and off, which is observed by the rate of changes in mRNA productions.

The team built their predictive model by measuring these changes. In a press release, co-senior author, Harvard professor Peter Kharchenko, described this process using a great analogy:

“Estimating RNA velocity—or the rate of RNA change over time—is akin to observing the cooks in a restaurant kitchen as they line up the ingredients to figure out what dishes they’ll be serving up next.”

The team verified their mathematical model by inputting other data that was not use in constructing the model. Karolinkska Institutet professor, Sten Linnarsson, the other co-senior author on the study, described how such a model could be applied to human biomedical research:

“RNA velocity shows in detail how neurons and other cells acquire their specific functions as the brain develops and matures. We’re especially excited that this new method promises to help reveal how brains normally develop, but also to provide clues as to what goes wrong in human disorders of brain development, such as schizophrenia and autism.”

The study appears in the journal Nature.

What makes an expert an expert?

When we launched our Facebook Live “Ask the Expert” series earlier this year we wanted to create an opportunity for people to hear from and question experts about specific diseases or disorders. The experts we turned to were medical ones, neurologists and neuroscientists in the case of the first two Facebook Live events, stroke and ALS.

Then we learned about a blog post on the ALS Advocacy website questioning our use of the word “expert”. The author, Cathy Collet, points out that doctors or scientists are far from the only experts about these conditions, that there are many people who, by necessity, have become experts on a lot of issues relating to ALS and any other disease.

Cathy Collet ALS

 

Here’s Cathy’s blog. After you read it please let us know what you think: should we come up with a different title for the series, if so what would you suggest?

 

 

 

“Over the years I’ve experienced many “Ask the Experts” sessions related to ALS.  It’s always a panel of neuroscientists who talk a lot about ALS research and then take a few questions.

The “Expert” crown defaults to them.  They speak from the dais.  We get to listen a lot and ask.  They are by default “The Experts” in the fight against ALS.

But wait, there are all kinds of people with superb and valuable knowledge related to ALS –

  • There are people who know a lot about insurance.
  • There are people who know a lot about communication technology.
  • There are people who know a lot about low-tech hacks.
  • There are people who know a lot about suction machines.
  • There are people who know a lot about breathing.
  • There are people who know a lot about the FDA.
  • There are people who know a lot about moving a person on and off a commode.
  • There are people who know a lot about taxes.
  • There are people who know a lot about drugs.
  • There are people who know a lot about data.
  • There are people who know a lot about choking.
  • There are people who know a lot about financing research.
  • There are people who know a lot about stem cells.
  • There are people who know a lot about feeding tubes and nutrition.
  • There are people who know a lot about what’s important in living with the beast ALS.
  • There are people who know a lot about primary care in ALS.
  • There are people who know a lot about constipation.

Our default implication for the word experts being neuroscientists is revealing. There are many people in the fight against ALS, including those living with it, who know a lot.  We still live in a hierarchy where people with ALS and caregivers are at the bottom.

Words matter.  “Expert” is not a royal title to be owned by anyone by default.

It’s time for simple changes to some traditions.  “Ask the Neuroscientists,” anyone?

 

By the way, our next Facebook Live “Ask the ?” feature is targeting Sickle Cell Disease. It will be from noon till 1pm on Tuesday August 28th. More details, and maybe even a new name, to follow.

 

The Five Types of Stem Cells

When I give an “Intro to Stem Cells” presentation to, say, high school students or to a local Rotary Club, I begin by explaining that there are three main types of stem cells: (1) embryonic stem cells (ESCs) (2) adult stem cells and (3) induced pluripotent stem cells (iPSCs). Well, like most things in science, it’s actually not that simple.

To delve a little deeper into the details of characterizing stem cells, I recommend checking out a video animation produced by BioInformant, a stem cell market research company. The video is introduced in a blog, “Do you know the 5 types of stem cells?” by Cade Hildreth, BioInformant’s founder and president.

Stem-Cell-Types

Image credit: BioInformant

Hildreth’s list categorizes stem cells by the extent of each type’s shape-shifting abilities. So while we sometimes place ESCs and iPSCs in different buckets because the methods for obtaining them are very different, in this list, they both belong to the pluripotent stem cell type. Pluri (“many”) – potent (“potential”) refers to the ability of both stem cell types to specialize into all of the cell types in the body. They can’t, though, make the cells of the placenta and other extra-embryonic cells too. Those ultimate blank-slate stem cells are called toti (“total”) – potent (“potential”).

When it comes to describing adult stem cells in my talks, I often lump blood stem cells together with muscle stem cells because they are stem cells that are present within us throughout life. But based on their ability to mature into specialized cells, these two stem cell types fall into two different categories in Hildreth’s list:  blood stem cells which can specialize into closely related cell types – the various cell types found in the blood – are considered “oligopotent” while muscle stem cells are “unipotent” because the can only mature into one type of cell, a muscle cell.

For more details on the five types of stem cells based on their potential to specialize, head over to the BioInformant blog. And scroll to the very bottom for the video animation which can also viewed on FaceBook.

CIRM-supported study shows promise in fighting acute myeloid leukemia

Chemotherapy

Chemotherapy

For years chemotherapy has been a mainstay in the war against cancer. While it can be very effective it can also come with some nasty side effects. Since chemo works by killing rapidly growing cells, it not only hits the cancer cells, but can also hit other rapidly growing cells too, including those in our hair roots, which is why many people undergoing chemo lose their hair.

So, the key to a truly effective anti-cancer therapy is one that does as much damage as possible to the cancer cells, and as little as possible to all the healthy cells in the body. A therapy being developed by Cellerant Therapeutics seems to have found that sweet spot in a new therapy targeting acute myeloid leukemia (AML).

AML starts in the bone marrow and quickly moves into the blood, where it can spread to other parts of the body. It is the second most common form of leukemia and claims around 10,000 lives in the US every year. Chemotherapy is the main weapon used against AML but it can also cause nausea, hair loss and other complications and in most cases has limited effectiveness because, over time, the leukemia cells get used to it.

Cellerant 2013In a study published in the journal Blood Advances, Cellerant researchers explain the limitations of existing treatments.

“The current standard of care for acute myeloid leukemia (AML) is largely ineffective with very high relapse rates and low survival rates, mostly due to the inability to eliminate a rare population of leukemic stem cells (LSCs) that initiate tumor growth and are resistant to standard chemotherapy.”

Cellerant has developed a therapy called CLT030 which targets CLL1, a marker found on the surface of leukemia cells but not on normal blood stem cells. Preclinical studies in mice show CLT030 is able to zero in on this surface marker and attack the leukemia but do little damage to blood or other surrounding cells.

In a news release, Ram Mandalam, President and CEO of Cellerant, said this is encouraging news:

“AML remains a significant unmet medical need, and our therapy, CLT030, that can target leukemic stem cells precisely while minimally affecting normal hematopoietic stem cells could improve outcomes while avoiding much of the toxicities associated with conventional chemotherapy and other targeted therapeutics.”

Mandalam says they are now doing the late-stage preclinical testing to be able to apply to the Food and Drug Administration for permission to start a clinical trial. CIRM is funding this stage of the research.

 

Stem cell therapy offers a glimpse of hope for a student battling a deadly cancer

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Daniel Apodaca Image credit: CNN

“About a week later they gave me a call and mentioned the word ‘cancer’ to me. For a long time, I was depressed and then, I guess you accept it and try to make the most out of the time you have now.’

That is not something you expect to hear from a 24 year old. But for Daniel Apodaca that became, very suddenly, his reality. He was diagnosed with a rare, soft tissue cancer called epithelioid sarcoma. Fortunately for Daniel help was at hand, and a lot closer than he could ever have possibly anticipated.

Daniel is a student at UCLA. CIRM is funding a clinical trial run by UCLA’s Dr. Antoni Ribas that targets the same cancer Daniel is battling. The therapy re-programs a person’s own immune system to help fight the disease.

Daniel became patient #1 in that trial.

CNN reporter Rachel Crane profiled Dr. Ribas and the treatment he hopes will save Daniel’s life.

 

 

Research Targeting Prostate Cancer Gets Almost $4 Million Support from CIRM

Prostate cancer

A program hoping to supercharge a patient’s own immune system cells to attack and kill a treatment resistant form of prostate cancer was today awarded $3.99 million by the governing Board of the California Institute for Regenerative Medicine (CIRM)

In the U.S., prostate cancer is the second most common cause of cancer deaths in men.  An estimated 170,000 new cases are diagnosed each year and over 29,000 deaths are estimated in 2018.  Early stage prostate cancer is usually managed by surgery, radiation and/or hormone therapy. However, for men diagnosed with castrate-resistant metastatic prostate cancer (CRPC) these treatments often fail to work and the disease eventually proves fatal.

Poseida Therapeutics will be funded by CIRM to develop genetically engineered chimeric antigen receptor T cells (CAR-T) to treat metastatic CRPC. In cancer, there is a breakdown in the natural ability of immune T-cells to survey the body and recognize, bind to and kill cancerous cells. Poseida is engineering T cells and T memory stem cells to express a chimeric antigen receptor that arms these cells to more efficiently target, bind to and destroy the cancer cell. Millions of these cells are then grown in the laboratory and then re-infused into the patient. The CAR-T memory stem cells have the potential to persist long-term and kill residual cancer calls.

“This is a promising approach to an incurable disease where patients have few options,” says Maria T. Millan, M.D., President and CEO of CIRM. “The use of chimeric antigen receptor engineered T cells has led to impressive results in blood malignancies and a natural extension of this promising approach is to tackle currently untreatable solid malignancies, such as castrate resistant metastatic prostate cancer. CIRM is pleased to partner on this program and to add it to its portfolio that involves CAR T memory stem cells.”

Poseida Therapeutics plans to use the funding to complete the late-stage testing needed to apply to the Food and Drug Administration for the go-ahead to start a clinical trial in people.

Quest Awards

The CIRM Board also voted to approve investing $10 million for eight projects under its Discovery Quest Program. The Quest program promotes the discovery of promising new stem cell-based technologies that will be ready to move to the next level, the translational category, within two years, with an ultimate goal of improving patient care.

Among those approved for funding are:

  • Eric Adler at UC San Diego is using genetically modified blood stem cells to treat Danon Disease, a rare and fatal condition that affects the heart
  • Li Gan at the Gladstone Institutes will use induced pluripotent stem cells to develop a therapy for a familial form of dementia
  • Saul Priceman at City of Hope will use CAR-T therapy to develop a treatment for recurrent ovarian cancer

Because the amount of funding for the recommended applications exceeded the money set aside, the Application Subcommittee voted to approve partial funding for two projects, DISC2-11192 and DISC2-11109 and to recommend, at the next full Board meeting in October, that the projects get the remainder of the funds needed to complete their research.

The successful applications are:

 

APPLICATION

 

TITLE

 

INSTITUTION

CIRM COMMITTED FUNDING
DISC2-11131 Genetically Modified Hematopoietic Stem Cells for the

Treatment of Danon Disease

 

 

U.C San Diego

 

$1,393,200

 

DISC2-11157 Preclinical Development of An HSC-Engineered Off-

The-Shelf iNKT Cell Therapy for Cancer

 

 

U.C. Los Angeles

 

$1,404,000

DISC2-11036 Non-viral reprogramming of the endogenous TCRα

locus to direct stem memory T cells against shared

neoantigens in malignant gliomas

 

 

U.C. San Francisco

 

$900,000

DISC2-11175 Therapeutic immune tolerant human islet-like

organoids (HILOs) for Type 1 Diabetes

 

 

Salk Institute

 

$1,637,209

DISC2-11107 Chimeric Antigen Receptor-Engineered Stem/Memory

T Cells for the Treatment of Recurrent Ovarian Cancer

 

 

City of Hope

 

$1,381,104

DISC2-11165 Develop iPSC-derived microglia to treat progranulin-

deficient Frontotemporal Dementia

 

 

Gladstone Institutes

 

$1,553,923

DISC2-11192 Mesenchymal stem cell extracellular vesicles as

therapy for pulmonary fibrosis

 

 

U.C. San Diego

 

$865,282

DISC2-11109 Regenerative Thymic Tissues as Curative Cell

Therapy for Patients with 22q11 Deletion Syndrome

 

 

Stanford University

 

$865,282

 

 

Headline: Stem Cell Roundup: Here are some stem cell stories that caught our eye this past week.

In search of a miracle

Jordan and mother

Luane Beck holds Jordan in the emergency room while he suffers a prolonged seizure. Jordan’s seizures sometimes occur one after another with no break, and they can be deadly without emergency care. Photo courtesy San Francisco Chronicle’s Kim Clark

One of the toughest parts of my job is getting daily calls and emails from people desperate for a stem cell treatment or cure for themselves or a loved one and having to tell them that I don’t know of any. You can hear in their voice, read it in their emails, how hard it is for them to see someone they love in pain or distress and not be able to help them.

I know that many of those people may think about turning to one of the many stem cell clinics, here in the US and in Mexico and other countries, that are offering unproven and unapproved therapies. These clinics are offering desperate people a sense of hope, even if there is no evidence that the therapies they provide are either safe or effective.

And these “therapies” come with a big cost, both emotional and financial.

The San Francisco Chronicle this week launched the first in a series of stories they are doing about stem cells and stem cell research, the progress being made and the problems the field still faces.

One of the biggest problems, are clinics that offer hope, at a steep price, but no evidence to show that hope is justified. The first piece in the Chronicle series is a powerful, heart breaking story of one mother’s love for her son and her determination to do all she can to help him, and the difficult, almost impossible choices she has to make along the way.

It’s called: In search of a miracle.

A little turbulence, and a French press-like device, can help boost blood platelet production

Every year more than 21 million units of blood are transfused into people in the US. It’s a simple, life-saving procedure. One of the most important elements in transfusions are  platelets, the cells that stop bleeding and have other healing properties. Platelets, however, have a very short shelf life and so there is a constant need to get more from donors. Now a new study from Japan may help fix that problem.

Platelets are small cells that break off much larger cells called megakaryocytes. Scientists at the Center for iPS Cell Research and Application (CiRA) created billions of megakaryocytes using iPS technology (which turns ordinary cells into any other kind of cell in the body) and then placed them in a bioreactor. The bioreactor then pushed the cells up and down – much like you push down on a French press coffee maker – which helped promote the generation of platelets.

In their study, published in the journal Cell, they report they were able to generate 100 billion platelets, enough to be able to treat patients.

In a news release, CiRA Professor Koji Eto said they have shown this works in mice and now they want to see if it also works in people:

“Our goal is to produce platelets in the lab to replace human donors.”

Stem Cell Photo of the Week 

Photo Jul 11, 6 00 19 PM

Students at the CIRM Bridges program practice their “elevator pitch”. Photo Kyle Chesser

This week we held our annual CIRM Bridges to Stem Cell Research conference in Newport Beach. The Bridges program provides paid internships for undergraduate and masters-level students, a chance to work in a world-class stem cell research facility and get the experience needed to pursue a career in science. The program is training the next generation of stem cell scientists to fill jobs in California’s growing stem cell research sector.

This year we got the students to practice an “elevator Pitch”, a 30 second explanation, in plain English, of what they do, why they do it and why people should care. It’s a fun exercise but also an important one. We want scientists to be able to explain to the public what they are doing and why it’s important. After all, the people of California are supporting this work so they have a right to know, in language they can understand, how their money is changing the face of medicine.

Stem cell gene therapy combination could help children battling a rare genetic disorder

Hunter Syndrome-2

A child with Hunter Syndrome

Hunter syndrome is devastating. It’s caused by a single enzyme, IDS, that is either missing or malfunctioning. Without the enzyme the body is unable to break down complex sugar molecules and as those build up they cause permanent, progressive damage to the body and brain and, in some instances, result in severe mental disabilities. There is no cure and existing treatments are limited and expensive.

But now researchers at the University of Manchester in England have developed an approach that could help children – the vast majority of them boys – suffering from Hunter syndrome.

Working with a mouse model of the disease the researchers took some blood stem cells from the bone marrow and genetically re-engineered them to correct the mutation that caused the problem. They also added a “tag” to the IDS enzyme to help it more readily cross the blood brain barrier and deliver the therapy directly to the brain.

In a news release Brian Bigger, the lead researcher of the study published in EMBO Molecular Medicine, said the combination therapy helped correct bone, joint and brain disease in the mice.

“We expected the stem cell gene therapy approach to deliver IDS enzyme to the brain, as we have shown previously for another disease: Sanfilippo types A and B, but we were really surprised to discover how much better the tag made the therapy in the brain. It turns out that the tag didn’t only improve enzyme uptake across the blood brain barrier, but also improved uptake of the enzyme into cells and it appeared to be more stable in the bloodstream – all improvements on current technology.”

While the results are very encouraging it is important to remember the experiment was done in mice. So, the next step is to see if this might also work in people.

Joshua Davies has made a video highlighting the impact Hunter syndrome has on families: it’s called ‘Living Beyond Hope’