Promising Approach to Curing Spina Bifida Gets $5.6 Million from Stem Cell Agency

300px-Spina-bifida

Every day in the U.S. four children are born with spina bifida. It is the most common cause of lifelong paralysis and also frequently leads to other serious health problems affecting the bowel and bladder. The impact on families is enormous. A new approach to repairing the defect that causes spina bifida was today awarded $5.66 million by the Board of the California Institute for Regenerative Medicine (CIRM).

In spina bifida the spinal cord doesn’t form properly, in many cases leaving a section of it open, exposing tissues and nerves. The current standard of care is surgery, but even this leaves almost 60% of children unable to walk independently. Diana Farmer MD, and Aijun Wang PhD at U.C. Davis will use mesenchymal stem cells, taken from a donor placenta, and place them on a form of synthetic scaffold over the injury site in the womb. Tests in animals show this approach was able to repair the defect and prevent paralysis.

“Spina bifida is a devastating condition for babies born with this disorder and the families who care for them,” says Maria T. Millan, MD, President & CEO of CIRM. “CIRM has funded this important work from its earliest stages and we are committed to working with Dr. Farmer’s team to moving this work to the stage where it can be tested in patients.”

The CLIN1 award will provide funding to enable the UC Davis team to do the final testing and preparations needed to apply to the FDA for permission to start a clinical trial.

Dr. Farmer says she and Dr. Wang, have been working on this approach for more than ten years and are excited about being able to take the next step.

“There were many times of frustration, many times when cell types we explored and worked with didn’t work,” says Dr. Farmer. “But it’s the patients, seeing them, talking to them and working with them, that keeps me motivated to do the science, to keep persevering.”

If this therapy is successful it will have a huge economic impact on California, and on the rest of the world. Because spina bifida is a lifelong condition involving many operations, many stays in the hospital and, in some cases, lifelong use of a wheelchair this has a huge financial, and psychological, burden on the family.

“It affects them in so many ways; parents having to miss work or take time off work to care for their child, other children in the family feeling neglected because their brother or sister needs so much attention,” says Dr. Farmer. “That’s why we are so grateful to CIRM. Because this is a rare disease and finding funding for those is hard. CIRM has been a perfect partner in helping bring this approach, blending stem cell therapy and tissue engineering, together to help these families.”

This video shows English bulldogs treated with this approach who are now able to walk:

Stem Cell Agency Invests in New Immunotherapy Approach to HIV, Plus Promising Projects Targeting Blindness and Leukemia

HIV AIDS

While we have made great progress in developing therapies that control the AIDS virus, HIV/AIDS remains a chronic condition and HIV medicines themselves can give rise to a new set of medical issues. That’s why the Board of the California Institute for Regenerative Medicine (CIRM) has awarded $3.8 million to a team from City of Hope to develop an HIV immunotherapy.

The City of Hope team, led by Xiuli Wang, is developing a chimeric antigen receptor T cell or CAR-T that will enable them to target and kill HIV Infection. These CAR-T cells are designed to respond to a vaccine to expand on demand to battle residual HIV as required.

Jeff Sheehy

CIRM Board member Jeff Sheehy

Jeff Sheehy, a CIRM Board member and patient advocate for HIV/AIDS, says there is a real need for a new approach.

“With 37 million people worldwide living with HIV, including one million Americans, a single treatment that cures is desperately needed.  An exciting feature of this approach is the way it is combined with the cytomegalovirus (CMV) vaccine. Making CAR T therapies safer and more efficient would not only help produce a new HIV treatment but would help with CAR T cancer therapies and could facilitate CAR T therapies for other diseases.”

This is a late stage pre-clinical program with a goal of developing the cell therapy and getting the data needed to apply to the Food and Drug Administration (FDA) for permission to start a clinical trial.

The Board also approved three projects under its Translation Research Program, this is promising research that is building on basic scientific studies to hopefully create new therapies.

  • $5.068 million to University of California at Los Angeles’ Steven Schwartz to use a patient’s own adult cells to develop a treatment for diseases of the retina that can lead to blindness
  • $4.17 million to Karin Gaensler at the University of California at San Francisco to use a leukemia patient’s own cells to develop a vaccine that will stimulate their immune system to attack and destroy leukemia stem cells
  • Almost $4.24 million to Stanford’s Ted Leng to develop an off-the-shelf treatment for age-related macular degeneration (AMD), the leading cause of vision loss in the elderly.

The Board also approved funding for seven projects in the 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.

Application Title Institution CIRM Committed Funding
DISC2-10979 Universal Pluripotent Liver Failure Therapy (UPLiFT)

 

Children’s Hospital of Los Angeles $1,297,512

 

DISC2-11105 Pluripotent stem cell-derived bladder epithelial progenitors for definitive cell replacement therapy of bladder cancer

 

Stanford $1,415,016
DISC2-10973 Small Molecule Proteostasis Regulators to Treat Photoreceptor Diseases

 

U.C. San Diego $1,160,648
DISC2-11070 Drug Development for Autism Spectrum Disorder Using Human Patient iPSCs

 

Scripps $1,827,576
DISC2-11183 A screen for drugs to protect against chemotherapy-induced hearing loss, using sensory hair cells derived by direct lineage reprogramming from hiPSCs

 

University of Southern California $833,971
DISC2-11199 Modulation of the Wnt pathway to restore inner ear function

 

Stanford $1,394,870
DISC2-11109 Regenerative Thymic Tissues as Curative Cell Therapy for Patients with 22q11 Deletion Syndrome

 

Stanford $1,415,016

Finally, the Board approved the Agency’s 2019 research budget. Given CIRM’s new partnership with the National Heart, Lung, Blood Institute (NHLBI) to accelerate promising therapies that could help people with Sickle Cell Disease (SCD) the Agency is proposing to set aside $30 million in funding for this program.

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Congresswoman Barbara Lee (D-CA 13th District)

“I am deeply grateful for organizations like CIRM and NHLBI that do vital work every day to help people struggling with Sickle Cell Disease,” said Congresswoman Barbara Lee (D-CA 13th District). “As a member of the House Appropriations Subcommittee on Labor, Health and Human Services, and Education, I know well the importance of this work. This innovative partnership between CIRM and NHLBI is an encouraging sign of progress, and I applaud both organizations for their tireless work to cure Sickle Cell Disease.”

Under the agreement CIRM and the NHLBI will coordinate efforts to identify and co-fund promising therapies targeting SCD.  Programs that are ready to start an IND-enabling or clinical trial project for sickle cell can apply to CIRM for funding from both agencies. CIRM will share application information with the NHLBI and CIRM’s Grants Working Group (GWG) – an independent panel of experts which reviews the scientific merits of applications – will review the applications and make recommendations. The NHLBI will then quickly decide if it wants to partner with CIRM on co-funding the project and if the CIRM governing Board approves the project for funding, the two organizations will agree on a cost-sharing partnership for the clinical trial. CIRM will then set the milestones and manage the single CIRM award and all monitoring of the project.

“This is an extraordinary opportunity to create a first-of-its-kind partnership with the NHLBI to accelerate the development of curative cell and gene treatments for patients suffering with Sickle Cell Disease” says Maria T. Millan, MD, President & CEO of CIRM. “This allows us to multiply the impact each dollar has to find relief for children and adults who battle with this life-threatening, disabling condition that results in a dramatically shortened lifespan.  We are pleased to be able to leverage CIRM’s acceleration model, expertise and infrastructure to partner with the NHLBI to find a cure for this condition that afflicts 100,000 Americans and millions around the globe.”

The budget for 2019 is:

Program type 2019
CLIN1 & 2

CLIN1& 2 Sickle Cell Disease

$93 million

$30 million

TRANSLATIONAL $20 million
DISCOVER $0
EDUCATION $600K

 

 

Maria Millan Opens Up About the Current State of CIRM

In an article published to the SF Chronicle last week, reporter Erin Allday and Joaquin Palomino discussed CIRM, and how our work as an agency has paid off since our inception in 2004.

The article, which is a part of a four part series, explores the hope and reality of the revolutionary science of stem cell therapy. It focuses on what has transpired since 2004, when California voters approved a $3 billion bond measure to fund stem cell research with the promise that it soon would produce new treatments for incurable diseases.

In four parts, it follows the stories of patients desperately seeking remedies; probes the for-profit clinics where unproven and unregulated treatments are being offered; takes you into the labs and hospital rooms where scientists are testing new therapies; and provides a comprehensive accounting of what California’s multi billion dollar bet on stem cells has achieved.

Our CEO Maria Millan shared her thoughts in response to some of the questions raised by this article.

Q: There have been many critics who say it’s taking too long for CIRM to deliver cures, and they expected more. What is your response to these people?

A: Many of us can relate that relief cannot come quickly enough for our relatives and friends who suffer from debilitating and devastating medical conditions— I believe that is why many of us are at CIRM, an organization whose mission is to accelerate stem cell treatments to patients with unmet medical needs. Through the years, we have enabled the creation of an incredible ecosystem of top scientists and researchers and partnered with patients and patient advocates to pursue this mission.  We continually strive to improve and to become more efficient  and we share the sense of urgency to harness the potential of stem cell biology to deliver relief to those in need.

Q: Given all of the differences between CIRM and the NIH, why do you think the reporter compared CIRM to the NIH?

A: The NIH is the largest health research funder world-wide, has been around a lot longer, has a much larger budget >$30B this past year alone and the NHLBI alone has a $3B annual budget—NHLBI is just one of the 27 NIH Institutes. The reason that CIRM was formed is that the advocates of Proposition 71 wanted to make sure that scientists and developers can pursue vital research opportunities that may not have access to funding by traditional funders, including the NIH. CIRM has a total budget of $3B available to fund research and support operations and we have been managing that budget since the passage of Proposition 71 in 2004.  If we consider  the number of stem cell trials for given available budget, CIRM has funded a disproportionately higher number of translational and clinical programs in stem cell and regenerative medicine. In fact, the NHLBI has entered into a collaboration with CIRM on their Cure Sickle Cell initiative because of CIRM’s specialization in funding and enabling cell-gene regenerative medicine research.  I take this as a validation of CIRM’s value proposition in this new area– acceleration, translation, and clinical trials.

Q: Given the visibility being given to stem cell tourism and direct to consumer marketing of unproven and unregulated therapies, what value does CIRM bring to patients?

A: CIRM is positioned as the trusted agency for delivering on high quality trials, for being on the side of patients for safe trials and treatments and is a credible partner for patient, industry, government stakeholders to tackle this issue of stem cell tourism. We only fund clinical trials backed by solid scientific data with FDA permission to test it in patients. We have funded research being conducted at top tier medical centers and created the Alpha Stem Cell Clinic Network to provide people with high quality clinical trials. We are remaining in close contact with our legislators, including Speaker Pro Tem Kevin Mullin, Chair of the Assembly Select Committee on Biotechnology, to evaluate potential ways to protect patients from illegitimate clinics while progress is being made with legitimate approaches.

Q: Do you feel that enough money is being put towards basic research?

A: If we had the budget to do so, we would like to be able to fund more discovery research as well as translational and clinical research.  However, approximately $880M CIRM funds have gone into basic research thus far.  CIRM has a specialized and very unique role that supports and fosters rigorous stem cell and regenerative medicine science but, CIRM has distinguished itself as an agency that specializes in accelerating the translation of this science to therapies for patients.

Dr. Millan also appeared with SF Chronicle reporter Erin Allday and UC Davis stem cell scientist Paul Knoepfler on a Facebook Live talk about the work of the stem cell agency.

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

 

 

CIRM invests in stem cell clinical trial targeting lung cancer and promising research into osteoporosis and incontinence

Lung cancer

Lung cancer: Photo courtesy Verywell

The five-year survival rate for people diagnosed with the most advanced stage of non-small cell lung cancer (NSCLC) is pretty grim, only between one and 10 percent. To address this devastating condition, the Board of the California Institute for Regenerative Medicine (CIRM) today voted to invest almost $12 million in a team from UCLA that is pioneering a combination therapy for NSCLC.

The team is using the patient’s own immune system where their dendritic cells – key cells in our immune system – are genetically modified to boost their ability to stimulate their native T cells – a type of white blood cell – to destroy cancer cells.  The investigators will combine this cell therapy with the FDA-approved therapy pembrolizumab (better known as Keytruda) a therapeutic that renders cancer cells more susceptible to clearance by the immune system.

“Lung cancer is a leading cause of cancer death for men and women, leading to 150,000 deaths each year and there is clearly a need for new and more effective treatments,” says Maria T. Millan, M.D., the President and CEO of CIRM. “We are pleased to support this program that is exploring a combination immunotherapy with gene modified cell and antibody for one of the most extreme forms of lung cancer.”

Translation Awards

The CIRM Board also approved investing $14.15 million in four projects under its Translation Research Program. The goal of these awards is to support promising stem cell research and help it move out of the laboratory and into clinical trials in people.

Researchers at Stanford were awarded almost $6 million to help develop a treatment for urinary incontinence (UI). Despite being one of the most common indications for surgery in women, one third of elderly women continue to suffer from debilitating urinary incontinence because they are not candidates for surgery or because surgery fails to address their condition.

The Stanford team is developing an approach using the patient’s own cells to create smooth muscle cells that can replace those lost in UI. If this approach is successful, it provides a proof of concept for replacement of smooth muscle cells that could potentially address other conditions in the urinary tract and in the digestive tract.

Max BioPharma Inc. was awarded almost $1.7 million to test a therapy that targets stem cells in the skeleton, creating new bone forming cells and blocking the destruction of bone cells caused by osteoporosis.

In its application the company stressed the benefit this could have for California’s diverse population stating: “Our program has the potential to have a significant positive impact on the lives of patients with osteoporosis, especially in California where its unique demographics make it particularly vulnerable. Latinos are 31% more likely to have osteoporosis than Caucasians, and California has the largest Latino population in the US, accounting for 39% of its population.”

Application Title Institution CIRM funding
TRAN1-10958 Autologous iPSC-derived smooth muscle cell therapy for treatment of urinary incontinence

 

 

Stanford University

 

$5,977,155

 

TRAN2-10990 Development of a noninvasive prenatal test for beta-hemoglobinopathies for earlier stem cell therapeutic interventions

 

 

Children’s Hospital Oakland Research Institute

 

$1,721,606

 

TRAN1-10937 Therapeutic development of an oxysterol with bone anabolic and anti-resorptive properties for intervention in osteoporosis  

MAX BioPharma Inc.

 

$1,689,855

 

TRAN1-10995 Morphological and functional integration of stem cell derived retina organoid sheets into degenerating retina models

 

 

UC Irvine

 

$4,769,039

 

Breaking down barriers to advance stem cell therapies – the view from the Vatican conference

Perry and the Pope

Pope Francis meets Katy Perry at the Unite to Cure conference at the Vatican

All hands were on deck at the “Unite to Cure” conference, organized by the Cura Foundation and the Vatican Pontifical Council,  and held at the Vatican on April 26-28. Religious leaders, scientists, physicians, philanthropists, industry leaders, government, academic leaders and members of the entertainment industry gathered to discuss how to improve human health and to increase access to relief of suffering for the under-served around the world.

Pope Francis spoke of “the great strides made by scientific research in discovering and making available new cures” but stressed that science also needs to have “an increased awareness of our ethical responsibility towards humanity and the environment in which we live.”

He talked of the importance of addressing the needs of children and young people, of helping the marginalized and those with rare, autoimmune and neurodegenerative diseases. He said:

“The problem of human suffering challenges us to create new means of interaction between individuals and institutions, breaking down barriers and working together to enhance patient care.”

So, it was appropriate that breaking down barriers and improving collaboration was the theme of a panel discussion featuring CIRM’s President and CEO, Maria Millan. She had been invited to attend the conference and participate on a panel focusing on “Public Private Partnerships to Accelerate Discoveries”.

As Dr. Millan put it, “Collaboration, communication, and alignment” is the winning formula for public/private partnerships.

She highlighted how CIRM exemplifies this new approach, how everything we do is focused on accelerating the field and that means partnering with the National Institutes of Health and the Food and Drug Administration to create new regulatory models. It also means working with scientists every step of the way; helping them prepare the best possible application for CIRM funding and, if they are approved, giving them the support they need to help them succeed.

It was a wide ranging, thoughtful, engaging conversation with David J. Mazzo, PhD, President & CEO of Caladrius Biosciences and David  Pearce, PhD, Executive VP for Research at Sanford Health. You can watch the discussion here.

People may find it surprising that government agencies, academic researchers and private companies can all collaborate effectively.  It is absolutely critical to do so in order to rapidly and safely advance transformative stem cell, gene and regenerative medicine to patients with unmet medical needs.  Pope Francis and the Pontifical Council at the Vatican certainly believe that collaboration is essential and the “Unite to Cure” Conference was a powerful demonstration of how important it is to work together for the future of humanity.

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.

 

 

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.

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.

MariaMillan-085_600px

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.

A road trip to the Inland Empire highlights a hot bed of stem cell research

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Gillian Wilson, Interim Vice Chancellor, Research, UC Riverside welcomes people to the combined Research Roadshow and Patient Advocate event

It took us longer than it should have to pay a visit to California’s Inland Empire, but it was definitely worth the wait. Yesterday CIRM’s Roadshow went to the University of California at Riverside (UCR) to talk to the community there – both scientific and public – about the work we are funding and the progress being made, and to hear from them about their hopes and plans for the future.

As always when we go on the road, we learn so much and are so impressed by everyone’s passion and commitment to stem cell research and their belief that it’s changing the face of medicine as we know it.

Dr. Deborah Deas, the Dean of the UC Riverside School of Medicine and a CIRM Board member, kicked off the proceedings by saying:

“Since CIRM was created in 2004 the agency has been committed to providing the technology and research to meet the unmet needs of the people of California.

On the Board I have been impressed by the sheer range and number of diseases targeted by the research CIRM is funding. We in the Inland Empire are playing our part. With CIRM’s help we have developed a strong program that is doing some exciting work in discovery, education and translational research.”

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CIRM’s Dr. Maria Millan at the Roadshow Patient Advocate event

CIRM’s President and CEO, Dr. Maria T. Millan, and our Board Chair, Jonathan Thomas then gave a quick potted history of CIRM and the projects we are funding. They highlighted how we are creating a pipeline of products from the Discovery, or basic level of research, through to the 45 clinical trials we are funding.

They also talked about the Alpha Clinic Network, based at six highly specialized medical centers around California, that are delivering stem cell therapies and sharing the experiences and knowledge learned from these trials to improve their ability to help patients and advance the field.

Researchers from both UCR then gave a series of brief snapshots of the innovative work they are doing:

  • Looking at new, more efficient and effective ways of expanding the number of human embryonic stem cells in the laboratory to create the high volume of cells needed for therapies.
  • Using biodegradable materials to help repair and regenerate tissue for things as varied as bone and cartilage repair or nerve restoration.
  • Exploring the use of epigenetic factors, things that switch genes on and off, to try and find ways to make repairs inside the body, rather than taking the cells outside the body, re-engineering them and returning them to the body. In essence, using the body as its own lab to manufacture replacement.

Another CIRM Board member, Linda Malkas, talked about the research being done at City of Hope (COH), where she is the associate chair of the Department of Molecular and Cellular Biology, calling it an “engine for discovery that has created the infrastructure and attracted people with an  amazing set of skills to bring forward new therapeutics for patients.”

She talked about how COH is home to one of the first Alpha Clinics that CIRM funded, and that it now has 27 active clinical trials, with seven more pending and 11 more in the pipeline.

“In my opinion this is one of the crown jewels of the CIRM program. CIRM is leading the nation in showing how to put together a network of specialized clinics to deliver these therapies. The National Institutes of Health (NIH) came to CIRM to learn from them and to talk about how to better move the most promising ideas and trials through the system faster and more efficiently.”

Dr. Malkas also celebrated the partnership between COH and UCR, where they are collaborating on 19 different projects, pooling their experience and expertise to advance this research.

Finally, Christine Brown, PhD, talked about her work using chimeric antigen receptor (CAR) T cells to fight cancer stem cells. In this CIRM-funded clinical trial, Dr. Brown hopes to re-engineer a patient’s T cells – a key cell of the immune system – to recognize a target protein on the surface of brain cancer stem cells and kill the tumors.

It was a packed event, with an overflow group watching on monitors outside the auditorium. The questions asked afterwards didn’t just focus on the research being done, but on research that still needs to be done.

One patient advocate couple asked about clinics offering stem cell therapies for Parkinson’s disease, wondering if the therapies were worth spending more than $10,000 on.

Dr. Millan cautioned against getting any therapy that wasn’t either approved by the Food and Drug Administration (FDA) or wasn’t part of a clinical trial sanctioned by the FDA. She said that in the past, these clinics were mostly outside the US (hence the term “stem cell tourism”) but increasingly they are opening up centers here in the US offering unproven and unapproved therapies.

She said there are lots of questions people need to ask before signing up for a clinical trial. You can find those questions here.

The visit was a strong reminder that there is exciting stem cell research taking place all over California and that the Inland Empire is a key player in that research, working on projects that could one day have a huge impact in changing people’s lives, even saving people’s lives.