Written by: Dr. Kouzi

Published: 14 November 2017

Duke University Medical Center has received permission from the FDA to offer cord blood therapy for conditions like autism spectrum disorder and cerebral palsy under an expanded access clinical trial. This protocol establishes an umbrella clinical trial NCT03327467registered on 31 Oct. 2017 which enables children who have these neurological disorders to receive therapy with their own cord blood or cord blood from a sibling, regardless of whether they qualify for a targeted clinical trial.

The registration of this clinical trial is a watershed moment, opening the door for many children who are afflicted with an acquired neurological disorder to travel to Duke University for cord blood therapy, provided they have a suffienctly matching cord blood unit in a family bank. Sibling therapy only requires a partial match, not a perfect match. This expanded access protocol is a triple win for patients, family cord blood banks, and Duke University Medical Center.

The FDA may grant permission for expedited access (aka expanded access) under one of four designation pathways. In order to be considered for the FDA's expedited access prorgram, the new therapy must treat a “serious condition” that that has substantial impact on day-to-day functioning. In addition, there must be an “unmet medical need” because the condition is not addressed adequately by therapeutic alternatives.

The Breakthrough Therapy designation for expedited access is based on the results of phase 2 clinical trials. Duke University has conducted multiple clinical trials investigating the use of both autologous and allogeneic umbilical cord blood (UCB) in the treatment of cerebral palsy (NCT01147653, NCT02599207) and autism spectrum disorder (NCT02847182). As stated in the new open access clinical trial, “The use of (UCB) in this fashion is based on safety and efficacy data from prior and ongoing clinical trials at Duke University Medical Center in over 700 patients with these diagnoses infused with autologous or sibling UCB over the past decade.”

In the United States, the Centers for Disease Control and Prevention reports that the prevalence of cerebral palsy is 1 in 323 children (0.3%) and the prevalence of autism spectrum disorder is 1 in 68 children (1.5%). However, in order to be eligible to participate in the new clinical trial, patients must have their own or a sibling’s cord blood preserved in a family bank. To date, the only study that has examined the prevalence of medical conditions among families with privately stored cord blood is a recent publication that surveyed clients of Cord Blood Registry^®(CBR^®). The authors found that, out of 94,803 respondent families, 4.23% reported at least one child with an indication for regenerative therapy with cord blood. For conditions similar to autism spectrum disorder and cerebral palsy, the combined prevalence was 2.18%.

Worldwide, there are projected to be tens of thousands of children who are eligible to take advantage of this new treatment pathway. In the United States alone, there are over a million cord blood units in family storage, so that if 2% of the inventory corresponds to children with eligible conditions, that potentially translates into 20,000 patients.

Breaking News: The Cord Blood Association (CBA) announces 13 Nov. 2017 they are seeking funds to convert this protocol into a multi-center clinical trial administered by CBA and Duke.

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Written by: Dr. Kouzi

Published: 11 October 2017

Wednesday, October 11th 2017 marks Stem Cell Awareness Day, which brings together organizations and individuals around the world working to ensure that we realize the benefits of one of the most promising fields of science in our time. The day is a unique global opportunity to foster greater understanding about stem cell research and the range of potential applications for disease and injury.

For the millions of people around the world who suffer from incurable diseases and injury, Stem Cell Awareness Day is a day to celebrate the scientific advances made to-date and be hopeful of what is yet to come.

Research and academic institutions and educators are encouraged to participate by hosting public talks and other activities and events in their community.  In particular, we encourage efforts to get stem cell researchers into high school classrooms that day.

Follow events on twitter: #StemCellAwarenessDay

Visit our Get Involved page to learn about stem cell research outreach and advocacy all year long

Questions? Contact This email address is being protected from spambots. You need JavaScript enabled to view it.

Lecturer Resources

·      Introduction to stem cells lecture, no videos [ppt]

·      Introduction to stem cells lecture, with videos [zip] (Note: this is a large file and may take a few minutes to download)

 

2017 Events Calendar

California Events

Alliance for Regenerative Medicine, Annual Public Forum

October 5th: 6:00-6:45 PM

·      As part of the Stem Cell Meeting on the Mesa, the Alliance is hosting a public forum, “TOWARDS A BETTER UNDERSTANDING OF NEUROPSYCHIATRIC DISORDERS: THE ROLE OF SYNAPSES” featuring Speaker and Nobel Laureate Thomas Sudof, M.D.

·      Networking event to follow.

·      Auditorium at the Sanford Consortium for Regenerative Medicine, 2880 Torrey Pines Scenic Drive | La Jolla, CA 92037

CIRM Patient Advocate Event (UC Davis MIND Institute, Sacramento, CA)

October 10th: 11 AM - 1 PM

·      The California Institute for Regenerative Medicine (CIRM) and UC Davis welcome you to a free patient advocate event about how stem cell therapies are helping patients with unmet medical needs. Stem cell research is now providing cures and life-changing treatments to patients facing a wide array of diseases and disorders. Learn about the exciting progress being made in stem cell research and what it could mean for you, your family and loved ones. This meeting is FREE and open to the public. We hope that you'll join us!

·      ​RSVP on Eventbrite.

University of California, Irvine

October 11th: 2 PM

·      Join the UC Irvine Stem Cell Research Center at their Stem Cell Awareness Day Open House. Learn about the scientific advances being made and see

the future of medicine at their public open house including self guided tours, meeting UCI scientists, and lab demonstrations. Tours from 2-3, 3:30-4:30 PM.
Registration not required, see UCI flyer for more details. 

University of California, Berkeley

October 14th: 9:00 AM - 5:00 PM

·      The UC Berkeley's Student Society of Stem Cell Research (SSSCR) is hosting its 5th annual Stem Cell Conference: Culturing a Stem Cell Community. This conference aims to bring together different aspects of stem cell research, from science to advocacy, to demonstrate the growth and success of the field.

·      RSVP on Eventbrite (tickets cost a small fee of $7 or $12 including lunch to support the cost of the SSSCR conference)

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Events set for 2018

It's never too early to make plans for Stem Cell Awareness Day 2017, October 10th!! Contact us at This email address is being protected from spambots. You need JavaScript enabled to view it. and we'll include your event here.

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Events for 2016

California Events

Alliance for Regenerative Medicine

October 6

·      5:45-6:45 pm: As part of the Stem Cell Meeting on the Mesa, the Alliance is hosting a public forum, “Stem Cells, A History of Freedom, Hope and Reality” featuring Elena Cattaneo of the University of Milan

·      Networking event to follow

·      Auditorium at the Sanford Consortium, 2880 Torrey Pines Scenic Drive, La Jolla, CA

University of California, Irvine

October 14: 8:30 a.m. - 6:30 p.m.

·      Annual Stem Cell Awareness Day Science Symposium featuring leading scientists leading scientists who are experts in the field of stem cell science and regenerative medicine. Postdoc and Grad Students are invited present their research and posters at the symposium.

·      Attendance is free but registration is required.

Buck Institute for Research on Aging, Novato

October 15: 9:00 a.m. - 10:30 a.m.

·      Learn about the promise of regenerative medicine and California’s unique role in moving potential therapies from the bench to the bedside. Meet communicators from the California Institute for Regenerative Medicine (CIRM) and talk to Buck researchers who are involved in stem cell research.

·      RSVP on Facebook

University of California, Berkeley

October 15th: 9:00 a.m. - 5:00 p.m.

·      The UC Berkeley's Student Society of Stem Cell Research (SSSCR) is hosting its 4th annual Stem Cell Conference: Culturing a Stem Cell Community. This conference aims to bring together different aspects of stem cell research, from science to advocacy, to demonstrate the growth and success of the field.

·      RSVP on Eventbrite (tickets cost a small fee of $7 to support the cost of the SSSCR conference)

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Events for 2015

California Events

University of California, Riverside

October 8: 5:00 p.m. to midnight

·      Three stem cell scientists will be among the several dozen artists, performers and other scientist presenting during the seven-hour “Long Night of Arts and Innovation.”

·      The organizers describe it this way: “This evening, Downtown Riverside is alive with the arts and sciences. You will experience exhibits in which the science is so beautiful that it feels like art, and others in which the art is so technical it feels like science.“

San Diego State University

October 14: 6:00 p.m.

·      A public symposium on the how stem cell research and regenerative medicine is transforming the way doctors treat disease.

·      San Diego State University, 5500 Campanile Drive

University of California, Irvine

The Stem Cell Research Center at UC Irvine is planning a number of activities at the Sue & Bill Gross Hall on campus:

October 14

·      High School Tours for Irvine and Tustin school districts

·      Open House for the surrounding community, tours 2:00-3:00pm or 3:30-4:30pm

·      The self guided tours include opportunities to meet the scientists and lab demonstrations.

Questions, contact This email address is being protected from spambots. You need JavaScript enabled to view it.

October 28

·      8:30am-3:15pm: Stem Cell Awareness Day Scientific Symposium: “Stem Cells, Skin Cells, Cancer Cells: Basic and Translational Challenges and Opportunities”

·      RSVP by 10/19/15

University of California, Berkeley

October 17

·      9:00am-5:00pm: Public Symposium, “Culturing a Stem Cell Community”

·      Room 245 Li Ka Shing Center on the UC Berekeley Campus

·      For more information, visit the symposium website

University of California, Santa Barbara

October 14

·      Wednesday, October 14th 2015 marks Stem Cell Awareness Day, which brings together organizations and individuals around the world working to ensure that we realize the benefits of one of the most promising fields of science in our time. The day is a unique global opportunity to foster greater understanding about stem cell research and the range of potential applications for disease and injury. UC Santa Barbara researchers participated in the event with local outreach talks in Santa Barbara at the Vista Del Monte Retirement Community, the annual meeting of the UCSB Board of Trustees, and at San Marcos High School. In addition, our latest research was presented at scientific meetings, including the Stem Cell Meeting on the Mesa, the American Association of Blood Banks, and the Alliance for Regenerative Medicine in London. UC Santa Barbara is at the forefront of research in stem cell biology and engineering, with advances being made in understanding molecular mechanisms of stem cell biology, the discoveries of novel biotechnologies, and translation of cellular therapies for ocular disease. For information, please see www.stemcell.ucsb.edu.

The Parkinson’s Institute

·      The research community at the Parkinson's Institute will post a special edition of “Alex’s Stem Cell Blog”

The Scripps Research Institute

October 14

·      Noon-1pm: A free educational program with short talks on the basics of stem cells, the development of therapies for Parkinson’s Disease and Multiple Sclerosis and sports injuries, as well as the perils of stem cell tourism.  

·      Pizza and Snacks provided

·      3030 Science Park Road, La Jolla, CA

·      contact for more information: Estela Aliaga, This email address is being protected from spambots. You need JavaScript enabled to view it.

Alliance for Regenerative Medicine

October 8

·      5:45-6:45pm: As part of the Stem Cell Meeting on the Mesa, organized in partnership with CIRM, the Alliance is hosting a public forum, “The Promise of Regenerative Medicine: Are We There Yet,” featuring Jennifer Elisseeff of Johns Hopkins University

·      Networking event to follow

·      Auditorium at the Sanford Consortium, 2880 Torrey Pines Scenic Drive, La Jolla, CA

National Events

Americans for Cures

Ongoing

·      As part of the group’s Proposition 71 “Report Back to the Public” it launched a series of webinars, the first two were held September 28 and 29

·      Website: www.americansforcures.org 
 

Texas Heart Institute

October 14

·      The Texas Heart Institute celebrates Stem Cell Awareness Day: A message from President James T. Willerson

·      The Texas Heart Institute will be conducting and online campaign posting educational links throughout the day.
 

Texans for Stem Cell Research

October 29

5:00p-10:00p: In honor of Stem Cell Awareness Day, the Texans for Stem Cell Research will launch a new campaign “Cancer’s New Champions” in Austin, Texas at a special launch party and appreciation roast with a mystery honoree.
Zilker Park Clubhouse
Austin, TX