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Technology transfer's important role in discovery-to-commercialization story of FDA-approved immunotherapy to treat rare childhood cancer

April 17, 2015

An immunotherapy drug 20 years in the making called dinutuximab (ch14.18) is providing a newly approved treatment for infants and children with neuroblastoma, a rare cancer. The NCI Technology Transfer Center played an important role in this discovery-to-commercialization story through negotiation of a Cooperative Research Agreement (CRADA). Specifically, ch14.18 was provided for NCI-funded clinical studies through NCI’s Biopharmaceutical Development Program (BDP), and produced under NCI contracts/sub-contracts.  A TTC-negotiated CRADA allowed for transfer of the NCI BDP's evolving drug production process to United Therapeutics Corp., (UTC), a company that would take the product to licensure. The TTC-negotiated CRADA also allowed for NCI and UTC’s cooperative conduct of late-stage clinical trials of Ch14.18 that supported commercial development of Ch14.18 as a treatment in children with high-risk neuroblastoma.

Read more to learn about how ch14.18, now commercially known as Unituxin, received FDA approval. 

Also see: Harnessing the Power of Our Immune Systems to Treat Neuroblastoma: Discovery of Ch14.18 Immunotherapy

Reprinted from the Poster, NCI Frederick. (See

FDA Approves Immunotherapy for a Cancer that Affects Infants and Children

By Frank Blanchard, Staff Writer

The U.S. Food and Drug Administration (FDA) recently approved dinutuximab (ch14.18) as an immunotherapy for neuroblastoma, a rare type of childhood cancer that offers poor prognosis for about half of the children who are affected. The National Cancer Institute’s (NCI) Biopharmaceutical Development Program (BDP) at the Frederick National Laboratory for Cancer Research produced ch14.18 for the NCI-sponsored clinical trials that proved the drug’s effectiveness against the disease.

“It’s pretty cool,” said Doug Gaum, director of Quality Assurance at BDP, which also provided technical and regulatory support in transferring the technology, under an NCI Cooperative Research and Development Agreement, to United Therapeutics Corporation of Silver Spring, Md., and Research Triangle Park, N.C., for commercial production.

Neuroblastoma arises in infants and young children as a tumor of immature cells of the peripheral nervous system. More than 650 cases are diagnosed each year in North America, mostly in children under age 5, and more than one-third of these cases are diagnosed in infants, according to NCI. Half of all patients have a high-risk form of the disease and a poor prognosis. Dinutuximab is designed to benefit these high-risk patients.

Approval Was 25 Years in the Making

It was a long road to clinical approval. The lead scientist and study chair of the clinical trials, Alice Yu, M.D., Ph.D., University of California, San Diego, began pursuing ch14.18 as a cancer treatment about 25 years ago. BDP began producing the antibody for human testing in 1997.

During the Phase III trial in 2009, an interim analysis of results showed that ch14.18, given along with two immune-stimulating agents, was definitively effective. Among the 226 patients (median age 3.8 years) who received the combined therapy, 66 percent were alive and free of neuroblastoma after two years, compared with 46 percent in the control group, who received only the standard therapy.

As a result, the control group began to receive the combined therapy right away, and this became the new standard of treatment for pediatric neuroblastoma. Patients from the United States, Canada, Australia, and New Zealand enrolled in the trial to receive the drug, and BDP manufactured additional supplies to meet the demand. Results of the clinical trial were published in the New England Journal of Medicine in September 2010.

“After decades of pursuits, I am pleased to see that dinutuximab has received FDA approval and may now benefit high-risk neuroblastoma patients,” Yu said in a news release from United Therapeutics Corporation, which will manufacture and market the drug under the brand name UnituxinTM.

“This is not only the first successful immunotherapeutic to target a non-protein antigen, but also to be developed from an Investigational New Drug application through Phase III trials largely through investigator-initiated effort and NCI support,” Yu said.

The FDA approval calls for using Unituxin in combination with two immune-boosting compounds (IL-2 and GM-CSF) and the anticancer drug cis-retinoic acid. Unituxin links to cells of the immune system (natural killer cells and granulocytes) and directs those cells to attack and kill tumor cells.

Several members of BDP have been instrumental in supporting this project over the years. For Beverly Keseling, manager, Cell Culture, it was one of her first assignments when she joined Leidos Biomed, then named SAIC-Frederick, in the 1990s. Steve Giardina, Ph.D., a former employee, worked directly with NCI, the FDA, and Health Canada on issues of patient safety and product characterization. Keseling, Samir Shaban, manager, Manufacture, and their teams managed the bioreactor and purification units that worked extraordinary hours to ensure that the supply of ch14.18 met the demand for the drug during the transitional period prior to FDA approval. Sheryl Ruppel, director, Regulatory Affairs, and her group worked extensively with NCI’s Cancer Therapy Evaluation Program, the FDA, and Health Canada in developing regulatory submissions and ensuring that reviews stayed on track.

Annual Report to the Nation Shows Continuing Declines in Most Cancer Death Rates (L3)

March 31, 2015

The latest Annual Report to the Nation on the Status of Cancer, 1975-2011 (ARN) is now available, and key highlights can be found in In addition to reporting rates and trends for the most common cancers, this year’s ARN includes a special section on breast cancer by molecular subtype. Breast cancer subtypes have major implications for determining treatment and may hold important clues to the origins of breast cancer.

This is an extraordinary time for the Technology Transfer Center at NCI facilitating interactions and partnerships between researchers, academia and industry to further develop cancer technologies. We believe that our efforts have had a positive impact on these findings. Major findings from this year's report include:

Continuing declines in cancer death rates for both men and women, for children, and for most major cancer sites.
Continuing declines in the combined annual rate of new cases of cancer for men and women.
Trends that require greater evaluation, including increasing rates of thyroid, kidney, liver, oral/oropharyngeal, and cervical cancers.
National data on the incidence of breast cancer subtypes.

The Annual Report to the Nation was published online March 30, 2015, in the Journal of the National Cancer Institute.

Award-Winning Breast Cancer Startup Challenge Inspires Neuro Startup Challenge Launch

November 20, 2014

In July 2014, the Breast Cancer Startup Challenge (BCSC) was selected by Health and Human Services (HHS) Secretary Sylvia Mathews Burwell as one of three "Secretary's Picks" in the  HHS Innovates competition, an HHS-sponsored award program that identifies and recognizes HHS employees for their innovative work. Read the HHS IDEA Lab blog to learn more about the progress of the BCSC and how it enabled the launch of a new challenge, the Neuro Startup Challenge.


NCI Researchers Receive National Medal of Technology and Innovation for HPV Vaccine Advances

October 7, 2014

The NCI scientists whose discovery was the basis of the technology transfer to commercially develop HPV vaccines are among the recipients of this year’s National Medal of Technology and Innovation. Douglas R. Lowy, M.D., and John T. Schiller, Ph.D., received their medals at a White House ceremony on Nov. 20, 2014

Hear NCI Scientists Discuss their Discoveries - Featured Videos (L3)

August 21, 2014

Five new videos profiling NCI researchers have been added to the NCI YouTube channel:

These technologies may contain NCI inventions that are available for licensing and/or co-development collaborations. Please contact the NCI Technology Transfer Center for more information.

Breast Cancer Startup Challenge announces ten winning teams of entrepreneurs

March 6, 2014

The winners of a world-wide competition to bring emerging breast cancer research technologies to market faster were announced yesterday by the Avon Foundation for Women, in partnership with the National Cancer Institute (NCI) and the Center for Advancing Innovation (CAI).  Avon is providing $250,000 in funding for this Challenge. The Breast Cancer Startup Challenge is comprised of ten research technologies that were judged to show great promise to advance breast cancer research. Nine of the inventions were developed at NCI and one is from an Avon Foundation-funded university lab; they include therapeutics, diagnostics, prognostics, a device, a vaccine, a delivery system and a health IT invention. Teams of business, legal, medical/scientific, engineering, computer science students and seasoned entrepreneurs evaluated these technologies to create business plans and start new companies to develop and commercialize them.

Two hundred teams expressed an interest in joining the challenge and 46 teams were accepted to compete on a range of business plan possibilities. In total, 478 people participated in the competition. Given the number of teams and people on each team, this challenge is one of the largest global university business plan challenges to date.  The 10 business plan winners and finalists include:

  • Challenge #1 - Diagnostic from Biopsies with Software Analysis, Winner - University of Cambridge; Lead Inventor - Tom Misteli, Ph.D. (NCI)
  • Challenge #2 - Immunotherapy Using Modified Self Tumor Cells, Winner - Washington University in Saint Louis; Lead Inventor - Dennis Klinman, M.D., Ph.D. (NCI)
  • Challenge #3 - Combination of Tissue Reconstruction and Recurrence Prevention, Winner - Tulane University; Lead Inventor - Karen Burg, Ph.D. (Clemson University), Finalist - Clemson University
  • Challenge #4 - Human Monoclonal Antibody Based Cancer Therapies, Winner - Stanford University; Lead Inventor - Mitchell Ho, Ph.D. (NCI)
  • Challenge #5 - Immunotherapy Using Granulysin Activated Monocytes, Winner - Northwestern University; Lead Inventor - Alan Krensky, M.D., Northwestern University (formerly of NCI)
  • Challenge #6 - Anti-cancer Toxin, Winner - Rutgers University; Lead Inventor - Nadya Tarasova, Ph.D. (NCI)
  • Challenge #7 - Versatile Delivery Method for Cancer Therapeutics, Winner - University of Cambridge; Lead Inventors - Stanislaw J. Kaczmarczyk, Ph.D. & Deb Chatterjee, Ph.D. (NCI), 
  • Finalist - Wake Forest University
  • Challenge #8 - Genomic-based Diagnostic Assay, Winner - University of California, Berkeley; Lead Inventor: Steven Libutti, M.D., FACS, Albert Einstein College of Medicine (formerly of NCI)
  • Challenge #9 - Tissue-based Diagnostic Assay, Winner - McGill University; Lead Inventor - Stephen M. Hewitt, M.D., Ph.D. (NCI)
  • Challenge #10 - Diagnostic Kit for Therapy Benefit Prediction, Winner - Tulane University; Lead Inventor - Sherry Yang, MD., Ph.D. (NCI)

“NCI has always had a strong interest in fostering young investigators and the fact that this challenge pairs each student team with entrepreneur-mentors to assist in the development of the business plans is another example of how we can bring new ideas and energy to cancer research,” said Douglas Lowy, M.D., NCI deputy director.

Winners and finalists in the Breast Cancer Startup Challenge will not only be recognized for creating a business plan and pitch, as other competitions require, but they will also be invited to launch a start-up, negotiate licensing agreements and raise seed funding to further develop these NCI and Avon Foundation grantee inventions.


The Avon Foundation for Women, the world’s largest corporate-affiliated philanthropy focused on issues that matter most to women, was founded in 1955 to improve the lives of women. Through 2013, Avon global philanthropy, led by the Avon Foundation, has donated more than $957 million in more than 50 countries for causes most important to women. Today, Avon philanthropy focuses on funding breast cancer research and access to care through the Avon Breast Cancer Crusade. Visit for more information.

The National Cancer Institute (NCI) leads the National Cancer Program and the NIH effort to dramatically reduce the prevalence of cancer and improve the lives of cancer patients and their families, through research into prevention and cancer biology, the development of new interventions, and the training and mentoring of new researchers. For more information about commercial co-development and collaborative research opportunities at NCI, please visit the NCI Technology Transfer Center's website at or call the NCI Technology Transfer Center Marketing Office at 240-276-5515.

The Center for Advancing Innovation (CAI) is a global public-private partnership non-profit focused on creating a virtuous circle of innovation and driving growth breakthroughs through novel, creative paradigms and models.  For more information about CAI, please visit

First Breast Cancer Startup Challenge business plan winners to be announced

February 28, 2014

WHAT:     The Center for Advancing Innovation (CAI), the Avon Foundation for Women, and National Cancer Institute (NCI), part of the National Institutes of Health, will announce 10 business plan winners and finalists for the first Breast Cancer Startup Challenge. Each winning team will receive a $5,000 award from the Avon Foundation for Women and CAI.

WHEN:     March 5, 2014 from 1 to 3 p.m. EST. Media availability is limited before, during and after event. Interviews with awardees and other speakers can be arranged upon request. The event can be viewed via a live webcast at

WHERE:    National Cancer Institute, Shady Grove Campus, 9609 Medical Center Drive, Room: TE406 (terrace level of East wing), Rockville, Md. 20850

WHY:         The Breast Cancer Start-up Challenge provides teams of business, legal, medical/scientific, engineering, and computer science students, as well as seasoned entrepreneurs, the opportunity to create strategic business plans and start new companies focused on developing and commercializing 10 inventions that could show benefit in breast cancer and potentially other diseases.

WHO:       National Cancer Institute: Douglas Lowy, M.D., deputy director

                  Avon Foundation for Women: Marc Hurlbert, Executive Director

                  NCI Inventors: Tom Misteli, head, Cell Biology of Genomes Group; Mitchell Ho, head, Antibody Therapy Section Investigator; others

                  The Center for Advancing Innovation: Rosemarie Truman, Founder, CEO

                  Winners: (up to 10 awardees)

Examples of three inventions for business consideration in the Challenge include:

Pairing patients with optimal breast cancer treatment: Approximately one in eight women will be diagnosed with breast cancer in their lifetime. The Challenge invention will potentially be used to identify specific therapies that patients will be most responsive to, saving the patient from undergoing unnecessary and costly treatments.

New treatment for triple-negative breast cancer subtype: 63,000 women are diagnosed with late stage triple-negative disease annually and 70 percent of those women will die within 5 years.  The Challenge invention will potentially be used to develop a new class of therapeutics using a naturally occurring aspect of the person's immune system, thereby allowing the body to naturally respond to the cancer.

Biodegradable breast reconstruction and cancer recurrence prevention: Up to 20 percent of women who have a breast cancer removed surgically will see the cancer recur.  The Challenge invention could be applied to reconstructing the breast with a three dimensional, biodegradable platform with anti-tumor properties that is injected directly after breast tissue removal. This new process will hopefully eliminate the need for invasive cosmetic surgery and reduce cancer recurrence.

The Breast Cancer Start-up Challenge: Update

December 20, 2013

05 Feb 2014:

The National Cancer Institute’s Technology Transfer Center, the Avon Foundation and The Center for Advancing Innovation have partnered to create a “first-of-a-kind” Breast Cancer Start-up Challenge. The Challenge is a business plan and start-up challenge among multi-disciplinary university-led teams. The finalists in the best business plan phase of the challenge will launch a start-up, compete for seed funding, and negotiate a license for the invention in the final phase of the challenge.

The primary goal of the Challenge is to stimulate the creation of start-up businesses based upon NCI and Avon Foundation grantee inventions. The Challenge features ten assets that are viewed as having commercial viability with significant public health benefit.

The Challenge is a global effort. 39 teams are participating with team members from universities from Canada, India, the Netherlands, New Zealand, United Kingdom, and the United States. Winners of the Business Plan phase will be announced in late February 2014, and the successful start-ups will be announced Summer 2014.

Although the Challenge is no longer accepting new teams, the public can support the effort by spreading the word and following the Challenge via Facebook (, YouTube (, or Twitter (  For questions, comments, or suggestions, please contact Dr. Rose Freel or Dr. Tom Stackhouse of the NCI TTC (TTC Staff Directory).

NCI SBIR-Technology Transfer program

March 11, 2013

The National Cancer Institute's Small Business Innovation Research (NCI SBIR) Development Center and the NCI Technology Transfer Center created the NCI SBIR-Technology Transfer program in FY 2011 with the goal of incentivizing small businesses to collaborate with researchers of the NCI Intramural Research Program. An SBIR-TT contract provides funding to work under research and commercialization licenses and/or co-development research agreements to commercialize the NCI-patented technology while working closely with the NCI inventor. The inventor will assist the contractor in a collaborative manner with reagents and confidential discussions during the entire award period, and the awarded contractor will automatically be granted a royalty-free, non-exclusive license to use NIH-owned and patented background inventions within the scope and term of the award. An SBIR offeror or SBIR contractor can apply for an exclusive or non-exclusive commercialization license to make, use, and sell products or services incorporating the NIH background invention. Details of the current year's contract funding opportunities are available at

The National Cancer Institute's Small Business Innovation Research (NCI SBIR) Development Center released thirteen funding opportunities for FY2013

September 26, 2012

Included are two NCI SBIR Technology Transfer (SBIR-TT) topics based on inventions from NCI intramural researchers: Topic 317: Wound Healing Preparations Incorporating Nitric Oxide-Releasing Materials; and Topic 318: Test to Predict Effectiveness of Docetaxel Treatment for Prostate Cancer. The SBIR-TT program awards small business contracts to translate NCI-invented technology towards commercialization. Additional details on the FY2013 contract funding opportunities are available at