In developing new drugs for rare diseases, non-profit steps in when pharmaceutical companies won’t


(WASHINGTON) —  A research team has reason to celebrate after the Food and Drug Administration granted it approval on Friday to begin a clinical trial for a new pediatric brain cancer drug, one that might have ended up overlooked by pharmaceutical companies.

The lead researcher on the team, Dr. Teresa Purzner has already beat impossible odds. The neurosurgeon and mom of three managed to get the approval in record time and with little money thanks to the help of a team of ‘scientific altruists’ called SPARK.

The development of new medications in the United States is driven by pharmaceutical companies; researchers at universities rarely bring their discoveries to the bedside. For every 10,000 potential new medicines sitting on laboratory shelves around the country, only one will ever reach patients in need, according to the National Institutes of Health. Why? Because the process can take 10 to 15 years, costing upwards of a billion dollars per drug.

As a result, the number of new medications approved by the FDA has remained stagnant at about 31 per year over the past 10 years. The majority of these medications are similar to already existing ones, and many target diseases for which there are large markets — like hypertension and high cholesterol — and therefore, a return on investment.

Enter SPARK, a non-profit program created in partnership between Stanford University and volunteers from the biotechnology, pharmaceutical, and investment industries, which helps academic researchers bring their discoveries to patients. Since its founding, SPARK has given special consideration to projects typically neglected by pharmaceutical companies, including rare diseases and diseases affecting children.

Purzner put her neurosurgery practice on pause to study medulloblastoma, a type of childhood brain cancer. Compared to diseases like hypertension and high cholesterol, which affect millions of Americans, medulloblastoma is rare, affecting only 250 to 500 children every year.

“There’s something especially poignant about seeing children —beautiful, wonderful, innocent things — and seeing the impact of the therapies we are giving them. The medications, the radiation therapies impact their cognition, their quality of life and their ability to function as independent adults in the future,” Purzner said in an interview with ABC News.

Purzner had a clear goal: to find a targeted therapy that could shut down the basic biochemical pathway responsible for the development of this cancer, and she did. She tested the potential drug in mice with good results, and she just received FDA approval to test it in clinical trials, which she will do through the Pediatric Brain Tumor Consortium. She did it all in five years and for a price tag of $500,000.

“To get from my initial findings in the lab to the point where the Pediatric Brain Tumor Consortium picked it up for clinical trials would have never happened without the help of SPARK… they gave me a clear pathway and made me believe it was possible,” said Purzner.

Every year, SPARK provides 10 teams with funding and expert mentorship to promote efficient and cost-effective drug development.

“This is a case of scientific altruism, scientific advisors who donate their time… these individuals are just remarkably generous,” said Dr. Kevin Grimes, professor of chemical and systems biology at Stanford University, practicing physician, and co-director of SPARK.

For SPARK’s founder, Dr. Daria Mochly-Rosen, a professor in chemical and systems biology at Stanford, the organization’s mission is personal.

“SPARK has been the better part of my life,” Mochly-Rosen told ABC news.

And like Purzner’s story, there are many. Sixty percent of SPARK projects have entered clinical trials or have been licensed to companies.

“SPARK addresses a black hole that neither NIH or biopharma can fill,” Dr. Roham Zamanian, associate professor of medicine and director of the Adult Pulmonary Hypertension Program at Stanford University, told ABC News. It allows researchers to move forward drugs that would otherwise have no path, addressing the high cost of drug development and bringing precision medicine to patients, explained Zamanian.

Zamanian’s own drug for the treatment of a rare lung disease, known as pulmonary hypertension, will be tested in a global clinical trial this year.

SPARK’s model has been replicated at 12 academic institutions around the world.

“It might be one way to have an impact on the health care cost crisis,” Zamanian said, “while bringing precision and personalized medicine that maximizes benefits and decreases toxicity for patients.”

Dr. Edith Bracho-Sanchez is a board-certified pediatrician, a Stanford Global Health and Journalism fellow and a member of the ABC News Medical Unit.

Copyright © 2019, ABC Radio. All rights reserved.

North Dakota State wins record seventh National Championship

Sports News North Dakota State wins record seventh National Championship

Bo Rader/Wichita Eagle/TNS via Getty Images(NEW YORK) — North Dakota State Bison have won their seventh Football Championship Subdivision National Championship in the past eight years with a 38-24 win over Eastern Washington.

The seven FCS national championships breaks the FCS record that was previously held by Georgia Southern.

It was the final game for fifth year head coach Chris Klieman, who is taking over at Kansas State. Klieman joins former Youngstown State head coach Jim Tressel as the only four-time winners.

Defensive coordinator Matt Entz has been named the next head coach.

Quarterback Easton Stick improved to 49-3 as a starter, breaking former NDSU QB Brock Jensen’s record for the most wins by an FCS QB.

The title was the school’s fifteenth total title, with three coming from their time in the College Divisions and five Division II crowns.

The Bison finish the season 15-0. They won 21 straight games, the current longest streak in Division One.

Since the Bison’s first title season in 2011, they’re 111-8.

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Posted On 05 Jan 2019

Knicks center Enes Kanter to skip trip to London

Sports News Knicks center Enes Kanter to skip trip to London

Matthew Stockman/Getty Images(NEW YORK) — New York Knicks Center Enes Kanter will not travel with the team to London when the team face’s the Washington Wizards on January 17 due to his ongoing going clash with Turkish president Recep Tayyip Erdogan.

Kanter, a native of Turkey, told the media he fears for his life.

“I talked to the front office and said I’m not going because of that freaking lunatic, the Turkish President,” he said following the Knicks 119-112 of the Lakers on Friday night. “There’s a chance that I could get killed out there.”

In 2016, Kanter spoke out against Erdogan following a bombing in Turkey’s capital.

The following year, his Turkish passport was canceled and he was held at the Bucharest airport upon landing in Romania.

Kanter also believes his father has been a target of the government because of Kanter’s stance against it.

Asked if he really believes he would be killed if he traveled Kanter said, “Oh yeah easy.”

Copyright © 2019, ABC Radio. All rights reserved.

Posted On 05 Jan 2019

Scoreboard roundup — 1/4/19

Sports News Scoreboard roundup -- 1/4/19

iStock(NEW YORK) — Here are the scores from yesterday’s sports events:
 Final  Utah           117  Cleveland     91
 Final  Minnesota      120  Orlando      103
 Final  Miami          115  Washington   109
 Final  Brooklyn       109  Memphis      100
 Final OT  Indiana        119  Chicago      116
 Final  Boston         114  Dallas        93
 Final  Milwaukee      144  Atlanta      112
 Final  L.A. Clippers  121  Phoenix      111
 Final  N-Y Knicks     119  L.A. Lakers  112
 Final  Oklahoma City  111  Portland     109

 Final  Pittsburgh   4  Winnipeg      0
 Final OT  Detroit      4  Nashville     3
 Final  Carolina     4  Columbus      2
 Final OT  Dallas       2  Washington    1
 Final SO  New Jersey   3  Arizona       2
 Final  Colorado     6  N-Y Rangers   1
 Final  Vegas        3  Anaheim       2

 Final  (20) Buffalo   74  E. Michigan   58

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Posted On 05 Jan 2019