The superhero Iron Man inspired a three-year-old hemophilia patient to continue his treatment at the Children’s Hospital of Philadelphia (CHOP), where he had been a patient nearly all his life, and adopt the nickname “Iron Max.”

Max Levy, now six years old, was diagnosed with hemophilia as a baby. He and his older sister Zoe, eight, put together March Madness brackets to raise money for charity. This past year, they created a calendar featuring 26 children from CHOP. Their target was to raise $5,000, and so far they’ve raised $8,394, or 168 percent of their goal.

 few days before,” explained Max’s father, Dan Levy. “I said to him, ‘Do you remember the scene where Tony Stark takes the port, takes the arc reactor out of his chest and puts the new one in? ... you’re going to get one of those.’”

Marvel, the creators of Iron Max, even reached out Dan Levy and let him know that Max would appear in one of the comics.

“I thought, one of these is a superhero and I don't think it's the guy who wears the suit,” Dan Levy said after seeing Max appear on the back cover of the book with Iron Man. Max was also featured in the pages as a cartoon character.

Here’s the letter Zoe wrote to all the readers of 

I first started reading a story about a girl who raised thousands of dollars for cancer research, and since my brother Max has a bleeding disorder called hemophilia, I wanted to do something to raise money for hemophilia research.

Max has had hemophilia his whole life. We found out about it when he was nine months old, and he had to wear a helmet every day for three years. Just last year he got a port—a metal disk in his chest like, Iron Man. Now we call him Iron Max.

I was thinking what to do to raise money and I wanted to include my friends. Finally I thought to make a calendar so I asked my friends to take photos for the calendar. I can’t believe how many said yes!

All of the pictures in our calendar were taken by kids in grade school. All of my friends, and some of my family too, wanted to help raise awareness for hemophilia.

Articles

The superhero Iron Man inspired a three-year-old hemophilia patient to continue his treatment at the Children's Hospital of Philadelphia (CHOP), where he had been a patient nearly all his life, and adopt the nickname "Iron Max."

"Iron Max" Fights Hemophilia with Cameo in "Iron Man" Comics

Milk from rabbits may be used to find a cure for hemophilia, according to a report 

 in Framingham, Mass. engineered rabbits and raised them in order to produce milk with the blood clotting factor VII (FVII) agent for use in human patients with hemophilia. Their idea is that the rabbit produced milk could compete with hemophilia treatments made the “old fashioned way,” from human blood. The tests using rabbit produced FVII were in progress at press time in human patients in France.

“We’re getting ourselves positioned for launch and commercialization operations,” veterinarian and LFB USA president William G. Gavin told the 

. “This is the time to get this facility up and running.”

Previously, the researchers developed 1,000 genetically engineered goats, which were capable of producing the human breakup clot protein called antithrombin III in their milk. That protein is sold as ATryn by LFB subsidiary rEVO Biologics. It is designed to prevent clotting problems in patients who lack antithrombin either before surgery or childbirth. rEVO wants to expand the use of the goat protein by eventually testing it as a treatment for preeclampsia.

 The developers wanted to create an animal produced FVII, but knew the goats would not be appropriate.

“The rabbit milk did the best job of the carbohydrate pattern and the decoration on that molecule,” Gavin said.

About 410 New Zealand white rabbits reside in the 12,000 square foot facility and are tended to by between eight and 10 researchers. The company hopes to reach 1,000 rabbits on site by 2017. The 

article added that female rabbits are ready for reproduction and milk production at about five to six months old, and have litters as big as 10 kits. The female mothers produce milk for about three weeks after giving birth. One estimate said the average output of a rabbit it about 200 milliliters per day.

The rabbits are kept indoors, but are visible to the workers through windows. While the goats are kept together and reside in barns and are milked like any regular cow dairy, the rabbits are taken to hammock like structures and hooked up to tubes. The milking takes about six minutes and then the rabbits are permitted to go back into their cages which contain perches for climbing and balls to occupy themselves.

“We don’t support the use of transgenic animals, especially in that field, because there are other alternatives those companies could use” for production, Pascaline Clerk, senior director of policy and advocacy for the US Humane Society’s animal research issues department, told the 

However, LFB is not the only company testing animal milk for hemophilia cures. The Dutch company Pharming NV uses rabbits to produce a treatment for hereditary angioedema.

The LFB researchers added that notable developments in hemophilia treatments, including ACE910, are important to FVII production for patients.

Researchers from the LFB USA Inc. in Framingham, Mass. engineered rabbits and raised them in order to produce milk with the blood clotting factor VII (FVII) agent for use in human patients with hemophilia.

Using Rabbit Milk for Hemophilia Treatment

The use of twice-weekly prophylactic regimens to treat hemophilia A was shown to be successful among patients treated with BAX 855, based on Advate, according to findings 

, built upon data originally presented in 2014. BAX 855, known in the United States as Adynovate, was assessed for safety and efficacy profiles for bleed prevention with a twice-weekly dosing schedule. The mean half-life extension increased between 1.4 and 1.5 fold compared to Advate.

“ADYNOVATE has the potential to offer an important new option for patients, providing the combination of bleed prevention with a simple, twice-weekly dosing schedule,” Leonard Valentino, MD, global head, Hematology Medical Affairs, Baxalta, said in a press release. “The results of this pivotal trial demonstrate the ability of ADYNOVATE to help further personalize treatment regimens to minimize the impact that hemophilia has on patients’ lives.”

A total of 137 patients aged 12 to 65 years were assigned to the twice-weekly prophylaxis group or on demand treatment. The twice-weekly group experienced a 95 percent reduction in a median annualized bleed rate compared to the on-demand treatment arm of the program (1.9 vs. 41.5, respectively). Additionally, BAX 855 was successful in treated all bleeding episodes — 95.9 percent of which were controlled with either one or two infusions.

Nearly all (96.1 percent) of the treatment episodes were rated excellent or good. Almost 40 percent of the prophylactic group experienced no bleeds. Reported side effects included headache, diarrhea, nausea, and flushing in seven patients, though no serious adverse events were reported.

“Upon approval, Adynovate will be one of the first new treatments that we bring to market as Baxalta, representing a major milestone toward our plan to achieve 20 new product launches by 2020,” Ludwig Hantson, chief executive officer and president, Baxalta, added in the statement. “With a decades-long heritage in hematology and commitment to innovation, we continue to build a patient-centric portfolio that provides new solutions for evolving patient needs.”

Studies of BAX 855 continue, the statement explained, especially for children under the age of 12 years with severe hemophilia A. BAX 855 is based on Advate, a drug that has been on the market for more than a decade. It is designed to prolong the amount of FVIII available for the body to use and the technology behind the drug is present in several treatments approved for chronic or otherwise serious conditions.

The use of twice-weekly prophylactic regimens to treat hemophilia A was shown to be successful among patients treated with BAX 855.

Twice-Weekly BAX 855 Treatment Viable for Hemophilia

Late last year, the US Food and Drug Administration (FDA) granted the bispecific antibody “ACE910” from Chugai Pharmaceutical Co, Ltd with a Breakthrough Therapy Designation as a treatment for hemophilia A, according to a company 

ACE910 is designed for the prophylactic treatment of people aged 12 years or older with hemophilia A with factor VIII (FVIII) inhibitors. The drug mimics the job responsibilities of FVIII, which the company describes as a novel concept for hemophilia A treatment.

Researchers are conducting phase I and II clinical trials to examine the safety and efficacy of ACE910 for preventing bleeding in once weekly subcutaneous injections in patients both with and without inhibitors. The 

 that in all patients (no matter the presence of inhibitors) once weekly ACE910 injections were effective, and bleeding was completely controlled in 50 percent of the patients during ACE910 administration.

“We are very pleased that the FDA has granted Breakthrough Therapy Designation to ACE910,” said Chugai’s Director and Executive Vice President, Dr. Yutaka Tanaka. “We believe with its new mode of action, ACE910 will contribute to the patients by offering new choice of treatment options.”

Adverse effects were reported in 18 patients, but they were all considered mild or moderate; there were six patients in which the researchers were unable to rule out as having a causal relationship with ACE910. The common symptom was injection site erythema. No evidence pointed to clinically relevant abnormalities of coagulation indicated by clinical findings or laboratory tests in any cohort, the researchers added. There were also no thromboembolic adverse events reported, even when ACE910 was administered concomitantly with FVIII products or bypassing agents as on demand therapy for bleeding events, the researchers concluded.

Swiss drug maker Roche is also involved in the production of ACE910. The company will prepare its own separate phase III trials throughout 2016, in similar patient cohorts to the Chugai studies. This FDA designation proves that the FDA is “recognizing an unmet need for patients with inhibitors and the promise of these early data,” Roche Chief Medical Officer Sandra Hornung 

Late last year, the US Food and Drug Administration (FDA) granted the bispecific antibody â€œACE910â€ from Chugai Pharmaceutical Co, Ltd with a Breakthrough Therapy Designation as a treatment for hemophilia A.

Hemophilia A Drug Granted Breakthrough Therapy Designation

All von Willebrand Disease patients successfully managed bleeding episode using BAX 111 treatment, according to a study conducted by Baxalta. The study was published in the journal 

The researchers observed the on demand treatment of 37 von Willebrand patients around the world to determine if BAX 111 would successfully treat bleeding episodes. They also evaluated the drug’s efficacy measures, pharmacokinetics, and the number of infusions and the number of units administered for each bleeding episode.

“Von Willebrand disease is the most common hereditary bleeding disorder, yet few treatment options exist,” John Orloff, MD, head of Research & Development and chief scientific officer, Baxalta, explained in a 

. “BAX 111 has the potential to transform the standard of care, especially for patients with severe von Willebrand disease, by offering an effective individualized treatment option.”

The patients reported a mean efficacy rating of <2.5 on a four-point scale — in the study, 1 represented excellent and 4 demonstrated no bleeding control. Using one infusion was sufficient to control a majority of the bleeds, 81.8 percent. A median of two infusions were required to treat major bleeds.

The researchers did not report any thrombotic events or severe allergic reactions. No patients reported anti von Willebrand factor binding or neutralizing antibodies to von Willebrand factor.

Side effects were reported in the trial, the most common of which included headache, nausea or vomiting, and anemia. These side effects were not considered related to the BAX 111 treatment. However, there was one patient who experienced two simultaneous series adverse events that were considered related to the treatment: chest discomfort and increased heart rate during infusion. Those adverse effects were resulted within three hours and no further symptoms were reported.

BAX 111 is different from other recombinant replacement treatments for managing von Willebrand disease patients’ bleeding episodes because it contains a higher concentration of ultra large molecular weight multimers — the statement added that these demonstrate high functional activity.

All von Willebrand Disease patients successfully managed bleeding episode using BAX 111 treatment, according to a study conducted by Baxalta. The study was published in the journal Blood. 

Drug Manages von Willebrandâ€™s Disease with 100 Percent Efficacy

The clotting factor deficiency causing hemophilia A may be reversed using pluripotent stem cells (iPSCs), according to findings 

Researchers from the Institute for Basic Science and Yonsei University in Korea used mice models of hemophilia A in order to prove the viability of iPSCs as a cure for hemophilia A. For the first time ever, the investigators collected urinary cells from patients with the chromosomal inversions causing hemophilia to make the iPSCs. The team used Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) associated protein 9 to apply Cas9 nucleases to the iPSCs.

“We used CRISPR RNA-guided engineered nucleases (RGENs) to repair two recurrent, large chromosomal inversions responsible for almost half of all severe hemophilia A cases,” study author Jin-Soo Kim said to 

The CRISPR Cas9 corrected the FVIII genes and permitted the iPSCs to mature into endothelial cells, in turn, producing the FVIII gene. The endothelial cells reversed the FVIII deficiency, the researchers explained.

But the team wanted to make sure the process worked. They transplanted the engineered endothelial cells with the corrected genes into FVIII deficient mice (those with hemophilia A). the mice produced FVIII on their own, which the study authors believes meant that the mice’s hemophilia A was essentially cured.

“To the best of our knowledge, this report is the first demonstration that chromosomal inversions or other large rearrangements can be corrected using RGENs or any other programmable nuclease in patient iPSCs,” study author Dong-Wook Kim explained.

Even more importantly, the researchers highlighted, was that there did not appear to be any additional accidental mutations from the iPSC transplants. The only parts of the genome that were altered were the parts the study authors targeted.

In the future, the team believes that further testing is necessary and may determine if human results are similar to the mice trials. They hinted that such studies with positive results appear promising.

Researchers report the clotting factor deficiency causing hemophilia A may be reversed using pluripotent stem cells (iPSCs).

March 2016

"Iron Max" Fights Hemophilia with Cameo in "Iron Man" Comics

Using Rabbit Milk for Hemophilia Treatment

Twice-Weekly BAX 855 Treatment Viable for Hemophilia

Hemophilia A Drug Granted Breakthrough Therapy Designation

Drug Manages von Willebrandâ€™s Disease with 100 Percent Efficacy

Corrected FVIII Deficiency in Mice with Hemophilia A